Research Centre for Pharmaceutical Care and Pharmaco-economics, KU Leuven, Leuven, Belgium.
Drugs. 2012 Jul 30;72(11):1437-43. doi: 10.2165/11635320-000000000-00000.
Orphan drugs are intended for diseases with a very low prevalence, and many countries have implemented legislation to support market access of orphan drugs. We argue that it is time to revisit the special market access status of orphan drugs. Indeed, evidence suggests that there is no societal preference for treating rare diseases. Although society appears to assign a greater value to severity of disease, this criterion is equally relevant to many common diseases. Furthermore, the criterion of equity in access to treatment, which underpins orphan drug legislation, puts more value on health improvement in rare diseases than in common diseases and implies that population health is not maximized. Finally, incentives for the development, pricing and reimbursement of orphan drugs have created market failures, including monopolistic prices and the artificial creation of rare diseases. We argue that, instead of awarding special market access status to orphan drugs, there is scope to optimize research and development (R&D) of orphan drugs and to control prices of orphan drugs by means of, for example, patent auctions, advance purchase commitments, pay-as-you-go schemes and dose-modification studies. Governments should consider carefully the right incentive strategy for R&D of orphan drugs in rare diseases.
孤儿药用于患病率非常低的疾病，许多国家已实施立法以支持孤儿药的市场准入。我们认为，现在是重新审视孤儿药特殊市场准入地位的时候了。事实上，有证据表明，社会并没有偏好治疗罕见病。尽管社会似乎更看重疾病的严重程度，但这一标准同样适用于许多常见疾病。此外，孤儿药立法所依据的治疗机会公平标准，对罕见病的健康改善赋予了比常见疾病更高的价值，这意味着人口健康没有实现最大化。最后，孤儿药的开发、定价和报销激励措施造成了市场失灵，包括垄断价格和人为制造的罕见病。我们认为，与其授予孤儿药特殊的市场准入地位，不如通过专利拍卖、预付款承诺、按疗效付费和剂量调整研究等方式，优化孤儿药的研发，并控制孤儿药的价格。政府应仔细考虑在罕见病的孤儿药研发方面的正确激励策略。
