LSHC Horizons Brochure 2024 - Flipbook - Page 95
Hogan Lovells | 2024 Life Sciences and Health Care Horizons | United States
Pediatric exclusivity and the Pediatric Research Equity Act
FDA’s pediatric framework, most notably
including the Best Pharmaceuticals for
Children Act (BPCA) and the Pediatric
Research Equity Act (PREA), has played a
pivotal role in incentivizing and regulating
pediatric drug development. The BPCA,
enacted in 2002, offers exclusivity (i.e.,
six additional months for some patents/
exclusivities) in exchange for conducting
pediatric studies based on a written request
(WR). PREA, enacted in 2003, mandates
pediatric studies for certain pharmaceuticals
and biologics. The BPCA incentives and the
PREA mandates are sometimes referred to as
the “carrot” and the “stick”. In May 2023, FDA
issued two draft guidance documents outlining
recommendations for sponsors investigating
products for pediatric indications. In these
guidances, FDA proposed significant changes,
particularly regarding the issuance of WRs for
pediatric exclusivity.
Historically, despite the critical roles the two
statutes play in pediatric drug development,
FDA had provided little comprehensive
guidance on the interplay between BPCA and
PREA. The new draft guidances seek to assist
industry in (1) complying with PREA and
qualifying for pediatric exclusivity, and (2)
developing data and obtaining information
needed to support approval of drug products in
pediatric populations.
Deborah Cho
Senior Associate
Washington, D.C.
The most consequential change is FDA’s newly
proposed policy on issuing WRs. The draft
guidance generally explains that a sponsor
must be in receipt of a WR from FDA to qualify
for exclusivity, how FDA determines whether
studies “fairly respond” to the WR such that
exclusivity would be granted, and the scope
of the exclusivity once granted. However, the
agency has proposed to limit the issuance of
WRs to only sponsors who conduct additional
pediatric studies beyond what is required under
PREA. Previously, a sponsor could benefit from
pediatric exclusivity even though it was doing
solely what was already required under PREA.
The WR would often mirror PREA-required
studies, allowing the sponsor to benefit from
the “carrot” while at the same time satisfying
requirements under the “stick”.
The result of the new proposed policy, which
the agency states will only go into effect upon
finalization of the guidance, would be an
expansion of studies required for pediatric
exclusivity beyond what has historically been
required. This may result in more expansive
WRs and may lead to fewer opportunities for
pediatric exclusivity.
We continuously monitor FDA’s actions in
this area, and are keeping a close eye on FDA’s
proposed approach to advise clients on how to
engage with FDA on WRs.
Bryan Walsh
Associate
Washington, D.C.
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