Drugs for neglected tropical diseases: availability of age-appropriate oral formulations for young children.

It is recognised that paediatric indications and age-appropriate formulations are required to ensure that paediatric populations receive appropriate pharmacotherapeutic treatment. The lack of information on dosing, efficacy and safety data (labelling) is a well-recognised problem for all diseases affecting children. For neglected tropical diseases, the fact that they affect to a large extent poor and marginalised populations in low- and middle-income countries means that there is a low economic return on investment into paediatric development activities compared to other diseases [e.g. human immunodeficiency virus (HIV)]. This review provides an introduction to issues affecting the availability and development of paediatric population-relevant data and appropriate formulations of drugs for NTDs. We are summarising why age-appropriate formulations are important to ensure treatment efficacy, safety and effectiveness, outline initiatives to increase the number of paediatric indications/labelling and age-appropriate formulations, provide an overview of publicly available information on the formulations of oral drugs for NTDs relative to age appropriateness and give an introduction to options for age-appropriate formulations. The review completes with 'case studies' of recently developed paediatric formulations for NTDs, complemented by case studies for fixed-dose combinations for HIV infection in children since such formulations have not been developed for NTDs.
(© 2022. World Health Organization.)