建模和模拟以及现实世界的证据的关键作用,以告知临床试验多样性规划中的挑战
Ananth Kadambi1, Elke H J Krekels2, Antal Martinecz3
1Certara USA, Radnor, Pennsylvania, USA.
Clinical and translational science
|June 17, 2025
概括
临床试验的多样性对于现实世界的患者安全至关重要. 建模和模拟 (M&S) 和现实世界的证据 (RWE) 可以优化多样性规划并改善药物开发结果.
科学领域:
- 药物开发 药物开发
- 临床试验 临床试验
- 健康 公平 卫生 公平
背景情况:
- 临床试验往往缺乏多样化的患者代表,导致批准后的安全问题.
- 监管机构强调在临床试验中需要多样化的群体,以获得现实世界的有效性.
- 目前的试验多样性规划方法需要改进,以满足监管的期望.
研究的目的:
- 在临床试验多样性规划中探索建模与模拟 (M&S) 和现实世界的证据 (RWE) 的战略价值.
- 确定未满足的需求,并突出利用M&S和RWE实现多样性的优先思想领导力.
- 提供对整个药物开发生命周期的多样性规划的见解.
主要方法:
- 在试验多样性中对M&S和RWE进行现有文献和案例的审查.
- 对有关临床试验多样性的监管指南的分析.
- 对将M&S和RWE整合到多样性计划中的战略方法的综合.
主要成果:
- M&S和RWE为优化临床试验多样性规划提供了强大的工具.
- 这些方法可以提高试验参与者的代表性,超出传统的流行病学数据.
- 早期整合M&S和RWE可以减轻批准后的风险,并提高药物的疗效.
结论:
- M&S和RWE对于制定强大且符合监管标准的临床试验多样性计划至关重要.
- 这些方法的战略实施可以改善患者安全和治疗结果.
- 需要对M&S和RWE进行进一步的研究和应用,才能充分发挥其在试验多样性方面的潜力.
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