Suggestions for Patient-Focused Drug Guidances
Pfizer and several other stakeholders are asking FDA to provide more detail on how it will incorporate patient experience into regulatory decisions such as benefit/risk considerations. “For example,” Pfizer says in a comment letter on an agency patient-focused drug development (PFDD) discussion document, “it would be helpful to know what level of evidence would be required to incorporate patient experience information into a label, as opposed to, for example, using patient perceptions and experience to inform procedures in a clinical trial protocol. In addition, we suggest the agency indicate where in a label the patient experience would be captured, if the information were to be incorporated into a label.”
Pharmaceutical Research and Manufacturers of America (PhRMA) recommends that FDA continue to prepare discussion documents in advance of upcoming PFDD workshops as it facilitates meaningful discussion and participation by all attendees. PhRMA also says that while it agrees with FDA that PFDD-related engagement with the agency is imperative and that FDA staff must be available to meet and provide input to sponsors in the drug development process, it has become challenging for sponsors to obtain PDUFA milestone meetings with FDA and sponsors have seen a notable increase in written-response-only to meeting requests.
In its response, the Arthritis Foundation recommends that FDA adopt a culture of “co-production,” working closely with patients and patient advocacy organizations from the ideation of the work to completion. It also recommends that FDA be explicit in its definitions, guidances, and actions. “Robust patient engagement depends on access to and use of robust, high quality patient-generated data in addition to meaningful partnership with individual patients and patient advocacy groups,” it says.
Hyman, Phelps and McNamara, which represents over 20 patient advocacy organizations, recommends that FDA (1) develop pathways for submitting patient experience data to FDA and engaging the agency; (2) provide greater transparency for using patient experience data for regulatory purposes; (3) describe various research goals and objectives most informative to regulatory decision-making; and (4) ensure opportunities for in-person engagement between patients and FDA officials.
Areas that the National Organization for Rare Disorders says should be expounded upon include (1) extensive guidance to patient organizations on what data collection efforts they should undertake and when; (2) awareness of the financial and staff limitations of rare disease patient organizations; and (3) additional guidance on sampling in rare diseases. The comment letter also includes additional considerations for the agency.
Janssen says the discussion guide for the first draft guidance is “helpful in terms of outlining many approaches to bridge from early discussions to tangible data.” The company also says the benefit to sponsors or other stakeholders in collecting patient experience data is missing from the discussion document, as is clarity on how the information will be used by the agency. And it says it is not clear what the process and procedure for submission, review, and discussion of patient experience data with the agency is expected to be.
Finally, FasterCures says it sees great value in the discussion document as it provides a shared vernacular for PFDD that has been lacking. “We also appreciate it as a strong signal that FDA is moving towards greater acceptance and use of patient experience data to inform regulatory decision-making and an acknowledgment that further technical guidance on how to collect these data is needed. And it makes specific comments for improving the document.