来源：NORD，翻译：纪寻
2013年1月4日，华盛顿特区。30年前的今天，罗纳德·里根（Ronald Reagan）总统签署了《孤儿药法案》（Orphan Drug Act），使之生效为法律，以鼓励研发惠及百万计的美国罕见病患者的治疗方法。
这项新法案代表了一种胜利：是病患倡导者与政府伙伴、媒体以及其他支持者合作，共同解决一个非常重要但未被满足的需要。
为了让法案通过，各种罕见病组织的领导人组成了一个特别的联盟，并命名为全国罕见疾病组织（NORD），教导他们如何为其所代表的绝望的病人和家庭做倡导。
演员杰克·克卢格曼（Jack Klugman）的兄弟死于一种罕见病。在他广受欢迎的电视节目《法医昆西》（Quincy, M.E.）中，克卢格曼以一集特别节目体现了罕见疾病患者的治疗需要，以示他对这一群体的支持。克卢格曼随后在一个国会下属委员会前发表公开声明，当该法案在国会受阻时，他播出了另一集《法医昆西》，帮助法案获得了通过。
就在里根总统签署该法案使之成为法律的整整4个月后，在1983年5月4日，联盟正式成立了NORD。它作为一个非盈利性的组织，为所有受罕见病影响的美国人提供倡导、教育、研究和病人/家属服务等工作。
“让这个当时迫切需要的法律生效是一件了不起的成就，”NORD的主席兼首席执行官彼得·索顿斯托尔（Peter L. Saltonstall）说，“从今天一直到整个2013年，我们将向帮助我们做到这一点的先驱们致敬，庆祝迄今取得的进展，并与合作伙伴共同寻找方法，以加快前进的步伐。”
索顿斯托尔补充说，最近杰克·克卢格曼（12月24日）的离世也为这次活动平添一份心酸与悲伤。
在NORD的第一个25年，作为主席的阿比·S·迈尔斯（Abbey S. Meyers）被广泛认为是孤儿药法案的主要患者倡导者。她在2008年退休，索顿斯托尔在那个时候加入了该组织。
美国国立卫生研究院（NIH）和美国食品和药物管理局（FDA）的政府合作伙伴通过其实施的法律，为罕见病社区提供了重要的支持和鼓励。
周一，FDA的“孤儿产品”开发办公室将为FDA工作人员和客人举办一场活动，以庆祝该法律的周年庆。FDA专员玛格丽特·汉伯格博士（Margaret Hamburg, MD），将和索顿斯托尔一起致辞，同时会播出迈尔斯的VCR问候。特别嘉宾包括由FDA评选出来的获得本年度的“罕见病英雄”（Rare Disease Heroes）荣誉的个人和组织代表。
NORD为期一年的30周年纪念活动将包括一个在其网站上特殊组成部分，罕见病患者、研究人员和其他人将被邀请分享罕见病的故事和成就。
在NORD主办的2013年罕见病日（2月28日）期间，全国都会有特别的活动，以纪念罕见病社区的历史。此外，NORD在5月的年度庆典和在10月的年度会议将有一个周年主题。还有其他的特殊活动贯穿全年。
《孤儿药法案》提供财政激励措施，如研发的药物治疗人群少于20万，该产品可以享受7年的市场独占权。 7000种疾病被认为是罕见病，它们在美国影响近3000万人，大约10个美国人中有1人是罕见病患者。
在该法律通过的十年前，只有10个治疗罕见病患者的新药物被行业研发出来。自1983年以来，有超过2700种潜在的治疗方法进入“孤儿药产品”的研究管道，其中400多种已被FDA批准。
自该法颁布以来的突出部分包括由NORD促进的《2002年罕见疾病法案》（Rare Diseases Act of 2002），该法案使罕见病临床研究网络落户NIH成为可能。以及最近的《2012年FDA安全和创新法案》（FDA Safety and Innovation Act of 2012），其中包括许多罕见病相关的重大规定。
索顿斯托尔说：“今年，我们将向迄今取得的进展致敬，但我们也将专注于一个事实，即仍然有许多工作要做”，“大多数罕见病患者仍然没有经FDA批准的有效治疗方法，而且，即使有治疗方法，许多患者获得治疗仍存在种种困难。”
“在这个周年之际，NORD将带着新的承诺向前迈进，希望研发出安全、有效的治疗方法，并保证所有严重医疗问题的病人可以获得他们所需要的治疗和服务。”
原文：
Launching a Year-Long Celebration: 30th Anniversary of the Orphan Drug Act and NORD
Washington DC, Jan. 4, 2013----Thirty years ago today, President Ronald Reagan signed the Orphan Drug Act into law to encourage the development of treatments for the millions of Americans with rare diseases.
The new law represented a triumph of patient advocates working with government partners, the media, and other supporters to address a critically important unmet need.
To get the law passed, an ad hoc coalition of leaders of rare disease patient organizations gave themselves a name -- the National Organization for Rare Disorders (NORD) -- and taught themselves how to be advocates for the desperate patients and families they represented.
Actor Jack Klugman, whose brother was dying of a rare disease, lent his support with an episode of his popular TV show, Quincy, M.E., dedicated to showing the need for treatments for rare disease patients. Klugman later testified before a Congressional subcommittee and, when the bill became stalled in Congress, aired another Quincy episode that helped get the bill passed.
Exactly four months after President Reagan signed the bill into law, on May 4, 1983, the coalition of patient advocates formally established NORD as a nonprofit organization to provide advocacy, education, research and patient/family services for all Americans affected by rare diseases.
"It was an amazing accomplishment to get this desperately needed law enacted at that time," says NORD President and CEO Peter L. Saltonstall. "Today and throughout 2013, we will be saluting all the pioneers who helped make it happen, celebrating progress to date, and working with our partners to find ways to accelerate the pace of progress."
Saltonstall added that the recent death of Jack Klugman (on Dec. 24th) added poignancy to the occasion.
NORD's president for its first 25 years, Abbey S. Meyers, is widely considered the primary patient advocate for the Orphan Drug Act. She retired in 2008 and Saltonstall joined the organization at that time.
Government partners at the National Institutes of Health (NIH) and Food and Drug Administration (FDA) have provided major support and encouragement to the rare disease community through their implementation of the law.
On Monday, the FDA Office of Orphan Products Development will host an event for FDA staff and guests to observe the anniversary of the law. FDA Commissioner Margaret Hamburg, MD, will speak, as will Saltonstall, with a video message from Meyers. Special guests will include individuals and representatives of organizations being honored as "rare disease heroes" by FDA during the anniversary year.
NORD's year-long observance of the 30th anniversary will include a special section on its website where rare disease patients, researchers, and others will be invited to share stories about rare disease milestones and achievements.
On Rare Disease Day 2013 (Feb. 28th), which NORD sponsors nationally, there will be special activities to honor the history of the rare disease community. In addition, NORD's annual gala in May and annual conference in October will have an anniversary theme. There will be other special activities throughout the year.
The Orphan Drug Act provides financial incentives such as seven years of marketing exclusivity for products developed to treat diseases affecting fewer than 200,000 Americans. There are 7,000 diseases considered rare in the U.S. affecting nearly 30 million Americans, or about 1 in 10 people.
In the decade before the law was passed, only 10 new drugs were developed by industry for patients with rare diseases. Since 1983, more than 2,700 potential treatments have entered the research pipeline as "orphan products" and more than 400 have been approved by FDA.
Highlights over the years since the law was enacted have included the Rare Diseases Act of 2002, promoted by NORD, which made possible a Rare Diseases Clinical Research Network at NIH, and most recently the FDA Safety and Innovation Act of 2012, which includes many major provisions related to rare diseases.
"This year, we will be saluting the progress made to date but we'll also be focusing on the fact that much remains to be done," Saltonstall said. "Most people with rare diseases still have no FDA-approved treatment. And, even when treatments exist, many patients have trouble accessing them.
"During this anniversary year, NORD will move forward with renewed commitment toward the development of safe, effective treatments for all, and assurance that all patients with serious medical problems will have access to the treatments and services they need."
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CONTACT:
Mary Dunkle (mdunkle@rarediseases.org)
