Rare Disease Treatment Market Size

Rare Disease Treatment Market size was over USD 224.6 billion in 2024 and is estimated to reach USD 652.9 billion by the end of 2037, expanding at a CAGR of 9.3% during the forecast timeline, i.e., 2025-2037. In 2025, the industry size of rare disease treatment is assessed at USD 245.4 billion. The rare disease treatment market is largely driven by advancements in biotechnology, increasing investment in orphan drug research, and greater government incentives for developing treatments for rare conditions. More than 400 million people globally suffer from a rare disease. 1 in every 10 people have a rare disease, and 1 in every 2 patients diagnosed with a rare disease is a child.  Over 10,000 distinct types of rare diseases exist in the world, and 8 in every 10 cases of rare diseases are genetic by nature.

These diseases often lack effective treatments due to high costs and complex drug development processes. However, regulatory support such as the U.S. Orphan Drug Act has spurred pharmaceutical companies to explore more in the market. For instance, in October 2021, U.S. FDA awarded 11 new clinical trial research grants, valued at USD 25 million through the FDA’s Congressionally-funded Orphan Products Grants Program. Rare disease treatment market companies are increasing their focus on gene and cell therapies, which offer promising one-time treatments for certain rare diseases. Additionally, digital health tools and personalized medicine approaches are gaining momentum as they allow for more targeted and efficient treatment options.