The waxing and waning of the speedy approval

As the FDA cools on the accelerated pathway, could biopharma’s annual approval count take a hit?

The FDA began restricting use of the accelerated approval pathway in earnest last year, and these efforts are now apparent in the data. Only 12% of novel drugs arrived on the market via this route last year, a substantial drop from the heady years of 2021 and 2020, an Evaluate Vantage analysis finds.

Heavy use of this fast-track regulatory pathway, particularly in the cancer field, gave a big lift to the agency’s annual approval tally in previous years. This peaked in 2021 when a quarter of its 58 novel, first-time drug approvals were granted on an accelerated basis. With dwindling use of this pathway, the FDA’s output is likely to take a hit.

This probably partly explains last year’s dip, when the agency’s green lights fell well below the 10-year average. True, there are many other moving parts to this picture, but biopharma watchers should perhaps not be too surprised if headline approval figures struggle to match records set in the last few years.

Industry foot-dragging on providing corroborative evidence contributed to the agency closing off this pathway to projects without a confirmatory trial under way. The latest look at the status of historic accelerated approvals suggests there is still much room for improvement here, with a substantial proportion of agents approved as long ago as 2017 still on the market on a conditional basis.  

This includes: Bayer’s Aliqopa, in second-line follicular lymphoma; Astrazeneca’s Calquence in mantle cell lymphoma; and four dangling accelerated approvals since 2017 for the anti-PD(L)1 class.

Until recently this list also included Johnson & Johnson’s and Abbvie’s Imbruvica in relapsed marginal zone lymphoma. The companies voluntarily withdrew that indication, and another mantle cell lymphoma indication granted in 2018, after two study failures.

Speedy approval in niche and underserved settings can of course benefit patients in the longer term, but the need to establish both the risks and benefits of these agents, as expeditiously as possible, remains.

The FDA’s internal data on accelerated approvals, which now runs to the end of 2022, lies behind chart above. The more recent years will of course contain fewer converted approvals, given the time it takes to run studies.

Kudos to Novartis nonetheless, which converted Scemblix’s October 2021 conditional approval, for a refractory form of Philadelphia chromosome-positive chronic myeloid leukaemia, in only 12 months.

Perhaps the picture above will improve with the FDA’s tighter requirements on the status of confirmatory trials. But with the agency also acting fast to pull drugs that fail to make the grade, expect the number of withdrawals to also rise in coming years.

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