FDA Publishes Draft Guidance - Labeling for Human Prescription Drug and Biological Products

February 1, 2019 Becky Hurt

Approved Under the Accelerated Approval Regulatory Pathway January 2019

Authored By: Mamata Gokhale, Principal Consultant, PAREXEL® Consulting.

The accelerated approval is one of several approaches used by the FDA to expedite the development of drugs for serious or life-threatening diseases and conditions upon determination that the product has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit, or on an intermediate endpoint that can be measured earlier than irreversible morbidity or mortality. This guidance focuses on how accelerated approval is represented in the Indications and Usage section of product labeling (USPI) and offers recommendations to sponsors on language that best conveys different circumstances specific to accelerated approval.

Indication and Usage statement for drugs approved under the accelerated approval framework differs from drugs with traditional approval by acknowledging that the drug was approved based upon accelerated approval and that continued approval for the drug (or indication) may be contingent upon verification and description of clinical benefit in a confirmatory trial or trials. Effect on surrogate endpoint or intermediate clinical endpoint such as response rate that supported the accelerated approval needs to be described in the Indication and Usage statement and should be cross referenced to the Clinical Studies section of the label. A similar presentation should also be used under the Indications and Usage heading in Highlights without a cross-reference to the Clinical Studies.

Continuation of accelerated approval is subject to the requirement that confirmatory trials verify the drug’s clinical benefit. Therefore the Indication and Usage statement also needs to state that continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). 

Following successful verification and description of clinical benefit in the post-marketing studies, the information in the INDICATIONS AND USAGE section should be revised to reflect the population and condition for which there is substantial evidence of effectiveness. The statements concerning limitations of usefulness and continued approval should be removed or revised, as appropriate. In addition, other sections of labeling (e.g., Adverse Reactions and Clinical Studies) should be revised, as appropriate, to reflect the new data. Approval of a drug or indication approved under accelerated approval may be withdrawn if confirmatory trial(s) fail to verify the clinical benefit or demonstrate that the use is not safe. If the accelerated approval indication is withdrawn, but the drug remains approved for other indications, the labeling must be revised.

PAREXEL has internal expertise (industry and ex-FDA) in accelerated drug development and corresponding labeling for drugs and biologics. Our labeling subject matter experts can develop and update the labels as well as facilitate labeling negotiations with the regulators.

 

 

 

 

 

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