廊桥遗梦看这里

小雪

Dear Sandra Zhuoli,



I hope this letter finds you well. I understand that you recently requested information on early access to dexpramipexole from our medical information department. Therefore, I wanted to respond directly to your request and explain Biogen Idec’s situation regarding this investigational agent for the treatment of amyotrophic lateral sclerosis (ALS).



As you know, ALS is a devastating disease with, as of yet, no optimal therapies or cure. Biogen Idec is focused on the urgent concerns of patients with this fatal and rapidly progressive disease and we are committed to advancing dexpramipexole as quickly as we can.  Our ultimate goal is to achieve regulatory approval of dexpramipexole so that all eligible patients may have equitable access to treatment. Rigorous and well conducted clinical trials are required to achieve this goal.



The safety and efficacy of dexpramipexole for the treatment of ALS is currently under evaluation in a large Phase III clinical trial (223AS302, ‘EMPOWER’; recruitment complete).



Unfortunately, at this time, we cannot make dexpramipexole available outside of a well-controlled clinical trial setting.  This was a deeply considered decision for our company based on the following:

•             The efficacy of dexpramipexole was only suggested, not determined, by the Phase II study.

•             There are examples of recent clinical trials in ALS in which experimental therapies actually worsened outcomes for patients, despite earlier hopeful signals.

•             Long-term safety of dexpramipexole in a large population of ALS patients has not been fully established and the potential risks of the drug have not been adequately identified.

•             Above all, we must not harm patients and their families by allowing access to a drug that has not been proven safe or by encouraging false hope with a drug that has not been proven effective.



Our position has been discussed with prominent experts, including bioethicists, regulators, and patient advocacy groups – all of whom support our decision.  However, we also recognize that our reasons for delaying an early access program for dexpramipexole may seem inadequate to a patient or a loved one; unfortunately, they are essential to fulfilling our obligations and mission of advancing a meaningful treatment for the benefit of all ALS patients as rapidly as possible. Please let me know if you have any additional questions.



Yours sincerely,



Sarah Kulke, MD



____________________________________________
Sarah Kulke, MD
Senior Medical Director

Global Medical Affairs - Dexpramipexole | Biogen Idec
ph: 781.464.3275 | fax: 866.577.8255 | mobile: 781.363.9474

晨雾

请英语高手翻译下邮件内容，谢谢

老牛

亲爱的桑德拉卓立，



我希望这封信找到你。我知道你最近要求早日进入dexpramipexole从我们的医疗信息部门的信息。因此，我想直接回应您的要求和解释Biogen Idec公司的情况，对本研究药物治疗肌萎缩性侧索硬化症（ALS）。



如你所知，ALS是一场毁灭性的疾病，还作为，没有最佳的治疗或治愈。 Biogen Idec公司的重点是对这种致命的和迅速恶化的疾病患者的迫切关注我们都致力于推进dexpramipexole的，我们可以尽快。我们的最终目标是实现dexpramipexole监管部门的批准，使所有符合条件的患者可能有公平获得治疗。要求严格，以及进行临床试验，以实现这一目标。



目前正在评估的安全性和疗效的治疗ALS dexpramipexole在一个大型的三期临床试验（223AS302，“EMPOWER的”;招聘完成）。



不幸的是，在这个时候，我们不能让dexpramipexole外提供一个良好控制的临床试验设置。这是我们公司的一个深思熟虑的决定，基于以下几点：

•的疗效dexpramipexole，只有建议，没有决心，通过第二阶段的研究。

有最近在ALS的临床试验，在实验性疗法，反而加重了患者的预后的例子，尽管早期的充满希望的信号。

尚未完全建立•长期的dexpramipexole安全的在人口众多的ALS患者和药物的潜在风险没有得到充分的确认。

•首先，我们必须不损害病人及其家属，允许访问一个尚未被证明是安全的药物，或鼓励虚假的希望，一个还没有被证明是有效的药物。



我们的立场已经讨论过的著名专家，包括生命伦理学家，监管机构和病人权益团体 - 其中所有支持我们的决定。然而，我们也认识到，我们为延缓为dexpramipexole的早期访问计划的原因，可能会显得不足，病人或亲人，不幸的是，他们是履行我们的义务和使命，推进所有ALS患者的利益为有意义的治疗至关重要尽快。请让我知道如果你有任何其他问题。



此致



萨拉Kulke，MD

老牛

中文(简体)英语日语
萨拉Kulke，MD
高级医务主任

全球医疗事务 -  Dexpramipexole Biogen Idec公司
PH值：781.464.3275|传真：866.577.8255|手机：781.363.9474

苏州丽丽

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