Health Technology & Policy Unit, School of Public Health, University of Alberta, Edmonton, AB, Canada.
Department of Medicine, University of Calgary, Calgary, AB, Canada.
Orphanet J Rare Dis. 2019 Jun 7;14(1):127. doi: 10.1186/s13023-019-1104-7.
In Canada, reimbursement recommendations on drugs for common and rare diseases are overseen by the Canadian Agency for Drugs and Technologies in Health (CADTH) and made through the pan-Canadian Oncology Drug Review (pCODR) and the Common Drug Review (CDR). While the agency specifies information requirements for the review of drug submissions, how that information is used by each process to formulate final reimbursement recommendations, particularly on drugs for rare diseases (DRDs) in which per patient treatment costs are often high, is unclear. The purpose of this study was to determine which factors contribute to recommendation type for DRDs.
Information was extracted from CDR and pCODR recommendations on drugs for diseases with a prevalence < 1 in 2000 from January 2012 to April 2018. Data were tabulated and multiple logistic regression was applied to explore the association between recommendation type and the following factors: condition/review process (cancer vs non-cancer), year, prevalence, clinical effectiveness (improvements in surrogate, clinical and patient reported outcomes), safety, quality of evidence (availability of comparative data, consistency between population in trial and indication, and bias), clinical need, treatment cost, and incremental cost-effective ratio (ICER). Two-way interactions were also explored.
A total of 103 recommendations were included. Eleven were resubmissions, all of which received a positive recommendation. Among new submissions (n = 92), DRDs that were safe or offered improvements in clinical or patient reported outcomes were more likely to receive positive reimbursement recommendations. No associations between recommendation type and daily treatment cost, cost-effectiveness, or condition (cancer or non-cancer) were found.
Clinical effectiveness, as opposed to economic considerations or whether the drug is indicated for cancer or non-cancer, determine the type of reimbursement recommendation.
在加拿大，常见和罕见疾病药物的报销建议由加拿大药品和技术评估机构（CADTH）监督，并通过全加肿瘤药物评审（pCODR）和通用药物评审（CDR）进行。虽然该机构为药物评审规定了信息要求，但每个流程如何使用这些信息来制定最终的报销建议，特别是对于每位患者治疗费用通常较高的罕见病（DRD）药物，目前尚不清楚。本研究旨在确定哪些因素会影响 DRD 的推荐类型。
从 2012 年 1 月至 2018 年 4 月，我们从 CDR 和 pCODR 对患病率<1/2000 的疾病药物的建议中提取信息。对数据进行制表，并应用多因素逻辑回归来探讨推荐类型与以下因素之间的关系：疾病/评审流程（癌症与非癌症）、年份、患病率、临床疗效（替代指标、临床和患者报告结局的改善）、安全性、证据质量（比较数据的可用性、试验人群与适应证之间的一致性以及偏倚）、临床需求、治疗费用和增量成本效益比（ICER）。还探索了双向交互作用。
共纳入 103 项建议。其中 11 项为重新提交，均获得了积极的建议。在新提交的建议中（n=92），安全或能改善临床或患者报告结局的 DRD 更有可能获得积极的报销建议。未发现推荐类型与每日治疗费用、成本效益或疾病（癌症或非癌症）之间存在关联。
临床疗效而非经济考虑因素或药物是否适用于癌症或非癌症决定了报销建议的类型。
