FDA “Available Therapy” Determination Is A Moving Target, Industry Groups Say
In comments on the agency’s expedited programs draft guidance, PhRMA and BIO say a decision about the relevant standard of care is needed earlier in the development process; groups call for more flexibility on accelerated approval than is suggested in the guidance and identify several issues involving breakthrough therapies that need more clarity.
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Sponsors of ‘breakthrough therapy’-designated investigational oncology products may need to identify a niche subpopulation that remains underserved when a competing product with accelerated approval in the same indication completes confirmatory trials.
Attorneys Sasinowski and Varond write that in some cases weak clinical evidence on the surrogate endpoint was not a barrier, but strong findings on unmet need and rarity usually are necessary for accelerated approval.
Letter being circulated by Rep. Bilirakis states the agency’s draft expedited approval guidance does not provide clarity for rare disease sponsors to use accelerated approval.