FDA Commissioner Dr. Martin Makary has proposed a dramatic expansion of the agency’s powers to speed up drug reviews, announcing on Tuesday a plan to evaluate new medicines that address U.S. “national interests,” like a health crisis, in just one to two months.
The initiative builds on the “priority review” program in place since 2007, which awards companies that bring to market treatments for rare infectious or pediatric diseases a voucher that speeds up a subsequent drug review.
The new program, however, is a pilot that doesn’t have congressional authority and, in its first year, will only grant a limited number of vouchers to companies “aligned with U.S. national priorities.” In a statement, the FDA said those priorities include addressing a U.S. health crisis, delivering “more innovative cures,” addressing unmet public health needs or boosting domestic drug manufacturing “as a national security issue.”
To qualify, companies have to submit the portion of their application covering a drug’s manufacturing process, as well as its proposed prescribing information, at least 60 days before their final submission, according to the FDA. They also have to be available for “ongoing communication with prompt responses.” The agency could additionally extend a review if the application is “insufficient or incomplete,” if study results are “ambiguous” or the review is “particularly complex.”
In a post on the social media site X, Makary said the voucher would permit drugmakers to pre-submit their entire data package, except the results of pivotal trials, to help expedite a review. The agency may increase the number of vouchers issued in subsequent years as well.
The FDA has granted more than 80 priority vouchers through existing programs. Those vouchers are valuable not only for their ability to streamline drug reviews, but also because companies can sell them for upwards of $100 million. The fast passes issued under the new initiative can’t be sold, but would stay with a drugmaker if it’s acquired.
Under the Prescription Drug User Fee Act, the FDA has a deadline of 10 months after a company files an application to make an approval decision. The evaluation period is shortened to six months if a company has been granted a priority review.
The FDA is proposing to chop that time to one to two months by convening cross-functional teams that Makary likened to “tumor boards” of multidisciplinary oncologists who discuss specific cases to quickly decide on a patient’s best treatment course. The agency could use those reviews to support “accelerated” approvals, if warranted by the supportive evidence.
In a note to clients, Rick Weissenstein, a Washington analyst for T.D. Cowen, wrote that the program “appears to borrow elements from various ongoing pilots and regulatory pathways at FDA.” Those include the Real Time Oncology Review pathway, which permits some cancer drug developers to begin submitting an application after data collection is complete but before a pivotal trial has read out, and the STAR program, which shortens the review window for experimental drugs in other disease areas by allowing data submission earlier in the process.
“The real value of the [new program] may be in the designation itself, explicitly stating commissioner-level interest and an inferred predisposition in favor of a fast approval, rather than the aspirational reduced review times,” Weissenstein wrote.
The new program “requires the government to pick winners,” similar to how it chooses who should receive National Institutes of Health grants, wrote David Ridley, the Duke University health economist who first proposed the concept of priority review vouchers, in an email. “The current priority review voucher program sidesteps this by rewarding success without pre-selecting participants.”
Makary also claimed the new program will enhance the communications between applicants and regulators, a back-and-forth that was enabled by the “breakthrough therapy designation” authorized in a 2012 law.
Unlike breakthrough therapy designations, however, the closer dialogue “can encompass a company's entire portfolio” rather than a single drug, Ridley noted.
