Rare essentials: drugs for rare diseases as essential medicines
Rare essentials: drugs for rare diseases as essential medicines
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DOI：
10.2471/blt.06.031518
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发表时间：
2006-09-01
期刊：
影响因子：
--
通讯作者：
Leufkens, Hubert GM
中科院分区：
文献类型：
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作者：
Stolk, Pieter;Willemen, Marjolein JC;Leufkens, Hubert GM
自 1977 年以来，世界卫生组织发布的世界卫生组织基本药物示范清单 (EML) 为那些难以决定应在其公共卫生系统内向患者提供哪些制药技术的会员国提供了建议。源自世界卫生组织之外，各国政府已针对罕见疾病药物（“孤儿药”）的开发制定了激励制度。随着药物研究（例如针对较窄适应症的药物）的进展，这些药物将更多地出现在未来的公共卫生议程中。然而，当严格应用当前基本药物选择的定义时，孤儿药不能成为世卫组织基本药物计划的一部分，从而造成世卫组织可能与该领域脱节的风险。我们认为，世卫组织应通过制定补充性孤儿药模型清单作为 EML 的补充，明确将孤儿药纳入其政策范围。这份“稀有必需品”的补充清单可以帮助新兴国家的政策制定者和患者改善这些药物的获取并刺激相关政策。此外，当前 EML 中关于罕见疾病药物的不一致问题也可以得到解决。在本文中，我们提出了孤儿药模型清单的选择标准，该标准可以成为未来广泛的世卫组织孤儿药计划工作的出发点。
Since 1977, the WHO Model List of Essential Medicines (EML), published by WHO, has provided advice for Member States that struggle to decide which pharmaceutical technologies should be provided to patients within their public health systems. Originating from outside WHO, an incentive system has been put in place by various governments for the development of medicines for rare diseases ("orphan drugs"). With progress in pharmaceutical research (e.g. drugs targeted for narrower indications), these medicines will feature more often on future public health agendas, However, when current definitions for selecting essential medicines are applied strictly, orphan drugs cannot be part of the WHO Essential Medicines Programme, creating the risk that WHO may lose touch with this field. In our opinion WHO should explicitly include orphan drugs in its policy sphere by composing a complementary Orphan Medicines Model List as an addition to the EML. This complementary list of "rare essentials" could aid policy-makers and patients in, for example, emerging countries to improve access to these drugs and stimulate relevant policies. Furthermore, inconsistencies in the current EML with regard to medicines for rare diseases can be resolved. In this paper we propose selection criteria for an Orphan Medicines Model List that could form a departure point for future work towards an extensive WHO Orphan Medicines Programme.