Peabody J W, Ruby A, Cannon P
General Internal Medicine Division, Veteran's Affairs Hospital, Los Angeles, California, USA.
Pharmacoeconomics. 1995 Nov;8(5):374-84. doi: 10.2165/00019053-199508050-00002.
This review of the US Orphan Drug Act (ODA) 1983 outlines how the ODA is intended to stimulate orphan drug research and development of drugs for rare diseases. We also evaluate the effectiveness of the ODA in the past decade and provide recommendations for ODA improvements in the future. The economic incentives embedded in the ODA are presented in a simple economic model, in which a guarantee of market exclusivity plays a central role in encouraging firms to pursue the development of orphan products. Some evidence suggests that this provision has been a major impetus for the rise in orphan drug applications and designations in the last decade. Market exclusivity is the key incentive for orphan drug research, and should be retained. Concerns about a limited number of highly successful 'blockbuster' orphan drugs should be evaluated in terms of the useful economic incentives. In the future, exceptionally high profits could be limited by more precise evaluation of disease prevalence, elasticity of demand, and the other uses of orphan compounds. We further recommend an expansion of the ODA tax credits and research grants programme and targeting of 'priority' diseases. We conclude that the ODA has been a valuable legislative initiative, but it can be strengthened with some simple extensions of the current incentives that it contains.
对1983年美国《孤儿药法案》（ODA）的这篇综述概述了该法案旨在如何刺激孤儿药研究以及针对罕见病药物的研发。我们还评估了《孤儿药法案》在过去十年中的成效，并为其未来改进提供建议。《孤儿药法案》中的经济激励措施通过一个简单的经济模型呈现，其中市场独占权保障在鼓励企业开展孤儿产品研发方面发挥着核心作用。一些证据表明，这一规定是过去十年中孤儿药申请和认定数量增加的主要推动力。市场独占权是孤儿药研究的关键激励因素，应予以保留。对于数量有限但极为成功的“重磅炸弹”孤儿药的担忧，应从有用的经济激励角度进行评估。未来，可通过更精确地评估疾病患病率、需求弹性以及孤儿化合物的其他用途来限制过高的利润。我们还建议扩大《孤儿药法案》税收抵免和研究资助计划，并针对“优先”疾病。我们的结论是，《孤儿药法案》是一项有价值的立法举措，但通过对其现有激励措施进行一些简单扩展，它可以得到加强。
