Stakeholder perspectives on clinical research related to therapies for rare diseases: therapeutic misconception and the value of research.
Stakeholder perspectives on clinical research related to therapies for rare diseases: therapeutic misconception and the value of research.
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DOI：
10.1186/s13023-020-01624-0
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发表时间：
2021-01-12
影响因子：
3.7
通讯作者：
Canadian Inherited Metabolic Diseases Research Network
中科院分区：
文献类型：
--
作者：
Tingley K;Coyle D;Graham ID;Chakraborty P;Wilson K;Potter BK;Canadian Inherited Metabolic Diseases Research Network
对于许多罕见疾病，很少有治疗方法得到强有力的证据支持。因此，患者、家庭成员、卫生保健提供者和政策制定者必须考虑是否接受、推荐或资助临床疗效不确定的治疗。他们还必须考虑是否以及如何参与可能涉及接受或提供正在评估的治疗的临床研究。了解罕见代谢疾病患者和家庭为什么以及如何选择是否参与研究以及他们如何使用和重视研究。我们对每个利益相关者群体进行了单独的焦点小组访谈（共三组）;两组通过电话进行，第三组亲自进行。参与者采用目的性抽样招募。我们分析了每个访谈记录顺序使用定性描述的方法，归纳确定的关键主题。采用了几种确保可信度和可信赖性的策略，包括在每个焦点小组之后举行汇报会，并让多名小组成员审查成绩单。四名患者/护理人员，六名医生和三名政策顾问参加了会议。我们的研究结果并不支持传统观点，即治疗误解（获得治疗）是患者/护理人员参与临床研究的主要动机因素。相反，患者/护理人员表示参与研究的原因包括为下一代推进科学，并有机会分享他们的经验。患者/护理人员和医生描述了在权衡接受没有证据充分支持的治疗的风险与受益时的困难。医生还报告说，他们作为患者顾问/倡导者和证据评估者的双重角色感到矛盾。政策顾问主要侧重于批判性地评估证据，为卫生系统提出建议。利益相关者对罕见病研究的观点各不相同，但在做出个人和人群层面的决策时，他们对治疗的风险与益处有着共同的担忧。
For many rare diseases, few treatments are supported by strong evidence. Patients, family members, health care providers, and policy-makers thus have to consider whether to accept, recommend, or fund treatments with uncertain clinical effectiveness. They must also consider whether and how to contribute to clinical research that may involve receiving or providing the therapy being evaluated. To understand why and how patients and families with rare metabolic diseases, specialist metabolic physicians, and health policy advisors choose whether to participate in studies and how they use and value research. We conducted separate focus group interviews with each stakeholder group (three groups in total); two groups were conducted by telephone and the third was held in-person. Participants were recruited using purposive sampling. We analyzed each interview transcript sequentially using a qualitative description approach to inductively identify key themes. Several strategies to ensure credibility and trustworthiness were used including debriefing sessions after each focus group and having multiple team members review transcripts. Four patients/caregivers, six physicians, and three policy advisors participated. Our findings did not support conventional perspectives that therapeutic misconception (gaining access to treatment) is the main motivating factor for patients/caregivers to participate in clinical research. Rather, patients’/caregivers’ expressed reasons for participating in research included advancing science for the next generation and having an opportunity to share their experiences. Patients/caregivers and physicians described the difficulties in weighing risks versus benefits of accepting treatments not well-supported by evidence. Physicians also reported feeling conflicted in their dual role as patient advisor/advocate and evaluator of the evidence. Policy advisors were primarily focused on critically appraising the evidence to make recommendations for the health system. Stakeholders differ in their perspectives on rare disease research but share concerns about the risks versus benefits of therapies when making individual- and population-level decisions.
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影响因子：
15.8
作者：
Henderson GE;Churchill LR;Davis AM;Easter MM;Grady C;Joffe S;Kass N;King NM;Lidz CW;Miller FG;Nelson DK;Peppercorn J;Rothschild BB;Sankar P;Wilfond BS;Zimmer CR
通讯作者：
Zimmer CR
影响因子：
1.9
作者：
DeWard, Stephanie J.;Wilson, Ashley;Mooney, Kimberly
通讯作者：
Mooney, Kimberly
影响因子：
1.7
作者：
Bradshaw, Carmel;Atkinson, Sandra;Doody, Owen
通讯作者：
Doody, Owen
影响因子：
5.4
作者：
Henderson, GE;Easter, MM;Nelson, DK
通讯作者：
Nelson, DK
影响因子：
3.7
作者：
Chow SC;Chang M
通讯作者：
Chang M