, Saskatoon, SK, Canada.
Canadian Health Policy Institute, Toronto, ON, Canada.
Orphanet J Rare Dis. 2022 Jun 13;17(1):218. doi: 10.1186/s13023-022-02390-x.
Since 2014, the Canadian Agency for Drugs and Technologies in Health (CADTH), which performs health technology assessments for all federal, provincial and territorial government drug programs (except Quebec's) and the pan-Canadian Pharmaceutical Alliance (pCPA), which conducts price negotiations with manufacturers for all government drug programs, have been aligning their processes.
To examine trends in CADTH recommendations for non-oncology drugs for rare disorders (DRDs) released between 2014 and 2021, results of pCPA negotiations for the same drugs, and listings in government drug plans to assess who benefits from the alignment.
Recommendations were positive in 87% of the reviews, although all included clinical criteria for use and/or economic conditions. Almost 90% of the DRDs with a positive recommendation had a successful price negotiation and 71% of those with a negative recommendation had no negotiation. Although no recommendation published before mid-2016 had a specified price reduction, almost 95% of those issued afterwards included the price reduction required to achieve a specific low cost-effectiveness threshold. The median time between the DRDs receiving marketing approval and a completed price negotiation was 663 days. Negotiations for DRDs completed after 2017 generally had fewer listings in government drug plans, but there was no distinct trend. The drug's price likely played a role in listing decisions. When DRDs were listed, drug plans had access criteria consistent with CADTH's or stronger for all the DRDs.
The governments who own, fund and manage CADTH and the pCPA benefit from their alignment. The alignment is less beneficial for patients waiting for access to the DRDs. The time taken by CADTH and pCPA actions and individual government drug plans to make listing decisions delays access. CADTH's clinical criteria have become more extensive and are applied rigorously by drug plans which restricts patient access to DRDs. Canadians with rare disorders urgently need their governments to implement a long-overdue, comprehensive rare disease strategy to ensure DRDs are reviewed and reimbursed quickly and equitably to provide adequate health care to all who need them.
自 2014 年以来，加拿大药品和技术评估机构（CADTH）一直在为所有联邦、省和地区政府药品计划（魁北克省除外）以及全加药品采购联盟（pCPA）进行健康技术评估，pCPA 与所有政府药品计划的制造商进行价格谈判。CADTH 和 pCPA 一直在调整他们的流程。
研究 2014 年至 2021 年间 CADTH 对罕见疾病药物（DRD）的非肿瘤药物建议的趋势、pCPA 对相同药物的谈判结果以及政府药品计划中的上市情况，以评估谁从调整中受益。
在 87%的审查中，建议是积极的，尽管所有建议都包含了使用的临床标准和/或经济条件。近 90%的阳性推荐 DRD 药物的价格谈判都取得了成功，而 71%的阴性推荐 DRD 药物的价格谈判则没有成功。虽然 2016 年年中之前发布的任何建议都没有指定降价，但之后发布的建议几乎都包含了达到特定低成本效益阈值所需的降价。DRD 获得市场批准到完成价格谈判的中位数时间为 663 天。2017 年后完成谈判的 DRD 药物在政府药品计划中的上市数量通常较少，但没有明显的趋势。药物的价格可能在上市决策中发挥了作用。当 DRD 药物上市时，药品计划的准入标准与 CADTH 或更严格的准入标准一致。
拥有、资助和管理 CADTH 和 pCPA 的政府从他们的调整中受益。这种调整对等待获得 DRD 的患者的益处较小。CADTH 和 pCPA 采取行动以及各个政府药品计划做出上市决策所花费的时间延迟了患者获得药物的机会。CADTH 的临床标准已经变得更加广泛，并且被药品计划严格执行，这限制了患者获得 DRD 的机会。患有罕见疾病的加拿大人迫切需要他们的政府实施一项姗姗来迟的、全面的罕见疾病战略，以确保迅速、公平地审查和报销 DRD，为所有需要的人提供足够的医疗保健。
