Orphan Drug Trends: A Data-Driven Analysis

Global orphan drugs market projected to grow from USD 212.59 billion in 2025 to USD 443.07 billion by 2031 at a CAGR of 13.02%, driven by regulatory incentives and genomics advances1.

Alternative forecasts estimate growth from USD 216.55 billion in 2025 to USD 610.24 billion by 2034 at 12.20% CAGR4.

In 2024, 52% of FDA's 50 novel drug approvals (26 drugs) were for orphan diseases, continuing a trend where over 50% of approvals are orphan drugs2.

Orphan drugs expected to comprise 20% of global prescription drug sales by 2030, doubling their share over the last decade3.

Key drivers include government incentives like tax credits and market exclusivity, with 28 of 55 novel FDA approvals in 2023 being orphan drugs12.

Challenges involve high treatment costs and reimbursement delays, with Europe averaging 578 days for patient access in 20241.

Trends feature AI integration in drug discovery, mergers/acquisitions, and rising gene therapies, with 5 new approvals in 20231.

In June 2025, FDA granted orphan drug designation to rilzabrutinib for sickle cell disease4.

Projections show 160-200 more orphan drugs entering FDA review by 2030, increasing competition5.

U.S. legislation like H.R.946 (ORPHAN Cures Act) modifies Medicare drug price negotiation for orphan drugs7.

Sources:

1. https://www.globenewswire.com/news-release/2026/01/22/3223571/28124/en/Global-Orphan-Drugs-Market-to-Surpass-USD-443-Billion-by-2031-Driven-by-Advances-in-Genomics-and-Precision-Medicine.html

2. https://www.dcatvci.org/features/orphan-drugs-still-showing-market-strength-or-not/

3. https://www.evaluate.com/thought-leadership/orphan-drugs-2025-report/

4. https://www.precedenceresearch.com/orphan-drugs-market

5. https://antonhealth.com/orphan-drugs-in-2026-are-you-prepared/

7. https://www.congress.gov/bill/119th-congress/house-bill/946