[罕见癌症药物开发的现状和问题]
Yuji Kashitani1, Hironori Takayama, Natsuko Hamada
1Regulatory Affairs Committee, Japan Pharmaceutical Manufacturers Association.
Gan to kagaku ryoho. Cancer & chemotherapy
|December 6, 2023
概括
日本 日本 日本 日本 日本.
科学领域:
- 制药发展 制药发展
- 监管事务 监管事务
- 罕见疾病是一种罕见的疾病.
背景情况:
- 日本的孤儿药物和孤儿医疗器械研发推广系统,成立于1993年,支持罕见疾病药物开发.
- 指定通常是影响<50,000名患者的疾病,其未满足需求和发展概率很高.
- 最近的趋势表明,由于严格的数据要求,孤儿药物名称在开发过程中出现较晚.
研究的目的:
- 分析日本孤儿药物开发系统的现状和挑战.
- 确定延迟指定的原因,并提出系统改革.
- 探索改善患者获得罕见疾病治疗的策略.
主要方法:
- 在日本对孤儿药物指定趋势的分析.
- 日本批准的孤儿药物与美国和欧盟的比较.
- 检查日本患者参与多区域临床试验 (MRCT) 的障碍.
主要成果:
- 孤儿药物名称越来越多地发生在晚期开发阶段.
- 在美国/欧盟批准的大量抗癌孤儿药在日本未获得批准.
- 日本对本地安全数据的要求可能会阻止及时参与全球临床试验,阻碍超罕见疾病的发展.
结论:
- 日本的孤儿药物系统需要进行根本性改革,以促进罕见病药物开发.
- 扩大孤儿药物指定和预算拨款是最初的步骤.
- 实施早期访问审查系统和促进MRCT参与对于及时的患者访问至关重要.
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