FDA explains the ins and outs of real-time oncology review program in new guidance

Regulatory NewsRegulatory News | 26 July 2022 |  By 

Cancer drugs under development that show substantial promise over existing therapies and have simple study designs, as well as easy to interpret endpoints, may qualify for a head-start review from the US Food and Drug Administration (FDA). While the drug will ultimately be reviewed under the same user fee time frame as other products, it may help speed up the review process for sponsors.
On 22 July, FDA published a draft guidance titled, Real-Time Oncology Review (RTOR) Guidance for Industry. The program is meant to help identify promising new cancer treatments that may qualify to be reviewed early by the Center for Drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research (CBER).
In 2018, FDA’s Oncology Center of Excellence and the Office of Oncologic Diseases (OOD) launched the RTOR program to allow sponsors to submit clinical trial top-line results and data of their cancer drugs in development sooner. The objective is to let FDA reviewers not only get a glimpse of the data before an official submission of the premarket application but also allow them to provide feedback on the clinical trial. (RELATED: OCE year 4: Progress despite a pandemic, Regulatory Focus 8 February 2022)
FDA notes that efficacy and safety data are submitted at the same time as other elements of the drug application during a typical drug review which can take months to assemble and complete.
“The OCE developed RTOR to facilitate earlier submission of critical efficacy and safety data to initiate FDA’s evaluation of the application, whereby components of individual modules (e.g., parts of the clinical module, etc.) may be submitted at separate times,” the agency notes. “RTOR is different than the existing mechanisms for rolling review in which, generally, complete modules (e.g., the complete clinical module) are submitted prior to a complete application submission. The intent of RTOR is to provide FDA reviewers earlier access to data, to identify data quality and potential review issues, and potentially provide early feedback to the applicant, which can allow for a more streamlined and efficient review process.”
However, to qualify for the program FDA says it needs three things from sponsors. They need to show their drug is likely to demonstrate substantial improvement over other available treatments or would qualify for the agency’s Expedited Programs. They would also need to have a straightforward study design as determined by the review division and the OCE, as well as easily interpreted clinical trial endpoints.
If the drug qualifies for the RTOR program it “does not alter the review performance goals and timelines associated with the applications, including as described in the Prescription Drug User Fee Amendments (PDUFA),” FDA notes. “Although early approvals have occurred with applications included in the RTOR, this may not be feasible for all applications due to specific issues that may be identified with the application or overall workload considerations.”
“Acceptance into the RTOR program does not guarantee or influence approval of the application, which is subject to the same statutory and regulatory requirements for approval as applications that are not included in RTOR,” the agency adds. “Participation by the applicant in this program is voluntary. If at any point FDA determines participation in the program is no longer appropriate, FDA may rescind acceptance and instruct the applicant to use routine submission procedures for their application.”
The draft guidance also lists the proposed process the RTOR program will take starting with submission of the top-line results from a pivotal trial when the data is available. Sponsors can submit a request to be considered for the program via email to their assigned Regulatory Project Manager (RPM) and to the Investigational New Drug Application (IND) according to FDA. They should include their top-line results and a written justification for why they should be included in the RTOR program.
“The applicant should also include a proposed timeline of when it will submit the various components of the RTOR application,” FDA says. “The review division director/deputy director, with input from the review team (including reviewers, team leaders, and management from all relevant review disciplines), will decide whether the application will be selected for the RTOR program. This decision will generally be made within 20 business days of receipt of the request and communicated to the applicant via email.”
Stakeholders can comment on the draft version of the guidance until 20 September on www.regulations.gov under docket no. FDA-2022-D-0823.


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