By Kurt R. Karst – Last week, FDA issued its highly anticipated Proposed PDUFA V Reauthorization Performance Goals and Procedures for Fiscal Years 2013 through 2017, which were hammered out between FDA and industry after long negotiations. The proposal includes several new provisions compared to the PDUFA IV Reauthorization Performance Goals. We’ll focus on a couple of them here. Although many of the goals for priority and standard NDA and BLA review (including Class 1 and Class 2 resubmissions, efficacy supplements and manufacturing supplements) remain the same as under PDUFA IV, the proposed PDUFA V agreement establishes a new review model that will apply to all New Molecular Entity (“NME”) NDAs and original BLAs received from October 1, 2012, through September 30, 2017, including applications that are resubmitted following a refuse-to-file action. The new review model, referred to as “the Program” in the PDUFA V proposal, is intended to “promote greater transparency and improve communication between the FDA review team and the applicant” and to “improve the efficiency and effectiveness of the first cycle review process and decrease the number of review cycles necessary for approval, ensuring that patients have timely access to safe, effective, and high quality new drugs and biologics.” The parameters of the Program include, among other things, a pre-submission meeting (which the agreement says is “strongly encouraged”), a mid-cycle communication “to provide the applicant with an update on the status of the review of their application,” and a late-cycle meeting at which the FDA review team and the applicant will discuss the status of the review of the application. At the pre-submission meeting, “FDA and the applicant will agree on the content of a complete application for the proposed indication(s), including preliminary discussions on the need for risk evaluation and mitigation strategies (REMS) or other risk management actions.” In addition, “FDA and the applicant may also reach agreement on submission of a limited number of application components not later than 30 calendar days after the submission of the original application,” such as the submission of updated stability data. The proposed agreement cautions that “[i]f the applicant does not have a pre-NDA/BLA meeting with FDA, and no agreement exists between FDA and the applicant on the contents of a complete application or delayed submission of certain components of the application, the applicant’s submission is expected to be complete at the time of original submission.” As part of the Program, priority and standard NME NDAs and original BLA will be subject to different review goals as compared to other applications. As shown in the table below, the proposed agreement says that FDA will review and act on 90% of standard NME NDA and original BLA submissions within 10 months of the 60 day filing date, and 90% of priority NME NDA and original BLA submissions within 6 months of the 60 day filing date. Use of the filing date instead of the receipt date essentially means that the reviews are slated to take place within 12 months (priority) and 8 months (standard) from submission. Another change of note is the treatment of “major amendments” to pending applications. Under PDUFA IV (and FDA’s regulations at 21 C.F.R. § 314.60), “[a] major amendment to an original application, efficacy supplement, or resubmission of any of these applications, submitted within three months of a goal date, may extend the goal date by three months. A major amendment to a manufacturing supplement submitted within two months of the goal date extends the goal date by two months” (emphasis added). Under the proposed PDUFA V agreement, however, “[a] major amendment to an original application, efficacy supplement, or resubmission of any of these applications, submitted at any time during the review cycle, may extend the goal date by three months” (emphasis added), and “[a] major amendment to a manufacturing supplement submitted at any time during the review cycle may extend the goal date by two months” (emphasis added). The proposed PDUFA V agreement says that a major amendment may include, among other things, “submission of a REMS with [elements to assure safe use (ETASU)] not included in the original application; or significant amendment to a previously submitted REMS with ETASU;” however, “changes to REMS that do not include ETASU and minor changes to REMS with ETASU will not be considered major amendments.” The PDUFA V proposal includes several items intended to enhance regulatory science and expedite drug development, including: (1) initiatives to enhance FDA-sponsor communications (e.g., the agreement says that “FDA will develop a dedicated drug development communication and training staff within the Office of New Drugs in CDER and augment the manufacturers assistance staff in CBER”); (2) approaches and methods for the conduct of meta-analyses; (3) advancing the use of biomarkers and pharmacogenomics; and (4) advancing the development of patient-reported outcomes and other endpoint assessment tools. One initiative, styled as “Advancing Development of Drugs for Rare Diseases,” appears to show the growing importance of orphan drugs. According to the proposal: By the end of FY 2013, FDA will complete a staffing and implementation plan for the CDER Rare Disease Program within the Office of New Drugs and a CBER Rare Disease liaison within the Office of Center Director. FDA will increase by five the staff of the CDER Rare Disease Program and establish and fill the CBER Rare Disease liaison position. On an ongoing basis, the staff in the Rare Disease Programs of the two Centers will develop and disseminate guidance and policy related to advancing and facilitating the development of drugs and biologics for rare diseases, including improving understanding among FDA reviewers of approaches to studying such drugs; considering non-traditional clinical development programs, study design, endpoints, and statistical analysis; recognizing particular challenges with post-market studies; and encouraging flexibility and scientific judgment, as appropriate, on the part of reviewers when evaluating investigational studies and marketing applications for drugs for rare diseases. By mid-FY 2014, FDA, through the Rare Disease Program, will conduct a public meeting to discuss complex issues in clinical trials for studying drugs for rare diseases, including such questions as endpoint selection, use of surrogate endpoints/Accelerated Approval, and clinical significance of primary endpoints; reasonable safety exposures; assessment of dose selection; and development of patient-reported outcome instruments. By the end of FY 2015, FDA will develop and implement staff training related to development, review, and approval of drugs for rare diseases. The training will be provided to all CDER and CBER review staff, and will be part of the reviewer training core curriculum. By the end of FY 2016, FDA, through the Rare Disease Program, will develop an evaluation tool to evaluate the success of the activities of the Rare Disease Program, including the reviewer training. Peter L. Saltonstall, president and CEO of the National Organization for Rare Disorders, applauded the PDUFA V proposal, saying that “[t]he document reflects a clear recognition that drugs for rare diseases warrant special consideration and special staff training.” Additional Reading: PhRMA Statement BIO Statement National Health Council Statement
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