转变客观参与型患者倡导
Pat Furlong1, Timothy R Franson2, Peter J Pitts3
1Parent Project Muscular Dystrophy (PPMD), Washington, DC, USA.
Therapeutic innovation & regulatory science
|July 21, 2025
概括
参与性患者倡导 (PPA) 使用数据来放大患者在罕见疾病药物开发中的声音. 这种战略方法通过将患者需求纳入研究和监管流程来加速治疗批准.
科学领域:
- 药理学和药物开发领域
- 患者倡导和参与 患者倡导和参与
- 监管科学与政策 监管科学与政策
背景情况:
- 传统的患者倡导正在向以数据为导向的战略方法发展,称为参与性患者倡导 (PPA).
- 通过集中患者的声音,PPA显著影响药物开发和监管过程,特别是在罕见疾病中.
- 父母项目肌肉发育不良 (PPMD) 作为一个关键的例子,展示了PPA如何加速杜申肌肉发育不良 (DMD) 的治疗开发和批准.
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