Comparative effectiveness research

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Comparative effectiveness research (CER) is the direct comparison of existing health care interventions to determine which work best for which patients and which pose the greatest benefits and harms. The core question of comparative effectiveness research is which treatment works best, for whom, and under what circumstances. [1] Engaging various stakeholders in this process, while difficult, makes research more applicable through providing information that improves patient decision making. [2]

Contents

The Institute of Medicine committee has defined CER as "the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The purpose of CER is to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels." [3]

Comparative effectiveness research adopts many of the same approaches and methodologies as cost-effectiveness analysis, including the use of incremental cost-effectiveness ratios (ICERs) and quality-adjusted life years (QALYs). An important component of CER is the concept of pragmatic randomised controlled trials. [4] These clinical research trials measure the benefit produced by the treatment in routine clinical practice.

In the United States

Researchers at the Dartmouth Institute for Health Policy, in addition to the Congressional Budget Office, have documented a large gap in the quality and outcomes and health services being delivered. Unwarranted variation in medical treatment, cost, and outcomes suggests a substantial area for improvement and savings in our health care system. Statistical findings show that "patients in the highest-spending regions of the country receive 60 percent more health services than those in the lowest-spending regions, yet this additional care is not associated with improved outcomes." [5] New models of shared decision making promise to bring greater emphasis to informed patient choice for "preference-sensitive" care, improving quality, safety, and effectiveness of health care by providing both patients and their health care providers with the evidence to assist in informed decision making. [5]

In 2009, $1.1 billion of President Barack Obama's stimulus package was earmarked for CER. [6] There was initial disagreement regarding whether CER will be used to limit patient health care options, [7] or help lower health care costs. [8] Ultimately, the bill approved by the Senate contained measures to use CER as a means for increasing quality while reducing rising costs. [9] [10]

Several groups have emerged to provide leadership in the area of Comparative Effectiveness Research. The Agency for Healthcare Research and Quality (AHRQ) is a federal agency focused on health care quality. The Institute for Clinical and Economic Review provides independent evaluation of the clinical effectiveness and comparative value of health care interventions, while also overseeing the New England Comparative Effectiveness Public Advisory Council (CEPAC), an independent body of physicians and patient representatives that aids patients, physicians and policymakers in the application and use of comparative effectiveness information to improve the quality and value of healthcare in the region.

The Patient-Centered Outcomes Research Institute (PCORI) was established to conduct comparative effectiveness research but the Patient Protection and Affordable Care Act (PPACA) prohibits it from using cost per QALY ICER thresholding. The PPACA states:

The Patient-Centered Outcomes Research Institute...shall not develop or employ a dollars per quality adjusted life year (or similar measure that discounts the value of a life because of an individual's disability) as a threshold to establish what type of health care is cost effective or recommended.

Comparing key measures utilized in comparative effectiveness research

The study of comparative effectiveness research (CER) is composed of measures useful in determining the value of various treatment options to help patients make more informed decisions in their own care. While each of these measures provides a useful comparison of one treatment option versus another, they require different inputs into their respective calculations, thus the potential for producing conflicting results. Additionally, some health conditions, such as for prostate cancer treatment, lack patient-centered outcomes to inform comparative effectiveness research. [11]

While there remains a widespread lack of understanding on the potential impact of CER in the U.S. and a reluctance to fully adopt the concept as part of our healthcare system, research studies within this area continue to expand across health conditions. [12] [13] [14]

Related Research Articles

Evidence-based medicine (EBM) is "the conscientious, explicit and judicious use of current best evidence in making decisions about the care of individual patients". The aim of EBM is to integrate the experience of the clinician, the values of the patient, and the best available scientific information to guide decision-making about clinical management. The term was originally used to describe an approach to teaching the practice of medicine and improving decisions by individual physicians about individual patients.

The incremental cost-effectiveness ratio (ICER) is a statistic used in cost-effectiveness analysis to summarise the cost-effectiveness of a health care intervention. It is defined by the difference in cost between two possible interventions, divided by the difference in their effect. It represents the average incremental cost associated with 1 additional unit of the measure of effect. The ICER can be estimated as:

<span class="mw-page-title-main">Quality-adjusted life year</span> Measure of disease burden

The quality-adjusted life year (QALY) is a generic measure of disease burden, including both the quality and the quantity of life lived. It is used in economic evaluation to assess the value of medical interventions. One QALY equates to one year in perfect health. QALY scores range from 1 to 0 (dead). QALYs can be used to inform health insurance coverage determinations, treatment decisions, to evaluate programs, and to set priorities for future programs.

A clinical decision support system (CDSS) is a health information technology that provides clinicians, staff, patients, and other individuals with knowledge and person-specific information to help health and health care. CDSS encompasses a variety of tools to enhance decision-making in the clinical workflow. These tools include computerized alerts and reminders to care providers and patients, clinical guidelines, condition-specific order sets, focused patient data reports and summaries, documentation templates, diagnostic support, and contextually relevant reference information, among other tools. CDSSs constitute a major topic in artificial intelligence in medicine.

Patient safety is a discipline that emphasizes safety in health care through the prevention, reduction, reporting and analysis of error and other types of unnecessary harm that often lead to adverse patient events. The frequency and magnitude of avoidable adverse events, often known as patient safety incidents, experienced by patients was not well known until the 1990s, when multiple countries reported significant numbers of patients harmed and killed by medical errors. Recognizing that healthcare errors impact 1 in every 10 patients around the world, the World Health Organization (WHO) calls patient safety an endemic concern. Indeed, patient safety has emerged as a distinct healthcare discipline supported by an immature yet developing scientific framework. There is a significant transdisciplinary body of theoretical and research literature that informs the science of patient safety with mobile health apps being a growing area of research.

A patient-reported outcome (PRO) is a health outcome directly reported by the patient who experienced it. It stands in contrast to an outcome reported by someone else, such as a physician-reported outcome, a nurse-reported outcome, and so on. PRO methods, such as questionnaires, are used in clinical trials or other clinical settings, to help better understand a treatment's efficacy or effectiveness. The use of digitized PROs, or electronic patient-reported outcomes (ePROs), is on the rise in today's health research setting.

The Drug Effectiveness Review Project (DERP) is a self-governed collaboration of state Medicaid and public pharmacy programs that commission high-quality evidence-based research products to assist policymakers and other decision-makers grappling with difficult drug coverage decisions. Housed at the Center for Evidence-based Policy at Oregon Health & Science University in Portland, Oregon, DERP produces concise, comparative, evidence-based research products that evaluate the efficacy, effectiveness, and safety of drugs in many widely used drug classes.

Health services research (HSR) became a burgeoning field in North America in the 1960s, when scientific information and policy deliberation began to coalesce. Sometimes also referred to as health systems research or health policy and systems research (HPSR), HSR is a multidisciplinary scientific field that examines how people get access to health care practitioners and health care services, how much care costs, and what happens to patients as a result of this care. HSR utilizes all qualitative and quantitative methods across the board to ask questions of the healthcare system. It focuses on performance, quality, effectiveness and efficiency of health care services as they relate to health problems of individuals and populations, as well as health care systems and addresses wide-ranging topics of structure, processes, and organization of health care services; their use and people's access to services; efficiency and effectiveness of health care services; the quality of healthcare services and its relationship to health status, and; the uses of medical knowledge.

In economics, supplier induced demand (SID) may occur when asymmetry of information exists between supplier and consumer. The supplier can use superior information to encourage an individual to demand a greater quantity of the good or service they supply than the Pareto efficient level, should asymmetric information not exist. The result of this is a welfare loss.

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 includes health services research that focuses on identifying variations in medical procedures and associated health outcomes. Though listed as a synonym for the National Library of Medicine MeSH term "Outcome Assessment ", outcomes research may refer to both health services research and healthcare outcomes assessment, which aims at Health technology assessment, decision making, and policy analysis through systematic evaluation of quality of care, access, and effectiveness.

<span class="mw-page-title-main">Quality of life (healthcare)</span> Notion in healthcare

In general, quality of life is the perceived quality of an individual's daily life, that is, an assessment of their well-being or lack thereof. This includes all emotional, social and physical aspects of the individual's life. In health care, health-related quality of life (HRQoL) is an assessment of how the individual's well-being may be affected over time by a disease, disability or disorder.

Patient participation is a trend that arose in answer to medical paternalism. Informed consent is a process where patients make decisions informed by the advice of medical professionals.

David M. Eddy is an American physician, mathematician, and healthcare analyst who has done seminal work in mathematical modeling of diseases, clinical practice guidelines, and evidence-based medicine. Four highlights of his career have been summarized by the Institute of Medicine of the National Academy of Sciences: "more than 25 years ago, Eddy wrote the seminal paper on the role of guidelines in medical decision-making, the first Markov model applied to clinical problems, and the original criteria for coverage decisions; he was the first to use and publish the term 'evidence-based'."

<span class="mw-page-title-main">Patient-Centered Outcomes Research Institute</span> Health research organization

The Patient-Centered Outcomes Research Institute (PCORI) is a United States-based non-profit institute created through the 2010 Patient Protection and Affordable Care Act. It is a government-sponsored organization charged with funding Comparative Effectiveness Research (CER) that assists consumers, clinicians, purchasers, and policymakers to make informed decisions intended to improve health care at both the individual and population levels, according to the Institute of Medicine. Medicare considers the Institute's research in determining what sorts of therapies it will cover, although the institute's authorizing legislation set certain limits on uses of the research by federal health agencies.

Unnecessary health care is health care provided with a higher volume or cost than is appropriate. In the United States, where health care costs are the highest as a percentage of GDP, overuse was the predominant factor in its expense, accounting for about a third of its health care spending in 2012.

Health care quality is a level of value provided by any health care resource, as determined by some measurement. As with quality in other fields, it is an assessment of whether something is good enough and whether it is suitable for its purpose. The goal of health care is to provide medical resources of high quality to all who need them; that is, to ensure good quality of life, cure illnesses when possible, to extend life expectancy, and so on. Researchers use a variety of quality measures to attempt to determine health care quality, including counts of a therapy's reduction or lessening of diseases identified by medical diagnosis, a decrease in the number of risk factors which people have following preventive care, or a survey of health indicators in a population who are accessing certain kinds of care.

The Comparative Effectiveness Research Translation Network (CERTAIN) is a learning healthcare system in Washington State focused on patient-centered outcomes research (PCOR) and comparative effectiveness research (CER), leveraging existing healthcare data for research and healthcare improvement, incorporating patient and other healthcare stakeholder voices into research, and facilitating dissemination and implementation of research evidence into clinical practice.

Learning health systems (LHS) are health and healthcare systems in which knowledge generation processes are embedded in daily practice to improve individual and population health. At its most fundamental level, a learning health system applies a conceptual approach wherein science, informatics, incentives, and culture are aligned to support continuous improvement, innovation, and equity, and seamlessly embed knowledge and best practices into care delivery

Harlan M. Krumholz, MD, SM is an American cardiologist, leading research scientist, and the Harold H. Hines, Jr. Professor of Medicine at Yale School of Medicine, where he has been on faculty since 1992. A pioneer in the development of the field of outcomes research, his groundbreaking contributions to science have directly led to improvements in healthcare outcomes for patients and populations. He is an international expert in the science to evaluate and improve the quality and efficiency of care, reduce disparities, improve integrity in medical research, promote better health policies and regulations, and promote patient-centeredness in research and clinical care. He is the founder and director of the Yale New Haven Hospital Center for Outcomes Research and Evaluation.

References

  1. ↑ Greenfield S, Rich E (January 2012). "Welcome to the Journal of Comparative Effectiveness Research". Journal of Comparative Effectiveness Research . 1 (1): 1–3. doi:10.2217/cer.11.13. PMID   24237290.
  2. ↑ Hong YD, Goto D, Mullins CD (May 2017). "Querying stakeholders to inform comparative effectiveness research". Journal of Comparative Effectiveness Research. 6 (3): 265–273. doi:10.2217/cer-2016-0082. PMC   5680160 . PMID   28485177.
  3. ↑ Initial National Priorities for Comparative Effectiveness Research. Washington, D.C.: National Academies Press. 2009-10-14. doi:10.17226/12648. ISBN   978-0-309-13836-9.
  4. ↑ Roland M, Torgerson DJ (January 1998). "What are pragmatic trials?". BMJ. 316 (7127): 285. doi:10.1136/bmj.316.7127.285. PMC   2665488 . PMID   9472515.
  5. 1 2 Institute of Medicine (2009). Initial National Priorities for Comparative Effectiveness Research. National Academies Press. doi:10.17226/12648. ISBN   978-0-309-13836-9.
  6. ↑ Pear R (February 15, 2009). "U.S. to Compare Medical Treatments". The New York Times. Retrieved July 16, 2016.
  7. ↑ Mundy A (February 10, 2009). "Drug Makers Fight Stimulus Provision". The Wall Street Journal. Retrieved July 16, 2016.
  8. ↑ Garber AM, Tunis SR (May 2009). "Does comparative-effectiveness research threaten personalized medicine?". The New England Journal of Medicine. 360 (19): 1925–7. doi:10.1056/NEJMp0901355. PMID   19420360. S2CID   16806907.
  9. ↑ Kotok A (March 22, 2010). "Comparative Effectiveness Research Boosted in Health Care Bill". Science Magazine. Retrieved July 16, 2016.
  10. ↑ Committee on Comparative Effectiveness Research Prioritization; Board on Health Care Services; Institute of Medicine (2009). Initial National Priorities for Comparative Effectiveness Research. The National Assemblies Press. doi:10.17226/12648. ISBN   978-0-309-13836-9.
  11. ↑ Jayadevappa R, Chhatre S, Wong YN, Wittink MN, Cook R, Morales KH, et al. (May 2017). "Comparative effectiveness of prostate cancer treatments for patient-centered outcomes: A systematic review and meta-analysis (PRISMA Compliant)". Medicine. 96 (18): e6790. doi:10.1097/MD.0000000000006790. PMC   5419922 . PMID   28471976.
  12. ↑ "PCORnet: the National Patient-Centered Clinical Research Network". PCORnet. Archived from the original on 2011-09-22. Retrieved 2019-09-05.
  13. ↑ Mushlin AI, Ghomrawi H (January 2010). "Health care reform and the need for comparative-effectiveness research". The New England Journal of Medicine. 362 (3): e6. doi:10.1056/nejmp0912651. PMID   20054035.
  14. ↑ Inge, Thomas H.; Coley, R. Yates; Bazzano, Lydia A.; Xanthakos, Stavra A.; McTigue, Kathleen; Arterburn, David; Williams, Neely; Wellman, Rob; Coleman, Karen J.; Courcoulas, Anita; Desai, Nirav K. (2018). "Comparative effectiveness of bariatric procedures among adolescents: the PCORnet bariatric study". Surgery for Obesity and Related Diseases. 14 (9): 1374–1386. doi:10.1016/j.soard.2018.04.002. PMC   6165694 . PMID   29793877.
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