Comparative Effectiveness Research and Patient‐Centered Outcomes Research

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 need this information to evaluate and select treatment options 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, including support for 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)3. 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 appropriate; however, researchers should be provided the necessary flexibility to conduct analysis to help patient and providers make informed healthcare decisions, including using available real-world evidence. Additionally, researchers should be free to evaluate 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 be publicly available to any interested party for use in making personal or patient care decisions. Sponsors of privately funded research should make their findings publicly available as well, regardless of results. To promote effective patient-provider collaboration in health care decision-making, it is critical for research results to be presented in a manner understandable to a lay person. Realistic outcome expectations and any side effects associated with the studied treatments should also be disclosed.

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.

Conclusion  
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.   
See also: 

AMCP Legislative and Regulatory Positions 

AMCP CER Glossary: Comparative Effectiveness Research - Glossary of Terms | AMCP.org 

Revised by the AMCP Board of Directors, December 2024

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