Opinion: A seemingly small semantic issue is a major roadblock to develop treatments for rare diseases
Clarifying this distinction will help accelerate the development of cures for rare diseases.
Finding a new medicine is never easy. But developing treatments for patients with rare diseases — conditions that afflict fewer than 200,000 people in the United States — is particularly challenging.
When there are only a few thousand or even a few hundred people living with a particular condition, finding enough patients for clinical trials can take years. Clinical trials also typically require collaboration between multiple academic health centers, and care is often not standardized due to disagreements among researchers about what a “successful” drug should do. Many biotech companies also shy away from rare disease research, since the potential financial returns can be comparatively low, given the small population of potential customers.
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