LSHC Horizons Brochure 2024 - Flipbook - Page 102
Hogan Lovells | 2024 Life Sciences and Health Care Horizons | United States
102
New approach to accelerated approval withdrawals in 2024
FDA’s accelerated approval pathway is
designed to allow earlier access to promising
drugs that treat serious or life-threatening
disease. The pathway permits approval based
on early clinical endpoints (i.e., interim
clinical or surrogate endpoints), but requires
a post-marketing confirmatory trial with an
established clinical endpoint to confirm the
product’s clinical benefit. If clinical benefit is
not confirmed by the confirmatory trial or if
the trial is not conducted in a timely manner,
the indication may be withdrawn. Originally
enacted to help address the HIV-AIDS
epidemic, the majority of accelerated approvals
in recent years have been in the oncology space.
Over the past several years, FDA has focused on
addressing increasing numbers of “dangling”
approvals where the confirmatory trials had
failed, been delayed, or were never completed.
FDORA, signed into law in December 2022,
made several changes to facilitate the timely
completion of confirmatory trials and included
provisions intended to expedited withdrawal
procedures for sponsors that fail to conduct
any confirmatory trial with “due diligence.”
Prior to FDORA, the withdrawal process, set
forth in regulation and intended to provide an
abbreviated process, could drag on for years
before an advisory committee was convened
and the approval was withdrawn.
Lynn Mehler
Partner
Washington, D.C.
Under FDORA, sponsors are not eligible for
a second advisory committee if a committee
was previously convened with respect to
withdrawal. While the intent is to further
streamline the withdrawal process, the
amended statute provides the sponsor with
additional opportunities to engage with
the agency prior to an advisory committee,
including opportunities to meet with the
Commissioner and for public comment. The
exact sequencing of steps remains unclear,
as FDA has not yet updated its regulations to
align with the statutory language. A July 2023
Notice of Proposed Withdrawal of Approval
for Pepaxto (melphalan flufenamide) suggests
sponsors will first have an opportunity to
meet with the Commissioner, followed
by opportunities to appeal and for public
comment on the proposed withdrawal.
The changes also raise questions regarding how
aggressive FDA will be in seeking withdrawal
of indications, and how long sponsors will
have before an indication will be subjected to
the process. Recent agency statements in the
oncology space suggest confirmatory trials
should be completed in two to four years, but
exactly how FDA will exercise its authority
to initiate the withdrawal process both in
oncology and in other disease areas is unclear.
We continuously monitor new developments in
this evolving area and advise clients on how to
engage with FDA on these issues.
Komal Karnik Nigam
Counsel
Washington, D.C.
Eva Marie Schifini
Associate
Los Angeles
Bryan Walsh
Associate
Washington, D.C.
