Sponsors Share Perspectives on Patient Experience Data at FDA’s Request
The collection, use and submission of patient experience data (PED) still requires significantly more FDA guidance, clarification and support, big-name sponsors told the agency in response to a request for information and comments on the subject.
The FDA presented five talking points in its request, issued May 2:
- Describe any perceived barriers to the use of PED for regulatory decision-making (e.g., benefit-risk assessment, product labeling);
- Describe any challenges and limitations experienced when selecting, modifying or developing fit-for-purpose clinical outcome assessment (COA) measures;
- Describe any challenges and statistical analysis considerations when constructing and selecting endpoints of interest and in understanding whether an estimated treatment effect corresponds to a real difference in patients’ lives;
- Describe any challenges and limitations experienced when developing and conducting patient preference studies to support regulatory submissions; and
- Describe any challenges and limitations when submitting PED to the FDA.
The FDA received nine responses from sponsors, nonprofits and Pharmaceutical Research and Manufacturers of America (PhRMA). AbbVie, Boehringer Ingelheim and GSK, for example, believe the FDA should offer more direction on the topic of PED, even at basic levels, and engage sponsors more in this area.
Boehringer Ingelheim feels the definition of PED itself is currently murky and advises the FDA to come up with a clear definition outlining what it is and isn’t and how the agency uses it to inform its decisions. Additionally, the sponsor pointed out the lack of a PED submission template and protocol development time constraints that make developing and adapting fit-for-purpose COAs a challenge, especially when modifications to measures must be made.
AbbVie believes there are significant hurdles for sponsors to clear within their own organizations, with one of the greatest being the investment of adequate time and resources in identifying and generating these data in a way that meshes with product development timelines. This is even more of a barrier when the value of PED is not clear across a company and to outside stakeholders, and the FDA can help build support in this area by showcasing real-life examples of its impact, the drugmaker said.
“We recommend … a future workshop to discuss examples or case studies where PED was an influential part of the risk-benefit decision-making process, highlighting applications across various types of PED, and share how PED was considered in the context of the full evidence package,” AbbVie said in its response. “If the industry can point to examples where PED was just as, if not more, influential than more traditional endpoints for demonstrating efficacy and safety, this could enhance advocacy for additional investment in this type of research.”
In addition, examples of times where PED may have made a difference in risk-benefit decision-making but was inadequate in quality or completely lacking would also be helpful, AbbVie said.
GSK expressed similar sentiments and stressed the need for greater agency clarity on PED’s utilization in benefit-risk assessments and clarity on what PED may be incorporated into approved product labeling. The lack of guidance on labeling makes planning for PED studies particularly difficult and limits sponsors’ efforts to identify “where there may be flexibilities to incorporate such data,” GSK says.
More guidance on the level and quality of evidence the agency would consider for incorporating PED into product labels, and insight on where this data might go on the label, would be of great benefit to sponsors, as would direction on things sponsors should be considering to be able to include qualitative patient data on labels, GSK said.
“A particular challenge for including PED in labeling is that such information does not always fit squarely within the existing required format for FDA-approved labeling,” PhRMA said in its comments. “Further discussion on where, and how, information relating to PED may be included in FDA-approved labeling would be beneficial and could help further advance the use of PED in drug development,” it said. “In addition, it is important that FDA staff across review divisions are equally familiar with the role and importance of PED in FDA-approved labeling and in drug development more broadly.”
The FDA says the comments received will inform a pair of PED workshops, one to be held no later than the end of fiscal 2024 and the other by the end of fiscal 2025.
Read the comments here.