Inhestern Laura, Zybarth David, Otto Ramona, Brandt Maja, Härter Martin, Bergelt Corinna
Department of Medical Psychology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
Department of Medical Psychology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
BMJ Open. 2020 Nov 30;10(11):e040470. doi: 10.1136/bmjopen-2020-040470.
Patients and families affected by a rare disease are burdened in multiple ways. Functional interface management can unburden patients or relatives from the need to be solely accountable for the navigation through the healthcare system. This study aims at (1) providing an assessment of approaches and interface management concepts in the care of rare diseases, (2) evaluating selected existing approaches and concepts and (3) developing best practice recommendations for interface management.
We will conduct a mixed-methods study with three phases. In phase 1, we will develop a tool to assess existing concepts of interface management for rare diseases based on a literature search and an expert workshop. The tool will be applied in a telephone survey with representatives of centres or clinics of expertise for rare diseases (target: n=100) and cooperating practitioners (target: n=60). Based on the results of phase 1, we will select four to six centres of expertise with interface management concepts, which will be evaluated extensively in phase 2. For the evaluation, we will conduct semistructured interviews with practitioners cooperating with centres or clinics for rare diseases (target: n=50), a paper-based survey including patients or parents/legal guardians (target: n=300) from the selected centres or clinics, and semistructured interviews with patients or parents/legal guardians (target: n=50). The final phase of the study will be an integration of results from phases 1 and 2 to develop best practice recommendations for interface management in healthcare of rare diseases. In a concluding expert workshop, recommendations will be presented and finalised.
This study was approved by the Local Psychological Ethics Committee of the Center for Psychosocial Medicine of the University Medical Center Hamburg-Eppendorf (LPEK-0062). The findings of our study will be presented on national and international conferences and published in scientific, peer-reviewed journals. To assure that centres for rare diseases get access to the study results, centres are invited to send a representative to a final expert workshop in phase 3. Moreover, an executive summary will be provided and sent to relevant stakeholders.
German Clinical Trials Registry (DRKS00020488).
受罕见病影响的患者及其家庭承受着多方面的负担。功能界面管理可以减轻患者或亲属独自应对医疗系统的负担。本研究旨在：（1）评估罕见病护理中的方法和界面管理概念；（2）评估选定的现有方法和概念；（3）制定界面管理的最佳实践建议。
我们将开展一项分为三个阶段的混合方法研究。在第一阶段，我们将基于文献检索和专家研讨会，开发一种工具来评估罕见病界面管理的现有概念。该工具将应用于对罕见病专业中心或诊所代表（目标：n = 100）以及合作从业者（目标：n = 60）的电话调查。基于第一阶段的结果，我们将选择四至六个具有界面管理概念的专业中心，在第二阶段对其进行全面评估。为进行评估，我们将与与罕见病中心或诊所合作的从业者（目标：n = 50）进行半结构化访谈，对选定中心或诊所的患者或家长/法定监护人（目标：n = 300）进行纸质调查，并与患者或家长/法定监护人（目标：n = 50）进行半结构化访谈。研究的最后阶段将整合第一阶段和第二阶段的结果，以制定罕见病医疗保健中界面管理的最佳实践建议。在最后的专家研讨会上，将展示并确定建议。
本研究已获得汉堡 - 埃彭多夫大学医学中心社会心理医学中心当地心理伦理委员会的批准（LPEK - 0062）。我们研究的结果将在国内和国际会议上展示，并发表在经过同行评审的科学期刊上。为确保罕见病中心能够获取研究结果，将邀请各中心派代表参加第三阶段的最终专家研讨会。此外，还将提供一份执行摘要并发送给相关利益相关者。
德国临床试验注册中心（DRKS00020488）
