TY - JOUR KW - Infectious Diseases KW - Parasitology KW - Paediatric indications KW - Paediatric age-appropriate formulations KW - Neglected tropical diseases (NTDs) KW - Oral Formulation KW - Preventive chemotherapy (PC) AU - Al-Obaidi I AU - Krome AK AU - Wagner KG AU - Pfarr K AU - Kuesel AC AU - Batchelor HK AB -

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.

BT - Parasites & Vectors DO - 10.1186/s13071-022-05546-7 IS - 1 LA - Eng N2 -

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.

PB - Springer Science and Business Media LLC PY - 2022 SP - 1 EP - 22 T2 - Parasites & Vectors TI - Drugs for neglected tropical diseases: availability of age-appropriate oral formulations for young children UR - https://parasitesandvectors.biomedcentral.com/counter/pdf/10.1186/s13071-022-05546-7.pdf VL - 15 SN - 1756-3305 ER -