Ensuring children have accelerated access to optimal drug formulations
Limited treatment options and sub-optimal formulations have contributed to poor adherence and outcomes for children living with HIV as well as other diseases that impact the lives of children living in resource limited settings. Despite the need for expanded and improved options for paediatric treatment, which are essential for sustained adherence and better treatment outcomes, barriers to incentivizing research and development remain.
Challenges for the development of paediatric drug formulations include:
- Small markets in high-income countries that do not stimulate development of formulations adapted to paediatric needs
- Current practice of sequential enrolment of different age groups into PK studies and clinical trials that delays progress
- Drug absorption, distribution, metabolism and elimination changes that lead to different PK/PD across ages
- Child-friendly formulations (taste-masked, scored tablets in dispersible, chewable or crushable forms or granules) covering the entire weight spectrum that are often not available
- Limited interaction of industry with the research and public health community on paediatric study plans (PIP/PSPs)
- Uptake and demand generation for new formulations, when developed, that remain slow due to lack of adequate prioritization of paediatric populations and the reluctance of health care providers who may not be comfortable with early adoption of novel paediatric formulations.
To move the field forward, there is a need for:
- Smarter coordination between existing processes to reduce the lag time between steps
- A portfolio approach to coordinate efforts on overall treatment needs across product lifecycles
- Donor engagement to share the manufacturers’ risk for niche products
- Market analytics to support decisions at various stages of program lifecycle, with product commercialization plans and means for implementation.
The Global Accelerator for Paediatric Formulations (GAP-f) aims to promote a faster, more efficient and more focused approach to paediatric formulation development. It is a collaborative framework that can help get better paediatric products quicker and cheaper by prioritizing products, streamlining the generation of clinical evidence, incentivizing manufacturers, accelerating product development and introduction, and coordinating procurement. The GAP-f formalizes collaboration across sectors to ensure accelerated development and uptake of the most needed drugs and formulations for children.
The GAP-f formalizes collaboration across sectors to ensure accelerated development and uptake of the most needed drugs and formulations for children. SRAs, stringent regulatory authorities; NRAs, national regulatory authorities (in high-burden countries).
Adapted from:Penazzato et al. Shortening the decade-long gap between adult and paediatric drug formulations: a new framework based on the HIV experience in low- and middle-income countries. J Int AIDS Soc, 2018, 21(S1), e25049 (see here).
GAP-f progress updates
We are glad to share updates on our continued efforts to advance the development of the Global Accelerator for Paediatric Formulations (GAP-f).
The GAP-f is an evolving collaboration between a number of partner organizations, including:
- Clinton Health Access Initiative (CHAI)
- Drugs for Neglected Diseases initiative (DNDi)
- Elizabeth Glaser Pediatric AIDS Foundation (EGPAF)
- Global Fund to Fight AIDS, Tuberculosis and Malaria
- ICAP at Columbia University's Mailman School of Public Health
- International AIDS Society (IAS, through its Collaborative Initiative for Paediatric HIV Education and Research – CIPHER - and Industry Liaison Forum – ILF)
- International Maternal Pediatric Adolescent AIDS Clinical Trials Group (IMPAACT)
- Medicines Patent Pool (MPP)
- Paediatric European Network for treatment of AIDS (PENTA)
- United Nations Children's Fund (UNICEF)
- U.S. President's Emergency Plan for Aids Relief (PEPFAR)
- World Health Organization (WHO).