调查日本医疗技术评估中制造商和公众分析之间的差异
Yoko Hirano1, Akira Yuasa1, Karin Matsumoto2
1Japan Access & Value Pfizer Japan, Inc., Tokyo.
Journal of health economics and outcomes research
|November 4, 2025
概括
日本的医疗技术评估 (HTA) 系统显示了制造商和公共成本效益分析之间的不一致性,特别是对于孤儿药物和独特的好处. 对于更可预测的药品定价评估,需要更明确的指导.
科学领域:
- 卫生经济学 卫生经济学
- 制药政策 制药政策
- 卫生技术评估 卫生技术评估
背景情况:
- 日本的药品定价制度还原了批准的药品,并于2019年推出了健康技术评估 (HTA) 系统.
- 医疗药品评估系统通过补偿后的成本效益评估来调整创新型,高预算药物的价格.
研究的目的:
- 为了分析制造商与公众 (C2H) 之间的差异,对日本HTA系统下的31种产品进行了成本效益分析.
- 确定导致这些差异的因素,重点关注孤儿药物和独特的产品属性.
主要方法:
- 对公开可用的制造商和C2H成本效益报告进行描述性分析.
- 对额外收益,增量成本效益比率 (ICER) 和重新分析项目的评估进行比较.
- 调查与孤儿药物和超出QALYs的实用性溢价产品相关的问题.
主要成果:
- 48.6%的分析群体显示,制造商和C2H之间的效益,结果或方法不一致.
- 在2022年4月指南修订后,不一致性增加了.
- ICER差异与QOL参数,基线假设,孤儿药物处理和非QALY属性有关.
结论:
- 在评估QOL,孤儿药物和非QALY属性方面,在制造商和C2H之间存在显著差异.
- 更明确的指导和考虑国际HTA系统可以提高评估的一致性.
- 这些发现旨在为更透明和可预测的日本药品定价评估提供精细信息.
相关概念视频
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
395
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
395
01:28Drug Products: Biologics, Biosimilars and Interchangeables
223
Body:Biologics, derived from living sources such as humans, animals, or microorganisms, represent a significant category of pharmaceuticals. These complex molecules, developed through advanced biotechnological methods or purified from natural sources, include essential medical treatments like insulin and growth hormones. The complexity of biologics arises from their large molecular structures and the intricate processes required for their production, making them distinct from conventional...
223
01:30Health Information Technology and Healthcare Information System
3.3K
Health Information Technology (HIT)
Health Information Technology, commonly called HIT, integrates advanced information systems and technology in healthcare settings. Its primary functions include:
Health Information Technology, commonly called HIT, integrates advanced information systems and technology in healthcare settings. Its primary functions include:
3.3K
01:13Bioequivalence studies: Biowaivers
214
Body:In certain scenarios, in vitro dissolution tests can replace in vivo bioequivalence studies. This is particularly true when a drug product, though available in varying strengths, maintains proportional similarity in its active and inactive ingredients. In such cases, the need for in vivo bioequivalence studies for lower strength variants may be waived, provided dissolution tests and in vivo studies on the highest strength yield satisfactory results.Bioequivalence can be indicated through...
214
01:19Pharmacovigilance
1.6K
Post-marketing surveillance is a critical component of pharmaceutical regulation, often uncovering unanticipated adverse drug reactions (ADRs) once a drug is widely used over an extended period.
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
1.6K
01:16Bioequivalence Data: Statistical Interpretation
187
Body:The statistical interpretation of bioequivalence data is a significant aspect of pharmaceutical research. Bioequivalence refers to the absence of any significant difference in the rate and extent to which the active ingredient in pharmaceutical products becomes available at the site of drug action when administered at the same molar dose under similar conditions. This helps determine if different drug products have similar absorption rates, ensuring their interchangeability.Statistical...
187