WASHINGTON, D.C. – Congressman Neal Dunn, MD (Florida-02) joined Congresswoman Doris Matsui (California-07), Congressman Mike Thompson (California-04), and Congressman Mike Kelly (Pennsylvania-16) in introducing the PROTECT Rare Act, a bill that will allow Medicare Part D and Medicaid to cover drugs for off-label use if there is peer-reviewed medical literature that indicates its use is appropriate in the treatment of a rare disease. This bill would also require private insurance plans to create an expedited review process for coverage requests for the off-label use of a drug for a rare disease.

“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. “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.”

Because rare diseases impact a very small patient population, there is low incentive to develop cures; however, medical professionals and researchers often can identify other drugs or drug combinations that are effective in treating the disease. Unfortunately, Medicare and Medicaid will not cover those.

“When I needed my off-label drug for Immune Thrombocytopenia, I had to jump through several hoops. Insurance kept denying the treatment, not even sending it to the proper third-party reviewers. They kept trying to push me to take IVIg infusions every other week, which were not working and were billing at $175,000 per treatment,” said Florida-02 constituent Candace Lerman. “There was also no formal PA or appeals process my doctor’s office could leverage. I ended up almost in critical care because my platelets dropped low enough to put me at serious risk of potentially fatal internal bleeding. Because this process took so long, it caused disease progression in a way that made recovery more difficult. Totally avoidable if we had proper protocols in place for rare disease patients to access off-label treatments.  I applaud Dr. Dunn for the work he is leading on the Protect Rare Act.  It can’t come soon enough!”

Read the full text of the bill here.

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