Introduction
The medical needs of the paediatric population and those of adults are very different. This population’s bodies function differently, and their treatments extend beyond simply modifying adult doses and therapies. The Federal Food, Drug, and Cosmetic Act categorises them as neonates (born within the first 28 days of life), infants (29 days to less than 2 years), children (2–12 years), and adolescents (12–21 years). Clinical trials are crucial to the development of medications and therapies tailored to the specific needs of paediatrics because of their distinct physiological and developmental differences.
Every year in Africa, common childhood illnesses—particularly pneumonia, diarrhoea, malaria, and newborn disorders—claim the lives of 2.6 million children before they turn five (Demby, 2023). This is a pretty high number for illnesses that, if age-appropriate treatments were available, could be avoided or treated.
The most effective way to produce the evidence-based knowledge needed to make illness-specific medicine popular in this area is through clinical trials. Nonetheless, the population of paediatric ages remains disadvantaged due to the disproportionately low quantity of clinical trials carried out on them to identify and enhance the most effective medical intervention currently available (Iroh Tam et al., 2020).
Why conduct paediatric clinical trials in Africa?
Given the influence of age on pharmacokinetics and pharmacodynamics, children’s prescriptions, which are based on extrapolated data from adults, can pose significant risks. The pharmaceutical industry is required by current laws and regulations, such as the Paediatric Research Equity Act (PREA), to conduct excellent research when developing medications to guarantee the availability of high-quality data about paediatric treatments (ICTRP, 2024). However, according to Clinical Trials Arena, over 50% of paediatric medical interventions lack any controlled trial data, and the majority of medications used by children are currently off-label (McLaren, 2024).
Paediatric clinical trials are a vital means of ensuring that children in Africa have access to safe and effective medications, particularly those who reside in remote areas where chronic and debilitating parasitic infections or other neglected tropical diseases, or NTDs, are prevalent (N’Goran et al., 2019). Notably, the World Health Organisation (WHO) has stated that the treatment of these diseases in the paediatric population cannot be supported by the current dosages and formulations of medications on their essential medicine list due to a lack of evidence.
The purpose of paediatric clinical trials is to help researchers find the most effective ways to treat children, which makes them extremely important. Still, when weighing the risks of research against the need for safe and proven therapies, the vulnerable nature of this population must be taken into account. This is supported by ICH E11 and ICH E6 (Good clinical practice), which specify that paediatric subjects in clinical trials should only be included if the possible benefits outweigh the risks. In order to protect participants’ rights and safety, trials should be planned to minimise risks, and the therapeutic benefits for the paediatric population should be reasonable.
As per the International Clinical Trials Registry Platform (2024), children are generally not able to give legally binding consent to participate in clinical trials due to specific ethical and clinical concerns. Given that the child is still a minor and that the parents may be legally obligated to give informed consent before the child can participate, it is crucial that both the parent and the child are aware of the benefits and risks of involvement. After being given an age-appropriate explanation of the trial, the child might then assent.
Paediatric disease prevalence in Africa
Major causes of death in children vary by age, but children under the age of five are particularly vulnerable to infectious diseases such as malaria, pneumonia, diarrhoea, HIV, and tuberculosis. For older children, non-communicable diseases, injuries and conflict pose more significant threats (UNICEF, 2016; Arora, 2024).
Sub-Saharan Africa is the region with the highest prevalence of diseases like pneumonia, which causes 16% of all deaths in children under five. More than 700,000 children under the age of five die from pneumonia each year—roughly 2,000 of them every day—making it the leading infectious cause of death in children globally (WHO, 2017; UNICEF Data, 2023). With 100,000 new cases of childhood cancer diagnosed annually, this disease is also becoming more and more significant. South Africa, West Africa, and East Africa report 21.7%, 8.5%, and 8.1% survival rates for all malignancies, respectively, while North Africa reports 30.3% (Van Heerden et al., 2020).
Common infectious diseases continue to kill a lot of young children, even though they are completely preventable and treatable. Despite the low overall survival rate for paediatric illnesses, this map by Mordor Intelligence (2024) demonstrates that the market for paediatric trials is still expanding slowly in the African region.
Paediatric clinical trials in Africa: A search for solutions
The Pan African Clinical Trials Registry reports that over 22 paediatric trials are now recruiting in Africa for a variety of illnesses, including cancer in Egypt, urogenital schistosomiasis in Gabon, childhood tuberculosis in Zambia, and perinatal asphyxia for newborns in the Democratic Republic of the Congo. Considering that this is in contrast to the 89 paediatric studies conducted in the United States and 54 conducted in the United Kingdom (Clinicaltrials.gov), the significant differences speak for themselves.
According to Clinical Trials Arena, there are several reasons why there are not enough paediatric-only and paediatric-inclusive trials. These include a lack of funding (which usually comes from government and nonprofit organisations), the moral dilemmas associated with studying minors, and the challenges associated with enrolling and keeping young patients in studies.
Clinical trials have the potential to enhance the precision of diagnosis, reduce treatment failure, and boost the efficacy of interventions. Africa has significant research and clinical trial potential, with a largely young population that can have up to 20 malignancies per 100,000 children annually (Van Heerden et al., 2020).
The previous traditional trial model of clinical trials burdened children and their carers excessively because it required frequent travel to the clinical site; concerns included the number of invasive treatments (like blood draws) that the child would need to undergo at the site, as well as the length and frequency of study visits. According to GlobalData’s Clinical Trials database, decentralisation has been used in paediatric trials more often than in non-paediatric trials recently (McLaren, 2024). In 2022, 9.6% of all paediatric trials included at least one decentralisation component, such as remote participant monitoring or mobile visits. This would increase the widespread adoption of clinical trials throughout Africa and lessen the burden associated with research participation.
Conclusion: The path forward
Millions of untreated paediatric populations in Africa require safe and effective medications, and it is becoming clearer that conducting paediatric clinical trials in accordance with relevant ethical and regulatory guidelines is a crucial first step in that direction.
To guarantee this, it is necessary to enhance the ability of regional ethics committees and the National Medical Regulatory Authority (NMRA) to supervise medical research, guaranteeing participant safety and the scientific accuracy of the clinical data. Even though many African nations have seen a progressive improvement in their regulatory capacity, according to WHO estimates, at least 30% of NMRAs in Africa have limited ability to carry out essential regulatory functions (N’Goran et al., 2019).
All things considered, one of the lowest health indicators for this population is a result of the frequent exclusion of children and adolescents from clinical trials and the dearth of products available that specifically target them. Efforts must indeed be made to improve the utilisation of currently available interventions in this population and to expand access to potentially life-saving, affordable interventions for the prevention and treatment of conditions in children.
References
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Demby, A. (2023, August 31). Calling all african health leaders: Ensuring the survival of africa’s children demands african leadership and accountability. Child Health Task Force. https://www.childhealthtaskforce.org/news/2023/calling-all-african-health-leaders-ensuring-survival-africas-children-demands-african
International Clinical Trials Registry Platform (ICTRP). (2024). Clinical trials in children. Www.who.int. https://www.who.int/clinical-trials-registry-platform/clinical-trials-in-children
Iroh Tam ,PuiYing, Angela, D., Labi ,AppiahKorang, Mujuru ,Hilda A, & Ogunbosi ,Babatunde O. (2020). Antimicrobial resistance among children in Africa: need for paediatric clinical trials. Expert Review of Antiinfective Therapy, 18(10), 955–956. https://doi.org/10.1080/14787210.2020.1782741
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Mordor Intelligence. (2024). Pediatric clinical trials market – share & insights. Mordorintelligence.com. https://www.mordorintelligence.com/industry-reports/pediatric-clinical-trails-market
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