[tr=transparent]让ALS患者的访问情况下的IND FDA的位置[tr=transparent]今天，美国食品和药物管理局与公共决策让肌萎缩性侧索硬化症患者共享，一个致命的神经退行性疾病，也被称为卢伽雷氏症，或ALS，获得药物的情况下被称为研究性新药（IND）申请。IPLEX（美卡舍明rinfabate [ rDNA来源]注射），是一个组合的两种物质：人胰岛素样生长因子1（IGF-1）和胰岛素样生长因子结合蛋白-3（rhIGFBP-3）。情况是由FDA仅在重度原发性IGF-1缺乏儿童生长障碍的治疗批准（原发性IGFD）和生长激素（GH）谁开发的GH中和抗体基因缺失。该药物目前没有销售因为一个专利侵权相关的法院命令。情况是由Insmed的生物制品公司，总部设在里士满，弗吉尼亚。
FDA的决定
[tr]FDA和Insmed同意访问情况患者调查使用ALS患者会发生在两方面研究性新药申请（IND）：
- [tr=transparent]单个病人只要求“IPLEX为命名的ALS患者治疗使用同情”，收到的日期由FDA的文件室的业务在3月6日关闭盖，
[tr]2009，将允许进行，和Insmed已同意向患者供应情况；及 - [tr=transparent]剩下的情况供应，这是很有限的，将由一个工业Insmed的其他ALS患者在接受治疗的人感兴趣的情况将被随机分配到通过摇号系统接受药物在进行临床试验的应用。
[tr=transparent]所有患者接受一个病人情况下或在Insmed IND临床试验必须充分利用他们的治疗医生的治疗可能带来的好处和风险告知。为了便于知情同意的过程中，FDA提供其他文件，如下所述，确保卫生保健提供者和患者获得更完整的信息，对治疗的潜在风险和收益情况相关。
[tr=transparent]FDA已经同意允许Insmed提交成本回收的要求，现有的工业法规下偏移进行临床试验计划相关的成本。医生为病人申请单提交的“同情使用”的情况将被要求参与数据收集的Insmed的临床试验，以最大限度地从药品的供给非常有限，数据收集。
Rationale for fda' s decision
[tr]FDA的决定是与ALS和药物的极其有限的供应带来的实际限制，患者需要认真的考虑后。该机构已经仔细审查了所有可用的研究和数据的潜在益处和风险ALS患者，以及需要有计划尽可能公平分配那些想得到它的患者的药物供应有限公司。
[tr]FDA已收到一些病人要求医生允许工业“同情使用”命名的ALS患者的情况。在允许调查使用的ALS患者的情况作出决定，FDA认为仅仅给予患者的药物种类单一下访问会快速消耗情况供应有限，使得它几乎不可能进行临床对照试验。这是至关重要的，因为没有足够的临床对照试验，不可能确定情况是有效的，或有害的，ALS患者。
[tr]FDA认为其决定代表尽可能公平的方式提供访问情况，第一，因为Insmed没有为每一个病人谁可能需要足够的药物，其次，因为它是最大化可以从药物的情况下，它有利益的剩余供应学习重要，并可能进一步发展通过广泛使用与ALS患者。
[tr]FDA已经试图平衡个体患者迫切寻求与需要学习如果药物实际上是有利的，或有害的这种破坏性疾病的治疗选择的需要，在ALS的治疗。这些因素都称过去几周通过FDA的科学家和医生，他们举行了一系列与Insmed和内部会议会议讨论的最佳途径。
年表
[tr]随着透明关于我们的决策意图，今天我们是让公众对ALS作为我们决策的依据患者情况使用现有的资料和研究结果。
Approximately 10 weeks ago, the FDA first received requests from physicians requesting access to Iplex for “compassionate use” treatment of named patients with ALS under single-patient INDs. After careful review and consideration, FDA initially denied those requests because the agency was not aware of any data that suggested that Iplex was beneficial in the treatment of ALS. In addition, FDA was aware of data from controlled trials for a very similar drug (IGF-1 or Myotrophin) that failed to demonstrate benefit, and in some trials suggested a worse outcome in patients treated with IGF-1 compared to patients treated with placebo (no treatment). The data for IGF-1 came from four randomized, double-blind, placebo-controlled trials as well as randomized access to IGF-1 through a Treatment IND. Reports of three of the controlled trials of IGF-1 have been published in the medical literature[tr]一; the fourth trial and the Treatment IND experience (both of which suggested harm of IGF-1) have not been published but were reported to FDA by the sponsor of the IGF-1 development program. As part of today’s web posting, FDA is making available a summary of the 控制数据对IGF-1在ALS治疗中的应用[tr]。
In addition, although it was not a basis for the denial of the single-patient IND requests, FDA also noted in those denials that the only way to determine if Iplex was beneficial or harmful in the treatment of ALS was to conduct a controlled clinical trial similar to those conducted for IGF-1. Given the variable natural history of ALS, it is not possible to interpret anecdotal experience that may arise from uncontrolled treatment use of the drug, such as was requested under the single-patient INDs. FDA’s regulations on access to investigational drugs for treatment use acknowledge this limitation and, in general, there is an expectation that a drug will be under development and adequate data will be available to evaluate the drug’s safety and effectiveness before single-patient access under an IND is granted. Finally, FDA was concerned that due to the devastating nature of the disease and lack of existing treatments, demand for access to Iplex under single-patient INDs might become widespread, and that such access to the drug would impair the ability to conduct a proper clinical trial to determine if the drug was beneficial or harmful in the treatment of ALS. This concern was cited in our communications to the physicians who submitted the single-patient INDs. We also made clear in those communications that we would welcome the opportunity to work with the ALS community to develop a controlled clinical trial of Iplex for ALS. These concerns over single-patient access became more acute when we subsequently learned, as discussed above, that the supply of the drug is very limited. [tr]FDA持续收到情况后访问单个病人的心智最初否认请求，我们将继续评估数据和追求一些行动并行。
First, FDA was aware of reports that Iplex was being made available for treatment use in Italy under a court order. FDA contacted its counterpart agency in Italy to request information about that program and to inquire about any data that may have been collected from that experience. FDA learned that more than 100 patients with ALS had received court-ordered treatment in Italy, and the agency received a summary report of the data provided to the Italian government by Insmed in March 2008. FDA reviewed that report, and while the data were not interpretable with regard to determining any benefit of Iplex in the treatment of ALS, the data were useful in that no serious, immediate drug-related toxicities were apparent (noting that these uncontrolled data are not interpretable for the potential adverse signals of increased mortality that were seen in the IGF-1 studies as reported above).[tr]Insmed已同意允许FDA做出意大利的汇总数据 available to the public as part of today’s announcement. FDA believes that access to these data, along with the summary of the available data on IGF-1, will allow physicians and patients to make better informed decisions about the use of Iplex. [tr]FDA还提供公开的文件的非官方翻译从英语IPLEX意大利政府的审查为ALS的治疗。[tr]FDA认为这些文件进一步提供上下文的意大利政府决定使用情况是有用的，并可能有助于澄清任何混乱情况在意大利访问。
[tr]FDA还引发了一系列的会议，以确定为IPLEX Insmed的商业开发用于治疗ALS的药物和他们的计划，他们计划制造活动清单，以及他们在开一个IND在ALS进行临床对照试验情况的兴趣。这些会议是非常翔实和生产力，并导致今天的声明关于申请来访问计划的情况下。
结论
The FDA understands that ALS is a fatal disease with limited to no treatment options. We are very sympathetic to the desperate situation of patients with this terrible disease, and their families, and we remain committed to facilitating the development of effective drugs to combat ALS. Sometimes therapies that appear promising in the preclinical phase (before studies in humans) do not lead to benefits in patients. Such early promise was not born out by controlled clinical trials for IGF-1 and for another product called recombinant human ciliary neurotrophic factor (rhCNTF)[tr]二[tr]，这表明没有效益和增加死亡率的临床对照试验。
[tr=transparent]FDA注意到需要罢工获得的未经证实的治疗的患者的治疗选择有限之间的平衡，并使这些患者与不可接受的风险或未经证实的好处，药物的伦理。今天我们选择的方式提供访问情况来尽可能多的患者，所有的考虑前面讨论的一致。
[tr=transparent]我们将继续在其临床试验Insmed的紧密合作，将使公众了解其现状。我们也有与任何其他公司或医生谁愿意探索发展了治疗ALS的讨论很感兴趣。
1. Neurology 1997;49:1621-1630; Neurology 1998;51:583-586; Neurology 2008;71:1770-1775
[tr=transparent]2。1996年纪事；39：256-260
[tr=transparent]本文来自美国FDA网站：
http://www.fda.gov/drugs/resourc ... onals/ucm118121.htm
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