Life Sciences Horizons Brochure 2025 - Flipbook - Page 112
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2025 Horizons Life Sciences and Health Care
Accelerated approval: FDA defines whether a confirmatory trial is “underway”
The accelerated approval (AA) pathway allows FDA to
approve drugs for serious conditions that address an
unmet need based on surrogate or intermediate
clinical endpoints that are reasonably likely to predict
clinical benefit or an effect on irreversible morbidity or
mortality. This pathway expedites the availability of
treatments for serious conditions while obligating
sponsors to conduct confirmatory trials post-approval
to verify clinical benefit.
Congress amended the AA statute in 2022 to provide FDA new
authorities. Among other things, the amended statute permits
FDA to require, as appropriate, confirmatory trial(s) to be
“underway” prior to approval or “within a specified time period
after the date of approval.”
In January 2025, FDA published a draft guidance, ”Accelerated
Approval and Considerations for Determining Whether a
Confirmatory Trial is Underway.” The draft guidance states that
FDA generally intends to require that confirmatory trial(s) be
“underway” prior to AA, although FDA explicitly recognizes that
drugs intended to treat some rare diseases may be excepted.
As sponsors consider the timing of submitting applications for
AA, we recommend engaging with FDA about study design,
practical limitations, and challenges in confirmatory trials as early
as possible. In addition to timely agreement on the protocol, these
discussions should include:
Expected timelines for enrollment and study completion.
Factors that may impact timeliness, including the accrual rate
and enrollment timeline, number of active trial sites, and rate
of additional site activation.
Objective benchmarks for measuring progress, e.g.,
recruitment goals or endpoint event accrual.
FDA may be concerned that commercial availability may hinder
recruitment. A sponsor unable to enroll a significant portion of the
anticipated patient population before approval should proactively
engage with FDA on enrollment plans and why timelines can be
met after commercial availability.
FDA has recently issued other draft guidance documents on AA
as well, including regarding withdrawal procedures where clinical
benefit was not confirmed and determining whether a surrogate
endpoint is reasonably likely
to predict clinical benefit.
Per the draft guidance, a trial is considered "underway" if:
it has a target completion date “consistent with diligent and
timely conduct of the trial,”
the sponsor’s plans “provide sufficient assurance to expect
timely completion of the trial,” and
enrollment has been initiated (patients are actively being
enrolled).
Lynn Mehler
Partner
Washington, D.C.
Komal
Karnik Nigam
Partner
Washington, D.C.
Eva Marie Schifini
Senior Associate
Los Angeles
Bryan Walsh
Senior Associate
Washington, D.C.
