Based on the provided FDA datasets, patient-reported outcomes (PROs) are frequently collected and submitted as part of drug applications to CDER, but their inclusion in the official prescribing information (PI) is uncommon and subject to strict criteria. The decision to include PRO data in the PI depends on the adequacy of the trial design, the quality and relevance of the PRO instruments, and the robustness of the data.

Key findings from the sources:

1. Guidance and Review Practice:

   • The FDA explicitly states that inclusion of PRO data in product labeling (i.e., the PI) depends on several factors, including the adequacy of the submitted data, the strengths and limitations of the instrument, and the design and conduct of the trial. Exploratory PRO findings are generally considered descriptive and are unlikely to support inclusion in product labeling unless they meet high evidentiary standards ^14295568.
   • For example, the FDA notes: "Inclusion of PRO data in the product label will depend on the adequacy of the design and conduct of the trial, the strengths and limitations of the instrument within the given context of use, and the quality of submitted data" ²⁹.

2. Recent Drug Approvals and PRO Data:

   • Many recent FDA reviews (2020–2024) document the collection and analysis of PRO data as secondary or exploratory endpoints. However, these reviews often state that PRO data were not included in the PI due to limitations such as lack of a control arm, insufficient statistical analysis, or the exploratory nature of the endpoints.
   • Examples where PROs were NOT included in the PI:
     • ABECMA (idecabtagene vicleucel): "The Applicant did not seek a labeling claim based on clinical outcomes assessment (COA) data and these data were not incorporated in the PI" ⁹.
     • ENJAYMO (sutimlimab-jome): "Given this was a single arm study with no comparator, the PRO endpoints were not included in the USPI" ⁵⁷.
     • AYVAKIT (avapritinib): "Efficacy claims cannot be made in the PRO outcomes... Any PRO results are only exploratory" ⁴⁴.
     • REZDIFFRA (resmetirom): "The Applicant did not seek labeling based on any of the collected patient experience data" ³⁴.
     • KISQALI (ribociclib): "The PRO results from this study cannot support any efficacy claims as PRO assessment was sparse, and there was no plan for formal testing of any PRO endpoints" ²⁵.
     • CARVYKTI (ciltacabtagene autoleucel): "The PRO data are considered exploratory as they have not been statistically evaluated according to the prespecified hierarchical testing strategy" ⁸⁵.

3. Rare Examples of Inclusion:

   • There are rare instances where descriptive PRO data have been included in the PI, typically in the ADVERSE REACTIONS section to provide additional detail about the nature, frequency, and severity of certain adverse events as reported by patients. For example, the FDA guidance notes: "Exploratory PRO results further describing the timing, frequency, and impact of visual disturbances were included in the ADVERSE REACTIONS section of labeling for XALKORI, to provide additional detail about the nature, frequency, and severity of vision disorders reported by clinicians" ¹⁴.
   • However, these inclusions are exceptions and are generally limited to descriptive information about adverse events rather than claims of efficacy or benefit.

4. Summary Table and Review Templates:

   • Many review templates and clinical reviews list PROs as part of the data submitted or considered, but do not indicate inclusion in the PI ^{61130485967697173}.
   • The standard language in these reviews often distinguishes between data submitted and data included in labeling.

Conclusion:

• PROs are routinely collected and reviewed by CDER, but their inclusion in the prescribing information (PI) is rare and typically limited to descriptive information about adverse events, not efficacy claims.
• In the last five years, there are no clear examples in the provided datasets of new drugs where PROs were included in the PI as efficacy claims. When included, it is usually in the context of adverse reactions and only when the data are robust and well-justified ^14295568.
• Most applications with PRO data do not result in PI inclusion, as documented in multiple recent reviews ^92534445785.

References:

• ⁹ ABECMA idecabtagene vicleucel
• ¹⁴ Core Patient-Reported Outcomes in Cancer Clinical Trials: Guidance for Industry
• ²⁵ KISQALI KISQALI FEMARA CO-PACK (COPACKAGED) LETROZOLE; RIBOCICLIB SUCCINATE RIBOCICLIB SUCCINATE
• ²⁹ Core Patient-Reported Outcomes in Cancer Clinical Trials: Guidance for Industry
• ³⁴ REZDIFFRA RESMETIROM
• ⁴⁴ AYVAKIT AVAPRITINIB
• ⁵⁵ Core Patient-Reported Outcomes in Cancer Clinical Trials: Guidance for Industry
• ⁵⁷ ENJAYMO SUTIMLIMAB-JOME
• ⁶⁸ EPSOLAY BENZOYL PEROXIDE
• ⁸⁵ CARVYKTI ciltacabtagene autoleucel