Lessons learned from drug trials in neurofibromatosis: A systematic review

Dhaenens, Britt A E; Ferner, Rosalie E.; Evans, D. Gareth; Heimann, Guenter; Potratz, Cornelia; Ketterij, Edwin van de; Kaindl, Angela M.; Hissink, Geesje; Carton, Charlotte; Nievo, Marco; Legius, Eric; Oostenbrink, Rianne

doi:10.1016/j.ejmg.2021.104281

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Platform Trials and Irps Must Address Key Gaps In Drug Devel...1

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“… 35 Moreover, most trials are single-country and in the case of NF2, single-arm. 36 Already successful experiences of collaborative platform trials in pediatrics exist, which allow to overcome/mitigate some of the barriers inherent to clinical research in pediatric patients, which are particularly challenging in the field of rare diseases, remarkably small sample sizes, 37 as well as fostering international collaboration.…”

Section: Platform Trials and Irps Must Address Key Gaps In Drug Devel...mentioning

confidence: 99%