The U.S. Food and Drug Administration (FDA) announced Tuesday a “Commissioner’s National Priority Voucher” (CNPV) program, which will provide a “limited number” of vouchers to sponsors of drug products that are “aligned with U.S. national priorities.” Below we summarize who may be eligible for a CPNV, along with the benefits of a CNPV, which most notably include cutting drug product review time from about 10-12 months to 1-2 months. As we outline below, the announcement leaves several significant unanswered questions about how the program will be implemented. We also note potential litigation FDA may face relating to its authority to implement the program, and to its decisions to either grant or deny a voucher application.
What are the benefits of a CNPV?
According to FDA’s press release, a voucher from the new CNPV program may be redeemed by a sponsor to shorten FDA’s review time from approximately 10-12 months to only 1-2 months after final submission of a drug application. Under the program, FDA will gather experts from various agency offices for a team-based review, rather than using the standard review system. The announcement then explains, “clinical information will be reviewed by a multidisciplinary team of physicians and scientists who will pre-review the submitted information and convene for a 1-day ‘tumor board style’ meeting.”
FDA may provide the voucher recipient with accelerated approval for the product, if it meets the applicable legal requirements. The new review program will also include enhanced communication with the sponsor throughout the process. Touting the program as “common sense,” FDA Commissioner Marty Makary said it “will allow companies to submit the lion’s share of the drug application before a clinical trial is complete so that we can reduce inefficiencies.” These benefits resemble those granted through FDA’s Oncology Center of Excellence’s Real-Time Oncology Review (RTOR) pilot program.
Who is eligible to receive a CNPV?
FDA did not say how many vouchers it intends to grant, but said it plans to issue a “limited number” in the first year, and that it may increase the number of CNPVs it gives following its one-year pilot phase. FDA explains that vouchers will be given “to companies aligned with U.S. national priorities,” including but not limited to:
- Addressing a health crisis in the U.S.
- Delivering more innovative cures for the American people.
- Addressing unmet public health needs.
- Increasing domestic drug manufacturing as a national security issue.
In FAQs issued alongside the press release announcing the program, FDA included the following insights:
- Vouchers can be directed by FDA towards a specific investigational new drug or granted to a company as an “undesignated voucher,” allowing that company to use the voucher for a new drug at the company’s discretion.
- The vouchers are non-transferrable but will remain valid through changes in company ownership.
- The voucher can be applied to a product at any stage in development but must be used within two years following receipt.
- The program does not currently apply to devices or combination drug-device applications.
- The CNPV program will operate separate and independent from existing FDA Priority Review Voucher (PRV) programs, and will not affect pre-existing expedited review programs, including fast track designation, breakthrough therapy designation, accelerated approval, and priority review designation.
- To qualify, sponsors must submit the chemistry, manufacturing, and controls (CMC) portion of the application and the draft labeling at least 60 days before submitting the final application.
Unanswered questions
The new program’s announcement leaves many questions unanswered, including whether (and if so, when) FDA will publish detailed guidelines on qualification criteria, how FDA will evaluate and make voucher grant decisions, and what these abbreviated reviews will look like in practice. Thus far, FDA has merely said it “will use specific criteria to make the vouchers available to companies” and “will provide information in the near future on how companies seeking to apply can indicate their alignment with the FDA Commissioner’s criteria to meet national priorities.” Among other things, it remains to be seen what decision-making process FDA will follow to decide who qualifies for a voucher, and how the criteria for program-specific and undesignated vouchers will differ.
The statutory authority for implementing this program also remains unclear. Endpoints quotes Grace Graham, FDA’s newly-appointed Deputy Commissioner for Policy, Legislation, and International Affairs, as saying on a conference panel before the program’s announcement that a pilot program to speed approvals would not require congressional authorization, but that “statutory language can make the approach more consistent.”
Legal challenges to FDA’s grants and denials of these vouchers seem quite likely, particularly given the potentially enormous value of a shortened review period; priority review vouchers granted under existing statutory programs are routinely sold for more than $100 million. The absence of clear and detailed criteria for qualifying for the program (and possibly of transparent adherence to those criteria) also suggest legal challenges will be launched.
The agency’s statement that the vouchers are not transferable but “will remain valid through changes in company ownership” appears to effectively allow a voucher to be transferred to a company that acquires a company granted a CNPV, which likely will significantly increase the value of the company holding the voucher, particularly “undesignated” vouchers that are not tied to a specific clinical program.
This new program raises questions about the impact on the already strained resources of a reduced FDA workforce, and could compromise FDA’s ability to meet its existing PDUFA commitments. How FDA will triage applications for this program, and whether the agency will have sufficient resources to carry through on the promise of the new program are important considerations.
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