A survey of new oncology drug pediatric approvals in the USA from 2000 to 2021: focus on the impact of pediatric formulation availability and pediatric dose selection

Authors:: Poster Author(s)

Chen, Meg FDA/CDER (Student); Shen, Guoxiang, FDA/CDER (Mentor)

Center:: Contributing Office

Center for Drug Evaluation and Research

Abstract

Poster Abstract

Objective

To determine the appropriateness of dosage formulations and dosing regimens of oncology medications indicated for use in pediatric populations, defined as less than 17 years of age. In addition, this research also aims to investigate the extent of clinical safety, efficacy and pharmacokinetic (PK) data to support pediatric dosing regimen selections.

Methods

A retrospective review of the Drugs@FDA database was conducted, using Drug Approval Reports organized by month. Package Inserts of Original New Drug Applications (NDAs) and Biologics License Applications (BLAs) from January 2000 to June 2021 were searched for oncology-related indications and pediatric approvals. Information pertaining to dosage form and dosage regimens was also collected from the labels. The NDA/BLA numbers of these medications were entered into DARRTs Resources, and clinical pharmacology reviews from DARRTs were searched for PK, safety, and efficacy data from studies with pediatric subjects and/or population PK modeling simulations from data collected from adult studies.

Results

Of 185 NDAs and BLAs with oncology indications, 32 (17%) are indicated for use in pediatric patients. Five of these NDAs/BLAs were developed specifically for pediatric populations. 24 (75%) have pharmacokinetic data to support pediatric dosing regimen, 28 (88%) have safety data, and 20 (62.5%) have efficacy data. 17 of the 32 (53%) have all three components. While 14 of the 32 approvals were BLAs and thus come in injectable forms, 18 were NDAs that require age-appropriate formulations (2 were for pediatric dosage forms of the same medications). Only 6 out of the 18 (33%) of NDAs have developed age-appropriate formulations for pediatric populations, and 11 (69%) have different dosing regimens for pediatric and adult populations. As children under a certain age are unable to swallow whole tablets or capsules, a lack of age-appropriate formulations, namely oral suspensions and intravenous injectables, may limit the use of oncology medications in younger age groups, as seen with Rozlytrek. In other cases, doses administered to children may need to be rounded due to fixed doses of tablets and capsules, such as in Xalkori.