PDUFA V Recommendations Promote Development of Innovative Therapies

The Prescription Drug User Fee Act [2] (PDUFA) was signed into law in 1992 and authorizes the U.S. Food and Drug Administration to collect fees from companies that produce human drugs and biologics. It was reauthorized in 1997, 2002 and 2007 and needs to be reauthorized again before the current agreement expires in September 2012.

The FDA met with representatives of BIO and the Pharmaceutical Research and Manufacturers of America (PhRMA) in order to craft recommendations for PDUFA V.  With an unprecedented level of public input, the Agency solicited feedback from patient groups and other stakeholders throughout that process and hosted two public meetings, the most recent of which was held in late October.

In that meeting, BIO Managing Director of Science and Regulatory Affairs Andrew Emmett expressed strong support for the PDUFA V recommendations [3] as they will enhance the drug development and review process through increased transparency and scientific dialogue, advance regulatory science, strengthen post-market surveillance, and provide patients and doctors with earlier access to breakthrough therapies.

“We as a nation need to focus policy discussions on how to unleash the promise of biotechnology so that American patients can realize the benefits it has to offer - better health, productivity, and well-being,” Andrew stated. “The PDUFA program is a key element of the overall innovation eco-system.  A fundamental part of biotechnology companies’ ability to innovate and raise private investment is having an FDA with the resources and mechanisms required to effectively and consistently review and approve innovative products in a timely manner based on the best available science.”

In PDUFA V, industry and FDA agreed upon a set of enhancements that seek to restore FDA’s review performance and get back-to-basics for patients.  Underlying the PDUFA V recommendations are the principles that a science-based, transparent, and well-managed review process that appropriately balances benefits and risks can enhance public trust and increase patient access to new medicines.

Key PDUFA V recommendations include:

• NME Review Program: A new review program for New Molecular Entity (NME) applications seeks to reduce overall approval times and improve patient access to novel therapies by increasing FDA-Sponsor interaction during the review, including a mid-cycle communication and late-cycle meeting, so that review issues can be identified and resolved in the first review cycle.
• Enhanced Communication during Drug Development:  To help advance American innovation and promote the development of the next generation of modern medicines, FDA has also committed to a philosophy under PDUFA V that timely, interactive communication with biotechnology and life science companies during drug development is a core Agency activity.  FDA will train staff on best practices for communicating with sponsors and additional FDA staff capacity will help ensure that questions are addressed in a timely manner.
• Modernizing Regulatory Science:  Additionally, the agreement makes new resources available to modernize regulatory science, for example, in the areas of personalized medicine and rare disease drug research. These proposals will also integrate more structured and systematic approaches to assessing benefits and risks of therapies, and allow FDA to better understand patient perspectives on disease severity and unmet medical need.
• Robust Drug Safety and Post-Market Surveillance Capacity: PDUFA V continues industry’s commitment to a lifecycle approach to product evaluation by strengthening FDA’s post-market surveillance and benefit/risk management capacity. Earlier discussion of risk management strategies, standardized approaches to Risk Evaluation and Mitigation Strategies (REMS), and further validation of the Sentinel Network will promote patient confidence in drug and biologics.

Other stakeholders, including the Alliance for Aging Research and National Health Council, also offered their support for the recommendations.

“FDA's processes for evaluating and approving new and innovative therapies for chronic diseases are critical to allow discoveries from basic science to become medical breakthroughs,” stated Daniel Perry, President and CEO of the Alliance for Aging Research [4].  “We recognize that the FDA can only realize this vital role if the proper resources and policies are in place. The current proposal under consideration for PDUFA V is a positive step toward enabling the Agency to conduct more patient-focused, scientifically sound, and timely reviews.”  

Under the agreement, the biotech and pharmaceutical industries reinforced their commitment to a well-funded drug and biologics program that supports sound, science-based regulation consistent with FDA’s public health mission.  However, user fees are intended to support limited FDA activities around the drug review process and were never intended to supplant a sound base of appropriations.  User fees currently account for nearly two-thirds of the cost of human drug review.  Several years ago, BIO joined with other industry, patient, and consumer groups to form the Alliance for a Stronger FDA [5] to urge Congress to support FDA’s mission and provide the resources it needs to meet its ever-expanding responsibilities.

The next step in the process is for the PDUFA V recommendations to be transmitted to Congress in January 2012.  It is critical for Congress to reauthorize PDUFA well in advance of PDUFA IV’s expiration in September 2012 in order to avoid a reduction in force at the FDA.  Even the threat of a downsizing at the FDA would be devastating to the Agency’s public health mission and its ability to review new drugs and biologics.

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