The Prescription Drug User Fee Act (PDUFA) authorizes the Food and DrugAdministration (FDA) to collect fees from Rx pharmaceutical companies to help fund the agency’s drug review work, hire FDA staff, and improve systems. The end goal of this user fee collection system is more timely approval of human drug applications. The regulated industry supports the fees because they help bring their products to the market more quickly and make the review process more predictable. Before the first prescription drug user fee program started in 1992 (PDUFA I), the median drug approval time was more than three years. Currently, the FDA must reach a decision on products within 12 months for a regular application and within eight months for a priority review.

Since the enactment of the original PDUFA measure, the law has been reauthorized four times with each renewal traditionally lasting for a five year period. Current law (PDUFA V) allows the agency to collect around $865 million annually, which is split up among the companies submitting applications. Congress will have the final say over the amount for the next reauthorization, which will be taken up in 2017, but those who have been involved in the negotiations estimate that PDUFA VI would bring the annual amount closer to $1 billion a year. FDA is also working on similar user fee agreements for medical devices and generic drugs. In recent years the combined fees have brought in around $2 billion per year, which represents more than 40 percent of the FDA’s nearly $5 billion annual budget.

During the lead up to each PDUFA reauthorization, as part of FDA’s agreement with industry, the agency commits to achieving certain performance goals to enhance the process of drug review. The document that memorializes this agreement is commonly referred to as the “goals” or “commitment” letter. The goals apply to the review processes, including the review of original new drug applications, resubmissions, and supplemental applications. In mid-July, the FDA released its draft goals-commitment letter for PDUFA VI. The prescription drug industry and patient advocates are praising this preliminary agreement, stating the deal could lead to shorter drug development timelines and the incorporation of patients’ perspectives into reviews. However, some consumer advocates are cautioning that the approach could result in products that are less safe or effective.

The proposed agreement would, among other things, allow the FDA to consider ways to promote new designs for clinical trials. Drug industry representatives believe that using “real-world” evidence and reducing an emphasis on randomized, controlled trials could help shorten the 12 to 15 years it often takes to develop a drug, but underscored that the new agreement takes a gradual approach. Under the draft agreement, FDA would first issue guidance on how real-world evidence can contribute to the agency’s assessment of a drug.

Sources for the new kind of evidence could include electronic health records, insurance data and other databases that can be mined after a drug has gone on sale. However, FDA officials have stated they do not envision using the new evidence for approving new drugs, but for more conservative uses, like allowing new uses of already-approved drugs. Even so, groups like the Public Citizen have expressed concerns that the use of the data could weaken FDA standards.

After a public comment period, the FDA will tweak the proposal before sending it to Congress in January for consideration. The change in administration is not expected to hinder the process, as Congress will need to pass the PDUFA VI legislation before the fiscal year ends on Sept. 30, 2017. Any delays to enactment by that date would put FDA’s drug reviews on hiatus, which could result in massive financial losses for the industry.

Given these high stakes, the PDUFA legislation is considered a must-pass bill, which also makes it a magnet for other FDA-related proposals. For instance, a separate ongoing effort in the House and Senate to pass medical innovation bills this year is delayed because of a dispute over funding for the National Institutes of Health. Most observers believe that legislation will not move this year, but that federal lawmakers will try to attach many of those FDA provisions to the PDUFA VI user-fee bill. Statutory changes to the existing OTC monograph system may also be wrapped into the larger PDUFA reauthorization measure next year. As a result of the significance of this FDA measure, the AAHP will be monitoring the progress of the 2017 PDUFA reauthorization legislation very closely for potential opportunities or threats to the homeopathic drug sector.