Dicerna Pharmaceuticals, Lexington MA.
Trinity Life Sciences, Waltham, MA.
J Manag Care Spec Pharm. 2022 Mar;28(3):316-323. doi: 10.18553/jmcp.2022.28.3.316.
Primary hyperoxaluria (PH) is a family of rare, life-threatening genetic liver disorders characterized by elevated production and excretion of oxalate. To date, the clinical and economic burden associated with PH has not been well characterized due to the rarity of the disease and previous challenges with diagnostic coding that prevented proper identification of patients with PH in claims data. To characterize the clinical and economic costs, as well as health care resource utilization (HCRU), associated with PH relative to a matched cohort of patients without PH. Data from the IQVIA PharMetrics Plus Database were used to conduct a retrospective matched-cohort study to compare differences in clinical characteristics, HCRU, and pharmacy and medical costs in patients with PH compared with a matched cohort of patients without PH from January 2014 to December 2019. Overall, 324 patients were included in the PH cohort and 1,620 patients were in the non-PH cohort. The mean age of PH patients was 48.1 years, and approximately 58% of the sample were male. Significantly more patients in the PH cohort than the non-PH cohort were diagnosed with stage 2 chronic kidney disease (CKD; 3.1% vs 0.4%, respectively; < 0.001), stage 3 CKD (4.6% vs 0.5%; < 0.001), stage 4 CKD (2.5% vs 0.1%; < 0.001), and stage 5 CKD or end-stage renal disease (ESRD; 2.2% vs 0.1%; < 0.001). PH patients had a significantly higher mean Charlson Comorbidity Index composite score than patients in the non-PH cohort (0.79 vs 0.37; < 0.001). HCRU was significantly higher in patients with PH. The PH cohort had a significantly higher proportion of patients with at least 1 visit to clinicians specializing in nephrology (19% vs 0.4%, respectively; < 0.001), cardiology (22% vs 12%; < 0.001), ophthalmology (16% vs 7%; < 0.001), general surgery (9% vs 6%; = 0.011), and urology (65% vs 6%; < 0.001) compared with patients without PH. Mean total annual health care costs in the PH cohort were 65% higher than in the non-PH cohort ($22,549 vs $7,852, respectively; < 0.001). Similar results were found for total prescription drug costs ($4,125 vs $2,464; = 0.012). Despite the rarity of PH, patients with this disease incur substantial clinical and economic burden and may cause financial strain on the health care system. Additional research is warranted to understand the economic and clinical burden of PH stratified by the 3 subtypes of the disease. Funding for this research was provided by Dicerna Pharmaceuticals. Mucha and Hoppe are employed by Dicerna Pharmaceuticals. Silber Miyasoto, Skaar, and Wang are employed by Trinity Life Sciences, which was contracted by Dicerna Pharmaceuticals to conduct the study analysis. Langman is consultant to Dicerna Pharmaceuticals. This study was presented as a poster at the AMCP Nexus 2020 (virtual), October 19-23, 2020, and American Society of Nephrology 2020 (virtual), October 19-25, 2020.
原发性高草酸尿症（PH）是一组罕见的危及生命的遗传性肝脏疾病，其特征为草酸盐的产生和排泄增加。迄今为止，由于疾病的罕见性以及以前在诊断编码方面存在的挑战，这些挑战阻止了在索赔数据中正确识别患有 PH 的患者，因此尚未很好地描述与 PH 相关的临床和经济负担。本研究旨在描述与未患有 PH 的匹配队列相比，PH 患者的临床和经济成本以及医疗保健资源利用（HCRU）。
使用 IQVIA PharMetrics Plus 数据库中的数据进行了一项回顾性匹配队列研究，以比较 2014 年 1 月至 2019 年 12 月期间 PH 患者与未患有 PH 的匹配队列患者在临床特征、HCRU、药房和医疗成本方面的差异。总体而言，324 名患者被纳入 PH 队列，1620 名患者被纳入非 PH 队列。PH 患者的平均年龄为 48.1 岁，约 58%的样本为男性。与非 PH 队列相比，PH 队列中有更多的患者被诊断为慢性肾脏病（CKD）2 期（3.1%比 0.4%；<0.001）、CKD3 期（4.6%比 0.5%；<0.001）、CKD4 期（2.5%比 0.1%；<0.001）和 CKD5 期或终末期肾病（ESRD；2.2%比 0.1%；<0.001）。PH 患者的Charlson 合并症指数综合评分明显高于非 PH 队列（0.79 比 0.37；<0.001）。PH 患者的 HCRU 明显更高。PH 队列中至少有 1 次就诊于肾脏病学专家的患者比例明显高于非 PH 队列（19%比 0.4%；<0.001）、心脏病学专家（22%比 12%；<0.001）、眼科专家（16%比 7%；<0.001）、普通外科专家（9%比 6%；=0.011）和泌尿科专家（65%比 6%；<0.001）。PH 队列的年总医疗保健费用比非 PH 队列高 65%（分别为 22549 美元和 7852 美元；<0.001）。处方药物总成本也有类似的结果（4125 美元比 2464 美元；=0.012）。
尽管 PH 较为罕见，但患有这种疾病的患者会承受巨大的临床和经济负担，并可能给医疗保健系统带来财务压力。需要进一步研究来了解根据疾病的 3 种亚型划分的 PH 的经济和临床负担。这项研究的资金由 Dicerna 制药公司提供。Mucha 和 Hoppe 受雇于 Dicerna 制药公司。Silber Miyasoto、Skaar 和 Wang 受雇于 Trinity Life Sciences，该公司受 Dicerna 制药公司委托进行研究分析。Langman 是 Dicerna 制药公司的顾问。这项研究以海报形式在 2020 年 AMCP Nexus（虚拟）上发表，2020 年 10 月 19-23 日，和 2020 年美国肾脏病学会（虚拟）上发表，2020 年 10 月 19-25 日。
