releases: https://kelly.house.gov/media/press-releases/reps-kelly-matsui-dunn-thompson-introduce-legislation-support-access-evidence
Data license: MIT · Data source: dwillis/congress-press
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| https://kelly.house.gov/media/press-releases/reps-kelly-matsui-dunn-thompson-introduce-legislation-support-access-evidence | Reps. Kelly, Matsui, Dunn, Thompson introduce legislation to support access to evidence-based care for rare disease patients | 2023-10-27 | 2023 | 2023-10 | Republican | House | PA | Mike Kelly | K000376 | kelly.house.gov | kelly | https://kelly.house.gov/media/press-releases | scraper | WASHINGTON, D.C. – This week, U.S. Reps. Mike Kelly (R-PA-16), Congresswoman Doris Matsui (D-CA-07), Reps. Neal Dunn (R-FL-02), and Mike Thompson (D-CA-04), introduced the Providing Realistic Opportunity To Equal and Comparable Treatment for Rare (PROTECT Rare) Act. This legislation will support patients with rare and ultra-rare diseases in getting evidence-based, medically necessary care covered by their health insurance. Over 90% 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. “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 PROTECT Rare Act will do just this, and guarantees patients have every opportunity to access life-saving cures in a way that works for them.” “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 Congresswoman 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 Congressman Dunn, MD. “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.” “Access to new and innovative treatments are essential to the health and well-being for seniors on Medicare who live with rare diseases,” said Rep. Thompson. “Too often, these people have very few options and new treatments or off-label drugs are often not covered under Medicare or Medicaid. The PROTECT Rare Act will allow Medicare and Medicaid to cover the cost of medication or treatment that doctors think would benefit their patients if appropriate, providing flexibility to Americans living with rare diseases.” | 1 | 2026-03-30T01:40:41Z | 2026-04-08T00:49:26Z |