在瘤学药物中,为患者群体的推不断发展:定量和定性分析
Milou A Hogervorst1, Rick A Vreman1, Theresa A Oduol2
1Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, The Netherlands.
Clinical pharmacology and therapeutics
|March 10, 2025
概括
关于瘤医学患者群体的决定在监管,HTA和指导机构之间存在很大差异. 这种不一致性使患者的访问和沟通复杂化,突出了对标准化报告框架的需求.
科学领域:
- 在瘤学瘤学.
- 卫生政策 卫生政策
- 制药医学 制药医学
背景情况:
- 瘤学药物的患者群体由制药开发商,监管机构,健康技术评估 (HTA) 组织和专业协会顺序定义.
- 这些定义从关键试验发展到通过监管提交,营销许可,HTA审查和临床指南开发.
- 定义患者群体的变化可能会影响患者获得和理解药物指示.
研究的目的:
- 评估瘤药物患者群体在药物生命周期的不同阶段以及各种决策机构如何定义.
- 识别和量化用于描述瘤患者群体的元素的变化.
- 制定一个框架,以便对瘤病患者群体进行一致的报告.
主要方法:
- 开发了一个框架,包括四个领域的20个元素 (疾病特征,患者特征,治疗位置,排除标准) 来描述瘤病患者群体.
- 该框架应用于25个瘤医学群体,涉及6个决策步骤 (试验,监管提交,营销许可,HTA提交,HTA建议,临床指南).
- 探索性分析对整个药物生命周期以及澳大利亚,加拿大,荷兰,英国和美国不同决策者的框架元素的表格变化进行了分析.
主要成果:
- 平均而言,在药物生命周期中,对患者群体定义进行了10次调整,每个决策者贡献了大约2.3次调整.
- 制药开发人员主要调整疾病规范,而监管机构,HTA组织和指导方针开发人员则专注于治疗位置.
- 决策者经常修改其他人先前调整的元素,表明人口定义缺乏一致的进展.
结论:
- 在不同决策机构和阶段对瘤药物患者群体的定义中存在显著的不一致性.
- 这些变异使与患者的沟通复杂化,并可能影响获得创新的癌症治疗方法的公平机会.
- 开发的框架提供了一个工具,以促进利益相关者和国际上对瘤病患者群体的一致和透明报告.
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