通过优化患者参与,帮助采用主协议
Raymond A Huml1, Deborah Collyar2, Zoran Antonijevic3
1Syneos Health Clinical Solutions, Morrisville, NC, USA. raymond.huml@syneoshealth.com.
Therapeutic innovation & regulatory science
|August 24, 2023
概括
将患者参与 (PE) 整合到总体协议 (MP) 中可以提高临床试验的成功率. 这种方法有利于患者,提供量身定制的治疗方法,增加选择,并加速获得创新的治疗方法,特别是对于罕见疾病.
科学领域:
- 临床试验设计 临床试验设计
- 药物开发 药物开发
- 以患者为中心的研究
背景情况:
- 总体协议 (MP) 是一种灵活的临床试验设计,允许多个子研究来评估疗法.
- 像FDA和EMA这样的监管机构强调了患者参与的重要性.
- 目前的MP框架可以通过结合患者的观点来改进.
研究的目的:
- 探索将患者参与原则纳入总体协议开发和行为中的好处.
- 为利益相关者,包括药物开发商和患者倡导团体,提供关于创建更有效的临床试验的见解.
- 突出患者的声音如何提高所有参与方的主协议的成功.
主要方法:
- 审查现有的文献和监管指导关于总体协议和患者参与的指导意见.
- 在MP框架内分析PE对患者,赞助商和提供者的潜在好处.
- 来自美国统计学会 (ASA) /DahShu主协议工作组 (MPWG) 人与患者参与 (PE) 小组的专家观点.
主要成果:
- 将PE纳入MP可以带来更适合患者需求的治疗方法.
- 患有PE的MP可以增加治疗的可用性,并加快患者获得新疗法的机会.
- 在MP中PE可以促进对罕见疾病和儿科患者的创新治疗方法的开发.
- 在MP中共享控制手臂可以降低患者接受安慰剂的可能性.
结论:
- 整合患者参与到总体协议中是一个科学合理的方法,以提高临床试验的效率和患者的结果.
- 这种整合为药物开发商,医疗保健提供者和患者提供了显著的优势,特别是在罕见疾病研究等领域.
- 建议在MP中进一步开发和实施PE,以最大限度地发挥其促进治疗发展的潜力.
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