为什么是瑞德西韦? 一封来自吉利德CEO Daniel O’Day的公开信
当冠状病毒的新闻首次出现时，吉利德立即开始了对瑞德西韦（Remdesivir）治疗潜力的研究。我们对多种抗病毒药物进行大量研究，其中，瑞德西韦是我们研究了很多年的一个药物。瑞德西韦从未被批准使用，但是，根据我们到目前为止的了解，我们知道它可能有潜力治疗新型冠状病毒。从那时起，我们带着最大的紧迫感和责任感努力工作，以确定瑞德西韦是否确实对新型冠状病毒肺炎有效。
这种紧迫感来自于对患者迫切需求的认知以及没有任何已批准治疗方案的现状。瑞德西韦是在研药物，将其运往全球使用之前确保它是有效且安全的，这是责任。
这也是我们一直在以前所未有的速度入组患者参与临床试验的原因。与监管机构合作，明确瑞德西韦的安全性和有效性，对于未来可能让更多患者获得治疗至关重要。有多项研究正在进行中，我们有望在接下来的几周获得初步数据。如果药物获得批准，我们将确保其可负担性和可及性，让有迫切需要的患者能够用上瑞德西韦。
与此同时，我们还为不能参加临床试验的重症患者提供了这一在研药物。“同情用药”程序通常只用于少数个例，但这一次的危机已突破常规，到目前为止，我们已向1000多名患者提供了瑞德西韦。这一程序由监管机构设计，要求必须对每个申请以个体为基础逐一审评。通常情况下，这个程序可以很好地运行，因为申请数量很有限。但是，该系统无法支持和处理我们在新型冠状病毒肺炎中遇到的巨大数量的申请。
为了解决这个问题，我们正在过渡到一种更加精简、可持续的方式，即“扩大可及”方案。同情用药程序将仅开放给儿童和孕妇患者，以将数量降低到系统可以应对的水平。通过扩大可及方案，医院或医生可以同时为多个重症患者申请瑞德西韦的紧急用药。虽然搭建站点网络需要一些时间，但扩大可及的方式最终将可以加速让更多患者紧急获得药物。截至昨天，美国的一些初始站点已经启动并运行，预计在其他国家的站点也将很快启动。
近些天来，许多人代表自己的朋友和亲人向吉利德提出要求使用瑞德西韦。我非常能体会在那种处境中的感受。我们最近每天在新闻中看到数字和统计结果，但是，我们知道每一个数字背后都是一个真实的、令人心碎的关于人的故事。我知道，当我说“我们多么希望能够帮助每一位有需要的患者”时，我代表着吉利德的每一位员工。现在，我们正在快速确立临时的扩大可及方案，同时明确瑞德西韦的潜在安全性和有效性，并确定瑞德西韦对哪些患者可能有效。
瑞德西韦仍然是一种在研药物，大家都在期待它可以被证明是一种安全、有效的治疗方案，我们也正在基于这种预期做相应的计划——与此同时，我们也在以合乎伦理道德且负责任的方式来确定结果是否如此。在这个过程中的每一步，我们会在公共卫生机构和生物伦理学家的指导下，结合我们数十年来为艾滋病病毒（HIV）和病毒性肝炎等疾病提供抗病毒治疗方案的经验来做出决定。
我们希望，通过与世界各地的许多团体合作，我们可以一起帮助新型冠状病毒肺炎患者。我们知道情况是如此危急，迫切需要确定瑞德西韦是否是一种安全有效的治疗方案。
我们挂念日以继夜奋战在抗击流行病第一线的医护人员，他们急需要有效的治疗方案。我们知道全世界的患者和他们的家人都在等待。全体吉利德人正在全力以赴，怀着最大的紧迫感和关爱之心，和瑞德西韦一起，履行我们的责任。
在继续努力推进上述工作的同时，我们也将及时提供最新进展信息，回应全球公众对瑞德西韦的高度关注。
An Open Letter from our Chairman and CEO
Daniel O’Day
When the news of the coronavirus first emerged, Gilead immediately began to investigate the potential of remdesivir, a medicine we had been studying for many years as part of our extensive research in antivirals. Remdesivir had never been approved for use but based on what we had learned to date, we knew it might have potential with the novel coronavirus. Since then, we have been working with the greatest sense of urgency and responsibility to determine whether remdesivir does indeed work against COVID-19.
The urgency comes from knowing the desperate need among patients and the lack of any approved treatment. The responsibility is to ensure that remdesivir, an investigational medicine, is effective and safe before it is distributed for use worldwide.
This is why we have been working at unprecedented speed to enroll patients in clinical trials. Establishing the safety and efficacy of remdesivir, in partnership with regulatory authorities, is essential to potentially enabling the treatment of many more patients in the future. Multiple studies are ongoing, and we are on track to have initial data in the coming weeks. If it is approved, we will work to ensure affordability and access so that remdesivir is available to patients with the greatest need.
In the meantime, we have made the investigational medicine available for severely ill patients who cannot enroll in a trial. This “compassionate use” program is typically reserved for a small number of individual cases but there is nothing typical about this crisis and to date we have provided remdesivir to more than 1,000 patients. The program is designed by regulatory authorities in such a way that each application has to be reviewed on an individual basis. This works well when there is only a limited number of requests – as is normally the case – but the system cannot support and process the overwhelming number of applications we have seen with COVID-19.
To address this, we are transitioning to what should be a more streamlined, sustainable approach with “expanded access” programs. The compassionate use program will continue for children and pregnant women only, reducing the numbers to a level where the system can cope. With expanded access, hospitals or physicians can apply for emergency use of remdesivir for multiple severely ill patients at a time. While it will take some time to build a network of active sites, this approach will ultimately accelerate emergency access for more people. Initial sites in the United States are up and running as of yesterday, and it is expected that sites in additional countries will be activated soon.
In recent days, many people have reached out to Gilead to advocate for access to remdesivir on behalf of friends and loved ones. I can only imagine how it must feel to be in that situation. We are used to seeing numbers and statistics in the news on a daily basis but we all know that behind each of those numbers is a real and often heartbreaking human story. I know I speak for everyone at Gilead when I say how much we all wish we could help every patient in need. Today we are working at speed to establish the temporary expanded access programs, while at the same time establishing the potential safety and efficacy of remdesivir and determining for which patients remdesivir may have activity.
Remdesivir is still an investigational medicine. We are planning for the outcome we all hope for – that it will prove to be a safe, effective treatment – and in the meantime we are taking the ethical, responsible approach to determining whether that is the case. At each step of the way, our decisions are informed by guidance from public health authorities and bioethicists, and by our decades of experience in making antiviral treatments for diseases such as HIV and viral hepatitis.
We hope that, in partnership with many groups around the world, we can play a part in helping patients with this disease. We know how much is at stake and the urgent need to determine whether remdesivir will be a safe and effective treatment.
We think about the healthcare workers who are on the front lines of fighting this pandemic around the clock and the urgent need to equip them with a treatment. We know that patients and their families around the world are waiting. All of us at Gilead are doing everything we can to meet our responsibility with remdesivir, with the greatest sense of urgency and care.
As we continue with those efforts, we will provide updates on our progress as soon as information becomes available, recognizing the significant public interest in remdesivir around the world.
