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

The U.S. Food and Drug Administration (FDA) on Tuesday provided updates on two programs negotiated under the Prescription Drug User Fee Act (PDUFA VII) to give sponsors an opportunity to engage the agency early and drive innovation . The programs address the use of Real-World Evidence (RWE) to support product efficacy or to meet post-authorisation study requirements and to implement innovative clinical study designs.

in one Federation Registration Notice, the FDA said it will begin enrolling applicants in its new Advancing Real-World Evidence Program. While the agency has long touted the potential of using RWE to ensure product safety and efficacy, the program specifically aims to understand how this 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 the approval of new indications of approved medical products, or to meet the requirements of post-approval studies.” (TIED TOGETHER: Industry commends PDUFA VII’s focus on innovation and transparency, Regulatory focus 08/26/2022)

Typically, reviewers only speak to sponsors after they have submitted their pre-application to the Center for Drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research. However, as part of the Advancing RWE program, sponsors selected by the FDA to participate have the opportunity to discuss the use of RWE even prior to protocol development and study initiation.

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The FDA notes that it will hold meetings with program participants during the PDUFA VII program, which covers fiscal years 2023-2027. It adds that sponsors developing cancer drugs may derive additional benefits from participating in the program if they receive early input from the oncology center of excellence.

Sponsors with an Investigational New Drug (IND) or Pre-IND, those with proposed RWE who are expected to meet regulatory requirements to demonstrate efficacy on labeling, and those who agree to publicly present their study designs can apply for the program in question. The FDA expressly points out that the knowledge gained from the program, e.g. B. new study designs, can be presented at future FDA public forums and in guidelines. However, due to resource constraints, the agency initially only accepts a small number of sponsors for the program.

“The FDA welcomes submissions related to all eligible RWE proposals,” the agency said. “However, given the limited number of applications accepted per submission cycle, FDA will select applications based on their potential for usability data, appropriate study design, and appropriate regulatory conduct. It also considers promoting the diversity of data sources, study designs, analytical methods, and regulatory indications, as well as the diversity of the diseases studied and the FDA centers and offices involved.”

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The FDA said it will accept initial meeting requests for the Advancing RWE program semi-annually, with submission deadlines falling on March 31 and September 30 each year. While sponsors can submit applications at any time, the agency will only review those applications in the previous six months. It also only accepts one to two primary meeting requests and up to two alternate requests per FY2023-2024 submission cycle. Ultimately, the FDA intends to double the number of eligible applicants for fiscal years 2025-2027.

“Sponsors will be notified approximately 45 days after the submission deadline whether to proceed with discussion of the disclosures or have their meeting denied,” the FDA said. “For each session request granted under the program, FDA will conduct an initial session and, if desired, up to three follow-up sessions.”

In addition to announcing the Advancing RWE Program, the FDA announced that it will continue with its Complex Innovative Design (CID) Paired Meeting Program, which was initially introduced as a pilot program under PDUFA VI.

“The goals of the early briefing discussions granted under this program are to provide advice on how a proposed CID approach may be used in a particular drug development program and to encourage innovation by allowing the FDA to review the study designs considered under the program publicly, including study designs for drugs that have not yet been approved by the FDA,” the agency said. “The FDA has committed to accepting up to eight proposals each fiscal year.”

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The agency accepts submissions for the program on an ongoing basis, however only those received by the end of a calendar quarter will be considered for the following quarter. Regulators also said they plan to review applications and provide a response within 45 days of the end of each quarter as to whether or not an applicant has been accepted into the program.

Earlier this year, the FDA highlighted three case studies showing how sponsors can incorporate innovative study designs into their clinical trials using the CID pilot program to help sponsors understand factors to consider when applying to the program . Lessons learned from this pilot program were used to develop the full CID Paired Meeting Program. (TIED TOGETHER: The FDA provides examples of innovative study designs that have been included in the CID pilot program, Regulatory focus January 20, 2022)


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