Posted by Michael Wonder on 07 Feb 2017
Assessing the value of drugs for rare conditions: Institute for Clinical and Economic Review to host orphan drug assessment and pricing summit
7 February 2017 - 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.
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 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.
