Realities of Rare Disease and the Role of Patient Investors

December 16, 2016 Guest Contributor

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.

For more information about Global Genes – Allies in Rare Disease, head to


EBD Group is committed to the rare disease community: To further partnering and collaboration opportunities between leaders from patient advocacy groups, industry, academia and government, the inaugural Cell & Gene Exchange event will be held in Washington, DC May 22–23, 2017. Companies and patient advocacy groups specializing in the cell and gene therapy sector will be gathering to network, exchange ideas, and forge meaningful partnerships to advance the cell and gene therapy sector. Find out more.


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