Bipartisan team proposes legislation to improve access to off-label drugs

Act would allow Medicare, Medicaid to consider data other than FDA approval

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A bipartisan team of congressional representatives is proposing new legislation that aims to make it easier for people with rare disorders like cold agglutinin disease (CAD) to access treatments that may be beneficial even if they haven’t been formally approved for that indication.

The legislation — called the Providing Realistic Opportunity To Equal and Comparable Treatment for Rare (PROTECT Rare) Act — was proposed last week by U.S. Reps. Mike Kelly (R-PA-16), Doris Matsui (D-CA-07), Neal Dunn, MD (R-FL-02), and Mike Thompson (D-CA-04), and endorsed by a number of rare disease advocacy organizations.

The PROTECT Rare Act would allow Medicare and Medicaid to consider data apart from U.S. Food and Drug Administration (FDA) approval, including peer-reviewed studies and clinical guidelines, when deciding whether to provide coverage for off-label therapies in rare diseases.

The act also would require private insurance companies to create an expedited review pathway for formulary exception, reconsideration, and/or appeals for denials of coverage for treatments prescribed to rare disease patients.

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More than 95% of rare diseases don’t have a treatment approved by the FDA, “forcing patients to rely on treatments approved in other diseases to alleviate their symptoms,” Annie Kennedy, chief of policy, advocacy, and patient engagement at the EveryLife Foundation for Rare Diseases, said in a press release. “Regrettably, insurers often obstruct access to these vital, life-sustaining treatments, even when they align with clinical guidelines.”

“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,” Dunn said. “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.”

Medicare and Medicaid are the government-funded programs that provide health insurance for the elderly and those who are disabled, and low-income people, respectively.

Therapies for other diseases used often for rare disease treatment

Many therapies approved for other conditions are prescribed off-label for rare diseases in which the medications have been shown to also provide benefits, but have not been FDA-approved for that use.

CAD is an example of this. First-line treatment for CAD is usually rituximab, which is well documented to be effective in the disease, but has not been approved by the FDA for the rare autoimmune condition. Rituximab is FDA-authorized to treat other autoimmune diseases and blood cancers, but its use in CAD is off-label.

“Unfortunately, often the off-label use of therapies is not covered by health insurance, creating costly barriers to necessary treatments,” said Peter L. Saltonstall, CEO of the National Organization for Rare Disorders.

“This is especially burdensome for patients in underserved communities, who may lack the resources to either pay out-of-pocket for needed therapies or navigate the health system to secure coverage,” said Jenifer Waldrop, executive director of the Rare Disease Diversity Coalition. “Greater access to off-label treatments will reduce burdens of diseases, improve long-term prospects, and save lives.”

“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,” Matsui said.

Thompson said that the legislation “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.”

The PROTECT Rare Act “will give rare disease patients like me access to the treatments my doctors think is best for me and are considered medically acceptable,” said Janet Segall, a constituent of Matsui who lives with a rare autoimmune skin condition called pemphigus. “Living with a rare disease is hard enough without having to be denied access to the treatments I need.”