Department of Clinical Epidemiology and Medical Technology Assessment, Maastricht University Medical Centre, Maastricht, The Netherlands.
Department of Respiratory Medicine, Erasmus Medical Centre, University Hospital Rotterdam, Rotterdam, The Netherlands.
Patient. 2017 Oct;10(5):629-642. doi: 10.1007/s40271-017-0234-z.
The aim of this study was to develop, together with the Lung Foundation Netherlands and Dutch Kidney Patients Association, patients and clinicians, a measure to evaluate patient experiences with the orphan drugs pirfenidone (for idiopathic pulmonary fibrosis [IPF]) and eculizumab (for atypical haemolytic uraemic syndrome [aHUS]), as well as a generic measure of patient experiences and satisfaction with medications.
Development of the Patient Experiences and Satisfaction with Medications (PESaM) questionnaire consisted of four phases: literature review (phase I); focus groups and individual patient interviews (phase II); item generation (phase III); and face and content validity testing (phase IV). Literature review aimed to identify existing disease-specific and generic patient experience measures to provide guidance on the domains of medication use relevant to patients, the number of items and type of response categories, and to generate an initial pool of items. Subsequent focus groups and patient interviews were conducted to gain insight into the perceived effectiveness of the therapies, the burden of side effects, and how the medication impacted on a patient's daily life. Focus groups and interviews were recorded and transcribed verbatim. Coding was carried out by highlighting passages in the text and assigning each passage a code representing the following predefined categories: (1) perceived effectiveness; (2) side effects; (3) ease of use; and (4) impact of medication. Using data from phase I and II, a panel of experts selected items relevant for inclusion in the questionnaire. Individual patient interviews with IPF and aHUS patients (n = 18), using a retrospective verbal probing technique, were conducted to assess face validity, time needed to fill out the questionnaire, and content validity.
The PESaM questionnaire that was developed consisted of two disease-specific modules that assessed patient experiences with pirfenidone for the treatment of IPF, and eculizumab for the treatment of aHUS, a generic module, applicable to any medication, and a module to assess patient expectations. Review of the literature identified multiple disease- or medication-specific questionnaires and two generic patient satisfaction questionnaires. Common domains across most questionnaires were effectiveness, side effects, ease of use and overall satisfaction. Patient interviews revealed the social impact (e.g. unable to go outside) of side effects such as photosensitivity associated with pirfenidone and the risk of infection associated with eculizumab. Each PESaM module focuses on patients' perceived effectiveness of the medication, side effects, and ease of use, and the impact these aspects have on physical and emotional health and daily life. The generic module additionally includes items related to satisfaction with the medication. Individual interviews with patients in phase IV confirmed, in general, that questions and response options of the modules were clear and content validity was good. The mean time to complete the modules ranged from 6 min for the disease-specific (aHUS) module to 9 min for the generic module.
We developed the PESaM questionnaire to quantitatively assess patient experiences and satisfaction with medications. A validation study is currently underway to examine the psychometric properties of the PESaM questionnaire.
本研究旨在与荷兰肺脏基金会和荷兰肾脏患者协会以及患者和临床医生共同开发一种评估药物使用体验的工具，用于评估孤儿药吡非尼酮（用于特发性肺纤维化[IPF]）和依库珠单抗（用于非典型溶血尿毒综合征[aHUS]）的患者体验，以及评估患者对药物的总体体验和满意度的通用工具。
患者对药物的体验和满意度（PESaM）问卷的开发分为四个阶段：文献回顾（第一阶段）；焦点小组和个别患者访谈（第二阶段）；项目生成（第三阶段）；以及表面和内容有效性测试（第四阶段）。文献回顾旨在确定现有的疾病特异性和通用患者体验评估工具，以提供与患者相关的药物使用相关领域、项目数量和响应类别类型的指导，并生成初始项目池。随后的焦点小组和患者访谈旨在深入了解治疗的有效性、副作用的负担，以及药物对患者日常生活的影响。焦点小组和访谈进行了记录并逐字转录。通过突出文本中的段落并为每个段落分配代表以下预定义类别的代码来进行编码：（1）感知效果；（2）副作用；（3）易用性；和（4）药物影响。使用第一和第二阶段的数据，一个专家小组选择了与问卷相关的项目。使用回顾性口头探查技术，对 18 名 IPF 和 aHUS 患者进行了个别患者访谈，以评估表面有效性、填写问卷所需的时间以及内容有效性。
开发的 PESaM 问卷由两个疾病特异性模块组成，用于评估治疗 IPF 的吡非尼酮和治疗 aHUS 的依库珠单抗的患者体验、适用于任何药物的通用模块，以及评估患者期望的模块。文献回顾确定了多个疾病特异性或药物特异性问卷和两个通用患者满意度问卷。大多数问卷的共同领域包括有效性、副作用、易用性和总体满意度。患者访谈揭示了与吡非尼酮相关的副作用（例如光敏感性）和与依库珠单抗相关的感染风险等副作用的社会影响。每个 PESaM 模块都侧重于患者对药物的有效性、副作用和易用性的感知，以及这些方面对身体和情感健康以及日常生活的影响。通用模块还包括与药物满意度相关的项目。第四阶段的个别患者访谈普遍证实，模块的问题和回答选项清晰，内容有效性良好。完成模块的平均时间从疾病特异性（aHUS）模块的 6 分钟到通用模块的 9 分钟不等。
我们开发了 PESaM 问卷来定量评估患者对药物的体验和满意度。目前正在进行一项验证研究，以检查 PESaM 问卷的心理测量特性。
