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20 November 2023

To mark World Children’s Day, the Global Accelerator for Paediatric Formulations Network (GAP-f) is highlighting some of its work to shape the global innovation and access landscape for better paediatric medicines.

Since 2015, the decline in infant and child mortality has been slowing, yet there is a lack of research into new life-saving tools targeted at children. This is partly due to the complexity of conducting studies including the youngest age groups.

Children cannot swallow tablets or capsules, often cannot bear the taste of liquid medicines and metabolize drugs differently as they develop and grow. Paediatric medicines need to be palatable, scored, crushable, dispersible (i.e. disintegrates quickly in water), chewable, sprinkled on food or mixed with breast milk. Appropriate medicines to save and improve the lives of infants and children often do not exist, are unavailable or are not quality assured, especially in low-resource settings.

Even with significant progress in child health, with 6 million fewer children under the age of 5 years dying in 2016 than in 1990, urgent action is needed to achieve the UN Sustainable Development Goals (SDG), particularly SDG 3, for good health and well-being, and the related target to achieve Universal Health Coverage (UHC) by 2030.

GAP-f network partners are working together to remove barriers to developing and delivering appropriate, quality, affordable and accessible medicines for children and contribute to UHC. GAP-f works by spurring collaboration across stakeholders to identify gaps, set priorities for needs and accelerate product investigation, development and delivery to improve and save the lives of children.

Below are key GAP-f events and documents being released to mark World Children’s Day:

Exploring innovative approaches for faster access to paediatric medicines for antimicrobial resistance

Our final webinar of 2023 in the GAP-f #BetterMeds4Kids webinar series focuses on exploring innovative approaches for faster access to paediatric medicines for antimicrobial resistance (AMR). The webinar will be hosted on 20 November 2023 at 1400 CET. Click here for more information.

Cefiderocol product brief

Despite huge progress, preventable and treatable infectious diseases remain the leading cause of death among under 5s. Bacterial infections, especially pneumonia, neonatal sepsis and gastrointestinal infections, are the main cause of infectious mortality in this age group worldwide. This problem is further exacerbated by the global rise of antimicrobial resistance.

WHO conducted an exercise to produce a Paediatric Drug Optimization (PADO) priority list of antibiotics for development, with all products included having an approved indication for children but for which age-appropriate formulations are missing.

Out of those on the list, the most ongoing work to date has focused on cefiderocol, which is the subject of a new product brief documenting the status of clinical trials, regulatory approval and research priorities, to further advance efforts to make it accessible to children.

Priority drugs for Neglected Tropical Diseases

Due to limited financial incentives, few new drugs are being developed for neglected tropical diseases (NTDs). Several NTDs disproportionately affect children versus adults. As is the case for most diseases affecting adults and children, the burden to children is compounded by their not being included in clinical trials, as well as a lack of age-appropriate dosing regimens and formulations.

To help address these challenges, WHO developed a PADO priority list for schistosomiasis, human African trypanosomiasis, scabies, onchocerciasis and visceral leishmaniasis.

Research and development pipeline analysis for childhood cancer

Childhood cancer remains a major cause of death among children globally, accounting for over 100,000 deaths every year. Despite major advances in research and development (R&D) in cancer, few clinical trials have addressed the effect of investigational medicine on tumour biology in children, particularly those living in in low- and middle-income countries.

To map gaps and barriers, WHO has developed a summary of the R&D landscape for childhood cancer, which shows where more investment is needed, using data from the WHO Global Observatory on Health R&D (GOHRD).

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