PROMISing development for standardising the assessment of self-reported health worldwide? An exploration of the potential application of U.S. developed patient-reported outcome measures to Germany

患者报告的结果（PROs）在评估医疗干预的利弊方面越来越重要。由于观察员（代理）不能准确地捕捉患者的观点，世界各地的监管机构建议在临床试验中应用PROs。然而，为了将这些数据用于跨研究和关键亚组的比较目的，必须制定评估自我报告健康的标准。该领域最近的出版物推荐人行横道表或在同一种通用度量标准上对不同仪器进行校准，这是对PROs进行标准化评估的开创性步骤。标准化PRO评估的主要先驱是2004年由美国国立卫生研究院发起的患者报告结果测量信息系统（PROMIS®）倡议。PROMIS基于这样的思想，即测量相同构造的项目可以合并到一个题库中，并根据相同的度量标准进行校准。自推出以来，PROMIS研究人员已经开发并验证了50多个题库。2012年，PROMIS开始向其他国家扩展，这是一个在全球范围内标准化pro的独特机会。然而，在全球范围内推出试题库之前，还需要探索一系列挑战。这些挑战涉及(1)文化充分性，(2)单维性和普遍性，(3)美国项目参数在其他语言版本中的可转移性，以及(4)基于美国普通人群的参考数据在规范目的中的适用性。因此，本提案旨在以PROMIS物理功能（PF）题库为例，对PROMIS项目进行全面测试。具体来说，为了回答研究问题(1)，我们对PF项目进行了定性测试。为了探究(2)和(3)，我们对来自风湿病学、心脏病学、心身医学三个临床样本的600名患者进行了PROMIS项目测试。首先，验证性因子分析探讨了构念的单一性。第二，差异项目功能（DIF）测试探索普遍性（疾病组DIF），第三，从德国数据中得出的新项目参数估计探索了美国项目参数到翻译版本（语言-DIF）的可转移性。最后，为了回答(4)，我们从德国普通人群中获得参考数据（n= 1000）。本项目的总体目标是为回答一些剩余的心理测量学挑战作出根本贡献，这些挑战涉及旨在测量自我报告健康的项目的翻译和文化适应。本研究不仅将为德国PRO评估的标准化做出重要贡献，而且将为主观幸福感评估的国际知识体系做出贡献。

Patient-reported outcomes (PROs) are of growing importance in the assessment of benefits and harms of medical interventions. As observers (proxy) do not accurately capture the patient perspective, regulatory authorities worldwide recommend the application of PROs in clinical trials. However, for these data to be used for comparative purposes across studies and crucial subgroups, it is imperative to develop standards for the assessment of self-reported health. Recent publications in this field recommend crosswalk tables or a calibration of different instruments on the same, a common, metric, which is a ground-breaking step towards standardised assessment of PROs. A major pioneer in the context of standardised PRO assessment is the Patient-Reported Outcomes Measurement Information System (PROMIS®) initiative, launched in 2004 by the U.S. National Institutes of Health. PROMIS is based on the idea that items measuring the same construct can be merged in one item bank and calibrated on the same metric. Since its launch, PROMIS researchers have developed and largely validated over 50 item banks. In 2012, PROMIS started expanding its efforts to other countries, which is a unique opportunity to standardise PROs worldwide. However, it comes with a range of challenges that need to be explored before item banks can be launched globally. These challenges relate to (1) cultural adequacy, (2) unidimensionality and generalisability, (3) transferability of U.S. item parameters to other language versions, and (4) applicability of reference data based on the U.S. general population for norming purposes. As a result, this proposal aims at a thorough testing of PROMIS items, using the PROMIS Physical Function (PF) Item Bank as an example. Specifically, to answer research question (1), the PF items are tested qualitatively. To explore (2) and (3), PROMIS items are tested in n=600 patients from three clinical samples (rheumatology, cardiology, psychosomatic medicine). First, confirmatory factor analyses explore unidimensionality of the construct. Second, tests of differential item functioning (DIF) explore generalisability (disease group-DIF), and third, new item parameter estimates derived from the German data explore transferability of the U.S. item parameters to the translated version (language-DIF). Finally, to answer (4), reference data from the German general population are obtained (n=1,000). The overarching aim of the present project is to make a fundamental contribution to answering some of the remaining psychometric challenges involved with the translation and cultural adaptation of items aimed at measuring self-reported health. This study shall make an important contribution to the standardisation of PRO assessment not only in Germany, but it shall contribute to the international body of knowledge of the assessment of subjective well-being.