FDA User-Fee Legislation - Import controls and risk strategies aim to promote quality and spur new drug development. - BioPharm International


FDA User-Fee Legislation
Import controls and risk strategies aim to promote quality and spur new drug development.

BioPharm International
Volume 25, Issue 8, pp. 10-15


This fifth version of the Prescription Drug User Fee Act (PDUFA V) offers additional assistance for research sponsors and greater transparency in the application review process to complete more first-cycle reviews. Patient advocates played a prominent role in pressing for provisions to accelerate FDA approval of new treatments for serious and life-threatening conditions, along with articulation of an FDA risk-benefit framework to clarify that new drugs are never absolutely safe, and that patients accept a certain amount of risk in using new treatments.

Specific provisions provide expedited review of fast-track products and clarification of evidence and endpoints to support accelerated approval of drugs for serious or life-threatening conditions. Grants for developing orphan-drug products will continue, and revised conflict-of-interest rules will make it easier to bring in knowledgeable experts to serve on advisory committees, especially those committees dealing with rare diseases.

Infectious disease experts gained incentives for developing new antibiotics: an added five years exclusivity will apply to specific "qualified infectious disease products," which FDA has to define and list.

Other provisions encourage development of new formulations and expanding labels for drugs for children. The Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act were made permanent, instead of requiring reauthorization every five years. A new pilot program will test whether an offer of priority review vouchers, which can be redeemed for a speedy FDA review of another product, will accelerate development of new therapies for rare childhood diseases.

A less-noticed but important provision requires electronic submission of applications for drugs, once FDA establishes policies and standards for doing so. In this part of the bill, legislators take the unusual step of permitting FDA guidance to set requirements, a strategy that aims to move this policy forward without a lengthy rulemaking process. And the final bill aims to reduce duplicative clinical studies by encouraging FDA to accept foreign clinical trial data to support new drug applications.

FDASIA also makes it easier for manufacturers to make minor modifications in risk evaluation and mitigation strategy (REMS) programs. Generic-drug makers, however, lost out in their campaign to prevent brand firms from using REMS to block access to products needed to test and develop new generic competitors, an issue that could affect biosimilar development down the road. However, other changes in generic exclusivity policy and procedures for handling citizens' petitions promise to enhance access to generic drugs.

Policymakers sought to add restrictions on the prescribing and sale of opioid painkillers to rein in the rampant abuse of these prescription products, but pharmacists objected strenuously. So instead, FDA will hold a public meeting on the need to reschedule hydrocodone and seek ways to exchange prescribing information across state lines to help pharmacists detect multiple painkiller prescriptions. These discussion are likely to address FDA's long-awaited REMS for extended-release opioids, which was issued just after FDASIA was finalized. The REMS stops short of mandating education for opioid prescribers, opening the door to further debate and legislative proposals on controlling these drugs.

Jill Wechsler is BioPharm International's Washington editor, Chevy Chase, MD, 301.656.4634,

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