-Paper presents recommendations for improving evidence generation and assessment methods for gene therapies; also analyzes the options for innovative payment mechanisms that can balance clinical value with affordability-

Boston, Mass., March 3, 2017 – The Institute for Clinical and Economic Review (ICER) has released a new white paper: Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value. The paper explores the scientific frontier being opened up by emerging gene therapies, with the potential for curing or substantially improving the lives of individuals who have had limited treatment options in the past. The paper also addresses the challenges associated with generating the evidence needed to make robust assessments of safety, effectiveness, and value of these treatments. In particular, the paper explores the need to consider new mechanisms for pricing, payment, and delivery, including an analysis of different options for amortizing over multiple years payment for one-time curative treatments.

The white paper was developed following a December 2016 meeting of ICER’s membership program, during which leaders from 20 payer and manufacturer organizations convened to share their perspectives on the future of gene therapy.

“Gene therapy offers tremendous promise in changing the way we treat and cure illnesses that affect millions of people,” noted Steven D. Pearson, MD, MSc, President of ICER. “However, it will take ongoing effort by all stakeholders involved to address the challenges surrounding evidence generation, value assessment, and payment strategies. Our policy summit and white paper have sought to highlight key challenges and opportunities, and it is our hope that this paper can be a starting point in developing the path forward. We will continue to convene important industry leaders to build on this collaborative dialogue.”

You can find the full white paper here.

The white paper is a product of the ICER membership program and was developed in collaboration with researchers at the Office of Health Economics in England. The 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.

The December 2016 meeting, where the ideas in the paper were developed, included leaders from 20 leading insurer and pharmaceutical companies participating in this collaborative effort. Importantly, no assertion, judgment, or recommendation included in the white paper should be viewed as representing the opinion of any participant or their company. ICER alone is ultimately responsible for the final content.

The meeting had representatives from the following organizations:

Payer Organizations: Aetna, Association of Health Insurance Plans, Anthem, Blue Shield of California, CVS Caremark, Express Scripts, Harvard Pilgrim Healthcare, Health Care Service Corporation (HCSC), OmedaRx, Premera, Prime Therapeutics, United Healthcare

Life Sciences Organizations: bluebirdbio, Genentech, GlaxoSmithKline, Johnson & Johnson, Merck, National Pharmaceutical Council, Sanofi, Spark Therapeutics

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.