Stakeholder perspectives on randomized clinical trials for children with poor-prognosis cancers

Stakeholder perspectives on randomized clinical trials for children with poor-prognosis cancers
JAMA Network Open; Nicholas Bird, MSc; Nicole Scobie; Pablo Berlanga, MD; Patricia Blanc, MBA; Vickie Buenger, PhD; Quentin Campbell-Hewson, MBChB; Michela Casanova, MD; Steven DuBois, MD; Julia Glade Bender, MD; Ann Graham; Delphine Heenen, LLM; Christina Ip-Toma, BSc; Donna Ludwinski, BSChem; Lucas Moreno, MD; Donna Neuberg, ScD; Antonia Palmer, MASc; Xavier Paoletti, PhD; Willemijn Plieger-van Solkema, LLM; Gregory Reaman, MD; Teresa de Rojas, MD; Claudia Rossig, MD; Anja Schiel, PhD; Sara Wakeling, BA; Gilles Vassal, MD; Andrew Pearson, MD; Leona Knox, BSc; 12/24
In poor-prognosis children’s cancers, new therapies may carry fresh hope for patients and parents. However, there is an absolute requirement for any new therapy to be properly evaluated to fulfill scientific, regulatory, and reimbursement requirements. Randomized clinical trials (RCTs) are considered the gold standard, but no consensus exists on how and when they should be deployed to best meet the needs of all stakeholders. The agreed-upon workshop conclusions provide a basis for key considerations while undertaking future drug development activities for children with poor-prognosis cancers, ensuring that the needs and perspectives of all stakeholders are factored in from the outset.