批准新药后完成加拿大卫生部要求的条件的时间：一项队列研究

IF 3.6 3区医学 Q1 HEALTH CARE SCIENCES & SERVICES

Health Policy Pub Date : 2025-04-02 DOI:10.1016/j.healthpol.2025.105314

Joel Lexchin

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引用次数: 0

摘要

背景：为了确保加拿大人及时获得治疗严重疾病的有希望的药物，加拿大卫生部可以有条件地批准这些药物，前提是公司承诺进行验证性研究以验证其益处。目的确定满足条件需要多长时间，以及是否有某些因素影响时间长度。方法从加拿大卫生部的数据库中编制到2024年底有条件批准的新药和现有药物的新适应症清单。孤儿药状态由美国食品和药物管理局数据库确定。构建Kaplan-Meier生存曲线来确定完成研究所需的时间。结果153例有条件批准，截至2025年1月18日，91例满足条件，45例未满足条件，17例被撤回。实现的中位时间为1200 （IQR 777, 1852）天。孤儿药状态以及有条件批准是新药还是现有药物的新适应症都不会影响完成时间。结论一些NOC/c需要相当长的时间才能实现。除特殊情况外，加拿大卫生部应要求在授予NOC/c时进行研究，并应对确认性研究的完成日期保持透明，并提供有关任何延误的详细报告。在无法证明合理的延误情况下，应赋予通过将NOC/c途径从政策转变为立法对制造商施加重大经济处罚的权力。

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Time to completion of conditions required by Health Canada after approving new drugs: A cohort study

Background

To ensure that promising drugs for serious illnesses reach Canadians in a timely manner, Health Canada can approve them conditionally provided companies commit to conducting confirmatory studies to verify the benefits.

Objective

To determine how long it takes until the conditions are fulfilled and if certain factors affect that length of time.

Methods

A list of conditional approvals for new drugs and new indications for existing drugs to the end of 2024 was compiled from Health Canada databases. Orphan drug status was determined from the US Food and Drug Administration databases. Kaplan-Meier survival curves were constructed to determine how long it took to complete the studies.

Results

There were 153 conditional approvals: 91 were fulfilled, 45 have not been fulfilled as of January 18, 2025 and 17 were withdrawn. The median time for fulfillment was 1200 (IQR 777, 1852) days. Orphan drug status and whether the conditional approval was for a new drug or a new indication for an existing drug did not affect the time to completion.

Conclusions

Some NOC/c take considerable time to be fulfilled. Health Canada should require studies to be underway at the time that a NOC/c is granted except in exceptional circumstances and it should be transparent about the completion date for confirmatory studies and provided detailed reports about any delays. In the case of delays that cannot be justified it should be given the power to impose significant financial penalties on manufacturers through the NOC/c pathway being converted from a policy into legislation.

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来源期刊

Health Policy 医学-卫生保健

CiteScore

6.40

自引率

6.10%

发文量

157

审稿时长

3-8 weeks

期刊介绍： Health Policy is intended to be a vehicle for the exploration and discussion of health policy and health system issues and is aimed in particular at enhancing communication between health policy and system researchers, legislators, decision-makers and professionals concerned with developing, implementing, and analysing health policy, health systems and health care reforms, primarily in high-income countries outside the U.S.A.