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患者的观点可纳入卫生技术评估:对加拿大药品和卫生技术局常见药物审查的探索性分析。

Patients' perspectives can be integrated in health technology assessments: an exploratory analysis of CADTH Common Drug Review.

作者信息

Berglas Sarah, Jutai Lauren, MacKean Gail, Weeks Laura

机构信息

CADTH, Ottawa, Canada.

London School of Economics, London, UK.

出版信息

Res Involv Engagem. 2016 Jun 7;2:21. doi: 10.1186/s40900-016-0036-9. eCollection 2016.

Abstract

PLAIN LANGUAGE SUMMARY

In Canada, the CADTH Common Drug Review helps ensure that scarce health care resources are used to fund the most effective drugs. Clinicians, researchers, payers, and patients all have important, but potentially different, ideas on what should be considered, to determine a drug's value. Since 2010, CADTH has invited patient groups to contribute their perspectives to the Common Drug Review. We explored whether, and how, insights offered by patient groups are integrated into assessment reports and Recommendations by the CADTH Canadian Drug Expert Committee. After examining 30 completed drug assessments, we found that:Patient insights are used by CADTH reviewers to frame an assessment and are used by the expert committee to interpret the evidence.Drug trials do not always measure outcomes that patients consider important.Survival, symptom relief, the process of recovery, and maintaining health are all important aspects to consider when determining value during health technology assessments.

ABSTRACT

Since 2010, Canadian patient groups have contributed to the CADTH Common Drug Review (CDR). CADTH conducts health technology assessments of new drugs to support publicly funded drug plans' reimbursement decisions. We explored whether, and how, patient insights were integrated into assessment reports and Recommendations by the CADTH Canadian Drug Expert Committee (CDEC). We descriptively analyzed 30 consecutive assessments. One researcher identified a set of issues, insights, and desired treatment outcomes provided by patient groups for each included drug assessment. We tracked the presence of each identified patient insight in the relevant assessment protocol, in clinical trials as reported in the assessment, and in the CDEC Recommendations. Additionally, patient insights were categorized by topic and grouped into a three-tier framework to explore the observed juxtaposition between immediate treatment outcomes as seen in clinical trials and the insights from patients living with a chronic condition. In 30 drug assessments, 119 patient insights were identified. Of these insights, 89 were included in assessment protocols; 61 in reported clinical trial data; and 67 insights were reflected upon within the CDEC Recommendations. Patient insights within the first framework tier (health status achieved) were frequently included in all aspects of CDR assessments. Within the second tier (progress of recovery), although two-thirds of patient insights were included in protocols, only one-third was reflected in reported trial data or in CDEC Recommendations. Insights within the third tier, which address the long-term consequences of illness and treatment, were even less frequently addressed in all aspects of CDR assessments. Patients' perspectives need not be "considered" in isolation. Patient insights are used by CADTH reviewers to frame an assessment and used by CDEC to interpret the evidence. As health technology assessments should address the indirect and unintended consequences of a technology, as well as its direct and intended effects, drug assessments should consider the progress of recovery and sustainability of health, in addition to survival and immediate health achieved.

摘要

通俗易懂的总结

在加拿大,加拿大药品和卫生技术评价机构(CADTH)的通用药品审查有助于确保稀缺的医疗资源用于资助最有效的药物。临床医生、研究人员、支付方和患者对于确定药物价值时应考虑的因素都有重要但可能不同的看法。自2010年以来,CADTH邀请患者群体为通用药品审查贡献他们的观点。我们探讨了患者群体提供的见解是否以及如何被纳入CADTH加拿大药品专家委员会的评估报告和建议中。在审查了30份完整的药物评估后,我们发现:CADTH的评审人员利用患者的见解来构建评估框架,专家委员会则利用这些见解来解释证据。药物试验并不总是衡量患者认为重要的结果。在卫生技术评估中确定价值时,生存、症状缓解、康复过程和保持健康都是需要考虑的重要方面。

摘要

自2010年以来,加拿大患者群体为CADTH通用药品审查(CDR)做出了贡献。CADTH对新药进行卫生技术评估,以支持公共资助药品计划的报销决策。我们探讨了患者的见解是否以及如何被纳入CADTH加拿大药品专家委员会(CDEC)的评估报告和建议中。我们对30项连续评估进行了描述性分析。一名研究人员为每项纳入的药物评估确定了患者群体提供的一系列问题、见解和期望的治疗结果。我们在相关评估方案、评估中报告的临床试验以及CDEC的建议中追踪了每个确定的患者见解的存在情况。此外,患者见解按主题分类并分组到一个三层框架中,以探讨临床试验中观察到的即时治疗结果与慢性病患者的见解之间的并列关系。在30项药物评估中,确定了119条患者见解。在这些见解中,89条被纳入评估方案;61条在报告的临床试验数据中;67条见解在CDEC的建议中得到了体现。第一个框架层级(实现的健康状况)中的患者见解经常被纳入CDR评估的各个方面。在第二个层级(康复进展)中,虽然三分之二的患者见解被纳入方案,但只有三分之一反映在报告的试验数据或CDEC的建议中。第三个层级中涉及疾病和治疗长期后果的见解在CDR评估的各个方面中得到讨论的频率更低。患者的观点不应孤立地“被考虑”。CADTH的评审人员利用患者的见解来构建评估框架,CDEC则利用这些见解来解释证据。由于卫生技术评估应解决技术的间接和意外后果以及其直接和预期效果,药物评估除了应考虑生存和实现的即时健康外,还应考虑康复进展和健康的可持续性。

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