Incorporating the patient's perspective into drug development and communication: An ad hoc task force report of the patient-reported outcomes (PRO) harmonization group meeting at the Food and Drug Administration, February 16, 2001
Incorporating the patient's perspective into drug development and communication: An ad hoc task force report of the patient-reported outcomes (PRO) harmonization group meeting at the Food and Drug Administration, February 16, 2001
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DOI：
10.1046/j.1524-4733.2003.65309.x
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发表时间：
2003-09-01
期刊：
影响因子：
4.5
通讯作者：
Rothman, M
中科院分区：
文献类型：
--
作者：
Acquadro, C;Berzon, R;Rothman, M
背景：以患者为基础的结果在多大程度上可以用来评估和传达新药和设备的效果，这是一个很有争议的主题。在美国和欧洲，许多科学家和组织提出了评估数据科学质量的标准，以支持与健康相关的生活质量(HRQL)和其他基于患者的标签和宣传主张。自2000年3月以来，一个由国际生活质量研究学会(ISOQOL)、国际药物经济学和结果研究学会(ISPOR)、制药商协会健康结果委员会(PhRMAHOC)和欧洲生活质量评估监管问题(ERIQA)成员组成的工作组开会，讨论和协调各自小组的各种建议，并解决在美国和欧洲监管机构内部和之间统一结果审查标准的必要性。随着时间的推移，讨论从HRQL结果扩展到包括基于患者或患者代理提供的数据的任何结果，即患者报告的结果(PRO)。方法：与美国食品和药物管理局(FDA)的一名成员合作，确定了四个需要澄清的关键问题：PRO如何定义并用于研究目的；PRO在药物审查和评价过程中的附加值；与PRO测量和研究方法相关的选定问题；以及决策者对PRO信息的兴趣和需求。2001年2月15日，支持协调小组的所有成员参加了在马里兰州罗克维尔举行的会议，进一步讨论了这四个问题，并于2001年2月16日向FDA各部门和审查部门的代表作了正式陈述。结果：所有参与者都同意，专业知识对于理解治疗对患者功能和健康的影响很重要。他们还强调，需要向关键决策者传达PRO信息，包括监管机构、临床医生、患者及其家人和支付者。总结：2001年2月16日的会议是协调各组织努力的重要一步，也是与FDA就药物评价过程中测量和解释PRO的方法学标准相关重大问题展开对话的重要一步。
Background: The extent to which patient-based outcomes can be used to evaluate and communicate the effect of new drugs and devices is a subject of much debate. Criteria for evaluating the scientific quality of data to support health-related quality of life (HRQL) and other patient-based labeling and promotional claims in the United States and Europe have been proposed by various scientists and organizations. Since March 2000, a working group composed of members of the International Society for Quality of Life Research (ISOQOL), the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the Pharmaceutical Manufacturer's Association Health Outcomes Committee (PhRMAHOC), and the European Regulatory Issues on Quality of Life Assessment (ERIQA) met to discuss and coordinate the various recommendations by their respective groups and address the need to harmonize outcomes review criteria within and across United States and European regulatory agencies. Over time, the discussion expanded from HRQL outcomes to include any outcome based on data provided by the patient or patient proxy, that is, patient-reported outcomes (PROs). The working group therefore became known as the PRO Harmonization Group.Methods: Working with a member of the US Food and Drug Administration (FDA), four key issues requiring clarification were identified: how PROs are defined and put into operation for research purposes; the added value of PROs in the drug review and evaluation process; selected questions related to the PRO measurement and research methodology; and the interest and demand for PRO information by decision makers. On February 15, 2001, all members of the PRO Harmonization Group attended a meeting in Rockville, Maryland, to discuss these four issues further, and on February 16, 2001, a formal presentation was made to representatives from various departments and reviewing divisions of the FDA. These presentations are summarized in this report.Results: All participants agreed that PROs are important for understanding the impact of treatment on patient functioning and well-being. They also stressed the need to communicate PRO information to key decision makers, including regulatory agencies, clinicians, patients and their families, and payers. Finally, the meeting resulted in plans for continuing the dialogue on PRO measurement and interpretation.Summary: The February 16, 2001, meeting represented an important step in harmonizing efforts across various organizations and in opening a dialogue with the FDA around major issues related to methodologic standards for measuring and interpreting PROs in the drug evaluation process.