One Voice: SCAD Alliance Rare Disease Advocacy in Washington, D.C

“Join together for better care.” The theme of the 2014 Rare Disease Day events in Washington, D.C. captured the spirit of the rare movement to the essence. Participants came together to speak as one voice on behalf of the rare disease community, which includes more than 7,000 rare diagnoses. To offer perspective, one in 10 Americans have a rare disease, of which 80% are genetic. Rare diseases impact more people than do AIDS and all cancers combined.

For SCAD Alliance, the advocacy week in D.C. was action-packed and full of surprises.  At the National Press Club on Tuesday, February 25, 2014, we gained valuable insight into the state of rare disease advocacy at the RDLA Legislative Conference. Hearing the stories of families whose battles revolve around drug trials and FDA approval gave perspective to the challenges facing rare disease patients everywhere.

The raw power of rare disease, clinical trials and advocacy came to life Tuesday evening, during a screening of the HBO documentary “Life According to Sam” at the Carnegie Institute of Science. Sam Berns, who died January 10, 2014, at age 17 from complications of Progeria, is a young man whose outlook and spirit offer lessons to each of us, showing that rare diseases DO matter. In a bittersweet turn of events, a ceremony after the screening honored Stephen C. Groft, Pharm. D., who recently retired as director of the NIH National Center for Advancing Translational Sciences (NCATS) Office of Rare Diseases Research (ORDR). SCAD Alliance was privileged to be among the many participants that signed our thanks and good wishes on his retirement gift.

Wednesday, it was on to Capitol Hill for meetings with members of Congress in our respective states to share our SCAD stories and ask for support of HR 1591 “CAL Undiagnosed Diseases Research and Collaboration Network Act,” HR 3116 “The MODDERN Cures Act,” and HR 460 “Patients’ Access to Treatments Act.” In addition, all advocates encouraged members to join the Rare Disease Congressional Caucus, and obtain a Senate Champion to Co-Chair the Caucus. The House Caucus is led by Representatives Leonard Lance (R-NJ) and Joseph Crowley (D-NY).

On Rare Disease Day, February 28, 2014, SCAD Alliance was an exhibitor at the National Institutes of Health (NIH) during a daylong conference on rare diseases. In addition to attending several fascinating presentations throughout the day, we were honored to meet briefly with Gary H. Gibbons, M.D., director of the National Heart, Lung, and Blood Institute (NHLBI). Dr. Gibbons expressed his support of and interest in SCAD, as well as his appreciation for patient involvement in advancing research.