Under the draft PDUFA V commitment letter, nearly as many deadlines were set for FY 2013 as the remaining four fiscal years of the program cycle. The agency’s efforts fall into six broad categories:

New review model
Risk-benefit structured assessment
Drug safety
Rare diseases
Drug development tools
Electronic application submission

Agency begins to take NME NDAs and novel BLAs into its new review model at the start of the year.

Draft five-year plan on further development and implementation of a structured risk-benefit assessment in the drug approval process due by the end of the first quarter. Implementation across review divisions to begin by the end of the year. Four meetings to be conducted per year, throughout the program cycle, on disease states that could benefit. (Although there are no other fixed deadlines for this effort in the commitment letter, FDA obviously will undertake considerable work on the project over the course of the five years.)

Draft guidance on standards and format for electronic application submission due by Dec. 31, 2012. Final guidance due one year after closing the comment period.

Assessment of review activity adjustment methods and recommendations for change, if necessary, due by the end of the second quarter, and also by the end of FY 2015.

Proposed project plan for clinical terminology standards development related to electronic application submissions due for review and comment by June 30, 2013; plans to be updated annually.

Draft guidance on formal meeting process between FDA and sponsors due by the end of the year.

CDER Office of New Drugs to develop a dedicated drug development communication and training staff and CBER to augment its manufacturer assistance staff to enhance communication with sponsors by the end of the year.

Public meeting to be held by the end of the year to talk about current and emerging scientific approaches for meta-analyses and to solicit feedback on their use in regulatory review.

Public meeting to be held by the end of the year on the status of biomarkers and pharmacogenomics and strategies for scientific exchange in regulatory and non-regulatory contexts.

FDA will complete staffing and implementation plans for the CDER Rare Disease Program (five additional employees) and CBER Rare Disease liaison position.

Guidance for applying statutory criteria on whether a REMS is necessary due by the end of the year.

At least one public meeting on standardizing REMS and reducing their implementation burden to be held by the end of the year.

Public workshops on assessment methods for whether REMS actually mitigate risks, their effectiveness and burden on the health care system to be held by the end of the year.

Public meetings on current and upcoming Sentinel Initiative projects to be held by the end of the year. Agency to fund four to six activities designed to further evaluate safety signals that in previous cases had been the basis for regulatory action or that will help determine the validity of Sentinel in evaluating signals in population-based databases throughout the program cycle.

Report identifying at least one priority project for integrating REMS into pharmacy systems, prescriber education, practice settings and transmission of risk-benefit information to patients due by the end of the first quarter.

Public meeting to talk about issues related to rare disease drug clinical trials, including endpoint selection and use of accelerated approval, required by the middle of the year.

OND drug development and communication staff to train CDER IND review staff in the philosophy of timely, interactive communication with sponsors, best practices for answering sponsors’ simple and clarifying questions, and the role of agency liaison staff in enhanced communication with sponsors by the end of the year.

Public meeting to be held by the end of the year to discuss agency qualification standards for drug development tools, including patient-reported outcomes, new measurement theory and their implications on multinational trials.

Guidance on REMS assessment methods, including whether elements to assure safe use is not unduly burdensome on patient access, due by the end of the year.

Assessment of the impact of electronic application submissions and data standards on the efficiency and performance of the drug review process is due by the beginning of the year.

Interim assessment of new review program for NME NDAs and novel BLAs to be published by March 31; public meeting for stakeholder comment to be held by June 30.

Draft guidance for FDA reviewers and industry on best practices for communication between the agency and IND sponsors during development to be published by the end of the second quarter. Final guidance due within 18 months of the end of the comment period.

Draft guidance on the agency’s intended approach to the use of meta-analyses in its regulatory review processes due by the end of the year; final guidance due 18 months after comment period closes.

Staff training related to development, review and approval of rare disease drugs required the end of the year.

Interim assessment of the strengths, limitations and appropriate use of Sentinel for managing safety issues due by the end of the year.

Evaluation tool for success of Rare Disease Program activities, including staff training, to be developed by the end of the year.

Standardized clinical data terminology and detailed implementation guides due.

Final assessment of new review program for NME NDAs and novel BLAs due by Dec. 31, 2016. Public meeting to be scheduled no later than March 30, 2017.

Second assessment of Sentinel’s strengths, weaknesses and appropriate use for managing safety issues due by the end of the year.

PDUFA V Implementation: A Year-By-Year Breakdown

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