FDA report highlights 2019 drug approvals

The Food and Drug Administration's Center for Drug Evaluation and Research’s annual report, “Advancing Health Through Innovation: New Drug Therapy Approvals,” highlights an abundance of FDA drugs that received special designations in 2019.

The agency reported that 20 of the 48 novel drugs approved in 2019, or 42% as first-in-class, which is one indicator of the drug’s potential for strong positive impact on the health of the American people. These drugs often have mechanisms of action different from those of existing therapies. 

CDER designated 17 of the 48 novel drugs, or 35% in 2019 as Fast Track. Fast Track-designated drugs have the potential to address unmet medical needs. Fast Track speeds new drug development and review by increasing the level of communication between FDA and drug developers, and by enabling CDER to review portions of a drug application ahead of the submission of the complete application.

Thirteen of the 48 novel drugs approved in 2019, or 27%, were designated as breakthrough therapies. A breakthrough therapy designation includes all the Fast Track program features, as well as more intensive FDA guidance on an efficient drug development program. Breakthrough therapy designation is designed to help shorten the development time of potentially important new therapies. Breakthrough therapies are drugs for serious or life-threatening diseases for which there is unmet medical need and for which there is preliminary clinical evidence demonstrating that the drug may result in substantial improvement on a clinically significant endpoint (usually an endpoint that reflects how the patient feels, functions or survives) over other available therapies.

Twenty-eight of the 48 novel drugs approved in 2019, or 58% were designated Priority Review. A drug receives a Priority Review if CDER determines that the drug could potentially provide a significant advance in medical care. The drug is reviewed in an expedited timeline, that is, within eight months instead of the standard 12 months. 

CDER approved nine of the 48 novel drugs, or 19%. in 2019 under the Accelerated Approval program. The application of accelerated approval brings drugs that can provide important advances to patients sooner than with traditional approvals. The Accelerated Approval program allows FDA more flexibility in what endpoints can be used to approve a drug that offers a benefit over current treatments for a serious or life-threatening illness. These accelerated approval endpoints may include ones that show benefits over a shorter duration of treatment (where longer-term demonstration of benefit is needed for full approval) or are considered as “reasonably likely” to predict an important clinical benefit. Subsequent confirmatory trials must be conducted to support full approval. 

Twenty-nine of the 48 novel drug approvals of 2019, or 60% were designated in one or more expedited categories of Fast Track, Breakthrough, Priority Review, and/or Accelerated Approval.

CDER approved 43 of the 48 novel drugs of 2019, or 90% on the “first cycle” of review, meaning without a “complete response” letter from FDA that requires re-submission with additional information, resulting in a delay to approval.

Although regulatory processes differ widely between FDA and those of regulatory agencies in other countries, 33 of the 48 novel drugs approved in 2019, or 69% were approved in the United States before receiving approval in any other country.