Cystic Fibrosis Target Product Profiles: Focus group data (clinical and people with CF)

Diagnostics are key to the appropriate detection, treatment and management of many conditions and associated health implications. People with cystic fibrosis are susceptible to recurrent pulmonary infections that over time contribute to deterioration and degeneration of the lung. Launched in 2019, the Cystic Fibrosis (CF) Antimicrobial Resistance (AMR) Syndicate was formed to accelerate the translation and adoption of new CF antimicrobials and diagnostics to the clinic through collaboration (https://cfamr.org.uk/), bringing better treatment options to people with CF, faster. To catalyse the development of new diagnostics, the CF AMR Syndicate, together with the Newcastle NIHR HealthTech Research Centre (formerly Newcastle NIHR MIC), has worked with the wider community to understand the unmet diagnostic needs and develop a suite of Target Product Profiles (TPPs) for CF lung infection diagnostics. The intention of this is to drive diagnostic discovery, focusing efforts where they are needed the most. Although developed in the UK with input from UK based experts and people with CF, we intend for the impact of these TPPs to be global. This project involved three main phases, sandwiched between planning and management activities, and a dissemination strategy programme, all guided by an expert advisory group. Phase 1 involved a landscape analysis to identify and prioritise unmet diagnostic needs via multiple focus groups of clinical experts and people with Cystic Fibrosis (pwCF), as well as a scoping review of the diagnostic space and available diagnostic tests (https://doi.org/10.1016/j.resinv.2024.07.005). Phase 2 involved drafting the TPPs using existing literature and regulatory documentation, focus groups, one-to-one interviews with key opinion leaders (KoLs), and a web-based survey, to define ‘minimal’ and ‘optimal’ characteristics for each TPP. Phase 3 refined and validated the TPP content through additional interviews, a two-round modified Delphi exercise, and a virtual symposium. These data are from phase 2 of this study and are the outputs of expert and patient focus groups discussing the CF care pathway, positive and negative aspects of clinical care, unmet needs in CF, and the perspectives of pwCF and clinical teams on optimal and desired characteristics for diagnostic tests in CF. This patient-centric methodology builds upon the experience and network of MDC and CF Trust in the development of TPPs to guide CF antimicrobial therapeutic development. These early stakeholder focus groups went on to inform all subsequent diagnostic TPP development work, and laid the foundation for subsequent stakeholder elicitation regarding content and focus. Focus group topic guides are available at: https://doi.org/10.25405/data.ncl.26477263. The final TPP documents (and a report from the virtual symposium) are available at: https://cfamr.org.uk/therapeutic-tpp/.诊断对于多种疾病及其相关健康影响的精准检测、治疗与管理至关重要。囊性纤维化（cystic fibrosis, CF）患者易罹患反复发作的肺部感染，随时间推移会导致肺组织损伤与退变。2019年启动的囊性纤维化抗菌药物耐药性（antimicrobial resistance, AMR）联盟旨在通过协作加速新型CF抗菌药物与诊断技术向临床的转化与落地（https://cfamr.org.uk/），以期更快为CF患者提供更优治疗方案。为推动新型诊断技术的研发，CF AMR联盟与纽卡斯尔英国国家健康与护理研究所（National Institute for Health and Care Research, NIHR）健康技术研究中心（前身为纽卡斯尔NIHR MIC）携手多方社群，明确未被满足的诊断需求，并针对CF肺部感染诊断开发了一系列目标产品特征（Target Product Profiles, TPPs）。此举旨在推动诊断技术研发，将资源集中于最亟需的领域。尽管该系列TPPs由英国专家与CF患者参与制定，但我们期望其影响能够辐射全球。本项目由专家咨询委员会全程指导，包含规划与管理环节、传播策略项目，以及夹在二者之间的三大核心阶段。第一阶段为现状分析：通过多轮临床专家与囊性纤维化患者（people with CF, pwCF）焦点小组讨论，以及对诊断领域与现有诊断技术的范围综述，明确未被满足的诊断需求并排序优先级（https://doi.org/10.1016/j.resinv.2024.07.005）。第二阶段为TPPs起草：依托现有文献与监管文件、焦点小组讨论、关键意见领袖（key opinion leaders, KoLs）一对一访谈，以及网络问卷调查，明确每份TPP的“最低要求”与“最优标准”特征。第三阶段为TPP内容优化与验证：通过补充访谈、两轮改良德尔菲法研讨，以及虚拟研讨会完成。本数据集来自本研究的第二阶段，包含专家与患者焦点小组的讨论成果，议题涵盖CF诊疗路径、临床诊疗的优劣之处、CF领域未被满足的需求，以及pwCF与临床团队对于CF诊断技术的最优期望特征与预期特征的观点。这种以患者为中心的研究方法，依托MDC与囊性纤维化基金会（CF Trust）在TPPs开发领域的经验与资源网络，用于指导CF抗菌治疗药物的研发。这些早期利益相关方焦点小组的成果为后续所有诊断类TPP的开发工作提供了依据，并为后续针对TPP内容与聚焦方向的利益相关方调研奠定了基础。焦点小组讨论指南可通过以下链接获取：https://doi.org/10.25405/data.ncl.26477263。最终版TPP文件（及虚拟研讨会报告）可通过以下链接获取：https://cfamr.org.uk/therapeutic-tpp/。