USA's PDUFA V sets new course for regulatory science, say BIO and PhRMA

With the implementation of the USA’s Prescription Drug User Fee Act (PDUFA V) on Monday, trade groups the Pharmaceutical Research and Manufacturers of America’s (PhRMA) president and chief executive John Castellani and Biotechnology Industry Organization’s (BIO) president and CEO Jim Greenwood issued a joint statement, saying they “stand together to usher in an exciting new era in regulatory science – a period that holds great promise for bringing new, innovative medicines to patients in a timely manner.”

They say: "A reliable, science-driven regulatory environment fosters innovation, promotes economic competitiveness and helps maintain high patient confidence in the integrity of our medicines. PDUFA V, reauthorized in July 2012 as part of the Food and Drug Administration Safety and Innovation Act (FDASIA), is a significant step in this direction. If implemented successfully, PDUFA V will refocus the program on its original intent – timely patient access to new medicines – while strengthening FDA’s high safety standards and helping to establish a new system-wide approach to regulatory science that embraces the scientific tools used in 21st century drug development.

Among its many forward-looking provisions, PDUFA V will enable more timely patient access to safe and effective new medicines through enhanced scientific communications with sponsors during drug development and throughout the drug review process. It also will support the development of a framework to facilitate benefit/risk evaluations of new medicines and integrate patient perspectives in the review process, with a particular focus on diseases with considerable unmet needs, they note.