Center for Individualized Medicine Grand Rounds

Description

Title: From Bedside to Bench and Back: How Patient Communities are Transforming Rare Disease Research and Driving Scientific Discoveries

Speaker: Tania Simoncelli, MS | Chan Zuckerberg Initiative

Rare diseases impact as many as 400 million people around the world.  Scientific progress is stymied by small patient populations, suboptimal collaboration, and inefficient data sharing. An increasing number of patient advocacy groups have emerged over the last two decades that seek to overcome these barriers.  Far beyond the education and awareness efforts of traditional advocacy groups, these organizations are actively driving research forward by building strong and highly informed patient communities, developing clinical registries and biobanks, funding research, and partnering with industry to develop treatments. This presentation will describe this emerging model of patient-driven research, highlight examples of how these groups are changing the way knowledge is produced and transforming the research landscape in their disease areas, and discuss what more is needed to fully unleash the power of patient communities to accelerate biomedical research and innovation.