ACCELERATE and European Medicines Agency Paediatric Strategy Forum for medicinal product development of checkpoint inhibitors for use in combination therapy in paediatric patients

Abstract

The third multistakeholder Paediatric Strategy Forum organised by ACCEL

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

“…The third forum, held in September 2018, was on the topic of immune checkpoint inhibitor combinations in pediatric malignancies. 9 The topic choice was driven by the success of immune checkpoint inhibition in adult malignancies not mirrored in pediatric malignancies. The objective was to consider opportunities for pediatric development in which checkpoint inhibitors are used in…”

“…It also concluded on the need for an international inter-company registry of pediatric-specific early and late adverse effects of immunotherapies, which is currently in the process of being set up. 9 The fourth forum on medicinal product development for acute myeloid leukemia (AML) was the first with the FDA contributing to the organization, recognizing that drug development in rare diseases, such as pediatric oncology, is only successful when approached globally. It was held in April 2019 and 26 products from 18 companies were discussed.…”

Discordance between recommended and delivered therapies for immunotherapy‐associated cutaneous toxicities in pediatric populations

“…Cutaneous immune‐related adverse events (cirAEs) represent one of the most common and disruptive toxicities of immune‐checkpoint inhibitor (ICI) therapy in the general population 1‐3 . Guidelines for cirAE management exist, but until recently, the patients receiving these therapies included only adults 1‐3 .…”

“…To date, though more than 400 children have received ICIs in clinical trials, only 15 cirAEs have been reported, and age‐specific treatment paradigms have not been developed 1,4 . How cirAEs are currently being managed by pediatric providers, and how these approaches compare to general treatment recommendations, remain unclear 1‐3 . We sought to address this gap by characterizing patterns of cirAE management in a multiinstitutional pediatric cohort, assessing discordance between delivered and recommended therapies using consensus guidelines.…”

Evaluation of the immunological response of patients with childhood cancer treated with the personalized peptide vaccine for refractory soft tissue sarcoma: An early phase II study

“…Recent advances in pediatric multidisciplinary cancer therapy have resulted in long-term survival and full recovery in most patients. 1,2 However, this impressive advancement is associated with late adverse effects owing to repeated intense treatments needed to achieve these results. One of the effects is the suppression of immune responses, which might in turn result in early recurrence 3,4 or the development of secondary cancers.…”