Spending User Fees: Biomarker Research, Drug-Device Combos?
This article was originally published in Scrip
Even though the technical negotiations between the FDA and representatives from the biopharmaceutical industry are just getting underway this month in earnest for the sixth round of the Prescription Drug User Fee Act (PDUFA VI), some regulators and drug makers already have their eyes set on how the money should be spent, or at least, some of it.
But a group of industry sources on Sept. 2 wanted to make one thing clear: User fees are intended to aid the regulatory application review process and are not for policymaking provisions and mandates.
Nonetheless, they acknowledged that in the rounds that followed the initial PDUFA in 1992, other "policy" measures, like most recently the FDA's breakthrough therapy designation, ended up in broader legislation on which the user fees bills rode through Congress.
The industry sources said manufacturers are keen on making it a priority under PDUFA VI to advance and broaden some of the measures put in place during PDUFA V, whose five-year run officially ends on Sept. 30, 2017.
Top of the list, they said, is figuring out how to incorporate patients' perspectives into the regulatory decision-making process by advancing the science of collecting, analyzing and interpreting that information – something the FDA heard several times over during a congressionally required July 15 public meeting, where the agency inaugurated PDUFA VI.
Indeed, at that July meeting, Sascha Haverfield, vice president of scientific and regulatory affairs at the Pharmaceutical Research and Manufacturers of America, noted that while great progress had been made through the Patient-Focused Drug Development initiative under PDUFA V, "additional clarity is needed on how to translate patient preference information into tangible outcomes to inform clinical research and the regulatory review and decision-making processes."
But Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, also has proposed another area where she'd like to see some of the user fees go: advancing the science of evaluating biomarker qualifications.
"Industry is realizing qualification of biomarkers could help move the field along," Woodcock said during a Aug. 21 symposium hosted by the FDA, in collaboration with the Critical Path Institute and the University of Maryland Center of Excellence in Regulatory Science and Innovation.
She noted one of the greatest barriers to advancing the science of evaluating biomarker qualifications is the lack of funding.
And unless the FDA, academia and industry can get a handle on it, the promise of precision medicine may never be a reality, Woodcock said.
"We look forward to further discussions with FDA and stakeholders about biomarkers and about the use of user fees to enhance regulatory science in general as part of the PDUFA process," industry sources told Scrip in a response to questions about Woodcock's suggestion. "Addressing FDA's review of biomarkers is an important issue, because the use of biomarkers can accelerate access by patients to new treatments. We are happy to discuss with FDA ways of improving the processes of qualifying and using biomarkers either in the context of PDUFA."
But others in industry, too, have shared their thoughts about how some of the user fees should be expended.
Jay Siegel, chief biotechnology officer and head of scientific strategy and policy at Johnson & Johnson, and a member of the board at the Biotechnology Industry Organization, told Scrip earlier this summer he'd like to see a user fees structure established for drug-device combination products.
Right now, the FDA center that has the lead on a drug-device product uses its fees to aid in the review. But when the product gets to the other center, it's not a priority because there are no user fees there to support it, Siegel said.
For instance, he explained, if a drug maker is seeking approval of a medicine that requires an auto-injector, the company pays the user fees to the FDA's drug or biologics centers.
But the agency's device center, which must sign off on the auto-injector, won't get any user fees to speed its review, so therefore, it doesn't have the same timeline and accountability obligations as the drug and biologics centers, ultimately holding up the product's entry to the US market, Siegel noted.
The advancement of drug-device combos and medicines and their companion diagnostics also will be necessary to bring precision medicine fully to fruition, he pointed out.
In response, the industry sources said they were "open to discussing any proposals brought up during the PDUFA VI negotiation process."
While it's unclear now how much the FDA will ultimately collect in users fees through PDUFA VI, the going rate for fiscal year 2016 is a little over $2.37m for an application requiring clinical data and around $1.18m for ones that don't.
House and the Senate appropriators have called for about $2bn in user fees for FY 2016 – bills that must still be reconciled before next year's budget is official.
The FDA and industry are expected to be deep into their PDUFA VI negotiations for the next several months – ongoing at the same time lawmakers also will be trying to get a major overhaul of the US drug agency and the National Institutes of Health (NIH) completed.
In July, acting FDA Commissioner Stephen Ostroff expressed concern over staring PDUFA VI without knowing what may ultimately wind up in the FDA-NIH biomedical legislation – called the 21st Century Cures Act in the House, which has already passed its measure, and Healthier Americans in the Senate.
"You can't have one set of discussions related to user fees and a completely separate set of discussions related to what may show up in these bills unless there's going to be total funding support for what comes out of the bills," Ostroff told the FDA's Science Board during a July 29 meeting.
"One has to somehow inform the other," he said, calling the pending crash of the bills "a bit of a conundrum" and a "very unusual constellation" of occurrences.
Ostroff insisted PDUFA VI "clearly will have to wait" until the biomedical bill is a done deal.
The FDA is set to finalize the draft of its PDUFA VI performance goals letter by March 2016 and submit it the next month to the Department of Health and Human Services, which has until June to clear it and send it on to the White House Office of Management and Budget (OMB).
The OMB's deadline to give its approval is September 2016.
The FDA is expected to hold another public meeting sometime in the fall of 2016.
And the final proposed PDUFA VI performance goals letter is supposed to be in the hands of Congress by Jan. 15, 2017.