U.S. Rep. Mike Kelly, R-16th, and a number of other representatives have introduced the Access to Rare Indications Act of 2021, which will help patients with rare and ultra-rare diseases receive necessary, evidence-based care and have it be covered by insurance.

The legislation will allow Medicare and Medicaid to use sources such as peer-reviewed literature and rare-disease expert opinion to satisfy medical standards and ensure coverage for treatment of the disease.

“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,” said Kelly. “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.”