Open AccessThe Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study
Author(s) -
Debra S. Regier,
Jennifer Weaver,
Nancy Cheng,
Mark L. Batshaw,
Mary Ottolini,
Michael E. Shy,
Marshall Summar
Publication year - 2022
Publication title -
translational science of rare diseases
Language(s) - English
Resource type - Journals
eISSN - 2214-6512
pISSN - 2214-6490
DOI - 10.3233/trd-210051
Subject(s) - thematic analysis , medical education , curriculum , population , psychology , descriptive statistics , qualitative property , qualitative research , test (biology) , medicine , computer science , pedagogy , sociology , social science , statistics , mathematics , environmental health , machine learning , paleontology , biology
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.