Center for the Evaluation of Value and Risk in Health, Tufts Medical Center, Boston, MA, USA.
Center for the Evaluation of Value and Risk in Health, Tufts Medical Center, Boston, MA, USA.
Value Health. 2024 Dec;27(12):1656-1661. doi: 10.1016/j.jval.2024.07.005. Epub 2024 Jul 31.
To examine ultraorphan drugs in terms of incremental health, costs, and cost-effectiveness compared with more prevalent disease drugs.
We identified Food and Drug Administration drug approvals from 1999 to 2019. For drugs approved for multiple indications, we considered each drug-indication pair separately. Utilizing Food and Drug Administration's orphan drug designation and US disease prevalence, we categorized drug-indication pairs as: ultraorphan (<10 000 patients), "other" orphan (≥10 000 and <200 000), and nonorphan (≥200 000). We searched the PubMed database for cost-effectiveness analyses and comparative effectiveness 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 with 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, Mann-Whitney U (MWU), and Kolmogorov-Smirnov (KS) tests.
Median incremental QALYs, costs, and ICERs differed across nonorphan, "other" orphan, and ultraorphan categories (Kruskal-Wallis P < .01). Compared with nonorphan drugs, ultraorphan drugs had larger QALY gains (0.700 vs 0.050, MWU P < .01, KS P < .01), larger costs ($172 231 vs $3360, MWU P < .01, KS P < .01), and larger ICERs ($1 216 184/QALY vs $114 061/QALY, MWU P < .01, KS P <.01). Compared with "other" orphan drugs, ultraorphan drugs had larger QALY gains (0.700 vs 0.310, MWU P =.65, KS P =.32), larger costs ($172 231 vs $69 308, MWU P = .03, KS P = .03), and larger ICERs ($1 216 184/QALY vs $223 472/QALY, MWU P <.01, KS P <.01).
Novel ultraorphan drugs typically offer larger incremental health gains than drugs for more prevalent diseases, but because of their substantial added costs, are typically less cost-effective.
根据与更常见疾病药物相比的增量健康、成本和成本效益，研究超孤儿药物。
我们确定了 1999 年至 2019 年期间食品和药物管理局批准的药物。对于批准用于多种适应症的药物，我们分别考虑每种药物-适应症组合。利用食品和药物管理局的孤儿药物指定和美国疾病流行率，我们将药物-适应症组合分类为：超孤儿（<10 000 例患者）、“其他”孤儿（≥10 000 例和<200 000 例）和非孤儿（≥200 000 例）。我们在 PubMed 数据库中搜索成本效益分析和比较有效性研究。我们排除了制造商资助的研究。我们提取了与批准时的标准护理相比，药物-适应症组合的增量健康获益（以质量调整生命年（QALY）表示）和增量成本的估计值。我们使用 Kruskal-Wallis、Mann-Whitney U（MWU）和 Kolmogorov-Smirnov（KS）检验比较 QALY 获益、附加成本和增量成本效益比（ICER）。
非孤儿、“其他”孤儿和超孤儿类别之间的中位增量 QALY、成本和 ICER 存在差异（Kruskal-Wallis P<.01）。与非孤儿药物相比，超孤儿药物的 QALY 获益更大（0.700 与 0.050，MWU P<.01，KS P<.01），成本更高（172 231 美元与 3360 美元，MWU P<.01，KS P<.01），ICER 更高（1 216 184 美元/QALY 与 114 061 美元/QALY，MWU P<.01，KS P<.01）。与“其他”孤儿药物相比，超孤儿药物的 QALY 获益更大（0.700 与 0.310，MWU P=.65，KS P=.32），成本更高（172 231 美元与 69 308 美元，MWU P=.03，KS P=.03），ICER 更高（1 216 184 美元/QALY 与 223 472 美元/QALY，MWU P<.01，KS P<.01）。
新型超孤儿药物通常比治疗更常见疾病的药物提供更大的增量健康获益，但由于其巨额附加成本，通常不太具有成本效益。
