Cooperative Major in Advanced Biomedical Sciences, Joint Graduate School of Tokyo Women's Medical University and Waseda University, 8-1 Kawadacho, Shinjuku-ku, Tokyo, Japan.
Healthcare and Wellness Division, Mitsubishi Research Institute, Inc., 10-3 Nagatacho 2-Chome, Chiyoda-ku, Tokyo, Japan.
Orphanet J Rare Dis. 2022 Nov 8;17(1):408. doi: 10.1186/s13023-022-02526-z.
Extremely high prices facilitate drug development for ultra-rare diseases (ultra-orphan drugs). However, various problems arise in terms of healthcare financing and fairness, and the status of ultra-orphan drug pricing remains ambiguous. In this study, we investigated ultra-orphan drug prices in Japan relative to that of other drugs. We examined the relationship between annual expected drug prices and expected sales, and the expected number of patients, for 393 drugs containing new active ingredients for therapeutic use that were listed on the National Health Insurance drug price list in Japan between April 16, 2010 and August 26, 2020. In addition, we compared prices, the drug price calculation method, and price calculation adjustment factors for ultra-orphan and other drugs.
Drug prices tended to increase as the expected number of patients to whom the drug was administered decreased; however, this trend diminished when the expected number of patients was less than 1000. On the other hand, the expected sales tended to decrease as the number of expected patients decreased, and this tendency was reinforced when the expected number of patients was less than 1000. The cost accounting method tended to be used for the price calculation of ultra-orphan drugs, but there were no price differences based on the drug price calculation method. Regarding the price calculation adjustment factors, the premium for usefulness tended to be higher for ultra-orphan drugs. The premium for marketability was higher for non-orphan drugs but did not differ from that for orphan drugs, except for ultra-orphan drugs.
The status of drug prices and expected sales differed beyond a threshold of 1000 expected patients, indicating that recovering the development cost for ultra-orphan drugs is difficult. In addition, the higher premium for usefulness for ultra-orphan drugs reflects the largely unmet need of the associated diseases. Scarcity among orphan drugs is not considered for marketability, highlighting the need for a new framework to promote the development of ultra-orphan drugs.
极高的价格有利于治疗超罕见疾病（超孤儿药）的药物开发。然而，在医疗保健融资和公平性方面出现了各种问题，超孤儿药定价的地位仍然不明确。在这项研究中，我们调查了日本超孤儿药的价格与其他药物的关系。我们检查了 393 种含有新活性成分的治疗用药物的年预期价格与预期销售额之间的关系，这些药物于 2010 年 4 月 16 日至 2020 年 8 月 26 日在日本国家医疗保险药品价格表上列出，且预期患者人数为 393 人。此外，我们比较了超孤儿药和其他药物的价格、药品定价方法和价格计算调整因素。
药物价格随着给药预期患者数量的减少而趋于增加；然而，当预期患者数量少于 1000 人时，这种趋势会减弱。另一方面，预期销售额随着预期患者数量的减少而趋于减少，当预期患者数量少于 1000 人时，这种趋势会加强。成本核算方法倾向于用于超孤儿药的价格计算，但基于药品定价方法没有价格差异。关于价格计算调整因素，有用性溢价往往更高对于超孤儿药。对于非孤儿药，市场性溢价较高，但除了超孤儿药外，与孤儿药没有区别。
在预期患者超过 1000 人这一门槛之外，药物价格和预期销售额存在差异，表明很难收回超孤儿药的开发成本。此外，超孤儿药的高有用性溢价反映了相关疾病的大量未满足需求。孤儿药的稀缺性在市场性方面没有得到考虑，这突出了需要一个新的框架来促进超孤儿药的开发。
