FDA Draft Guidance on Patient Experience Data Incomplete, Critics Argue

Regulatory NewsRegulatory News | 25 March 2019 |  By 

Industry group BIO, the National Association for Rare Disorders (NORD) and the National Center for Health Research (NCHR) called for clarification on how regulators will use draft guidances proposed by external stakeholders on patient experience data.

The comments on the US Food and Drug Administration (FDA) draft guidance from December 2018 on developing and submitting proposed draft guidances related to patient experience data showed some consensus around including further regulatory clarifications on various aspects of the framework, particularly as it relates to FDA’s use of proposed draft guidances.

Commenters appreciated the framework set forth via FDA’s draft guidance and supported the agency’s efforts to increase the use of data on patients’ perspectives and experiences in drug development and regulation. But they cautioned that the framework is a complex undertaking due to a number of factors, including financial ties between patient groups and industry, a wide range of patient needs, proficiency and preferences, as well as resource-intense draft guidance development.

“As a first step, FDA should provide additional information on how and under what conditions it would use proposed draft guidances to inform regulatory decisions,” argued NCHR. “Transparency in how 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.”

BIO and NORD echoed NCHR’s request to the agency.

NORD vice president of regulatory and government affairs Rachel Sher argued the opportunity to propose draft guidances “may be more appealing if FDA were to offer examples of how FDA can use the information submitted by patient communities and how this information has been used historically."

In the event of an FDA decision to only use a portion of a proposed draft guidance, BIO director of science and regulatory affairs Danielle Friend cited the time and resources needed for an external stakeholder to develop a draft guidance, saying “it would be helpful for FDA to share the rationale as to why only portions of the submitted draft guidance will be supported by the agency.”

BIO also argued that additional clarifications are needed in the agency’s draft guidance to further support strong communication between FDA staff, patients and patient organizations and sponsors.

Clarifications around mechanisms to receive FDA feedback, comment on and update or withdraw proposed draft guidances should be included in the guidance as well, according to all three commenters. 

Further, NCHR cautioned on the potential for financial interests to manipulate patient experience data.

“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,” NCHR noted. “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.” NCHR argued a proposed draft guidance should include a description of its development, including data source and breadth, and conflicts of interest to address this concern.



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