Advocacy
The Importance of the Access to Rare Indications Act Marc Yale
T
he Institute for Clinical and Economic Review (ICER) recently released a white paper on rare diseases that offers several potentially misguided proposals that would not benefit our community or help bring new treatments to market for pemphigus and pemphigoid (P/P). A far more thoughtful proposal from Reps. Doris Matsui (D-CA), Mike Thompson (D-CA), Mike Kelly (R-PA) and Markwayne Mullin (R-OK) is H.R. 6160, the Access to Rare Indications Act, which recognizes the fact that the vast majority of rare disease patients still don’t have an FDA-approved treatment. This illustrates that clinicians shouldn’t have to struggle to convince payers that medically accepted off-label treatments are “medically necessary.” Most importantly, it gives rare disease patients the same opportunity to get an off-label treatment that non-rare patients enjoy.
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Quarterly | Q2 2022
Medical necessity is the yardstick by which insurance companies, including Medicare and Medicaid, decide if they will pay for a particular treatment. When drugs are prescribed, payers look first to its label to see if it treats or manages the particular condition in question. If the prescribed use is off-label, they look to evidence-based compilations of drugs and their uses (compendia). This seems to work well for common conditions and in most cancers, which have robust compendia to address heavy reliance on off-label uses. Congress also ensured Medicare and Medicaid cancer patients have access to promising off-label uses that are not yet incorporated into compendia but are supported in published peer-reviewed literature. Like cancer, off-label use is often the only treatment option for P/P patients. Treatment decisions are based on clinical guidelines, published research, and
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