Assessing the Value of Drugs for Rare Conditions: Institute for Clinical and Economic Review to Host Orphan Drug Assessment and Pricing Summit

— Discussion will be anchored by an ICER Briefing Paper evaluating the evidence on the comparative clinical effectiveness of Spinraza (nusinersen) for spinal muscular atrophy, and examining the options for assessing value and establishing value-based prices for orphan drugs —

Boston, Mass., February 7, 2017 – The Institute for Clinical and Economic Review (ICER) will host a multi-stakeholder policy summit in Washington, DC this May to discuss methods for assessing the value of new drugs for rare conditions, including how these methods can be applied to recommend fair prices that reflect the value of orphan drugs to patients and the health system, allowing for broader insurance coverage for innovative new treatments. To facilitate the discussion, ICER will produce a Briefing Paper on Spinraza™ (nusinersen, Biogen), a new drug for spinal muscular atrophy (SMA) recently approved by the FDA, and will also examine the policy context surrounding other recent orphan drug approvals, such as Exondys-51™ (eteplirsen, Sarepta Therapeutics) for Duchenne muscular dystrophy (DMD).

The Briefing Paper will review the existing evidence on the clinical effectiveness of Spinraza. It will seek to understand the costs of development for Spinraza as a case study, and will also compare methods used by academic researchers and others to evaluate the long-term value of drugs for rare conditions, including conditions affecting children. The Briefing Paper will not include an ICER value-based price benchmark for Spinraza or other drugs, nor will the meeting involve a vote on the value of these drugs. Instead, the meeting will seek broad input from all 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.

Controversy over drug pricing and costs is an important national issue. The high prices for orphan drugs, exemplified by a first-year launch price of $625,000 to $750,000 for Spinraza, 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 dialog about how its methods should reflect and serve the ultimate aim of providing sustainable access to high-value care for all patients.

Representatives from the rare disease patient community, life sciences, clinical, and payer communities have been invited to join a Working Group to advise ICER on the development of the Briefing Paper and the content of the Summit itself. The Orphan Drug Assessment and Pricing Summit is free and open to the public. Registration is now open. Because seating will be limited, the Summit will also be webcast for those unable to attend in Washington, DC. Webcast registration is available here.

Key Dates for the ICER Orphan Drug Assessment and Pricing Summit:
Meeting registration is open today  through May 24, 2017.
  • Briefing Paper Posted: May 10, 2017
  • Public Comments Accepted: May 10 – May 24, 2017
  • Meeting Date and Location: Wednesday, May 31, 2017
    • 8:30am – Registration
    • 4:00pm – Meeting concludes
    • Kaiser Permanente Center for Total Health
      700 Second St. NE
      Washington, DC 20002
  • Final Recommendations and Briefing Paper: June 14, 2017

Find the latest information about the ICER Orphan Drug Assessment and Pricing Summit 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.