FDA Issues Draft MDUFA Performance Goals and ProceduresMarch 22, 2012
On March 20, 2012, FDA published a notice in the Federal Register announcing a public meeting to discuss proposed recommendations (here and here) for the reauthorization of the Medical Device User Fee Act (“MDUFA”) for fiscal years 2013 through 2017. 77 Fed. Reg. 16239 (March 20, 2012). The recommendations, dated February 17, 2012, stem from meetings between FDA, the medical device industry, and other stakeholders, and may be revised based upon input received at the public meeting and from written comments.
The draft recommendations include the following proposals:
1. Process Improvements
2. Review Performance Goals
3. Shared Outcome Goals
5. Independent Assessment of Review Process Management
6. Performance Reports
7. Discretionary Waiver
We highlight below the proposals that seem intended to respond specifically to industry’s concerns about FDA’s management of the pre-market review process.
There are eight separate items within this one proposal, two of which appear to potentially have the greatest impact on pre-market review: pre-submissions and patient safety and risk tolerance.
Pre-Submissions. In this proposal, FDA has agreed to institute a “structured process” for managing pre-submissions, which include “a formal written request from an applicant for feedback from FDA which is provided in the form of a formal written response or, if the manufacturer chooses, a meeting or teleconference in which the feedback is documented in meeting minutes.”
As part of this structured process, FDA states that it will provide initial feedback to the applicant by email at least three business days prior to the meeting. This feedback will include “written responses to the applicant’s questions; FDA’s suggestions for additional topics for the meeting or teleconference, if applicable; or, a combination of both.”
Most notably, FDA states that it “intends that feedback the Agency provides in a Pre-Submission will not change, provided that the information submitted in a future investigational device exemption (“IDE”) or marketing application is consistent with that provided in the Pre-Submission and that the data in the future submission do not raise any important new issues materially affecting safety or effectiveness.” While this still gives FDA substantial flexibility to change its mind, the intent that the feedback is final is a change from current practice, in which FDA often feels free not to adhere to the preliminary advice it gave to a sponsor. For sponsors to benefit from this new approach, they will need to clearly think through all the issues on which they would like FDA’s feedback, frame very specific questions to address those issues, and be sure to clarify any answers that do not appear to respond adequately to the question posed.
Patient Safety and Risk Tolerance. FDA states that it intends to “fully implement final guidance on the factors to consider when making benefit-risk determinations in medical device premarket review.” FDA issued draft guidance on this topic in August 2011, discussed here. In addition to finalizing the guidance, FDA states that it will meet with patient groups “to better understand and characterize the patient perspective on disease severity or unmet medical need” and will also “increase its utilization of FDA’s Patient Representatives as Special Government Employee consultants to CDRH to provide patients’ views early in the medical product development process and ensure those perspectives are considered in regulatory discussions.”
Taking patient perspectives into consideration may be helpful, particularly in the premarket review of innovative devices intended to address unmet medical needs. In addition to meeting with patients, FDA should carefully consider the comments it received on the draft guidance prior to its implementation, and incorporate and revise the guidance as needed to address those comments.
Review Performance Goals
A common concern expressed by industry is that FDA takes too long to complete its premarket review. In this recommendation, FDA is proposing to “ramp-up” the percentage of submissions that will meet the stated review time goals during each of the years in the five year MDUFA time frame.
The length of the review time is often due to the number of deficiency letters a sponsor may receive. In this proposal, FDA states that, when it issues a deficiency letter, the letter will be based “upon a complete review of the submission and will include all deficiencies. Any subsequent deficiencies will be limited to issues raised by the information provided by the applicant in its response, unless FDA concludes that the initial deficiencies identified do not adequately address important new issues materially relevant” to the pending determination. Though FDA will still have the ability to send more than one deficiency letter, perhaps this goal will encourage FDA to do a very thorough review of the submission and consider all factors before sending the letter. Managers should carefully review proposed additional deficiency letters to ensure the reviewers are not raising new issues that could have been raised in the original letter, but, due to the reviewer’s own mismanagement or oversight, were not.
Independent Assessment of Review Process Management
FDA will award a contract to a private, independent consulting firm that will publish findings on high-priority recommendations within 6 months of the contract award, and will publish its final comprehensive findings and recommendations within 1 year of the contract award. FDA will publish an implementation plan within six months of receipt of each set of recommendations, and will then incorporate the findings and recommendations into its management of the premarket review program. Though on its face it seems like a good idea to have a third party assess the review process management, this idea is not completely dissimilar from FDA’s contract with the Institute of Medicine (“IOM”) to review the 510(k) program and provide recommendations. It is not clear that FDA has, at this time, implemented any of the recommendations provided by the IOM, and there is no guarantee that it will act differently with regard to the third party recommendations. Hiring a consulting group certainly will not have any short-term beneficial impact. While an outside assessment may be valuable, there are many ways in which this can become an exercise in generating a report that gets lots of attention but has no impact on actual practices.
The draft proposals appear intended to address several of the key concerns raised by industry about premarket review. As with all FDA proposals, only time will tell whether FDA will follow its own recommendations, and, if so, whether implementation of those recommendations will have the desired results.