Alnylam Pharmaceuticals recently announced a new Value Based Agreement (VBA) framework for their newly approved RNAi medicine to treat acute hepatic porphyria, a group of rare genetic diseases due to one or more deficiencies in the heme biosynthesis pathway in the liver. The framework is supposed to make it easier for payers to sign agreements to make this very expensive medication (costs are estimated at $400,000-650,000 per year for treatment of attacks) available to their patients. VBAs have been touted in the news this year as a solution to medicine affordability challenges, particularly with new gene therapies. However, we have no data yet on whether they will actually improve access as formulary listing is just the first step in a long path towards actual treatment. Even Harvard Pilgrim, the payer at the forefront of making VBA deals, is uncertain what impact they will have. It would be interesting to hear the perspective of patient advocacy groups such as Patients for Affordable Drugs or the National Organization for Rare Disorders.
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Text Copyright © 2020 Katrina Rogers