FDA, Regulatory

FDA finalizes accelerated approval labeling guidance

The FDA on Tuesday finalized guidance on labeling drugs and biologics approved under the accelerated approval pathway.

The FDA’s accelerated approval program allows the agency to approve products to treat serious or life-threatening conditions based on surrogate or intermediate clinical endpoints “that are reasonably likely to predict clinical benefit.” When granting accelerated approval, FDA will require a sponsor to complete postmarketing studies to confirm the product’s benefits.

The nine-page guidance finalizes a draft version released for comment in 2014 and focuses on the Indications and Usage section of labeling for products granted accelerated approval based on a surrogate endpoint or a clinical endpoint other than survival or irreversible morbidity.

The guidance also provides labeling recommendations for products granted accelerated approval that have subsequently had their clinical benefit confirmed, as well as labeling considerations for products that have had an indication with accelerated approval withdrawn while other indications for the same product remain approved.

Scott Gottlieb

“To make sure this pathway remains robust, we’re taking new steps to help ensure that a product’s labeling provides actionable and complete information about the clinical evidence supporting an accelerated approval and clearly states that post-market commitments may have to be met for an indication to remain approved,” said FDA Commissioner Scott Gottlieb.

While the content and format of labeling for products granted accelerated approval “is in most ways the same as labeling for drugs with traditional approval,” products granted accelerated approval based on a surrogate or intermediate clinical endpoint are required to include a “succinct description of the limitations of usefulness of the drug and any uncertainty about anticipated clinical benefits,” the guidance says.

The FDA also says that the Indications and Usage section should acknowledge that product was granted accelerated approval that may be contingent upon verification of clinical benefit.

In some cases, FDA says that simply detailing the endpoint that was used to support accelerated approval is sufficient to convey the limitations of usefulness and clinical benefit uncertainty. However, the agency says that in other cases “additional context about the approval should be included by identifying the clinical outcome(s) that are expected … but not yet established.”

Sponsors are also advised to include a brief summary of any required postmarketing studies to “further emphasize the limitation of the clinical study results supporting the accelerated approval.”

After clinical benefit has been verified, sponsors are instructed to revise the information in the Indications and Usage section to “reflect the population and condition for which there is substantial evidence of effectiveness, including any new or remaining limitations of use.”

Additionally, the FDA says that sponsors should revise other sections of the labeling to reflect data from the confirmatory studies, such as the Adverse Reactions and Clinical Studies sections.

For products whose accelerated approval is withdrawn that remain approved for other indications, FDA says that sponsors must update the labeling to remove information related to the withdrawn indication.

However, in certain circumstances, sponsors may be required to add new information to the labeling concerning a withdrawn indication, such as when an indication is withdrawn for lack of evidence or when an indication is withdrawn for safety issues.


First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email news@raps.org for more information.


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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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