EP 1117B – Inside The Orphan Drug Revolution

Rare diseases are also called ‘orphan diseases,’ so named because they were long abandoned (or orphaned) by a profit-driven pharmaceutical industry. So why a book about orphan drugs, which by definition treat only rare diseases? Did they really trigger a revolution important enough to care about? And if so, why is that so little known? In fact, orphan drugs have revolutionized society, for reasons ranging from the deeply personal to the broader cultural and political.

Rare diseases deserve our attention because they’re merciless, causing families often far worse human suffering than common diseases. And unlike many common diseases, few can be prevented or treated by a good diet or exercise. Perhaps most importantly, a rare genetic disease could someday strike any family—and an orphan drug become of utmost urgency to anyone with a child or grandchild, niece or nephew.

A biotechnology executive, Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception. His book is in part a history, with eyewitness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made the revolution happen. And it tells deeply personal stories of patients and parents willing to risk new, untried therapies. But Geraghty also uses his exceptional experience and vantage point to look forward, to the immense promise of the newest technologies like gene therapy and gene editing for the treatment of patients today and tomorrow.