— Report contains a technical brief on Spinraza® and will inform discussions at ICER pricing summit on May 31st; summit participants will explore options for assessing value and establishing value-based prices for orphan drugs —

Boston, Mass., May 10, 2017 – The Institute for Clinical and Economic Review (ICER) today posted a briefing paper on Assessing the Effectiveness and Value of Drugs for Rare Conditions. The briefing paper seeks to provide background information to help frame the issues to be discussed during ICER’s Orphan Drug Assessment and Pricing Summit, a multi-stakeholder policy meeting to be held in Washington, DC on May 31st.

Controversy over drug pricing and costs is generating important national discussion. High prices for orphan drugs are among the most difficult examples of the tension between the goals of patient access, affordability, and sustained incentives for innovation. As an organization performing assessments of new drugs and other health services, ICER is committed to fostering a broader public dialogue about how its methods should reflect and serve the ultimate aim of providing sustainable access to high-value care for all patients. As part of this goal, participants at ICER’s pricing summit will discuss methods for assessing the value of new drugs for rare conditions, including how these methods can capture the value of orphan drugs to patients and the health system.  Options will also be examined for how best to translate this information into recommendations for fair prices that can maintain the balance between rewards for innovation and affordability so that patients will be able to benefit fully from the growing pipeline of treatments for rare and ultra-rare conditions.
Serving as the basis for this discussion, ICER’s briefing paper seeks to understand the commercial, regulatory, and reimbursement environments for rare drug development, and compares methods used by academic researchers and international agencies to evaluate the long-term value of drugs for rare conditions. The paper includes a short case study on nusinersen (Spinraza®, Biogen), approved by the FDA earlier this year for treatment of spinal muscular atrophy. During the pricing summit, participants will share perspectives on how the methods used by ICER and other groups can best be tailored to meet the challenge of assessing the value of orphan drugs, and provide an objective foundation for discussions about fair pricing, insurance coverage, and payment mechanisms.


Public Comment Process
ICER will not be accepting formal public comment on the briefing paper; however, following the Orphan Drug Assessment and Pricing Summit ICER will develop a draft proposal outlining specific modifications to its assessment process when applied to treatments for rare conditions, and will post this document with a request for public comment over a 60-day timeframe.
The Orphan Drug Assessment and Pricing Summit is free and open to the public; however, registration is currently at capacity. For those unable to attend in person, a live webcast of the meeting will be available. Webcast registration is available here.
Find the latest information about the ICER Orphan Drug Summit, including a current agenda, on ICER’s website.
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.