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ACCELERATE WORKSHOP

Approaches to Child-First Trials in Pediatric Oncology

28 and 29 September 2026
London, UK


This Child First workshop will explore the opportunities and challenges involved in advancing novel anti-cancer therapies directly into paediatric clinical trials without data from early phase adult cancer trials. It will bring together academic experts, patient advocates, regulators and industry to consider the ethical, regulatory, scientific and patient-centred requirements for responsible child-first development.


The workshop will focus on practical approaches to designing and conducting child-first trials, drawing on experience from paediatric oncology and other rare, life-threatening childhood diseases. Its aim is to develop a shared, globally coordinated framework that supports timely access to innovative therapies while ensuring rigorous safeguards, transparent communication and meaningful participation by patients and families.


Programme committee members:

  • Steven DuBois (chair)
  • Martha Donoghue
  • Dominik Karres
  • Nicole Scobie
  • Gilles Vassal
  • Peter Adamson


ACCELERATE wishes to extend its gratitude to LifeArc for hosting the forum and to C-FURTHER for providing funding. Our sincere thanks to both organisations for their generosity.

Statement of Need

First-in-child clinical trials of anti-cancer drugs almost always follow initial testing in adult cancer populations, providing critical safety, pharmacokinetic, and dosing data to inform the paediatric trial. Advances in precision paediatric oncology and immunotherapy, such as innovative and paediatric specific cell therapies, may require development of new agents only relevant to paediatric and not adult cancer populations. To ensure timely evaluation of novel agents for children with life-threatening malignancies, we must anticipate a need for “child-first” trials that advance to the clinic without data from early phase adult cancer trials. Such development raises unique ethical, regulatory, scientific, and patient-centred challenges. This invitation only workshop aims to address these challenges by developing a shared framework for responsible, efficient, and globally-coordinated child-first drug development.

 

Issues related to child-first trials also impact the development of innovative therapies for rare or ultra rare paediatric diseases, such as patient specific in vivo gene editing to treat an inborn error of metabolism. The workshop will be inspired by lessons learnt in this field and develop recommendations.

 

From the patient and family perspective, delays in accessing targeted therapies and personalized immunotherapies, lack of alternatives, and reliance on adult-centric models all contribute to treatment inequities and missed therapeutic opportunities. A child-first approach must therefore be crafted with rigorous safeguards, transparent communication, and meaningful participation of patients and families.


Objectives

  1. Frame ethical principles relevant to conducting child-first cancer clinical trials.
  2. Understand how child-first trials have been conducted in other rare paediatric life-threatening conditions
  3. Understand scope of child-first trials in paediatric oncology
  4. Develop a multistakeholder framework for the design and conduct of child-first trials in oncology
  5. Identify and propose potential solutions to facilitate the conduct of child-first trials.