MedCity News July 24, 2023
In 2020, 46.9% of all novel drugs approved by the FDA were for rare diseases, as compared to 23.5% in 2012. Investment is expected to continue trending upward, with estimates suggesting that global spending on rare disease therapies will reach $260 billion by the end of 2025.
Research and development for rare diseases has traditionally been the province of small biotech companies and targeted at very much a niche market. But in recent years, there has been a growing interest in the space among investors, researchers, and pharmaceutical companies—giving hope to the 30+ million Americans with diseases considered “rare.”
In the last several years, the FDA has approved new treatments for chronic graft-versus-host disease, Duchenne muscular dystrophy, relapsed or refractory...
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