CHICAGO (EGMN) – A new online resource will help seriously ill patients and their physicians request expanded access to investigational therapies when they have exhausted all other treatment options and the patients are not eligible for a clinical trial, it was announced June 4 at a joint news conference of the American Society of Clinical Oncology and the U.S. Food and Drug Administration.

“Most clinical trials have quite stringent eligibility criteria, and many patients are not eligible to enroll,” said ASCO President-Elect George W. Sledge Jr., of Indiana University Simon Cancer Center, Indianapolis.

“In those instances when preliminary scientific evidence suggests that treatment with an unapproved therapy might be beneficial, the FDA’s expanded access regulations allow physicians to request access to an investigational agent, outside of the clinical trial setting, for seriously ill patients,” he said.

Expanded access has been allowed since the 1970s, but many physicians have been uncertain about how to request it, what regulations apply, and the legal ramifications. All of these issues are clarified in the new Web site.

“It’s been clear to the agency that there was room for progress,” said FDA Principal Deputy Commissioner Dr. Joshua M. Sharfstein, addressing the press conference on the opening day of ASCO’s annual meeting.

“This is about the opportunity for the FDA to work with a medical specialty society, in this case ASCO, in what really should be a model for the agency – and work together to develop a program to reach out to clinicians and help them understand something that can be a little confusing at first,” he said.

The new resources are available immediately to all patients and physicians, free of charge, through ASCO’s physician education Web site, ASCO University (www.university.asco.org).

The material is presented in three online modules, which introduce the expanded access regulations, outline the process for requesting expanded access, and explain the physicians’ legal responsibilities for treating patients with an investigational agent outside of a clinical trial.

The modules also contain resources such as a glossary, an expanded access request checklist, and various templates (letter to manufacturer, consent form, etc.) to help physicians with the paperwork.

In August 2009, the FDA issued updated regulations and attempted to clarify the rules in two publications, “Expanded Access to Investigational Drugs for Treatment Use,” and “Charging for Investigational Drugs.” The new Web site goes a step further.

To clarify the new rules for patients and the medical community, ASCO consulted the main stakeholders, including representatives from patient advocacy groups, manufacturers, institutional review boards, and the FDA, as well as physicians. Although these tools were made with the oncology community in mind, these resources should be helpful to doctors from other medical specialties as well.

Copyright (c) 2009 Elsevier Global Medical News. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.

芝加哥(EGMN)——美国临床肿瘤学会(ASCO)和美国食品药品管理局(FDA)在6月4日召开的联合新闻发布会上宣布，将推出一种新的网络资源以便重症患者及其医生在用尽了其他所有治疗方案而患者又没有资格参与临床试验时，申请放宽试验用药的获取途径。

 

作为ASCO候任主席，美国印第安纳波利斯印第安纳大学Simon癌症中心的George W. Sledge Jr.博士说：“大部分临床试验都设有非常严格的纳入标准，因此许多患者都没有资格参与试验。”

 

Sledge博士说：“如果有初步的科学证据表明一种尚未获批的治疗药物可能对患者有益，那么FDA所制定的放宽获取途径的法规便允许医生在临床试验范围之外申请获取试验用药以治疗重症患者。”

 

早在20世纪70年代就允许放宽试验用药的获取途径，但许多医生一直都不清楚应如何申请，应参照哪些法规以及相关的法律细节。所有这些问题在新建立的网站上都将一一得以解答。

 

在ASCO 2010年年会开幕当天，FDA副局长Joshua M. Sharfstein博士在新闻发布会上表示：“FDA很清楚，仍有改进的余地。”

 

Sharfstein博士说：“这是FDA与医学专业学会合作的机会，共同制定一个能惠及临床医生的项目，帮助其了解最初显得有些令人困惑的事情。这次是与ASCO合作，希望这次能真正成为FDA的一个成功范例。”

 

所有患者和医生很快就能通过ASCO医生教育网站，即ASCO大学网站(university.asco.org)，免费获取这项新的网络资源。

 

网站上分3个模块介绍了相关事宜，分别介绍了关于放宽获取途径的法规、申请放宽获取的程序以及医生在临床试验范围之外采用试验用药来治疗患者所应承担的法律责任。

 

模块中还含有一些其他资源，如术语表、放宽获取申请表以及各种模板(写给生产商的信函、同意书等)，以帮助医生完成相关的文字工作。

 

2009年8月，FDA发布了新版法规并试图通过两份文件阐明相关规则，即“放宽试验用药的获取途径以供治疗用途”和“试验用药的收费”。新推出的网站又向前迈进了一大步。

 

为了向患者和医疗界阐明新的法规，ASCO曾咨询过主要的利益相关者，包括患者倡导组织的代表、生产商、机构审查委员会、FDA和医生。虽然这些工具出自肿瘤界，但这些资源应该对其他专科领域的医生也同样有用。