Farris Maria, Goodall Stephen, De Abreu Lourenco Richard
Astra Zeneca, Health Economics, Macquarie Park, NSW, Australia.
Centre for Health Economic Research and Evaluation, https://ror.org/03f0f6041University of Technology Sydney, Ultimo, NSW, Australia.
Int J Technol Assess Health Care. 2025 Jul 8;41(1):e61. doi: 10.1017/S0266462325100226.
Researchers propose wider individual and societal benefits (or broad elements of value) be included in economic evaluations (EEs) of medicines. This study investigates opinions of Australian stakeholders regarding the inclusion of broader value elements in reimbursement decisions for medicines for rare diseases in Australia.
Stakeholders were invited via email to complete an online survey about their views on broader elements of value in HTA. Responses were summarised using descriptive statistics and compared using chi-square statistics.
Forty-four respondents (academia (n=11), private sector (n=33)) completed the survey between October 2023 and May 2024. Only 27% of stakeholders agree the current information about the sources of value considered in reimbursement decisions is sufficient. Stakeholders consistently agree labour productivity (>50%), adherence (>80%), reducing uncertainty due to a new diagnostic (>70%), disease severity (>71%), value to caregivers (>70%), and equity (>70%) should be considered in HTA. The majority (>70%) agreed managed entry agreements (MEA), risk share arrangements (RSA), and multi criteria decision analysis (MCDA) be used in reimbursement decision making for medicines for rare diseases. Significantly fewer academic stakeholders (40%) versus private sector (77%), believe an increased willingness-to-pay threshold be applied to medicines for rare disease.
Academic and private sector stakeholders hold similar views when considering medicines for non-rare and rare diseases. Stakeholders favour considering more value elements in HTA than referred to in the Pharmaceutical Benefits Advisory Committee (PBAC) guidelines. This study highlights further advice is needed on the factors considered in reimbursement decisions and how that would influence guidelines.
研究人员提议在药品的经济评估（EEs）中纳入更广泛的个人和社会效益（或广泛的价值要素）。本研究调查了澳大利亚利益相关者对于在澳大利亚罕见病药品报销决策中纳入更广泛价值要素的看法。
通过电子邮件邀请利益相关者完成一项关于他们对卫生技术评估（HTA）中更广泛价值要素看法的在线调查。使用描述性统计对回复进行总结，并使用卡方统计进行比较。
44名受访者（学术界（n = 11），私营部门（n = 33））在2023年10月至2024年5月期间完成了调查。只有27%的利益相关者认为报销决策中考虑的当前价值来源信息是充分的。利益相关者一致认为，卫生技术评估应考虑劳动生产率（>50%）、依从性（>80%）、降低新诊断带来的不确定性（>70%）、疾病严重程度（>71%）、对护理人员的价值（>70%）以及公平性（>70%）。大多数（>70%）人同意在罕见病药品报销决策中使用管理式进入协议（MEA）、风险分担安排（RSA）和多标准决策分析（MCDA）。与私营部门（77%）相比，学术界利益相关者（40%）认为应提高罕见病药品支付意愿阈值的人数明显更少。
在考虑非罕见病和罕见病药品时，学术界和私营部门利益相关者持有相似观点。利益相关者倾向于在卫生技术评估中考虑比药品福利咨询委员会（PBAC）指南中提及的更多价值要素。本研究强调在报销决策中考虑的因素以及这将如何影响指南方面需要进一步的建议。
