Realities of Rare Disease and the Role of Patient Investors

December 20, 2016

For the EBD Group, Guest post by Nicole Boice, Founder & CEO, Global Genes – Allies in Rare Disease

Rare Disease represents the largest disease community on the planet!  With over 7,000 identified diseases, impacting over 350 million people worldwide and 1 in 10 Americans, the impact on our global population is real.  Today there are no cures and less than 5% of the 7,000 diseases have a treatment or effective therapy; it is clear that there are needs on both the business and human level.

When presented with the not-so-encouraging stats and facts, Patient and Parent advocates have had to become Change Agents for their disease communities. They have become drivers and innovators in the quest for cures.  Today, we are seeing a paradigm shift with patients and their roles within the drug development lifecycle. Patient Activists have begun identifying promising science, have given seed funding to early investigators, have creatively built virtual biotechs, have created venture funds and have become very savvy investors and biotech executives. The momentum has increased as Patient Activists have seen increasing success; early investors turned biotech CEOs like John Crowley, CEO of Amicus and Brad Margus, CEO of Cerevance have paved the way for others like Ilan Ganot, CEO, Solid Biosciences.

Rare Disease has become a validated segment and area of investment within the life sciences.  It is a very encouraging time within rare disease, and Global Genes continues to try and build programs to impact and educate patients so that they can continue to be drivers and architects of their health and the health of the diseases communities that they care about. The reality is that Patients have become very active investors, and for those that have seen success, they are paving the way for others. To help, Global Genes continues to develop partnerships and collaborations that will help advance these efforts and impact for all of those in the rare disease fight.


Join Global Genes for a unique partnering opportunity for rare disease innovators at “Rare in the Square (RITS)” event taking place January 9–11, 2017, in San Francisco’s Union Square. The inaugural RITS will coincide with the JP Morgan Healthcare Conference (JPM), creating the opportunity for rare disease focused companies and organizations to network at the epicenter of JPM-related activities in a unique, one-of-a-kind space.


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