FDA “Available Therapy” Determination Is A Moving Target, Industry Groups Say
Executive Summary
In comments on the agency’s expedited programs draft guidance, PhRMA and BIO say a decision about the relevant standard of care is needed earlier in the development process; groups call for more flexibility on accelerated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.
You may also be interested in...
Accelerated Approval Roadblock: Agenus’ PD-1 Stumble Reflects Pathway’s Regulatory Risk
Agenus was banking on US FDA taking the full six months to act on Keytruda application in same cervical cancer indication where it was seeking accelerated approval; agency took less than two months, making it among the fastest oncology supplemental approvals in the last two years.
Accelerated Approval Conversion Could Mean ‘Breakthrough’ Loss
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.
FDA Flexible With Accelerated Approval Evidence, Analysis Finds
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.