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Two of the FDA's expedited programmes: Fast Track gives drugs for serious unmet needs more frequent FDA meetings and rolling review; RMAT is the equivalent for cell and gene therapies. Neither guarantees approval or changes the evidence standard.
Fast Track (1997) is granted on preclinical or early clinical data and is the most common designation; Breakthrough Therapy (2012) requires preliminary evidence of substantial improvement and brings intensive guidance; RMAT (2016, 21st Century Cures Act) applies breakthrough-like benefits to cell therapies, gene therapies and tissue-engineered products, including CAR-T; Priority Review shortens the review clock; Accelerated Approval permits surrogate endpoints. The EMA's PRIME and Japan's Sakigake are counterparts. Designations are frequently announced in press releases and can be rescinded; roughly a third of Fast Track drugs are eventually approved.
Shares FDASIA 2012 and the origins of the expedited programmes, Breakthrough Therapy / Priority Review / Priority Voucher, BLA / NDA / MAA (marketing applications) and PDUFA dates, Accelerated approval.
Shares Sakigake designation (Japan), Japan conditional early approval and time-limited approval, Regulation (EC) No 726/2004: the EU centralised procedure, Accelerated approval.
Shares Orphan drug designation, BLA / NDA / MAA (marketing applications) and PDUFA dates, Accelerated approval.
Shares ILAP (Innovative Licensing and Access Pathway), BLA / NDA / MAA (marketing applications) and PDUFA dates, Regulation (EC) No 726/2004: the EU centralised procedure.
Shares Sakigake designation (Japan), Japan conditional early approval and time-limited approval, ILAP (Innovative Licensing and Access Pathway), BLA / NDA / MAA (marketing applications) and PDUFA dates.
Shares Japan conditional early approval and time-limited approval, BLA / NDA / MAA (marketing applications) and PDUFA dates, Accelerated approval.
Shares 21st Century Cures Act, Regulation (EC) No 726/2004: the EU centralised procedure, Accelerated approval.
Shares Japan conditional early approval and time-limited approval, FDASIA 2012 and the origins of the expedited programmes, Accelerated approval.