Designing drug products suitable for the youngest patient. Enabling access to medicines for the most vulnerable and complex patient population.

开发两岁以下儿童口服配方对开发两岁以上儿童口服配方带来了许多不同的多方面挑战，例如赋形剂负担、剂量量、给药装置要求、对喂养的影响等。事实上，这部分儿科人口在某种程度上仍然是治疗孤儿，这不仅是因为临床需求未得到满足，而且还因为缺乏经过验证的剂型或技术，无法实现口服给药。这项以化学、数据收集、工程和材料科学为界面的制药技术研究提供了一个绝佳的机会，可以通过以下方式大大改善患者的治疗效果：1)了解这一独特患者群体的需求/研究能力；2)根据这些要求评估当前可用的口服剂型类型；3)开发适合这一年龄组的平台。这类药物输送平台还需要考虑其他因素，如市场因素，如成本；需要个性化给药，如肿瘤、慢性或急性给药和文化方面的考虑。创造性和变革性研究方法的例子包括“凝胶分散片剂”的可行性，该片剂可在勺子上形成凝胶，以帮助新生儿给药，这可能符合低成本地区的要求；分散迷你片剂或颗粒剂的可行性；orodispersible电影;或者基于泡沫的输送系统可能更适用于剂量灵活性至关重要和/或成本限制不是关键驱动因素的利基疗法。这一高度优先的卫生保健挑战是儿科给药的一个关键领域，对该领域知之甚少，而且已证实的平台的可用性有限。它真正鼓励了转化研究，并开辟了影响的途径。

Developing oral formulations for children under 2 years of age pose many different multifaceted challenges to those associated with the development of oral formulations for children over 2 years of age e.g. excipient burden, dose volume, dosing device requirements, impact on feeding etc. In fact, this subset of the paediatric population remain somehow therapeutic orphans not only due to unmet clinical needs but also due to a lack of proven dosage forms or technology, to enable oral dosing. This pharmaceutical technology focused research at the interface of chemistry, data gathering, engineering and materials sciences provides a fantastic opportunity to greatly improve patient outcomes by i) understanding the needs/research capabilities of this unique patient population ii) evaluating the current available oral dosage form types against these requirements andiii) developing a platform that is appropriate for this age group. Such drug delivery platforms need to also consider other factors such as market considerations e.g. cost; need for personalised dosing e.g. oncology, chronic or acute dosing and cultural considerations. Examples of creative and transformational approaches to investigate include the feasibility of a 'gelling dispersible tablet' that forms a gel on a spoon to aid dosing to neonates which may meet the requirements of low cost territories; feasibility of dispersible mini tablets or granules; orodispersible films; or foam based delivery systems that might be more applicable to niche therapies where dose flexibility is critical and/or cost constraints are not a key driver. This high priority healthcare challenge is a critical area of paediatric drug delivery of which little is known and where availability of proven platforms is limited. It truly encourages translational research and opens up pathways to impact.