Measuring treatment impact: a review of patient-reported outcomes and other efficacy endpoints in approved product labels

Richard J. Willke , Laurie B. Burke , Pennifer Erickson
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引用次数: 300

Abstract

Context

The term “patient-reported outcomes” (PROs) has evolved to include any endpoint derived from patient reports, whether collected in the clinic, in a diary, or by other means, including single-item outcome measures, event logs, symptom reports, formal instruments to measure health-related quality of life (HRQL), health status, adherence, and satisfaction with treatment. This term coincides with the explicit interest from drug development researchers and regulatory authorities in the appropriate utilization and reporting of treatment impact measures.

Objective

To determine the level and nature of use of PROs compared to other types of effectiveness endpoints in approved product labeling for new drugs recently approved in the United States.

Design and sources

Review and analysis of effectiveness endpoints as reported in clinical study descriptions in approved product labeling of new molecular entities (NMEs) approved in the United States from 1997 through 2002.

Main outcome measures

Effectiveness study endpoints reported in approved product labeling that fall into the following categories of measurement: PROs, clinician-reported outcomes (CROs), and laboratory test/device measurement endpoints.

Results

PROs were reported in 64 (30%) of the 215 product labels reviewed. Clinician-reported outcomes were reported most frequently (62%) followed by laboratory/device endpoints (50%). PROs were the only type of endpoint used in the FDA-approved label for 23 products. Formal multiitem PRO scales were cited 22 times. Use of PROs is most common in antiinflammatory, CNS, gastrointestinal, respiratory, allergic conjunctivitis, and urologic therapy areas. The frequency of reported PRO use over this period did not change.

Conclusion

PROs, although quite variable as a class of study endpoints, were found to have a significant role in the development and evaluation of new medicines. More formal guidance from the FDA about use of such measures along with continued collaboration by PRO researchers to develop and disseminate standards will enhance the appropriate use of PROs in future drug development and labeling.

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测量治疗效果:对已批准产品标签中患者报告的结果和其他疗效终点的回顾
“患者报告的结局”(pro)一词已经发展到包括从患者报告中得出的任何终点,无论是在诊所、日记中还是通过其他方式收集的,包括单项结果测量、事件日志、症状报告、测量健康相关生活质量(HRQL)、健康状况、依从性和治疗满意度的正式工具。这一术语与药物开发研究人员和监管当局对适当利用和报告治疗影响措施的明确兴趣相吻合。目的在美国最近批准的新药标签中,与其他类型的有效性终点相比,确定使用PROs的水平和性质。设计和来源回顾和分析1997年至2002年在美国批准的新分子实体(NMEs)产品标签中临床研究描述中报告的有效性终点。主要结果测量在批准的产品标签中报告的有效性研究终点分为以下测量类别:PROs、临床报告的结果(cro)和实验室测试/设备测量终点。结果在审查的215个产品标签中,有64个(30%)报告了pros。临床报告的结果报告最频繁(62%),其次是实验室/设备终点(50%)。在fda批准的23种产品的标签中,PROs是唯一一种终点。正式的多条目PRO量表被引用22次。PROs最常见于抗炎、中枢神经系统、胃肠道、呼吸道、过敏性结膜炎和泌尿系统治疗领域。在此期间,报告的PRO使用频率没有变化。结论作为一类研究终点,pros虽然变化很大,但在新药的开发和评价中具有重要作用。FDA关于使用此类措施的更正式的指导,以及PRO研究人员在开发和传播标准方面的持续合作,将增强PRO在未来药物开发和标签中的适当使用。
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