BILL TO CREATE “RARE DISEASE DAY” IN MASSACHUSETTS PASSES LEGISLATURE, SIGNED BY GOVERNOR
(Boston–2/6/2023) The Massachusetts State Legislature has passed legislation filed by Senator Paul R. Feeney and Representative Brian M. Ashe to establish a “Rare Disease Day” in Massachusetts.
S.2058/ H.3101, “An Act designating the last day of February as rare disease day in the Commonwealth,” designates the last day of February as “Rare Disease Day” in the Commonwealth and recommends annual observance by the public to increase public awareness of rare diseases not only by the people, but also to recognize the impact on medical professionals, researchers, educators and others who serve the rare disease community.
"Rare diseases affect millions of Americans each year and present special struggles to patients and their families, an added burden to the challenges they already face such as the lack of access to needed therapies and treatments. With the passage of this bill and the establishment of "Rare Disease Day", we have the opportunity to raise awareness of the unique challenges faced by patients, families and medical professionals in the Rare Disease community," said Senator Paul Feeney (D-Foxborough). “Thank you to the incredible Rare Disease advocates and constituents that have shared their personal stories of struggle and perseverance with me and my staff over the years. And thank you to House bill sponsor, State Representative Brian Ashe, for partnering with me to bring this bill to the finish line.”
"Having Rare Disease Day officially recognized by the Commonwealth will go a long way to bring awareness, research, and hopefully cures to some of these diseases. Just because a disease is rare doesn't make it any less debilitating or important than one more familiar. This has been a passion of mine since I first filed it ten years ago and couldn't be more proud. Thank you to my counterpart in the Senate, Senator Paul Feeney (D-Foxborough) as well as the many individuals and groups for their continued support and advocacy. This was truly a team effort,” said Representative Brian Ashe (D- Longmeadow).
"On behalf of the US rare disease community and the National Organization for Rare Disorders (NORD), we are very grateful to Senator Feeney and Representative Ashe for championing legislation to officially recognize February 28th as Rare Disease Day in Massachusetts. It is likely that every Massachusetts resident has a personal connection to someone who is living with a rare disease. Rare Disease Day is a fantastic opportunity to raise awareness and support for a more equitable future in which all Americans, including the more than 25 million Americans living with a rare disease, have access to the care and treatment they need to thrive,” said Peter Saltonstall, President and CEO, NORD.
“Rare New England is beyond thrilled for the passing of the bill dedicating February 28th as Rare Disease Day in Massachusetts. This wonderful recognition will promote acknowledgement and increase support and advocacy efforts for such an underserved community. With an already challenged healthcare system, rare disease patients and families face daily obstacles as they search for diagnosis and scarce treatments to improve quality of lives. Often feeling isolated, patients and families often rely on hope, and lean on their communities through their diagnostic journeys. Rare New England hopes that every year we can increase public engagement to help the rare disease community thrive,” said Nicole White, Executive Director, Rare New England and Julie Gortze, President, Rare New England.
“MassBio applauds Senator Feeney, Representative Ashe, and the Legislature for making Rare Disease Day official in Massachusetts, a move that will bring more awareness to those living with a rare disease, and the researchers working to discover treatments and cures,” said Kendalle Burlin O’Connell, CEO and President of MassBio. “Each year MassBio hosts an event at the Statehouse to bring attention to the groundbreaking, patient-focused research happening in Massachusetts and recognize the organizations, patients, and families who are bravely telling their stories and advocating for policies that promote access and advance research.”
“Rare diseases are not that rare. One in every 10 people is affected by rare disease – half of whom are children, and fewer than 10% of rare diseases have an FDA-approved treatment. Celebrating Rare Disease Day each year renews hope for these patients and their loved ones and reminds them they are not forgotten. Thank you to the legislators, patient advocates, researchers, and leaders across Massachusetts for shining a light on the rare disease community; together we can accelerate the development of effective therapies to transform the lives of people living with rare disease,” said Emil D. Kakkis, M.D., Ph.D., founder and CEO of Ultragenyx
In the United States, rare diseases are classified as diseases that affect fewer than 20,000 people. According to the National Institute of Health, there are over 6,500 known rare diseases affecting the lives of approximately one out of every ten Americans. Rare diseases are notoriously hard to diagnose meaning that patients often face years of incorrect diagnoses and wrong treatment plans before arriving at the correct diagnosis, if ever.
This long path to a correct diagnosis can greatly reduce the patient's quality of life and result in long-term disability, difficulty accessing accommodations and resources, large undue medical expenses and more. The creation of Rare Disease Day and annual observance by the public will bring attention to this often-overlooked community to bring their experiences to the forefront and provide support and resources to patients still searching for answers.
In the words of the National Organization for Rare Disorders, “when you hear hoof beats, it isn't always a horse. Sometimes, it's a zebra."
“An Act designating the last day of February as rare disease day in the Commonwealth” was signed into law by Governor Baker on 12/28/22 as Chapter 328 of the Acts of 2022.
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