Rare Daily Staff

A bipartisan group of Congressional representatives introduced legislation they said would provide support to patients with rare and ultra-rare diseases getting evidence-based, medically necessary care covered by their health insurance.

Representatives Mike Kelly (R-Pennsylvania), Doris Matsui (D-California), Markwayne Mullin (R-Oklahoma), and Mike Thompson (D-California), introduced the Access to Rare Indications Act of 2021.

More than 90 percent 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.”

Rep. Mullin said that rare conditions are labeled “rare” for a reason. Because of that, they are often forced to seeks alternative treatments aside from those deemed “standard.” “Every American deserves the right to quality care, especially those walking a road seldom traveled,” she said.

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