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Genentech's Hemlibra: Clinical Outcome Assessment Data Only Partially Swayed US FDA

 
• By Sue Sutter

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.

Drug Review Profile regular column

Clinical outcome assessments that address clinically relevant concepts meaningful to patients, and are sensitive to change because of treatment, give sponsors the best chance to get meaningful patient experience data into labeling.

That's one lesson that can be drawn from the US FDA's review of Genentech Inc.'s hemophilia A treatment Hemlibra (emicizumab-kxwh).

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