外来书信

廊桥遗梦

Dear Sandra Zhuoli,
亲爱的卓丽


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).
我希望这封信对你有所帮助,我知道你最近请求得到我们医疗信息部关于dexpramipexole的信息.因此,我想直接回答你的提问并解释Biogen Idec公司把这项研究作为治疗ALS的的基本情况.

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.
你也知道,ALS是一种毁灭性的疾病, 现在没有最佳的治疗或治愈方法, Biogen Idec公司目前也关心着这些有致命的并病情迅速发展的病人, 我们正致力于加快dexpramipexole的研究进程. 我们的最终目标就是实现监管dexpramipexole的部门的批准,以便所有符合条件的患者公平地获得治疗. 要求严谨，以及进行临床试验来达到这个目标.

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).
dexpramipexole治疗ALS的安全性和有效性目前下在一个大型的三期临床试验的评价(招募工作已经完成)

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: 不幸的是, 我们不能在良好控制的临床试验设置外使用这药 (也就是说,还不能对外使用这药物)  这是我们公司考虑再三的一个决定, 理由如下:

•             The efficacy of dexpramipexole was only suggested, not determined, by the Phase II study.这药物在第二期试验中仅仅证明有效，还没有完全决定是否有效
•             There are examples of recent clinical trials in ALS in which experimental therapies actually worsened outcomes for patients, despite earlier hopeful signals.最近的一些治疗ALS的临床试验的例子实际上使病人恶化，尽管早期的迹象充满希望。

•             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.使用这药的大批患者的长期安全性还没有完全认定，药物的潜在危险性还没有得到充分确定。

•             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.
首要的是，我们不可以拿这些没有得到证明是安全的、没有证明是有效的药去伤害患者及家人，


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.
我们一直在跟一些杰出专家讨论这问题，包括生物伦理学家、监管机构、病人权益团体。所有人都支持我们的决定。然而，我们也意识到，推迟使用这药物的理由对患者和家属来说也许是不合适的。不幸的是，为了所有ALS患者尽快得到这药物的治疗，他们有必要要履行我们的义务和推进一个有意义的治疗的使命。如果你还有问题，请告诉我，

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

电话：7814643275，传真：8665778255，手机：7813639474

MOMO

这是一种诚恳而且负责任的态度，希望药物能早日研制成功！

青松

严谨而负责，为患者考虑。

lepus

感谢廊桥~~~