关于孤儿药物定价的辩论:一个元叙事文献综述
1iHuman, Social Research Institutes, University of Sheffield, The Wave, 2 Whitham Road, S10 2AH, Sheffield, UK. m.s.hanchard@sheffield.ac.uk.
Orphanet journal of rare diseases
|March 7, 2025
概括
罕见疾病药品成本上升给医疗保健系统带来了压力. 本综述确定了关于孤儿药物定价的三个叙述,发现需要以福利经济学和患者包容为中心的改革.
科学领域:
- 卫生经济学 卫生经济学
- 制药政策 制药政策
- 社会科学研究 社会科学研究
背景情况:
- 罕见疾病的流行率增加和孤儿药的高成本在全球范围内给医疗保健系统带来了压力.
- 有关孤儿药物定价的利益相关者之间存在重大紧张关系,缺乏统一的文献评论.
- 关于孤儿药物定价的现有话语缺乏与各种利益相关者叙述及其基本原则的关键接触.
研究的目的:
- 批判性地审查和综合围绕孤儿药物定价的各种叙述.
- 确定孤儿药物定价辩论中的核心论点,基础假设和争议点.
- 提出一个统一的元叙事,并建议为孤儿药物创新和获取政策改革.
主要方法:
- 进行了一项元叙事文献审查,以分析关于孤儿药物定价的叙事.
- 检查学术,活动家,倡导,行业和政策文献.
- 综合发现,以确定共同的主题,分歧和潜在的共识领域.
主要成果:
- 确定了关于孤儿药物定价的三个核心叙述: 1) 批评由于开发成本和断资本主义而导致的高价格, 2) 为市场健康和创新辩护高价格, 3) 建议替代创新和估值模型.
- 一个一致的改革呼吁出现,强调福利经济学和资源分配.
- 目前的激励措施和法规被认为不足以解决孤儿药物定价和获取的复杂问题.
结论:
- 监管机构应该探索由社会科学为基础的替代创新模式,以改善孤儿药物的评估,覆盖和报销流程.
- 增加患者参与和透明度对于将社会因素纳入孤儿药物获取考虑因素至关重要.
- 改革孤儿药物政策需要向福利经济学转变,并更广泛地理解传统市场指标之外的价值.
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