LSHC Horizons Brochure 2024 - Flipbook - Page 22
Hogan Lovells | 2024 Life Sciences and Health Care Horizons | Cell, Tissue, and Gene Therapies
FDA’s efforts to support CTGT products
The U.S. Food and Drug Administration
(FDA) has increased efforts to support the
development and approval of cell, tissue,
and gene therapy (CTGT) products. This is
evidenced by the upswing in CTGT approvals
in the last few years and the unprecedented
approval, on the same day in 2023, of a novel
gene editing product and another gene therapy
product for sickle cell disease. Although
FDA’s approval rate is still far behind the
one to two applications FDA receives each
week, the agency continues to demonstrate
commitment to the accelerated development of
these therapies. The agency recently increased
its repertoire of tools for accelerating the
development of CTGT products by providing
additional opportunities for early interactions
with sponsors:
• The START Program. Under FDA’s
Support for clinical Trials Advancing Rare
disease Therapeutics (START) program,
sponsors with active INDs for certain CTGT
products may be able to seek more frequent
advice and have more communication
with FDA on issues such as clinical study
design, choice of control group, and choice
of population, if they meet certain eligibility
criteria. FDA is accepting applications
for the program until 1 March 2024. FDA
may repeat the program, depending on
stakeholder feedback.
• The Voluntary Consensus Standards
(VCS) Recognition Program. This
program permits sponsors to recommend
Yetunde Fadahunsi
Senior Associate
Washington, D.C.
novel scientific standards applicable
to their products if the standard meets
certain criteria. Developed in response to a
mandate under the 21st Century Cures Act
and modeled after a similar program for
medical devices, this program is especially
helpful for regenerative therapies, including
CTGT products, whose complex nature
makes regulatory standards challenging
and unpredictable. FDA has started
accepting standard recognition requests
and will maintain a public list of recognized
standards.
• Platform Technology Designation
Program. This designation is intended to
accelerate review of products for sponsors
who have multiple products that use a
similar technology or platform, like adenoassociated viral vectors. Designation
gives sponsors additional opportunities
for early FDA interactions and may also
permit a sponsor to reference data from the
designated technology.
Other recent efforts include FDA’s advocacy for
greater use of the accelerated approval pathway
under its Split Real Time Application Review
(STAR) pilot program for new uses of approved
therapies to address unmet medical need, as
well as a Rare Disease Endpoint Advancement
(RDEA) pilot program.
Collectively, these FDA initiatives should lead
to more CTGT approvals in the next few years.
Mike N. Druckman
Partner
Washington, D.C.
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