LS&HC Horizons 2023 - Flipbook - Page 12
Hogan Lovells | 2023 Life Sciences and Health Care Horizons | Clinical Trials
Advancing expanded access programs
FDA continues to strengthen and modernize
the regulatory framework for expanded access
programs. Expanded access, often referred to
as compassionate use, allows patients with
serious conditions who are not otherwise
eligible for a clinical trial to obtain access to
an investigational drug product. Over the
past few years, FDA has made it substantially
easier for individual patients and their health
care providers to apply for expanded access.
In 2020, the agency launched an electronic
portal for expanded access requests. In a
guidance document FDA issued in November
2022, the agency provided additional insights
into expanded access requirements and best
practices. In 2022, FDA also updated its
guidance related to the limited circumstances
under which a sponsor can charge for its
investigational products (including in
expanded access programs). Further, both
publicly and privately, the agency has been
actively encouraging sponsors to make their
policies for evaluating and responding to
expanded access requests publicly available
as required by the FDA Reauthorization Act
of 2017 and 21st Century Cures Act.
Eva Marie Schifini
Associate, Los Angeles
Taking FDA’s lead, the pharmaceutical industry
is increasing investments into expanded
access programs. Many companies have
built accessible expanded use request portals
and formalized policies and procedures for
reviewing such requests. Often, expanded
access initiatives are being embraced by the
industry as a part of their humanitarian efforts.
Not only do these programs give an opportunity
to provide a novel treatment to a hopeful
patient in dire need, these initiatives can
provide meaningful real world data. In fact,
in 2022, the agency granted accelerated
approval to a drug based on “real-world”
efficacy data from its expanded access program.
Although expanded access programs can
present real risk to clinical development
(e.g., by putting additional pressure on
limited drug supply), in 2023, we anticipate
that companies will continue to bolster their
expanded access programs and, perhaps, find
more opportunities to use these programs
to support commercial development.
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