Accelerating access to paediatric medicines: lessons learned from the Global Accelerator for Paediatric Formulations,...

Area of Science:

• Global Health
• Paediatric Pharmacology
• Medicines Access

Background:

• Neonatal and child mortality reduction efforts are ongoing, yet equitable access to age-appropriate medicines remains a significant challenge, especially in low- and middle-income countries.
• The Global Accelerator for Paediatric Formulations (GAP-f), established in 2020, is a WHO-hosted network comprising 33 partners dedicated to enhancing innovation and access to child-friendly medicines.

Purpose of the Study:

• To outline the focus and learnings from Phase 2 (2022-24) of GAP-f, concentrating on therapeutic areas with misaligned stakeholder efforts.
• To identify key strategies for improving paediatric formulation development and access, including clinical trial methodology, regulatory engagement, novel delivery technologies, and product roll-out collaboration.

Main Methods:

• Review of GAP-f's Phase 2 activities, focusing on stakeholder alignment, clinical trial innovation, regulatory engagement, technology identification, and collaborative product roll-out.
• Analysis of learnings to inform future prioritization, platform trial implementation, incentives for paediatric formulation development, and strategies for efficient medicine delivery to children.

Main Results:

• Adaptable prioritization processes and a focus on high-impact areas based on unmet needs are crucial.
• Platform trials show promise for accelerating evidence generation but require regulatory guidance, industry collaboration, and overcoming operational/funding hurdles.
• Incentivizing paediatric formulation development for small markets and proactively matching innovations to children's needs are essential.

Conclusions:

• Successful paediatric medicine development requires political will and community engagement for demand creation, roll-out support, and equitable use.
• Dedicated funding and targeted programs are vital for driving systemic and sustainable improvements in paediatric medicine access.
• Lessons from GAP-f implementation will guide future efforts to accelerate progress in providing essential medicines for children.