{"database": "press", "table": "releases", "rows": [["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. \u2013 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.\n\nOver 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 \u201cmedically accepted indications\u201d 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.\n\n\u201cModern 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\u2019t have the opportunity to benefit from these new cures,\u201d said Rep. Kelly. \u201cIn some instances, there simply aren\u2019t enough patients with a rare disease to run a full clinical trial. That\u2019s 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.\u201d\n\n\u201cAccess 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,\u201d said Congresswoman Matsui. \u201cThe 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\u2019s 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.\u201d\n\n\u201cI 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,\u201d said Congressman Dunn, MD. \u201cTo 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.\u201d\n\n\u201cAccess to new and innovative treatments are essential to the health and well-being for seniors on Medicare who live with rare diseases,\u201d said Rep. Thompson. \u201cToo 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.\u201d", 1, "2026-03-30T01:40:41Z", "2026-04-08T00:49:26Z"]], "columns": ["url", "title", "date", "year", "month", "party", "chamber", "state", "member_name", "bioguide_id", "domain", "scraper", "source", "date_source", "text", "has_text", "collected_at", "updated_at"], "primary_keys": ["url"], "primary_key_values": ["https://kelly.house.gov/media/press-releases/reps-kelly-matsui-dunn-thompson-introduce-legislation-support-access-evidence"], "units": {}, "query_ms": 0.8058249950408936, "source": "dwillis/congress-press", "source_url": "https://github.com/dwillis/congress-press", "license": "MIT", "license_url": "https://github.com/dwillis/congress-press/blob/main/LICENSE"}