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

Area of Science:

• Pediatric Oncology
• Drug Development
• Regulatory Science

Background:

• Timely evaluation and approval of new anti-cancer drugs are crucial for improving survival and reducing long-term side-effects in pediatric cancer patients.
• The international multi-stakeholder organization ACCELERATE was established to expedite the investigation of novel anti-cancer drugs for this population.

Purpose of the Study:

• To outline the initiatives and impact of ACCELERATE in advancing pediatric cancer drug development.
• To highlight the importance of multi-stakeholder collaboration, mechanism-of-action driven approaches, and early patient engagement.

Main Methods:

• Facilitating enhanced communication and understanding between academia, industry, patient advocates, and regulators.
• Developing Paediatric Strategy Forums to prioritize medicinal products and strategize drug development.
• Championing early assessment of drugs in adolescents via inclusion in adult early phase trials.
• Promoting alignment between regulatory agencies (EMA, FDA) and identifying unmet medical needs.

Main Results:

• Improved communication and collaboration among key stakeholders in pediatric oncology drug development.
• Establishment of prioritized drug development strategies and focused sequential approaches.
• Successful integration of adolescents into early phase trials for promising new drugs.
• Strengthened alignment between regulatory bodies and identification of critical unmet needs.

Conclusions:

• Early and continuous engagement of all stakeholders, including patient advocates, is essential for developing innovative, patient-centric clinical trials.
• Innovative trial designs and early discussions with sponsors and regulators are necessary.
• These collaborative efforts enable children and adolescents with cancer to benefit rapidly from novel therapies, improving outcomes and reducing treatment burdens.