Patients having rare diseases would receive evidence-based, medically necessary care covered by their health insurance under a bipartisan bill introduced by U.S. Rep. Mike Kelly (R-PA).
“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,” Rep. Kelly said. “In some instances, there simply aren’t enough patients with a rare disease to run a full clinical trial.”
On Oct. 26, the congressman cosponsored the Providing Realistic Opportunity To Equal and Comparable Treatment for Rare Act, also known as the PROTECT Rare Act, H.R. 6094, with bill sponsor U.S. Rep. Doris Matsui (D-CA) and two other original cosponsors. The legislation would 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.
“We need to empower doctors to make the best decision for their patients, using all the clinical data which is currently available,” said Rep. Kelly. “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.”
H.R. 6094 also would 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 that is prescribed for a patient with a rare disorder, according to the summary provided by Rep. Kelly’s staff.
“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,” Rep. Matsui said. “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.”
The bill has garnered support from numerous entities, including the Haystack Project, the EveryLife Foundation for Rare Diseases, the National Organization for Rare Disorders, the Rare Disease Diversity Council, the United Mitochondrial Disease Foundation, the United Porphyrias Association, the Autoinflammatory Alliance, the Barth Syndrome Foundation, the Biomarker Collaborative, the Cares Foundation, and many others.