Rep. Mike Kelly Introduces Bipartisan Legislation to Support Access to Treatments for Rare Disease Patients

Statement

Today, Rep. Mike Kelly (R-PA) along with Reps. Doris Matsui (D-CA), Markwayne Mullin (R-OK) and Mike Thompson (D-CA), announced the introduction of Access to Rare Indications Act of 2021, to support patients with rare and ultra-rare diseases 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 illness to rely on off-label use of drugs approved by the FDA for more common conditions. In addition to the FDA-approved label, this legislation will allow Medicare and Medicaid to use additional sources including compendia, peer-reviewed literature and opinion of rare disease experts to meet medically accepted criteria that ensure coverage for rare disease treatment. It will also require private payers to create an expedited review pathway for formulary exception, reconsideration, and/or appeal of any denial of coverage for 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 decision for their patients, using all the clinical data which is currently available. The Access to Rare Indications 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, and too often these patients cannot get the evidence-based care they need," said Congresswoman Matsui. "The community of rare patients deserve access to medically necessary treatments, 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 our rare diseases. That's why I introduced this bill to ensure that rare and ultra-rare patients are no longer denied access to the care prescribed by their doctors."

"Rare conditions are labeled "rare' for that very reason -- that there are indeed so few patients with these conditions, that as a result they are forced to seek alternative treatments aside from those deemed "standard,'" said Rep. Mullin. "Every American deserves the right to quality care, especially those walking a road seldom traveled."

"Constituents have told me that expanding access to treatment for rare diseases is going to make a big difference for patients in our district and nationwide," said Rep. Thompson. "That's why I'm proud to be a lead coauthor of the Access to Rare Indications Act that will improve access to treatment and lower costs for patients living with rare disease."

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

"I live in Erie, PA and not only have a very rare condition, but an atypical form of it," said Mark P., a constituent of Congressman Kelly. "Because I present with different symptoms, the diagnostic process has taken nearly 10 years, causing a significant financial and emotional burden. Worst of all, there is no cure or even effective treatment for my condition. Legislation like the Access to Rare Indications Act is critical for our community of rare patients, and we thank you for your leadership here."

Another constituent of Congressman Kelly, Vivesh P., said "I was diagnosed with a rare autoimmune blistering skin disease called Mucous Membrane Pemphigoid in 2017 when I was just 14 years old. Unfortunately, there is no FDA approved therapy for me. There are off-label drugs being used to treat this disease, but it's nearly impossible to get them. Even though they are prescribed by my doctor and they're commonly used for Pemphigoid, and there is evidence and studies and peer-reviewed journal publications showing the treatments are beneficial. Rare disease patients like me are experiencing unnecessary delays in life-changing treatments that cause worsening of symptoms, physical and mental pain and a difficult quality of life. The Access to Rare Indications Act will give rare disease patients like me access to needed therapies that work, are safe and effective. Being diagnosed with a rare disease at such a young age was hard enough but being denied access to life-saving treatments shouldn't be such a challenge."

"We look forward to the enactment of this important legislation so that our community of rare patients can get the treatments their providers think best for them," said Deanna Darlington, the incoming Haystack Project CEO.

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