PDUFA PASSES THROUGH CONGRESS
AND AWAITS PRESIDENT'S SIGNATURE
Legislation Provides New Encouragement for Patients
and Biomedical Researchers

This week the United State Congress reauthorized the Prescription Drug User Fee Act (PDUFA) as a component of The Food and Drug Administration Amendments Act of 2007, legislation that will go a long way toward making the drug evaluation and research component of the U.S. Food and Drug Administration (FDA) stronger and more efficient. This legislation will not only provide essential resources to this agency, but it will also provide medical professionals and patients greater access to cutting-edge treatments and will promote greater innovation in biomedical research.

The bill garnered great support from both houses of Congress, passing 405 to 7 in the House and unanimously in the Senate, largely due to effective collaboration between lawmakers, researchers, health care providers and patient advocates. The President is expected to sign the bill into law shortly.

LEGISLATION KEY COMPONENTS

Renewal of Prescription Drug Use Fees
This legislation reauthorizes the prescription drug user fee program, which allows the FDA to collect vital funds from product sponsors to offset the cost of application review. The reauthorization also calls for increased funding (to adjust for a larger workload and keep pace with inflation); the total user fees collected will increase to $392.8 million for Fiscal Year (FY) 2008, an $87.4 million (22.3%) increase over the current base.

Enhancement of Post-Market Surveillance
The FDA will establish a surveillance system to better identify potential adverse effects of drugs on the market. This will be done through enhanced interactions between the FDA and public and private sources such as databases maintained by the Department of Veterans Affairs, large insurance provider groups, or Medicare programs. Scientists will routinely analyze data on millions of patients, looking for signals that drugs pose serious risks. This type of active surveillance will help the FDA continue to gain information on products as they are used by a large, diverse population and provide data that is virtually impossible to collect through pre-market trials.

Creation of the Reagan-Udall Foundation
This legislation creates the Reagan-Udall foundation for the FDA in order to establish a public-private partnership to advance the FDA’s Critical Path Initiative, which will modernize product development, accelerate innovation and increase product safety. The Foundation will also be responsible for identifying unmet needs in the sciences of developing, manufacturing, and evaluating the safety and effectiveness of diagnostics, devices, biologics, and drugs.

Establishment of Risk Evaluation and Mitigation Strategies
This bill codifies the FDA’s authority to require further studies of medical products once they have reached the market. This can be in the form or either observational or clinical studies if new safety information is detected. In addition, the bill creates a “Risk Evaluation and Mitigation Strategy” (REMS) process. If the FDA determines a REMS is necessary to ensure that the drug’s benefits outweigh its risks such a strategy would be applied. A REMS could include elements ranging from package inserts, enhanced communication efforts, or certain restrictions on distribution and use. In order to create a favorable benefit to risk balance, a REMS could be used for new drug or biologic license applications, drugs and biologics that have already been approved, drugs and biologics seeking accelerated approval, and supplemental applications seeking approval of a new indication for use of the drug. If product sponsors violate the required studies or REMS, the bill gives the FDA the authority to apply civil monetary penalties to the company involved.

Regulation of Conflict of Interest
In order to preserve the unbiased nature of the expert committees that advise the FDA on scientific matters, the bill reduces the number of committee members that can have personal financial interests in the products they are reviewing. Currently all individuals under consideration to serve on an advisory committee must disclose all financial interests that may be affected by the committee’s actions. The FDA may grant waivers to potentially conflicted members whose expertise is essential for the committee discussion. The bill requires a 5% reduction in the total number of waivers granted for each of the next 5 consecutive years

CONTRIBUTION FROM RESEARCH ADVOCATES

Last year, Friends convened an expert working group chaired by Fox Chase Cancer Center Chancellor Robert Young, MD, to address pressing issues surrounding drug safety. Earlier this year, the independent working group released a special report: “Drug Safety and Efficacy: Two Sides of the Same Coin.” The report was created to ensure that the voices of scientists, physicians, and patients were heard in this important debate.

With the support of numerous cancer organizations and other research advocates, this report served as a valuable foundation for the drug safety debate. Many of the committee’s recommendations helped shape the elements of the legislation. Friends is deeply appreciative of the committee’s efforts and community support.

ADDITIONAL INFORMATION

• Cure article by Ellen Sigal
• Washington Post Article
• Wall Street Journal Article
• Statement by FDA Commissioner Andrew C von Eschenbach
• Statement by Senator Edward Kennedy
• Complete text of The Food and Drug Administration Amendments Act of 2007 (PDF)