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罕见疾病的研究和开发景观不断发展:日本对孤儿药物滞后的担忧越来越大
Kazuaki Enya1, Itsuki Kageyama2, Yoshiyuki Kobayashi2
1Graduate School of Design and Architecture, Nagoya City University, Nagoya, Japan.
Clinical pharmacology and therapeutics
|February 4, 2025
概括
由于制药公司的影响,日本的药物滞后状况正在恶化.
科学领域:
- 罕见疾病药物开发 罕见疾病药物开发
- 药品监管事务 药品监管事务
- 全球卫生政策 全球卫生政策
背景情况:
- 在全球范围内,患有罕见疾病的患者经历了大量未满足的医疗需求.
- 与其他国家相比,日本药物批准的延迟仍然是一个关键的公共卫生问题.
- 由于制药公司战略的转变,孤儿药物研发 (R&D) 的景观正在演变.
研究的目的:
- 分析2005年至2021年美国孤儿药物 (OD) 批准趋势.
- 检查导致日本OD滞后复苏的因素.
- 评估当前的研发模式,并提出简化日本OD开发的战略.
主要方法:
- 美国孤儿药物批准的回顾性分析 (2005-2021年).
- 检查大型制药公司和中小型企业 (SME) 使用的研发策略.
- 对影响日本的执照实践和全球发展战略的分析.
主要成果:
- 尽管自2018年以来,美国的OD批准数量增加,但日本未经批准的OD数量有所增加.
- 孤儿药物滞后,经过一段时间的下降,自2017年以来再次出现.
- 制药公司的研发策略,特别是美国和欧洲的中小企业的起以及它们的授权做法,大大导致了日本的OD滞后增加.
结论:
- 大型制药公司越来越多地采用全球发展战略,从中小企业获得早期候选药物的许可.
- 日本制药公司经常为日本市场提供后期候选人的许可证,加剧了OD滞后.
- 日本制药公司和中小企业之间的积极的全球合作伙伴关系对于解决日益增长的OD滞后至关重要.
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