Hong Jihyung, Bae Eun-Young, Cha Sohee, Lee Joohyun
Department of Healthcare Management, Gachon University, Seongnam-Si, Gyeonggi-Do, Republic of Korea.
College of Pharmacy, Gyeongsang National University, 501 Jinju-Daero, Jinju, 52828, Republic of Korea.
BMC Health Serv Res. 2025 Jan 14;25(1):74. doi: 10.1186/s12913-025-12207-9.
Innovative health technologies have increasingly emerged as a promising solution for patients with untreatable or challenging conditions. However, these technologies often come with expensive costs and limited evidence at the time of launch. This study assessed how these high-priced drugs with limited evidence were appraised and introduced in South Korea, England, Australia, and Canada, where cost-effectiveness analysis (CEA) generally plays a central role in pricing and reimbursement decisions.
The study analysed 22 high-priced drugs (24 indications) introduced in South Korea, which were granted CEA waivers due to difficulties in evidence generation and high unmet needs. Data, including funding arrangements and evidence assessed, were derived from national health technology assessment (HTA) documents and other public domains in the four countries.
Nearly all drugs received positive recommendations, largely through managed entry agreements (MEAs), particularly in England. Single-arm trials were more common in South Korea and England. Indirect comparison was the primary source of comparative effectiveness in England (70.0%), emphasising alignment with current practices. Australia and Canada utilised both indirect comparison and head-to-head trial data in similar proportions. Except for South Korea, all countries still required CEA data for these drugs. Data collection for coverage with evidence development was necessary in 55.0% of cases in England, and less in other countries.
HTA agencies increasingly accept the uncertainty of high-priced drugs with high unmet needs through MEAs. To ensure timely access and value for money, implementing full value assessment and uncertainty management, while strengthening national and international collaboration for effective data collection, is imperative.
创新医疗技术日益成为治疗无法治愈或具有挑战性疾病患者的一种有前景的解决方案。然而，这些技术在推出时往往成本高昂且证据有限。本研究评估了这些证据有限的高价药物在韩国、英国、澳大利亚和加拿大是如何被评估和引入的，在这些国家，成本效益分析（CEA）通常在定价和报销决策中发挥核心作用。
该研究分析了在韩国引入的22种高价药物（24种适应症），这些药物因证据生成困难和未满足的高需求而获得CEA豁免。包括资金安排和评估证据在内的数据来自四个国家的国家卫生技术评估（HTA）文件和其他公共领域。
几乎所有药物都获得了积极推荐，主要是通过管理式进入协议（MEA），尤其是在英国。单臂试验在韩国和英国更为常见。间接比较是英国比较有效性的主要来源（70.0%），强调与当前实践保持一致。澳大利亚和加拿大以相似比例同时使用间接比较和头对头试验数据。除韩国外，所有国家仍要求提供这些药物的CEA数据。在英国，55.0%的案例需要收集证据开发覆盖范围的数据，其他国家则较少。
HTA机构越来越多地通过MEA接受高未满足需求的高价药物的不确定性。为确保及时获得药物并实现物有所值，实施全面价值评估和不确定性管理，同时加强国家和国际合作以有效收集数据，势在必行。
