Comparative Data On Inhibitor Side Effects Sought By Hemophilia Patients
FDA hears calls for more post-approval monitoring of products at drug-development meeting, a reminder to sponsors that patient pressure can prod the agency for more than just streamlined approvals.
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Hemophilia A drug's labeling reflects data on physical function improvement because FDA deemed that portion of the Haem-A-QoL instrument fit for purpose, while other questions were viewed as insensitive to change or irrelevant. Review documents suggest agency was unimpressed with results from a health status instrument frequently used in economic analyses.
A cautious patient population with long memories could thwart the potential of new hemophilia A treatments, even as the market leaders promise Wall Street that switching patients will be no problem.
Part 2: High costs are built into the rare disease business model. Participants at a roundtable discussion led by Scrip at the J.P. Morgan Healthcare Conference talked about how much they are feeling the pressure on pricing.