Praise for FDA Patient Experience Draft Guidance Draft
Stakeholders are praising FDA for its draft guidance on developing and submitting proposed draft guidance relating to patient experience data. The Biotechnology Innovation Organization (BIO) says it believes that “strong trilateral communication between FDA, patients and patient organizations, and sponsors around patient experience will result in incorporation of patient experience data throughout the product lifecycle, including drug development and review.” The association asks that the agency clearly outline a process by which it will communicate and provide feedback to stakeholders that submit draft guidance or other patient experience data. It also asks FDA to communicate how proposed draft guidance submitted by external stakeholders is leveraged to influence thinking related to development of draft guidance by FDA.
The National Organization for Rare Disorders (NORD) comments that several patient communities within the broader rare disease patient community have already submitted proposed draft guidance to FDA and “have found the process and final product to be valuable.” NORD says it is optimistic that the agency draft guidance will lead to an increase in submission of proposed guidances by other rare disease communities and ultimately accelerate therapeutic development for patients.
The Amyotrophic Lateral Sclerosis Association notes the “value of bringing together a diverse group of stakeholders, namely, patients, caregivers, industry and government representatives, academics, and patient advocacy organizations to develop the content necessary to produce an externally-led draft drug development guidance. It was our experience that the rich conversations and learnings regarding the state of the science and patient experience in areas such as natural history, clinical trial design, biomarkers, outcome measures, benefit-risk assessment, and others had broad impact on the field well beyond the draft guidance itself.”
The Lupus Foundation of America asks FDA to clarify that patient groups and other stakeholders have other opportunities to provide feedback once a public docket is closed. It also urges the agency to provide more clear opportunities for ongoing engagement and feedback when a patient organization is developing a proposed draft guidance. “This will help ensure that the content that patient groups ultimately submit to the agency is useful to the agency and could potentially be used by FDA to publish their own draft guidance,” the foundation says.
Finally, the National Center for Health Research says FDA should provide additional information on how and under what conditions it would use proposed draft guidances to inform regulatory decisions. “Any metrics that FDA may use to determine the usefulness of a proposed guidance can also be used to develop better guidances,” it says. “It should also note the extent to which certain types of stakeholders, data sources, or topics are prioritized.”