A landscape review to identify what matters to patients with thrombotic cardiovascular diseases and patient-reported outcome instruments which can be used to capture the patient experience.

IF 3.3 3区医学 Q1 HEALTH CARE SCIENCES & SERVICES Quality of Life Research Pub Date : 2024-11-22 DOI:10.1007/s11136-024-03790-1

Alexandra I Barsdorf, John Fastenau, Shannon Lee, Xiaoyan Li, Ellen O'Brien, Blue Stevenson, Brandon Becker

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Abstract

Purpose: Thrombotic cardiovascular diseases profoundly impact patients' health-related quality of life (HRQoL). However, patient-reported outcome (PRO) instruments that are disease-specific or antithrombotic-treatment focused, developed according to US Food and Drug Administration (FDA) guidance on PROs, and can be used in clinical trials, are lacking. The aim of this study was to understand concepts important to patients diagnosed with coronary artery disease (CAD) or acute coronary syndrome (ACS), atrial fibrillation (AF), or stroke who require antithrombotic treatment for reducing risk of future thrombotic events (indications being evaluated for an investigational new drug), identify PROs that measure relevant symptoms and impacts, and determine acceptability of PROs from a health technology assessment (HTA) perspective.

Methods: A landscape review, conducted between January 2009 and October 2020, included a search of qualitative literature (OVID), a review of PRO instruments using multiple sources (e.g., OVID and clinical trials databases), and a survey of HTA decisions for antithrombotic medications.

Results: The qualitative literature review identified 27 publications used to develop a high-level conceptual summary of symptoms and HRQoL impacts reported by patients. The instrument landscape review indicated that generic PROs have been utilized for thrombotic indications, but disease-specific, fit-for-purpose instruments are lacking, and the HTA review revealed that although HTA agencies discussed PRO instruments, evidence of specific recommendations was not found.

Conclusion: To ensure patients' experiences, perspectives, and priorities are incorporated into drug development and evaluation, a core set of PROs for thrombotic indications that meet health authority guidance and are acceptable to HTA agencies is needed.

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进行一次情况回顾，以确定对血栓性心血管疾病患者重要的是什么，以及可用于获取患者体验的患者报告结果工具。

目的：血栓性心血管疾病严重影响患者的健康相关生活质量（HRQoL）。然而，目前还缺乏针对特定疾病或以抗血栓治疗为重点、根据美国食品药品管理局（FDA）关于患者报告结果（PRO）的指南开发、可用于临床试验的患者报告结果（PRO）工具。本研究旨在了解对确诊为冠状动脉疾病（CAD）或急性冠状动脉综合征（ACS）、心房颤动（AF）或中风且需要抗血栓治疗以降低未来血栓事件风险的患者（正在接受新药研究评估的适应症）而言非常重要的概念，确定可测量相关症状和影响的PROs，并从健康技术评估（HTA）的角度确定PROs的可接受性：方法：在 2009 年 1 月至 2020 年 10 月期间进行了一次全面回顾，包括定性文献（OVID）检索、使用多种来源（如 OVID 和临床试验数据库）对 PRO 工具进行回顾，以及对抗血栓药物的 HTA 决策进行调查：定性文献综述确定了 27 篇出版物，用于对患者报告的症状和 HRQoL 影响进行高级概念总结。工具情况回顾表明，血栓适应症已经使用了通用的PRO，但缺乏针对特定疾病的、适合目的的工具；HTA回顾表明，虽然HTA机构讨论了PRO工具，但没有发现具体建议的证据：结论：为确保将患者的经验、观点和优先事项纳入药物开发和评估，需要为血栓适应症制定一套核心的PRO，这些PRO应符合卫生当局的指导意见，并为HTA机构所接受。

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来源期刊

Quality of Life Research 医学-公共卫生、环境卫生与职业卫生

CiteScore

6.50

自引率

8.60%

发文量

224

审稿时长

3-8 weeks

期刊介绍： Quality of Life Research is an international, multidisciplinary journal devoted to the rapid communication of original research, theoretical articles and methodological reports related to the field of quality of life, in all the health sciences. The journal also offers editorials, literature, book and software reviews, correspondence and abstracts of conferences. Quality of life has become a prominent issue in biometry, philosophy, social science, clinical medicine, health services and outcomes research. The journal''s scope reflects the wide application of quality of life assessment and research in the biological and social sciences. All original work is subject to peer review for originality, scientific quality and relevance to a broad readership. This is an official journal of the International Society of Quality of Life Research.