My Anniversary Wish for the Rare Disease Community

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Dear Friends, 

Happy anniversary to us, the rare disease community. Forty years ago today marked a turning point for people living with rare diseases and their families. The Orphan Drug Act became law on this day in 1983, creating the world’s first law incentivizing drug development for rare diseases. Forty years ago, there were fewer than 40 rare disease treatments; today, more than 1,100 have been FDA approved. 

The existence of the Orphan Drug Act proves our community has the power to change history. The law came to be because an impromptu group of individuals dealing with rare diseases in their personal lives urged Congress to help; they and their loved ones were suffering, there were no treatments, and none were on the horizon in the US. Their advocacy grew into a grassroots movement. Legislators listened, held hearings, and passed legislation. President Reagan signed the bill into law. The Orphan Drug Act has been part of every rare disease breakthrough over the past 40 years and will continue to do so in the future. 

On this special day for our community, we thank the legislators, past and present, who take note and help, the companies and researchers who deliver innovations, and we are continually indebted to the life-changing work of the first group of parent and patient advocates who put our community of 25 million Americans on their backs and would not take no for an answer. When they decided their work was not done, they formed NORD, building the first organization to foster belonging and support for all rare diseases and a platform for awareness and collective impact. We are grateful for the dedication and passion of the patients, caregivers and their loved ones, advocates, volunteers, supporters, and partners who have been with us since. 

That is only a piece of our story so far. The other piece is you. Thank you. You are a part of our community. You are why the next treatment will be approved, why a clinical trial will begin, why a doctor or medical student will have awareness to suspect a rare disease and order the diagnostic test, and why someone in our community feels a little bit less alone.  

Your impact does not stop there. It is at the core of our culture to speak up, step up, and not wait for someone else to do the hard work. We refuse to back away because something is hard or has not been tried; we would not exist otherwise. We have so much more work to do, and that is why today I urge you to make your voice heard. Every member of the rare disease community has the power to shape the future and make a lasting impact. Let us pause, in this new year and on this important day in our history, to envision the next ten years. We want to hear from you on what you reimagine is possible for yourself, your loved one, and the entire community affected by rare disease. What would make a difference in your rare journey or for those in your life? What change would you like to see for yourself, or for all rare diseases? Share your vision and ideas today as your gift to our community.  

Through the decades, we’ve shown by coming together we can address tough challenges and create change. Let us use this anniversary year to boldly reimagine the future, because people living with rare diseases do not have more time to wait. I look forward to your response and gathering throughout the year to celebrate and forge our next era of progress. 

Your partner in rare disease, 

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Peter L. Saltonstall
President and Chief Executive Officer