PDUFA V Sets New Course for Regulatory Science – Joint BIO/PhRMA Statement

Washington, D.C. (October 1, 2012) — Pharmaceutical Research and Manufacturers of America (PhRMA) President and CEO John J. Castellani andBiotechnology Industry Organization (BIO) President and CEO Jim Greenwood today released the following joint statement on the implementation of the Prescription Drug User Fee Act (PDUFA V):

“Today, as implementation of PDUFA V officially commences, PhRMA and BIO 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.

“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.

“Under PDUFA V, FDA will have increased resources and staffing to validate the use of new scientific tools, such as pharmacogenomics and biomarkers, that can help demonstrate therapeutic benefits more rapidly. The Agency also will have dedicated resources to evaluate the use of meta-analyses and provide guidance on standardized methodologies for their use in drug review and safety monitoring.

“In addition, FDA will have the resources to further strengthen its already robust focus on patient safety by standardizing risk evaluation and mitigation strategies (REMS) and helping to make them less burdensome to implement for the healthcare community.

“PDUFA V reflects unprecedented collaboration and transparency among healthcare stakeholders, including PhRMA, BIO, FDA and, importantly, the patients we serve. It also reflects strong bipartisan efforts within Congress to reauthorize the program in a timely manner.

“Unlike prior PDUFA agreements, the architecture of PDUFA V provides opportunities for continuous engagement and assessment for all stakeholders that will allow us to build toward the next reauthorization in 2017. It is incumbent upon us to help ensure that PDUFA V lives up to its vast potential.

“BIO and PhRMA look forward to working with FDA on the implementation of PDUFA V, as well as other elements of FDASIA, in a collaborative, constructive manner that helps elevate regulatory science and fosters the development of new, innovative therapies for patients.”