赞助商与FDA倡导的儿童临床试验协议的变化:关联参与者负担的比较
Susan Abdel-Rahman1, Zoe Sund2, Cheryl Alderman2
1Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA. susan.abdel-rahman@fda.hhs.gov.
Therapeutic innovation & regulatory science
|February 23, 2025
概括
临床试验协议修正案中的一半增加了参与者的负担,FDA提出的变化往往带来更高的分数. 在修改过程中重新评估试验元素可以减轻额外的负担.
科学领域:
- 临床试验 临床试验
- 儿科研究 儿科研究
- 监管科学 监管科学
背景情况:
- 临床试验的风险优先于负担,影响招生和保留.
- 美国食品和药物管理局 (FDA) 强调负担是参与研究的障碍.
- 这项研究调查了FDA在儿科标签研究中提出的变化所带来的负担.
研究的目的:
- 检查FDA在儿科,非专利,标签试验中提出的研究修改的感知负担.
- 为了比较赞助商发起的和FDA推的协议变更之间的参与者负担.
- 评估儿科临床试验者和儿童之间的负担得分的一致性.
主要方法:
- 审查了21项儿科试验网络 (PTN) 研究 (2013-2023) 与PTN-FDA正式沟通.
- 将影响参与者负担的协议变化提取并分类为11个主题.
- 被盲目的试验者和儿童使用利克特级别分配感知负担得分.
主要成果:
- 确定了123个影响负担的协议变化;77个由赞助商发起,46个由FDA提出.
- 增加或扩展协议特征的变化 (64%) 比减少它们的变化 (36%) 更常见.
- 食品和药物管理局的变化,主要是为了安全,比赞助商的变化有更高的平均负担得分.
结论:
- 协议修正经常改变参与者的负担,往往增加它.
- 美国食品和药物管理局推的变化虽然以安全为重点,但往往会增加负担.
- 建议在修改过程中重新评估所有协议元素,以管理参与者负担.
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