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Types of outcomes in clinical research
Tipos de desfecho em pesquisa clínica, juliana carvalho ferreira.
1 . Methods in Epidemiologic, Clinical and Operations Research-MECOR-program, American Thoracic Society/Asociación Latinoamericana del Tórax, Montevideo, Uruguay.
2 . Divisão de Pneumologia, Instituto do Coração - InCor - Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo, São Paulo, Brasil.
Cecilia Maria Patino
3 . Department of Preventive Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
PRACTICAL SCENARIO
In a randomized trial evaluating the efficacy of a new drug for pulmonary arterial hypertension (PAH), patients were randomly assigned to receive the new drug or a placebo. The primary composite outcome was the time to the first PAH-related event (worsening of symptoms, initiation of treatment with prostanoids, lung transplantation, or atrial septostomy) or to death. Secondary outcomes included changes in the 6-minute walk distance (6MWD) and adverse events.
DEFINITIONS
Outcomes (also called events or endpoints) are variables that are monitored during a study to document the impact that a given intervention or exposure has on the health of a given population. Typical examples of outcomes are cure, clinical worsening, and mortality. The primary outcome is the variable that is the most relevant to answer the research question. Ideally, it should be patient-centered (i.e., an outcome that matters to patients, such as quality of life and survival).
Secondary outcomes are additional outcomes monitored to help interpret the results of the primary outcome: in our example, an increase in the 6MWD is inversely associated with the need for lung transplantation. They can also provide preliminary data for a larger study. For example, a preliminary trial that uses 6MWD as the primary outcome may include mortality as a secondary outcome if the power of the study to detect a difference in mortality is low. Although investigators may be tempted to monitor several outcomes, the effort and cost to monitor various outcomes may be prohibitive. Therefore, it is essential to decide which outcome(s) to monitor ( Table 1 ).
PAH: pulmonary arterial hypertension; 6MWD: six-minute walk distance; and PASP: pulmonary artery systolic pressure.
Surrogate outcomes are biomarkers intended to substitute for a clinical outcome, for example, 6MWD as a marker of disease severity in PAH. Surrogate outcomes are typically continuous variables and occur earlier than does the clinical outcome, reducing costs, study duration, and size. Surrogates are commonly used as the primary outcome in phase I and II clinical trials. However, they may lead to false interpretations of the efficacy of the intervention if the surrogate is not a very good predictor of the clinical outcome.
Composite outcomes are made up of multiple variables. In our practical scenario, the primary outcome was composed of several clinical outcomes related to disease progression. Using composite outcomes has the advantage of increasing the power of the study when each of the events is rare and when events are competitive (patients who die cannot have a lung transplant). However, the interpretation of results can be misleading: if the intervention reduces the occurrence of the composite outcome, it does not necessarily mean that it reduces the occurrence of all of its components.
IMPORTANT CONSIDERATIONS
- The study outcomes should be stated a priori (before the researcher looks at the results) in order to avoid the risk of drawing false conclusions by testing every possible variable until one is statistically significant.
- The sample size calculation should be carried out to detect a clinically relevant effect of the intervention on the primary outcome, although calculations can also be made for secondary outcome variables, which may increase the sample size but also increase trial validity.
- More importantly, the choice of the most suitable outcome should be based on the research question and the corresponding hypothesis.
RECOMMENDED READING
Research Outcome Measures
- First Online: 24 March 2020
Cite this chapter
- Fiona Mellor 2 &
- Karen Knapp 3
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An outcome is a consequence or result thus an outcome measure in research is a standardised way of measuring the consequences or results of research. Outcome measures are widely used in clinical practice, service evaluation, and research, however their selection and use may be complicated by the dearth of information available. The selection of outcome measures should be undertaken at a research design stage and should fit with the methodology, aims, and objectives of the research. There are many factors influencing the choice of outcome measure to report results and is important to know whether the chosen outcome measure is valid and reliable, researchers are therefore cautioned against developing their own. This chapter is designed to aid a researcher in understanding classifications and considerations when selecting outcome measures. Advice is presented along with information on how to report outcome measures in the write up of research. Outcome measures used in radiography research are also presented in this chapter.
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Mellor, F., Knapp, K. (2020). Research Outcome Measures. In: Ramlaul, A. (eds) Medical Imaging and Radiotherapy Research: Skills and Strategies. Springer, Cham. https://doi.org/10.1007/978-3-030-37944-5_11
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Outcomes Research: What It Is, What It's Not, and Why It's Important For FITs
February 15, 2017 | Dhaval Kolte, MD, PhD Education
The term "outcomes research" was first introduced in 1998 by Clancy and Eisenberg in their article in Science , which stated that "outcomes research the study of the end results of health services that takes patients' experiences, preferences, and values into account is intended to provide scientific evidence relating to decisions made by all who participate in health care." Realizing the importance and need for outcomes research in cardiovascular diseases, the National Heart, Lung, and Blood Institute of the National Institutes of Health convened a working group on 'Outcomes Research in CVD' in 2004 . The working group defined outcomes research as "applied clinical research that generates knowledge to improve clinical decision-making and health care delivery to optimize patient outcomes. It is the study of the delivery and consequences of health care on outcomes from the perspective of patients, providers, and the health care system."
While these two definitions elegantly capture the essence of what outcomes research is, there are still some common misunderstandings and misconceptions about this field. These have been previously addressed in much detail by Harlan M. Krumholz, MD, SM, FACC , in his article in Circulation: Cardiovascular Quality and Outcomes . I will briefly highlight two of these, which in my experience, are still the most common misconceptions about outcomes research among residents and FITs. First, outcomes research is not defined by its use of observational methodology and existing databases. Rather, it is defined by its objective or the very nature of the question it seeks to answer. Thus, outcomes research can utilize methodology of observational studies (retrospective or prospective), randomized trials, cost-effectiveness/economic analysis, surveys, or meta-analysis, whichever is most suited to the specific research question. Second, outcomes research is not a fad, but is now a well-established field that has grown exponentially over the past decade and a half. This is evident from the increasing number of centers for cardiovascular outcomes research across the country as well as internationally, some of which now even offer formal training programs in outcomes research.
So, why is it important for FITs to engage in cardiovascular outcomes research? As FITs and cardiologists we are trained to practice evidence-based medicine. However, despite our extensive knowledge of randomized controlled trials and best practices, we often lack basic information about contemporary patterns of care, the effectiveness of different clinical strategies in actual clinical practice (at the community, state, national and international level), and the barriers to delivering the best available care and achieving the maximal potential of various interventions. We need information about how to engage patients in decision making, the cost-effectiveness of treatments, and the impact of health care policy decisions on various patient outcomes.
Outcomes research can provide answers to most if not all of these questions. Outcomes research is not a single field, but rather allows you to work with experts across disciplines including basic/translational scientists, clinicians, epidemiologists and biostatisticians. It increases your knowledge and understanding of various research methodologies as mentioned before. Finally, at the individual or personal level, engaging in outcomes research also enables you to collaborate and share ideas with peers, and most importantly, seek mentors both at and outside your institution with whom you can develop life-long relationships. As someone once said, "If you want to be incrementally better: be competitive. If you want to be exponentially better: be cooperative."
This article was authored by Dhaval Kolte, MD, PhD , a Fellow in Training (FIT) at Brown University in Providence, RI, and the FIT Member of the ACC/AHA Task Force on Performance Measures.
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Related Programs
Measurement, design, and analysis methods for health outcomes research, related articles, the value of applying health outcomes research to improve treatment results, how health care executives can use health outcomes research for business decision making, genomic medicine in the next decade, using health outcomes research to improve quality of care.
by Lisa Ellis
There is growing pressure in health care to provide high quality, cost-effective, patient-centered care. Healthcare professionals are increasingly turning to health outcomes research for the evidence-based guidance they need to improve care. Clinicians and executives use this information to assess and improve their business performance or for insight into the most effective treatment options to consider for diverse populations with a range of diagnoses.
What is Health Outcomes Research?
Health outcomes research is a methodology used to identify and measure the link between treatments or interventions delivered and the actual outcomes achieved. Put simply, health outcomes studies help determine what works and what doesn’t in health care. Unlike clinical trials or other highly regulated scientific studies that consider only concrete, measurable data (such as mortality rates), health outcomes research takes a broader view to also incorporate clinical outcomes, financial impact, and a range of functional measures, including patients’ reported quality of life and satisfaction. The data gathered can come from a number of avenues and methodologies including from medical records, insurance databases, patient questionnaires, and more. By looking at a greater range of measures, health outcomes studies can provide guidance on a broader set of interventions and decisions than can clinical trials.
Who is Using Health Outcomes Research?
The Agency for Healthcare Research and Quality (AHRQ) is a federal agency that tracks health outcomes research through a number of initiatives, including through the Healthcare Cost and Utilization Project (HCUP) and the Medical Expenditure Panel Survey (MEPS) as part of its widespread efforts to increase patient safety, decrease medical errors, and identify and address gaps in healthcare quality. The information it provides is used across the country to hold healthcare providers accountable for providing the highest quality care, ensuring cost containment, and achieving optimal outcomes.
AHRQ also works with the Centers for Medicare and Medicaid Services (CMS) on the Medicare Health Outcomes Survey (HOS), which uses patient-reported information to monitor and assess the performance of its managed care plans and to identify and reward those that meet the highest standards.
How Is the Data Applied?
The data compiled by AHRQ, CMS, and others is regularly applied in a number of ways and settings. For instance, some regulating agencies are using outcome data to establish reimbursement structures that encourage collaboration and efficiency, as well as to provide a mechanism to identify successful programs and to share their lessons learned.
Health outcomes data is also being used to create measurement tools that can be used to provide a mechanism to compare different facilities and performance ratings. This makes it possible to identify areas of weakness in facilities, as well as to assess gaps in treatments or interventions being performed, and can help healthcare providers to address these issues and make necessary improvements.
There is growing pressure to provide high quality, cost-effective, patient-centered health care
As the health field continue to shift toward adopting a population health strategy that focuses on addressing the health needs and outcomes of specific populations, clinicians are also applying health outcomes data to identify those interventions that are most effective for specific conditions and populations so the lessons can be shared on a broader scale. For example, if you have a group of people living with both heart disease and diabetes and you add exercise to the treatment plan and this approach leads to optimal outcomes, this finding can inform clinicians to recommend a similar approach for other patients who meet the diagnostic criteria of co-existing diabetes and heart disease. By scaling the approach, it can have a broad impact on the greatest number of people.
Supporting Patient-Centered Care
Health outcomes research can also help to empower patients. For instance, cost and outcome data gathered on different clinicians, facilities, and interventions can help consumers to make educated decisions on who they want to perform their care, and in what setting, and which procedures they want to undergo. As patient satisfaction is becoming an increasingly important element in reimbursement structures, so is capturing the patient experience adequately and offering information patients can use to partner with medical providers to become more involved in their own care decisions.
Moving Forward
Many health systems today are effectively incorporating evidence-based findings, including health outcomes research, to direct their best practices and improve coordination among different providers, departments, and locations. Health outcomes research will continue to provide a valuable methodology for clinicians to streamline their efforts and ensure that they—and their health systems—are meeting regulatory requirements and the demand for high value care.
Harvard T.H. Chan School of Public Health offers Measurement, Design, and Analysis Methods for Health Outcomes Research , an introductory course in health outcomes research. To learn more about this opportunity, click here .
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A framework for implementing patient-reported outcomes in clinical care: the PROTEUS-practice guide
- Norah L. Crossnohere ORCID: orcid.org/0000-0002-2811-1330 1 ,
- Nicola Anderson 2 ,
- Judith Baumhauer ORCID: orcid.org/0000-0003-2142-7778 3 ,
- Melanie Calvert ORCID: orcid.org/0000-0002-1856-837X 2 ,
- Rebecca Esparza 4 ,
- Sandi Gulbransen 5 ,
- Lotte Haverman 6 ,
- Yuchen Li 7 ,
- Carolyn Petersen ORCID: orcid.org/0000-0002-2136-1273 8 ,
- Ameeta Retzer ORCID: orcid.org/0000-0002-4156-8386 2 ,
- Christopher Sidey-Gibbons 9 ,
- Angela M. Stover 10 ,
- Elissa Thorner 11 ,
- Garrett Ursin 12 ,
- Galina Velikova 13 ,
- Elliott Sparkman Walker 14 ,
- Michael Brundage 15 &
- Claire Snyder 16
Nature Medicine ( 2024 ) Cite this article
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- Health services
- Quality of life
Patient-reported outcomes (PROs) are a powerful tool for clinical practice that directly measure patient experiences, enabling providers to tailor care to patients’ needs and priorities. PROs are patients’ own reports of how they feel, function, and live their lives 1 , collected using standardized, validated questionnaires. Compelling evidence demonstrates the value of using PROs in clinical practice, whereby a healthcare provider and a patient together use the patient’s PROs to inform care. Implementing PROs as part of routine care can facilitate patient–provider communication, help to identify and address healthcare problems, increase efficiency, and improve patient outcomes, such as symptom burden, functioning, quality of life, and even survival 2 . Measurement of PROs is an integral part of innovative payment models, including the Centers for Medicare and Medicaid Services ‘Enhancing Oncology Model’ and ‘Comprehensive Primary Care Plus’ (CPC+) model 3 , and contributes to quality metrics such as Newsweek ’s 2023 ranking of the world’s best specialized hospitals 4 .
Despite being enthusiastic to use PROs to inform care, many healthcare systems lack understanding of the considerations involved in designing, implementing, and managing PRO systems. Furthermore, there is no one-size-fits-all approach to integrating PROs into clinical practice, and PRO systems can (and should) vary according to factors such as healthcare setting, PRO system goals, and patient characteristics. Established resources provide data-driven and consensus-based guidance on specific aspects of using PROs in clinical practice. However, a unified framework with updated and comprehensive guidance is needed to support the use of PROs in clinical practice.
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Acknowledgements
We thank members of the PROTEUS Consortium and the original developers of the foundational resources synthesized in the PROTEUS-Practice Guide for their contributions.
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Division of General Internal Medicine, The Ohio State University College of Medicine, Columbus, OH, USA
Norah L. Crossnohere
Centre for Patient Reported Outcomes Research, NIHR Birmingham Biomedical Research Centre and NIHR Applied Research Collaboration West Midlands, Institute of Applied Health Research, University of Birmingham, Birmingham, UK
Nicola Anderson, Melanie Calvert & Ameeta Retzer
Department of Orthopedics and Physical Performance, University of Rochester Medical Center, Rochester, NY, USA
Judith Baumhauer
Patient Advocate, National Coalition for Cancer Survivorship, Silver Spring, MD, USA
Rebecca Esparza
University of Utah Health, Salt Lake City, UT, USA
Sandi Gulbransen
Psychosocial Department, Emma Children’s Hospital Academic Medical Center, Amsterdam, The Netherlands
Lotte Haverman
Princess Margaret Cancer Center, Toronto, Ontario, Canada
Department of Artificial Intelligence and Informatics, Mayo Clinic, Rochester, MN, USA
Carolyn Petersen
MD Anderson Center for INSPiRED Cancer Care, University of Texas, Houston, TX, USA
Christopher Sidey-Gibbons
University of North Carolina, Department of Health Policy and Management, and Lineberger Comprehensive Cancer Center, Chapel Hill, NC, USA
Angela M. Stover
Patient Advocate, The PROTEUS Consortium, Baltimore, MD, USA
Elissa Thorner
Epic Systems, Verona, WI, USA
Garrett Ursin
University of Leeds, Leeds, UK
Galina Velikova
Strategic Communications & Planning, Philadelphia, PA, USA
Elliott Sparkman Walker
Queen’s University Cancer Research Institute, Cancer Care and Epidemiology, Kingston, ON, Canada
Michael Brundage
Johns Hopkins Schools of Medicine and Public Health, Baltimore, MD, USA
Claire Snyder
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Correspondence to Norah L. Crossnohere .
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Competing interests.
The PROTEUS Consortium is funded by unrestricted support from Pfizer Inc., who had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication. M.C. receives funding from the National Institute for Health and Care Research (NIHR), UKRI, NIHR Biomedical Research Centre, NIHR Applied Research Collaboration West Midlands, NIHR Birmingham–Oxford Blood and Transplant Research Unit in Precision Transplant and Cellular Therapeutics, GSK and Merck, and has received personal fees from Aparito, CIS Oncology, Halfloop, Merck, Pfizer, Vertex and Gilead outside the submitted work. A.M.S. has, in the past two years, received related funding from Pfizer Global and unrelated funding from UroGen Pharma Ltd, Sivan Innovation, Hematology/Oncology Pharmacy Foundation, Cancer and Aging Research Group (CARG) Infrastructure grants (R21AG059206 and R33AG059206), and the American Society of Clinical Oncology, and was a paid consultant with Navigating Cancer in 2021. C.S. has consulting funding from Shionogi. N.C. has unrelated consulting support from Boehringer Ingelheim.
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Crossnohere, N.L., Anderson, N., Baumhauer, J. et al. A framework for implementing patient-reported outcomes in clinical care: the PROTEUS-practice guide. Nat Med (2024). https://doi.org/10.1038/s41591-024-02909-8
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Published : 16 April 2024
DOI : https://doi.org/10.1038/s41591-024-02909-8
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Typical examples of outcomes are cure, clinical worsening, and mortality. The primary outcome is the variable that is the most relevant to answer the research question. Ideally, it should be patient-centered (i.e., an outcome that matters to patients, such as quality of life and survival). Secondary outcomes are additional outcomes monitored to ...
Abstract. An outcome is a consequence or result thus an outcome measure in research is a standardised way of measuring the consequences or results of research. Outcome measures are widely used in clinical practice, service evaluation, and research, however their selection and use may be complicated by the dearth of information available.
Outcomes research is a branch of public health research which studies the end results of the structure and processes of the health care system on the health and well-being of patients and populations.According to one medical outcomes and guidelines source book - 1996, Outcomes research [full citation needed] includes health services research that focuses on identifying variations in medical ...
In research evaluation, outcomes can be disaggregated into academic outcomes, which refers to influences and changes within the academic realm, and societal outcomes, which refers to changes ...
Outcomes research has become a fashion, meaning all things to all people, and runs the risk of becoming meaningless. This article attempts to reduce the confusion by clarifying what outcomes research is and delineating its several levels, along with the methods, tools, and examples appropriate to each level. The contributions of outcomes ...
Abstract. Outcomes research is a broad umbrella term without a consistent definition. However it tends to describe research that is concerned with the effectiveness of public-health interventions and health services; that is, the outcomes of these services. Attention is frequently focused on the affected individual − with measures such as quality of life and preferences − but outcomes ...
Outcomes research (OR), which is a growing field that combines principles of epidemiology, clinical research, health economics, quality of life assessment, and health policy, is critical in obtaining this evidence.1 Whereas clinical trials aim to generate knowledge on safety and efficacy, OR studies the benefits, risks, and costs that guide ...
Abstract. Outcomes research is a broad umbrella term without. a consistent definition. However it tends to describe. research that is concerned with the effectiveness of. public-health ...
Definition. Outcomes research evaluates the quality and effectiveness of health care by measuring parameters such as mortality or quality of life. For example, it can provide evidence on the ...
While these two definitions elegantly capture the essence of what outcomes research is, there are still some common misunderstandings and misconceptions about this field. ... Rather, it is defined by its objective or the very nature of the question it seeks to answer. Thus, outcomes research can utilize methodology of observational studies ...
Outcomes-Based Research 469 A randomized clinical trial is a form of a cohort study in which the investigators manipulate or control the exposure variables. They are often considered the "gold standard" for evaluating the efficacy or safety of an intervention. How- ever, for outcomes research, randomized clinical trials have many limitations.
Health outcomes research is a methodology used to identify and measure the link between treatments or interventions delivered and the actual outcomes achieved. Put simply, health outcomes studies help determine what works and what doesn't in health care. Unlike clinical trials or other highly regulated scientific studies that consider only ...
research outcomes with others is a beneficial method to . ensure that the outcomes of a research study are not beyond . ... definitions when thinking about outcomes and what th ey .
Outcomes research is a study that maps the effects of a certain medical procedure, medication or lifestyle, considering both short-term and long-term time spans. Medical professionals perform outcomes research in order to ensure that their patients receive the best care possible. If you work in the healthcare field or have an interest in ...
Patient-reported outcomes (PROs) are a powerful tool for clinical practice that directly measure patient experiences, enabling providers to tailor care to patients' needs and priorities.
Research outcome. definition. Open Split View. Cite. Research outcome means the results and conclusions arrived at as the result of particular research activities and includes products, processes, experimental methods and supporting data. Sample 1 Sample 2. Based on 2 documents. Remove Advertising.