Improving Clinical Trials for Rare Disease Therapeutics

With millions worldwide living with rare diseases that have few or no current therapeutic options, clinical trials are essential in treating the conditions faced by this diverse patient population. However, running these trials are expensive, hard to recruit for, and often require novel trial formats to account for small participant pools. Regardless, hope is on the horizon for patients.

According to Emily Wiggington, “The pharmaceutical industry and investors are showing increasing interest in developing treatments for rare diseases due to their potential for high ROI and the possibility of securing market exclusivity through the Orphan Drug Act”

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(Source: PM Live, March 23^rd, 2023)