The Centers for Medicare and Medicaid Services (CMS) may soon finalize proposed changes to the Medicaid Drug Rebate Program (MDRP) that, if implemented, would have a dramatically negative impact on vulnerable patients and future innovation for cell and gene therapy.
During a recent Congressional briefing hosted by the Institute for Gene Therapies (IGT) and the Alliance for Regenerative Medicine (ARM), a panel of experts including Erik Paulsen (IGT), Amanda Krezepicki (Autoimmune Association), Madelyn Schloss (Gaucher Community Alliance), Kim Phelan (Hemophilia B Coalition), Erica Cischke (ARM), and John Feore (IGT), spoke to the threats patients face from the proposed MDRP changes.
Patients with rare diseases such as Gaucher and hemophilia B are seeing a new wave of possibilities with the development and approval of gene therapies. Schloss discussed how invaluable these therapies can be to the patient communities:
However, innovation and access to gene therapies could be stunted with the implementation of the proposed changes to the MDRP rule. As Phelan and Krezepicki explain, this would be detrimental for patients:
The MDRP rule as is would make the path to treatment for cell and gene therapies more arduous, create additional payment issues – going against the value-based payment arrangements states, manufacturers, patients, and CMS have been advocating for, and unfairly scrutinize cell and gene therapy treatment options.
Congress can help by supporting legislation like the the MVP Act and encouraging CMS to withdraw this rule. While CMS has recently announced the agency will not finalize the proposed best price stacking provision in the proposed rule, several other concerning provisions remain. Patients and their providers need leaders in Congress and the Administration to work together to mitigate barriers faced in trying to access and provide the best possible care.