How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis.
How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis.
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DOI：
10.1186/s13023-023-02682-w
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发表时间：
2023-04-17
影响因子：
3.7
通讯作者：
中科院分区：
文献类型：
--
作者：
直到最近，孤儿药开发患者经历的多样性一直被忽视，现有文献报告了一些患者的经历，而不是其他患者。目前的证据基础（目前最好的研究）是由定量调查和研究人员定义的患者报告的结果措施。在使用定性方法进行数据收集和分析的研究中，使用内容分析和自动文本分析来研究患者体验，而不是深入的定性分析方法。对患者参与孤儿药开发的系统评价也排除了定性研究。本文的目的是回顾有关患者和其他公众成员如何参与孤儿药开发的定性文献。我们对描述一系列患者参与实践和经验的定性论文进行了系统检索，并进行了识别和筛选。由两名独立研究人员使用经验证的工具（CASP）对纳入的论文进行评估，并辅以报告指南（COSTO）。262篇论文被发现13篇论文报告了一系列定性数据收集方法。许多人将患者和公众参与和参与（PPIE）与定性研究混为一谈。患者通常通过他们的医生或患者组织招募。我们发现缺乏总体的哲学或方法框架，知情同意过程的细节有限，缺乏可识别的数据分析方法。我们的叙述性综合表明，患者和护理人员需要参与试验设计的各个方面，包括选择捕获更广泛结局的临床终点，确定扩大试验参与的方法，开发面向患者的材料以优化其决策，以及将患者纳入试验结果的传播。这种叙述性的定性综合确定了在罕见病患者的研究中明确需要严格的方法学（例如，适当和创新地使用定性方法或PPIE，而不是将其混为一谈）;努力捕捉服务不足、研究不足或很少听取有罕见疾病经验的社区的观点（例如创造性的招聘和更广泛地采用后殖民实践）;以及重新调整研究议程（例如使用共同设计，使患者能够设定议程，而不是对他们所提供的内容做出反应）。在线版本包含补充材料，可通过10.1186/s13023-023-02682-w获得。
The diversity of patient experiences of orphan drug development has until recently been overlooked, with the existing literature reporting the experience of some patients and not others. The current evidence base (the best available current research) is dominated by quantitative surveys and patient reported outcome measures defined by researchers. Where research that uses qualitative methods of data collection and analysis has been conducted, patient experiences have been studied using content analysis and automatic textual analysis, rather than in-depth qualitative analytical methods. Systematic reviews of patient engagement in orphan drug development have also excluded qualitative studies. The aim of this paper is to review qualitative literature about how patients and other members of the public engage with orphan drug development. We conducted a systematic search of qualitative papers describing a range of patient engagement practices and experiences were identified and screened. Included papers were appraised using a validated tool (CASP), supplemented by reporting guidance (COREQ), by two independent researchers. 262 papers were identified. Thirteen papers reported a range of methods of qualitative data collection. Many conflated patient and public involvement and engagement (PPIE) with qualitative research. Patients were typically recruited via their physician or patient organisations. We identified an absence of overarching philosophical or methodological frameworks, limited details of informed consent processes, and an absence of recognisable methods of data analysis. Our narrative synthesis suggests that patients and caregivers need to be involved in all aspects of trial design, including the selection of clinical endpoints that capture a wider range of outcomes, the identification of means to widen access to trial participation, the development of patient facing materials to optimise their decision making, and patients included in the dissemination of trial results. This narrative qualitative synthesis identified the explicit need for methodological rigour in research with patients with rare diseases (e.g. appropriate and innovative use of qualitative methods or PPIE, rather than their conflation); strenuous efforts to capture the perspectives of under-served, under-researched or seldom listened to communities with experience of rare diseases (e.g. creative recruitment and wider adoption of post-colonial practices); and a re-alignment of the research agenda (e.g. the use of co-design to enable patients to set the agenda, rather than respond to what they are being offered). The online version contains supplementary material available at 10.1186/s13023-023-02682-w.
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影响因子：
3.7
作者：
Tingley K;Coyle D;Graham ID;Chakraborty P;Wilson K;Potter BK;Canadian Inherited Metabolic Diseases Research Network
通讯作者：
Canadian Inherited Metabolic Diseases Research Network
影响因子：
3.6
作者：
Menon, Devidas;Stafinski, Tania;Dunn, Andrea;Wong-Rieger, Durhane
通讯作者：
Wong-Rieger, Durhane
影响因子：
3.7
作者：
Denton N;Molloy M;Charleston S;Lipset C;Hirsch J;Mulberg AE;Howard P;Marsh ED
通讯作者：
Marsh ED
影响因子：
3.6
作者：
Campbell, R.;Pound, P.;Donovan, J.
通讯作者：
Donovan, J.
影响因子：
5.4
作者：
Baggott, Rob;Jones, Kathryn
通讯作者：
Jones, Kathryn