LSHC Horizons Brochure 2024 - Flipbook - Page 103
Hogan Lovells | 2024 Life Sciences and Health Care Horizons | United States
103
Spotlight on single-trial approvals
In general, standalone marketing applications
must present substantial evidence of
effectiveness (or safety, purity, and potency)
to support a New Drug Application (NDA) or
Biologics License Application (BLA). FDA has
interpreted this to mean two or more adequate
and well-controlled trials (AWCTs), albeit with
notable exceptions (for drugs and biologics
with well-known, longstanding uses). Since
1997, FDA has had the statutory authority to
accept one AWCT plus confirmatory evidence
as sufficient for approval. FDA has issued
several guidance documents intended to
clarify the characteristics of a single AWCT
and the amount and type of data required for
confirmatory evidence.
The most recent draft guidance, issued
in September 2023, outlines the quality
and quantity of evidence required for an
application supported by a single AWCT,
plus confirmatory evidence. It introduces
the concept of a sliding scale for the amount
of confirmatory evidence needed to support
approval, based on the features and results
of the AWCT. The guidance also provides
examples of categories of confirmatory
evidence. These categories include clinical
evidence from a related indication, mechanistic
or pharmacodynamic evidence, evidence from
a relevant animal model, evidence from drugs
in the same pharmacological class, natural
history evidence, real-world data/evidence,
and evidence from expanded access use of an
investigational drug.
David M. Fox
Partner
Washington, D.C.
One important consideration, particularly for
BLA sponsors, is whether the confirmatory
evidence intended to support a single-study
application relies on data that the sponsor
does not own or to which the sponsor does
not have a right of reference. FDA specifically
notes in the draft guidance that use of certain
sources of information may not be permitted
under certain regulatory pathways. This likely
refers to the fact that, unlike for NDAs, there is
no “hybrid” 505(b)(2) pathway available for a
standalone BLA submitted under section 351(a)
of the Public Health Service Act to rely on
data not owned by the sponsor. BLA sponsors
must therefore be cautious about submitting
confirmatory evidence that the agency may
view as impermissible for a standalone
application.
Relatedly, FDA draft guidance from May 2023
describes the extent to which an application
may rely on “generally accepted scientific
knowledge” for certain non-clinical data
requirements.
We expect that questions regarding the
sufficiency of the clinical and non-clinical data
package for both BLAs and NDAs will continue
to be of significant focus in 2024. We look
forward to continuing to assist our clients in
crafting strong scientific and legal arguments to
bring critical therapies to market.
Lynn Mehler
Partner
Washington, D.C.
Komal Karnik Nigam
Counsel
Washington, D.C.
Bryan Walsh
Associate
Washington, D.C.
