论文部分内容阅读
在商业化的药品研发领域,罕见病患者一直都没有得到足够的关注。为了改善这一状况,一些地区制定了具体的法律法规,以鼓励那些不具商业价值的孤儿药(orphan drug,也称罕见病药)的研发。然而,由于其市场狭小,且价格昂贵,根据卫生技术评估(HTA)中经济学评价的标准方法,孤儿药通常不会被证明是符合成本效益的,再加上其成本高,意味着这种药的筹资和患者可及性都是受到限制的。但是,这些限制可能与社会偏好不一致。因此,本研究主要探讨HTA标准方法是否足以在孤儿药的筹资和患者可及性上协助决策,其次概述孤儿药相关研究事项,以期帮助了解孤儿药社会价值及期在研发、筹资和使用等方面的相关问题。
In the field of commercial drug research and development, rare patients have not been enough attention. To improve the situation, some regions have specific laws and regulations to encourage the development of orphan drugs that are not commercially valuable. However, because of its small market size and its high cost, orphanage is often not proven to be cost-effective according to the standard method of economic evaluation in the Health Technology Assessment (HTA), which, coupled with its high cost, implies that this Drug financing and access to medicines are limited. However, these restrictions may not be in line with social preferences. Therefore, the present study mainly explored whether the HTA standard method is sufficient to assist decision-making in fund-raising and patient accessibility of orphan drugs, followed by an overview of orphan drug-related research issues with a view to helping to understand the social values of orphan drugs and their implications for research and development, financing and use. Related issues.