目的:从增量健康的角度研究超孤儿药,成本,与更普遍的疾病药物相比,成本效益。
方法:我们确定了1999-2019年的FDA药物批准。对于批准用于多种适应症的药物,我们分别考虑了每个药物-适应症对.利用FDA的孤儿药名称和美国疾病患病率,我们将药物适应症对分类为:超孤儿(<10,000名患者),\'其他\'孤儿(≥10,000和<200,000),非孤儿(≥200,000)。我们在PubMed数据库中搜索了成本效益和成本效用研究。我们排除了制造商资助的研究。与批准时的护理标准相比,我们从质量调整生命年(QALYs)和与药物适应症对相关的增量成本方面提取了增量健康收益的估计值。我们比较了QALY收益,增加的成本,和使用Kruskal-Wallis(KW)的增量成本效益比(ICER),曼恩-惠特尼大学(MWU),和Kolmogorov-Smirnov(KS)测试。
结果:增量QALYs中位数,成本,ICER在非孤儿中有所不同,\'其他\'孤儿,和超孤儿类别(KWp<0.01)。与非孤儿药相比,超孤儿药的QALY收益较大(0.700vs.0.050,MWUp<0.01,KSp<0.01),更大的成本(172,231美元与$3,360,MWUp<0.01,KSp<0.01),和更大的ICER($1,216,184/QALY与114,061美元/质量,MWUp<0.01,KSp<0.01)。与“其他”孤儿药相比,超孤儿药的QALY收益较大(0.700vs.0.310,MWUp=0.65,KSp=0.32),更大的成本(172,231美元与$69,308,MWUp=0.03,KSp=0.03),和更大的ICER($1,216,184/QALY与223,472美元/质量,MWUp<0.01,KSp<0.01)。
结论:新型超孤儿药通常比治疗更普遍的疾病的药物提供更大的增量健康收益,但是由于他们大量增加的成本,通常成本效益较低。
OBJECTIVE: To examine ultra-orphan drugs in terms of incremental health, costs, and cost-effectiveness compared to more prevalent disease drugs.
METHODS: We identified FDA drug approvals from 1999-2019. For drugs approved for multiple indications, we considered each drug-indication pair separately. Utilizing FDA\'s orphan drug designation and US disease prevalence, we categorized drug-indication pairs as: ultra-orphan (<10,000 patients), \'other\' orphan (≥10,000 and <200,000), and non-orphan (≥200,000). We searched the PubMed database for cost-effectiveness and cost-utility studies. We excluded manufacturer-funded studies. We extracted estimates of incremental health gains in terms of quality-adjusted life-years (QALYs) and incremental costs associated with drug-indication pairs compared to the standard of care at the time of their approval. We compared QALY gains, added costs, and incremental cost-effectiveness ratios (ICERs) using the Kruskal-Wallis (KW), Mann-Whitney U (MWU), and Kolmogorov-Smirnov (KS) tests.
RESULTS: Median incremental QALYs, costs, and ICERs differed across non-orphan, \'other\' orphan, and ultra-orphan categories (KW p<0.01). Compared to non-orphan drugs, ultra-orphan drugs had larger QALY gains (0.700 vs. 0.050, MWU p<0.01, KS p<0.01), larger costs ($172,231 vs. $3,360, MWU p<0.01, KS p<0.01), and larger ICERs ($1,216,184/QALY vs. $114,061/QALY, MWU p<0.01, KS p<0.01). Compared to \'other\' orphan drugs, ultra-orphan drugs had larger QALY gains (0.700 vs. 0.310, MWU p=0.65, KS p=0.32), larger costs ($172,231 vs. $69,308, MWU p=0.03, KS p=0.03), and larger ICERs ($1,216,184/QALY vs. $223,472/QALY, MWU p<0.01, KS p<0.01).
CONCLUSIONS: Novel ultra-orphan drugs typically offer larger incremental health gains than drugs for more prevalent diseases, but due to their substantial added costs, are typically less cost-effective.