Rare Disease Treatment Market Outlook 2037

The global rare disease treatment market was valued at USD 232.2 billion in 2024 and is expected to grow significantly, reaching approximately USD 792.8 billion by 2037. This growth reflects a projected compound annual growth rate (CAGR) of 10.3% over the forecast period from 2025 to 2037. By the end of 2025, the market size is anticipated to reach around USD 243.1 billion. The rare disease treatment market is witnessing strong momentum due to scientific breakthroughs in genetics, cell therapy, and mRNA technology. With thousands of rare conditions lacking effective treatment, pharma companies are fast-tracking orphan drug development, supported by regulatory incentives such as the U.S. Orphan Drug Act and the EU’s PRIME scheme. The growing adoption of precision medicine and advanced diagnostics is enabling earlier intervention and targeted therapies. Meanwhile, biopharmaceutical investments and cross-border collaborations are expanding access to novel treatments, especially for ultra-rare and pediatric conditions.