Centre for Health Economics Research and Evaluation, University of Technology Sydney, PO Box 123 Broadway NSW 2007, Australia.
Centre for Health Economics Research and Evaluation, University of Technology Sydney, PO Box 123 Broadway NSW 2007, Australia.
Mult Scler Relat Disord. 2018 Oct;25:144-149. doi: 10.1016/j.msard.2018.07.020. Epub 2018 Aug 1.
In Australia, the Pharmaceutical Benefits Advisory Committee (PBAC) advises on the reimbursement of drugs to be subsidised through the Pharmaceutical Benefits Scheme (PBS). This study aims to provide insights into the PBAC process and key considerations regarding the reimbursement of MS drugs in Australia.
The factors considered by the PBAC and its advice on whether to reimburse a drug are documented in public summary documents (PSDs). Qualitative content analysis of PSDs was conducted for all MS drugs considered by the PBAC between January 2006 and January 2018. Key issues identified by the PBAC were extracted and categorised. Common issues were identified and compared between drugs indicated for MS.
A total of 23 submissions were evaluated relating to 13 MS drugs. Eight were recommended for reimbursement; an approval rate of 35% per submission and 62% per drug. Approval rates were higher for disease modifying treatments (73% per drug) than for symptomatic drugs (0% for nabiximols and fampridine submissions). The most frequently discussed issues in PSDs, irrespective of PBAC decision, were: (1) the validity of the indirect comparisons formed (n = 11); (2) the validity of the approach to obtain utilities (n = 6); (3) the lack of appropriate/long-term safety data (n = 8); and (4) the time horizon used in the economic models (n = 3).
A small but important number of issues have been consistently identified by the PBAC in relation to submissions for reimbursement of MS drugs. Drug developers and clinical trial investigators who are aware of these issues will be able to anticipate data requirements for reimbursement decision-making and thus potentially improve the evidence submitted for listing of MS drugs in Australia.
在澳大利亚，药品福利咨询委员会（PBAC）就药物通过药品福利计划（PBS）补贴的报销问题提供建议。本研究旨在深入了解 PBAC 流程以及澳大利亚对多发性硬化症（MS）药物报销的关键考虑因素。
PBAC 考虑的因素及其关于是否报销药物的建议都记录在公共摘要文件（PSD）中。对 2006 年 1 月至 2018 年 1 月期间 PBAC 审议的所有 MS 药物的 PSD 进行了定性内容分析。提取并分类了 PBAC 确定的关键问题。对用于 MS 的药物之间的常见问题进行了识别和比较。
共评估了与 13 种 MS 药物相关的 23 项申请。有 8 种药物被推荐报销；每提交一份的批准率为 35%，每药物的批准率为 62%。治疗疾病的药物（每药物 73%）比对症药物（纳比昔醇和金刚烷胺申请的批准率为 0%）的批准率更高。无论 PBAC 决定如何，PSD 中讨论最多的问题都是：（1）形成的间接比较的有效性（n=11）；（2）获得效用的方法的有效性（n=6）；（3）缺乏适当/长期安全性数据（n=8）；以及（4）经济模型中使用的时间范围（n=3）。
PBAC 一直在与 MS 药物报销申请相关的问题上持续确定一些重要但数量较少的问题。了解这些问题的药物开发商和临床试验研究者将能够预测报销决策所需的数据，从而有可能改善澳大利亚 MS 药物上市的申报证据。
