Paediatric Strategy Forum for medicinal product development for acute myeloid leukaemia in children and adolescents: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration

European Journal of Cancer : Official Journal for European Organization for Research and Treatment of Cancer (EORTC) [and] European Association for Cancer Research (EACR)
Andrew D J PearsonGilles Vassal

Abstract

The current standard-of-care for front-line therapy for acute myeloid leukaemia (AML) results in short-term and long-term toxicity, but still approximately 40% of children relapse. Therefore, there is a major need to accelerate the evaluation of innovative medicines, yet drug development continues to be adult-focused. Furthermore, the large number of competing agents in rare patient populations requires coordinated prioritisation, within the global regulatory framework and cooperative group initiatives. The fourth multi-stakeholder Paediatric Strategy Forum focused on AML in children and adolescents. CD123 is a high priority target and the paediatric development should be accelerated as a proof-of-concept. Efforts must be coordinated, however, as there are a limited number of studies that can be delivered. Studies of FLT3 inhibitors in agreed paediatric investigation plans present challenges to be completed because they require enrolment of a larger number of patients than actually exist. A consensus was developed by industry and academia of optimised clinical trials. For AML with rare mutations that are more frequent in adolescents than in children, adult trials should enrol adolescents and when scientifically justified, effic...Continue Reading

Citations

Sep 8, 2020·British Journal of Clinical Pharmacology·Mark A TurnerCarlo Giaquinto
Sep 30, 2020·European Journal of Cancer : Official Journal for European Organization for Research and Treatment of Cancer (EORTC) [and] European Association for Cancer Research (EACR)·Andrew Dj PearsonGilles Vassal
Apr 13, 2021·Pediatric Blood & Cancer·Grace EganJohann Hitzler
Aug 23, 2021·Paediatric Drugs·LaQuita M JonesTodd Cooper
Nov 19, 2021·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·Nicholas BirdDonna Ludwinski

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Methods Mentioned

BETA
flow
PCR

Software Mentioned

ACCELERATE
EUpAL

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