Fit for Purpose? New NPC, AcademyHealth Framework Closes Gaps Between Payer Needs and Research Answers

For Immediate Release
Contact: Andrea Hofelich,, 202-827-2078 (NPC)
Kristin Rosengren,, 202-689-9067 (AcademyHealth)

(Washington, DC, September 21, 2015)—Despite multi-million dollar public and private investments to tap into big data and improve the evidence available for health care decision-making, gaps remain between the questions explored by researchers and the issues that health care coverage decision-makers must address. A new paper, published in the September issue of The American Journal of Managed Care, introduces a framework developed by researchers at the National Pharmaceutical Council (NPC) and AcademyHealth that could help close those gaps by harmonizing research with decision-maker priorities.

The push for improved quality and value in health care delivery is driving collaborative efforts to develop more, and better, evidence to inform decision-making. Despite increased availability of research, the “supply” of evidence developed by researchers may not always meet the “demand” for evidence by payers.

This gap stems from payers traditionally favoring the use of evidence from randomized controlled trials over other study designs, such as real-world or observational studies based upon electronic health records, patient registries or administrative claims. A growing demand has emerged for better evidence to demonstrate how treatments work in the real world, among typical patient populations or compared to other treatment alternatives. This type of evidence can be better generated through real-world studies than through more narrowly defined randomized controlled trials.

“This gap between supply and demand is complicated by the tendency of decision-makers to assess evidence based on study design, rather than whether the evidence credibly answers the question that’s being asked,” said Jennifer Graff, PharmD, NPC’s vice president for comparative effectiveness research.

To help align evidence with the needs of payers, authors developed and tested a decision-making framework to understand when real-world evidence is “fit for purpose,” or answers specific payer needs.

Authors then 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.

“What’s clear is that there isn’t one type of study that’s fit for purpose,” Raj Sabharwal, MPH, director, AcademyHealth, pointed out. “Real-world evidence can often provide the data and insights payers need to answer salient questions.” Payer feedback on the framework demonstrated that, in some cases, the existing data is sufficient. In other cases, payers might need different questions answered, such as information on long-term safety from registries or from real-world studies. In other circumstances, payers may need studies with different comparators or populations in order to define coverage polices.

Authors noted that 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. “The need for smart, value-based coverage policies has never been greater, and payers need appropriate evidence to succeed. We hope this framework can contribute to the dialogue between researchers and payers and make the process of generating evidence more efficient for all,” said Dr. Graff.

About the National Pharmaceutical Council
The National Pharmaceutical Council is a health policy research organization dedicated to the advancement of good evidence and science, and to fostering an environment in the United States that supports medical innovation. Founded in 1953 and supported by the nation’s major research-based biopharmaceutical companies, NPC focuses on research development, information dissemination, and education on the critical issues of evidence, innovation and the value of medicines for patients. For more information visit and follow NPC on Twitter @npcnow.

About AcademyHealth
AcademyHealth is a leading national organization serving the fields of health services and policy research and the professionals who produce and use this important work. Together with our members, AcademyHealth offers programs and services that support the development and use of rigorous, relevant and timely evidence to increase the quality, accessibility, and value of health care, to reduce disparities, and to improve health. A trusted broker of information, AcademyHealth brings stakeholders together to address the current and future needs of an evolving health system, inform health policy, and translate evidence into action.