FasterCures Comments on reauthorization of FDA's Prescription Drug User Fee Act (PDUFA-VI)
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 in response to Federal Register Notice FDA‐2010‐N‐0128‐0078, Prescription Drug User Fee Act; Reopening of Comment Period.
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.
We believe PDUFA‐VI presents a tremendous opportunity to make meaningful progress toward this goal. FasterCures was very encouraged by FDA’s presentation at the October 29, 2015 stakeholder meeting on Patient‐Focused Drug Development. In particular, we are pleased to see FDA exploring how to move beyond discrete PFDD meetings to a “more systematic collection of patients’ input.” In addition, FasterCures has consistently advocated for incorporating patient perspective data throughout the medical product lifecycle, and we are glad to see FDA asking questions about how to effectively incorporate the “voice of the patient” into all aspects of medical product development – beginning with early stage research and extending through post‐market assessments. Furthermore, we applaud FDA for emphasizing the need to focus on identifying what “matters most to patients” and for recognizing that patient input goes beyond a patient’s tolerance for risk and expectation of benefits, and includes patient perspectives on unmet medical need, disease burden, individual preferences, and more.
Finally, we agree that providing guidance on “pragmatic and methodologically sound strategies, pathways, and methods to gather and use patient input,” is a logical and critical next step. To that end, with this comment, we include a proposal for your consideration as PDUFA‐VI negotiations move forward. This proposal is designed to help improve and enhance the integration of patient perspective data into medical product development and regulatory decision‐making and was informed through our work to advance the science of patient input, including dialogue with our Benefit‐Risk Advisory Council, consultations with our TRAIN network of venture philanthropy organizations, convenings of key opinion leaders with diverse expertise at our annual Partnering for Cures conference, a Benefit‐Risk Boot Camp, webinars, and active participation on numerous boards, steering committees and advisory councils hosted by other stakeholders including the Alliance for a Stronger FDA, Medical Device Innovation Consortium, Patient‐Centered Outcomes Research Institute, National Health Council, DIA, and the Clinical Trials Transformation Initiative. To facilitate incorporation into PDUFA VI, the language used here is modeled after analogous provisions found in the PDUFA V commitment letter.
Along with the proposal that follows, we highlight the following specific recommendations to further advance the science of patient input.
- To ensure that stakeholders have a common understanding of the many different ways patients can inform the medical product lifecycle, our proposal includes a robust definition of the term “Patient Perspective Data.”
- We believe terminology is important, and encourage consistent use of the term “patient perspective data” to refer to the types of information contemplated here. Specifically, we believe the term “patient perspective data” is favorable to the term “patient experience data,” which is used by CMS to describe a wholly different type of data than what is described in FDA’s October 29th presentation.
- We recognize that this proposal is submitted in connection with PDUFA VI, but we believe optimal impact will be achieved if this approach is applied not just to drugs, but to the development and review of all medical products.
We thank FDA for the opportunity to submit these comments and for opening a public docket for the duration of the PDUFA‐VI negotiations. While the stakeholder meetings are a useful vehicle for facilitating participation from multiple stakeholders, the public docket establishes another important avenue for sharing information which will further enhance transparency and help elevate the voice of key stakeholders who do not have a place at the negotiating table. 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 and we welcome the opportunity to explore these comments in greater detail with agency staff.