Individual parents and patients are increasingly doing more to fund, discover and develop treatments for rare and ultra-rare diseases that afflict their children, themselves or their friends. They are performing many roles in driving the science that are equivalent to professional scientists. Through their efforts and that of the collaborative networks which they have developed, they may be in position to disrupt drug discovery. But what is missing is accessibility to scientific data and publications. This can be illustrated using three different ultra-rare disease parent / patient advocate groups and the diseases for which they are developing treatments. Each group encountered difficulties in accessing information. Perhaps what could be proposed is an open data commons or database dedicated to rare and ultra-rare diseases. This could encompass chemistry and biology data. Our efforts with the mobile app Open Drug Discovery Teams (ODDT) will be described to catalyze such efforts. Challenges will likely come from industry which sees this as a lucrative field. Our aim is to discuss the pros and cons of Open Data for Ultra-Rare and Rare Disease Drug Discovery and suggest mechanisms for this to occur to benefit everyone.

Why there needs to be open data for ultrarare and rare disease drug discovery

248th National Meeting (2014)

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