Determining Commonalities in the Experiences of Patients with Rare Diseases: A Qualitative Analysis of US Food and Drug Administration Patient Engagement Sessions.

IF 3.4 3区医学 Q1 HEALTH CARE SCIENCES & SERVICES

Patient-Patient Centered Outcomes Research Pub Date : 2024-01-01 Epub Date: 2023-10-13 DOI:10.1007/s40271-023-00648-5

Catherine Mease, Lewis J Fermaglich, Karen Jackler, Shawn Shermer, Kathleen L Miller

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引用次数: 0

Abstract

Background: Rare diseases are estimated to affect more than one in ten Americans. However, most patients with a rare disease face significant emotional, physical, and social challenges. To better understand the burden of disease and unmet needs, the US Food and Drug Administration (FDA) conducts and supports multiple patient engagement platforms. We analyzed summaries from these discussions to identify commonalities among patients with disparate rare diseases, the results of which could inform priorities for cross-disease policies and medical product development.

Methods: We conducted a qualitative analysis of patient engagement session summaries to investigate shared experiences across rare diseases. Cross-disease similarities were identified within four dimensions: product development/regulatory, clinical/physical, social/psychological, and economic/financial. Summaries from 29 rare diseases were included in our analyses.

Results: Within the product development/regulatory dimension, we observed that patients and caregivers across rare diseases shared the desire for development of medical products that cured their disease or improved their overall quality of life. In the clinical/physical dimension, we found that patients had numerous common symptoms, including pain and fatigue. In the social/psychological dimension, we observed significant negative impact on mental health. Within the economic/financial dimension, patients and caregivers shared that disease burden caused significant financial hardships.

Conclusion: We found remarkable similarities among patients with rare diseases across all four dimensions. Our results indicate that, even among rare diseases with diverse etiologies, patients share numerous commonalties due to their diseases: a lack of effective treatment options, certain physical symptoms, mental health challenges, and financial concerns.

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确定罕见病患者经历的共性：美国食品药品监督管理局患者参与会议的定性分析。

背景：据估计，超过十分之一的美国人患有罕见疾病。然而，大多数罕见病患者都面临着巨大的情感、身体和社会挑战。为了更好地了解疾病负担和未满足的需求，美国食品药品监督管理局（FDA）建立并支持多个患者参与平台。我们分析了这些讨论的总结，以确定不同罕见病患者的共性，其结果可以为跨疾病政策和医疗产品开发的优先事项提供信息。方法：我们对患者参与会议总结进行了定性分析，以调查罕见病的共同经验。在四个维度上确定了跨疾病的相似性：产品开发/监管、临床/物理、社会/心理和经济/金融。我们的分析包括了29种罕见病的总结。结果：在产品开发/监管层面，我们观察到罕见病患者和护理人员都渴望开发治愈疾病或提高整体生活质量的医疗产品。在临床/身体方面，我们发现患者有许多常见症状，包括疼痛和疲劳。在社会/心理方面，我们观察到对心理健康的显著负面影响。在经济/财务层面，患者和护理人员分担了疾病负担造成的重大财务困难。结论：我们发现罕见病患者在所有四个维度上都有显著的相似性。我们的研究结果表明，即使在病因多样的罕见病中，患者也因其疾病而有许多共同点：缺乏有效的治疗选择、某些身体症状、心理健康挑战和经济问题。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Patient-Patient Centered Outcomes Research HEALTH CARE SCIENCES & SERVICES-

CiteScore

6.60

自引率

8.30%

发文量

审稿时长

>12 weeks

期刊介绍： The Patient provides a venue for scientifically rigorous, timely, and relevant research to promote the development, evaluation and implementation of therapies, technologies, and innovations that will enhance the patient experience. It is an international forum for research that advances and/or applies qualitative or quantitative methods to promote the generation, synthesis, or interpretation of evidence. The journal has specific interest in receiving original research, reviews and commentaries related to qualitative and mixed methods research, stated-preference methods, patient reported outcomes, and shared decision making. Advances in regulatory science, patient-focused drug development, patient-centered benefit-risk and health technology assessment will also be considered. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in The Patient may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances. All manuscripts are subject to peer review by international experts.