FDA Commissioner Marty Makary may not have the legal authority to implement his new priority review program that will speed up review times for companies that serve U.S. “national interests,” including by lowering drug prices, according to a legal expert who formerly worked at FDA, while stakeholders at an FDA user fee meeting Monday (July 14) expressed worries the agency might overlook safety and efficacy concerns as part of the new initiative.
Makary introduced the Commissioner’s National Priority Review Voucher (NPRV) June 30 as an initiative that will provide companies a voucher, intended to speed up the final review of their product to 1-2 months, if they align with administration priorities like addressing major unmet public health needs, delivering innovative cures and increasing domestic drug manufacturing. Last week, Makary said the vouchers could also be available to companies that voluntarily equalize their U.S. drug prices with those in other wealthy countries.
Reacting to Makary’s remarks last week, Paul Kim, a consultant at firm Kendall Square Policy Strategies and board member of the Friends of Cancer Research, said on LinkedIn the proposal is legally questionable. “There is no statutory authority for Makary’s proposal to award expedited reviews based on drug pricing assurances,” Kim, who is a former FDA policy staffer, wrote.
Some of FDA’s decisions have significant economic implications, Kim wrote, including approvals of biosimilars and generic drugs and of state drug importation plans. But approval of state importation plans requires a determination that the plan must not pose a threat to public health and safety, while FDA hasn’t included in the same disclaimers in speaking about the CNPRV program, which Kim said is based on a “naked bargain of economic advantage of efficiency.”
“FDA’s premarket reviews are public health decisions invested by Congress upon the Agency to be made on the basis of black letter legal standards, against which FDA must weigh scientific evidence to assess risks and benefits, and ultimately decide on whether a new drug is safe and effective for its intended uses,” Kim wrote.
He added that stakeholders are seeking further information on how the CRPV program will be implemented and Congress needs to weigh in to ensure the program is overseen properly.
On Monday, FDA held a meeting for its Prescription Drug User Fee Amendments (PDUDFA) program, including a public comment period. While commenters didn’t focus on the NPRV program, several did express concerns that FDA is prioritizing faster approvals even for drugs that should get additional scrutiny. Some referenced the accelerated approval drug Elevidys for Duchenne’s muscular dystrophy; the sponsor of the controversial drug recently reported a second patient death associated with its use.
Irene Ulrich, a senior policy scientist at the Center for Science in the Public interest, said HHS officials including Assistant Secretary Jim O’Neill have backed the idea of making drugs availability without clear evidence of efficacy.
“At FDA, Commissioner Makary has emphasized accelerating cures, which appears based on an assumption that promising early study results will translate into actual clinical benefit, but data show this is often not the case . . . Meanwhile, and as others have noted, FDA is already the fastest major regulator in the world,” she said.
Patricia Kelmar, senior director of health care campaigns at the U.S. Public Interest Research Group, said FDA needs to make its processes more accessible and use more plain language so that more people can participate in processes, like user fee reauthorization, that will impact their health care.
“You’re under increasing industry pressure to move swiftly to approve new drugs, but speedy approvals must be balanced against the public’s desire and FDA mission to ensure safety and efficacy of new drugs before and after they hit our pharmacy shelves, significantly broaden who’s at the table and implement less intimidating ways than this for the public to express their concerns about new drugs and get answers to their questions,” Kelmar said.
Amanda Berhaupt of the National Center for Health Research agreed the user fee renewal process is not focused on including patient perspectives and that the agency needs to convey its top priority is patient safety. “Speed should be secondary, because when drugs are ineffective or unsafe, patients lose confidence in relationships with their doctors and the FDA and may seek advice from other sources, like social media and where they may find erroneous and harmful advice,” she said.
Other speakers at the meeting, particularly those representing rare disease organizations, said accelerated approvals remain important for patient access and urged FDA to ensure it has the resources to continue prioritizing treatments for small patient populations. — Jessica Karins (jkarins@iwpnews.com)