FDA offers details on PDUFA VII STAR review pilot

Regulatory NewsRegulatory News | 04 October 2022 |  By 

The US Food and Drug Administration (FDA) has announced details on its Split Real Time Application Review (STAR) pilot, a program designed to shorten the review time of existing drugs and biologics where there is an unmet medical need so patients can access the therapies earlier.
The program would apply to “efficacy supplements across all therapeutic areas and review disciplines that meet specific criteria,” the agency wrote on a new web page. The program includes supplemental new drug applications (sNDAs) and supplemental biologics license applications (sBLAs) with new potential uses for already approved therapies.
Much of the information on the eligibility criteria for the STAR pilot had already been outlined in the agency’s commitment letter containing performance goals and procedures for the Prescription Drug User Fee Act (PDUFA) for 2023–2027, also known as PDUFA VII (RELATED: Rare diseases, STAR expansion considered in PDUFA VII premarket negotiations, Regulatory Focus 16 April 2021). However, FDA added a few clarifying points to the website elaborating on eligibility criteria for STAR pilot applicants.
The pilot program proposes splitting applications into two parts where submissions are sent approximately 2 months apart; the PDUFA clock starts once FDA receives the second part of the application. The first part of the submission will contain NDA/BLA efficacy supplement components apart from the final clinical study reports and a document with topline results for the investigations, while the second part contains the final clinical study reports and electronic common technical document (eCTD) module 2 clinical summaries not in the first part.
FDA said applications are eligible for the pilot program if the drug may be a substantial improvement for clinically relevant endpoints compared with other therapies as measured by “evidence adequate and well-controlled investigation(s),” is intended to treat an unmet medical need, is unlikely to require a long review time and would not require a foreign inspection due to its chemistry, manufacturing or control information.
The agency offered a few clarifications on the STAR pilot program web page for applicants looking to prove substantial improvement over existing therapies. While the therapy being considered for the pilot program is not required to have Breakthrough Therapy Designation (BTD) or Regenerative Medicine Advanced Therapy Designation (RMAT), FDA is using the same qualifying standard for substantial improvement as in BTD and RMAT, and the supplement must already qualify for priority review.
If the drug or biologic is being evaluated in a non-inferiority trial, it is eligible for the STAR pilot program if there is a built-in superiority analysis showing substantial improvement compared with other therapies. However, real-world evidence used to justify substantial improvement of efficacy supplements are not eligible, the agency said. Each applicant must also include an attestation statement in their STAR pilot program request indicating there were no foreign manufacturing sites involved with the drug or biologic, which FDA said they will verify during the first part of the submission.
The agency noted that being accepted into the STAR pilot program is not a guarantee of an expedited review or an application approval.
Interested parties can apply for the program by initiating a STAR entry request as a standalone teleconference request or a Type B sNDA/sBLA meeting request with topline trial results, a rationale for why the application meets the criteria of the program, and any proposed labeling information.  
“If an applicant is disqualified from the STAR pilot program due to incomplete submissions, they may still submit a complete application at a later time; however, they would not receive the benefits of the STAR program review,” FDA wrote.
STAR pilot program
PDUFA VII Commitment Letter


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