Bipartisan legislation introduced by U.S. Rep. Mike Kelly (R-PA) would ensure health insurance coverage of treatments for Americans having rare and ultra-rare diseases.
“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, noting that the proposed bill “guarantees patients have every opportunity to access life-saving cures in a way that works for them.”
The Access to Rare Indications Act of 2021, H.R. 6160, which Rep. Kelly cosponsored on Dec. 7 alongside bill sponsor U.S. Rep. Doris Matsui (D-CA), would amend both the Social Security Act and the Public Health Service Act to provide for coverage of certain drugs used in the treatment or management of a rare disease or condition, according to the congressional record bill summary.
“In some instances there simply aren’t enough patients with a rare disease to run a full clinical trial,” Rep. Kelly said. “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 Access to Rare Indications Act will do just this.”
If enacted, H.R. 6160 would require a label denoting approval by the Food and Drug Administration for certain rare disease treatments. In turn, this would allow Medicare and Medicaid to use additional sources — including compendia, peer-reviewed literature, and opinion of rare disease experts — to meet medically accepted criteria that ensure coverage for rare disease treatments, according to information provided by the lawmakers.
The bill 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, the information says.
“Access to the best medical care is crucial for patients living with rare and ultra-rare diseases, and too often these patients cannot get the evidence-based care they need,” Rep. Matsui said. “The community of rare patients deserve access to medically necessary treatments, the same way patients with more common conditions are covered by their health insurers.”
Patients, as well as healthcare providers, physicians, and nonprofit organizations endorsed the bill, which is under consideration by members in both the U.S. House Energy and Commerce Committee and the U.S. House Ways and Means Committee.