Woodcock, Califf Give Thumbs Up To Certain 21^st Century Cures Provisions

US regulators offer their support for law's hiring provisions, limited population pathway for antimicrobials, and patient-focused drug development aspects; but remain mum on the funding levels.

President's Obama's Dec. 13 signing of the 21^st Century Cures Act has opened the door for FDA to break its silence on the law, with top agency regulators giving their blessing to the legislation – at least to some extent.

Center for Drug Evaluation and Research Director Janet Woodcock touted several aspects of the law as being beneficial to FDA. Speaking at the 2016 FDA/CMS Summit, Woodcock said the agency believes, for example, that the law's limited population pathway provision for antimicrobials – which would afford FDA the flexibility to approve antimicrobial drugs based on a limited population if the drug treats a life-threatening infection – would be "helpful."

Woodcock also propped up the law's hiring provisions, which aim to help FDA fill its hundreds of staffing vacancies. The Cures legislation specific allows for the FDA commissioner to make pay more competitive by determining and fixing the annual pay rate up to a limit to help attract and retain employees.

"The hiring and the new pay structure that is put in the legislation – if ever implemented – would be very advantageous to us in attracting talent and being able to get people in, in a more efficient manner," Woodcock said.

Staffing was also a major topic of PDUFA VI discussions, particularly regarding staff for reviewing combination products. The commitment letter requires that additional staff "capacity and capability" be developed during PDUFA VI to improve combination product reviews across the medical product centers and Office of Combination Products. (Also see "PDUFA First: Fees To Support Device Center Staff" - Pink Sheet, 24 Aug, 2016.)

Woodcock further touted the law's focus on patient-focused drug development, which she said "substantiates [FDA's previous efforts] in law." The CDER director noted that the agency has been focusing significantly on patient-focused drug development throughout the years of PDUFA V. The Cures legislation includes several FDA requirements on the subject, including:

• The inclusion of a statement regarding any patient experience data that was used at the time a drug is approved;
• The issuance of guidance regarding how to collect patient experience data; and
• The issuance of a report on the agency's review of patient experience data and information on patient-focused drug development tools.

Patient involvement was a major focus of PDUFA V and will continue under PDUFA VI. The agency agreed to conduct a number of disease-focused meetings that gave patients the opportunity to discuss unmet needs and risk tolerance as part of PDUFA V. (Also see "Patient-Focused Drug Development At 10: Where Does It Go From Here?" - Pink Sheet, 20 Oct, 2014.) Under PDUFA VI, the next step is to make those opinions quantifiable so they can augment a drug application. (Also see "You Just Have To Wait: FDA Can't Hurry PDUFA VI Guidances" - Pink Sheet, 15 Aug, 2016.)

Woodcock did not say whether the agency was satisfied with the level of funding the law provides. The Cures legislatiton provides FDA with a $500 million funding boost over the next 10 years, although it is not mandatory, and must be reauthorized every year.

"I will say that we will try to accomplish everything that is being asked of us," Woodcock said. "We are chronically under resourced… We try to do what we are asked to do. We have a lot of unfunded mandates. Money is contingent each ear on appropriation."

She added that implementing the provisions of the Cures legislation will be one of CDER's top priorities for the coming year.

Commissioner Califf Concurs

FDA Commissioner Robert Califf agreed with Woodcock's assessments. Writing in a Dec. 13 FDA blog post, Califf also gave the thumbs up to the limited population pathway for antimicrobials. He noted that the limited population statement and the additional labeling statements describing the data required by the law "will help assure these drugs are used narrowly to treat these serious and life-threatening infections while additional evidence is generated to assess safety and effectiveness for broader use."

Califf also touted the law's hiring provisions.

"One of our ongoing challenges has been recruiting and retaining the experts we need in specialized areas to allow us to get our work done and meet our growing responsibilities," Califf wrote. "This is an especially important need given the tremendous advances in biological sciences, engineering, information technology and data science. Preventive, diagnostic and therapeutic strategies will become more complex with much greater potential for benefit and in some cases greater risk if used without adequate evidence to exclude risks that exceed potential benefits."

The commissioner additionally propped up a few additional benefits of the law that Woodcock did not mention, including its support for modernizing clinical trial designs and its increased efficiency for regenerative medicine products.

Neither Woodcock nor Califf mentioned the extension and expansion of the priority review voucher program that came with the law, but FDA has made it clear that it is not a proponent of the program. The agency expressed its displeasure with the program in a March report by the Government Accountability Office. FDA says the program has adversely impacted the ability to set its public health priorities and effectively manage its workload. It also contended that it hasn't seen evidence that the program has accomplished its objective of encouraging the development of new treatments. GAO said it is too early to gauge the program's effectiveness. (Also see "Review Voucher Program For Rare Pediatric Diseases Should Not Be Reauthorized, US FDA Says" - Pink Sheet, 3 Mar, 2016.)

CDER's Other Priorities

Another top priority for CDER will be to continue its five-year implementation plan, which began in fiscal year 2013. Woodcock says that the use of formalized data standards has been "extremely helpful" for the agency, but added that there are parts of CDER's work that are not yet standardized.

Woodcock also mentioned the importance of getting the three drug-related user fee programs reauthorized as a priority. She noted that "whatever kinds of interaction we need to have, which are unknown at this point, we are ready, and we stand ready to do whatever is needed to be done to get those programs reauthorized."

Additionally, the CDER director said the center will participate in any process that will help to modernize the OTC monograph process.