a Desmond Tutu TB Centre, Department of Paediatrics and Child Health, Faculty of Medicine and Health Sciences , Stellenbosch University , Cape Town , South Africa.
b Treatment Action Group , New York , NY , USA.
Expert Rev Clin Pharmacol. 2018 Mar;11(3):233-244. doi: 10.1080/17512433.2018.1421067. Epub 2017 Dec 27.
New and repurposed antituberculosis drugs are urgently needed to more safely and effectively treat multidrug-resistant (MDR) tuberculosis (TB) in children. Multiple challenges limit timely access to new MDR-TB treatments in children. Areas covered: Diagnosis of MDR-TB in children remains a barrier, with few children with MDR-TB diagnosed and treated. Other barriers to timely access to new and repurposed drugs are discussed, and include delayed initiation of paediatric trials, limited funding for paediatric drug development, fragmented regulatory systems and operational challenges. The status of access to current repurposed and novel drugs is presented. Expert commentary: More timely initiation of paediatric trials is needed and paediatric work should happen and be funded in parallel with each phase of adult trials. Better quality data, increased regulator resources and expertise, harmonization of regulatory requirements across borders/organisations and registration fee waivers would improve registration timelines. Improved diagnosis, recording and reporting will establish better demand. Improved systems for procurement and supply chain management would reduce in-country operational barriers to getting medications to children. The challenges must be addressed to ensure timely and equitable access to new drugs and regimens that are urgently needed for effective, safe and shorter treatment of children with MDR-TB.
新的和重新利用的抗结核药物迫切需要更安全、更有效地治疗儿童耐多药结核病（TB）。多种挑战限制了儿童获得新的耐多药结核病治疗的及时性。
儿童耐多药结核病的诊断仍然存在障碍，只有少数儿童被诊断和治疗。讨论了及时获得新的和重新利用的药物的其他障碍，包括儿科试验启动延迟、儿科药物开发资金有限、监管系统分散和运营挑战。介绍了当前重新利用和新型药物的获取状况。
需要更及时地启动儿科试验，并且应该在成人试验的每个阶段同时进行儿科工作并为其提供资金。更好的质量数据、增加监管机构的资源和专业知识、协调跨境/组织的监管要求以及免除注册费将缩短注册时间表。改进诊断、记录和报告将建立更好的需求。改进采购和供应链管理系统将减少国内获得药物的运营障碍，使儿童能够获得药物。必须解决这些挑战，以确保及时和公平地获得新的药物和方案，这些药物和方案对于有效、安全和缩短儿童耐多药结核病的治疗时间是迫切需要的。
