Background: Clinical trials during drug development are generally conducted on a subset of the target population. To enhance the generalizability of findings from the drug development and to facilitate optimal use of new drugs/biologics, the study population should ideally reflect the patient population. The impact of intrinsic and extrinsic factors related to demographics on exposure or response is well recognized. The goal here was to conduct a landscape analysis on the availability of exposure and response information in the labeling of new molecular entities (NMEs) for specific populations such as race, ethnicity, sex, and age to facilitate a broader understanding of the gaps and opportunities for therapeutic individualization. Methods: For 159 NMEs approved in 2018-2020, clinical exposure and/or response information in subpopulations of race, ethnicity, sex, and age (pediatrics and older adults) were collected from Drugs@FDA using initially approved and the latest (before 12/31/2023) updated labeling. Categories used to catalog resulting information: silent; no clinical information available (i.e., explicitly stated in labeling); no clinical information available but recommendation provided (explicit/implicit); clinical data available; and not applicable. Postmarketing requirements or commitments (PMR/PMC) were identified using the approval letters. Results: For race/ethnicity, older adults and sex, exposure and/or response information was available in more than half of the labeling (60%, 64%, and 68%, respectively) (Figure 1). For pediatrics, 67% labeling had no clinical information. Of these, 34% had deferred studies for all or some age groups and 66% had waived or exempt studies. The updated labeling for 12% NMEs included pediatric age group expansion or dosage recommendations not in the initial label. 123 PMR/PMC were established for these subpopulations. Conclusion: Overall, no instances of dose adjustments were identified based on race, ethnicity, and sex. For older adults, some labeling noted differences in adverse events and 16% of the labeling contained dosing considerations. For pediatrics, fewer NMEs had information at initial approval, however, additional information was accrued postapproval. This study could be a valuable benchmarking resource for specific population related information.
