A message from Eddie Pauline, President & CEO
A mission that matters: Improving and prolonging life for those with rare diseases
It can be tough amidst the day-to-day responsibilities to keep sight of the “why.” Why we are doing the work, why it matters, and whether it aligns with our goals and values. But sometimes, we get to see and reflect on how our industry’s work changes and, in many cases, saves lives. This is one of those times.
Today, Feb. 28, is Rare Disease Day, an internationally recognized day that raises awareness about these often-misdiagnosed diseases. Our industry is deeply connected with people who have rare diseases. Researchers work on therapies to treat those affected, and providers care for them. Our membership includes nonprofit organizations that support individuals and families impacted by rare diseases, and OLS advocates for legislation and policies that improve access to and affordability of life-saving treatments.
Talking with parents of children who have rare diseases is one of the most inspiring things I’ve been able to do in my time as president and CEO of OLS. It drives home for me the reason why this work — our work — matters so much. Without research, without advocacy, without manufacturers, without care providers, many of these children would not survive.
I was reminded of that recently when I joined leaders from Forge Biologics and Sarepta Therapeutics to present in front of the Ohio Rare Disease Advisory Council, a group of 31 people appointed by Gov. Mike DeWine, the Speaker of the Ohio House and the Ohio Senate President to advise the Ohio General Assembly on rare disease research, diagnosis, and treatment efforts across Ohio.
Forge and Sarepta, like many of our member organizations, are working to develop and manufacture gene and cell therapies that target the precise genes responsible for rare diseases. This is important: Rare diseases are, by their nature, difficult to diagnose, and patients are, in fact, often misdiagnosed. And they are hard to treat. In many cases, no therapies are even available.
Rare diseases affect some 300 million people around the world, but the individual diseases themselves are often extremely rare. Any disease affecting fewer than 200,000 people in the U.S. is considered rare. That means rare diseases often do not have the same coordinated, critical-mass advocacy efforts that help fund and drive research that leads to treatments.
This is where trade organizations like OLS can and must play a crucial role.
A strong trade organization is a unifying voice that brings together researchers, treatment manufacturers, medical device creators, business leaders, families, advocates, and patients to push for financing, policies, and legislation that makes it easier and more affordable to develop treatments. Two great examples are the Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs, federal initiatives that provide important seed funding for research. Both were in danger of not being renewed, but OLS, alongside other life sciences organizations and our national trade organization, BIO, advocated for its renewal — and we were successful.
We need a strong trade organization so the voice of the life sciences industry, with all its many facets, is not lost in the ocean of other voices clamoring for the attention of people in power.
I am biased, to be sure, but I think our industry has a reason to be loud here in Ohio, and not just because it is our industry. Not only are we growing rapidly and creating excellent jobs for Ohioans, but we are also developing and disseminating the treatments that save people’s lives. Those are missions I think all Ohioans should get behind.
As always, I would welcome your thoughts. In the meantime, I hope you will take time today to learn more about rare diseases affecting people across the planet — and the therapies we are developing here that might one day save their lives.
Eddie Pauline
President & CEO
Ohio Life Sciences