Comparative Effectiveness Research and Patient‐Centered Outcomes Research
Where We Stand:
The Academy of Managed Care Pharmacy (AMCP) supports scientifically-sound research that compares the effectiveness and value of medications and treatments, as well as research aimed at tailoring treatment options to individual patients’ needs. Prescribers and patients value this information to evaluate treatment options and select treatments most likely to achieve a desired therapeutic outcome. Health care decision-makers use this information when designing benefits to ensure that safe and effective medications with the best value are provided for all stages of treatment. Such research encourages optimal medication use while also promoting the prudent management of financial resources within the health care system. AMCP believes that the following principles apply to both comparative effectiveness research (CER)1 and patient‐centered outcomes research (PCOR)2 in the context of managed care: the appropriate role of the federal government and other entities (both public and private) in coordinating, funding, and disseminating the results of such research; the characteristics of research design; and the use of research results by patients, providers, and health care decision-makers.
The Role of the Federal Government and Other Entities (Both Public and Private) in Coordinating, Funding, and Disseminating Research
AMCP has supported efforts by Congress in the past that have advanced the role of the federal government in coordinating, funding, and disseminating the results of such research. AMCP was also supportive of the Patient‐Centered Outcomes Research Institute (PCORI), which was established by the Patient Protection and Affordable Care Act (P.L. 111‐148). The Institute, which operates as a non‐governmental, public‐private partnership and receives dedicated, continuous funding from both the Medicare trust fund as well as fees paid by health insurers, provides needed leadership in the further development of both comparative effectiveness and patient‐centered outcomes research. AMCP is also supportive of research conducted by other entities, such as academic institutions, managed care organizations, other stakeholder partnerships, and research organizations, such as the Institute for Clinical and Economic Review (ICER). In instances of privately funded research, sponsors of the studies and any potential conflicts of interest should be clearly disclosed.
Characteristics of Research Design
Perhaps most importantly, it is essential that both CER and PCOR adhere to optimal research design and transparency standards. Randomized, controlled trials or their meta-analyses are preferred over observational studies when practicable. Additionally, research designers should be free to examine the relative values of the treatments studied, including direct and indirect costs, in addition to patient outcomes. In instances where several treatment options lead to similar outcomes of safety and effectiveness, other factors such as cost, convenience, or patient preference serve an important role in informing conclusions regarding the relative value of medications and treatments.
The Use of Research Results by Providers, Patients, and Health Care Decision-Makers
All research funded by the federal government should rightfully become part of the public domain and should be available to any interested party for use in making personal or patient care decisions. Ideally, sponsors of privately funded research would make their findings publicly available as well, regardless of results. To best help patients participate in their own health care decision-making in collaboration with providers, research results should also be presented in such a manner that they are understandable to a lay person, with a clear explanation of realistic outcome expectations, as well as any side effects that could be associated with the treatments studied.
AMCP does not support any government mandate that would require coverage of medications and treatments based solely on the results of any CER or PCOR study. For years, health care decision-makers have been using CER and similar research as one of several variables when evaluating treatment options for coverage. Health care decision-makers serve diverse patient populations and therefore must retain flexibility to use the research results in the manner they deem most appropriate for the benefits designed for the populations they serve.
The results of CER and PCOR have the potential to provide invaluable insight to patients and providers searching for optimal treatment options, as well as health care decision-makers as they design affordable benefits to best suit their patient population. Such research must be well‐designed and should include a comprehensive view of all treatments studied, including relative value and patient satisfaction. However, it is important for health care decision-makers to retain the flexibility to use such research as one of several variables in the evaluation and determination of coverage for medications and treatments.
AMCP Where We Stand series: https://www.amcp.org/policy-advocacy/policy-advocacy-focus-areas/where-we-stand-position-statements
AMCP CER Glossary: Comparative Effectiveness Research - Glossary of Terms | AMCP.org
Revised by the AMCP Board of Directors, April 2021
Revised by the AMCP Board of Directors, February 2012
Revised by the AMCP Board of Directors, April 2008
Approved by the AMCP Board of Directors, March 2004