Impact of ACCELERATE Paediatric Strategy Forums: a review of the value of multi-stakeholder meetings in oncology drug development

Abstract

In a landscape of an increasing number of products and histology and age agnostic trials for rare patient cancer, prioritisation of products is required. Paediatric Strategy Forums, organised 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 paediatric 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 eleven Paediatric Strategy Forums since 2017 have made specific and general conclusions to accelerate drug development. Conclusions on product prioritisation 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% 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 prioritisation resulted in an increase in waivers for non-prioritised B cell products (44% to 75%) and a decrease in monotherapy trials, proposed in 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.