The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study.
The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study.
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DOI：
10.3233/trd-210051
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发表时间：
2022
期刊：
影响因子：
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通讯作者：
Summar, Marshall L
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文献类型：
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作者：
Regier, Debra S;Weaver, Jennifer A;Cheng, Nancy;Batshaw, Mark L;Ottolini, Mary;Shy, Michael E;Summar, Marshall L
罕见病临床研究人员对于确保快速增长的罕见病人群得到适当的诊断、护理和治疗至关重要。由于这些研究人员分布在许多专业领域，学习罕见疾病研究（RDR）的独特技能可能是一个障碍，并可能阻碍该领域的进展。在NIH资助的罕见疾病临床研究网络的学员需求评估中，确定了许多专业和背景的研究人员需要一个以RDR为重点的培训计划。基于这些信息，开发了罕见疾病研究学者计划（RDRSP）。我们描述了需求评估，课程设置，学者招聘和成果评估的基础上四年的计划数据（2015-2019）。这个为期一年的RDRSP使用混合方法，包括面对面、基于网络、同步和异步学习。我们使用定量和定性方法对RDRSP进行了评估。定量措施包括前和后的知识，自我效能和意图留在RDR问卷。采用描述性统计和配对t检验分析数据。定性半结构化访谈探讨了RDR学者的RDRSP的看法，主题分析研究的文本数据。定量的前后测量在以下方面具有统计学意义：1）RDR中的知识内容得到改善，2）临床研究中的自我效能得到增强，3）留在RDR领域的意图。定性数据分析发现，该计划支持学者的研究技能的发展，以及“软技能”。通过将RDR独特技能的培训与不同专业参与者之间的领导力、指导和协作等更一般的主题相结合，我们创建了一个支持下一代罕见疾病临床研究人员发展的计划，并作为培训的典范。在其他利基研究领域。
Rare disease clinician investigators are essential to ensure appropriate diagnosis, care, and treatment for the rapidly growing rare disease population. As these researchers are spread across many specialties, learning the unique skill set for rare disease research (RDR) can be a hurdle and may hinder progress in the field. The need for an RDR focused training program for investigators in many specialties and backgrounds was identified in a needs assessment of trainees in the NIH funded Rare Diseases Clinical Research Network. Based on this information, the Rare Disease Research Scholars Program (RDRSP) was developed. We describe the needs assessment, curriculum creation, scholar recruitment, and outcome evaluation based on four years of programmatic data (2015–2019). This one year-long RDRSP uses a blended approach that includes in-person, web-based, synchronous and asynchronous learning. We evaluated the RDRSP using quantitative and qualitative approaches. Quantitative measures included pre and post questionnaires about knowledge, self-efficacy, and intent to remain in RDR. Data were analyzed using descriptive statistics and a paired t-test. Qualitative semi-structured interviews explored the RDR scholars’ perceptions of the RDRSP; thematic analysis examined the textual data. Quantitative pre- and post-measures were statistically significant in the following areas: 1) improved knowledge content in RDR, 2) enhanced self-efficacy in clinical research, and 3) intent to remain in the field of RDR. Qualitative data analysis found the program supported the development of the scholar’s research skills as well as ‘soft-skills’. By combining training of skills unique to RDR with the more general topics of leadership, mentorship and collaboration among participants in diverse specialties, we created a program that supports the development of the next generation of rare disease clinician investigators and serves as a model for training in other niche research areas.