我国罕见病的伦理学和政策研究
摘要
罕见病是一种患病率极低的疾病,由于患病人数少,因而很容易被社会所忽视。随着科技的进步,特别是人类基因组工程,给更多的罕见病患者的治疗带来了新的希望,使他们的生存状态可以显著改变。目前,有一些酶缺乏的罕见遗传性疾病已经可以通过酶替代疗法治疗,并可以使这些患者过上正常人的生活。
根据世界卫生组织(WHO)对罕见病的定义(患病人数占总人口的0.65‰-1‰的疾病或病变),考虑到我国人口基数大,罕见病患者人数也不容乐观。因此,关注罕见病不仅是关注少数患者的健康权和生命权的问题,也是一个公共卫生问题。目前我国医疗卫生和社会保障仍处于完善和发展阶段,由于罕见病的发病率相对极低,因而尚未引起卫生行政部门、临床医学、医药研发和生产企业及社会各界应有的重视。在罕见病及孤儿药政策研究和医疗保障上,我们不仅落后于发达国家,甚至与一些发展中国家也存在不小差距。罕见病患者这个弱势群体中特别弱势的群体的特殊权益亟待引起社会的关注和解决。
本文意在通过对一些国家的罕见病定义及其罕见病相关的法律法规政策的梳理和研究,以及对我国罕见病现状的透视,分析我国罕见病现状以及罕见病控制和临床治疗中若干伦理问题,提出评价罕见病政策伦理学框架,并对可能的政策建议进行分析和论证。
A rare disease is any disease that affects a small percentage of the population. It is also known as orphan disease. Due to the size of affect population, it is most likely to be overlooked by the society. With the improvement of science technology, especially Human Genome Project [HGP], it brings more hopes and significant changes for people suffering rare diseases. Recently, with the therapy of enzyme replacement, certain patients, born with generic disorder due to enzyme deficiency, are able to lead a nearly normal life.
In accordance with the legal definition of Rare Disease by WHO, there are more than 10 millions of population suffering rare diseases in our country. Focusing on rare diseases is not only a concern on patient's right to health and life, but also an issue on public health. In China, the overall of health care, medical hygiene and social security is still in the stage of ongoing development and improvement. Due to the low rate, rare diseases have not drawn sufficient attention from the health administrative departments, research institutes, pharmaceutical enterprises, and other sectors of the society. Comparing to some other developing countries, our country remains fall behind on the research study of rare diseases and related health care policy and health care security, not to mention the developed country. For the population with rare diseases is the one of the most vulnerable groups, their rights and interests deserve to be specially concerned and demand to be promptly protected..
This paper will focus on analyzing existing problems associated with rare diseases and relevant ethical and policy issues in China with the study of experiences in other countries on the definition of rare disease, and the ACT for rare diseases, policies, regulations and laws associated with rare diseases. Recommendations will be provided based on the results of the study.
根据世界卫生组织(WHO)对罕见病的定义(患病人数占总人口的0.65‰-1‰的疾病或病变),考虑到我国人口基数大,罕见病患者人数也不容乐观。因此,关注罕见病不仅是关注少数患者的健康权和生命权的问题,也是一个公共卫生问题。目前我国医疗卫生和社会保障仍处于完善和发展阶段,由于罕见病的发病率相对极低,因而尚未引起卫生行政部门、临床医学、医药研发和生产企业及社会各界应有的重视。在罕见病及孤儿药政策研究和医疗保障上,我们不仅落后于发达国家,甚至与一些发展中国家也存在不小差距。罕见病患者这个弱势群体中特别弱势的群体的特殊权益亟待引起社会的关注和解决。
本文意在通过对一些国家的罕见病定义及其罕见病相关的法律法规政策的梳理和研究,以及对我国罕见病现状的透视,分析我国罕见病现状以及罕见病控制和临床治疗中若干伦理问题,提出评价罕见病政策伦理学框架,并对可能的政策建议进行分析和论证。
A rare disease is any disease that affects a small percentage of the population. It is also known as orphan disease. Due to the size of affect population, it is most likely to be overlooked by the society. With the improvement of science technology, especially Human Genome Project [HGP], it brings more hopes and significant changes for people suffering rare diseases. Recently, with the therapy of enzyme replacement, certain patients, born with generic disorder due to enzyme deficiency, are able to lead a nearly normal life.
In accordance with the legal definition of Rare Disease by WHO, there are more than 10 millions of population suffering rare diseases in our country. Focusing on rare diseases is not only a concern on patient's right to health and life, but also an issue on public health. In China, the overall of health care, medical hygiene and social security is still in the stage of ongoing development and improvement. Due to the low rate, rare diseases have not drawn sufficient attention from the health administrative departments, research institutes, pharmaceutical enterprises, and other sectors of the society. Comparing to some other developing countries, our country remains fall behind on the research study of rare diseases and related health care policy and health care security, not to mention the developed country. For the population with rare diseases is the one of the most vulnerable groups, their rights and interests deserve to be specially concerned and demand to be promptly protected..
This paper will focus on analyzing existing problems associated with rare diseases and relevant ethical and policy issues in China with the study of experiences in other countries on the definition of rare disease, and the ACT for rare diseases, policies, regulations and laws associated with rare diseases. Recommendations will be provided based on the results of the study.
引文
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26此处所说的特殊情况,是指疾病患病率超过卫生署的公告标准,或难以计算,而其诊断治疗所需的方法或药物取得确有困难,经罕见疾病及药物审议委员会认定者。
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43类似“两害相较取其轻”。即我们按选择对象可能产生的最坏结果来排列选择对象的次序,然后我们将采用这样一个选择对象：它的最坏结果优于其它对象的最坏结果。
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23需要提及的一个重要内容是,在日本,如果一个疾病的患者接近5万人(例如4.5万人),厚生劳动省可能会决定不认定治疗该疾病的药品为孤儿药。
24财团法人罕见疾病基金会网站,Http://www.tfrd.org.tw/intro/index.php?kind_id=12010年9月13日访问。
25叶秀珍,曾敏捷,林志鸿：《各国罕见疾病相关法案之比较研究》,财团法人罕见疾病基金会,2002年5月,第115-118页.
26此处所说的特殊情况,是指疾病患病率超过卫生署的公告标准,或难以计算,而其诊断治疗所需的方法或药物取得确有困难,经罕见疾病及药物审议委员会认定者。
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41是罗尔斯为了排除使人们陷入争论的各种偶然因素的影响一种方法,假定各方不知道他们自己在社会中的具体情况,包括种族、性别、经济状况,以及其他特征；没有人知道他的善的观念,合理生活计划的特殊性,甚至他的心理特征：也没有人知道这一社会的经济或政治状况,或者它能达到的文明和文化水平。
42是罗尔斯假设的一种的状态。它是一种期间所达到的任何契约都是公平的状态,是一种各方在其中都是作为道德人的平等代表、选择的结果不受偶然因素或社会力量的相对平衡所决定的状态。原初状态旨在建立一种公平的程序,以使任何被一致同意的原则都是公正的。
43类似“两害相较取其轻”。即我们按选择对象可能产生的最坏结果来排列选择对象的次序,然后我们将采用这样一个选择对象：它的最坏结果优于其它对象的最坏结果。
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