Paying for Safety

FDA legislation to boost user fees even more to support new testing and disclosure requirements
Sep 01, 2007

Jill Wechsler
As expected, must-pass legislation to reauthorize the Prescription Drug User Fee Act (PDUFA IV) has provided a vehicle to renew numerous programs and to establish new policies to ensure the safe use of medications. The measure implements a user-fee agreement issued in January 2007 as well as a similar user-fee plan for medical devices. It also continues incentives for sponsors to study pediatric uses of drugs and devices and gives FDA authority to require postapproval studies. Manufacturers will have to disclose more information about ongoing clinical trials and research results and must revise product labels within a set time frame. There is more funding for FDA oversight of postmarket drug use and for agency information systems. And manufacturers of more risky medications will have to adopt a range of pharmacovigilance activities to ensure appropriate product use.

More Fees for Services

In adopting PDUFA IV, Congress boosts the annual fees paid by pharma to adjust for inflation and the increased cost of overseeing the drug-development-and-review process in an efficient and timely fashion. FDA and industry initially agreed on a fee program that would collect nearly $400 million in 2008, with steady increases over the program's five-year period.

A new fee program also bolsters FDA review of direct-to-consumer (DTC) broadcast drug advertising, as discussed in Leading Indicators, April 2007. Some members of Congress sought to ban consumer advertising of new drugs for some months or years after coming to market, noting that even a more efficient and comprehensive review of DTC ads would remain voluntary. Instead, the legislators agreed to authorize stiffer fines and penalties for companies that fail to ensure the accuracy and fair balance of drug ads. All parties will be watching closely to see if misleading commercials aggravate drug safety concerns.

Some of the PDUFA increases will be used to expand drug safety oversight and assessment throughout a product's life cycle—and not just during the first two or three years after product approval, as currently allowed. The payments will support additional staffers in FDA's Office of Surveillance and Epidemiology in the Center for Drug Evaluation and Research and enhance the safety office's role in evaluating postapproval risk information and labeling changes. One specific project is to improve FDA's system for assessing proposed product names, which aims to reduce medication errors from look-alike and sound-alike names.

Push for More Appropriations
Congress is raising the fees even more to support these and other initiatives. And FDA will need all the added fee revenues the legislators provide because its appropriated funding is barely keeping up with inflation (see "Push for More Appropriations").

Managing Risks

One of the legislators' pet provisions permits FDA to require companies to develop a Risk Evaluation and Mitigation Strategy (REMS) to ensure safe use of particularly high-risk drugs. Initially, the legislators wanted to require a REMS for all new drugs but agreed with manufacturers that such a broad mandate could waste time and resources if applied to relatively safe products.

Manufacturers may submit a REMS plan in a new or supplemental application, or FDA may require such a process based on new safety signals for marketed products. A REMS would evaluate the need to:

•conduct additional postapproval studies and clinical trials

•require labeling changes to disclose new safety concerns

•prepare and distribute a Medication Guide or patient package insert

•develop a communication plan for disseminating additional information to healthcare professionals

•seek FDA prereview of advertising and marketing materials to ensure that ads describe serious risks appropriately and fairly

•limit drug prescribing and distribution. FDA may determine that certain treatments should be prescribed only by specially trained health professionals and only to certain patients. Distribution similarly may be limited to select wholesalers and pharmacists. Additional laboratory testing, patient monitoring, and/or patient enrollment in a registry may be required.

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