FDA report examines patient experience data in drug development and regulatory review

A new FDA assessment has examined how patient experience data are used across drug development and regulatory review, including their role in trial design, benefit-risk assessment, and product labeling.

The Baseline

• A new FDA assessment examines how patient experience data are used throughout drug development and regulatory review.
• Patient experience data were referenced in almost two-thirds of the 800 regulatory reviews examined, with use increasing over the assessment period.
• The report highlights early sponsor engagement, clear documentation, and appropriate methodological expertise as important for supporting the use of patient experience data.

The US FDA has released a new report outlining the agency’s use of patient experience data (PED) in drug development and regulatory decision-making. The report, Assessment of the Use of Patient Experience Data in Regulatory Decision-Making, is the second of three independent assessments required under the 21st Century Cures Act. Conducted by Booz Allen Hamilton, it summarizes focus group interviews with FDA review staff on how patient experience data are considered in drug development and regulatory review, alongside an examination of reviewer-generated documents and approved product labeling for 800 NDA, BLA, and efficacy supplement submissions approved from February 5, 2021, to December 31, 2025.

FDA defines PED as information intended to describe patients’ experiences with a disease or condition, including its “physical and psychosocial impacts” on their lives and their “preferences with respect to treatment.” FDA describes PED as an “integral component of the drug development and review process,” with the assessment finding that these data can inform multiple stages, from early interactions between FDA and sponsors to the review of new drug applications (NDAs), biologics license applications (BLAs), and efficacy supplements.

During the investigational new drug phase, PED were reported to inform four main areas:

1. Endpoint selection
2. Clinical trial design
3. Clinical outcome assessment (COA) selection and development
4. Considerations for the planned benefit-risk assessment

Reviewers also described drawing on Patient-Focused Drug Development meetings, patient listening sessions, Voice of the Patient reports, natural history studies, and peer-reviewed literature for additional context.

Early engagement emerged as a recurring theme. The report states that reviewers “universally mentioned the importance of early sponsor engagement” before clinical trials begin and encouraged adherence to FDA’s Patient-Focused Drug Development guidance series. Sponsors were also encouraged to clearly identify submitted PED, its location, and intended purpose within the Reviewer’s Guide to reduce the risk that relevant information is overlooked.

The document review found that PED were referenced in almost two-thirds of the 800 regulatory reviews examined, with references becoming more frequent over the assessment period. Among original new molecular entity (NME) submissions, references became more frequent over the assessment period, reaching 85% of reviews in 2025. Mentions of PED were particularly common in reviews of products with orphan drug designation, at 91.5%. FDA reviewers suggested this may reflect the importance of patient input in rare diseases, where information on natural history, endpoints, and disease burden can be limited.

Patient-reported outcomes were the most frequently documented type of COA, appearing in around 80% of original NME reviews that referenced PED. PED were also reflected in product labeling: approximately 40% of approved NME labels associated with reviews mentioning PED referenced this information, compared with 30% in the 2021 assessment.

The assessment also identified continuing challenges, including inconsistent methodological quality, underrepresentation of some patient groups, uncertainty over which review disciplines should assess particular data types, and workload constraints. Reviewers cited issues including “unclear saturation methods, poor interview design” and insufficient statistical planning in some submissions. The report highlighted early sponsor engagement, clearer documentation of PED within submissions, and access to appropriate multidisciplinary expertise as important considerations for supporting their use in drug development and regulatory review.