The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study

Debra S Regier; Jennifer A Weaver; Nancy Cheng; Mark L Batshaw; Mary Ottolini; Michael E Shy; Marshall L Summar

doi:10.3233/TRD-210051

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The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study

Journal article

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The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study

Debra S Regier, Jennifer A Weaver, Nancy Cheng, Mark L Batshaw, Mary Ottolini, Michael E Shy and Marshall L Summar

Translational science of rare diseases, Vol.6(1-2), pp.1-11

12/17/2021

DOI: 10.3233/TRD-210051

PMCID: PMC9108859

PMID: 35601643

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Abstract

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.

Details

Title: Subtitle: The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study
Creators: Debra S Regier - Children’s National Hospital, Washington, DC, USA
Jennifer A Weaver - Colorado State University
Nancy Cheng - Children’s National Hospital, Washington, DC, USA
Mark L Batshaw - Children’s National Hospital, Washington, DC, USA, Children’s National Research Institute, Children’s National Hospital, Washington, DC, USA
Mary Ottolini - Barbara Bush Children’s Hospital
Michael E Shy - University of Iowa
Marshall L Summar - Children’s National Hospital, Washington, DC, USA
Resource Type: Journal article
Publication Details: Translational science of rare diseases, Vol.6(1-2), pp.1-11
DOI: 10.3233/TRD-210051
PMID: 35601643
PMCID: PMC9108859
ISSN: 2214-6490
eISSN: 2214-6512
Language: English
Date published: 12/17/2021
Academic Unit: Neurology; Molecular Physiology and Biophysics; Stead Family Department of Pediatrics; Iowa Neuroscience Institute
Record Identifier: 9984202255702771

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