On July 9, 2012, the President signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA) (Public Law 112-144).  This new law includes the reauthorization of the Prescription Drug User Fee Act (PDUFA) that provides FDA with the necessary resources to maintain a predictable and efficient review process for human drug and biological products. This is the fifth authorization of the Act (otherwise known as “PDUFA V”), which was, as directed by Congress, developed in consultation with drug industry representatives, patient and consumer advocates, health care professionals, and other public stakeholders. FDASIA ensures that FDA will continue to receive a source of stable and consistent funding during fiscal years 2013-2017 that will help the Agency to fulfill its mission to protect and promote public health.  These resources will help bring to market critical new medicines for patients.   

Benefits to Patients:

• A new opportunity for greater engagement to inform drug development and review:o FDA will engage in a series of public meetings over the course of the next 5 years, holding meetings on 20 different identified disease areas.
  • FDA will seek to hear more patients’ perspectives on the range of severity of their illness and what matters most to patients at different stages of disease progression. 
  • FDA will ask patients about their perspective on and experiences with currently available therapies, and the degree to which available therapies meet their needs.
  • This information will help inform future risk-benefit assessments for new drugs to treat that disease, and may identify the need for the development and qualification of additional measures of benefit such as patient-reported outcomes that would matter to patients and the quality of their lives.
• PDUFA V provides continuing opportunities to participate in public workshops and inform FDA efforts on a number of key initiatives including enhancing benefit-risk assessment in regulatory decision making

Benefits to Industry:

• PDUFA V includes the development of a new review program for certain drugs and biologics, to enhance communication and transparency between FDA and sponsors through mid-cycle communications and late cycle meetings during FDA’s application review.
• PDUFA V will enhance benefit-risk assessment to integrate a more structured approach in FDA’s regulatory decision-making.
• PDUFA V will enhance and modernize the FDA Drug Safety System by:
  1. Initiate public process to standardize REMS in an effort to reduce the burden on the health care system while ensuring that REMS achieve their intended goals
  2. Using Sentinel to actively target and evaluate any drug safety issues
• PDUFA V requires standardized, fully electronic submissions in an effort to improve the quality of industry applications and the efficiency of FDA’s reviews

PDUFA V Performance Goals and Efficiency Improvements:

• PDUFA V will provide FDA with a 6% increase over FY 2012 fee revenue.
• The FY 2013 fee revenue is set at a base of $693 million and will be adjusted for inflation and workload associated with the review of human drug and biologic applications.  
• FDA Goals include:
  • Reviewing and acting upon 90% of priority applications within 6 months
  • Reviewing and acting upon 90% of standard applications within 10 months

Enhancing Regulatory Science and Expediting Drug Development:

• PDUFA V establishes a team of liaisons that will be available as an additional resource to enhance communication between FDA and sponsors during drug development. 
• PDUFA V advances the science of meta-analyses by developing best practices for conducting meta-analyses and approaches for their application in regulatory review.
• PDUFA V advances the use of biomarkers and encourage their use in clinical trials by enhancing FDA’s ability to address and incorporate these complex issues in application review.
• PDUFA V enhances the development of patient-reported outcomes (PROs) to ensure that these endpoint measures are well-defined and reliable to support claims of treatment and benefit.
• PDUFA V increases FDA’s capacity to work with both sponsors and reviewers to speed the development of drugs for rare diseases.