TY - JOUR
T1 - Impact of ACCELERATE Paediatric Strategy Forums
T2 - a review of the value of multi-stakeholder meetings in oncology drug development
AU - Pearson, Andrew D J
AU - de Rojas, Teresa
AU - Karres, Dominik
AU - Reaman, Gregory
AU - Scobie, Nicole
AU - Fox, Elizabeth
AU - Lesa, Giovanni
AU - Ligas, Franca
AU - Norga, Koen
AU - Nysom, Karsten
AU - Pappo, Alberto
AU - Weigel, Brenda
AU - Weiner, Susan L
AU - Vassal, Gilles
N1 - © The Author(s) 2023. Published by Oxford University Press.
PY - 2024/2/8
Y1 - 2024/2/8
N2 - In a landscape of an increasing number of products and histology and age agnostic trials for rare patient cancer, prioritization of products is required. Paediatric Strategy Forums, organized by ACCELERATE and the European Medicines Agency with participation of the US Food and Drug Administration, are multi-stakeholder meetings that share information to best inform pediatric drug development strategies and subsequent clinical trial decisions. Academia, industry, regulators, and patient advocates are equal members, with patient advocates highlighting unmet needs of children and adolescents with cancer. The 11 Paediatric Strategy Forums since 2017 have made specific and general conclusions to accelerate drug development. Conclusions on product prioritization meetings, as well as global master protocols, have been outputs of these meetings. Forums have provided information for regulatory discussions and decisions by industry to facilitate development of high-priority products; for example, 62% of high-priority assets (agreed at a Forum) in contrast to 5% of those assets not considered high priority have been the subject of a Paediatric Investigational Plan or Written Request. Where there are multiple products of the same class, Forums have recommended a focused and sequential approach. Class prioritization resulted in an increase in waivers for non-prioritized B-cell products (44% to 75%) and a decrease in monotherapy trials, proposed in Paediatric Investigation Plans (PIP) submissions of checkpoint inhibitors from 53% to 19%. Strategy Forums could play a role in defining unmet medical needs. Multi-stakeholder forums, such as the Paediatric Strategy Forum, serve as a model to improve collaboration in the oncology drug development paradigm.
AB - In a landscape of an increasing number of products and histology and age agnostic trials for rare patient cancer, prioritization of products is required. Paediatric Strategy Forums, organized by ACCELERATE and the European Medicines Agency with participation of the US Food and Drug Administration, are multi-stakeholder meetings that share information to best inform pediatric drug development strategies and subsequent clinical trial decisions. Academia, industry, regulators, and patient advocates are equal members, with patient advocates highlighting unmet needs of children and adolescents with cancer. The 11 Paediatric Strategy Forums since 2017 have made specific and general conclusions to accelerate drug development. Conclusions on product prioritization meetings, as well as global master protocols, have been outputs of these meetings. Forums have provided information for regulatory discussions and decisions by industry to facilitate development of high-priority products; for example, 62% of high-priority assets (agreed at a Forum) in contrast to 5% of those assets not considered high priority have been the subject of a Paediatric Investigational Plan or Written Request. Where there are multiple products of the same class, Forums have recommended a focused and sequential approach. Class prioritization resulted in an increase in waivers for non-prioritized B-cell products (44% to 75%) and a decrease in monotherapy trials, proposed in Paediatric Investigation Plans (PIP) submissions of checkpoint inhibitors from 53% to 19%. Strategy Forums could play a role in defining unmet medical needs. Multi-stakeholder forums, such as the Paediatric Strategy Forum, serve as a model to improve collaboration in the oncology drug development paradigm.
KW - Adolescent
KW - Child
KW - Humans
KW - Drug Development
KW - Neoplasms/drug therapy
KW - Medical Oncology/methods
KW - B-Lymphocytes
UR - http://www.scopus.com/inward/record.url?scp=85182869128&partnerID=8YFLogxK
M3 - Review
C2 - 37975877
SN - 1460-2105
VL - 116
SP - 200
EP - 207
JO - JNCI-Journal of the National Cancer Institute
JF - JNCI-Journal of the National Cancer Institute
IS - 2
ER -