Legislation introduced to support access to evidence-based care for rare disease patients

rare disease patients6

October 27, 2023

WASHINGTON, D.C. – This week, U.S. Reps. Mike Kelly (R-PA-16), Congresswoman Doris Matsui (D-CA-07), Reps. Neal Dunn (R-FL-02), and Mike Thompson (D-CA-04), introduced the Providing Realistic Opportunity To Equal and Comparable Treatment for Rare (PROTECT Rare) Act. This legislation will support rare disease patients. In getting evidence-based, medically necessary care covered by their health insurance.

Over 90% of rare diseases have no FDA-approved treatment. Leaving many rare and ultra-rare disease patients facing serious and life-threatening illnesses. To rely on off-label use of drugs approved by the FDA for more common conditions.

This legislation will allow Medicare and Medicaid to use additional sources. Including peer-reviewed literature when considering whether rare disease therapies meet the standard for “medically accepted indications” to provide coverage. It will also require private payers to create an expedited review pathway. For a formulary exception, reconsideration, and/or appeal of any denial of coverage. Aimed at a drug or biological prescribed for a patient with a rare disorder.

“Modern medicine has the potential to address many of the most serious diseases we face, but too often people with rare and ultra-rare diseases don’t have the opportunity to benefit from these new cures,” said Rep. Kelly. “In some instances, there simply aren’t enough patients with a rare disease to run a full clinical trial. That’s why we need to empower doctors to make the best decisions for their patients. Using all the clinical data that is currently available. The PROTECT Rare Act will do just this. And guarantees patients have every opportunity to access life-saving cures in a way that works for them.”

“Access to the best medical care is crucial for patients living with rare and ultra-rare diseases. Yet too often these patients cannot get the evidence-based care they need,” said Congresswoman Matsui. 

“The community of rare patients deserves coverage for medically necessary treatments. In the same way, patients with more common conditions are covered by their health insurers. Our current system for determining coverage of certain drugs is limited when it comes to rare diseases. That’s why I introduced the PROTECT Rare Act. To ensure that rare and ultra-rare disease patients are no longer denied access to the care prescribed by their doctors.”

“I hear from many of my constituents who fear that restrictions on off-label drugs threaten not only their quality of life but also their chance of survival. It is important that rare disease patients have access to proven courses of treatment that include off-label uses of drugs when no other viable treatment options exist,” said Congressman Dunn, MD. “To ensure that these patients have access to appropriate care. Medicare and Medicaid must be given increased flexibility to cover such treatments. If widely available research suggests their use is appropriate.”

“Access to new and innovative treatments are essential to the health and well-being of seniors on Medicare who live with rare diseases,” said Rep. Thompson. “Too often, these people have very few options. New treatments or off-label drugs are often not covered under Medicare or Medicaid. The PROTECT Rare Act will allow Medicare and Medicaid to cover the cost of medication or treatment that doctors think would benefit their patients. If appropriate, providing flexibility to Americans living with rare diseases.”

Full text of the bill is available HERE.

Patients, as well as health care providers, physicians, and non-profit organizations, are applauding the introduction of this legislation. Which will have a huge impact on their daily lives.

“Although there is only one FDA-approved therapy for my rare condition, pemphigus, there are several other off-label drugs that are used as standard of care for patients like me,” said Janet Segall, a constituent of Congresswoman Matsui. “Unfortunately, the off-label treatments are not covered. Also, there isn’t even an appeal process that’s easily accessible. Even though my doctors tell me these treatments are standard of care for my disease.

In the meantime, all the delays make my symptoms worse, and cause a lot of pain. And make things nearly impossible financially. The PROTECT Rare Act will give rare disease patients like me access to treatments. Treatments that my doctors think are best for me. Doctors consider these treatments medically acceptable for pemphigus. This will help me and so many other patients. So thank you for tackling this important issue for all of us. Living with a rare disease is hard enough without having to be denied access to the treatments I need.”

“We look forward to the enactment of this important legislation, and for the work the sponsors have put into this so far,” said Chevese Turner, CEO of the Haystack Project. “We are confident that this will make a significant difference in the lives of the rare disease patients we represent and work with every day.

“No disease is too rare to deserve treatment. Yet 95% of rare diseases lack a single FDA-approved product. Forcing patients to rely on treatments approved in other diseases to alleviate their symptoms,” said Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement of the EveryLife Foundation for Rare Diseases.

“Regrettably, insurers often obstruct access to these vital, life-sustaining treatments, even when they align with clinical guidelines. Further exacerbating the impact of living with a rare disease as patients and their families already bear a significant share of the nearly $1 trillion annual economic impact of rare diseases in the U.S. The EveryLife Foundation for Rare Diseases extends our heartfelt gratitude to Representatives Matsui and Dunn for championing the PROTECT Rare Act, a crucial step toward delivering affordable treatments to those who need them most. We look forward to Congress acting on this important legislation.”

“More than 95% of rare diseases lack an FDA-approved therapy. This means that rare disease patients are frequently prescribed therapies off-label by their trusted health care providers,” said Peter L. Saltonstall, CEO of the National Organization for Rare Disorders. “Unfortunately, often the off-label use of therapies is not covered by health insurance, creating costly barriers to necessary treatments. The PROTECT Rare Act would help those living with a rare disease obtain access to orphan drugs. By allowing clinical guidelines and peer-reviewed literature to be used to assess coverage for treatments for rare diseases. This legislation will help more rare disease patients obtain timely coverage for much-needed therapies. And NORD applauds Rep. Matsui for her leadership to support rare disease patients.”

“Over 90 percent of the over 10,000 known rare diseases still have no FDA-approved therapy,” said Jenifer Waldrop, Executive Director of the Rare Disease Diversity Council. 

“For the more than 30 million Americans living with a rare disease off-label drug use is often the only treatment option. This is especially burdensome for patients in underserved communities, who may lack the resources to either pay out-of-pocket for needed therapies or navigate the health system to secure coverage. For rare disease patients impacted by social determinants of health, these barriers are compounded. Greater access to off-label treatments will reduce burdens of diseases, improve long-term prospects, and save lives.”

The following entities endorse this legislation:

Haystack Project, Everylife Foundation for Rare Diseases, National Organization for Rare Disorders, Rare Disease Diversity Council, 5P Minus Society, Alpha-1 Foundation, American Brian Coalition, Arachnoiditis Chronic Meningitis Research Network, Association for Creatine Deficiencies, Autoinflammatory Alliance, Barth Syndrome Foundation, Biomarker Collaborative, Cares Foundation, Children’s PSC Foundation, Choroideremia Research Foundation, CLL Society, CSNK2A1 Foundation, CRI DU CHAT Research Foundation, Cutaneous Lymphoma Foundation, Danny’s Dose, DEE-P Connections, Desmoid Tumor Research Foundation, Dup15Q Alliance, FACES The National Craniofacial Association,

Galactosemia Foundation, Hairy Cell Leukemia Foundation, HCU Network America, Healthtree Foundation, Hermansky-Pudlak Syndrome Inc., Histiocytosis Association, Hope for Stomach Cancer, International Cancer Advocacy Network, International Fibrodysplasia Ossificans Progressiva Association, International Foundation for CDKL5 Research, International Pemphigus Pemphigoid Foundation, International SCN8A Alliance, LGS Foundation, MET Crusaders, MitoAction, MLD Foundation, Myasthenia Gravis Foundation of America, National Ataxia Foundation, NBIA Disorders Association, No Stomach for Cancer, NTM Info & Research, Organic Acidemia Association, PDL1 Amplified, Rare and Black, SADS Foundation, Share and Care Cockayne Syndrome Network, TEAM for Travis, The Exon 20 Group, The Foundation for Casey’s Cure, The Global Foundation for Peroxisomal Disorders, The Mast Cell Disease Society, Inc., TSC Alliance, United Mitochondrial Disease Foundation, United Porphyrias Association, Usher 1F Collaborative, VHL Alliance.

We would like to thank Every Life Foundation, for sharing this educational story with us.  Please share your thoughts and/or comments on this or any other article.  If you would like to get involved and share your experience with Ataxia, please get in contact with us and join our community today.  A place where we empower you to build a healthy lifestyle and raise overdue Ataxia Awareness.  Experience transformative storytelling and share your story to inspire positive change.


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