September 28, 2025

Abstract IA002: Challenges in accessing innovation for children with cancer

Puntos clave

Access to novel therapies remains limited due to lengthy clinical trial timelines and strict regulatory barriers.
Only 1.4% of new molecular entities received FDA approval within 10 years of clinical testing initiation.
Barriers in pediatric oncology include the need for age-appropriate formulations and challenging biological factors.
Strategies like the RACE act aim to eliminate histology-based waivers, potentially improving trial access.

Resumen

Abstract Children with cancer can access novel therapies through a range of mechanisms, including on- and off-label prescriptions, compassionate access, and clinical trials. A number of barriers have historically posed challenges to clinical trial development in pediatric oncology, including rare populations, young ages requirement child-friendly formulations, and unique underlying diagnoses and associated biology. Prior regulations also limited requirements for pediatric trials through waivers based upon common adult cancer histologies not being seen in children. These factors have led to a long lag time from first-in-human clinical trials to first-in-child clinical trials (median 6. 5 years in one analysis). Moreover, the proportion of trials and proportion of trial accruals supported by the pharmaceutical industry is lower for pediatric cancer trials compared to medical oncology trials. Not surprisingly then, the probability of a new molecular entity ultimately being US FDA approved was only 1. 4% by 10 years after initiating clinical testing. In this context, several strategies have been proposed to improve access to innovative medicines for children with cancer. Key examples include lowering the minimum age of eligibility for adult trials from 18 to age 12 years when shared biology between adult and adolescent tumors and the RACE act that has removed histology-based waivers. Early data on the impact of the RACE act show a similar proportion of drugs entering pediatric clinical trials, though the timeline for trial initiation appears earlier than pre-RACE. Citation Format: Steven DuBois. Challenges in accessing innovation for children with cancer abstract. In: Proceedings of the AACR Special Conference in Cancer Research: Discovery and Innovation in Pediatric Cancer— From Biology to Breakthrough Therapies; 2025 Sep 25-28; Boston, MA. Philadelphia (PA): AACR; Cancer Res 2025;85 (18Suppl₂): Abstract nr IA002.

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