NCHR Comment on Proposed Draft Guidance Relating to Patient Experience Data

National Center for Health Research; March 21, 2019

National Center for Health Research’s Public Comment on
Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data; Draft Guidance for Industry and Other Stakeholders; Availability

Thank you for the opportunity to comment on the draft guidance “Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data.”  We support the efforts of the Food and Drug Administration (FDA) to consider and encourage the use of patients’ perspectives and experiences in the development and regulation of medical products.  However, this is a complex undertaking stakeholders involved; the financial ties of some patients and many patient organizations to industry; and the range of patients’ needs, priorities, knowledge, and preferences.

Transparency is necessary but not sufficient to ensure that the inclusion of patient experience data is not manipulated by financial interests.

As a first step, the FDA should provide additional information on how and under what conditions it would use proposed draft guidances to inform regulatory decisions.  Any metrics that the FDA may use to determine the usefulness of a proposed guidance can also be used to develop better guidances. It should also note the extent to which certain types of stakeholders, data sources, or topics are prioritized.

Subsequently, FDA should cite proposed guidances when they are used in regulatory decisions.  This improves understanding of FDA decisions and informs future proposed guidances.

Proposed draft guidances should clearly describe the people and/or groups involved in their development.  This should include scrutiny and public acknowledgement of any conflict of interests, financial or otherwise, including the source(s) of funding for development of the guidance and funding provided to the individual(s)/group(s) involved, regardless of whether the funding is specifically for guidance development.

An inherent bias that will arise in this process is that industry funds and trains many patient groups and recruits many patient advocates.  Therefore, most patient advocates have ties to industry and views that align with industry, such as speeding up the FDA review process and lowering safety criteria.  This could lead to a potentially dangerous shift in the quality of medical products if endpoints and levels of effect or safety are modified because industry has persuaded patients to support those changes and not because they are consistent with most patients’ preferences.

Contrast this with patients who have been harmed by medical products.  These patients have repeatedly informed FDA Advisory Committees, FDA staff, and our think tank that they feel that their concerns do not receive equal respect from the FDA.  They feel that their experiences are treated as anecdotes, while patients urging faster FDA approval and fewer safeguards are treated as important sources of information.

The proposed draft guidances should describe how the guidance was developed.  This should include the methods used to develop the guidance, including the source and breadth of the data and the extent of patient input.  Information on guidance development and conflicts of interest are necessary to determine the extent to which the document is generalizable to all patients with the condition and any inherent biases.  This will especially be important as information about the condition and treatments evolve.

It is unclear from the draft guidance if there will be mechanisms in place to comment on proposed draft guidances.  These processes could improve FDA’s understanding of the context of a proposed draft guidance, including its generalizability and biases.  It is also unclear if there will be a mechanism for updating or withdrawing a guidance based on such comments or new information or treatments.

Patient perspectives and experience are important considerations for medical product development and regulation.  Patients, caregivers, and patient and consumer groups can provide a valuable resource for product developers and the FDA.  Transparency in how the FDA evaluates and uses proposed draft guidances as well as who and how they are developed will be important if they are to provide meaningful and accurate information about patients.

NCHR can be reached through Stephanie Fox-Rawlings, PhD at