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ICER Hemophilia Gene Therapy Reviews Show Clear ‘Net Health Benefit’ But Caution On Patient, Payer Fronts Given Upfront Costs

 
• By Ramsey Baghdadi

Restrictive interpretations of FDA labels for BioMarin and CSL Behring products by payers could signal slow launch of gene therapies seeking to supplant existing treatments.

hemophilia gene therapy
• Source: Shutterstock

The Institute for Clinical & Economic Review advisory panel review of gene therapies from BioMarin Pharmaceutical Inc. and CSL Behring for hemophilia demonstrate that the therapies may face a cautious patient community and restrictive interpretations by payers of the FDA label.

Though there was clear excitement for the potential of hemophilia gene therapies to replace existing treatments, such as factor prophylaxis therapy or novel antibodies like Genentech, Inc

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