WHO priorities | R&D of age-appropriate medicines for treatment of NTDs
The WHO released the first ever list of priority paediatric formulations for five NTDs to target research and development in addressing the specific needs of infants and children. The diseases are human African trypanosomiasis, onchocerciasis, scabies, schistosomiasis and visceral leishmaniasis.
The list results from a “ Paediatric Drug Optimization” (PADO) exercise carried out by WHO, the purpose of which is to identify priority medicines and formulations to be investigated and developed with a time horizon of 3–5 years (PADO priority list) or with a time horizon of 5–10 years (PADO watch list). Prioritization is a necessary first step to enable a targeted approach to research and development, which needs to be undertaken in conjunction with developing and updating normative guidance on the use of medicine for treating and preventing communicable and noncommunicable diseases. As such, WHO leads and convenes this work and develops it in collaboration with appropriate stakeholders in each disease area.
There is a severe lack of pharmacokinetic data for NTD medicines, which are required to optimize doses for children. Only 11% of pharmacokinetic studies for NTD medicines included children and, for most medicines, these data are not available. Generally, limited financial incentives have resulted in fewer new medicines being developed for NTDs. Paediatric formulations have faced added challenges because of the limited participation of children in clinical trials and the absence of dosing and treatments tailored to their age. Research conducted in 2022 showed that, overall, less than half of WHO-recommended medicines for NTDs are approved for children, highlighting the urgent need to increase research activity for NTDs in this area.