June 02, 2016

The Rare Diseases Clinical Research Network (RDCRN) sponsored the 4th Conference on Clinical Research for Rare Diseases (CCRRD) on Thursday, November 3, 2016 in Washington, D.C.

The CCRRD is supported by an NCATS conference grant with additional support from several other NIH Institutes and by RDCRN patient advocacy groups.

This unique conference focuses on research methodology for rare diseases and should be of particular interest to trainees and junior faculty engaged in research in rare diseases. Among the 2016 conference speakers were investigators from the RDCRN, and representatives of the US FDA, the biomedical industry, and patient advocacy groups. The meeting agenda included the following topics: design issues for clinical trials of small sample sizes, systems biology and big data in rare diseases, working with the FDA and industry for rare diseases research, novel approaches to rare diseases study design, and the challenges and opportunities involved in developing an academic career studying rare diseases. A poster session allowed for presentation of research in rare diseases with informal review and comments provided by senior investigators and NIH program officials attending the meeting.

Travel awards up to $850 were available on a competitive basis, and the award-winning abstracts were presented in a plenary session.

Prior attendees of this Conference have found its content and format extremely helpful and informative.

For more information about the conference, visit the conference website: www.rarediseasesnetwork.org/conference/2016/registration/index.htm.

by Peter A. Merkel, MD, MPH and Carol McAlear, MA
Fall 2016