传统的主要基于主要结果指标来评估治疗方法的模型在将其应用于罕见疾病方面步履蹒跚。罕见病临床试验面临的方法学挑战,异质的患者群体和相对较少的验证，疾病特异性结果测量。将定性研究纳入罕见疾病临床试验可能有助于赞助商，监管者,付款人,和处方者更好地了解潜在治疗的现实世界和患者特定的影响。本文提供了使用患者和护理人员感知变化（PPC和CPC）评估的方法学概述，该评估利用患者和护理人员视频访谈来补充罕见疾病临床试验中传统终点捕获的数据。
将定性的患者和护理人员视频访谈纳入临床试验，可以严格捕捉患者体验和护理人员观察。由主要利益相关者的输入提供的访谈指南提供了机会，以征求有关之前经验的结构化反馈，during,在临床试验之后.患者和护理人员可以在研究移动应用程序中完成他们的视频访谈，面试记录由独立编码员分析。主题由治疗组和个体患者总结，这为患者的感受和功能的临床结果指标增加了背景，以及阐明对患者和护理人员有意义的变化程度。定性结果可以与临床试验中捕获的数据进行比较，以评估数据一致性。
以足够的严谨性捕获患者经验数据，使其能够为监管机构使用的证据做出贡献，付款人,和开药者的决策。将PPC和CPC评估添加到罕见疾病临床试验中，提供了一种创新而强大的方式来利用患者及其家人的独特见解，以更全面地了解患者在临床试验中的经历。
隐形生物治疗公司.
The traditional model of evaluating treatments based primarily on primary outcome measures has stumbled in its application to rare disease. Rare disease clinical trials face the methodological challenges of small, heterogeneous patient populations and relatively few validated, disease-specific outcome measures. Incorporating qualitative research into rare disease clinical trials may help sponsors, regulators, payers, and prescribers to better understand the real-world and patient-specific impact of a potential therapy. This paper provides a methodologic overview of the use of Patient and Caregiver Perception of Change (PPC and CPC) Assessments utilizing patient and caregiver video interviews to complement the data captured by traditional endpoints in rare disease clinical trials.
Incorporating qualitative patient and caregiver video interviews into clinical trials allows for the rigorous capture of patient experiences and caregiver observations. Interview guides informed by input from key stakeholders provide the opportunity to solicit structured feedback on experiences before, during, and after the clinical trial. Patients and caregivers can complete their video interviews in a study mobile application, and interview transcripts are analyzed by independent coders. Themes are summarized by the treatment group and individual patient, which adds context to the clinical outcome measures of how patients feel and function, as well as elucidates the degree of change that is meaningful to patients and caregivers. The qualitative results can be compared to the data captured in clinical trials to assess data concordance.
Capturing patient experience data with sufficient rigor allows it to contribute to the body of evidence utilized in regulatory, payer, and prescriber decision-making. Adding PPC and CPC Assessments to rare disease clinical trials offers an innovative and powerful way to tap into the unique insights of patients and their families to develop a fuller picture of the patient experience in the clinical trial.
Stealth BioTherapeutics Inc.
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