Decelerated approval

ODAC supports FDA proposal to raise the bar for accelerated approval

After years of hints, complaints and informal discussion, FDA has explicitly asked its Oncologic Drugs Advisory Committee - and ODAC has agreed - to raise the hurdle cancer drugs will have to clear in order to receive accelerated approval.

The panel agreed with FDA that, except in rare circumstances, single-arm trials are not a sufficient basis for accelerated approval. ODAC also backed FDA's desire for two confirmatory trials to verify clinical benefit, instead of the single confirmatory study that has been customary.

The accelerated approval process was established in 1992 as a way to speed access to drugs that show promise for treating serious or life-threatening diseases. In the cancer setting, FDA has typically granted accelerated approval on the basis of data from a single trial measuring a surrogate endpoint, such as tumor shrinkage or progression-free survival (PFS).

If the proposals discussed last week become policy, one randomized trial will become the standard for accelerated approval. While the agency argues this should not hinder compounds where the response is unambiguous, in practical terms, it most likely means a longer wait for patients who might be helped by the next Gleevec imatinib. Novartis AG's Gleevec, which received accelerated approval in 2001 based on a single-arm trial, transformed CML from a fatal to a chronic disease.

Indeed, of the 49 accelerated approvals granted for cancer, 29 have been based on data from single-arm trials. The most recent example, in November of last year, was the approval of an sNDA from Bristol-Myers Squibb Co. for Sprycel dasatinib to treat newly diagnosed Philadelphia chromosome-positive (Ph+) chronic myelogenous leukemia (CML) in chronic phase.

BMS and Otsuka Pharmaceutical Co. Ltd. were already marketing the oral small molecule inhibitor of BCR-ABL and Src kinases to treat all phases of CML in patients who are resistant or intolerant to prior therapy and for Ph+ acute lymphoblastic leukemia (ALL) patients who are resistant or intolerant

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