Asbell Penny, Messmer Elisabeth, Chan Colin, Johnson Gary, Sloesen Brigitte, Cook Nigel
Ophthalmology, Icahn School of Medicine at Mount Sinai, New York City, New York, USA.
Department of Ophthalmology, Ludwig Maximilians University, Munich, Germany.
BMJ Open Ophthalmol. 2019 Dec 5;4(1):e000315. doi: 10.1136/bmjophth-2019-000315. eCollection 2019.
Dry eye disease is a multifactorial chronic disease, leading to ocular discomfort and visual disturbance with a substantial impact on quality of life. Therefore, the patient's perspective should be taken into account early in the drug development process. We have developed a step-by-step methodology based on the self-explicated conjoint approach to assess the needs and preferences of patients with moderate-to-severe dry eye disease.
Following a literature review and social media listening (step 0), qualitative phone call interviews were conducted with 12 patients (step 1). Patients' responses underwent content analysis and were coded, quantified and displayed as charts. Based on the emerging trends and attributes identified as relevant in steps 0 and 1, a quantitative online questionnaire was designed and conducted with 160 patients across four countries (step 2).
The online questionnaire was rated as easy/very easy to understand by 60% of respondents, 62% rated the survey as easy/very easy to complete and 71% rated it as interesting/very interesting. Treatment satisfaction was the most important aspect for patients, and the three most relevant attributes were as follows (with the most important indexed to 100%): 'treatment effectiveness on symptoms of dry eyes' (100%), 'frequency of treatment use' (96%) and 'how the treatment works' (95%).
Our methodology was well received by patients, and the results will help inform future clinical trial development and discussions with health technology assessment bodies and regulators on unmet needs and product attributes that are of most value to patients with dry eye disease.
干眼症是一种多因素慢性疾病,会导致眼部不适和视觉障碍,对生活质量有重大影响。因此,在药物研发过程中应尽早考虑患者的观点。我们基于自我解释联合分析法开发了一种逐步评估方法,以评估中重度干眼症患者的需求和偏好。
在进行文献综述和社交媒体舆情监测(步骤0)之后,对12名患者进行了定性电话访谈(步骤1)。对患者的回答进行内容分析,并进行编码、量化并以图表形式展示。根据在步骤0和1中确定的相关新兴趋势和属性,设计了一份定量在线问卷,并在四个国家对160名患者进行了调查(步骤2)。
60%的受访者认为在线问卷易于/非常易于理解,62%的受访者认为该调查易于/非常易于完成,71%的受访者认为该调查有趣/非常有趣。治疗满意度对患者来说是最重要的方面,三个最相关的属性如下(最重要的属性指标为100%):“对干眼症状的治疗效果”(100%)、“治疗使用频率”(96%)和“治疗作用方式”(95%)。
我们的方法受到了患者的好评,研究结果将有助于为未来的临床试验开发提供参考,并有助于与卫生技术评估机构和监管机构就未满足的需求以及对干眼症患者最有价值的产品属性进行讨论。