“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).

“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).

February 15, Washington, DC – Today, the National Organization for Rare Disorders (NORD) President and CEO Peter Saltonstall issued the following statement following the confirmation of Robert Califf as Commissioner of the Food and Drug Administration (FDA): 

“NORD and the rare disease community commend the Senate for the confirmation of Robert Califf as Commissioner of the FDA. Commissioner Califf’s previous experience at FDA gives him critical first-hand knowledge of the unique challenges associated with developing safe and effective treatments for rare diseases, as well as the importance of meaningful partnerships between the FDA and rare disease patient organizations such as NORD. 

“NORD is excited to continuing to drive rare disease innovation alongside FDA and ensuring the patient voice is incorporated into regulatory decision making. More than 90% of rare diseases lack an FDA-approved treatment, and NORD is looking forward to working alongside Commissioner Califf and the FDA to further the identification, treatment, and cure of rare disorders.” 

Since its establishment in 1983, NORD has advocated for policy changes to improve the lives of Americans impacted by rare diseases at the federal and state levels. To learn more about NORD’s advocacy and policy positions, visit the NORD website. 

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 Applauds Confirmation of Robert Califf as Commissioner of the Food and Drug Administration appeared first on NORD (National Organization for Rare Disorders).

“Although NORD is encouraged by many provisions that will help improve access to and affordability of critical health programs and services for rare disease patients, the House-passed Build Back Better Act continues to include devastating changes to the Orphan Drug Tax Credit (ODTC). In a bill that would otherwise do so much good for so many Americans, it is unconscionable that Congress would target this critical incentive that has, for decades, successfully driven the development of new cures and hope for the rare disease community.”

“There are over 7,000 rare diseases and over 90% do not have an FDA-approved drug specifically used for these diseases. Thousands of NORD’s Rare Action Network advocates have contacted their legislators to share their concerns that limiting the availability of the Orphan Drug Tax Credit would jeopardize the development of new therapies for their conditions. NORD urges the Senate to remove this harmful provision when it considers the Build Back Better Act. We are committed to working together with Congress to protect individuals with rare diseases and their hope for a better future.”

NORD has helped champion several provisions included in the Build Back Better Act that would improve access and affordability of other critical health programs and services for rare disease patients. The rare community will benefit from many aspects of the House-passed bill, including: 

• A $2,000 annual cap on Medicare beneficiaries’ out-of-pocket prescription drug costs;
• A national paid family and medical leave program that would provide individuals with up to four weeks of paid leave to recover from a serious illness or care for a family member with a serious illness;
• Significant additional funding to improve patient access to home and community-based services through the Medicaid program;
• Provisions to improve access to health care coverage for people living in the 12 states that have chosen not to expand Medicaid eligibility;
• Permanent funding for the Children’s Health Insurance Program (CHIP); and
• An extension of the increased advance premium tax credits through 2025 to help make private health insurance coverage offered through the exchanges more affordable.

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’s Response to the House Passage of the Build Back Better Act appeared first on NORD (National Organization for Rare Disorders).