FasterCures Comments on MDUFA IV Commitment LetterDivisions of Dockets Management (HFA-305)Food and Drug Administration5630 Fishers Lane,Room 1061 Rockville, Maryland 20852
RE: Federal Register Notice FDA-2016-N-2872 Medical Device User Fee Act; Public Meeting; Request for Comments
FasterCures, a center of the Milken Institute, is a non-profit, non-partisan action tank driven by a singular goal -- to save lives by speeding up and improving the medical research system. We thank you for the opportunity to submit these comments, researched, prepared and authored by FasterCures’ staff, in response to Federal Register Notice FDA-2016-N-2872, Medical Device User Fee Act; Public Meeting; Request for Comments.
At FasterCures, we believe it is critical for stakeholders to develop more systematic ways of capturing and integrating patient perspectives into medical product development and regulatory decision-making. Since its inception in 2003, FasterCures has been working to put patients forward as partners in the biomedical research enterprise. To that end, through our Patients Count program, we are focused on expanding opportunities for patients’ perspectives to shape the processes by which new therapies are discovered, developed and delivered. It has been a privilege to work on behalf of patients throughout the stakeholder process for MDUFA IV.
Overall, FasterCures believes the recommendations put forward by FDA in its Proposed Commitment Letter will continue to enhance and improve the device review program as a whole. Our comments focus on three proposed enhancements outlined in the goals letter, that we believe are specifically relevant to enabling FDA to advance its efforts to deepen and expand engagement with patients, patient advocates and family caregivers.
1. Patient Engagement & the Science of Patient Input (pg 16)
The Center for Devices and Radiological Health (CDRH) has been a leader when it comes to prioritizing patient input in medical product development and regulatory decision-making. In particular, it has issued final guidance on the use of patient preference information in applications seeking regulatory approval for medical devices. Moreover, CDRH has cited the need to “collect and use patient input in regulatory decision-making” as a regulatory science priority for 2017.
We commend FDA for its commitment in the MDUFA IV goals letter to “advance patient input and involvement in the regulatory process.” We believe that the proposed activities outlined in this section will help all stakeholders be better positioned to effectively integrate patient perspectives leading to more efficient and effective development of medical devices that address the needs of patients. We highlight a few specific elements below.
- To ensure the advancement of patient input in the regulatory process, it is critical that FDA have appropriate staff to execute the underlying work. Accordingly, we are pleased to see a commitment to enhance staff capacity and expertise to respond to submissions containing patient preference information (PPI) or patient-reported outcomes (PROs).
- We applaud FDA for requiring public engagement as it explores how to more effectively integrate PPI and PRO evidence into regulatory submissions and decision-making. Input from external experts, including patients, is critical to meaningful advancement of these initiatives. In particular, we think the activity to identify priority areas where decisions are preference-sensitive such that patient perspective data can be used to inform regulatory decision-making, will help the field move forward in a more productive and purposeful way. We encourage FDA to regularly revisit these priority areas as the science of patient input continues to develop, to ensure that resources are allocated to areas of highest impact.
- We are pleased to see a commitment to modify existing guidance on the use of Patient Reported Outcomes (PROs) to outline a framework flexible enough to fit multiple regulatory use cases. We encourage FDA to have ongoing dialogue with external stakeholders, including patients, as it moves forward to develop those modifications.
- FasterCures is particularly supportive of the development of a model for bridging studies that would facilitate the improvement or adaptation of existing PROs. Bridging studies offer the potential to maximize the use of currently validated PRO measures and focus the development of new PROs only where truly needed.
2. Scientific and Regulatory Review Capacity (pg 11)
To carry out its mission and execute the important goals articulated in the MDUFA IV commitment letter, FDA must be able to hire and retain staff with appropriate skills and expertise. Commissioner Califf has cited strengthening the FDA workforce as one of his top priorities and we applaud both FDA and industry for specifically applying user fee revenues to “enhance and supplement scientific review capacity” through focused recruitment, hiring, and retention efforts.
As detailed in our FDA Workforce Fact Sheet, we believe there is more that can be done outside of the boundaries of the MDUFA process to strengthen the agency’s workforce, but we are grateful to see this issue addressed so explicitly in the performance goals letter. We hope to have continuous and ongoing dialogue with stakeholders at FDA and beyond to explore and evaluate the outcomes of these enhancements to hiring and retention.
3. Real World Evidence (pg 17)
We applaud FDA’s commitment to continue to explore how real world evidence (RWE) can be more effectively used in regulatory decision-making for medical devices, including the use of user fee revenue to support the National Evaluation System for health Technology (NEST). This national system for evaluating medical devices is intended to generate evidence across the total product lifecycle of medical devices, leading to improved quality of real-world evidence that health care providers and patients can use to make better informed treatment decisions. There are however, many challenges associated with collecting and analyzing RWE and it is critical for FDA to engage with external experts, including patients, as they explore these challenges. In particular, we would like to see a commitment to include patients on the governing board of NEST to better ensure that the patient perspective is considered as this work moves forward.
Moreover, we think it is also important for FDA to engage with other government agencies (e.g., ONC, CMS, etc.) and non-governmental entities making infrastructure investments, such as PCORI’s PCORnet, where appropriate, to share learnings and discuss what systems are needed to collect RWE. FasterCures looks forward to sharing outputs of its own work in this area in the coming months, as we engage with patients and other stakeholders to build resources to help patients understand how their health-related data becomes part of Real World Evidence and informs decision-making by regulators and other entities.
We thank FDA and industry for the tremendous time and effort put forward to develop this comprehensive and thoughtful commitment letter. We are grateful for the opportunity to submit these comments, and to participate in the formal stakeholder process throughout the course of the MDUFA IV negotiations. FasterCures is committed to being a ready, reliable, and informed partner with the agency to help realize the promise of greater patient participation in regulatory decision-making as a means to improve public health. We welcome the opportunity to explore these comments in greater detail with agency staff.