Rare Disease Management: Trends from 2024 Approvals

IPD Analytics has released a new report “Rare Disease Management: Trends from 2024 Approvals,” analyzing management trends for rare disease therapies approved in 2024, building on our earlier publication, “Rare Disease Drug Approvals—2024 Year in Review.” With more than 7,000 rare diseases affecting over 30 million people in the United States, rare conditions represent a growing area of clinical and payer focus. In 2024 alone, the FDA approved 33 new molecular entities and biologics with Orphan Drug designation, including seven cell and gene therapies.

Our latest analysis reviews coverage policies across four national commercial payers and one Medicare payer for select rare disease drugs, highlighting key utilization management trends such as prior authorization, step therapy, and quantity limits.