BOSTON – April 10, 2018 – The Institute for Clinical and Economic Review (ICER), in collaboration with the Office of Health Economics (OHE), has released two new white papers to provide guidance to payers and manufacturers on the development and use of real world evidence for drug coverage and formulary decisions. The papers were generated as part of the annual ICER Policy Summit, where life science and payer organizations meet to develop collaborative approaches to addressing key policy issues related to the generation and application of evidence to improve value in the US health care system.

“Both manufacturers and payers share common goals in applying real world evidence to decision-making,” noted Steve D. Pearson, MD, MSc, President of ICER. “However, discussions at our Policy Summit highlighted the need for a common framework to help all parties gain a mutual understanding of how to tailor the entire evidence development process to achieve the best fit with key contextual factors that determine the level of evidentiary certainty decision-makers will require. There is no doubt that real world evidence will play a larger role in coverage and formulary decisions in the future, and we hope these two papers will support payers and the life science industry in their efforts to find common ground in how to maximize the positive role this evidence can play in guiding decisions that will improve patient outcomes and overall value for the health system.”

The first paper, entitled Real World Evidence for Coverage Decisions: Opportunities and Challenges, was developed as the background paper for the December 2017 Policy Summit of ICER’s membership program, where leaders from 22 payer and life science organizations convened to share their perspectives on challenges and opportunities for this type of evidence. This paper highlights the challenges associated with wider use of real world evidence, including increased potential for bias, the risks of incomplete data, and the lack of universally accepted methodological standards.

A companion paper, Understanding the Context, Selecting the Standards: A Framework to Guide the Optimal Development and Use of Real World Evidence for Coverage and Formulary Decisions, was written to address the key issue that emerged from discussion during the Summit: the need for a common approach to tailoring the development of real world evidence to meet the perceived evidentiary needs of the decision-maker. This paper sets out a conceptual framework to guide the generation and adoption of real world evidence in light of a shared understanding of key contextual issues, such as whether the evidence is intended to support an argument of superiority of a drug versus similar comparators, or whether the evidence will be used to argue for increased drug spending in hopes of lowering overall health system costs.

The discussion white paper and companion white paper are available on ICER’s website.

Both papers are a product of the ICER Policy Summit, a key feature of the ICER membership program, and were developed in collaboration with leading researchers at the OHE, a not-for-profit organization based in the UK that conducts research on the economics of innovation and the life sciences industry, the organization and financing of health care, and the role for outcomes research and health technology assessment.

The ICER membership program brings together a small, influential group of evidence leaders from insurers, pharmacy benefit management firms, health technology assessment groups, and life science companies to address key controversies in evidence methods and policy. Working together in a balanced, non-adversarial environment, ICER members gain the skills and insights in evidence policy necessary to strengthen their competitive position in the marketplace.

No assertion, judgment, or recommendation included in the white paper should be viewed as representing the opinion of any participant in the ICER membership program or their company. ICER alone is ultimately responsible for the final content.

Representatives from the following organizations attended the Policy Summit:

Payer Organizations: Aetna, America’s Health Insurance Plans (AHIP), Anthem Blue Cross Blue Shield, Blue Shield of California, CVS Caremark, Express Scripts, Harvard Pilgrim Health Care, Health Care Service Corporation (HCSC), Kaiser Permanente, MedSavvy, Premera Blue Cross, Prime Therapeutics, United Healthcare

Life Sciences Organizations: Alnylam, AstraZeneca, Editas, Genentech, GlaxoSmithKline, Janssen/Johnson & Johnson, Merck, National Pharmaceutical Council, Sanofi


About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.