Drug development for neglected diseases must account for kids

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When three-year-old Faith heard her name called by the nurse in the visceral leishmaniosis ward in Kacheliba Sub-County Referral Hospital in West Pokot, Kenya, she burst into tears. Her cry echoed through the ward as her 25th and 26th painful injections were administered. She had already spent 13 days in the hospital and still had four more days and eight more injections to endure.

Through my work as a paediatrician and at the Drugs for Neglected Diseases initiative (DNDi), I have heard adults wince, and lay face down for an hour after their shots, trying to bear the searing pain. Imagine what a child feels.

Lack of child-friendly treatments means health workers are forced to improvise. Sometimes, tablets meant for adults are split into smaller pieces by children’s caregivers, or crushed and dissolved, hoping the child can swallow it. In the case of injections, adult dosages are sometimes adjusted by estimating the child’s weight or age. These improvised treatments carry real risks—including incorrect dosing, stronger side effects, and reduced efficacy.

For Faith, every injection was a harrowing ordeal. Her experience is not unique. In Eastern Africa, more than 60% of visceral leishmaniasis (also known as kala-azar) patients are children under 15 – and all subjected to the same painful treatment regimen. The injections are excruciating and carry toxic side effects.

While the treatment is free, the 17-day hospital stay takes a heavy toll. Caregivers often lose income, and children miss more than three weeks of school. These treatments are only available in a few specialized hospitals, forcing families to travel long distances.

Africa continues to fall short in developing safe, accessible, and child-friendly treatments for many diseases. For example, while optimal paediatric antiretroviral-based treatment eventually became available, for years caregivers had to manage bitter, difficult-to-administer syrups, turning every dose into a daily struggle. These child-specific formulations came decades after those for adults, highlighting a troubling delay in drug development where children were treated as an afterthought. And not much has changed: a 2019 study¹ found that only 17% of over 360 late-stage clinical trials for neglected diseases included participants under 18.

The argument that children must be protected from tests of new drugs, and they must be tested on adults first is a very valid concern. Paediatric clinical trials raise complex ethical issues. Children cannot provide informed consent and are considered vulnerable. But excluding them is not a solution either. New treatments under evaluation for children must go through extremely rigorous (and highly regulated) validation processes to ensure they are safe and do not carry risks. Growing evidence and well-established ethical frameworks show how to include children in trials safely and responsibly. Both the European Union’s Paediatric Committee and the U.S. FDA’s Pediatric Research Equity Act mandates testing when diseases affect children and offer clear protocols for safe inclusion.

An African-led regulations that borrow best practices and are tailored to Africa’s context is essential to protect children without excluding them from the promise of medical innovation. This will encourage regulatory authorities in different countries to authorize trials involving children. As African nations, we must urgently strengthen regulatory environments, both nationally and regionally, with enforceable guidelines that require pharmaceutical companies to take children’s unique needs into account when drugs are developed.

But regulation alone isn’t enough.

Africa also needs to incentivize research and development. This systemic failure also stems from a market-driven model where children, especially those in low- and middle-income countries, are regarded as unprofitable. As a result, pharmaceutical companies have little financial motivation to develop treatments for them, despite the huge needs. Governments can help change this by creating a policy environment that invests in and encourages pediatric research, by public, private, and not- for-profit organizations.

The continent has made some progress. Child-friendly tuberculosis medicines have become more widely available, and dispersible malaria tablets tailored for children are now used in many countries, an examples that show what’s possible when the needs of children are prioritized.

Currently the Drugs for Neglected Diseases initiative (DNDi), together with their partners, is developing a promising new oral treatment for kala-azar,

aimed at replacing the current painful and toxic injectable regimens. The organisation is also working on a single-dose oral treatment for children with both Stage 1 and Stage 2 sleeping sickness.

There are other initiatives trying to shift the tide. The World Health Organization’s Global Accelerator for Paediatric Formulations Network (GAP-f) has been established to address the long-standing gap between diseases that affect children and the medical research dedicated to them. GAP-f has published a list of priority pediatric formulations urgently needed across a range of diseases, including neglected tropical diseases, to accelerate the development of safer, more effective treatments for children.

Every child has a right to health, which includes the right to medicine tailored to their bodies. The impact of NTDs on children are significant-including disability, interfering with school attendance, cognitive and developmental delays and perpetuation of poverty. Our children cannot wait.