LSHC Horizons Brochure 2024 - Flipbook - Page 101
Hogan Lovells | 2024 Life Sciences and Health Care Horizons | United States
Increased scrutiny of the accelerated approval program
In recent years, FDA’s accelerated approval
program, which permits the agency to approve
certain drugs that treat serious or lifethreatening diseases before confirmatory trials
are completed, has come under intense public
scrutiny. There have been increasing concerns
about the rising number of drugs approved
under this pathway and the perceived delays
in drug sponsors completing confirmatory
trials. Congress recently enacted program
reforms aimed to reduce delays in confirmatory
trials and increase transparency, and the U.S.
Department of Health and Human Services
(HHS) could take additional steps to penalize
lags in confirmatory trials.
Increased FDA authority: The Consolidated
Appropriations Act of 2023 included several
new tools for FDA to exert more power over the
accelerated approval pathway, including by:
• allowing the agency to require a postapproval study to be underway prior to
granting accelerated approval;
• permitting FDA to pursue the expedited
withdrawal procedures if a sponsor fails to
conduct any required post-approval study of
the product with due diligence; and
• requiring sponsors to submit periodic
progress reports on post-approval studies at
least every 180 days.
Potential pricing penalties: To identify
strategies to address prescription drug
affordability, HHS recently examined a pricing
model to reduce Medicare Part B payments for
some drugs approved through the accelerated
approval program when sponsors fail to
timely complete their post-approval studies.
For now, HHS appears to have set aside this
proposal, reporting that it will “continue to
monitor” developments in the accelerated
approval program. HHS, Centers for Medicare
& Medicaid Services Innovation Center’s
One-Year Update on the Executive Order to
Lower Prescription Drug Costs for Americans
(11 October 2023). If, however, there is more
public outcry over controversial approvals,
delays in confirmatory trials, or delayed product
withdrawals, HHS may revisit pricing strategies
to penalize delays in confirmatory trials.
Tips for innovators: For innovator sponsors
pursuing the accelerated approval pathway, we
recommend the following:
• communicate early and often with the agency
about both scientific and practical challenges
in designing and completing confirmatory
trial(s);
• assume that the agency will require one or
more confirmatory trials to be underway
prior to approval;
• reach alignment with the agency on the
adequacy of the confirmatory trial(s)
design prior to submission of the new drug
application; and
• manage timing expectations for confirmatory
approval with key stakeholders, including the
public, following accelerated approval.
Eva Marie Schifini
Associate
Los Angeles
101
