Marketing Authorisations with Limited Clinical Evidence: A Review of Small-Scale Cancer Drug Studies

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Key Points

The aim is to evaluate regulatory approvals for oncology drugs based on studies with limited patient enrollment.
Performed an electronic search of the EMA database and reviewed FDA notifications.
Focused on oncology drug approvals from 2010 to 2025 for studies enrolling 50 or fewer patients.
Identified disparities in age approvals between FDA and EMA for paediatric indications.
14 oncology drugs were approved by either EMA or FDA, with only one being a randomized comparative study.
FDA approved 13 drugs for paediatric use, while EMA approved only 8; both approved avapritinib for adults.
Efficacy outcomes for paediatric indications were based on overall response rates (ORR), which were below 50% in 2 studies.

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Review analyzes oncology drug approvals with limited studies enrolling fewer than 50 patients, highlighting implications.

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Curtis et al. (2026) studied this question.

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