Clinical development of new drugs for adults and children with cancer, 2010-2020
Menée à partir de données de la base "AdisInsight" et du site internet de la "U.S. Food and Drug Administration", cette étude évalue, sur la période 2010-2020, le nombre de nouveaux médicaments autorisés par la FDA pour le traitement des cancers chez l'adulte et l'enfant
Résumé en anglais
Many new molecular entities (NMEs) enter clinical development to evaluate potential therapeutic benefits for oncology patients. We characterized adult and pediatric development of the set of NMEs that started clinical testing in 2010-2015 worldwide.We extracted data from AdisInsight, an extensive database of global pharmaceutical development, and the FDA.gov website. We followed the cohort of NMEs initiating First in Human (FIH) phase I clinical trials in 2010-2015 up to the end of 2020. For each NME, we determined whether it was granted FDA approval, studied in a trial open to pediatric enrollment, or stalled during development. We characterized the cumulative incidence of these endpoints using statistical methods for censored data.The 572 NMEs starting FIH studies in 2010-2015 were studied in 6,142 trials by the end of 2020. Most NMEs were small molecules (N = 316, 55.2%), antibodies (N = 148, 25.9%), or antibody-drug conjugates (N = 44, 7.7%). After a mean follow-up of 8.0 years, 173 NMEs did not advance beyond FIH trials, and 39 were approved by the FDA. NMEs had a 10.4% estimated probability (95% Confidence Interval: 6.6%-14.1%) of being approved by the FDA within 10 years of FIH trials. After a median of 4.6 years since start of FIH trials, 67 (11.7%) NMEs were tested in trials open to pediatric patients and 5 (0.9%) were approved for pediatric indications. More efficient clinical development strategies are needed to evaluate new cancer therapies, especially for children, and incorporate approaches to ensure knowledge gain from investigational products that stall in development.
