Connect with us

Hi, what are you looking for?


Neal Dunn, Doris Matsui Team Up on Bill to Expand Coverage for Rare Disease Treatments

Last week, U.S. Rep. Neal Dunn, R-Fla., threw his support behind U.S. Rep. Doris Matsui’s, D-Calif., “Providing Realistic Opportunity to Equal and Comparable Treatment for Rare (PROTECT Rare) Act.

The bill “will support patients with rare and ultra-rare diseases in getting evidence-based, medically necessary care covered by their health insurance” and the California Democrat’s office noted some of the reasons for the proposal.

“Over 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. This legislation will allow Medicare and Medicaid to use additional sources, including peer-reviewed literature, when considering whether rare disease therapies meet the standard for ‘medically accepted indications’ to provide coverage. 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,” Matsui’s office noted.

“Access to the best medical care is crucial for patients living with rare and ultra-rare diseases, yet too often these patients cannot get the evidence-based care they need,” said Matsui. “The community of rare patients deserves coverage for 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 rare diseases. That’s why I introduced the PROTECT Rare Act to ensure that rare and ultra-rare patients are no longer denied access to the care prescribed by their doctors.”

“I hear from many of my constituents who fear that restrictions on off-label drugs threatens 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 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.”

Other backers include U.S. Reps. Mike Kelly, R-Penn., and Mike Thompson, D-Calif.

The bill was sent to the U.S. House Energy and Commerce and the Ways and Means Committees. So far, there is no companion measure over in the U.S. Senate.


  • Kevin Derby

    Originally from Jacksonville, Kevin Derby is a contributing writer for Florida Daily and covers politics across Florida.

    View all posts
Share Story Via Text, E-Mail, Facebook or Twitter

Follow Us on Social Media

Ed Dean: Publisher


Ed Dean is a leading radio and news media personality including hosting the #1 statewide radio talk show in Florida. Contact

Article Archives

Related Stories