Biotech Could Miss a Multi-Billion Dollar Opportunity in Rare Disease | Keegan Johnson

March 6, 2015

Drug development is expensive.  Drug development is risky.  Drug development takes a long time.

The key to changing this is leveraging the fact that no one is more motivated to eliminate rare disease than those who are directly affected by it.

I know this because, as the founder of Give RARE, I have talked to parents of children with Tay Sachs disease who know their children will die within a few years unless something changes.  I have worked with people whose lives have been completely disrupted by their rare disease.  They are unable to work, have enormous medical bills, have difficulty maintaining social relationships, and can feel like they have stopped living a long time ago and are barely surviving from day to day.  I also know from personal experience because my son was diagnosed with a rare disease and I have experienced the transformative power of realizing there was literally no one in the world as motivated to save our children as the parents in our rare disease community.

How does that motivation help with drug development, Clinical treatment, and drug reimbursement?  It means everything.

Patient engagement can significantly reduce drug development costs, but this is just the beginning.  One of the biggest misunderstood limiting factors right now is the fear of the rare disease community.  With the right encouragement and systems, I have consistently seen people get through the initial fear and turn into $100,000 fundraisers for their rare disease community over the years.  I experienced this transformation myself.  I do not have a medical or non-profit background, but I have raised millions of dollars for rare disease research.  I have also seen first-hand the transformation in our community from fearful and not believing any change was possible to an engaged group on a mission to eliminate the challenges of Prader-Willi Syndrome.  I know we can make this transformation for the entire rare disease community a reality.

In the Art of War, Sun-Tzu writes “When your army has crossed into hostile territory, you should burn your boats, in order to make it clear to everybody you have no option of retreat” (paraphrase).  In the battle against rare disease, everybody has an option to retreat … except for the person with the rare disease.  Going into battle rare disease, without the rare disease community ‘leading’ the charge, is like playing football without a quarterback.  You are leaving your biggest weapon at home.

Think of it this way, an NFL quarterback has trained his entire life to become the best quarterback in competition.  Malcom Gladwell states we need to practice for 10,000 hours before we become an expert.  Someone with a rare disease has practiced for exactly zero hours when they get the diagnosis.

Fighting a rare disease is exactly like picking a random person out of 100,000 people and putting them in at quarterback in the Superbowl. Crazy.  The current response is to simply play without a quarterback.  We can still move the ball down the field, but it is going to be very difficult to win the big game.

I propose a different model.  I suggest we focus on turning our rare disease community into all-star quarterbacks as fast as we can.  Here is how we get started.

On March 3rd, 2015 we will host “Give Rare”, the world’s first Rare Disease Giving Day (  This movement will grow as we are joined by people, organizations, and institutions motivated to eliminate the challenges of rare disease.  Any rare disease non-profit can participate, set up a fundraising page, and compete for corporate sponsorship funds.  Rare disease groups benefit, by not only winning additional funds, but more importantly, by leveraging an existing fundraising model.  No need for each rare disease group to create the marketing, technology, training and communications necessary to raise significant funds.

The biggest winners might be the bio-tech industry.  Our first bio-tech partner, Genzyme, has been a pioneer in the rare disease space for years and was eager to help Give RARE become a reality.   Jamie Ring, Vice President, Global Patient Advocacy and Humanitarian Programs – Rare Diseases at Genzyme said, “Genzyme’s legacy of partnering with advocacy groups is rooted in finding areas of common interest.  Investing in the capacity of rare disease patient associations is something we have done throughout our 30+ year involvement in rare diseases.  Time and time again, we’ve seen that working together produces the best results for patients both in accelerating the drug development process and in helping to raise disease awareness.  We are excited to be supporting this inaugural year of Give Rare’s efforts to help further support the entire rare disease community.”

We are looking for those people truly motivated to eliminate rare disease.  If you are one of those people, I would like to ask you to join Give Rare and Genzyme as we all create this movement together.

Join now at

Buckle up … the movement is about to begin.

Thanks to Bio-Tech Now for this article.

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