Can a Multistakeholder Prioritization Structure Support Regulatory Decision Making? A Review of Pediatric Oncology Strategy Forums Reflecting on Challenges and Opportunities of this Concept

Abstract

Timely and successful drug development for rare cancer populations, such as pediatric oncology, requires consolidated efforts in the spirit of shared responsibility. In order to advance tailored development efforts, the concept of multistakeholder Strategy Forum involving industry, academia, patient organizations, and regulators has been developed. In this study, we review the first five pediatric oncology Strategy Forums co‐organized by the European Medicines Agency between 2017 and 2020, reflecting on the outcomes and the evolution of the concept over time and providing an outline of how a “safe space” for multistakeholder engagement facilitated by regulators could be of potential value beyond pediatric oncology drug development.

ACCELERATE – Five years accelerating cancer drug development for children and adolescents

“…The importance of industrysupported, academic-sponsored international platform trials and agreement for sequenced development efforts has been reinforced. Both the EMA [30,32] and the FDA [33] have highlighted the value of Paediatric Strategy Forums.…”

“…Prioritisation is key, as there are too many molecules to evaluate in children [30]. It is paramount to focus competition when too many trials with similar patient enrolment criteria for drugs within the same class or for the same molecular targets exist.…”

“…It is paramount to focus competition when too many trials with similar patient enrolment criteria for drugs within the same class or for the same molecular targets exist. It is important to note that FDA [32] and EMA [30,31] report that companies take into account the scientific recommendations from ACCELERATE's Paediatric Strategy Forums to guide prioritisation for the development of drugs for children in high value targets. There is a need for clear, multistakeholder endorsed criteria for second or third generation products to be developed.…”

The Changing Face of Oncology Research, Drug Development, and Clinical Practice: Toward Patient‐Focused Precision Therapeutics

“…[29][30][31] In addition, the partnering of regulatory agencies with other key stakeholders early in the drug development process and clinical trial design process will be critical for the advancement of precision cancer therapeutics. [29][30][31][32]…”

Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases

“…Regulatory authorities and public health research agencies have also sponsored scientific meetings on product development for these therapeutic classes and engaged in public–private collaborations to advance global drug development for rare diseases. One example is the Pediatric Strategy Forums which focus on developmental strategies to support regulatory decision making for various types of targeted therapies for pediatric cancers [ 66 ]. Another example is the International Neonatal Consortium (INC), a global public–private partnership formed to accelerate the development of therapies to prevent and treat a variety of neonatal conditions, most of which are rare diseases [ 67 ].…”