clinical research 101

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• Clinical Trials: What Patients Need to Know

Basics About Clinical Trials

What are clinical trials.

Clinical trials are research studies in which people volunteer to help find answers to specific health questions. When carefully conducted, they are the safest and fastest way to find new treatments and ways to improve health.

Clinical trials are conducted according to a plan, called a protocol, which describes:

• the types of patients who may enter the study
• the schedules of tests and procedures
• the drugs involved
• the dosages, or amount of the drug
• the length of the study
• what the researchers hope to learn from the study.

Volunteers who participate in the study must agree to the rules and terms outlined in the protocol. Similarly, researchers, doctors, and other health professionals who manage the clinical trials must follow strict rules set by the FDA. These rules make sure that those who agree to participate are treated as safely as possible.

Learn more about the basics of clinical trial participation, read first hand experiences from actual clinical trial volunteers, and see explanations from researchers at the NIH Clinical Research Trials and You Web site.

Why are clinical trials done?

Clinical trials are conducted for many reasons:

• to determine whether a new drug or device is safe and effective for people to use.
• to study different ways to use standard treatments or current, approved treatments so that they will be more effective, easier to use, or decrease certain side effects.
• to learn how to safely use a treatment in a population for which the treatment was not previously tested, such as children.

Who should consider clinical trials and why?

Some people participate in clinical trials because none of the standard (approved) treatment options have worked, or they are unable to tolerate certain side effects. Clinical trials provide another option when standard therapy has failed. Others participate in trials because they want to contribute to the advancement of medical knowledge.

Ensuring people from diverse backgrounds join clinical trials is key to advancing health equity. Participants in clinical trials should represent the patients that will use the medical products. This is often not the case—people from racial and ethnic minority and other diverse groups are underrepresented in clinical research. This is a concern because people of different ages, races, and ethnicities may react differently to certain medical products. Learn more about the clinical trial diversity initiative from the Office of Minority Health and Health Equity.

All clinical trials have guidelines, called eligibility criteria, about who can participate. The criteria are based on such factors as age, sex, type and stage of disease, previous treatment history, and other medical conditions. This helps to reduce the variation within the study and to ensure that the researchers will be able to answer the questions they plan to study. Therefore, not everyone who applies for a clinical trial will be accepted.

It is important to test drugs and medical products in the people they are meant to help. It is also important to conduct research in a variety of people, because different people may respond differently to treatments.  FDA seeks to ensure that people of different ages, races, ethnic groups, and genders are included in clinical trials. Learn more about FDA’s efforts to increase diversity in clinical trials .

Where are clinical trials conducted?

Clinical trials can be sponsored by organizations (such as a pharmaceutical company), Federal offices and agencies (such as the National Institutes of Health or the U.S. Department of Veterans Affairs), or individuals (such as doctors or health care providers). The sponsor determines the location(s) of the trials, which are usually conducted at universities, medical centers, clinics, hospitals, and other Federally or industry-funded research sites.

Are clinical trials safe?

FDA works to protect participants in clinical trials and to ensure that people have reliable information before deciding whether to join a clinical trial. The Federal government has regulations and guidelines for clinical research to protect participants from unreasonable risks. Although efforts are made to control the risks to participants, some may be unavoidable because we are still learning more about the medical treatments in the study.

The government requires researchers to give prospective participants complete and accurate information about what will happen during the trial. Before joining a particular study, you will be given an informed consent document that describes your rights as a participant, as well as details about the study, including potential risks. Signing it indicates that you understand that the trial is research and that you may leave at any time. The informed consent is part of the process that makes sure you understand the known risks associated with the study.

What should I think about before joining a clinical trial?

Before joining a clinical trial, it is important to learn as much as possible. Discuss your questions and concerns with members of the health care team conducting the trial. Also, discuss the trial with your health care provider to determine whether or not the trial is a good option based on your current treatment. Be sure you understand:

• what happens during the trial
• the type of health care you will receive
• any related costs once you are enrolled in the trial
• the benefits and risks associated with participating. 

What is FDA’s role in approving new drugs and medical treatments?

FDA makes sure medical treatments are safe and effective for people to use. We do not develop new therapies or conduct clinical trials. Rather, we oversee the people who do. FDA staff meet with researchers and perform inspections of clinical trial study sites to protect the rights of patients and to verify the quality and integrity of the data.

Learn more about the Drug Development Process .

Where can I find clinical trials?

One good way to find out if there are any clinical trials that might help you is to ask your doctor. Other sources of information include:

• FDA Clinical Trials Search. Search a database of Federally and privately supported studies available through clinicaltrials.gov. Learn about each trial’s purpose, who can participate, locations, and who to contact for more information.
• Clinicaltrials.gov. Conduct more advanced searches
• National Cancer Institute or call 1–800–4–CANCER (1–800–422–6237). Learn about clinical trials for people with cancer.
• AIDS Clinical Trials and Information Services (ACTIS) or call 1–800–TRIALS–A (1–800–874–2572). Locate clinical trials for people with HIV.
• AIDSinfo. Search a database of HIV/AIDS trials, sponsored by the National Institutes of Health’s National Library of Medicine.

What is a placebo and how is it related to clinical trials?

A placebo is a pill, liquid, or powder that has no treatment value. It is often called a sugar pill. In clinical trials, experimental drugs are often compared with placebos to evaluate the treatment’s effectiveness.

Is there a chance I might get a placebo?

In clinical trials that include placebos, quite often neither patients nor their doctors know who is receiving the placebo and how is being treated with the experimental drug. Many cancer clinical trials, as well as trials for other serious and life-threatening conditions, do not include placebo control groups. In these cases, all participants receive the experimental drug. Ask the trial coordinator whether there is a chance you may get a placebo rather than the experimental drug. Then, talk with your doctor about what is best for you.

How do I find out what Phase a drug is in as part of the clinical trial?

Talk to the clinical trial coordinator to find out which phase the clinical trial is in. Learn more about the different clinical trial phases and whether they are right for you.

What happens to drugs that don't make it out of clinical trials?

Most drugs that undergo preclinical (animal) research never even make it to human testing and review by the FDA. The drug developers go back to begin the development process using what they learned during with their preclinical research. Learn more about drug development .

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Understanding Clinical Trials

Clinical research: what is it.

Your doctor may have said that you are eligible for a clinical trial, or you may have seen an ad for a clinical research study. What is clinical research, and is it right for you?

Clinical research is the comprehensive study of the safety and effectiveness of the most promising advances in patient care. Clinical research is different than laboratory research. It involves people who volunteer to help us better understand medicine and health. Lab research generally does not involve people — although it helps us learn which new ideas may help people.

Every drug, device, tool, diagnostic test, technique and technology used in medicine today was once tested in volunteers who took part in clinical research studies.

At Johns Hopkins Medicine, we believe that clinical research is key to improve care for people in our community and around the world. Once you understand more about clinical research, you may appreciate why it’s important to participate — for yourself and the community.

What Are the Types of Clinical Research?

There are two main kinds of clinical research:

Observational Studies

Observational studies are studies that aim to identify and analyze patterns in medical data or in biological samples, such as tissue or blood provided by study participants.

Clinical Trials

Clinical trials, which are also called interventional studies, test the safety and effectiveness of medical interventions — such as medications, procedures and tools — in living people.

Clinical research studies need people of every age, health status, race, gender, ethnicity and cultural background to participate. This will increase the chances that scientists and clinicians will develop treatments and procedures that are likely to be safe and work well in all people. Potential volunteers are carefully screened to ensure that they meet all of the requirements for any study before they begin. Most of the reasons people are not included in studies is because of concerns about safety.

Both healthy people and those with diagnosed medical conditions can take part in clinical research. Participation is always completely voluntary, and participants can leave a study at any time for any reason.

“The only way medical advancements can be made is if people volunteer to participate in clinical research. The research participant is just as necessary as the researcher in this partnership to advance health care.” Liz Martinez, Johns Hopkins Medicine Research Participant Advocate

Types of Research Studies

Within the two main kinds of clinical research, there are many types of studies. They vary based on the study goals, participants and other factors.

Biospecimen studies

Healthy volunteer studies.

Clinical trials study the safety and effectiveness of interventions and procedures on people’s health. Interventions may include medications, radiation, foods or behaviors, such as exercise. Usually, the treatments in clinical trials are studied in a laboratory and sometimes in animals before they are studied in humans. The goal of clinical trials is to find new and better ways of preventing, diagnosing and treating disease. They are used to test:

Drugs or medicines

New types of surgery

Medical devices

New ways of using current treatments

New ways of changing health behaviors

New ways to improve quality of life for sick patients

 Goals of Clinical Trials

Because every clinical trial is designed to answer one or more medical questions, different trials have different goals. Those goals include:

Treatment trials

Prevention trials, screening trials, phases of a clinical trial.

In general, a new drug needs to go through a series of four types of clinical trials. This helps researchers show that the medication is safe and effective. As a study moves through each phase, researchers learn more about a medication, including its risks and benefits.

Is the medication safe and what is the right dose?   Phase one trials involve small numbers of participants, often normal volunteers.

Does the new medication work and what are the side effects?   Phase two trials test the treatment or procedure on a larger number of participants. These participants usually have the condition or disease that the treatment is intended to remedy.

Is the new medication more effective than existing treatments?  Phase three trials have even more people enrolled. Some may get a placebo (a substance that has no medical effect) or an already approved treatment, so that the new medication can be compared to that treatment.

Is the new medication effective and safe over the long term?   Phase four happens after the treatment or procedure has been approved. Information about patients who are receiving the treatment is gathered and studied to see if any new information is seen when given to a large number of patients.

“Johns Hopkins has a comprehensive system overseeing research that is audited by the FDA and the Association for Accreditation of Human Research Protection Programs to make certain all research participants voluntarily agreed to join a study and their safety was maximized.” Gail Daumit, M.D., M.H.S., Vice Dean for Clinical Investigation, Johns Hopkins University School of Medicine

Is It Safe to Participate in Clinical Research?

There are several steps in place to protect volunteers who take part in clinical research studies. Clinical Research is regulated by the federal government. In addition, the institutional review board (IRB) and Human Subjects Research Protection Program at each study location have many safeguards built in to each study to protect the safety and privacy of participants.

Clinical researchers are required by law to follow the safety rules outlined by each study's protocol. A protocol is a detailed plan of what researchers will do in during the study.

In the U.S., every study site's IRB — which is made up of both medical experts and members of the general public — must approve all clinical research. IRB members also review plans for all clinical studies. And, they make sure that research participants are protected from as much risk as possible.

Earning Your Trust

This was not always the case. Many people of color are wary of joining clinical research because of previous poor treatment of underrepresented minorities throughout the U.S. This includes medical research performed on enslaved people without their consent, or not giving treatment to Black men who participated in the Tuskegee Study of Untreated Syphilis in the Negro Male. Since the 1970s, numerous regulations have been in place to protect the rights of study participants.

Many clinical research studies are also supervised by a data and safety monitoring committee. This is a group made up of experts in the area being studied. These biomedical professionals regularly monitor clinical studies as they progress. If they discover or suspect any problems with a study, they immediately stop the trial. In addition, Johns Hopkins Medicine’s Research Participant Advocacy Group focuses on improving the experience of people who participate in clinical research.

Clinical research participants with concerns about anything related to the study they are taking part in should contact Johns Hopkins Medicine’s IRB or our Research Participant Advocacy Group .

Learn More About Clinical Research at Johns Hopkins Medicine

For information about clinical trial opportunities at Johns Hopkins Medicine, visit our trials site.

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Nih clinical research trials and you.

The NIH Clinical Trials and You website is a resource for people who want to learn more about clinical trials. By expanding the below questions, you can read answers to common questions about taking part in a clinical trial. 

What are clinical trials and why do people participate?

Clinical research is medical research that involves people like you. When you volunteer to take part in clinical research, you help doctors and researchers learn more about disease and improve health care for people in the future. Clinical research includes all research that involves people.  Types of clinical research include:

• Epidemiology, which improves the understanding of a disease by studying patterns, causes, and effects of health and disease in specific groups.
• Behavioral, which improves the understanding of human behavior and how it relates to health and disease.
• Health services, which looks at how people access health care providers and health care services, how much care costs, and what happens to patients as a result of this care.
• Clinical trials, which evaluate the effects of an intervention on health outcomes.

What are clinical trials and why would I want to take part?

Clinical trials are part of clinical research and at the heart of all medical advances. Clinical trials look at new ways to prevent, detect, or treat disease. Clinical trials can study:

• New drugs or new combinations of drugs
• New ways of doing surgery
• New medical devices
• New ways to use existing treatments
• New ways to change behaviors to improve health
• New ways to improve the quality of life for people with acute or chronic illnesses.

The goal of clinical trials is to determine if these treatment, prevention, and behavior approaches are safe and effective. People take part in clinical trials for many reasons. Healthy volunteers say they take part to help others and to contribute to moving science forward. People with an illness or disease also take part to help others, but also to possibly receive the newest treatment and to have added (or extra) care and attention from the clinical trial staff. Clinical trials offer hope for many people and a chance to help researchers find better treatments for others in the future

Why is diversity and inclusion important in clinical trials?

People may experience the same disease differently. It’s essential that clinical trials include people with a variety of lived experiences and living conditions, as well as characteristics like race and ethnicity, age, sex, and sexual orientation, so that all communities benefit from scientific advances.

See Diversity & Inclusion in Clinical Trials for more information.

How does the research process work?

The idea for a clinical trial often starts in the lab. After researchers test new treatments or procedures in the lab and in animals, the most promising treatments are moved into clinical trials. As new treatments move through a series of steps called phases, more information is gained about the treatment, its risks, and its effectiveness.

What are clinical trial protocols?

Clinical trials follow a plan known as a protocol. The protocol is carefully designed to balance the potential benefits and risks to participants, and answer specific research questions. A protocol describes the following:

• The goal of the study
• Who is eligible to take part in the trial
• Protections against risks to participants
• Details about tests, procedures, and treatments
• How long the trial is expected to last
• What information will be gathered

A clinical trial is led by a principal investigator (PI). Members of the research team regularly monitor the participants’ health to determine the study’s safety and effectiveness.

What is an Institutional Review Board?

Most, but not all, clinical trials in the United States are approved and monitored by an Institutional Review Board (IRB) to ensure that the risks are reduced and are outweighed by potential benefits. IRBs are committees that are responsible for reviewing research in order to protect the rights and safety of people who take part in research, both before the research starts and as it proceeds. You should ask the sponsor or research coordinator whether the research you are thinking about joining was reviewed by an IRB.

What is a clinical trial sponsor?

Clinical trial sponsors may be people, institutions, companies, government agencies, or other organizations that are responsible for initiating, managing or financing the clinical trial, but do not conduct the research.

What is informed consent?

Informed consent is the process of providing you with key information about a research study before you decide whether to accept the offer to take part. The process of informed consent continues throughout the study. To help you decide whether to take part, members of the research team explain the details of the study. If you do not understand English, a translator or interpreter may be provided. The research team provides an informed consent document that includes details about the study, such as its purpose, how long it’s expected to last, tests or procedures that will be done as part of the research, and who to contact for further information. The informed consent document also explains risks and potential benefits. You can then decide whether to sign the document. Taking part in a clinical trial is voluntary and you can leave the study at any time.

What are the types of clinical trials?

There are different types of clinical trials.

• Prevention trials look for better ways to prevent a disease in people who have never had the disease or to prevent the disease from returning. Approaches may include medicines, vaccines, or lifestyle changes.
• Screening trials test new ways for detecting diseases or health conditions.
• Diagnostic trials study or compare tests or procedures for diagnosing a particular disease or condition.
• Treatment trials test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
• Behavioral trials evaluate or compare ways to promote behavioral changes designed to improve health.
• Quality of life trials (or supportive care trials) explore and measure ways to improve the comfort and quality of life of people with conditions or illnesses.

What are the phases of clinical trials?

Clinical trials are conducted in a series of steps called “phases.” Each phase has a different purpose and helps researchers answer different questions.

• Phase I trials : Researchers test a drug or treatment in a small group of people (20–80) for the first time. The purpose is to study the drug or treatment to learn about safety and identify side effects.
• Phase II trials : The new drug or treatment is given to a larger group of people (100–300) to determine its effectiveness and to further study its safety.
• Phase III trials : The new drug or treatment is given to large groups of people (1,000–3,000) to confirm its effectiveness, monitor side effects, compare it with standard or similar treatments, and collect information that will allow the new drug or treatment to be used safely.
• Phase IV trials : After a drug is approved by the FDA and made available to the public, researchers track its safety in the general population, seeking more information about a drug or treatment’s benefits, and optimal use.

What do the terms placebo, randomization, and blinded mean in clinical trials?

In clinical trials that compare a new product or therapy with another that already exists, researchers try to determine if the new one is as good, or better than, the existing one. In some studies, you may be assigned to receive a placebo (an inactive product that resembles the test product, but without its treatment value).

Comparing a new product with a placebo can be the fastest and most reliable way to show the new product’s effectiveness. However, placebos are not used if you would be put at risk — particularly in the study of treatments for serious illnesses — by not having effective therapy. You will be told if placebos are used in the study before entering a trial.

Randomization is the process by which treatments are assigned to participants by chance rather than by choice. This is done to avoid any bias in assigning volunteers to get one treatment or another. The effects of each treatment are compared at specific points during a trial. If one treatment is found superior, the trial is stopped so that the most volunteers receive the more beneficial treatment.  This video helps explain randomization for all clinical trials .

" Blinded " (or " masked ") studies are designed to prevent members of the research team and study participants from influencing the results. Blinding allows the collection of scientifically accurate data. In single-blind (" single-masked ") studies, you are not told what is being given, but the research team knows. In a double-blind study, neither you nor the research team are told what you are given; only the pharmacist knows. Members of the research team are not told which participants are receiving which treatment, in order to reduce bias. If medically necessary, however, it is always possible to find out which treatment you are receiving.

Who takes part in clinical trials?

Many different types of people take part in clinical trials. Some are healthy, while others may have illnesses. Research procedures with healthy volunteers are designed to develop new knowledge, not to provide direct benefit to those taking part. Healthy volunteers have always played an important role in research.

Healthy volunteers are needed for several reasons. When developing a new technique, such as a blood test or imaging device, healthy volunteers help define the limits of "normal." These volunteers are the baseline against which patient groups are compared and are often matched to patients on factors such as age, gender, or family relationship. They receive the same tests, procedures, or drugs the patient group receives. Researchers learn about the disease process by comparing the patient group to the healthy volunteers.

Factors like how much of your time is needed, discomfort you may feel, or risk involved depends on the trial. While some require minimal amounts of time and effort, other studies may require a major commitment of your time and effort, and may involve some discomfort. The research procedure(s) may also carry some risk. The informed consent process for healthy volunteers includes a detailed discussion of the study's procedures and tests and their risks.

A patient volunteer has a known health problem and takes part in research to better understand, diagnose, or treat that disease or condition. Research with a patient volunteer helps develop new knowledge. Depending on the stage of knowledge about the disease or condition, these procedures may or may not benefit the study participants.

Patients may volunteer for studies similar to those in which healthy volunteers take part. These studies involve drugs, devices, or treatments designed to prevent,or treat disease. Although these studies may provide direct benefit to patient volunteers, the main aim is to prove, by scientific means, the effects and limitations of the experimental treatment. Therefore, some patient groups may serve as a baseline for comparison by not taking the test drug, or by receiving test doses of the drug large enough only to show that it is present, but not at a level that can treat the condition.

Researchers follow clinical trials guidelines when deciding who can participate, in a study. These guidelines are called Inclusion/Exclusion Criteria . Factors that allow you to take part in a clinical trial are called "inclusion criteria." Those that exclude or prevent participation are "exclusion criteria." These criteria are based on factors such as age, gender, the type and stage of a disease, treatment history, and other medical conditions. Before joining a clinical trial, you must provide information that allows the research team to determine whether or not you can take part in the study safely. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy volunteers. Inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe, and to help ensure that researchers can find new information they need.

What do I need to know if I am thinking about taking part in a clinical trial?

Risks and potential benefits

Clinical trials may involve risk, as can routine medical care and the activities of daily living. When weighing the risks of research, you can think about these important factors:

• The possible harms that could result from taking part in the study
• The level of harm
• The chance of any harm occurring

Most clinical trials pose the risk of minor discomfort, which lasts only a short time. However, some study participants experience complications that require medical attention. In rare cases, participants have been seriously injured or have died of complications resulting from their participation in trials of experimental treatments. The specific risks associated with a research protocol are described in detail in the informed consent document, which participants are asked to consider and sign before participating in research. Also, a member of the research team will explain the study and answer any questions about the study. Before deciding to participate, carefully consider risks and possible benefits.

Potential benefits

Well-designed and well-executed clinical trials provide the best approach for you to:

• Help others by contributing to knowledge about new treatments or procedures.
• Gain access to new research treatments before they are widely available.
• Receive regular and careful medical attention from a research team that includes doctors and other health professionals.

Risks to taking part in clinical trials include the following:

• There may be unpleasant, serious, or even life-threatening effects of experimental treatment.
• The study may require more time and attention than standard treatment would, including visits to the study site, more blood tests, more procedures, hospital stays, or complex dosage schedules.

What questions should I ask if offered a clinical trial?

If you are thinking about taking part in a clinical trial, you should feel free to ask any questions or bring up any issues concerning the trial at any time. The following suggestions may give you some ideas as you think about your own questions.

• What is the purpose of the study?
• Why do researchers think the approach may be effective?
• Who will fund the study?
• Who has reviewed and approved the study?
• How are study results and safety of participants being monitored?
• How long will the study last?
• What will my responsibilities be if I take part?
• Who will tell me about the results of the study and how will I be informed?

Risks and possible benefits

• What are my possible short-term benefits?
• What are my possible long-term benefits?
• What are my short-term risks, and side effects?
• What are my long-term risks?
• What other options are available?
• How do the risks and possible benefits of this trial compare with those options?

Participation and care

• What kinds of therapies, procedures and/or tests will I have during the trial?
• Will they hurt, and if so, for how long?
• How do the tests in the study compare with those I would have outside of the trial?
• Will I be able to take my regular medications while taking part in the clinical trial?
• Where will I have my medical care?
• Who will be in charge of my care?

Personal issues

• How could being in this study affect my daily life?
• Can I talk to other people in the study?

Cost issues

• Will I have to pay for any part of the trial such as tests or the study drug?
• If so, what will the charges likely be?
• What is my health insurance likely to cover?
• Who can help answer any questions from my insurance company or health plan?
• Will there be any travel or child care costs that I need to consider while I am in the trial?

Tips for asking your doctor about trials

• Consider taking a family member or friend along for support and for help in asking questions or recording answers.
• Plan what to ask — but don't hesitate to ask any new questions.
• Write down questions in advance to remember them all.
• Write down the answers so that they’re available when needed.
• Ask about bringing a tape recorder to make a taped record of what's said (even if you write down answers).

This information courtesy of Cancer.gov.

How is my safety protected?

Ethical guidelines

The goal of clinical research is to develop knowledge that improves human health or increases understanding of human biology. People who take part in clinical research make it possible for this to occur. The path to finding out if a new drug is safe or effective is to test it on patients in clinical trials. The purpose of ethical guidelines is both to protect patients and healthy volunteers, and to preserve the integrity of the science.

Informed consent

Informed consent is the process of learning the key facts about a clinical trial before deciding whether to participate. The process of providing information to participants continues throughout the study. To help you decide whether to take part, members of the research team explain the study. The research team provides an informed consent document, which includes such details about the study as its purpose, duration, required procedures, and who to contact for various purposes. The informed consent document also explains risks and potential benefits.

If you decide to enroll in the trial, you will need to sign the informed consent document. You are free to withdraw from the study at any time.

Most, but not all, clinical trials in the United States are approved and monitored by an Institutional Review Board (IRB) to ensure that the risks are minimal when compared with potential benefits. An IRB is an independent committee that consists of physicians, statisticians, and members of the community who ensure that clinical trials are ethical and that the rights of participants are protected. You should ask the sponsor or research coordinator whether the research you are considering participating in was reviewed by an IRB.

Further reading

For more information about research protections, see:

• Office of Human Research Protection
• Children's Assent to Clinical Trial Participation

For more information on participants’ privacy and confidentiality, see:

• HIPAA Privacy Rule
• The Food and Drug Administration, FDA’s Drug Review Process: Ensuring Drugs Are Safe and Effective

For more information about research protections, see: About Research Participation

What happens after a clinical trial is completed?

After a clinical trial is completed, the researchers carefully examine information collected during the study before making decisions about the meaning of the findings and about the need for further testing. After a phase I or II trial, the researchers decide whether to move on to the next phase or to stop testing the treatment or procedure because it was unsafe or not effective. When a phase III trial is completed, the researchers examine the information and decide whether the results have medical importance.

Results from clinical trials are often published in peer-reviewed scientific journals. Peer review is a process by which experts review the report before it is published to ensure that the analysis and conclusions are sound. If the results are particularly important, they may be featured in the news, and discussed at scientific meetings and by patient advocacy groups before or after they are published in a scientific journal. Once a new approach has been proven safe and effective in a clinical trial, it may become a new standard of medical practice.

Ask the research team members if the study results have been or will be published. Published study results are also available by searching for the study's official name or Protocol ID number in the National Library of Medicine's PubMed® database .

How does clinical research make a difference to me and my family?

Only through clinical research can we gain insights and answers about the safety and effectiveness of treatments and procedures. Groundbreaking scientific advances in the present and the past were possible only because of participation of volunteers, both healthy and those with an illness, in clinical research. Clinical research requires complex and rigorous testing in collaboration with communities that are affected by the disease. As research opens new doors to finding ways to diagnose, prevent, treat, or cure disease and disability, clinical trial participation is essential to help us find the answers.

This page last reviewed on October 3, 2022

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Introduction to Clinical Trials

This training program provides the foundational knowledge upon which one can develop their competence as a clinical research professional..

This 2-hour online course details how medical products are developed, how volunteer patients are protected, and who plays key roles in the development, research, review, and approval of medical products.

In addition to supporting clinical research professionals and organizations, this program is an ideal tool for organizations seeking to help educate the public about clinical research and to raise awareness of clinical trials and the clinical research profession.

Introduction to Clinical Trials is an ideal program for all novice clinical researchers, those interested in the profession, or those indirectly involved in clinical trials.

Upon completion of this training program, participants should be able to:

• Analyze the study and site activities and requirements.
• Explain the core ethical principles of clinical research.
• Discuss how the research subject’s safety, rights and welfare are protected in clinical trials.
• Describe the development process for medical products and the related regulations.
• Determine good clinical practices for ensuring the safety of the research subject and the validity of a clinical trial.

This course helps clinical researchers of all types build competency in Clinical Trial Operations (GCPs), Ethical and Participant Safety Considerations, and Scientific Concepts and Research Design. Explore Competencies >

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Clinical Trials 101

Clinical trial awareness starts with education..

Get answers to your questions and orient yourself to some of the most common clinical trial terminology.

Clinical Trial FAQs

Check out this great answer from the National Institute on Aging !

FTLOScience.com has an excellent rundown of the steps necessary for developing and approving new drugs. We especially love the drug development funnel illustration about halfway down the page!

There are many different ways to find a clinical trial. Carebox and Antidote are great sources to get you started, and you can also speak with your doctor if they currently run clinical trials. Here’s a great breakdown of the process from Pfizer and another from the National Institutes of Heath . 

There are several different factors to consider here. One is that your usual personal healthcare costs remain the same, but some lab work, X-rays, research drugs, and other medical care related specifically to the study you participate in will be covered by the sponsor of that study. Cancer.gov has a handy explanation here . 

Another factor you may be wondering about is whether you’ll have to miss work and therefore miss paychecks due to participating in a clinical trial. This is certainly something to consider. Ask about the trial’s timing and the in-person commitment needed from you during sign-up. Additionally, some trials do offer compensation to participants , but not all share this practice. 

When it comes to travel to and from research sites, some trials cover these costs for patients and others don’t. Be sure to ask about this when signing up for a specific trial. 

Finally, you may be able to find a financial navigator to help you sort out costs related to your illness, including any costs associated with participating in research. Here’s an example of how a Financial Navigation Program works with cancer patients and families. 

Your primary care physician is your ally in tapping into clinical research as a care option. Some questions to ask include: 

• Can you help me find a clinical trial that is a good fit for my health condition? 
• Are you serving as a principal investigator in any current or upcoming clinical trials you’d like me to be aware of? 
• What support should I count on from you and your practice during and after I become a medical studies participant? 
• Here are some more great questions to ask from the Memorial Sloan Kettering Cancer Center… 
• …and even more in this excellent clinical trials overview article from Patient Advocate Foundation ! 

Yes! There are many decentralized clinical trials (those that wholly or in part rely on data that can be collected remotely) and hybrid clinical trials (those that rely on some remote data and some onsite data collection). When considering a study, you can talk to your doctor or the study enrollment team to better understand whether travel is required, and if so, how much. 

The Carebox link in question #3 is a great place to start in your case, as well! You can also check out the Pediatric Trials Network and Pfizer’s Pediatric Clinical Trials hub . 

As shared by the National Heart, Lung, and Blood Institute, healthy volunteers, patient volunteers, diverse volunteers, and children should all consider clinical trial participation. The NHLBI page elaborates here . 

Altruistic support of global wellness, research as a care option, and financial benefits are some of the reasons that people choose to participate in clinical trials. See what Johns Hopkins Medicine has to say about this . 

Common Clinical Trial Terminology

Controlled study/controlled trial.

A controlled study is a way to test if something, like a new medicine, works. There are two groups in a controlled study: the treatment group and the control group. The treatment group gets the new medicine being tested. The control group gets something else, like a fake drug (the “placebo”) or a different treatment. The researchers watch the two groups to see if the treatment group does better than the control group. If it does, then the new medicine being tested probably works. 

DCT stands for “decentralized trial”. It’s a type of clinical trial where some or all of the study happens outside of a hospital or clinic. This means that participants can do their study visits from home or another location, like their doctor’s office. For example, a company is testing a new drug for treating asthma. They want to make it easy for people with asthma to participate in the trial, so they offer the option to do study visits from home. Participants in the trial can use a special app to track their symptoms and report them to the study team. They can also do video calls with the study team if they have any questions or concerns. 

Double-Blind Study

A double-blind study is a type of scientific study where neither the people taking part (the subjects) nor the people running the study (the investigators) know who is getting the real treatment and who is getting a fake treatment (a placebo). This helps make sure the results are not influenced by people’s expectations. If people know they are getting the real treatment, they might be more likely to get better, even if the treatment doesn’t work. And if people know that they are getting a placebo, they might be less likely to get better, even if the placebo does work. Double-blind studies are some of the most reliable ways to test if a new treatment works.  

FDA (Food and Drug Administration)

The FDA stands for Food and Drug Administration. It’s a government agency in the United States that helps to make sure that food, drugs, and medical devices are safe and that they work. The FDA has the final say on whether a new drug can be sold in the United States. They do this by reviewing the drug’s safety and effectiveness (if it works). Other countries have their own food and drug agencies, like the European Medicines Agency (EMA) in Europe.  

Hybrid Trial

A hybrid trial is a type of clinical trial in which some of the study activities are done remotely, like at a patient’s home or doctor’s office, while others are done at a traditional clinical research site.  

Investigational Drug

A drug that is still being tested in people. It has not been approved by the FDA yet, so it is not available to the public. Investigational drugs are being studied to see if they are safe and effective for treating a certain disease or medical condition. They are tested in clinical trials, which are studies that look at how well a drug works and what side effects it might have. 

Medical Device Trial

A medical device trial is a clinical study that tests if a new medical device is safe and if it works. Medical devices can be anything from a bandage to an implant, like a pacemaker.  

Phase I-III Clinical Trials

Phase I-III clinical trials are studies that test new drugs or treatments in people. They are done in stages, called phases.  

Phase I trials are the first time a new drug or treatment is given to people. The goal of Phase I trials is to find the safe dose of the drug or treatment.  

Phase II trials are done to see how well the drug or treatment works. They are usually done with a small group of people who have the disease or health condition that the drug or treatment is meant to treat.  

Phase III trials are done to compare the drug or treatment to other treatments that are already available. They are usually done with a large group of people who have the disease or health condition that the drug or treatment is meant to treat  

Some trials also have a Phase 0, which is a very early stage of testing. In Phase 0, researchers give a very small amount of the drug or treatment to people to see how it works in the body.  

After a drug or treatment is approved, Phase IV trials may be done to continue to monitor its long-term effects. 

Principal Investigator

Principal Investigator, or PI, is the person in charge of a clinical study. They are responsible for designing the study, finding people to be in the study, and collecting data during the study. They also work with the sponsor of the study to make sure it follows the FDA’s rules. PIs are usually doctors or scientists with experience in clinical research.  

The protocol is a clinical trial plan. It tells what the study will do, how, and why. It includes information about the number of patients who will be in the trial, who is qualified to be in the trial, what they will be given, what tests they will receive, and when, and what information will be collected. 

Randomization

Randomization is a way to make sure that the groups of people in a clinical study are as similar as possible. This is done by randomly assigning participants to different groups. For example, if a study is comparing two different treatments, the participants will be randomly assigned to either the treatment group or the control group. This means that there is an equal chance that any participant will be assigned to either group. Randomization is important because it helps to make sure that the results of the study are accurate. If the groups are not similar, then it may be difficult to tell if the difference in results is due to the treatment or to other factors. 

Reimbursement

Reimbursement is when you get money back for something you bought. In a clinical trial, reimbursement is money that you get back for things you bought because of the trial, like bus fare, parking fees, or lost wages. 

A site is where a trial happens. This could be a doctor’s office, a hospital, a dedicated clinical research facility, or another medical facility. 

The sponsor is the person, company, or organization that pays for a clinical trial. The sponsor also has to make sure that the trial is run properly and that the participant’s rights are protected. 

Itching to Learn More About Participating in Clinical Trials?  

Click the button to visit our library of information!   

Clinical Trials 101: The Ultimate Guide to Understanding Clinical Research

You have probably heard about clinical trials, but you may have many questions about what they are, how they work, and how to get started as a participant. Are they just for people with cancer? Are they safe? Am I eligible? How would I find one? There is a lot to ask about—and you are not alone. We have created  Clinical Trials 101  to answer your questions about clinical trials so you can have a clear understanding of trial participation is the right fit for you.

What are clinical trials?

Clinical trials are research studies that evaluate new—and existing—medicines, vaccines, and devices for effectiveness and safety. In many clinical trials, the new treatment is compared to the current standard treatment to learn if the new treatment is more effective and/or has fewer side effects.

What are clinical trial phases?

• Phase I  trials involve a small number of people (often less than 100) to evaluate the dosage amount, side effects, and safety.
• Phase II  trials enroll more participants (usually 100 to 300) to evaluate effectiveness. The researchers want to see if the new treatment or device works for the condition or disease, and phase II may last for two or three years.
• Phase III  includes more patients, from several hundred to 3,000, to try different doses in a larger group. The effectiveness of the new treatment is the number one priority, followed by its safety and side effects. Phase III often compares the new treatment to the standard treatment. If results are encouraging, researchers will ask for FDA approval.
• Phase IV  is conducted after approval to study the benefits and safety.

Why are clinical trials important?

Clinical trials are the reason why new—and sometimes life-saving—medicines, vaccines, and devices are available to the world. It is hard to completely underscore the importance enough. From the Advil you have on your bathroom shelves to the chemo that could save someone’s life, all those drugs once started out in clinical research with participants like you or someone you know. Even now after COVID-19, we are seeing the global effects of clinical research: over five million people participated in trials that went to help the world at large.

Clinical trials are also offered as an additional care option to many who face an illness or disease. That is why we believe in connecting as many patients and physicians to available clinical research opportunities as possible—everyone deserves to know what their options are and what is out there. 

Why do people join clinical trials?

Everyone has different reasons for joining a trial. Some of the most common reasons people decide to join are:

• Access to a new treatment option
• Personalized and attentive monitoring throughout the trial, often improving the patient-provider relationship
• Advance research for other people and bring new medicines, vaccines, and devices to the world
• Advance our global understanding of a condition
• Gain access to support groups and helpful resources
• Access to a low- or no-cost care option. In most cases, trial participants do not have to pay out-of-pocket when participating in a trial. Research-related costs are typically covered by the clinical trial sponsor. 

How to get started with clinical research

A familiar quote comes to mind as anyone starts with clinical research and joins a clinical trial: "Sometimes the hardest thing to do is to take the first step." You will probably have more questions than answers, but eventually, you will see a clearer path and the direction you want to go.

If you're considering a clinical trial, talking to your doctor is a good first step. A trial might be conducted at their facility that would be appropriate for you, and they may be able to direct you where to start. If your physician isn't aware of any clinical trials, it may be up to you to be proactive and take charge of finding a suitable clinical trial. That’s what  TrialJourney  is for. Here, you can find any available clinical trial and filter by criteria such as your location, disease or ailment, or top-rated research sites. You can also see what other participants have said about their experiences which can help you make a truly informed decision about where to go. 

How do you know if a clinical trial is right for you?

Before enrolling in a particular trial, you will usually meet with someone from the clinical trial research team who will explain all the aspects of the trial. They may also ask some basic screening questions to ensure you meet the criteria to participate in the study. 

This is also the perfect time to ask questions to help you make your decision. Write down questions before meeting with the research team and take notes about the answers to your questions. Then you, and anyone who may help inform your decision, can help you determine if a clinical trial is a good option. It is worth noting, participants in clinical trials can leave the study at any time and for any reason.

What should you expect when participating in a clinical trial?

If you choose to enroll in a clinical trial and you are eligible,you may hear about a clinical trial protocol. A clinical trial protocol is a document that details how the trial will be conducted. The protocol includes the purpose/objective, the details, the methods to be used, and how the statistics/outcomes will be calculated.

• Purpose of a protocol: A trial protocol helps assure patient safety. The document helps ensure the integrity of the data collected and the ethical interpretation of the study.
• Inclusion/Exclusion criteria: every clinical trial has criteria for eligibility of a particular trial. This criteria is very specific and may include age, gender, ethnicity, smoking status, BMI, and other chronic medical conditions. The criteria are important to increase the chances that the study will produce accurate, reproducible, and reliable results.

Who is involved in the clinical trial process?

Throughout your time in a clinical trial, there will be key people involved, some of whom are always in the background, while others are patient-facing.

• Research Coordinator.  The researcher coordinator utilizes the trial protocol as their guidance — they recruit and screen patients and ensure the safety and security of trial medications. They oversee the collection of all patients' data, tests, side effects, and outcomes. The coordinator pays particular attention to any adverse events that patients experience.
• Research Assistant.  The research assistant helps the coordinator fulfill the protocol requirements — from recruiting patients to patient interviews and preparing mid-trial reports to help prepare final reports.
• Principal Investigator.  A principal investigator, or PI, is a physician who coordinates the medical side of the trial — ensuring the patient's access to potentially more effective treatment while watching for adverse events. The investigator interfaces with the drug company as they evaluate the efficacy and safety of a new treatment.
• Sponsors.  Sponsors help ensure the necessary funding for the clinical trial and direct the ongoing process of the trial. The sponsor communicates with the FDA, other regulatory agencies, and ethics boards over any concerns about the treatment's safety or benefits. Sponsors include government agencies, biotech, medical device, pharmaceutical companies, healthcare entities, or private individuals or entities.
• CROs.  CRO stands for Contract Research Organization, which provides clinical trial management services for biotech, medical device, and pharmaceutical companies. A CRO has extensive experience in the day-to-day operation of clinical trials to maintain a well-run trial. You may hear about a CRO but never interact with them directly.

Participant protection and safety measures

Patient safety is the most important thing throughout every clinical trial. Each trial goes through an exacting review, and the staff is supervised—including the doctors, research team, and procedures. Potential participants receive one-on-one counseling about the trial and the potential risks and benefits. You will have a chance to ask questions to ensure that you understand the risks and the extensive measures taken by the trial team and oversight organizations to ensure your safety. As mentioned earlier, participants are always allowed to stop participating in the trial at any time for any reason.

Informed consent

Patients receive a printed document containing information with sufficient details of the trial for patients to decide. Each patient meets with the doctor, research staff, or both to answer any questions you may have. The information provided includes:

• Your treatment options, including deciding not to join
• The clinical trial treatment and how it differs from the standardized treatment
• The potential benefits and predictable risks, including how the risks may be different from the risks of standard treatment
• How many doctor visits, the frequency of visits, and medical tests
• The costs that you may have to pay
• How your privacy and personal information is protected
• If compensation is available for travel and/or overnight expenses
• How to contact the staff

Participants are given time to take the consent form home to discuss with their family members or trusted friends. Should you decide to join a trial, you will receive a signed copy to keep as part of your permanent records. In some trials, this process is done electronically and may include videos, quizzes, or electronic signatures (also known as eConsent).

IRB (Institutional Review Board)

The Institutional Review Board comprises several physicians, a scientist, a non-scientist, and a representative from the community where the trial is located. An IRB is required for every research project that involves humans as study participants.

They serve as an ethics review to ensure that the rights of research participants are protected—and protect the rights of future patients who might benefit from this trial research. The IRB ensures that informed consent forms are provided to all clinical trial participants and that they have been properly signed.

Office of Human Research Protections (OHRP)

OHRP is an organization that furnishes leadership, clarification, and guidance for clinical trials and other biomedical and behavioral research. In other words, OHRP safeguards the rights, well-being, and safety of participants. The Federal Drug Administration (FDA) oversees trials to ensure that each one is designed, managed, evaluated, and presented according to good clinical practice (GCP) regulations and federal law.

The oversight from OHRP ensures that rigorous medical research is conducted while ensuring the safety of patients.

Food and Drug Administration (FDA)

The Food and Drug Administration is charged with overseeing both the effectiveness and safety of treatments that either cure, control symptoms, or delay the progression of diseases in humans. Treatments include drugs, medical devices (like insulin pumps), or biologics (vaccines, blood products, or gene therapy). Not every treatment is completely effective for every patient, so the search continues for new and more effective treatments with fewer side effects.

While a new treatment may appear "promising" in the laboratory, it is impossible to know how effective or safe it will be until it is used within humans. The goal of the FDA is to protect human participants from excessive risks while allowing clinical trial patients the opportunity to benefit from a new treatment and help advance the cause of science.

Where can I find resources for clinical trials? 

There are many clinical trial resources, but you will have to learn which ones are "trusted" resources; with so many options, it may seem overwhelming. You may not find a trial at first, but do not be discouraged—new trials begin regularly, so continue looking until you find a trial that is right for you.

Clinicaltrials.gov  is one of the most known resources for clinical trial participation, though it may not be the most helpful in your search.

ClinicalTrials.gov is the largest repository of clinical trials globally, and over 325,000 trials from 209 countries are listed. 

While the concept that "bigger is better" sounds good, this concept may not be necessarily true for ClinicalTrials.gov. Its large size limits its usefulness to patients searching for a clinical trial that is actively enrolling, nearby, and might be right for them. And there are three other areas where it is lacking:

• Non-registration of some trials
• Registration of trials with incomplete information
• Non-reporting of trial results

Get started with TrialJourney

TrialJourney  was founded by physicians, nurses, researchers, and computer scientists who realized the difficulties and frustrations patients faced when searching for a clinical trial.

While information is abundant online, it is not always well-organized or easy to use. The terminology used is difficult for many patients to understand—TrialJourney provides a user-friendly search method.

How to use TrialJourney

The most important feature of TrialJourney is the  Find a Clinical Trial  tool. It is easy to use, with the ability to search nearby cities; you can also extend your search area to a wider distance, the 100-mile or 300-mile radius, to include suburbs or centers in adjoining states. You can narrow your search by closest location or highest ratings, by gender, or by age. Be sure to check back regularly because clinical trial research facilities add new trials throughout the year.

Why are site reviews and ratings important?

Patient reviews are extremely valuable to patients considering a clinical trial. Knowing that someone like you has a good/positive experience is encouraging and uplifting. Seeing a negative review alerts a potential patient to ask lots of questions to help ensure that they are making a wise decision.

What information is available?

Every research center in the United States that has registered a clinical trial on ClinicalTrials.gov is included in our database. TrialJourney offers its website visitors a variety of resources:

• What You Should Know Before Participating in a Clinical Trial
• Definitions
• Frequently Asked Questions
• The clinical trials landscape series

So, what is the next step? 

If you think you are ready to participate, we thank you! If you qualify, you will be helping to bring new treatment options and learnings to the world. You can think about the kind of trial that you may be looking for, and then look through trial opportunities near you. It is also recommended that you consult your provider for their recommendations. 

If you still need more time or understanding before you feel ready to participate, that is okay too! Clinical research is not for everyone, and there is so much to learn. Read through our  Nest hub to keep learning  about how clinical research works, and it is always worth looking through available opportunities in your area to see if anything could be the right fit in the future.  

Foundations of Clinical Research

This Harvard Medical School six-month, application-based certificate program provides the essential skill sets and fundamental knowledge required to begin or expand your clinical research career.

Associated Schools

Harvard Medical School

What you'll learn.

Understand and apply the foundational concepts of biostatistics and epidemiology

Develop a research question and formulate a testable hypothesis

Design and begin to implement a clinical research study

Cultivate the skills required to present a clinical research study

Critically evaluate the research findings in medical literature

Synthesize crucial statistical analyses using Stata software

Course description

The Foundations of Clinical Research program is rooted in the belief that clinical research training is critical to professional development in health care. Clinical research training not only creates potential independent investigators, but also enables clinicians to advance their careers through a greater understanding of research evidence. Designed to provide learners with the foundational knowledge and skill sets required to produce high-quality clinical research, our program will lay the fundamental groundwork in epidemiology and biostatistics required for a multifaceted career in clinical research.

The overarching goal of the Foundations of Clinical Research program is to equip the next generation of researchers with the skill sets essential to evaluating evidence, understanding biostatistics, and beginning their clinical research careers. Our aim is to ensure that learners develop a strong foundation in the design, implementation, analysis and interpretation of clinical research studies.

During the program, our innovative active learning approach emphasizes the traditional tutorial system with weekly live video tutorials, seminars and symposia anchored by 3 live intense weekend online workshops.  The Foundations of Clinical Research program’s six-month online curriculum emphasizes real-time skill-based learning. 

Participants will be eligible for Associate Alumni status upon successful completion of the program. Early tuition and need-based tuition reductions may be available.

Course Outline

Live Workshops

The interactive workshop curriculum will focus on hands-on skill development through active learning. To that end, the intensive schedule is designed to accelerate the growth of high-yield clinical research skills via individual and team-based workshop exercises. Students will be immersed in a dynamic learning environment that encourages collaboration and collegial networking with faculty and peers. 

Essential elements of the workshop include instruction and practical exercises in the core concepts of biostatistics, epidemiology and research question development, as well as critical assessment of the medical literature and practical training in statistical software using real-life datasets. In addition to providing training in mentorship, academic career development and leadership, we create a supportive and active learning environment where opportunities for knowledge retention and networking abound.

Live Symposia, Tutorials and Seminars

Symposia, tutorials and seminars are mandatory and will be delivered live online and organized according to eight specific clinical research topics. 

Eight 3-Hour Symposia

• Instruction on a specific clinical research topic (e.g., cohort study design and interpretation)
• In-depth discussion on a related epidemiology concept (e.g., odds ratio)
• Hands-on guidance for implementing the related analysis with statistical programming in Stata

Eight 1-Hour Tutorials

• Interpret and report on papers related to the specific clinical research topic

Eight 1-Hour Special-Topic Seminars

• The biostatistical and epidemiological concepts to specific clinical research topics with concrete examples

Assignments

All students will be expected to complete all assignments by the due dates. Assignments will be graded as either “pass” or “fail.”

Individual Assignment 1

Individual Research Question and Study Design

• Generate a novel research question in the evidence-based PICO format
• Receive expert faculty review

Individual Assignment 2

Design, Implement and Present an Original Abstract

• Design and implement a clinical research study based on a publicly available dataset
• Analyze and create data visualizations via a user-friendly R Shiny web app
• Write a formal 350-word abstract suitable for submission to an international conference
• Present a digital poster to faculty at Workshop 3

Online Lectures

Research Study Introduction 

• Designing a Clinical Research Study I–III
• Introduction to Evidence-Based Medicine, Systematic Review and Meta-Analysis
• Study Design 1 – Observational
• Study Design 2 – Randomized Controlled Trials
• Study Design 3 – Quasi-Experimental Studies
• Introduction to Biostatistics
• An Investigator’s Responsibility for Protection of Research Subjects
• How to Search PubMed
• Overview of Evidence-Based Medicine

Statistical Programming in Stata

• Loading Data
• Basic Programming Commands
• Data Cleansing
• Data Analytics I – Central Tendency
• Data Analytics II – Statistical Testing
• Data Analytics III – Regression Testing

Instructors

Jamie Robertson

Djøra Soeteman

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A Consumer’s Guide to Clinical Trials

A clinical trial is a research study that involves human volunteers. Heralded as the best, if not the only way, to advance medicine, clinical trials are designed to evaluate new ways to prevent, detect, or treat disease. ( 1 , 2 )

Every prescription medicine on the market and every vaccine in use was once the subject of a clinical trial. Countless lives have been saved by penicillin and the smallpox vaccine, to reach back in history and name just two. Current advances include a long list of immunotherapies now approved for use by the U.S. Food and Drug Administration (FDA) to treat lung cancer , as well as other cancers.

Only prescription medications require clinical trials before approval by FDA. Over-the-counter medications and supplements typically do not require a clinical trial, according to the Congressional Research Service.

The number of clinical trials being conducted in the United States and around the world is higher than it’s ever been. A total of 439,377 studies are currently registered with ClinicalTrials.gov , one of the largest databases of clinical trials in existence. Of these, 63,922 are actively recruiting participants. That’s up from just 1,255 trials, in total, when the National Institutes of Health (NIH) website first became available to the public in early 2000. ( 3 , 4 )

Yet despite efforts by both pharmaceutical companies and public health agencies to educate people about the potential benefits of clinical trials and to recruit volunteers, public awareness about clinical trials remains relatively low — as does participation, particularly among women, children, and minorities, who tend to be underrepresented in research. ( 5 , 6 , 7 )

One study found that while women were well-represented in trials of drugs for hypertension and atrial fibrillation , the numbers of those participating in studies involving heart failure, coronary artery disease , and acute coronary syndrome were significantly lower than the relative numbers of women affected by those conditions in the general population. ( 8 )  Cardiovascular disease continues to be the leading cause of death among women (and men) in the United States.

Indeed, recruitment in general is considered a major challenge for those leading these trials, and many studies never get off the ground due to a lack of volunteers. Studies involving cancer therapies are known for being hard hit. Over 18,000 cancer clinical trials are in the recruitment stage , but estimates put the rate of participation among cancer patients at as low as 6 percent of potential trial candidates.

Among commonly cited reasons why some cancer patients may not enroll in clinical trials: simple lack of awareness among potential volunteers, mistrust of the process and fear of being a “guinea pig,” fear of not receiving the new therapy but rather the “standard of care” comparison therapy, restrictive inclusion criteria — for example, those who have had prior chemotherapy may be ineligible — and fear of side effects of new drugs. ( 9 , 10 )

Clinical Trials Past and Present

The history of clinical trials is said to date back to 1747, when British physician James Lind conducted a systematic trial among British sailors with scurvy — a disease now known to be caused by a deficiency in vitamin C — by dosing some sailors on board a long sea voyage with a combination of orange and lemon juice to see if it helped cure the condition.

A variety of studies soon followed, but it wasn’t until the twentieth century, when randomized trials came into play, that things took a big leap forward. ( 11 ) Randomization involves dividing participants into separate groups by chance, not by selecting them for certain features, to compare different treatments. The first recognized randomized trial was carried out in the mid-1940s by the British Medical Research Council to test the efficacy of the antibiotic streptomycin against tuberculosis .

This trial also featured two other methodological advances in experimentation in that it was double-blind (neither investigators nor patients knew whether they were getting the active drug) and placebo-controlled (some patients received an inactive substance).

Today, most large clinical trials involving drugs that are likely en route to FDA approval are randomized, double-blind, and placebo-controlled — a combination known in medicine as the gold standard. Still, trials involving medical devices and surgeries are frequently not randomized, and many, if not most, devices aren’t subject to the same clinical rigor as drugs.

The vast majority of clinical trials involve drugs or biologics — medicinal preparations like vaccines and immunotherapies that stimulate the immune system to fight infection and disease. At last count, 209,104 such trials were registered with ClinicalTrials.gov , more than double the number of studies involving surgical procedures and medical devices combined. (4)

The Phases of Clinical Trials: 1, 2, 3, and 4

Clinical trials are usually carried out in humans after laboratory and animal testing demonstrate that a treatment is promising. The trials move through a series of phases and follow protocols including plans to balance potential risks with benefits. Trials are typically conducted in four phases:

Phase 1  Researchers test a treatment in a small group of people (about 20 to 80), for a period of several months. The main purpose is to learn about safety and to identify side effects, often as dosage levels are increased. About 70 percent of drugs pass this phase of testing.

Phase 2  Usually (but not always) a larger group of people (100 to 300) receive the treatment to further assess efficacy as well as safety and side effects. About one-third of experimental drugs successfully complete both phase 1 and phase 2.

Phase 3  The treatment is given to still larger numbers of people (1,000 to 3,000) and can last several years. This phase is intended to provide investigators — and, ultimately, the FDA — with more complete information on effectiveness and the range of possible adverse reactions. Between 25 and 30 percent of drugs entering phase 3 studies succeed. After a successful phase 3 trial, a pharmaceutical company can request FDA approval to market the drug or biologic.

Phase 4  This phase, also referred to as post-marketing surveillance, occurs after a treatment has been approved by the FDA and is made available to the public. At this stage, researchers are supposed to track safety in the general population and gather information on benefits and optimal use. If problems occur, restrictions may be placed on the drug or result in its being taken off the market. ( 12 , 13 )

Should You Participate in a Clinical Trial?

People say they participate in clinical trials for a variety of reasons, including to play an active role in their healthcare; to help others by contributing to medical research; to gain access to treatments before they are widely available; to get medical attention and care they might not ordinarily receive; and even to earn money. ( 14 , 15 )

Satisfaction rates among people who have participated in a clinical trial run high: One report found that 95 percent of those who had taken part in a trial in the past said they would consider joining another in the future. ( 16 )

Still, many potential first-time volunteers never move forward. There are a number of reasons for that. Some, particularly minorities, are disturbed by memory of missteps in the past, primarily the Tuskegee Syphilis Experiment (1932–1972), in which African-American men were told by the U.S. Public Health Service that they were being treated (at no charge) for syphilis , but who in fact received no treatment at all for the communicable and often deadly disease. ( 17 )

Still others are cognizant of past trials that resulted in products or treatments that subsequently turned out to be problematic for some reason, such as thalidomide , the sedative marketed to pregnant women in the late 1950s for morning sickness that caused thousands of severe birth defects; and the Dalkon Shield, the intrauterine device (IUD) used in the early 1970s that prompted more than 200,000 lawsuits by women harmed by the device.

Other commonly cited concerns include: fears about side effects; doubts that the therapy will be any better than the standard of care; and worry about receiving a placebo — as in, why would anyone want to risk getting a placebo, especially if they are very ill? (14 )

While experts in the field acknowledge that the risks of enrolling in a clinical trial are real and entail many unknowns, they also say that some concerns are unfounded. For example, placebos are rarely used in trials involving patients with more advanced or serious illness, says Kenneth Getz , founder of CISCRP and deputy director of the Tufts Center for the Study of Drug Development, explaining that most use an active comparison drug, typically the FDA-approved standard of care . In addition, volunteers are free to withdraw from a trial at any time and for any reason. ( 18 )

Understanding Informed Consent

Over the years, and often in response to mistakes from the past, practices have been put into place to protect and inform patients. For example, the Tuskegee tragedy led to a process known as informed consent, which involves informing prospective volunteers about the intervention in question and the potential risks involved. Informed consent is now considered both a critical component of patients’ rights and clinical trials themselves. (6)

During the informed consent process, people should be provided with information that enables them to make an informed and educated decision about whether to enroll in the trial. That includes explaining to prospective subjects the purpose of the research, what their role would be and how the trial will work, and allowing them adequate time to ask questions and discuss things with family and friends. The FDA also notes that it’s important for people to understand their role as a “subject of research” — not as a patient. ( 19 )

Finding a Trial That Suits Your Needs

Clinical studies are conducted in a variety of types of locations, including hospitals, universities, doctors’ offices, and community clinics. The location depends on who’s conducting the study. Funders can include governmental agencies, industry groups, individuals, universities, and other organizations, such as foundations and not-for-profits. Locations now exist in all 50 states and in more than 200 countries around the world. (4)

Trials can be found for virtually every condition imaginable. While many of the most commonly publicized trials often involve high-stakes therapies, such as those for late-stage cancer, lower-stakes trials are plentiful. Registered trials include treatments for everything from acne to insomnia to anxiety. (4)

And they’re not all about pharmaceuticals. Many clinical trials involve interventions with therapies such as botanicals, mineral supplements, and acupuncture — even aromatherapy and yoga for hot flashes, for example. (4)

People with a preference for lifestyle interventions to prevent or treat illness can help play a role in moving those disciplines forward as well. A number of trials involving diet and exercise have been registered.

These include studies that have now been completed assessing the effects of low-fat vegan versus  Mediterranean diets  on body weight and insulin sensitivity, run by the Physicians Committee for Responsible Medicine, and the effects of  intermittent fasting  in subjects consuming a Mediterranean or Western diet, performed by the Washington University School of Medicine on the prevention and treatment of age-related diseases. (4)

In the end, whether to participate in a trial is a personal decision, but perhaps the most important takeaway is that the decision be an informed one. Whether the thinking is “buyer beware” or “well-informed is well-armed,” the consensus is that it’s best to gather as much information and input as possible from trusted and valued sources, including doctors and other health professionals as well as family and friends.

“Our motto is ‘education before participation,’” says Getz. “We encourage patients and their families and friends to gather the facts, speak with other patients and professionals, and ask a lot of questions.”

There are several searchable databases that offer listings of available clinical trials, including:

• ClinialTrials.gov

Additionally, you may find searchable listings at local healthcare centers, on the websites of disease specific organizations, and even advertised in local newspapers. (14,19, 20 , 21 , 22 )

Things to Keep in Mind When Considering a Clinical Trial

In most cases, participating will be at no cost to you. Sometimes, insurance may be billed for some devices, drugs, or services. These should be spelled out in the informed consent process.

What to Ask Can you spell out what the cost will be to me?

Some clinical trials will not require travel. Others will require regular check-ins. Find out what will be required of you beforehand.

What to Ask  Will my travel costs be covered?

A treatment trial may be comparing a new treatment to one that is the standard, or to a placebo. You have no control over which arm of the trial you are assigned to.

What to Ask  Am I willing to participate if I’m not getting the new treatment?

You can opt out of a trial at any time.

What to Ask  How do I handle the situation if I decide to opt out part way through the trial?

Some trials involve years of follow-up.

What to Ask  When does the trial end, and how long will my obligation last?

Editorial Sources and Fact-Checking

Everyday Health follows strict sourcing guidelines to ensure the accuracy of its content, outlined in our editorial policy . We use only trustworthy sources, including peer-reviewed studies, board-certified medical experts, patients with lived experience, and information from top institutions.

• NIH Clinical Research Trials and You.  National Institutes of Health .
• What Are Clinical Trials and Studies?  National Institute on Aging . March 22, 2023.
• Learn About Clinical Studies.  ClinicalTrials.gov . March 2019.
• Trends, Charts, Maps.  ClinicalTrials.gov . February 2, 2023.
• Survey: 50% of Americans Not Aware of Clinical Trials.  Clinical Leader . April 20, 2017.
• Clinical Trial Diversity.  U.S. Food and Drug Administration . November 4, 2022.
• Women and Health Research: Ethical and Legal Issues of Including Women in Clinical Studies: Volume I.  National Library of Medicine . 1994.
• Scott PE, Unger EF, Jenkins MR, et al. Participation of Women in Clinical Trials Supporting FDA Approval of Cardiovascular Drugs.  Journal of the American College of Cardiology . May 2018.
• Survey of Clinical Trial Awareness and Attitudes [PDF]. SubjectWell .
• How to Maximize Patient Recruitment in Oncology Trials [PDF].  BioPharmaDive.com .
• Junod SW. FDA and Clinical Drug Trials: A Short History [PDF].  U.S. Food and Drug Administration . 2008.
• NIH Clinical Research Trials and You: Glossary of Common Terms.  National Institutes of Health . April 20, 2023.
• About Clinical Trials.  CenterWatch .
• Volunteering for a Clinical Trial.  CenterWatch .
• Deciding Whether to Be Part of a Clinical Trial.  American Cancer Society . August 22, 2022.
• 2017 Perceptions and Insights Study: Public and Patient Perceptions of Clinical Research [PDF].  The Center for Information & Study on Clinical Research Participation .
• Washington HA. Medical Apartheid: The Dark History of Medical Experimentation on Black Americans From Colonial Times to the Present . January 8, 2008.
• Become a Research Volunteer [PDF]. U.S. Food and Drug Administration Office of Minority Health .
• Informed Consent for Clinical Trials. U.S. Food and Drug Administration . January 4, 2018.
• Questions to Ask Before Participating in Clinical Research.  National Institute on Aging . March 22, 2023.
• Getting Answers: Questions to Ask Before Enrolling in a Clinical Trial [PDF].  The Center for Information & Study on Clinical Research Participation .
• Women in Clinical Trials = Hope [PDF].  U.S. Food and Drug Administration . 2016.

Additional Sources

• Unger JM, Fleury M. Nationally Representative Estimates of the Participation of Cancer Patients in Clinical Research Studies According to the Commission on Cancer. Journal of Clinical Oncology . October 1, 2021.
• The Drug Development Process, Step 3: Clinical Research. U.S. Food and Drug Administration . January 4, 2018.
• Standard of Care. National Cancer Institute .
• FDA Regulation of Over-the-Counter (OTC) Drugs: Review and Issues for Congress. Congressional Research Service . December 10, 2021.

Clinical Trials 101

Video of webinar for public health professionals introducing clinical trials and explaining key concepts, June 2011.

• Clinical Trial FAQs

Clinical Trials 101

On This Page:

Phases of clinical trials, types of ovarian cancer clinical trials, costs of clinical trials, clinical trials process, what is a clinical trial .

A clinical trial is a highly-controlled biomedical research study, which aims to test the safety and effectiveness of new treatments, diagnostics, and methods of screening and prevention. Once an experimental treatment reaches the clinical trial stage, it has already gone through a lengthy, rigorous scientific process. Clinical trials are all strictly regulated for eligibility and safety.

Every treatment available today is the result of a clinical trial. Yet only three percent of U.S. adults with cancer (all cancer types) participate in clinical trials, but it is estimated that 20 percent are eligible. 

A patient is eligible to participate in a clinical trial at any point in their experience with ovarian cancer. Many people think of clinical trials as a “last resort” option – one to explore only  after  other treatments have failed – but this isn’t the case at all. Many equally important trials are available for patients earlier in their fight against ovarian cancer.

Clinical trial participants will  never  receive just a placebo. Everyone who participates will receive either standard of care treatment, or the new treatment that is being tested. In this way, clinical trials offer two invaluable benefits: for the individual, they have potential to succeed where other treatments have failed – and at the same time, they accelerate scientific progress for everyone. 

If you are interested in a clinical trial, you should not hesitate to ask your doctor about whether there are trials that might be right for you. If your physician does not know how to find trials, refer them to the  Find a Clinical Trial  section of this site. Our  Questions to Ask Your Doctor  will help prepare you to discuss clinical trials with your medical team.

Find Gynecologic Cancer Clinical Trials

OCRA’s Clinical Trial Finder is a one-stop service to find information on current clinical trials for ovarian cancer and other gynecologic cancers in the U.S. and Canada. Search online, or connect with a real person on the phone to guide your search. You’ll receive a personalized list of available clinical trials specific to your diagnosis, treatment history, and location, and get updates when new studies open that match your profile.

Visit OCRA’s Clinical Trial Finder

Every clinical trial has a “sponsor” that oversees the conduct of the trial. The  National Cancer Institute  and other parts of the  National Institutes of Health  and the Department of Defense sponsor and conduct ovarian cancer clinical  trials.

In addition, organizations or individuals, such as medical institutions, pharmaceutical and biotech companies, and even individual physicians sponsor clinical trials.

Clinical trials take place in all kinds of locations where people receive medical care across the United States.

• Doctors’ offices
• Major hospitals and cancer centers
• Community hospitals and clinics

Clinical trials occur in sequential phases, each of which serve a different function. Here is how the National Cancer Institute describes the trial phases:

In  Phase I  trials researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

In  Phase II  trials, the experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

In  Phase III  trials, the experimental study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.

In  Phase IV  trials, post marketing studies delineate additional information including the drug’s risks, benefits, and optimal use.

There are many different types of clinical trials for ovarian cancer patients. Trials can focus on prevention, screening, diagnostic, treatment, quality of life and supportive care, or genetics.

Prevention trials test ways to reduce the  risk of ovarian cancer . They typically enroll healthy women at high risk for developing ovarian cancer or survivors who want to prevent its  return  or reduce the chance of developing a new type of cancer.

Screening trials look for ways to detect ovarian cancer at an early stage in healthy women.

Diagnostic trials seek to develop better ways to diagnose and care for patients with ovarian cancer. They usually enroll patients who have already had ovarian cancer or who have  signs and symptoms  of it. Many of the current diagnostic trials in ovarian cancer focus on proteomics, which involves evaluating the levels of different proteins in the blood.

Treatment trials determine what new treatments or combinations of  existing treatments  can help women with ovarian cancer. They evaluate the effectiveness of new treatments or new ways to use existing treatments. (A “treatment” may be a drug, therapy vaccine, surgery or any combination of these.) Various treatment trials exist, most of which explore the effectiveness of different combinations of surgery and drug therapies in fighting ovarian cancer.

These trials aim to improve the quality of life for ovarian cancer patients, survivors, and their families. These may include issues like  side effects from chemotherapy  like neuropathy or nausea, or need for pain medication.

Usually part of another clinical trial, genetics trials attempt to determine how a woman’s genetic makeup can influence the detection, diagnosis, prognosis and treatment of ovarian cancer. (Family-based genetic research studies exist that differ from cancer clinical trials; in these studies, multiple high-risk family members may give blood and tissue and agree to be evaluated on an annual basis.)

Many people wonder, what are the costs associated with enrolling in a clinical trial? The clinical trial sponsor pays for the costs associated with the treatment under study, such as drugs being compared, and will pay the extra costs associated with additional testing or doctor visits required by the trial’s protocol. Routine patient costs, such as doctor visits, hospital stays, laboratory tests, are the costs of medical care you would have received if you were not in the trial. Some health insurance plans may not cover routine patient care costs in a clinical trial. You should work with the clinical trial research coordinator to help you find out if routine costs are covered by your insurance plan. To try to help get an insurance company to pay for a trial, the National Cancer Institute, suggests that you ask the research coordinator to provide you with medical literature that show potential benefits of the treatment tested, a letter of clinical necessity, documentation from the researchers explaining the clinical trial, and support letters from an advocacy group to submit to an insurer.

Health insurers use several criteria to determine whether to cover the costs of a clinical trial. According to the National Cancer Institute:

• If you live in a state that requires coverage for clinical trials. For more information go to States that Require Health Plans to Cover Patient Care Costs in Clinical Trials
• If the policy allows coverage of routine patient care in a clinical trial That the trial is medically necessary, which is decided usually on a case-by-case basis
• If the trial is a phase III, the insurer might cover it because the treatment has already had success in many people
• If the routine costs of care in the trial are comparable to the costs for standard treatment
• The trial is sponsored by the National Institutes of Health or one of the groups it funds.

There are also resources available from not-for-profit organizations to help with the costs of clinical trials. You may be able to find assistance by searching through this  directory on the NCI website .

Eligibility

Before you can join a clinical trial, your eligibility to participate has to be determined. Inclusion and exclusion criteria are used so that patient safety is maintained, particular research questions can be answered, and credible results are obtained. Your age, health, previous medication history, and  type  and  stage of ovarian cancer , are factors used to determine your eligibility.

Informed consent

Before participating in a clinical trial, research coordinators will explain the purpose, procedures, risks and benefits of a trial. Translators are available for people who do not speak English. Once you fully understand what the trial entails, you will be asked to sign a document that includes in writing details about the study. The informed consent document is not a contract and you can leave a trial any time you wish. You can also always ask the staff members of a trial any question you have while the clinical trial is ongoing.

Patient protection

Federal rules and local laws ensure that clinical trials are performed ethically and respect the safety and rights of the patient. A clinical trial is evaluated by scientific review panels and Institutional Review Boards (IRB) before it can begin. A Scientific Review Panel, comprised of experts, determines whether a clinical trial protocol has sufficient scientific merit before patients participate. Members of an IRB, including physicians, statisticians, researchers, community activists and others, also review a trial’s protocol before patients are enrolled to make sure the trial’s benefits outweigh its risks. IRBs and Data Safety and Monitoring Boards for Phase III trials monitor ongoing trial progress. DSMBs, which include physicians, statisticians and other experts, work to minimize risks, ensure data is unbiased and accurate, and stop a trial if safety concerns arise.

Trial locations

Trials take place in cities and towns throughout the United States, and other countries. Doctors’ offices, cancer centers, medical centers, community hospitals, clinics and veterans’ and military hospitals are all possible venues for clinical trials. Some trials may only take place in one facility, such as a large medical center, while other trials occur at multiple locations.

Trial sponsors

There are a variety of sponsors of clinical trials, including government agencies, such as the National Cancer Institute, organizations, physicians, academic medical centers, foundations, and biotechnology and pharmaceutical companies. Various groups work together to form networks, such as the Gynecologic Oncology Group, which designs and manages the majority of the ovarian cancer clinical trials in the United States. OCRA, for example, co-sponsors clinical trials.

The research team

A research team, including doctors, nurses, research assistants, and data analysts, manages the trial. They work with other health care professionals, including laboratory technicians, pharmacists, dieticians and social workers to provide medical and supportive care for participants. The research team checks your health at the beginning of the trial, gives instructions regarding the protocol you are participating in, monitors your health carefully during the trial and stays in touch after the trial is completed. They will also respond to any side effects you may be experiencing. Some clinical trials require more tests and doctor visits than you would have gotten normally. Trial success depends on adhering to the protocol strictly and maintaining frequent contact with the research staff. You will also stay in contact with your regular health care provider, who communicates with the research team.

After a clinical trial is completed

After a clinical trial is over, the data collected is analyzed to understand the results and whether it is necessary to plan additional research. The results are usually published in peer-reviewed scientific journals so expert medical “peers” can assess the integrity of the research findings. Sometimes important results are announced at scientific meetings, which get covered in the media, before the findings are published. After an intervention has proven safe and effective in a clinical trial, it may become a new standard of care.

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Deborah D. Gordon has spent her career trying to level the playing field for healthcare consumers. She is co-founder of Umbra Health Advocacy , a marketplace for patient advocacy services, and co-director of the Alliance of Professional Health Advocates , the premiere membership organization for independent advocates. She is the author of "The Health Care Consumer's Manifesto: How to Get the Most for Your Money," based on consumer research she conducted as a senior fellow in the Harvard Kennedy School's Mossavar-Rahmani Center for Business and Government. Deb previously spent more than two decades in healthcare leadership roles, including chief marketing officer for a Massachusetts health plan and CEO of a health technology company. Deb is an Aspen Institute Health Innovators Fellow, an Eisenhower Fellow and a Boston Business Journal 40-under-40 honoree. Her contributions have appeared in JAMA Network Open, the Harvard Business Review blog, USA Today, RealClear Politics, The Hill and Managed Care Magazine. She earned a BA in bioethics from Brown University and an MBA with distinction from Harvard Business School.

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How to Get into a Clinical Trial

It helps to know how to advocate for yourself and who to turn to for help.

If you or a loved one has a serious disease or condition, joining a clinical trial may help you get access to new treatments. It also lets you contribute to society by being part of scientific discovery that could help others.

But how do you join a clinical trial? You can’t just sign up. It’s a decision that should be made with your healthcare team. And, then, you have to qualify. It’s also important to think about the costs, timing, travel and how being in the trial will work with your ongoing care.

These questions may feel overwhelming — but figuring them out could save your life.

Here’s what you should know about getting into a clinical trial.

Why join a clinical trial?

When you join a clinical trial, you may be able to get access to new drugs or treatments that you might not otherwise receive. You will also be a part of the research that goes into developing new treatments that could help other people.

"If you have a tough-to-treat cancer that is historically not responsive to standard of care, participating in a trial could increase your chances of getting access to innovative therapy before it becomes available on the market," said Dana Hutson, a board-certified patient advocate and founder of Cancer Champions , a medical advocacy firm that helps patients and families navigate cancer. "Sometimes, access to the most promising and innovative treatments for hard-to-treat cancers can only be accessed through a clinical trial."

In a 2023 HealthyWomen survey, 54% of women surveyed said they would consider joining a clinical trial if there were potential benefits.

In addition to the benefits, there can also be downsides. First, there's no guarantee that the new treatment will work for you. And the new treatment could cause side effects. You also may not get the new treatment in the trial. In a clinical trial, some people get the experimental treatment while others don't, so scientists can see how old and new treatments compare. But, according to Hutson, you'll get at least the standard of care if you have cancer.

"You will not be treated like a guinea pig," she said. "Technology has come such a long way that scientists and clinicians know so much about a new therapy before it even enters into the clinical trial setting, even if it is a 'first-in-human' trial."

How to find a clinical trial

To find a study for your condition, you can search publicly and privately funded clinical trials at ClinicalTrials.gov . You can also check with disease-specific advocacy or patient groups.

According to Hutson, the clinical trial landscape is always changing. "There are trials opening and closing all the time," she said.

Searching on your own might be overwhelming. Each study has specific rules for who can participate, which may be hard to figure out.

"There are usually strict criteria for acceptance into a trial," Hutson said. "If you embark on your own search, it is important to understand, at a minimum, what kind of cancer you have (cell type) — not just 'breast cancer': where it is in your body, the number and kind of prior therapies you have had, the genomic profile of the tumor, and your overall health — before beginning your search."

Several companies help match patients with trials, including some that use AI to do so . The services are usually free for patients, although you'll probably have to consent to sharing your medical records.

It's important to discuss clinical trials as an option with your healthcare team. Your healthcare provider (HCP) might know of relevant trials or help you find one. Other HCPs who specialize in your condition might be good sources of information about specific trials for you. (Those specialists might even be conducting the research themselves.)

The HealthyWomen survey showed that almost 3 out of 4 women would want to learn about clinical trials from their HCP. And nearly half (46%) of survey respondents said they'd consider participating in research if their HCP recommended it.

But not every HCP knows about available trials for your condition.

"Your physician may not bring up participation in a clinical trial," Hutson said. "If it is something you would like to know more about, you may have to ask. And even if they tell you they don't have anything for you, that does not mean there is not a trial out there that may be perfect for your situation. You often have to be proactive and do the research yourself."

Questions to ask before signing up for a clinical trial

Before signing up for a clinical trial, it's important to understand what you're getting into. Study organizers must get your informed consent. That means they have to share specific details with you about the purpose of the research, how the trial will work, the patient's role, potential risks and the patients' financial responsibility — all before you agree to participate.

"These [informed consent] documents are often long, and it is advisable for the patient to take them home and discuss [them] with their family, friends or an advocate prior to signing," Hutson said.

Hutson suggests several questions to consider, including:

• Has the drug been studied before?
• Is there any published information about the drug being studied?
• How many other patients are in or have been in the trial? What have their experiences been?
• Is the study drug paid for by the research sponsor or will insurance cover it?
• What other financial obligations are there for participants?
• What are the risks of participating in the trial?
• What benefits or outcomes could participants expect?
• Are there at-home options or do participants have to travel?

The bottom line, according to Hutson: "Ask questions about anything you do not understand."

Your HCP's role

Although you may be able to participate in a clinical trial from home, most involve some centralized testing or treatment at a study site, or a combination of at-home and on-site procedures.

Ideally, your HCP(s) and the trial organizers will coordinate your care. Hutson suggests setting up a call between your HCP(s) and the study coordinator when you first sign up and as needed to make sure everyone involved in your care knows what’s going on. If you’re comfortable communicating electronically, you can use your patient portal to communicate updates with your HCP. You also may be able to link your patient portal to the trial site’s portal.

Ultimately, if all the legwork involved in getting into a clinical trial is too overwhelming, you can contact a care coordinator or patient navigator service through your insurance company (if they offer one) or hire a private patient advocate. These professionals can help with all of the stages involved in finding, navigating and participating in a clinical trial, including facilitating and coordinating communication between HCPs.

This resource was created with support from Pfizer, a HealthyWomen Corporate Advisory Council member.

• Why Diversity in Clinical Trials Is Important ›
• I Was Immunocompromised and a Clinical Trial Changed My Life ›
• Why We Need More Black and Hispanic Women in Breast Cancer Clinical Trials ›
• What You Need to Know About Clinical Trials ›
• Steps to Find a Clinical Trial - NCI ›
• How to Join a Clinical Trial | Memorial Sloan Kettering Cancer Center ›
• Finding a Clinical Trial | National Institutes of Health (NIH) ›

The Great Girlfriends Podcast: Clinical Trials 101

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Video Jul | 28 | 2021

Clinical Trials 101

Learn about clinical trials in this video with Elizabeth Abraham, RN, clinical research nurse at Mass General Cancer Center. Elizabeth describes the various aspects involved with clinical trials in order to help improve your understanding so you can better navigate the process of being enrolled in a clinical trial.

Related Pages

• Cancer Center Clinical Trials
• Termeer Center for Targeted Therapies

Centers and Departments

Related news and articles.

• Nov | 17 | 2020

Video: Termeer Center for Targeted Therapies

With its focus on innovative clinical trials, the Henri and Belinda Termeer Center for Targeted Therapies at the Mass General Cancer Center is committed to offering its patients the best and most advanced treatments available.

• Jul | 7 | 2020

Clinical Trials and Research

Dr. Rebecca Heist and Casandra McIntyre, RN discuss what clinical trials are and why they are important. Learn how clinical trials are benefitting patients and how we are keeping enrollees safe.

• Jan | 1 | 2020

Leapfrog Moments in Cancer Therapies with Keith Flaherty, MD

Go behind the scenes with our Director of Clinical Research, Dr. Keith Flaherty to learn how teams of researchers and clinicians are applying scientific insights to propel the creation of new therapies for patients, and get to know more about what motivates him to do what he does every day.

An integral part of one of the world’s most distinguished academic medical centers, the Massachusetts General Hospital Cancer Center is among the leading cancer care providers in the United States.

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Clinical Research in Primary Care

Program snapshot.

There is a critical need to extend research participation opportunities to broader communities. This is, in part, because people who are historically underrepresented in research are also often medically underserved and disproportionately impacted by disease and illness (e.g., certain racial and ethnic groups, gender minority groups, people who live in rural environments, older adults, persons experiencing challenging social determinants of health and related experiences). When research study demographics don’t match the demographics of the illness or condition being studied, generalizability of the evidence is compromised, further compounding these health disparities. While most have not talked with their doctor about participating in health research, Americans increasingly agree that opportunities for participation in clinical trials should be a part of regular healthcare. 

Therefore, NIH is proposing to establish a network to conduct research in primary care settings. This network will address barriers to access to clinical research participation by implementing a sustained infrastructure that integrates innovative research with routine clinical care in real world settings, with a focus on sustained engagement with communities that are traditionally underrepresented in clinical research. Improving access to clinical studies will facilitate and accelerate research advances for adoption and implementation into everyday clinical care, improving health outcomes and advancing health equity for all Americans. 

The four goals of CARE for Health are to: 

• Pilot and implement the infrastructure for a network on primary care-focused clinical research to serve all ICs, particularly those without their own large-scale networks.
• Establish a foundation for sustained engagement with communities underrepresented in clinical research (e.g., racial and ethnic minority groups, gender minority groups, people who live in rural environments, older adults, persons experiencing challenging social determinants of health and related experiences).
• Implement innovative study designs that address common health issues, including disease prevention.
• Engage with community and health care systems to integrate innovative science with routine clinical care and change clinical practice as a result of the research outcomes

The network will involve the following components: 

• Providing oversight of the studies/protocols and site and study selection.
• Providing statistical and data management support
• Developing innovative clinical study designs and implementation strategies to minimize burden on participants and providers in primary care settings.
• Operations Center – conducting site feasibility assessments, site agreements/contracting, and coordination of study operations (protocol development; compliance with Food and Drug Administration (FDA) and Office of Human Research Protections (OHRP) regulatory and participant protection requirements; communications; training; auditing; quality assurance; and data monitoring)
• Independent Review and Monitoring Boards - including Data and Safety Monitoring Board (DSMB), Observational Study Monitoring Board (OSMB), and the Central Institutional Review Board (IRB)
• Network Research Hubs – leveraging existing research networks and partnerships with Clinical Sites to conduct clinical research in primary care settings.
• Community Engagement – providing support, advice, and resources, in part through partnerships with existing entities, to facilitate sustained participant and community engagement, community-driven research, and integration of studies in primary and community care settings.
• Industry Partnerships – engaging for-profit partners for collaborative knowledge sharing and potential participation in/use of the infrastructure.

Announcements

On April 5, 2024, The NIH Council of Councils approved the concept of Establishing a Network for Research in Primary Care. View the presentation slides presented by NIH Director, Dr. Monica Bertagnolli here . And watch the presentation videocast . 

Do you have questions? Email us at: [email protected]

This page last reviewed on May 6, 2024

Clinical Trials 101: A guide to Understanding Clinical Research Language

Clinical research  relies upon an ongoing process that evolves as new data becomes available. Any and all  medical treatments  that are used now first underwent multiple phases of clinical studies in order to become FDA-approved.

There is always a diverse variety of new information being discovered , which can be fascinating to keep up with. However, you shouldn’t have to conduct your own research just to figure out what it all means. We’ve compiled our own glossary of clinical research terminology, so you don’t need to have a career in clinical research  in order to understand the basics.

Understanding Clinical Research vs. Clinical Trials

Clinical research is a type of medical research which uses human volunteers to develop and improve medical treatments. While clinical researchers will continue to evaluate the data post-study, clinical trials are simply the individual studies in which data is collected.

Inclusion & Exclusion Criteria

Inclusion and exclusion criteria refers to the volunteer requirements  for participating in a clinical trial. These factors can include age, gender, medical history, etc., and vary according to each clinical trial. Inclusion criteria are the factors which allow someone to participate as a volunteer, while exclusion criteria disqualify participation.

Healthy volunteer vs. Patient Volunteer

A healthy volunteer is someone with no significant health problems related to the study, while a patient volunteer is someone with a diagnosis or health problem related to the clinical trial in question. Both types of volunteers are important for testing and helping to better understand treatments.

Informed Consent

One clinical research language term you will hear often is informed consent . This is an agreement (in writing) which includes a summary of the clinical trial, treatment procedures and schedules, and risks and benefits. It also lays out all of one’s rights as a volunteer. This includes the right to leave the trial at any time.

Phases of Clinical Studies

There are four phases  of clinical trials: the first phase being a test in a small group of 20-80 volunteers, the second with 100-300 volunteers, and the third with 1,000-3,000 volunteers. The fourth phase is continued research following the FDA approval. OCRC specializes more in Phase 1 clinical trials.

Placebo A placebo  is a part of a treatment which visually appears as the new treatment being investigated but does not have any active ingredients. Volunteers generally do not know whether they are being given a placebo or active treatment; therefore the effects can be compared side-by-side.

Principal Investigator A Principal Investigator  is the person in charge of overseeing a clinical trial. In addition to PIs, there are also several other paths  that can be taken within the clinical research career.

Randomization

Randomization  is when two or more treatments are assigned to volunteers by chance rather than choice, comparing new treatments and standard therapy. This is done in order to prevent bias, such as when doctors assign volunteers to trial groups based upon their medical history or diagnosis.

Single-Blind vs. Double-Blind

A single-blind study means that the participants in a clinical trial do not know whether they are receiving a placebo or the active treatment; a double-blind study means that both the participants and the researchers do not know whether the treatment is the placebo or not. This is done to reduce the risks of bias or a placebo effect , which can produce superficial results.

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Trials to watch: Four ALS drugs to keep an eye on

There are a total of 12 candidates in mid to late-stage trials for ALS poised to enter the market in the next five years.

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It has been a turbulent few weeks for patients with amyotrophic lateral sclerosis (ALS) after Amylyx Pharmaceuticals pulled its lead drug Relyvrio from the market after a Phase III trial did not meet its primary endpoint.

The drug was pegged to be revolutionary for patients as it was said to slow disease progression, however Phase III data showed no difference between the candidate and placebo.

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Despite this setback, chief research officer at the Muscular Dystrophy Association, Dr Sharon Hesterlee, says that there are plenty of novel candidates in the pipeline. “The good news is the pipeline is healthy for ALS, which is important, especially given what has happened with Amylyx’s drug which was disappointing for everyone,” Hesterlee explains.

There are only six FDA approved drugs for ALS – just one of which, riluzole, is a disease modifying therapy, leaving a great need for more treatment options.

Momna Ali, neurology analyst for GlobalData says: “There are 12 assets in their mid-late-stage development (Phase II–III) which are poised to enter the market within the next five years. With numerous pipeline agents that have a diverse mechanism of action under investigation, key opinion leaders interviewed by GlobalData in January 2024 anticipate the treatment landscape to evolve significantly within the next decade.”

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HEALEY Platform trial investigates Calico and AbbVie drug

The HEALEY ALS Platform Trial (NCT04297683) is a perpetual multi-centre, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.

Regimen F is a Phase II/III trial (NCT05740813) which will evaluate the safety and efficacy of a single study drug, ABBV-CLS-7262, in patients with ALS. ABBV-CLS-7262 has been developed in a partnership between Calico Life Sciences and AbbVie .

Regimen F plans to enrol 300 patients with a primary completion date of September 2024.

ABBV-CLS-7262 is an oral candidate which acts by targeting eukaryotic translation initiation factor 2 subunit beta (EIF2B).

The primary endpoint is change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score at 24 weeks.

Secondary endpoints include muscle strength, respiratory function, and disease progression biomarker at 24 weeks.

“In the last couple of years, investigators have really narrowed in on the role of TDP 43 in ALS,” Hesterlee explains. “It will be very interesting to see if blocking that aggregation would be useful. Until we've done the study we're not going to know, but it's certainly nice to see a drug that is trying this.”

Co-director of Massachusetts General Hospital’s Neurological Clinical Research Institute (NCRI), and co-principal investigator in the HEALEY Platform trial, Dr. Sabrina Paganoni, agrees that the candidate has an interesting target.

“This appears to be a promising drug and a very interesting target. I am hopeful if there are positive signals that the candidate could be explored further.”

AL-S Pharma candidate for both sporadic and familial patients

AL-S Pharma is investigating its candidate AP-101 in a Phase IIa trial (NCT05039099) in patients with familial amyotrophic lateral sclerosis (fALS) and sporadic amyotrophic lateral sclerosis (sALS).

The multicentre, randomised, double-blind, placebo-controlled study is evaluating safety, tolerability, pharmacodynamic (PD) markers, and pharmacokinetics (PK) of AP-101.

The study has a primary completion date in June 2024 with an estimated 63 patients.

AP-101 is an intravenous monoclonal antibody which acts by activating superoxide dismutase-1 (SOD1). Mutations in SOD1 leads to ALS.

The primary endpoints in the study are adverse events and abnormalities in vital signs while secondary endpoints will explore levels of cerebrospinal fluid (CSF), changes in neurofilament light chain and phospho-neurofilament heavy chain levels in CSF and plasma.

“It’s a bit of a different way of going after it. It's always good to try these pathways in different ways,” Hesterlee says.

RAPA Therapeutics investigates cell therapy

Rapa Therapeutics is running a Phase II/III trial (NCT04220190) of its candidate RAPA-501 . The trial is an open-label, non-randomised, multi-centre study evaluating RAPA-501 T cell therapy in patients with ALS.

RAPA-501 is an autologous T cell therapy which protects motor neuron cells from inflammation. The cells are manufactured ex vivo using epigenetic reprogramming to yield a T cell population that is enriched for a dual anti-inflammatory phenotype based on hybrid TREG and Th2 differentiation.

Patients are dosed with up to four infusions six weeks apart over the 30-week study. There are 41 patients planned to be enrolled in the trial which has a primary completion in July 2025.

“There is some evidence that T regulatory cells can be helpful in ALS, but it usually requires some kind of reprogramming,” says Hesterlee. “In this case, they're not doing any gene engineering but exposing the cells to change their expression patterns, and in theory become helpful in fighting the inflammation that you see in ALS. This looks interesting and is worth following to see how it works.”

Clene’s CNMAu-8 could be a ‘sprinkle of gold dust’

Clene’s CNMAu-8 is being investigated in a Phase II trial with a Phase III due to be launched in 2024 .

The Phase II trial (NCT05299658) is an open-label extension trial investigating the candidate in patients with early symptomatic ALS on stable background therapy. The oral candidate is administered once daily over a 48-week period. The trial is due to enrol 40 patients. The trial has a completion date in December 2024.

CNMAu-8 is an oral candidate developed with clean-surfaced, catalytically-active gold nanocrystals. The nanocrystalline suspension inhibits the ability of damaging events leading to dysmyelination and negatively regulates myelination and protects the neurons.

“CNM-Au8 has the ability to cross the blood-brain barrier, which allows it to successfully reach and protect neurons in the central nervous system,” Ali says. “Thus far, it has shown to be safe, well-tolerated and most importantly displays survival benefits, more than some other pipeline agents. Clene is all set for a Phase III to be launched in 2024. If it all works out for Clene, it could quite literally be a sprinkle of ‘’gold dust’’ for patients with ALS.”

Paganoni said that Clene’s candidate was investigated as one of the earlier regimens in the HEALEY trial. “It received a good signal in the platform trial and the company is pursuing that. It is definitely one to watch,” Paganoni explains.

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Efficacy and Safety of Nail Psoriasis Targeted Therapies: A Systematic Review

• Systematic Review
• Published: 20 May 2023
• Volume 24 , pages 695–720, ( 2023 )

Cite this article

• Jonathan K. Hwang   ORCID: orcid.org/0000-0002-5066-5057 1 ,
• Jose W. Ricardo   ORCID: orcid.org/0000-0002-8355-4193 1 &
• Shari R. Lipner   ORCID: orcid.org/0000-0001-5913-9304 1  

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Introduction

Nail changes are frequent clinical findings in patients with cutaneous psoriasis and psoriatic arthritis, often causing significant impairments in quality of life. Numerous targeted therapies have been previously studied for treatment of nail psoriasis, however, newer agents have not been captured in prior systematic reviews. With over 25 new studies published since 2020, the landscape of nail psoriasis systemic treatments is rapidly evolving, warranting analysis of recently approved therapies.

An updated systematic review of all PubMed and OVID database studies assessing efficacy and safety of targeted therapies for nail psoriasis was performed, with the goal of incorporating clinical data of recent trials and newer agents, namely brodalumab, risankizumab, and tildrakizumab. Eligibility criteria included clinical human studies reporting at least one of the nail psoriasis clinical appearance outcomes (Nail Psoriasis Severity Index, modified Nail Psoriasis Severity Index).

A total of 68 studies on 15 nail psoriasis targeted therapeutic agents were included. Biological agents and small molecule inhibitors included TNF-alpha inhibitors (adalimumab, infliximab, etanercept, certolizumab, golimumab), IL-17 inhibitors (ixekizumab, brodalumab, secukinumab), IL-12/23 inhibitors (ustekinumab), IL-23 inhibitors (guselkumab, risankizumab, tildrakizumab), PDE-4 inhibitors (apremilast), and JAK inhibitors (tofacitinib). These agents all demonstrated statistically significant improvements in nail outcome scores, compared with placebo or with baseline values, at weeks 10–16 and weeks 20–26, with some studies assessing efficacy up to week 60. Safety data for these agents were acceptable and consistent with known safety profiles within these timepoints, with nasopharyngitis, upper respiratory tract infections, injection site reactions, headache, and diarrhea being the most reported adverse events. Specifically, the newer agents, brodalumab, risankizumab, and tildrakizumab, showed promising outcomes for treatment of nail psoriasis on the basis of current data.

Numerous targeted therapies have shown significant efficacy in improving nail findings in patients with psoriasis and psoriatic arthritis. Data from head-to-head trials have shown greater efficacy of ixekizumab over adalimumab and ustekinumab, as well as brodalumab over ustekinumab, while prior meta-analyses have demonstrated superiority of ixekizumab and tofacitinib to other included agents at various assessed timepoints. Further studies on the long-term efficacy and safety of these agents, as well as randomized controlled trials involving comparison with placebo arms, are needed to fully analyze differences in efficacy of newer agents compared with previously established therapies.

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A Comprehensive Review of Ixekizumab Efficacy in Nail Psoriasis from Clinical Trials for Moderate-to-Severe Psoriasis and Psoriatic Arthritis

Ixekizumab and Ustekinumab Efficacy in Nail Psoriasis in Patients with Moderate-to-Severe Psoriasis: 52-Week Results from a Phase 3, Head-to-Head Study (IXORA-S)

Nail Psoriasis: A Review of Treatment Options

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Hwang, J.K., Ricardo, J.W. & Lipner, S.R. Efficacy and Safety of Nail Psoriasis Targeted Therapies: A Systematic Review. Am J Clin Dermatol 24 , 695–720 (2023). https://doi.org/10.1007/s40257-023-00786-4

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Accepted : 11 April 2023

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Issue Date : September 2023

DOI : https://doi.org/10.1007/s40257-023-00786-4

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A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Giredestrant Plus Palbociclib Compared With Anastrozole Plus Palbociclib for Postmenopausal Women With Estrogen Receptor-Positive and HER2-Negative Untreated Early Breast Cancer (coopERA Breast Cancer)

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This is a randomized, multicenter, open-label, two-arm, Phase II study to evaluate the efficacy, safety, and pharmacokinetics of giredestrant versus anastrozole (in the window-of-opportunity phase) and giredestrant plus palbociclib compared with anastrozole plus palbociclib (in the neoadjuvant phase) in postmenopausal women with untreated, estrogen receptor (ER)-positive, human epidermal growth factor receptor-2 (HER2)-negative, early breast cancer.

The study consists of a screening period of up to 28 days, a window-of-opportunity phase for 14 days, followed by a neoadjuvant treatment phase for 16 weeks (four 28-day cycles), surgery, and an end of study visit (28 days after the final dose of study treatment).

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