The Rare Diseases Clinical Research Network (RDCRN) and the Clinical and Translational Science Awards (CTSA) programs co-sponsored the 2nd Conference on Clinical Research for Rare Diseases (CCRRD) which was held in September 2010 in Bethesda, Maryland. The Conference was again planned and organized by Dr. Peter Merkel of the Boston University School of Medicine. This unique conference focused on research methodology for rare diseases and with a particular emphasis on helpful information for trainees and junior faculty engaged in such research. There were 270 meeting attendees, including trainees, junior and senior faculty, NIH program officials, and leaders of patient advocacy groups.
Among the conference speakers were investigators from the RDCRN and CTSA program, and representatives of the US FDA, the biomedical industry, and patient advocacy groups. Topics covered included creating research networks for rare diseases, design issues for clinical trials of small sample sizes, pathways to FDA approval of orphan drugs, partnering on rare disease research with the pharmaceutical industry, utilizing the resources of the CTSAs, understanding unique issues of conflict of interest in rare disease research, and the challenges and opportunities involved in developing an academic career studying rare diseases. Over 90 scientific posters were presented and award-winning abstracts were presented in a plenary session. The conference ended with a lecture by Mr. John Crowley, whose account of his successful professional efforts to promote the development of a therapy to treat Pompe disease, which affects two of his children, was inspirational for all attendees.
The CCRRD was supported by generous grants from the following NIH offices/institutes: ORDR, NINDS, NCRR, NIAID, and NICHD. Additional support for trainees was provided by several patient advocacy groups.
The Conference on Clinical Research for Rare Diseases was a great success. Plans are underway for CCRRD 2012.
Peter A. Merkel, MD, MPH
Principal Investigator of the Vasculitis Clinical Research Consortium
Professor of Medicine Section of Rheumatology and the Clinical Epidemiology Unit
Director, Vasculitis Center
