Knoppers Terese, Cosquer Marie, Hagan Julie, Nguyen Minh Thu, Knoppers Bartha Maria
Centre of Genomics and Policy, McGill University, Montreal, QC, Canada.
Department of Sociology, Laval University, Quebec, QC, Canada.
Front Med (Lausanne). 2022 Mar 23;9:841887. doi: 10.3389/fmed.2022.841887. eCollection 2022.
Making bench to bedside advances in cystic fibrosis (CF) care requires the sustained engagement and trust of people living with CF. However, there is a scarcity of studies exploring their concerns and priorities regarding research and its end products. The aim of this qualitative study was to generate empirical evidence regarding patient and caregiver perspectives on cystic fibrosis research and personalized medicine to foster developments in translational research in Canada.
A total of 15 focus groups were conducted, engaging 22 adults with CF and 18 caregivers (e.g., parents, siblings and partners) living in Canada. Inductive thematic analysis relied on an iterative process involving themes derived from both participant meaning-making and existing scientific literature. Participant perspectives were considered along intrapersonal, intracommunity, interpersonal, and structural lines.
Overall, participants described a relationship to CF research inextricable from the lived experience of CF as a lifelong progressive and terminal disease and from the goal of advancing medical science. They were enthusiastic and excited about the emergence of CFTR modulators, although they had some knowledge gaps regarding the associated research. They largely spoke to positive experiences with researcher communication but had feedback regarding informed consent processes and the return of study results. Participants also voiced concerns about structural access barriers to research and to its end products. Extensive histories of research participation, a relatively small and intercommunicative CF community, and structural overlap between research and care settings contributed to their perspectives and priorities.
Study findings are valuable for researchers and policy-makers in CF and rare or progressive diseases more broadly. Continuing to solicit and listen to the voices of patients and caregivers is crucial for research ethics and the translation of new therapies in the area of personalized medicine.
要在囊性纤维化（CF）护理方面实现从实验室到临床的进展，需要CF患者的持续参与和信任。然而，探索他们对研究及其最终成果的担忧和优先事项的研究却很匮乏。这项定性研究的目的是生成关于患者和护理人员对囊性纤维化研究和个性化医疗的观点的实证证据，以促进加拿大转化研究的发展。
共进行了15个焦点小组讨论，参与人员为22名成年CF患者和18名居住在加拿大的护理人员（如父母、兄弟姐妹和伴侣）。归纳主题分析依赖于一个迭代过程，该过程涉及从参与者的意义构建和现有科学文献中得出的主题。从个人内部、社区内部、人际和结构层面考虑参与者的观点。
总体而言，参与者将与CF研究的关系描述为与CF作为一种终身渐进性和终末期疾病的生活经历以及推进医学科学的目标密不可分。他们对CFTR调节剂的出现充满热情和兴奋，尽管他们在相关研究方面存在一些知识空白。他们大多谈到了与研究人员沟通的积极经历，但对知情同意过程和研究结果的反馈提出了意见。参与者还表达了对研究及其最终产品的结构准入障碍的担忧。广泛的研究参与历史、相对较小且相互交流的CF患者群体以及研究与护理环境之间的结构重叠促成了他们的观点和优先事项。
研究结果对CF以及更广泛的罕见或进行性疾病的研究人员和政策制定者具有重要价值。持续征求和倾听患者及护理人员的声音对于研究伦理和个性化医疗领域新疗法的转化至关重要。
