Regulatory Focus™ > News Articles > 2021 > 6 > FDA releases first Cures Act report on patient experience data in regulatory decision-making

Regulatory NewsRegulatory News
Posted 30 June 2021 | By Jeff Craven 

FDA releases first Cures Act report on patient experience data in regulatory decision-making

2819 The US Food and Drug Administration (FDA) has published its first report on how it uses patient experience data in regulatory decision-making.
As part of the 21st Century Cures Act of 2016, FDA is required to publish patient data assessment reports in 2021, 2026, and 2031. The agency contracted Eastern Research Group, Inc. to conduct the assessment and publish the report for 2021.
When assessing how FDA uses the experience of patients in their decision-making, Eastern Research Group (ERG) said the “primary mandate” is making sure the product’s benefits outweigh its risks, and that the product itself is based on “sound science.” Patient-reported outcomes (PROs) and clinical outcome assessments (COAs) are the most common uses of patient experience data, which are used as “secondary or exploratory endpoints.” However, when FDA decisions involve PROs and COAs as primary endpoints, this data “tend to play a central role,” according to the report.
Patient-focused drug development (PFDD) is an area where “variability in FDA’s use of patient experience data likely reflects the wide range of diseases it regulates, and the state of the science and evolution of PFDD,” the report stated. In these situations, patient experience data is used as supporting information, rather than proof of efficacy. “Many interviewees observed that industry and FDA are in the middle of a learning curve—where many COAs have been established, and at the same time the science has not yet caught up with the need and desire for more and better integration of patient input in product development and review,” ERG noted in the report.
While stakeholders such as FDA staff and reviewers have said they see use of patient experience data as evolving, other stakeholders such as applicants, patients, clinicians, caregivers, and advocacy and/or research organization representatives “do not know how FDA uses patient experience data in regulatory decision-making,” according to the report. These stakeholders, however, acknowledged the agency is taking steps to communicate how it uses patient experience data and expressed an interest in understanding how FDA uses this data conceptually and for specific products.  
Areas for improvement
ERG also investigated how FDA might improve the way it uses patient experience data. Communication, developing a data analysis plan, clinical trial design, consultation with COA experts, sharing of data with patients, and framing the current report were all suggestions offered by FDA staff, applicants, and other stakeholders.
“When pursuing a drug/biologic development program, consult FDA guidance, other PFDD resources, and FDA staff early and often to discuss the potential value of patient experience data, types of data to develop, fit-for-purpose tools to use, approaches to collecting complete data, and a data analysis plan,” ERG recommended in the report.
These groups also identified opportunities to collaborate in all-stakeholder meetings, development of PFDD tools, endpoints, outcome measures, use of patient data in application reviews, and setting expectations for diversity of patients in PFDD as other areas for potential improvement.
“In collecting and reviewing patient experience data, some applicants and FDA staff focus on endpoints that are easily measured or of primary interest to clinicians,” whereas “[p]atients would like greater attention to psychosocial, quality of life, and measures of ability to function,” according to the report.
ERG recommended FDA consult with different patient organizations to “find diverse perspectives” and collect data that “ensures its representativeness across the full diversity of the patient population.”
FDA Assessment of Use of Patient Experience Data in Regulatory Decision-Making


© 2022 Regulatory Affairs Professionals Society.

Discover more of what matters to you