Back-to-Basics with PDUFA V

As Congress considers renewal of the Prescription Drug User Fee Act (PDUFA V), it must not lose sight of its most important beneficiary: patients. Patients deserve a clear, efficient, and predictable drug-review process that is science-based, judicious, timely, and appropriately balances benefits and risks. Patients and their families also must have the utmost confidence that the system will speed their access to new, safe, and effective cures and treatments. PDUFA, first passed in 1992, must be reauthorized every five years, and the Biotechnology Industry Organization (BIO) supports a "back-to-basics," patient-centric approach to PDUFA reauthorization. Over the past 20 years, the PDUFA program has contributed to the approval of more than 1200 new medicines and reduced review times for the newest, most innovative drugs by more than a year. In the past year alone (fiscal year 2011), biopharmaceutical companies have successfully brought to market new therapies to treat hepatitis C, melanoma, lung cancer, lupus, and rare genetic disorders.

Sara Radcliffe

However, PDUFA review performance experienced considerable stress in the early years of PDUFA IV due to a number of factors, including the scientific complexities of new drug and biologic applications; implementation of the FDA Amendments Act of 2007, and of Risk Evaluation and Mitigation Strategies (REMS); more frequent Advisory Committee meetings; and the profound impact of globalization, including the occurrence of more foreign inspections. As a result, FDA's review performance diminished, review times lengthened, and more applications went into multiple review cycles, leaving patients waiting longer for new therapies. Although FDA review performance has improved in recent years, there is much more that FDA and industry can do to strengthen and reinforce the PDUFA program.

After intensive technical discussions informed by an unprecedented level of public input, the biotechnology and pharmaceutical industries and FDA agreed on a set of PDUFA enhancements that seek to reinforce the agency's review performance. Highlights include: creating a new molecular entity review program for novel drugs and biologics that strengthens scientific dialogue and transparency between FDA and sponsors so that potential review issues can be identified and resolved early in the process; ensuring timely and interactive communication with companies during drug development; making new resources available to modernize regulatory science; and strengthening FDA's postmarket surveillance and benefit-risk management capacity through earlier discussion of risk-management strategies, standardized approaches to REMS, and further validation of the Sentinel Network.

In addition, there are several specific issues that BIO believes are crucial to modernizing the regulatory environment, promoting biomedical innovation, and encouraging investment. For example, user-fee legislation pending in both the House and Senate include provisions to modernize FDA's Accelerated Approval pathway, based upon Senator Kay Hagan's Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act (S. 2113), and the Faster Access to Specialized Treatments (FAST) Act (H.R. 4132), sponsored by Reps. Cliff Stearns (R-FL) and Ed Towns (D-NY).

These provisions would expedite the development of modern, targeted, and personalized therapies for patients suffering from serious and life-threatening diseases, while preserving FDA's robust standards for safety and effectiveness. These reforms would expand the accelerated approval pathway to conditions beyond just HIV/AIDS and oncology, better utilize modern drug-development tools and new surrogate and intermediate clinical endpoints, and take into account the severity and rarity of a condition subject to approval under accelerated approval. In short, it will help make the drug-development process as efficient, effective, and streamlined as the biotech companies producing the next generation of scientific breakthroughs. We hope Congress will fully support FDA's human drug review program—both through user fees and the appropriated budget—and pass PDUFA V as expeditiously as possible.

The BIO International Convention will return to the Boston Convention and Exhibition Center, June 18-21, 2012. Highlights include an FDA Town Hall, a Super Session on PDUFA V, and a Breakout Session on the Analysis and Impact of PDUFA V.

Sara Radcliffe is executive vice-president for health at the Biotechology Industry Organization (BIO).