September 2015 was the launch of The Rare Disease Research Training Program with our first in-person 2 day meeting.

The training program, supported by an RDCRN R25 grant, is a 12 month series of in-person and webinar-based opportunities allowing for synchronous and asynchronous learning opportunities to better understand the practical skills required to succeed in rare disease clinical research.

The goals of the program are to:

  1. Improve comfort with a clinical research career and perceived capacity to succeed in research.
  2. Promote success in rare disease research.
  3. To enhance the ability of clinical researchers to communicate effectively with rare disease stakeholders.

The cohort for 2015-2016 consists of 21 stellar early career investigators. Participants from most of the clinical research consortiums with varied backgrounds, some with MD and others with PhD training, joined the group, leading to diversity in the field of study and backgrounds.

The first two day in-person session consisted of didactics and discussions focused on the essentials of clinical research. Our opening address by Dr. Chris Austin from NCATS highlighted the need to support this stellar group of young investigators. Then, we heard from experts on topics including small cohort statistics, PCORI, the FDA, budgeting for rare disease research, and from the National Organization of Rare Diseases. In the second day, we spent time discussing the involvement of patient advocacy groups in research and went to Capitol Hill to learn about the role of federal legislation on rare disease research.

Upon completion of the in-person meeting in Washington, DC, we launched into the asynchronous course materials. Those course materials are wide-ranging. For example, materials include an introduction to interactions with industry, finding a rare disease niche, and "selling" the rare disorder to the "common" disorder world. The bi-monthly webinars have offered both didactic and discussion-based opportunities for all the participants to think about rare disease clinical research in new ways, both in real time and asynchronously.

We are now announcing the application request from the 2016-2017 cohort. Please share the announcement with any early career clinical researchers who might be interested in participating in the next group. For more information, please see www.rdcrn.org/train.


How to submit an application

Applications must include the following:

  • A letter of recommendation from a mentor
  • A letter of interest describing a clinical research project in rare disease (1 page)
  • CV

Please send all of the above to the Education Director: Debra Regier, MD, PhD (dregier@childrensnational.org).

Applications are due on or before April 15.

In reply, the official application form (1 page) will be returned for completion. Decisions will be communicated to applicants by June 1.


by Debra Regier, MD, PhD 
Spring 2016

January 03, 2016