PDUFA Offers New Tools to Support Drug Development & Review

As sponsors and FDA officials press for Congressional action on the latest version of the Prescription Drug User Fee Act (PDUFA VI), they are highlighting key provisions in the program designed to accelerate drug development and application review. Congressional staffers have set a goal for finalizing user fee reauthorization measures by June, which would fit the timetable sought by FDA leaders and industry officials eager to avoid disruptions in the program.

The Senate Health, Education, Labor and Pensions (HELP) Committee and the House Energy & Commerce Committee plan to hold hearings in the coming months on PDUFA and similar user fee performance goals for generic drugs, medical devices, and biosimilars. The aim is to reach agreement on funding levels for FDA programs and related policy changes before Congress’ summer recess. But “we’re not there yet,” observed Theresa Mullin, director of the Office of Strategic Programs in the Center for Drug Evaluation and Research (CDER), at this week’s DIA forum on regulatory submissions, information, and document management. She acknowledged a number of challenges facing FDA in continuing the gains achieved in the drug review process under the current user fee program, and the need for new processes and resources to do so.

Sponsors requested nearly 3,000 development-phase meetings last year, Mullin pointed out, and new scientific discoveries require further modernization of user fee programs so that FDA can bring in experts with newly-needed technical specialties. PDUFA VI proposes additional resources to support the breakthrough therapy program, early consultation on biomarker use, and enhanced review of drugs for rare diseases. The review of combination products would gain from clarification of internal procedures for evaluating these therapies, including protocols for human factors studies, plus further guidance on bridging studies and patient-oriented labeling.

CDER and the Center for Biologics Evaluation and Research (CBER) plan a more systematic approach for incorporating the “Patient’s Voice” into drug development. The aim is to build on the series of public workshops under the current patient-focused drug development program to produce guidances on assessing burden of disease, treatment impacts most important to patients, impact measures, clinical outcome assessments, and endpoints to inform drug development and regulatory decisions.

A main initiative is to advance the appropriate use of complex adaptive, Bayesian and other novel clinical trial designs, including those where simulation approaches are necessary. FDA plans dedicated meetings and a public workshop to discuss innovative clinical trial designs, which would lead to additional guidance on these study methods. A similar strategy is planned for advancing the use of less common statistical models to advance model-informed drug development.

FDA also seeks to improve the review and predictability of the biomarker qualification process through staff development, public meetings, guidance development and enhanced communication with sponsors on this process. This approach also will be applied to enhancing the use of real world evidence in evaluating drug safety and effectiveness. FDA may initiate pilot studies or develop new methods on this topic, plus guidance on how real-world data can support product assessment.
 
With more applications submitted in fully-standardized electronic form, FDA aims to improve the capacity of its staff to assist and analyze new data standards and relevant procedures. A main goal is to further develop and update electronic data standards, while ensuring transparency and security for data submitters.