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