Alone we are Rare. Together we are strong. This motto exemplifies the mindset of the rare disease community and is as true today as it was 40 years ago. The deep bond created by a shared sense of purpose was palpable when rare disease advocates from around the country assembled on Capitol Hill last night to celebrate the 40th Anniversary of the Orphan Drug Act, building on a long and successful history of partnership to bring life-changing therapies to patients living with rare diseases.
40 years ago, a small, dedicated group of rare disease patients and caregivers, led by NORD’s founder, Abbey Meyers, drew upon their passion, determination, and creativity to change the future for rare disease patients. Last night, before a packed room, Kyle Bryant, a rare disease patient and advocate, movingly shared his journey as someone living with Friedreich’s Ataxia. He discussed how his diagnosis eventually led him to start a wildly successful bicycle ride fundraiser, rideAtaxia, which has raised awareness and $11 million for research into the disease. Kyle also shared what the prospect of an effective treatment means to patients like him and their families saying, “Without the Orphan Drug Act, who knows if the incentives would be in place to have taken us to this point. This first treatment represents so much hope for my community, but thanks in part to the Orphan Drug Act, it is just the first of several therapies currently in development.” That first treatment for patients with Friedreich’s ataxia is now before the FDA, with a decision expected by February 28th – so fittingly, Rare Disease Day.
In their remarks, Congressman Gus Bilirakis (R-FL) and Congresswoman Doris Matusi (D-CA), Co-Chairs of the Rare Disease Congressional Caucus, both emphasized the long history of broad bipartisan support for rare disease policy, and reinforced their commitment to help break down barriers for rare disease patients, including the long diagnostic odyssey and timely access to care.
The lead sponsor of the Orphan Drug Act, retired Congressman Henry Waxman, provided virtual remarks looking back at how a constituent impacted by a rare diseases first made him aware of the market failures leading to so few rare disease therapies and highlighted how thanks to the Orphan Drug Act, and the continued work of many rare disease champions and advocates, more than 600 drugs have been approved to treat over 1100 rare indications since 1983.
NORD was proud to co-host this reception honoring the 40th Anniversary of the Orphan Drug Act with Alexion, AstraZeneca Rare Disease. In their remarks, Peter Saltonstall, President and CEO of NORD, and Scott Weintraub, SVP and Head of US Commercial Operations at Alexion, AstraZeneca Rare Disease celebrated our community’s accomplishments, but also looked to the future, knowing there is still so much more work that needs to be done. It still takes a rare disease patient an average of 5-7 years to get an accurate diagnosis; of the 7000 known rare diseases, more than 90% do not have an FDA approved therapy; and many rare disease patients struggle to access affordable treatment options.
As Peter Saltonstall, NORD’s President and CEO said, “We work to see a world where rare disease patients have access to the services and therapies they need to thrive. Together, we will make this dream a reality.”
Thank you to the rare disease community for coming to Capitol Hill to celebrate together last night, and for giving rare diseases a voice every day!
