PDF(Pubmed)
Abstract：
UNASSIGNED: An expanded access clinical trials (EACTs) provides exceptional patient access to investigational new drugs for life-threatening diseases for which no effective treatment exists. Based on public information, we have studied EACTs since 2016, when the EACT system was launched in Japan. In this study, we investigated the reality of EACTs by interviewing pharmaceutical companies and clarifying how they view them.
UNASSIGNED: We conducted semi-structured interviews with 10 pharmaceutical companies developing new drugs. This study aims to clarify the status of EACTs, so we selected pharmaceutical companies that develop innovative drugs for which they may perform EACTs (however, experience in conducting EACTs was optional).
UNASSIGNED: All those surveyed were aware of EACTs. Twelve access clinical trials were conducted, and the EACT implementation rate for pivotal clinical trials was 2.5%. The most common reason for implementing an EACT was \"requests from physicians and medical institutions\" (nine companies, 90.0%), and the most common reason for not implementing an EACT was \"the applicability of the system\" (five companies). Improvements to EACTs were identified by eight companies (80.0%); financial assistance by six companies (60.0%); reducing the scope of data to be collected and simplifying the procedure by six companies (60.0%). Seven companies (70.0%) responded that a Single Patient Investigational New Drug Application should be conducted, suggesting that the system should be revised.
UNASSIGNED: An interview survey of ten pharmaceutical companies developing new drugs in Japan regarding expanded access clinical trials indicated that there were issues with the system. Many wished to improve the system by establishing a single patient access system, supporting resources, and simplifying procedures. Based on our interviews with 10 Japanese pharmaceutical companies, it was found that the system needed to be improved by introducing a single patient access system, providing supporting resources, and simplifying procedures. In Japan, about eight years have passed since EACT was established, and it appears a revision of the EACT legislation is due.
UNASSIGNED: We conducted semi-structured interviews with 10 pharmaceutical companies developing new drugs. This study aims to clarify the status of EACTs, so we selected pharmaceutical companies that develop innovative drugs for which they may perform EACTs (however, experience in conducting EACTs was optional).
UNASSIGNED: All those surveyed were aware of EACTs. Twelve access clinical trials were conducted, and the EACT implementation rate for pivotal clinical trials was 2.5%. The most common reason for implementing an EACT was \"requests from physicians and medical institutions\" (nine companies, 90.0%), and the most common reason for not implementing an EACT was \"the applicability of the system\" (five companies). Improvements to EACTs were identified by eight companies (80.0%); financial assistance by six companies (60.0%); reducing the scope of data to be collected and simplifying the procedure by six companies (60.0%). Seven companies (70.0%) responded that a Single Patient Investigational New Drug Application should be conducted, suggesting that the system should be revised.
UNASSIGNED: An interview survey of ten pharmaceutical companies developing new drugs in Japan regarding expanded access clinical trials indicated that there were issues with the system. Many wished to improve the system by establishing a single patient access system, supporting resources, and simplifying procedures. Based on our interviews with 10 Japanese pharmaceutical companies, it was found that the system needed to be improved by introducing a single patient access system, providing supporting resources, and simplifying procedures. In Japan, about eight years have passed since EACT was established, and it appears a revision of the EACT legislation is due.
摘要：
■扩大的访问临床试验（EACTs）为患者提供了出色的机会，以治疗尚无有效治疗方法的威胁生命的疾病的研究新药。根据公共信息，自2016年在日本推出EACT系统以来，我们一直在研究EACT。在这项研究中,我们通过采访制药公司并澄清他们如何看待EACTs来调查EACTs的现实。
■我们对10家开发新药的制药公司进行了半结构化访谈。本研究旨在阐明EACTs的现状，因此，我们选择了开发创新药物的制药公司，他们可以执行EACTs(然而，进行EACT的经验是可选的)。
■所有被调查的人都知道EACT。进行了12项访问临床试验，关键临床试验的EACT执行率为2.5%.实施EACT的最常见原因是“医生和医疗机构的要求”(9家公司，90.0%)，没有实施EACT的最常见原因是“系统的适用性”(五家公司)。8家公司（80.0%）对EACT进行了改进；6家公司（60.0%）提供了财政援助；6家公司（60.0%）减少了收集数据的范围，简化了程序。七家公司（70.0％）回应说，应进行单患者研究新药申请，建议修改该系统。
■对十家在日本开发新药的制药公司进行的关于扩大访问临床试验的访谈调查表明，该系统存在问题。许多人希望通过建立单一患者进入系统来改善该系统，支持资源,简化程序。根据我们对10家日本制药公司的采访，发现需要通过引入单个患者进入系统来改进该系统，提供配套资源,简化程序。在日本,自EACT成立以来已经过去了大约八年，似乎应该对EACT立法进行修订。
■我们对10家开发新药的制药公司进行了半结构化访谈。本研究旨在阐明EACTs的现状，因此，我们选择了开发创新药物的制药公司，他们可以执行EACTs(然而，进行EACT的经验是可选的)。
■所有被调查的人都知道EACT。进行了12项访问临床试验，关键临床试验的EACT执行率为2.5%.实施EACT的最常见原因是“医生和医疗机构的要求”(9家公司，90.0%)，没有实施EACT的最常见原因是“系统的适用性”(五家公司)。8家公司（80.0%）对EACT进行了改进；6家公司（60.0%）提供了财政援助；6家公司（60.0%）减少了收集数据的范围，简化了程序。七家公司（70.0％）回应说，应进行单患者研究新药申请，建议修改该系统。
■对十家在日本开发新药的制药公司进行的关于扩大访问临床试验的访谈调查表明，该系统存在问题。许多人希望通过建立单一患者进入系统来改善该系统，支持资源,简化程序。根据我们对10家日本制药公司的采访，发现需要通过引入单个患者进入系统来改进该系统，提供配套资源,简化程序。在日本,自EACT成立以来已经过去了大约八年，似乎应该对EACT立法进行修订。
