FasterCures comments on FDA's Patient Preference Information
Divisions of Dockets Management (HFA-305)
Food and Drug Administration
5630 Fishers Lane, Room 1061
Rockville, Maryland 20852
RE: Federal Register Notice FDA-2015-D-1580
Patient Preference Information – Submission, Review in PMAs, HDE Applications, and De Novo Requests, and Inclusion in Device Labeling; Draft Guidance for Industry, FDA Staff, and Other Stakeholders
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-2015-D-1580, Patient Preference Information – Submission, Review in PMAs, HDE Applications, and De Novo Requests, and Inclusion in Device Labeling; Draft Guidance for Industry, FDA Staff, and Other Stakeholders (“Guidance”). These comments have been informed through our ongoing efforts to advance the Science of Patient Input, in collaboration with stakeholders from across the biomedical ecosystem.
We commend FDA for its initiatives to include patient perspectives in regulatory decision-making, from the activities outlined in the Food and Drug Administration Safety and Innovation Act (FDASIA), to the 2013 Public Workshop hosted by the Center for Devices and Radiologic Health (CDRH) on “The Patient Preference Initiative: Incorporating Patient Preference Information into the Medical Device Regulatory Processes,” to this draft Guidance issued by CDRH and the Center for Biologics Evaluation and Research (CBER). We applaud FDA for its commitment to developing “a systematic way to help to ensure that patients are represented and patient perspectives are considered in the regulatory decision-making process.” (Guidance, pg. 4).
Our comments on this Guidance are designed to highlight areas we believe represent a significant step forward in FDA’s effort to deepen and expand its engagement with patients, patient advocates and family caregivers. We also take this opportunity to identify some specific areas we think could benefit from more guidance and clarity from FDA.
Philosophy of Engagement with Patients
As a regulatory agency subject to stringent statutory requirements that define its interface with the public, FDA’s engagement with patients will be subject to some unique restrictions. In previous submissions, we have outlined four guiding principles that we believe could be applied to patient engagement activities across the agency, to serve as both a compass and a scorecard for current and future initiatives. As explained in our December 2014 comments on “Patient Participation in Medical Product Discussions,” we believe that at minimum FDA’s patient engagement activities should be:
- Purposeful – Engagement should be designed and executed with the intent to inform the agency’s mission, strategy, and operations, including policy and regulatory decisions. It should be valued by staff at all levels of the agency as integral to their role in protecting and promoting public health.
- Reciprocal – Whenever possible, engagement should be predicated on fostering a two-way information exchange. This can occur without violating privacy or confidentiality boundaries that safeguard participants.
- Dynamic – Much of FDA’s interaction with patients and patient communities is currently episodic or cross-sectional, limited to annual forums or single events. Patient needs within a community and across communities change in response to scientific and technologic advances and other circumstances. Engagement activities should seek to build ongoing relationships.
- Transparent – In addition to required reporting on patient engagement activities through vehicles such as publicly available meeting agendas and minutes, the outcomes of patient engagement should be visible to the community, particularly when they affect or influence decisions or policy.
We believe this Guidance represents an important step towards enhancing the science of patient input by defining important terms and clarifying agency views on approaches to collecting patient preference information and indeed reflects many of the guiding principles outlined above. We recognize the synergistic effect of multiple initiatives aimed at better defining patient-centricity and the successful practices that support it throughout the development of both medical devices and drug products. For example, Sec. 2001 of H.R. 6 focuses on Patient Focused Drug Development. We believe many of the concepts in this draft guidance will be useful to advancing the process outlined in H.R. 6 for developing a guidance for collecting “patient experience data” to enhance FDA’s structured benefit-risk assessment framework.
CDRH’s active collaboration with and funding support of the Medical Device Innovation Consortium to develop a “Framework for Incorporating Information on Patient Preferences Regarding Benefit and Risk into Regulatory Assessments of Medical Technology,” represents another significant development in this space. Although this Guidance and the MDIC framework explicitly apply to medical devices, we think the principles set forth have potential for widespread application beyond the immediate intended audience. Indeed, the MDIC framework was referenced in BIO’s June 2015 white paper entitled, “A Lifecycle Approach to FDA’s Structured Benefit-Risk Assessment Framework,” suggesting that stakeholders outside the device industry recognize the potential of these outputs in the context of drug development.
Defining Patient Preferences
- We commend FDA for introducing more clarity to this space by providing a definition of “patient preference” information. However, we echo the sentiment of other stakeholders, that patient preference information is just one element of the overall patient perspective. Other patient perspective information includes, for example: symptoms experienced, burden of managing or living with a disease, strengths and weaknesses of currently available therapeutic options, unmet medical needs, and disease natural history. FDA should continue to work with stakeholders to identify and collect information reflecting all aspects of the patient experience.
- Further, we applaud FDA for helping sponsors identify what constitutes a “preference-sensitive” scenario and making clear that patient preference information “may not be relevant or appropriate for all device types.” (Guidance, pg. 3).
Integrating Patient Perspectives throughout the Lifecycle
- Enhancing the science of patient input will require meaningful integration of patient perspectives throughout the product development lifecycle. We applaud the agency for expressly acknowledging that both FDA and sponsors may consider patient preference information throughout the total product lifecycle, and not just during review of PMAs, HDE applications and de novo requests. (Guidance, pg. 10).
- Moreover, we commend FDA for highlighting the importance of communicating patient preference information in labeling. Such transparency is critical to empowering patients and caregivers to make informed decisions while also helping to clarify how the agency is using and evaluating such information.
- Patient preference information can be of huge value to Advisory Committee discussions, as it can help inform the benefit-risk profile of a particular product. We appreciate the example provided on page 23 illustrating the benefit of preference information to an advisory committee. FDA might consider clarifying in the final guidance whether such information, when available, will be routinely incorporated into Advisory Committee briefing documents and discussions.
Outlining Qualities of Patient Preference Studies
- We agree with FDA that the “Qualities of Patient Preference Studies” identified in this Guidance are important considerations when evaluating the scientific validity of patient preference information. Further, we applaud FDA for taking the initiative to outline standards and offer clarity around how FDA evaluates the quality of such studies. In particular, we are pleased to see FDA identify patient-centeredness as a measure of quality and echo FDA’s sentiment that “patient preference studies should ensure that the patient, not the health care professional, is the central part of the study.” (Guidance, pg. 13).
- We share FDA’s perspective that it is too early to carve out absolutes in this area and we encourage CDRH and FDA to continue to engage with stakeholders in exchanging information and providing feedback to ensure high quality submissions.
Approving Products for a Subset of Patients
- We strongly support FDA’s recognition that “patient preference studies may help FDA identify a subset of patients in whom the benefits outweigh the risks, and the approval would not be for the general population but instead would be limited to the population where FDA determines the benefits outweigh the risks.” (Guidance, pg. 17). Such an approach, coupled with appropriate conditions of approval, can facilitate access to promising treatments for patient who need them most.
Clarifying Opportunities for Non-Product Sponsors
- We encourage FDA to specifically outline a clear mechanism for FDA to receive and consider patient preference information from entities who are not directly affiliated with a particular product or sponsor. Such submissions appear to be contemplated by this Guidance as demonstrated by the reference to a patient group who “submits patient preference information from a study of parents of patients.” (Guidance, pg. 23). However, we respectfully request that FDA provide more clarity around how this information would be submitted and reviewed, including identifying a point of contact within the agency, to encourage the collection of patient preference information from non-product sponsors.
We thank FDA for the opportunity to submit these comments and encourage FDA to finalize and implement this draft document. 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.