加拿大孤儿药常见药物评估建议：建议基础及与魁北克、澳大利亚、苏格兰和新西兰相似评估的比较。

Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand.

机构信息

McKesson Specialty Health, 4705 Dobrin Street, Quebec, Saint-Laurent, H4R 2P7, Canada.

McKesson Specialty Health, 6355 Viscount Road, Mississauga, ON, L4V 1W2, Canada.

出版信息

Orphanet J Rare Dis. 2018 Jan 30;13(1):27. doi: 10.1186/s13023-018-0759-9.

DOI:10.1186/s13023-018-0759-9

PMID:29382371

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5791218/

Abstract

BACKGROUND

Public payer reimbursement for non-oncology drugs in Canada, including orphan drugs, is based on recommendations by the Common Drug Review (CDR) (with the exception of Quebec). CDR has been criticized for negative recommendations for orphan drugs and contributing to delays in patient access to these drugs. However, it is unclear how CDR makes recommendations for orphan drugs and the role clinical and economic factors play in decision making. The objective of the present study was to analyze the basis for CDR orphan drug recommendations and to compare recommendations to those in other jurisdictions.

METHODS

A list of orphan drugs reviewed by CDR (between 2004 and 2017) was compiled and final recommendations (list/do not list) assessed. The basis of each recommendation was categorized as clinical only, price only or combined clinical and price factors, based on the ranking of clinical and price parameters in recommendation summaries. The reimbursement status of the same drugs was determined in Quebec and other jurisdictions and level of agreement with CDR decisions assessed using a kappa analysis.

RESULTS

Sixty eight orphan drug submissions were identified in the CDR database. Clinical, clinical and price and price parameters were the basis of 48.5%, 44.1% and 7.4% of the reviews, respectively, and corresponding positive recommendation rates were 45.5%, 86.7% and 40.0% (p = 0.0008); overall positive recommendation rate was 63.2%. Positive recommendation rate increased from 50.0% for drugs reviewed between 2004 and 2009 to 86.7% in 2016; however, 84.6% of the latter were conditional on a price reduction. Of the drugs reviewed by CDR, 80.9%, 88.2%, 80.9% and 58.8% were reviewed for the same indications by health technology assessment agencies in Quebec, Scotland, Australia and New Zealand, respectively, with positive listing rates ranging from 60.0% (Quebec) to 92.7% (Australia) with fair (kappa coefficient 0.3307) to poor (kappa coefficient 0.0611) agreement with CDR in listing decisions, respectively.

CONCLUSIONS

The positive CDR recommendation rate for orphan drugs was highest when clinical and price parameters supported the assessment. Over time there has been an increase in CDR positive recommendation rates for orphan drugs, although most are conditional on a price reduction. It is unclear if this change in CDR recommendations will impact equitable and timely access to orphan drugs across Canada.

摘要

背景

加拿大的公共支付者对非肿瘤药物（包括孤儿药）的报销是基于共同药物评审（CDR）的建议（魁北克省除外）。CDR 因对孤儿药的负面建议以及导致患者获得这些药物的时间延迟而受到批评。然而，CDR 如何为孤儿药提出建议，以及临床和经济因素在决策中的作用尚不清楚。本研究的目的是分析 CDR 孤儿药建议的依据，并将其与其他司法管辖区的建议进行比较。

方法

编制了 CDR（2004 年至 2017 年）评审的孤儿药清单，并评估了最终建议（列入/不列入）。根据建议摘要中临床和价格参数的排名，将每项建议的依据分为仅临床、仅价格或临床和价格综合因素。确定了魁北克省和其他司法管辖区相同药物的报销情况，并使用 Kappa 分析评估与 CDR 决策的一致性程度。

结果

在 CDR 数据库中确定了 68 种孤儿药申请。临床、临床和价格以及价格参数分别是 48.5%、44.1%和 7.4%的审查的依据，相应的阳性推荐率分别为 45.5%、86.7%和 40.0%（p=0.0008）；总体阳性推荐率为 63.2%。阳性推荐率从 2004 年至 2009 年审查药物的 50.0%上升至 2016 年的 86.7%；然而，其中 84.6%是基于降价条件。在 CDR 审查的药物中，有 80.9%、88.2%、80.9%和 58.8%在魁北克、苏格兰、澳大利亚和新西兰的卫生技术评估机构被审查用于相同的适应证，阳性列入率从 60.0%（魁北克）到 92.7%（澳大利亚）不等，与 CDR 在列入决策方面的一致性程度从适度（kappa 系数 0.3307）到较差（kappa 系数 0.0611）不等。