Fleshing Out FDASIA

Aug 01, 2012

Jill Wechsler
There was much back-slapping and high-fives on Capitol Hill in late June, as leading legislators reached across party lines to quickly approve the Food and Drug Administration Safety and Innovation Act (FDASIA). The bill (S. 3187), which President Obama signed into law last month, enables FDA to collect some $6 billion in fees over the next five years from pharmaceutical and medical device companies. It's much cleaner than the 2007 user-fee measure, which set so many requirements on FDA that it slowed drug approvals for a year.

Now the real work begins, as FDA moves to implement new policies and prepare a host of required guidances and reports. As with most 300-page bills, FDASIA has something for everyone: patient advocates gained incentives for developing critical medicines; providers applauded policies to curb drug shortages; and FDA and industry cheered renewal of user fees that will keep the agency going for another five years.

Accepting risk

On the "spurring innovation" side, this fifth version of the Prescription Drug User Fee Act (PDUFAV) offers assistance for sponsors and greater transparency in the application review process. An important theme is that FDA and pharma should consider patient willingness to accept risk in product development and regulation. Patient advocates played a prominent role in pressing for faster FDA approval of certain 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 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 dealing with rare diseases.

Infectious disease experts won 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 stabilized programs that encourage development of new formulations and expanded labels for pediatric treatments, and a new pilot 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 and biologics, once FDA establishes policies and standards for doing so. Here, FDA and the legislators take the unusual step of making agency guidance mandatory, a strategy that seeks 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 that supports new drug applications.

FDASIA also allows manufacturers to make minor modifications in Risk Evaluation and Mitigation Strategy (REMS) programs. Generic drug makers lost out in their campaign to prevent brand companies 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 accelerate consumer access to generics.

Some Senators sought to add restrictions on the prescribing and sale of opioid painkillers to rein in the rampant abuse of these products, but ran into strong objections from pharmacists. Instead, FDA will hold a public meeting on this thorny topic. That could address whether FDA's long-awaited REMS for extended-release opioids, which was released last month, does enough to control abuse, in that it fails to require prescribers to meet certain educational requirements.

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