Public comments


FasterCures Comments on the Proposal to Create an Office of Patient Affairs at FDA

June 12, 2017

Divisions of Dockets Management (HFA-305)                                                               Submitted electronically
Food and Drug Administration
5630 Fishers Lane, Room 1061
Rockville, Maryland 20852

RE: Federal Register Notice FDA-2017-N-0455
Enhancing Patient Engagement Efforts Across FDA; Establishment of a Public Docket; 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 in response to Federal Register Notice FDA-2017-N-0455, Enhancing Patient Engagement Efforts Across FDA; Establishment of a Public Docket; Request for Comments. This letter was prepared and authored by FasterCures staff, and was informed by discussions with patient organizations, representatives of life sciences companies, and other stakeholders.

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 foster more frequent and effective partnerships with patients in the biomedical research enterprise. To that end, through our Patients Count program, we are focused on creating a science of patient input and expanding opportunities for patients’ perspectives to shape the processes by which new therapies are discovered, developed, and delivered.

FasterCures has long been supportive of efforts at the U.S. Food and Drug Administration (FDA) to enhance patient engagement. For example, we outlined a series of targeted recommendations in our December 2014 “Comments on FDA Activities for Patient Participation in Medical Product Discussions,” and actively participated in stakeholder meetings for the Prescription Drug User Fee Act (PDUFA) and Medical Device User Fee Amendments (MDUFA), where we advocated for the inclusion of commitments designed to advance the science of patient input. (See examples of our comments here, here, and here). We applaud the agency for its ongoing efforts to “enhance mechanisms for patient engagement at FDA.” However, we are concerned that this proposal to establish an Office of Patient Affairs (OPA) may have the opposite of its intended effect. Without more clarity from FDA as to how this Office will be resourced, how it will interact with FDA’s medical product review centers, and how it will influence existing patient engagement efforts, FasterCures is not able to offer its support for the establishment of this Office at this time.

We stand ready to work with FDA and other stakeholders to satisfy the goals articulated in the Federal Register Notice through existing structures and offices at FDA, or to refine the structure and objectives of the Office of Patient Affairs such that its mission and purpose are appropriately tailored to address those needs. 

We describe our concerns in more detail below.

I. The establishment of a separate Office of Patient Affairs could negatively impact existing patient engagement efforts.

Given the tremendous progress FDA has made in recent years to advance the science of patient input, it is critical that we avoid creating mechanisms that could impede or set back these advancements. There are a number of initiatives underway at FDA designed to enhance patient engagement, including:

  • Patient Preference Initiative at the Center for Devices and Radiological Health (CDRH): Launched in 2013, this initiative aims to “develop a systematic way of eliciting, measuring, and incorporating patient preference information” into the Total Product Lifecycle. Last fall, CDRH issued final guidance to address the incorporation of patient preference information in premarket submissions.
  • CDRH Patient Engagement Advisory Council: Announced in the fall of 2015, this Council will work across products and disease areas to advise FDA senior leadership on complex issues relating to medical devices, their regulation, and their use by patients.  This is just one component of CDRH’s broad strategic goal to more effectively “Partner with Patients” in 2016-2017.
  • Patient-Focused Drug Development (PFDD) initiative: The Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Review have been committed to enhancing patient engagement through the PFDD program. This initiative, established under PDUFA V, enables reviewers to hear directly from patients and their caregivers about their diseases, treatment options, and unmet needs. Guidelines for FDA participation in externally-led PFDD meetings have served to expand the scope of this initiative beyond the 24 federally supported meetings.
  • Drug Trials Snapshots Program: CDER launched this program in 2015 to provide patients and families with easily accessible information about who participated in the clinical trials that supported FDA approval of new drugs. This initiative has been another example of FDA’s efforts to engage with patient and consumers in new ways.

These and other patient engagement efforts have evolved and progressed in ways relevant to the specific medical products reviewed and monitored by each center. This might not be possible if all patient engagement activities were funneled through a centralized Office of Patient Affairs. Moreover, in this early stage of emerging practices in patient engagement, the varied and complementary nature of activities undertaken by the medical product review centers is useful and additive to the field. We’re concerned a centralized office could blunt existing creativity in an effort to standardize practice.

The negotiation process for both PDUFA VI and MDUFA IV also yielded strong, targeted commitments from FDA and industry to advance patient input and involvement in the regulatory process within both CDER and CDRH. For example, FDA has committed to issue new guidance documents clarifying how patient data can be collected and how they can inform drug development, while also committing to undertake activities designed to improve the regulatory predictability and impact of patient-reported outcomes. In addition, both the MDUFA IV and PDUFA VI letters contain commitments to enhance staff capacity to facilitate the use and development of patient-focused data in regulatory decision-making. It is important that those activities move forward without the potential for confused lines of authority that the establishment of an Office of Patient Affairs could cause.

Finally, many patients and patient representatives already have strong relationships with FDA staff within specific review divisions and other offices. We are concerned that the Office of Patient Affairs could create new administrative hurdles, or otherwise serve as a barrier to continued interaction between these entities, hampering existing efforts. Better visibility of the existing Office of Health and Constitute Affairs on FDA’s website and stronger external engagement across the board could help to direct inquiries of patients who are new to the agency and facilitate better access with the agency.

II. Establishing the Office of Patient Affairs could isolate patient engagement efforts from medical product review centers.

While we applaud the agency’s effort to “improve coordination and support for patient engagement across medical product centers,” we are concerned that the establishment of the Office of Patient Affairs could actually serve to disconnect patient engagement efforts from the medical product review process. The Federal Register Notice announcing this proposal indicates that the Office will be “directly accountable” to the medical product review centers; however, there is no detail about how this office will interact with the review divisions.

The entire agency can benefit from interaction with patients and incorporating patient-centric methodologies into their work. Therefore, these interactions should be integrated throughout the organization – just as biostatistics and genomics are – not restricted to one individual office that may operate in isolation from the “on the ground” agency staff who are the technical experts tasked with writing guidance documents, developing regulatory science, and reviewing medical product applications.

III. The Office of Patient Affairs could divert resources away from ongoing patient engagement efforts in an environment where financial resources and human capital are already scarce.

There is certainly more that can be done to ensure patients, especially individual patients who are not affiliated with a patient organization, better understand how to navigate and interact with FDA. Given FDA’s considerable funding and human resource limitations (within the past year, just one Center had more than 700 openings), we are concerned about ensuring sufficient personnel and budgetary resources for this new office to operate effectively. We suggest instead working within the existing framework of the Patient Liaison Program in the Office of Health and Constituent Affairs to enhance existing resources or develop new tools to assist patients and patient groups seeking to engage with the agency. 

Additionally, the innovative regulatory science activities needed to advance patient-centricity, such as the development of key guidance documents directed at advancing the science of patient input, require the medical and technical expertise that currently resides in the review centers. As explained above, it is not clear how the Office of Patient Affairs will interact with the medical product review centers, and we are concerned that these experts may not be readily accessible by this Office.

Finally, while not explicitly identified as an expected responsibility in the Federal Register Notice, by establishing itself as the “single, central entry point” to FDA for the patient community, this Office could become responsible for fielding inquiries related to single-patient expanded access requests. FDA fields over 1,000 formal requests for all types of expanded access per year, many of which are of an urgent nature, requiring an immediate response. Accordingly, without sufficient resources and personnel, handling inquiries related to these requests could crowd out other activities and responsibilities, heightening many of the concerns we’ve outlined above.

FasterCures is committed to partnering with FDA and other stakeholders to advance the science of patient input and ensure that the patient perspective is reflected in medical product development and regulatory decision-making. We appreciate FDA’s continued efforts to think creatively and proactively about how to do this most effectively. Indeed, we do think there could be significant benefit to developing better mechanisms to help patients and patient advocates navigate the agency and identify appropriate contacts for particular issues as well as establishing a central agency repository for contact information and best practices. However, we urge FDA to provide more detail about how this Office would accept and respond to requests, how it will be resourced to do so, and what role it will play with respect to existing programs, offices, and initiatives at FDA before establishing such an Office.

We welcome the opportunity to explore these comments in greater detail with agency staff.


Maureen Japha, JD
Director, Regulatory Policy
FasterCures, a Center of the Milken Institute

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