描述实验性药物的扩大使用及同情用药项目。
Characterizing expanded access and compassionate use programs for experimental drugs.
作者信息
Miller Jennifer E, Ross Joseph S, Moch Kenneth I, Caplan Arthur L
机构信息
Division of Medical Ethics, Department of Population Health, NYU School of Medicine, 227 East 30th Street, Office 723, New York, NY, 10016, USA.
Bioethics International, New York, USA.
出版信息
BMC Res Notes. 2017 Jul 28;10(1):350. doi: 10.1186/s13104-017-2687-5.
OBJECTIVE
We sought to determine the characteristics of "expanded access" and "compassionate use" programs registered in ClinicalTrials.gov and to determine the percentage of drugs provided through these programs that ultimately received FDA marketing approval.
RESULTS
We identified 398 expanded access and compassionate use programs (hereafter referred to as expanded access programs) registered on ClinicalTrials.gov. Industry funded 61% (n = 241) of programs individually or collaboratively, while NIH and the US Federal Government rarely funded programs (3% [n = 11] and 2% [n = 6], respectively). Most programs provided access to drugs (71% [n = 282]), 11% to biologics (n = 43), and 10% to medical devices (n = 40). These programs covered 460 unique conditions, the most common being HIV (n = 26), leukemia (22), and multiple myeloma (n = 14). Only 2% of programs reported results in ClinicalTrials.gov. Most programs (82%) were open to enrolling adults and seniors (n = 326). These programs provided access to 210 unique experimental drugs, of which 76% have received FDA approval.
目的
我们试图确定在ClinicalTrials.gov上注册的“扩大使用”和“同情用药”项目的特征，并确定通过这些项目提供的最终获得美国食品药品监督管理局（FDA）上市批准的药物所占比例。
结果
我们在ClinicalTrials.gov上识别出398个扩大使用和同情用药项目（以下简称扩大使用项目）。行业单独或合作资助了61%（n = 241）的项目，而美国国立卫生研究院（NIH）和美国联邦政府很少资助项目（分别为3% [n = 11]和2% [n = 6]）。大多数项目提供药物使用途径（71% [n = 282]），11%提供生物制品使用途径（n = 43），10%提供医疗器械使用途径（n = 40）。这些项目涵盖460种独特病症，最常见的是艾滋病病毒（HIV）（n = 26）、白血病（22）和多发性骨髓瘤（n = 14）。只有2%的项目在ClinicalTrials.gov上报告了结果。大多数项目（82%）对招募成年人和老年人开放（n = 326）。这些项目提供了210种独特实验药物的使用途径，其中76%已获得FDA批准。
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