On July 9, 2012, the President signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012. This new law includes the reauthorization of the Prescription Drug User Fee Act (PDUFA) that provides FDA with stable and consistent funding during fiscal years 2013-2017 to maintain a predictable and efficient review process for human drug and biologic products. The PDUFA program has revolutionized the review process in the United States and enabled FDA to help bring to market critical new medicines for patients while maintaining FDA’s high standards for safety, efficacy, and quality. Since its enactment in 1992, PDUFA has produced significant improvements in public health by providing patients with faster access to over 1,500 new drugs and biologics, including treatments for cancer, infectious diseases, neurological and psychiatric disorders, and cardiovascular diseases.
This fifth authorization of PDUFA is embodied by Title I of FDASIA and the performance goals and procedures for PDUFA V which resulted from extensive public input, negotiations with industry representatives, and consultation with public stakeholders that began in April 2010. FDA then transmitted the recommendations for PDUFA V to Congress in January 2012. These enhancements include a new review program for new molecular entity new drug applications and original biologics license applications, proposals to enhance regulatory science and expedite drug development, enhanced benefit-risk assessment, modernization of FDA’s drug safety system, and new requirements for electronic submissions with standardized application data.
These topics and more will be on the agenda of a two-day conference that will provide an opportunity for FDA, industry, patient, consumer, and healthcare professional group representatives, and technology vendors to discuss the key PDUFA V commitments, current plans for their implementation, and potential impact on all stakeholders.
Structured Benefit-Risk Assessment in Regulatory Decision Making, Patient-Focused Drug Development, and Advancing the Use of Patient Reported Outcome (PRO) Measures
The major benefit-risk considerations of FDA’s regulatory decision-making have taken shape in a structured benefit risk framework that the agency will implement in drug review during PDUFA V. This framework describes the basis for FDA’s decision-making in terms of severity of condition, the existing treatments options, demonstrated benefits, safety concerns, and risk management. Two of these considerations—the severity of the condition and the existing treatment armamentarium—could benefit from an expanded effort to understand the patient perspective on these issues.
In PDUFA V, FDA will conduct a series of meetings focused on individual disease areas to understand the perspective of those who live with the disease. Developing a good understanding of what benefits truly matter to patients with the disease could lead to future development of patient reported outcome measures, an area of clinical science development that will also be further enhanced in PDUFA V.
Continuing Education Credits are not Available for Archived Recordings
This Archived Recording was recorded live during the PDUFA V Conference. Subsequently there may be short periods of time where the sound level may vary for any given presentation. During these short intervals, you may want to turn up the speaker volume on your computer.
Multiple speakers participated in each session. Some presenters did not provide slides, so only the audio presentation is available.