年轻患者参与组合终点方法的开发,对儿科口服剂型的可接受性进行研究
Sibylle Reidemeister1, Begonya Nafria Escalera2,3,4, Daniel Marín2
1Global Drug Development, Novartis Pharma AG, WSJ-188 10 001, 4056, Basel, Switzerland.
Research involvement and engagement
|November 30, 2023
概括
年轻患者证实了一种评估药物可接受性的新方法的价值,他们更喜欢口感和可吞性评估. 他们的投入指导着儿童友好的药物的开发.
科学领域:
- 儿科医学的发展.
- 患者参与制药研究的参与.
- 药物配方的可接受性 药物配方的可接受性
背景情况:
- 欧洲药品管理局 (EMA) 要求在儿童药物开发中对药物的可接受性进行评估.
- 开发了一种标准化的方法来评估"可吞性"和"可食性"成为可接受性的复合终点.
- 需要年轻患者进行批判性审查,以验证这种新的可接受性测试方法.
研究的目的:
- 评估复合终点对年轻患者接受度方法的适用性.
- 调查患者参与药物开发和可接受性方法创建的重要性.
- 确定评估儿童和青少年药物可接受性的最相关因素.
主要方法:
- 与KIDS巴塞罗那青年咨询小组 (16-23岁) 举行了焦点小组会议.
- 电子问卷被用于调查患者参与和接受性评估的偏好.
- 使用贴纸和问卷评估了EMA列出的可接受性要素的相关性.
主要成果:
- 对年轻人参与方法开发的评价很高.
- 对于6-11岁的孩子来说,5分微笑式的利克特尺度是首选的,对于青少年来说,视觉模拟尺度是首选的.
- 味道和吞性被确定为最相关的,而药品颜色是最不相关的.
结论:
- 该研究加强了让年轻人参与药物生命周期和可接受性方法开发的价值.
- 计划在欧洲各地与多样化的年轻人群体进行进一步的焦点小组.
- 这项研究支持开发更可接受的儿科药物.
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