FDA starts PDUFA VII programs for real-world evidence, innovative trial designs

Regulatory NewsRegulatory News | 19 October 2022 |  By 

The US Food and Drug Administration (FDA) on Tuesday provided updates on two programs negotiated under the Prescription Drug User Fee Act (PDUFA VII) meant to give sponsors an opportunity to engage the agency early and spur innovation. The programs address the use of real-world evidence (RWE) to support product effectiveness or to meet post-approval study requirements and implementing innovative clinical trial designs.
In a Federal Register notice, FDA said it will start accepting applicants into its new Advancing Real-World Evidence Program. While the agency has long touted the potential of using RWE to ensure the safety and efficacy of products, the program is specifically aimed at understanding how that can be achieved. Regulators said the intent of the program is to “improve the quality and acceptability of RWE-based approaches in support of new intended labeling claims, including approval of new indications of approved medical products or to satisfy post-approval study requirements.” (RELATED: Industry lauds PDUFA VII focus on innovation, transparency, Regulatory Focus 26 August 2022)
Typically, reviewers only talk to sponsors after they’ve submitted their premarket application to the Center for Drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research. However, under the Advancing RWE Program, sponsors selected by FDA to participate will have the opportunity to discuss the use of RWE even before protocol development and study initiation.
FDA notes that it will hold meetings with program participants during the PDUFA VII program, which covers FY2023-2027. It adds that sponsors developing cancer drugs may see added benefits of participating in the program by getting early input from the Oncology Center of Excellence.
Sponsors with an investigational new drug (IND) or pre-IND, those with proposed RWE intended to meet regulatory requirements for how to present effectiveness in labeling and those who agree to publicly present their study designs may be eligible for the program. FDA specifically notes that lessons learned from the program, such as new study designs, may be presented at future FDA public forums and in guidances. However, the agency is only initially accepting a small number of sponsors for the program due to resource constraints.
“FDA welcomes submissions related to any eligible RWE proposal,” the agency said. “Given the limited number of requests accepted per submission cycle, however, FDA will select requests based on their potential regarding fit-for-use data, adequate study design, and appropriate regulatory conduct. Consideration will also be given to promoting diversity of data sources, study designs, analytical methodologies, and regulatory indications, as well as to diversity of diseases under study and FDA Centers and Offices involved.”
FDA said it will accept initial meeting requests for the Advancing RWE Program semi-annually, with submission deadlines falling on 31 March and 30 September each year. While sponsors can submit applications at any time, the agency will only review those applications in the preceding six-month period. It will also only accept one to two primary meeting requests and up to two alternates requests per submission cycle from FY2023-2024. Eventually, FDA intends to double the number of eligible applicants for FY2025-2027.
“Sponsors will be notified whether they will proceed to discussion of disclosures or their meeting has been denied approximately 45 days after the submission deadline,” FDA said. “For each meeting request granted as part of the Program, FDA will conduct an initial meeting and, if requested, up to three follow-up meetings.”
Alongside the Advancing RWE Program announcement, FDA said it will continue its Complex Innovative Design (CID) Paired Meeting Program, which was first instituted as a pilot program under PDUFA VI.
“The goals of the early meeting discussions granted under this program are to provide advice on how a proposed CID approach can be used in a specific drug development program and to promote innovation by allowing FDA to publicly present the trial designs considered through the program, including trial designs for drugs that have not yet been approved by FDA,” said the agency. “FDA has committed to accepting up to eight proposals each fiscal year.”
The agency will accept submissions for the program on a rolling basis however only those received by the end of a calendar quarter will be considered for the following quarter. Regulators also said that they plan to review the applications and provide a response within 45 days of the end of each quarter on whether an applicant has been accepted into the program or not.
Earlier this year, FDA highlighted three case studies showing how sponsors can incorporate innovative study designs in their clinical trials using the CID pilot program meant to help sponsors understand factors that should be considered when applying for the program. The lessons from that pilot program have been used to develop the full CID Paired Meeting Program. (RELATED: FDA offers examples of innovative study designs accepted into CID pilot, Regulatory Focus 20 January 2022)


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