Lessons Learned From Clinical Studies in Centronuclear Myopathies: The Patient Perspective—A Qualitative Study

IF 3.2 4区 医学 Q2 PHARMACOLOGY & PHARMACY
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Abstract

Background

Since 2014, several clinical studies focusing on centronuclear myopathies have been conducted, including a prospective natural history study, a gene transfer clinical trial and a clinical trial using an antisense oligonucleotide. Dedicated patient organizations have played an important role in this process. The experience of members of these organizations, either as a study participant, parent or as a patient organization member communicating with the sponsors are potentially very informative for future trial design.

Methods

We investigated the burden of and the lessons learned from the first natural history studies and clinical trials from a patient perspective using a qualitative approach. We arranged 4 focus groups with a total of 37 participants from 3 large international patient organizations: ZNM-ZusammenStark!, the Myotubular Trust, and the MTM-CNM Family Connection. 4 themes, based on a systematic literature search were discussed: Expectations and preparation, Clinical study participation, Communication and Recommendations for future clinical trials. The focus group recordings were transcribed, anonymized, and uploaded to Atlas-ti version 8.1 software. The data were analyzed using a thematic content analysis.

Results

Overall, participants were realistic in their expectations, hoping for small improvements of function and quality of life. The realization that trial participation does not equate to a treatment was challenging. Participating in a clinical study had a huge impact on many aspects of daily life, both for patients and their immediate families. First-hand insights into the burden of the design and its possible effect on performance were provided, resulting in numerous compelling recommendations for future clinical studies. Furthermore, participants stressed the importance of clear communication, which was considered to be especially vital in cases of severe adverse events. Finally, while patients were understanding of the importance of adhering to the regulations of good clinical practice, they indicated that they would strongly appreciate a greater understanding and/or acknowledgment of the patient perspective and a reflection of this perspective in future clinical trial design.

Conclusion

The acknowledgment and inclusion of patients’ perspectives and efficient and effective communication is expected to improve patient recruitment and retention in future clinical studies, as well as more accurate assessment of the patient performance related to suitable planning of the study visits.
从中心核肌病临床研究中汲取的经验教训:患者视角--定性研究
背景自 2014 年以来,已开展了多项以中心核肌病为重点的临床研究,包括一项前瞻性自然史研究、一项基因转移临床试验和一项使用反义寡核苷酸的临床试验。在这一过程中,专门的患者组织发挥了重要作用。这些组织的成员,无论是作为研究参与者、家长还是作为患者组织成员与申办者沟通的经验,都可能为未来的试验设计提供非常有价值的信息。方法我们采用定性方法,从患者的角度调查了首批自然史研究和临床试验的负担和经验教训。我们安排了 4 个焦点小组,共有来自 3 个大型国际患者组织的 37 人参加:ZNM-ZusammenStark!"、"肌管信托 "和 "MTM-CNM 家庭联系"。根据系统的文献检索,讨论了 4 个主题:期望与准备、临床研究参与、沟通以及对未来临床试验的建议。焦点小组的录音经过转录、匿名处理后上传到 Atlas-ti 8.1 版软件。结果总体而言,参与者的期望是现实的,他们希望自己的功能和生活质量能有小幅改善。参与试验并不等于接受治疗,这一认识具有挑战性。参与临床研究对患者及其直系亲属日常生活的许多方面都有巨大影响。与会者提供了关于设计负担及其对表现可能产生的影响的第一手见解,从而为未来的临床研究提出了许多令人信服的建议。此外,与会者还强调了清晰沟通的重要性,认为这在发生严重不良事件时尤为重要。最后,虽然患者理解遵守良好临床实践规范的重要性,但他们表示,如果能在未来的临床试验设计中更多地理解和/或承认患者的观点并反映出这一观点,他们将非常感激。结论承认并纳入患者的观点以及高效和有效的沟通有望改善未来临床研究中患者的招募和保留情况,并能更准确地评估与研究访问的适当规划有关的患者表现。
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来源期刊
Clinical therapeutics
Clinical therapeutics 医学-药学
CiteScore
6.00
自引率
3.10%
发文量
154
审稿时长
9 weeks
期刊介绍: Clinical Therapeutics provides peer-reviewed, rapid publication of recent developments in drug and other therapies as well as in diagnostics, pharmacoeconomics, health policy, treatment outcomes, and innovations in drug and biologics research. In addition Clinical Therapeutics features updates on specific topics collated by expert Topic Editors. Clinical Therapeutics is read by a large international audience of scientists and clinicians in a variety of research, academic, and clinical practice settings. Articles are indexed by all major biomedical abstracting databases.
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