Orphan Drug Development – A Pragmatic Approach To Developing A Rare Disease Drug

Challenges and considerations associated with the expedited development of orphan drugs, and how to successfully work through them

Most medicines in development today target diseases affecting large sections of the global population. But now we are slowly beginning to see a rise in the interest of developing "orphan drugs," or pharmaceutical agents intended to treat rare diseases. In the United States, a rare disease is defined as any disease affecting fewer than 200,000 individuals; however, this definition varies by country.¹ Though these rare diseases can be incredibly debilitating and present a truly unmet medical need, in some cases pharmaceutical companies have been reluctant to develop treatments.