Tallahassee Sun

WASHINGTON, D.C. - A group of bipartisan lawmakers, including Congressman Neal Dunn, MD, Congresswoman Doris Matsui, Congressman Mike Thompson, and Congressman Mike Kelly, have introduced the PROTECT Rare Act. This bill aims to broaden coverage for rare disease treatments under Medicare Part D, Medicaid, and private insurance plans.

The PROTECT Rare Act specifically focuses on allowing Medicare Part D and Medicaid to cover drugs for off-label use if there is peer-reviewed medical literature that supports their effectiveness in treating a rare disease. Additionally, private insurance plans would be required to establish an expedited review process for coverage requests for the off-label use of drugs for rare diseases.

Congressman Dunn highlighted the importance of expanding access to off-label drugs for rare disease patients, stating, "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."

The motivation behind this legislation stems from the fact that rare diseases affect a very small patient population, making it unprofitable for pharmaceutical companies to develop specific cures. However, medical professionals and researchers often discover that existing drugs or combinations of drugs can effectively treat these rare diseases. Unfortunately, Medicare and Medicaid currently do not cover these off-label treatments.

One Florida constituent, Candace Lerman, shared her personal experience with the challenges of accessing off-label drugs for rare diseases. Lerman stated, "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." Lerman further explained the consequences of this denial, saying, "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."

The PROTECT Rare Act aims to address these challenges and provide rare disease patients with the necessary access to off-label treatments. By expanding coverage under Medicare Part D and Medicaid, as well as implementing an expedited review process for private insurance plans, this bill seeks to improve the quality of life and chances of survival for individuals with rare diseases.

The full text of the PROTECT Rare Act can be found on the official website. This legislative effort represents a significant step forward in expanding coverage for rare disease treatments and addressing the unique needs of this patient population.

For additional details, please follow this link: https://dunn.house.gov/press-releases?ID=E51323C4-4BBD-4BC5-848D-F41168F17D3A