University of Buenos Aires, Faculty of Pharmacy and Biochemistry, Department of Pharmaceutical Technology, The Group of Biomaterials and Nanotechnology for Improved Medicines, 956 Junín St., Buenos Aires CP1113, Argentina.
Expert Opin Drug Deliv. 2012 Mar;9(3):303-23. doi: 10.1517/17425247.2012.655268. Epub 2012 Jan 19.
Due to a lack of approved drugs and formulations, children represent the most vulnerable patients. Magistral, unlicensed formulations obtained by the manipulation of solid forms should undergo clinical evaluation to ensure bioequivalence. The development of new pediatric medicines is complex and faces technological, economic and ethical challenges. This phenomenon has contributed to the emergence of an adult-children gap. To improve the situation, the World Health Organization launched the global campaign 'Make medicines child size' and a number of international initiatives have been established. The situation is more critical in the case of poverty-related diseases (PRDs) that mainly affect poor countries.
This review critically discusses different strategies to develop pediatric formulations and drug delivery systems (DDS) in PRDs and their potential implementation in the current market. Readers will gain an updated perspective on the development of pediatric medicines for the treatment of PRDs and the proximate challenges and opportunities faced to ensure an effective pharmacotherapy.
There is an urgent need for the development of innovative, scalable and cost-viable formulations to ensure pediatric patients have access to appropriate medications for PRDs. The guidelines of the International Conference on Harmonisation constitute a very good orientation tool, as they emphasize physiological and developmental aspects that need to be considered in pediatric research. It is important to consider cultural, economic and ethical aspects that make developing nations facing PRDs different from the developed world. Thus, the best strategy would probably be to conceive and engage similar initiatives in the developing world, to address unattended therapeutic niches.
由于缺乏批准的药物和制剂，儿童是最脆弱的患者群体。通过对固体形式进行操作获得的非法定剂必须经过临床评估，以确保生物等效性。新儿科药物的开发复杂且面临技术、经济和伦理挑战。这种现象导致了成人-儿童差距的出现。为了改善这种情况，世界卫生组织发起了全球运动“让药物适合儿童”，并建立了许多国际倡议。在与贫困相关的疾病（PRD）的情况下，情况更为关键，这些疾病主要影响贫穷国家。
本文批判性地讨论了在 PRD 中开发儿科制剂和药物输送系统（DDS）的不同策略，以及它们在当前市场中的潜在实施。读者将获得关于治疗 PRD 的儿科药物开发的最新观点，以及为确保有效的药物治疗而面临的直接挑战和机遇。
迫切需要开发创新、可扩展和具有成本效益的制剂，以确保儿科患者能够获得针对 PRD 的适当药物。人用药品注册技术要求国际协调会议的指导原则是一个很好的导向工具，因为它们强调了儿科研究中需要考虑的生理和发育方面。考虑到使面临 PRD 的发展中国家与发达国家不同的文化、经济和伦理方面非常重要。因此，最好的策略可能是在发展中国家构思并参与类似的倡议，以解决未得到满足的治疗需求。
