Clinical development of new drugs for adults and children with cancer, 2010-2020
Dr. Florence Bourgeois
Analysis of new molecular entities (NMEs) entering first-in-human (FIH) phase 1 trials from 2010 to 2015 found that after a mean follow-up of 8.0 years 173 of 572 NMEs did not advance beyond FIH trials, and 39 were approved by the FDA. NMEs had a 10.4% estimated probability (95% CI 6.6-14.1) of being approved by the FDA within 10 years of FIH trial. After a median of 4.6 years since start of FIH trials, 11.7% of NMEs were tested in trials open to pediatric patients and 0.9% were approved for pediatric indications. The authors conclude that more efficient clinical development strategies are needed to evaluated new cancer therapies, especially for children.
