College of Pharmacy, Chung-Ang University, Seoul, Republic of Korea.
Expert Rev Pharmacoecon Outcomes Res. 2023 Jun;23(5):519-525. doi: 10.1080/14737167.2023.2192481. Epub 2023 Mar 20.
This study aims to analyze the effect of the copayment reduction system on accessibility to orphan drugs (ODs) in South Korea.
Data on approval and reimbursement for drugs designated as ODs for the last 10 years (2012-2021) in South Korea were extracted. Among them, with 136 approved products as of 31 December 2022, the reimbursement rates and lead time to reimbursement between drugs for rare diseases (DRDs) and nondrugs for rare diseases (non-DRDs) were analyzed. The pricing and reimbursement (P&R) pathways between drugs for only rare diseases (DORDs) and drugs for rare and cancerous diseases (DRCDs) were compared.
The reimbursement rates for DRDs and non-DRDs were 54.8% and 33.3%, respectively, and the lead time to reimbursement for DRDs and non-DRDs were 16.1 months and 31.2 months, respectively. The P&R pathways for DORDs and DRCDs were pharmacoeconomic evaluation waivers (21.7% and 52.6%), weighted average price (52.2% and 13.2%), and risk-sharing agreement (30.4% and 81.6%).
The copayment reduction system may act as a driver and also barrier for the reimbursement of ODs. To expand treatment accessibility to ODs, it is necessary to consistently grants benefits in all processes from OD designation to market access.
本研究旨在分析自付额降低制度对韩国罕见病药物（ODs）可及性的影响。
提取韩国过去 10 年（2012-2021 年）指定为 ODs 的药物的批准和报销数据。截至 2022 年 12 月 31 日，共批准了 136 种产品，分析了罕见病药物（DRD）和非罕见病药物（非-DRD）之间的报销率和报销时间。比较了仅罕见病药物（DORD）和罕见病和癌症药物（DRCD）之间的定价和报销（P&R）途径。
DRD 和非-DRD 的报销率分别为 54.8%和 33.3%，DRD 和非-DRD 的报销时间分别为 16.1 个月和 31.2 个月。DORD 和 DRCD 的 P&R 途径分别为药物经济学评估豁免（21.7%和 52.6%）、加权平均价格（52.2%和 13.2%）和风险分担协议（30.4%和 81.6%）。
自付额降低制度可能既是 ODs 报销的推动因素，也是障碍。为了扩大 ODs 的治疗可及性，有必要从 OD 指定到市场准入的所有流程中持续给予福利。
