The PSC Partners Patient Registry Origin Story

Little did we know that attending a workshop in Washington D.C. in 2010 would be instrumental in moving PSC Partners towards the unimaginable patient-generated and patient-driven research. In 2010, it was unthinkable that our rare primary sclerosing cholangitis could create a loud enough voice to have a measurable impact on PSC research. In 2009, PSC Partners, in its fifth year of existence, had awarded its first research grants, and we were excitedly searching for more ways of creating PSC awareness.

Ricky and RachelsmallThe National Institutes of Health (NIH) workshop, “Advancing Rare Disease Research,” held in Washington D.C., was a pivotal experience for PSC Partners. Hundreds of physicians, academics, patient advocacy groups, drug developers, and regulators from the Food and Drug Administration (FDA) attended this meeting. It was unanimously agreed that having a patient-owned and patient-controlled standardized registry to locate dispersed rare-disease patients was key to increasing engagement in rare-disease research and in facilitating and stimulating research on our neglected diseases. 

We jumped right in. Starting a registry required knowledge and experience in building and running registries, and, of course, serious funds. PSC Partners Founder and President Ricky Safer and I were volunteers who did not have these important pre-requisites. However, one powerful asset we could count on was our boundless, infinite passion. Shortly after, we were selected to join a registry pilot program run by the NIH Office of Rare Diseases Research (ORDR). We needed medical support. Dr. Christopher Bowlus, Head of Hepatology at the University of California, Davis, and Co-chair of PSC Partners’ Scientific/Medical Advisory Committee (SMAC), never left our side. For a full two years, he attended all meetings with us and became our unfaltering medical advisor and friend. We launched the PSC Partners Patient Registry in 2014. 

The registry is still in its infancy, and yet has made huge strides. You can explore what's been accomplished to date on the IMPACT page. The 2020 registry upgrade will undoubtedly open many new doors. The registry will no longer be a static picture of a single moment in the PSC journey. This upgrade will allow us to observe changes over time, to add new surveys, and to collect clinical data on each participant. 

If you are new to this page and to PSC Partners, we encourage you to join our effort to speed up PSC research and to take part in our PSC Partners community by choosing from the myriads of community activities that are wide open to you. Visit the PSC Partners website at www.pscpartners.org.

Our registry coordinators are always ready to guide you. You can reach them by email at registrycoordinator@pscpartners.org

We’re together in this fight … whatever it takes!

Rachel Gomel
Registry Director and Coordinator
PSC Partners Patient Registry 

Other members of the registry team:
Sharon Nanz – Registry Assistant Coordinator
Mary P. Vyas – Registry Strategist
Dr. Christopher Bowlus – Registry Advisor