# Rare Diseases Clinical Research Scholar’s Program

> **NIH NIH R25** · CHILDREN'S RESEARCH INSTITUTE · 2020 · $60,000

## Abstract

Project Title: Rare Diseases Clinical Research Scholar's Program:
PROJECT SUMMARY
Rare diseases (RD) are unique as a medical field, comprising a large number of disorders with
a small number of patients in each. They primarily affect children and most have a genetic
basis. The methods employed in rare disease research (RDR) require models and analytical
tools that are different from more common conditions. This proposal seeks to develop a blended
learning curriculum employing specific tools and methods unique to rare diseases. It will
continue to be work closely with the NIH funded Rare Diseases Clinical Research Network
(RDCRN), a collaborative of 240 sites at universities and academic medical centers. It is
expected that this program will continue to attract and retain new investigators in the field of rare
disease through network development, research success, and exposure to patient and family
groups. The Rare Disease Scholar's Program is a curriculum provided over an academic year
that has 6 components: 1.) an initial two day face-to-face RDR methods and policy workshop
that will bring together the trainees, mentors, patient advocacy groups, federal agencies and
biotech/pharma. 2.) a series of semi-monthly interactive, web-based seminars on methodologies
in RDR including study design, statistics, bioethics, therapeutics, policy and approaches to
interacting with other key contributors to the field, including patient advocacy groups, federal
agencies and biotech/pharma. 3.) a semi-monthly “chalk board” videoconference focusing on
individual trainee RDR projects with an emphasis on practical design and analytical tools. 4) a
web portal with course content, opportunities for interaction among trainees and between
trainees and teachers/mentors, and other interactive features. 5) a “capstone” research project
presentation by each of the trainees at the semi-annual RDCRN meeting that will bring together
the trainees, patient advocacy groups, federal agencies and biotech/pharma. 6.) Utilizing the
CTSA tracking and evaluation system and a pre- and posttest to quantify understanding of the
topics involved in RDR prior to and following the curriculum. These tools will also be used to
monitor the quality of the program and instruction. We will track the program's outcome by
following the trainees in terms of academic career choice, retention in the field of RDR, and
other measures of success. Twenty to twenty five trainees will continue to matriculate each
year. The program involves 80 hours of direct teaching and at least 40 hours of prep work
(homework) as well as the work required to draft and carry out a research study protocol, and
write up the results. Trainees completing the program will receive a certificate in RDR from the
RDCRN.

## Key facts

- **NIH application ID:** 10198146
- **Project number:** 3R25HD078229-05S1
- **Recipient organization:** CHILDREN'S RESEARCH INSTITUTE
- **Principal Investigator:** Debra S Regier
- **Activity code:** R25 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $60,000
- **Award type:** 3
- **Project period:** 2020-06-26 → 2022-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10198146

## Citation

> US National Institutes of Health, RePORTER application 10198146, Rare Diseases Clinical Research Scholar’s Program (3R25HD078229-05S1). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10198146. Licensed CC0.

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