Drug Development for Rare Diseases is Littered with Regulatory Roadblocks

A rare disease is defined as a condition that affects fewer than 200,000 people but collectively, rare diseases are actually quite common. In fact, one in every 10 Americans is diagnosed with a rare disease.

The regulatory path for companies who wade into this drug development space is often fraught with challenges. From a lack of effective tools or endpoints to measure clinical success, to a limited number of available trial participants to ethical considerations, many challenges lay ahead.