Why iBIO Supports Rare Disease Day

Today is rare disease day, an annual event on the last day of February. It’s a day to raise awareness for rare diseases and the challenges faced by people who live with them. Rare diseases are diseases or conditions that affect fewer than 200,000 people, and while each rare disease may only affect a small number of people, collectively, they impact many. In total rare diseases affect 30 million Americans, that’s one in ten Americans. Rare diseases are often difficult to diagnose and treat and can significantly impact public health.


That is why iBIO and our member companies are passionate about supporting patients and families in fighting Rare Diseases. iBIO has made it part of our mission to support Rare Disease efforts in Illinois. We are helping to raise awareness with policymakers and the public while advocating for policies like creating the Illinois Rare Diseases Commission that gives rare disease patients a voice in Springfield and now improving access and support for patients in clinical trials in Illinois.

Innovation in our industry is critical for rare disease patients. 95% of the identified 7,000 rare diseases do not have an FDA-approved medicine. 85% to 90% of rare diseases are critical or life-threatening and disproportionately impact children (50%). Gene therapy is particularly relevant to rare disease patients, as over 80 percent of rare diseases have a known monogenic (single-gene) cause. That is why we must fight for policies that support innovation for patients, like increasing funding for research, strengthening intellectual property protections, supporting a science-based regulatory system, and implementing coverage and payment policies that support and encourage medical innovation.

During the Annual Rare Disease Event in Springfield, I met patients, and I saw the immense impact that innovation by iBIO’s members like Horizon, Astellas, Xeris, Jaguar Gene Therapy, Novartis, Amgen, Biogen, Genentech, Ultragenyx, Vertex, and so many more companies have on rare disease patients and their families. That is why we shared stories like the ones below.

The Power of a Clinical Trial

Successful therapy for their daughter leads a couple to advocate for the next generation of children By Jed Weiner, iBIO News Brief Contributor “Elizabeth was like a sound machine that helped her brother sleep. Her labored, nasally breathing was constant. But one...

Life-saving drug gives hope to a young girl – and her parents

By Jed Weiner iBIO News Brief Contributor One of iBIO’s legislative priorities in 2019 is the passage of Illinois House Bill 2259. If signed into law, HB2259 would establish panels of experts to support the needs of the state’s Drug and Therapeutic Advisory Board...

This year, iBIO is hosting the Illinois Rare Disease Day at the Capitol on March 9th at the Illinois State Library. It is a great event, bringing together rare disease advocates from across the state to make their voices heard by their Illinois elected officials. Participants are educated on policy proposals impacting the rare disease community and provided opportunities to advocate for policy changes directly to their state elected officials.

I hope you can join iBIO and the Rare Disease community in Springfield next week, and if you cannot, I hope you take time today to learn more about Rare diseases and Rare Disease Day and find a way to support the Rare Disease Community.

John Conrad
iBIO President & CEO



2023 Illinois Rare Disease Day at the Capitol