“This is a great day for the Connecticut rare disease community! The Connecticut RDAC coalition would like to thank Governor Lamont, Lt. Governor Susan Bysiewicz, the Connecticut General Assembly Public Health Committee, especially Committee Chair Representative Jonathan Steinberg and Ranking Member William Petit for all their support and hard work on this legislation,” said Lesley Bennett, National Organization for Rare Disorders (NORD) Connecticut Rare Action Network Volunteer Ambassador. “This RDAC will give patients, families, caregivers, health care providers, advocates, researchers, and other stakeholders an opportunity to make formal recommendations to state agencies and our legislature on ways to develop public policy and health care legislation that will improve the lives of those impacted by a rare disease in Connecticut.”

NORD established Project RDAC in 2020 to increase the number of RDACs across the country and help optimize existing RDACs. Including Connecticut, a total of 23 states have signed legislation into law creating a Rare Disease Advisory Council, with RDAC legislation being signed into law in nine states since the start of 2021 alone.

“Sincerest appreciation to the coalition of lawmakers, patients, caregivers, and other members of the rare disease community who worked together to establish a permanent Rare Disease Advisory Council here in Connecticut,” said Peter Saltonstall, NORD President and CEO. “NORD has its roots in this state – through our founder, Abbey Meyers, and our founding nearly four decades ago. It’s incredibly empowering that Connecticut will have a permanent RDAC to help give its residents a stronger voice in their government and provide further partnership opportunities.”

Any condition that affects fewer than 200,000 Americans is considered rare. There are more than 7,000 known rare diseases, affecting 25-30 million Americans across a broad spectrum of medical conditions.  People living with rare diseases face many challenges, including delays in obtaining an accurate diagnosis, finding a health care provider with expertise in their condition, and a lack of affordable access to therapies and medications used to treat rare diseases.

For more information on NORD’s Project RDAC, visit rarediseases.org/projectrdac. To learn more about NORD’s policy work, visit: bit.ly/Policy-Issues.

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About the National Organization for Rare Disorders (NORD)

The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 300 disease-specific member organizations, more than 17,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit rarediseases.org.

The post Connecticut Establishes a Permanent Rare Disease Advisory Council appeared first on NORD (National Organization for Rare Disorders).

In addition to running, NORD’s Boston Marathon team is paired with a community partner in the rare disease space to raise money for the Undiagnosed Diseases Network (UDN).

Thank you to our Boston Marathon team:

• Jorge Beunaventura was able to earn an Abbott Medal by completing six world marathons thanks to being a part of NORD’s running team! Support Jorge’s fundraiser by clicking here.
• Lena Ceballos ran the 2021 NYC and Boston Marathons for NORD and is dedicated to running for her community partner Declan, who is three years old. Support Lena’s fundraiser by clicking here.
• Aprill Lane who runs for her son Mark who has a rare disorder. Support Aprill’s fundraiser by clicking here.
• Liri Mustafa is a college student who has partnered with Shaylee, a Pompe disease patient. Support Liri’s fundraiser by clicking here.
• Graciello Reyes is from the Phillipines and was partnered with Nicole, a PNH patient and licensed social worker. Support Graciello’s fundraiser by clicking here.
• Robert Szvizzero is a law enforcement professional who runs for his Uncle Joe who has PLS. Support Robert’s fundraiser by clicking here.
• Vivienne Wei Xiong is a scientist for Regenxbio and is also partnered with Shaylee. Support Vivienne’s fundraiser by clicking here.

Interested in Running for Rare? You can apply to be added to the 2023 NORD Running for Rare Boston Marathon waitlist. Bibs will be distributed in late fall and selected runners will be responsible for their mandatory fundraising minimum of $7,500, must wear NORD apparel while running, and be paired with a community partner. View the website to learn more.

The post The Running for Rare Team Crossed Another Finish Line appeared first on NORD (National Organization for Rare Disorders).

Peter Saltonstall, CEO and President of NORD, stated: “As negotiations continue on the Build Back Better Act, NORD urges Congress to remove this harmful provision which would gut a key incentive from the 1983 Orphan Drug Act. Attacking the Orphan Drug Tax Credit (ODTC) will result in fewer cures for the 30 million U.S. rare disease patients, the majority of whom do not have access to an FDA-approved treatment.”

“Section 138141 of the Build Back Better Act would dramatically curtail the Orphan Drug Tax Credit for qualified clinical testing expenses by removing this critical incentive for all but the first approved orphan use of a new drug. Over 90% of rare diseases lack an FDA-approved treatment indicated for the specific rare disease. Each time a new orphan use of a drug is added to the label of a drug, more rare disease patients receive assurance that the drug is safe and effective for them. The importance of FDA approval for rare disease patients cannot be understated.

“The ODTC can help to offset the cost of developing and testing orphan therapies as they move through the clinical trial process. This longstanding incentive is particularly important for the many smaller companies focused exclusively on rare diseases.

The ODTC was already diminished in 2017 in the Tax Cut and Jobs Act when Congress reduced the total amount of the tax credit for qualifying clinical testing expenses from 50% to 25%.  Given the significant time it takes to conduct clinical trials, the full impact of the changes made by the 2017 law are still unknown.  To further reduce availability of the tax credit will hurt rare disease patients and hinder their ability to access treatments found to be safe and effective to treat their specific condition.”

Take Action! Tell Congress to Protect the Orphan Drug Tax Credit Today!

About the National Organization for Rare Disorders (NORD)
The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 300 disease-specific member organizations, more than 15,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit rarediseases.org. 

The post NORD Response to New Draft of the Build Back Better Act appeared first on NORD (National Organization for Rare Disorders).

After Bryce’s first surgery, we were made aware that the mass was clear cell sarcoma and the surgeon recommended a sarcoma specialist at Washington MedStar. The sarcoma specialist then sent us to an oncology surgeon in Georgetown. The sarcoma specialist told Bryce that there was nothing that could be done, and that he would have to be on high alert for the rest of his life. This was unacceptable to us, as I’m sure it would be to any parent.

Our medical and pharmaceutical backgrounds led us to go on a mission to find out everything we could about clear cell sarcoma. For weeks, we scoured the internet for any information about the disease. It was through these searches that we found Sara’s Cure. Through a post on the Sara’s Cure Facebook page, we were made aware of the Care Oncology protocol.

Bryce is currently receiving excellent care from the sarcoma team at Johns Hopkins in Baltimore, MD. One of our main frustrations as we educated ourselves about the disease was that we found no stories of CCS in black people. In fact, one article even mentioned that CCS is predominantly in white people. Our experience with Sara’s Cure has shown us that this is not the case at all. Clear cell sarcoma does not discriminate and does not care about your ethnic background.

No parent wants to hear the word “cancer” when it comes to their child. More importantly, no person should have to hear that they or a loved one has cancer that has no treatment or cure. Our goal is to not only raise awareness that will lead to a cure for clear cell sarcoma, but to also have some type of nationwide protocol for the disease. We also want to raise awareness for people of color affected with rare diseases because CCS affects so many different communities.

The National Organization for Rare Disorders (NORD) is committed to telling the stories of patients and families with rare or undiagnosed diseases and helping them live their best rare lives. If you would like to share your story, contact NORD here.

The post Rare Cancer Day 2021: Bryce’s Clear Cell Sarcoma Story appeared first on NORD (National Organization for Rare Disorders).

1. How did you get started in the rare disease community?

I was motivated by my father who died of advanced metastatic melanoma when I was a teenager. I always wanted to make a difference in cancer since it’s very personal. The NBTS encompasses a collection of rare diseases that make up a bigger family of brain tumors. About 700,000 people are living with a primary brain tumor and a few hundred thousand more are living with metastatic cancer. I found my way to NBTS as a personal mission, as being in rare disease and cancer is a very compelling way to help people.

2. How has your experience as an attorney representing patients led you to leading the National Brain Tumor Society today?

I am an attorney with experience in public policy and nonprofit management. All the work I have done goes back to creating change for people facing life-threatening diseases, disorders, and conditions. When I saw the opportunity to join NBTS, it was based on prior experiences of helping people in different ways personally and professionally. It’s important to help and advocate for people living with brain tumors as they deserve to have a voice, and be represented.

3. How does the National Brain Tumor Society set itself apart from other related organizations?

NBTS is clear about what we are trying to do to cure brain tumors, reduce the burden of brain tumors, help individuals and families, and help the field of neurooncology. Sometimes there are differences or similarities between us and other organizations, but we try to communicate with other organizations to avoid overlap and duplication. We are passionate about honoring every donor’s dollar and making each dollar uniquely impactful. We constantly strive to deliver our mission effectively, by making sure it is on point, evidence-based, grounded in space, and keeps patients first.

4. How does the National Brain Tumor Society include diversity and health equity principles in its drug development and research initiatives?

First, we must recognize how inequities influence the whole system. Understanding the reasons for health disparities and knowing where those exist is important. We are working towards carrying out our mission in a way that is inclusive and improves equity. With all that in mind, we address how to leverage diversity and be more inclusive in our own organization internally. Externally, we focus on our fundraising and advocacy efforts. Over the last two years, we helped pass public policy that would help Medicaid-enrolled patients in clinical trials have access to coverage, since Medicaid typically leaves that coverage out and it can discriminate against low-income populations. We joined other cancer and health organizations to pass the Clinical Treatment Act (H.R.913) that became law last year. We want the community to know that NBTS is open to everyone, and that we want to help everyone regardless of any demographic. Brain tumors do not discriminate.

5. How do you remain hopeful that rare disease/cancer/brain tumor research and funding can continue to save lives?

We remain hopeful because we have looked at the track record for the last 10 years. For example, metastatic melanoma used to have very few treatment options. In the middle of the decade, the discovery was made about immuno-therapies, and we see strong survival rates as a result. We see advances across rare diseases thanks to genomics, including breaking diseases into smaller and smaller sub-diseases characterized by genetic subtype to match the right drug to the right patient at the right time. Determination, persistence, compassion, and drive to be inclusive to the people living with rare diseases must all be present. We have tried to implement these elements in our new Venture Philanthropy Fund, which injects seed capital into biotechs to incentivize them to enter the brain tumor space and partner with great medical institutions. This is a very giving society, and our cause and purpose truly make a difference in the brain tumor community.

6. In what ways does the National Brain Tumor Society highlight and amplify the patient voice in its own and unique way?

It is very important to amplify the patient’s voice, and through this we can create change. NBTS offers opportunities for the community to directly participate in advocacy and affect change. An example is the annual Head to the Hill advocacy day, which brings over 300 patients and caregivers to Capitol Hill (during the pandemic, hundreds more joined virtually) to help communicate with Congress about our public policy agenda. NBTS holds events across the country to bring between 60-100,000 people together to walk or ride together as one brain tumor community. It is also important that we help people come together and talk about their experiences. Once a month we host a large online support group which is free and has no agenda – you just come, show up, and you can bring up any topic you would like in a safe space. Speaking up and connecting with others is powerful and there are many ways to be heard through NBTS.

7. Is there a specific project or initiative in the past few years that you are particularly passionate about?

Our work over the past five years through our public policy program has resulted in 100 million more dollars coming into brain tumor research than past funding. As our advocacy program has grown, millions more have joined due to the intense focused pressure we have put on Congress and the Executive Branch to both raise the level of funding and do more with that funding. We’re proud of this accomplishment. The federal government is the largest source of brain tumor research, so that is where our policy efforts are directed. In addition to policy, we are passionate about our philanthropic programs such as Defeat GBM, Defeat Pediatric Brain Tumors, our Venture philanthropy fund, and more.

8. What’s the last book you read?

It was a gift from a friend: The Splendid and The Vile by Erik Larson. It is about Winston Churchill, the prime minister of England during some of the toughest battles of World War Two. I enjoy biographies and this felt appropriate to read in the past year as we have been under unique organizational and societal stresses with the pandemic, social unrest and injustice, and the election.

David most frequently checks his Twitter account: @dfarons

Interview by Alexandra Folleco, NORD’s 2021 summer intern for the Membership department.

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