FDA finalizes guidance on real-time review of oncology drugs

The US Food and Drug Administration (FDA) has finalized its guidance that outlines the eligibility and submission requirements for applicants interested in submitting new drug applications (NDAs) or biologics license applications (BLAs) for its real-time oncology review pilot (RTOR). In the final version, the agency rejected calls to expand the guidance to cover cell and gene therapies.

Overall, the guidance is little changed from the earlier draft issued in July 2022 (RELATED: FDA explains the ins and outs of real-time oncology review program in new guidance, Regulatory Focus 26 July 2022).

FDA said the final version clarifies some terminologies in the draft, clarifies the eligibility and submission process, and better aligns the guidance with the RTOR website.

The FDA Oncology Center of Excellence (OCE) announced the RTOR pilot in 2018 to facilitate earlier review of promising new treatments; the program enables sponsors to submit clinical trial data to FDA as it becomes available to support an earlier start to the application review.

In the final guidance, the agency points out that this program is different from its other expedited review programs, including the fast-track designation program, breakthrough therapy designation program and priority review program. Instead, the program is limited to drugs that have clinical trial endpoints that can be “easily interpreted,” demonstrate substantial improvements over available therapies, and will not require an advisory committee meeting or a new Risk Evaluation and Mediation Strategy (REMS).

The final version incorporates Pfizer’s suggestion to clarify the eligibility requirements. The draft stated that those eligible for the program must have “straightforward study designs as determined by the review division and the OCE.” In its comments on the draft guidance, Pfizer noted that it would “be helpful for FDA to include several examples of the types of study designs that the review division and OCE consider straightforward.”

The final version states that eligible products must have clinical trial endpoints that can be “easily interpreted” in terms of overall survival and response rates as determined by the review division and OCE.

FDA rejected calls from the Alliance for Regenerative Medicines (ARM), Pfizer and the Pharmaceutical Research and Manufacturers of America (PhRMA) to expand the pilot to cell and gene therapies (RELATED: Industry groups: Expand FDA’s RTOR guidance to cell and gene therapies, Regulatory Focus 27 September 2022).

Industry officials offered some tips for preparing applications at RAPS Convergence 2022 meeting. They noted that the RTOR program is a labor-intensive process and for companies to have an “all-hands on deck mentality” as soon as clinical data becomes available (RELATED: Convergence: Upfront planning, communication critical to ensuring success in real-time oncology reviews, Regulatory Focus 14 September 2022).

At the same meeting, FDA officials said about 27% of oncology applications are now submitted through the RTOR, while 28% of supplemental applications are submitted through RTOR.

Final guidance

FDA finalizes guidance on real-time review of oncology drugs

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