Developing Evidence that is Fit for Purpose: A Framework for Payer and Research Dialogue


A study published in the September 2015 issue of The American Journal of Managed Care introduces a framework developed by the National Pharmaceutical Council (NPC) and AcademyHealth that could help close the gaps between the questions explored by researchers and the issues that health care coverage decision-makers must address. 

This gap stems from payers traditionally favoring randomized controlled trials over other methods, such as observational studies and indirect treatment comparisons. In particular, though growing demand has emerged for better evidence to demonstrate how treatments work in the real world, studies have shown that few payers use real-world evidence, including data from electronic health records or observational studies, in coverage or formulary decisions.

The framework is intended to help harmonize the evidence payers desire for coverage and formulary decisions with the evidence received from researchers, and help guide researchers as to what types of evidence needs to be developed in the future.

Authors tested the framework among payers using three clinical scenarios and the associated decisions payers face: 1) determination of coverage for a new hepatitis C therapy (sofosbuvir); 2) confirming coverage for oral anticoagulants (rivaroxaban, dabigatran, apixaban); and 3) limiting use of a multiple sclerosis (MS) therapy (natalizumab) that has known safety concerns. Payers applied the framework to these cases and provided key insights on the evidence needed to inform their decision-making:

  • Payers need more impact, not more evidence: Payers are not necessarily seeking more evidence about treatment efficacy. Their primary need is evidence that illustrates meaningful differences between treatment alternatives and more relevant outcomes for real-world use.
  • Timing matters: Payers need additional evidence most when treatment standards are in flux, for example when a new drug is approved or clinical practice shifts when new treatments emerge.
  • Payers take a broader view of outcomes: Payers are interested in obtaining new evidence that goes beyond efficacy, and includes effectiveness, longer-term safety or the impact of a treatment on subpopulations or impact on the delivery system.
  • Payers need real-world evidence: Payers noted that real-world evidence can provide the insights needed to design population-specific management programs or fine-tune coverage strategies.