To support optimal use of all available high quality medicines, MMV is working with partners to generate and disseminate post approval evidence of their safety and efficacy in the real world, especially in children. The West African Network for Clinical Trials of Antimalarial Drugs (WANECAM) carried out the first trial to investigate and confirm the real-life safety and effectiveness of four ACTs, when used for repeated treatment over a 2-year period.
Further to this study, the Central African Network on Tuberculosis, HIV/AIDS and Malaria (CANTAM) is conducting a post-approval study exploring the safety and tolerability in real-life settings of Pyramax® tablets and granules in adults and children.
Dr Ghyslain Mombo-Ngoma Head of Clinical Operations Department, CERMEL (Centre de Recherches Médicales de Lambaréné), Gabon, and an investigator on the CANTAM study explains the potential of Pyramax® granules in benefiting young children.
1. What are the unmet treatment needs in malaria for infants and children, and how do child-friendly medicines help?
At the moment, there are limited child-friendly antimalarial options and a lack of evidence-based guidelines for treatment of malaria in the very young (≤ 6 months of age or ≤ 5 kg). Incorrect treatment – for example the wrong dose or failure to take the full course of treatment – can increase the risk of severe malaria and death in this already vulnerable population. Formulations that make treatment easier to administer have received a positive response from caregivers and patients alike. It is of paramount importance to have good child-friendly formulations available to improve compliance.
2. Why is it important to have a range of quality-assured treatment options for children?
First, quality-assurance of treatments by the WHO and inclusion in the Model List of Essential Medicines for Children are important steps in the process of improving uptake of child-friendly medicines. Second, giving non-child-friendly medicines to children significantly increases the risk of noncompliance, and, as a consequence, the risks of severe malaria and death. Third, the availability of multiple first-line treatment combinations, and partner drugs with different mechanisms of action, might also delay the appearance of resistance.
The introduction of Pyramax granules will provide additional treatment options for children in Central Africa. Pyramax tablets and granules provide particular benefit as dosing is unaffected by food intake, an important consideration in this region.
The cost of the child-friendly formulation compared with the adult one is a potential limitation, however, as some regions may be less likely to switch to the more expensive formulation. MMV has already had a positive impact in this area by increasing the market supply of child-friendly formulations, but it is important that this and other work to address the issue continues.
3. What is the goal of the CANTAM study and how is it progressing?
The CANTAM study is a phase IIIb/IV study, designed to gather safety and tolerability information about Pyramax in ‘real-life’ settings, including in young children (under 1 year). The study was requested by the EMA to be carried out in parallel to the launch/ scale-up of Pyramax in malaria-endemic countries.
The increased breadth of pharmacovigilance and range of safety data collected is expected to support the wider uptake of both Pyramax tablets and granules at country level. Recruitment has been progressing very well, with over 3,000 patients, including 70 children under 1 year of age, recruited by the end of May 2018 in the five participating countries, including almost 800 in Gabon, up to April 2018 (planned recruitment: 8,572 malaria episodes). The results are expected in 2020.
4. What is it like to partner with MMV on this study?
It is a privilege to partner with MMV. MMV’s involvement with antimalarial trials and their drug development pipeline are very important factors. The improved laboratory facilities and good clinical practice-compliant environment created for the study with support from MMV, make a great contribution to capacity building in our African research settings – I look forward to further collaborations with MMV.