LSHC Horizons Brochure 2024 - Flipbook - Page 75
Hogan Lovells | 2024 Life Sciences and Health Care Horizons | Cross-jurisdictional
75
Possibly significant changes to U.S. and EU orphan drug
regulation may be looming
Events over the past year suggest 2024 will bring
heightened attention – and perhaps meaningful
changes – to the orphan drug regulatory schemes
in the U.S. and the EU. Authorities seem to be
seeking to adjust the balance between creating
incentives for orphan drug development and
allowing competing products to come to market.
Recent events that may signal upcoming
significant changes include:
U.S.
• in December 2023, FDA finalized a guidance
document providing additional flexibility in
the regulatory standards for drugs intended
for subsets of rare diseases, including with
regard to nonclinical data and programs for
expediting development.
• a recent FDA decision – being challenged in
court – suggests the agency is changing the
standard for breaking a competitor’s orphan
exclusivity and awarding a new exclusivity
period by a finding of clinical superiority by
way of a major contribution to patient care. A
number of core FDA and administrative law
issues are at play here.
• in denying a rare pediatric disease priority
review voucher for a gene therapy product,
FDA took an approach to defining sameness
of active ingredient that seems difficult to
reconcile with views on the topic expressed in
a relatively recent guidance.
• a provision in the Inflation Reduction Act
(IRA) that exempts certain orphan products
from being subject to price negotiations is
Hein van den Bos
Partner
Amsterdam
leading some companies to rethink strategies
for product development. The provision, which
would seem intended to create incentives
for orphan drug development, may actually
be leading companies to stop investigating
products for second orphan uses.
EU
• The European Commission published
proposed legislation that would reduce the
availability and benefits of orphan market
exclusivity. Proposed changes include:
– reducing the standard orphan exclusivity
period to nine years (from 10, which would
be reserved for products
addressing a high unmet need).
– limiting applicants to a maximum of
two one-year extensions of exclusivity
for subsequent orphan approvals
(rather than the full additional period
of exclusivity currently available with
each such approval).
– allowing competitors to apply for marketing
authorization during the exclusivity period
(rather than waiting until the end of
exclusivity before submission).
– dropping the two-year extension of
exclusivity for completing pediatric studies.
Especially because the nature, extent, and
timing of any changes are uncertain, the strategic
implications can be important for products in all
stages of development, and merit attention.
Komal Karnik Nigam
Counsel
Washington, D.C.
Yetunde Fadahunsi
Senior Associate
Washington, D.C.
