The Access to Rare Indications Act could be a game changer for millions of Americans

By Darcy Krueger and Emanual Maverakis

June 1, 2022

The term “medically necessary” is the yardstick by which insurance companies, including Medicare and Medicaid, decide if they will pay for a particular treatment. For the millions of Americans living with rare diseases, most of which do not have FDA-approved treatments, identifying a treatment as medically necessary can be a lifeline — or the end of the line.

A white paper released in April by the influential Institute for Clinical and Economic Review offers several misguided proposals that attempt to balance public interest in incentivizing innovation with payer cost containment goals in rare diseases. The proposals are unlikely to benefit people living with rare diseases, clinicians like us who care for them, or those trying to bring rare treatments to market.

A far more thoughtful approach is embodied in the Access to Rare Indications Act (H.R. 6160), introduced in the House of Representatives by Reps. Doris Matsui (D-Calif.), Mike Thompson (D-Calif.), Mike Kelly (R-Penn.) and Markwayne Mullin (R-Okla.). The bill recognizes that the vast majority of people with rare diseases don’t have an FDA-approved treatment, meaning their disease is not listed on the treatment’s label, and their clinicians shouldn’t have to struggle to convince payers that medically accepted off-label treatments are medically necessary. Specifically, this bill seeks to give people with rare diseases the same level of insurance coverage for off-label treatments as patients with “common” conditions such as cancer and insomnia.

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