Would Pulling A "Breakthrough" Break The Program?
FDA retains the right to pull a breakthrough designation if subsequent data do not back up the promise seen in early clinical results, but a few failures are not expected to unduly hurt the expedited regulatory program.
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FDA confidence to devote time and resources for a breakthrough product resonates with investors and analysts, but the real program value in terms of product sales is yet to be seen. Sponsors also must be careful to manage investor expectations once the breakthrough designation is secured.
Pharmaceutical manufacturers and FDA have enthusiastically embraced the “breakthrough therapy” program as a way to more quickly bring new drugs with substantial effects to patients in need. With two years of program experience now under their belts, agency and industry representatives talk about learnings and best practices that could be applied to the development and review of drugs outside the program.
CDER Manual of Policy and Procedures describes specific actions FDA review staff will take once a product has received “breakthrough therapy” designation, with an emphasis on more meetings, more frequently.