As US policymakers consider options to address rising health care costs while still meeting the needs of patients, a new report from IQVIA has found that rare diseases account for just 11% of medical invoice spending in the United States, and 79% of all orphan products treat only rare diseases. The report also highlights recent progress in drug development that has resulted in more people with rare diseases having access to critical treatments. As of January 1, 2020, 564 orphan products were approved by the US Food and Drug Administration (FDA) to treat 838 rare diseases (or “indications”). Thirty percent of all orphan indications were approved in the three years prior to the study, which is based on 2019 data. Multiple uses for the same medicine have become increasingly common, particularly in cancer and autoimmune diseases, as improved understanding of biological pathways allows for a single therapy to treat numerous rare and common conditions. Still, most orphan products treat only one rare disease.
For further information, see NORD (https://rarediseases.org/new-report-finds-medical-treatments-for-rare-diseases-account-for-only-11-of-us-drug-spending-nearly-80-of-orphan-products-treat-rare-diseases-exclusively/)
