Bovaira-García M J, Soler-Company E
Servicio de Farmacia, Consorcio Hospitalario Provincial de Castellón, Castellón, España.
Rev Calid Asist. 2012 Mar-Apr;27(2):92-102. doi: 10.1016/j.cali.2011.09.007. Epub 2011 Dec 1.
Patient-reported outcome (PRO) measures complement traditional biomedical outcome measures. The purpose of this study was to evaluate the use of PRO measures including health-related quality of life (HRQoL) questionnaires as a measurement of efficacy and the frequency of inclusion of economic variables related to direct and indirect costs in the design of clinical trials and phase IV observational studies. Moreover, for the trials quality score were measured, and if there were any relationship between the quality study design score and the PRO inclusion.
Retrospective observational study of the clinical trials and phase IV observational studies approved by a Clinical Research Ethics Committee (2008-2010). We gathered data concerning general aspects including medical specialty, pathology, methodological quality based on Jadad scale (0-5), inclusion of PRO and economic variables. For clinical trials including HRQoL measurements, we analysed the type of questionnaire in use. Where there were no HRQoL measurements, we analysed if their inclusion would have been proper or not.
A total of 70 protocols (59 CTs and 11 phase IV observational studies) were analysed; 37 (52.8%) included PRO measures, and 3 protocols (4.3%) used them as a primary endpoint. Data analysis by therapeutic area showed that PRO measures were most commonly studied in the fields of endocrinology, neurology, digestive diseases, and cardiology. The average quality score for the trials was 2.8. The trials with more PRO inclusion in their end points had a significantly higher quality score. Only 13 (22%) clinical trials and 2 (18.2%) phase IV observational studies included economic variables.
The emergence of economic variables in clinical trials and phase IV observational studies evaluated was low, however, more than half of the revised protocols have included PRO measures, reflecting the importance of these parameters in the assessment of the effectiveness of drug treatments, although its use is still not systematic.
患者报告结局（PRO）指标是对传统生物医学结局指标的补充。本研究旨在评估PRO指标（包括健康相关生活质量[HRQoL]问卷）在疗效测量中的应用，以及在临床试验和IV期观察性研究设计中纳入与直接和间接成本相关的经济变量的频率。此外，对试验质量评分进行了测量，并探究质量研究设计评分与PRO纳入之间是否存在任何关联。
对经临床研究伦理委员会批准的临床试验和IV期观察性研究（2008 - 2010年）进行回顾性观察研究。我们收集了有关一般方面的数据，包括医学专科、病理学、基于雅达量表（0 - 5）的方法学质量、PRO和经济变量的纳入情况。对于包含HRQoL测量的临床试验，我们分析了所使用问卷的类型。在没有HRQoL测量的情况下，我们分析其纳入是否合适。
共分析了70项方案（59项临床试验和11项IV期观察性研究）；37项（52.8%）纳入了PRO指标，3项方案（4.3%）将其用作主要终点。按治疗领域进行的数据分析表明，PRO指标在内分泌学、神经病学、消化系统疾病和心脏病学领域研究最为普遍。试验的平均质量评分为2.8。终点中纳入更多PRO的试验质量评分显著更高。只有13项（22%）临床试验和2项（18.2%）IV期观察性研究纳入了经济变量。
在评估的临床试验和IV期观察性研究中，经济变量的出现频率较低，然而，超过一半的修订方案纳入了PRO指标，这反映了这些参数在药物治疗效果评估中的重要性，尽管其使用仍不系统。
