BIO Principles on Comparative Effectiveness

Comparative effectiveness research should be conducted through an open and transparent process involving all stakeholders, starting from the research planning stage. Different comparative effectiveness studies evaluating the same item or service can produce widely disparate results, depending on the methods and assumptions used in the analysis. To enhance the credibility and usefulness of any comparative effectiveness study, stakeholders should be afforded the opportunity to provide meaningful input into all steps along the study process, including the identification of priority areas to research, study design and research methods, and dissemination of results. Any approach to comparative effectiveness study design and research should encourage the conduct of independent studies that meet certain standards and that can serve as a basis for consideration by the Food and Drug Administration, in addition to the Centers for Medicare and Medicaid Services and other payers. Careful consideration should be given and rigorous standards applied to the research method selected, such as randomized controlled trials, observational studies, or data synthesis. In their final form, comparative effectiveness research studies should include a concise description of the research question, transparency as to inclusion or exclusion of evidence or clinical information, transparent analytical methods, discussion of limitations in the quality of the evidence and overall conclusions and recommendations for areas to further research or more fully develop evidence.

Comparative effectiveness studies should capture all relevant aspects of diseases and their treatments using the highest possible standards of evidence. Comparative effectiveness analyses often ignore many important aspects of treatment interventions that affect patients, or may not account for spectra of disease severities. For example, many of the unique benefits provided by drugs and biologics, such as increased safety and improved patient quality of life, are often not captured in comparative effectiveness analyses, or when evaluated do not reflect the use of well-validated methodologies. Increased worker productivity and savings to other parts of the health care system are also important benefits that may not be reflected in studies conducted with a narrow perspective. Risk-adjusted comparisons of treatment interventions are also important to attempt to understand differences among patient subgroups in treatment efficacy, safety, tolerability, quality of life, and health economic outcomes.

BIO’s members remain committed to advancing innovation and developing new therapies to improve patient health. BIO welcomes the opportunity to continue to work with policymakers and other stakeholders on ways to improve value in the health care system by ensuring that the most appropriate care is delivered to each patient.