The Evolving Orphan Drug Commercial Landscape:
Expansion and Innovation in Rare Disease Therapies
The passage of the Orphan Drug Act (ODA) in 1983 is widely regarded as a key turning point leading to greater focus and availability of therapies for rare diseases, defined in the US as those with fewer than 200,000 affected patients. Prior to the ODA, the Food and Drug Administration (FDA) had approved only 38 orphan drugs, and rare diseases were not considered a commercially attractive segment in the pharmaceutical industry.
In the 40-years since the Act’s approval, a recent study by the FDA tallied that their agency approved orphan drugs for approximately 5% of rare diseases. Over this period, up to 15% of the identified and described rare diseases have had at least one orphan drug designation. In recent years, multiple therapies have been FDA-approved for rare diseases for which there had been no or few earlier treatment options. With many recently approved orphan drugs having competing indications, manufacturers can find themselves competing for patients and provider focus in small addressable markets. Understanding these recent trends is essential to your go-to-market strategy within commercial drug development.
Download our Evolving Orphan Drug Commercial Landscape guide to learn:
- Recent trends in the rare disease commercial landscape, based on interviews with rare disease experts
- The challenges of earlier disease identification
- The expanded focus on supporting patient lifetime management
- The changing competitive landscape including the acquisition of orphan assets by mid-sized and large pharmaceutical companies