Accelerated introduction of better medicines often means doing so despite limited clinical data specifically collected in a paediatric population. As a result, a safety net needs to be put in place to rapidly capture any adverse events that may only be detected when new medications are rolled out. Leveraging work in HIV as a proof of concept, GAP-f aims to foster and enhance the use and interpretation of safety and effectiveness data for new medicines. The Paediatric Data Hub will be designed and implemented to improve the availability, quality and use of real-world data to monitor and optimize treatments. This project will leverage electronic medical records systems at various levels of maturity in countries and complement other more traditional sources of effectiveness and safety data such as following up of randomized control trials and observational cohorts. 

Adapting the use of existing adult formulations and informing the design of new paediatric formulations

Identifying the optimal dose of drugs for children and infants is often challenging as direct extrapolation from adults doses is not possible. At GAP-f, we are utilizing pharmacokinetic modelling across current therapeutic areas to accelerate the development of paediatric products, but also to expand use of existing adult formulations wherever possible with the goal of reducing market fragmentation and contributing to supply continuity.

Better Medicines for Children project

GAP-f has recently started a collaboration with the Bill & Melinda Gates Foundation to identify and prioritize missing paediatric formulations for the WHO Model List of Essential Medicines for Children (EMLc). This scoping exercise will enable us to assess the appropriateness of existing paediatric formulations and to identify overall gaps. This process will feed into future EMLc revisions and inform inclusion of additional missing formulations in the GAP-f portfolio. This will ensure that in the long-term, children in low and middle income countries will be able to access a range of essential medicines in appropriate formulations.

Eliminating bitterness as one of the causes of poor adherence 

A drug’s potency is often correlated to its bitterness. As innovators develop more potent and effective drugs, they are also inadvertently developing more bitter compounds. Some paediatric drugs are so bitter that caregivers struggle to get their children to take their medicine. Children from different parts of the world sense bitterness differently.

GAP-f and members together with Monell Chemical Senses Center are working to identify bitter blockers for specific drugs in the GAP-f portfolio that should work for all children. In due course, this partnership will address other bitter drugs for paediatric use. The initial priority drugs include tenofovir alafenamide (TAF) for HIV, sofosbuvir (SOF) for hepatitis C, primaquine for malaria, cotrimoxazole for prevention and treatment of opportunistic infections in HIV-exposed and HIV-infected children, and cloxacillin for treatment of Staphylococcus aureus infections. Success from this work would establish a proof of concept whose principles can then be applied to other bitter drug products for paediatric markets worldwide.