National Heart and Lung Institute, Imperial College London Department of Medicine, London, UK
Department of Respiratory Medicine, Royal Brompton Hospital, London, UK.
BMJ Open Respir Res. 2024 Sep 3;11(1):e002368. doi: 10.1136/bmjresp-2024-002368.
Health research bodies recommend patient involvement and engagement in research and healthcare planning, although their implementation is not yet widespread. This deficiency extends to progressive pulmonary fibrosis (PPF), where crucial aspects remain unknown, including causal mechanisms, curative treatments and optimal symptom management. This study addresses these gaps by seeking stakeholders' perspectives to guide research and treatment directions.
A priority-setting partnership was established to explore stakeholders' priorities in the diagnosis, treatment, management and care of PPF, including idiopathic pulmonary fibrosis which is the archetypal PPF. Stakeholders included people living with PPF, their carers, relatives and healthcare professionals involved in their management.
Through an online open-ended survey, 2542 responses were collected from 638 stakeholders. Thematic analysis identified 48 specific research questions, which were then cross-referenced with academic literature to pinpoint research gaps. Following the evidence check, 44 unanswered questions were shortlisted by 834 stakeholders in a second online survey. Ultimately, a top 10 priority list was established through consensus.The prioritised research questions include (1) improved diagnosis accuracy and timing, (2) development of new treatments, (3) enhanced accuracy in primary care, (4) optimal timing for drug and non-drug interventions, (5) effective cough treatment, (6) early intervention for PPF, (7) improved survival rates, (8) symptom reduction, (9) impact of interventions on life expectancy and (10) new treatments with reduced side effects.
Stakeholders' priorities can be summarised into five areas: early diagnosis, drug and non-drug treatments, survival and symptom management. Ideally, these topics should guide funding bodies and health policies.
健康研究机构建议患者参与研究和医疗保健规划，尽管这些建议尚未得到广泛实施。这种缺陷也存在于进行性肺纤维化（PPF）中，在这种疾病中，许多关键方面仍不为人知，包括病因机制、治疗方法和最佳症状管理。本研究通过寻求利益相关者的观点来解决这些问题，以指导研究和治疗方向。
建立了一个优先事项设定伙伴关系，以探讨利益相关者在 PPF 的诊断、治疗、管理和护理方面的优先事项，包括特发性肺纤维化，这是 PPF 的典型代表。利益相关者包括患有 PPF 的人、他们的照顾者、亲属以及参与他们管理的医疗保健专业人员。
通过在线开放式调查，从 638 位利益相关者中收集了 2542 份回复。主题分析确定了 48 个具体的研究问题，然后与学术文献交叉引用，以确定研究差距。经过证据检查，834 位利益相关者在第二次在线调查中对 44 个未回答的问题进行了简短的提名。最终，通过共识确定了前 10 个优先事项清单。优先考虑的研究问题包括：（1）提高诊断准确性和及时性；（2）开发新的治疗方法；（3）提高初级保健中的准确性；（4）药物和非药物干预的最佳时机；（5）有效的咳嗽治疗；（6）对 PPF 的早期干预；（7）提高生存率；（8）减少症状；（9）干预对预期寿命的影响；（10）减少副作用的新治疗方法。
利益相关者的重点可以概括为五个方面：早期诊断、药物和非药物治疗、生存和症状管理。理想情况下，这些主题应该指导资金机构和卫生政策。
