Testimony Analyzes the FDA’s Implementation of the FDASIA

The Food and Drug Administration Safety and Innovation Act (FDASIA) (P.L. 112 144), which reauthorizes user fee programs for innovator drugs and medical devices and establishes two new user fee programs for generic drugs and biosimilar biological products was the subject of a Congressional hearing on November 15, as Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research (CDER) and Dr. Jeffrey Shuren, Director of the Center for Devices and Radiological Health (CDRH), at the FDA testified about its implementation. According to their testimony, the FDA has made significant progress in implementing FDASIA and is on track to meet most due dates established in the FDA’s three-year implementation plan.

User Fee Program Implementation

Included in the FDASIA is the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), which was first enacted in 1992, and the third authorization of the Medical Device User Fee Act (MDUFA III), which was first enacted in 2002. Enhancements to the PDUFA V include increased interaction during regulatory review of New Molecular Entity New Drug Applications (NME NDAs) and original Biologics License Applications (BLAs); regulatory science enhancements to expedite drug development; the development of important new guidance for drug developers; a commitment to develop a structured framework for benefit-risk assessment; various enhancements to the drug safety system; and requirements for electronic submissions and standardization of electronic application data.

Since last July, the FDA has put a new Generic Drug User Fee Act (GDUFA) program to expedite the availability of low-cost, high-quality generic drugs. To date, the backlog of pre-GDUFA applications has been significantly reduced by approximately 40 percent and review efficiencies have been enhanced. The FDA has also conducted completeness assessments for over 900 drug master files and launched the creation of a public list of these files, which are available for reference. This will expedite review of applications containing referenced active pharmaceutical ingredients.

The medical device user fee program (MDUFA III) was reauthorized to help expedite the availability of innovative new products in the marketplace by boosting the medical device’s regulatory review capacity through hiring new staff. The FDA has worked towards its commitment to increase the efficiency of regulatory processes in order to reduce the total time it takes to make decisions on safe and effective medical devices by meeting significantly enhanced performance goals for the device review process. The FDA believes that it has the potential to meet all of its FY 2013 MDUFA III performance goals and expects to see a 25 percent decrease in the backlog of 510(k) submissions. In addition, the FDA believes there will be a decrease in average total time for review of 510(k) submissions and Premarket Approval (PMA) Applications due to the enhancements.

The Biologics Price Competition and Innovation Act (BPCI Act), enacted under the Patient Protection and Affordable Care Act, established a new abbreviated approval pathway for biological products shown to be “biosimilar to” or “interchangeable with” an FDA-licensed biological product. Through this Act, clinicians and their patients should have access to more affordable treatments that are biosimilar or interchangeable.

The testimony noted, however, that, due to the sequestration, approximately $56 million in fiscal year (FY) 2013 user fee funds from PDUFA, MDUFA, the Generic Drug User Fee Amendments (GDUFA), and the Biosimilar User Fee Act (BsUFA) are now unavailable to the FDA. For example, the small 6 percent increase in PDUFA user fees was essentially canceled out by the amount sequestered.

Other FDASIA Provisions

In addition to the implementation of user fees, the FDASIA also includes provisions intended to strengthen the drug supply chain; enhance patient engagement with FDA; address the problem of drug shortages; promote innovation; promote the development of antibacterial drugs; encourage the development of drugs and devices for use in pediatric populations; and enhance FDA’s medical device premarket review program.

Global Drug Supply Chain

Title VII of the FDASIA also promotes drug safety by giving the FDA the authority to protect the integrity of the global drug supply chain. Given that almost 40 percent of finished drugs and 80 percent of active pharmaceutical ingredients are imported, this is of the utmost importance. The FDASIA: (1) increases the FDA’s ability to collect and analyze data to enable risk-informed decision-making; (2) advances risk-based approaches to facility inspection; (3) partners with foreign regulatory authorities; and (4) drives safety and quality throughout the supply chain through strengthened enforcement tools.

Considerable work has been done to implement the Title VII supply chain authorities. The FDA has issued a proposed rule which would extend the FDA’s administrative detention authority to include drugs intended for human or animal use, in addition to the FDA’s current authority for foods, tobacco, and devices. Draft guidance which defines conduct the FDA considers delaying, denying, limiting, or refusing inspection, resulting in a drug being deemed adulterated has been issued. Other recently issued draft guidance also addresses specification of the unique facility identifier system for drug establishment registration. The FDA has successfully worked with the U.S. Sentencing Commission on higher penalties relating to adulterated and counterfeit drugs.

Other Activities

The FDA has also initiated the Patient-Focused Drug Development Program, which is a five-year effort to more systematically obtain a patient’s perspective on a disease and its impact on patients’ daily lives, the types of treatment benefit that matter most to patients, and the adequacy of the available therapies for the disease. The FDA is also actively working towards preventing drug shortages by requiring that manufacturers notify the FDA early if they identify issues that could lead to a potential shortage or a manufacturing disruption. The testimony noted that these identifications have increased “six fold” since October 2011.

A new “break-through therapy” has been designated by the FDA, which will expedite the development of new drugs based on preliminary clinical evidence that indicates the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. The FDA is also working to improve the safety and effectiveness of drugs, biological products, and medical devices intended for use in pediatric populations by addressing the lack of sufficient labeling information for neonates and reauthorizing grant funding for non-profit consortia to stimulate pediatric device development. Woodcock and Shuren went on to address further ways the FDA is working towards implementing the FDASIA, including new device identification and classification systems, health information technology advances and humanitarian device exemptions.