New Priority Paediatric Formulations for Neglected Tropical Diseases

New Priority Paediatric Formulations for Neglected Tropical Diseases

The World Health Organization (WHO) has recently unveiled a groundbreaking list of priority paediatric formulations for neglected tropical diseases (NTDs). This significant development aims to address the specific needs of infants and children when it comes to treating these diseases. The list, resulting from a comprehensive exercise called “Paediatric Drug Optimization” (PADO) carried out by the WHO, focuses on five NTDs: human African trypanosomiasis, onchocerciasis, scabies, schistosomiasis, and visceral leishmaniasis.

The release of this priority list is an essential step in targeting research and development efforts to meet the healthcare needs of children aged 3 to 5 years (PADO priority list) or within a timeframe of 5 to 10 years (PADO watch list). Prioritization allows for a more focused approach to developing new medicines and formulations while aligning with the ongoing work of updating treatment guidelines for communicable and noncommunicable diseases. WHO spearheads this vital initiative in collaboration with relevant stakeholders in each disease area.

Neglected tropical diseases encompass a diverse group of infectious diseases characterized by varying epidemiology, local prevalence, control strategies, and challenges related to the availability of treatments suitable for children. Insufficient pharmacokinetic data for NTD medicines has been a persistent issue, making it difficult to optimize doses for pediatric patients. Shockingly, only 11% of pharmacokinetic studies for these medicines have focused on children, and the data for most drugs remain unavailable. Moreover, limited financial incentives have impeded the development of new medicines for NTDs. Children’s participation in clinical trials has been limited, which further complicates the development of pediatric formulations tailored to their specific age group. A recent study conducted in 2022 revealed that less than half of the WHO-recommended medicines for NTDs are approved for use in children, highlighting the urgent necessity for increased research in this area.

Dr. Ibrahima Socé Fall, Director of the WHO Global NTD Programme, expressed enthusiasm for extending the PADO concept to NTDs, emphasizing the need to target all age groups, including children, to interrupt NTD transmission and achieve elimination. He called for greater investments to support the development, production, and accessibility of these formulations, particularly for vulnerable populations.

The newly released PADO priority list for NTDs includes six medicines across five disease areas, such as acoziborole for human African trypanosomiasis and ivermectin and moxidectin for scabies and onchocerciasis. Additionally, the PADO watch list features three promising compounds: emodepside and oxfendazole for scabies and onchocerciasis, and LXE408 for visceral leishmaniasis.

Investments and coordinated efforts in research and development are crucial to bridge the nearly decade-long gap in pediatric medicine development compared to adult formulations. The WHO, along with its partners in the Global Accelerator for Paediatric Formulations Network (GAP-f), has intensified its activities to meet this global commitment following the adoption of resolution WHA69.20 by the World Health Assembly. WHO’s successful PADO work in identifying key priority products for research and development has previously been implemented for HIV, hepatitis C, tuberculosis, and antibiotics, demonstrating its potential to accelerate access to optimal formulations in small and fragmented markets for pediatric medicines.

Sustained investment in the research and development of improved pediatric formulations for priority NTD medicines is vital to expedite actions toward equitable access to appropriate treatments for infections worldwide.

Frequently Asked Questions (FAQ)

What are neglected tropical diseases (NTDs)?

NTDs are a diverse group of infectious diseases that primarily affect populations in tropical and subtropical regions. These diseases often have limited treatment options and primarily affect communities with limited access to healthcare.

Why is it important to develop pediatric formulations for NTDs?

Developing pediatric formulations for NTDs is crucial because these diseases disproportionately affect children in endemic areas. Tailoring treatments to their specific needs ensures more effective outcomes and supports the goal of interrupting disease transmission and achieving elimination.

What challenges hinder the development of pediatric formulations for NTDs?

There are several challenges in developing pediatric formulations for NTDs. These include limited participation of children in clinical trials, insufficient pharmacokinetic data for optimizing doses, limited financial incentives for research and development, and the absence of dosing and treatments tailored to different age groups.

What is the significance of the PADO priority list?

The PADO priority list is a crucial step in directing research and development efforts towards addressing the specific needs of children with NTDs. By identifying priority medicines and formulations, it enables a targeted approach to optimize treatments and increase access to quality healthcare for pediatric patients.

How can investment in research and development be increased for NTDs?

Increasing investment in research and development for NTDs requires collective efforts from various stakeholders, including governments, pharmaceutical companies, and international organizations. Financial incentives, collaborative partnerships, and policy support can help foster advancements in pediatric formulations and promote more equitable access to treatment for NTDs.

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