“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).

“Catalyst Pharms., Inc. v. Becerra threatens to undermine 40 years of practice and progress enabled by the Orphan Drug Act, which has benefited millions of Americans and their families facing rare disease diagnoses,” said Peter L. Saltonstall, President and CEO, NORD. “Still, there are approximately 25-30 million Americans living with a rare disease and more than 90% of the 7,000 known rare diseases do not yet have an FDA-approved treatment. We are very encouraged by the legislation in both the House and Senate to clarify that orphan drug exclusivity is awarded only for the specific use that is approved by the FDA. This clarification will maintain appropriate incentives to encourage rare disease drug development as intended in the Orphan Drug Act with a goal of addressing the still significant unmet needs of many in the rare disease community.” 

The ODA provides a set of incentives to support research and development into drugs for rare diseases, which had been largely neglected by the pharmaceutical industry before the 1983 law. One of the key incentives is a seven-year term of “exclusivity,” or market protection from competition, for the orphan drug once approved and marketed. The law established a two-part process for obtaining orphan drug exclusivity. First, at an early stage in development, a company can request that FDA “designate” the drug as an orphan drug to prevent, diagnose or treat a rare disease or condition. Once a company receives this designation, the company can access other ODA incentives, including tax credits for the research and clinical testing on the drug. Second, after completing the necessary clinical studies and obtaining FDA approval, the drug is then awarded exclusivity that protects the specific use of the drug that is approved. 

However, the recent 11th Circuit decision in the case of Catalyst Pharms., Inc. v. Becerra, if allowed to stand, would turn this system on its head. The Court rejected FDA’s decades-long interpretation of the ODA that the exclusivity protects the “use or indication” ultimately approved. The Court instead held that the rare disease designated at the outset of the drug development process dictates the scope of the orphan drug exclusivity. NORD believes this is an incorrect interpretation of the statute and is grateful to see Congress working to correct and clarify the law.  

In the absence of a legislative fix, NORD believes there would be fewer orphan drugs approved for fewer special patient populations. That is not the goal of the ODA, and it is not in the best interest of the rare disease community. In most cases, the orphan designation is intentionally broader than the use ultimately approved. For instance, a drug might be designated for the treatment of Fabry’s disease, a rare lysosomal storage disorder. After conducting studies in the disease, the sponsor may have only obtained data sufficient to support a narrower population than the entire patient population with Fabry’s disease and seek approval for the treatment of only adults with the disease. Similarly, many orphan drugs designated for cystic fibrosis are designated for the disease broadly but are only approved for use in patients with a specific mutation.  

If the 11th Circuit decision is left in place, the law would create an incentive for sponsors to seek broader designations for an entire rare disease at the outset, leaving little incentive to continue to study the safety and efficacy that drug in special populations, like children. More than half of people with rare diseases are children, so the implications of this Court ruling have the potential to be significant. 

“NORD is grateful that Energy and Commerce Committee leadership and Senators Baldwin and Cassidy have recognized the threat posed by the 11th Circuit’s interpretation of the Orphan Drug Act,” said Heidi Ross, Vice President, Policy and Regulatory Affairs, NORD. “This issue must be addressed swiftly to ensure access to safe and effective drugs for specific rare disease populations is maintained and future research and development efforts for new treatments for rare diseases are appropriately incentivized.”

To learn more about NORD’s policy work and how to get involved, visit: http://bit.ly/Policy-Issues. 

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 NORD Applauds Congressional Efforts to Restore Intent of the Orphan Drug Act appeared first on NORD (National Organization for Rare Disorders).

May 6, 2022, Atlanta, GA – This week, Governor Brian Kemp signed legislation into law to establish a Rare Disease Advisory Council (RDAC) in Georgia. This advisory body will be comprised of a variety of stakeholders including patients, caregivers, health care providers, researchers, and patient advocacy organizations and will advise the Georgia General Assembly and other state agencies and departments on the needs of individuals with rare diseases living in Georgia. The RDAC will be tasked with activities such as conducting surveys to better understand common challenges rare disease patients or caregivers face, consulting with experts to improve access to quality health care, and compiling resources related to rare diseases.

There are more than 7,000 known rare diseases affecting approximately 25-30 million Americans, more than half of which are children. People 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.

“Tuesday marked an incredible milestone for the rare community in Georgia. I would like to extend my heartfelt thanks to Governor Kemp who signed the Rare Disease Advisory Council legislation into law. It is truly an honor to serve and advocate on behalf of children and adults with rare and complex diseases,” said Beth Nguyen, RN, the National Organization for Rare Disorders (NORD) Georgia Rare Action Network Volunteer State Ambassador.

“This newly established RDAC will provide a platform for the rare community to have a stronger voice in the state of Georgia. These advocate-built councils are a great display of community engagement and partnership between families and the government. NORD would like to express our appreciation to Representative Mike Cheokas for being a champion for Georgia’s rare disease community by sponsoring this important legislation and ensuring it got over the finish line,” said Heidi Ross, Vice President, Policy and Regulatory Affairs, NORD.

NORD launched a new initiative, Project RDAC, in 2020 with the goal of increasing the number of RDACs across the country and helping to optimize existing RDACs. A total of 21 states have signed legislation into law creating a Rare Disease Advisory Council, including six in 2021 alone.

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

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 Georgia Becomes 22nd State to Establish a Rare Disease Advisory Council appeared first on NORD (National Organization for Rare Disorders).

“Over his many years in Congress, Senator Hatch demonstrated his commitment to public health and his concern for the millions of Americans affected by rare diseases through his leadership on numerous issues,” said Peter L. Saltonstall, President and CEO, NORD. “This includes the key role he played in shepherding the Orphan Drug Act through the Senate in the early 1980s to encourage development of treatments for rare diseases. He recognized, at a time when few others did, the crisis for individuals with rare diseases who had no hope of a treatment or cure.”   

In grateful appreciation of Senator Hatch’s support for the 30 million Americans affected by rare diseases, NORD honored him in 1987 and again in 2016 with its National Health Leadership Award.  

In addition to Senator Hatch’s leadership on the Orphan Drug Act, his many important contributions over the years to public health and support for those with rare diseases have included:  

• In 2000, Senator Hatch and Senator Ted Kennedy introduced the Rare Diseases Act, for which NORD advocated in support of, to enhance federal funding for rare disease research and accelerate the development of treatments. The legislation ultimately was split into two bills – the Rare Diseases Act and the Orphan Products Development Act.  
• In 2002, the Rare Diseases Act was signed into law to codify the NIH Office of Rare Diseases Research and provide for the establishment of the NIH Rare Diseases Clinical Research Network with $55 million in funding for rare disease research.  
• In 2015, Senator Hatch was named co-chair of the Rare Disease Congressional Caucus.  
• In 2016, he was a co-sponsor of the OPEN Act, legislation that was introduced to provide incentives for companies to “repurpose” previously approved drugs for the treatment of rare diseases. 

To learn more about NORD’s policy work, visit: http://bit.ly/Policy-Issues. 

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 NORD Pays Tribute to Life and Service of Senator Orrin Hatch appeared first on NORD (National Organization for Rare Disorders).

April 8, 2022, Washington, DC – This week, the National Organization for Rare Disorders (NORD), along with 90 other patient organizations sent letters to Health and Human Services (HHS) Secretary Xavier Becerra and Congressional leaders urging them to seize opportunities before them to protect patient access and strengthen the accelerated approval pathway.  

One letter is directed toward Congressional leadership urging them to incorporate into legislation to reauthorize the Prescription Drug User Fee Act (PDUFA) several policy recommendations to strengthen the accelerated approval.

Another letter is directed to HHS Secretary Becerra, urging him to support the efforts underway within Congress and FDA to strengthen the accelerated approval pathway and reject policies that could limit patient access to rare disease and other therapies that utilize accelerated approval. 

“Accelerated approval is vital to patients with rare diseases and other life-threatening medical conditions where no treatment currently exists,” said Heidi Ross, Acting Vice President, Policy and Regulatory Affairs, NORD. “However, NORD and the other 90 co-signing organizations believe reforms are necessary to help ensure patients have timely access to rare disease treatments that have been FDA approved through the accelerated approval pathway. NORD looks forward to working with Congress and HHS to ensure changes to the accelerated approval pathway ultimately benefit the rare disease community.” 

To learn more about NORD’s policy work, visit: http://bit.ly/Policy-Issues. 

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 ICYMI: Rare Disease Community Takes Action to Strengthen and Reform Accelerated Approval  appeared first on NORD (National Organization for Rare Disorders).