罕见病治疗药物研发的挑战和机遇（英文）

发布时间：2018-01-19 09:31

本文关键词： 罕见病药物研发激励措施单基因病概念验证　出处：《国际药学研究杂志》2017年02期 　论文类型：期刊论文

【摘要】：尽管每一种罕见病的发生率极低,但已确认的罕见病6000余种,累及人群约占总人口的6%~8%。对患者和整个社会而言,针对罕见病的治疗有重大意义,但远未满足患者需求。罕见病的新药开发同样面临着巨大的挑战,例如对开发新药的企业来说,存在罕见病药物潜在市场小、注册路径常不明晰、临床验证终点和患者自评结果缺乏、疾病相关数据不足等问题。现行的一些特殊的激励措施,如市场独占权、注册费减免、协助撰写临床试验方案,以及税收抵扣等,均可促进罕见病药物的开发。这些措施的最终目的是不论疾病的流行病学情况如何,都要有助于罕见病患者获得恰当的治疗方法。此外,罕见病通常有清晰的发病机制,这代表了一种独特的药物开发机会,即随后可在有相同发病机制的疾病中扩展适应证。尽管已有了越来越多的治疗罕见病的药物,但在这类特殊人群中仍然存在着极大的未能满足的医学需求。
[Abstract]:Although the incidence of each rare disease is extremely low, more than 6,000 rare diseases have been identified, affecting about 6% of the population. The treatment of rare diseases is of great significance, but far from meeting the needs of patients. The development of new drugs for rare diseases also faces great challenges, for example, there is a small potential market for rare disease drugs for companies that develop new drugs. The registration path is often unclear, the clinical verification end point and the patient self-evaluation result is lacking, the disease related data is insufficient and so on. Some special incentive measures, such as market exclusive right, registration fee reduction. Assistance in the preparation of clinical trial programmes, as well as tax credits, can facilitate the development of drugs for rare diseases. The ultimate objective of these measures is to promote the epidemiology of the disease. In addition, rare diseases often have clear pathogenesis, which represents a unique drug development opportunity. Although more and more drugs have been used to treat rare diseases, there is still a great unmet medical need in this special population.
【作者单位】：瑞士巴塞尔诺华生物医学研究中心;
【分类号】：R95
【正文快照】： "Rare diseases are only rare for the doctors.If their incidence is extremely low and physicians very seldom see them,they may be considered insignificant;even textbooks mention them only briefly."These were the initial words of a Preface we wrote in a sp

【相似文献】