Overview

"Advances in medicine have made possible better treatments for widespread, familiar human illnesses like cancer, diabetes, and heart disease. Yet there are thousands of much less common diseases, most of genetic origin, each classed as ""rare"" because it afflicts only a small number of people. These patient groups were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Yet these ""orphaned"" diseases collectively caused misery and expense, often far greater than did more common ailments, for tens of millions of individuals and their families. Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic triggered additional activism, compounded when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. And the third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen employing then-new genetic engineering instead of conventional approaches to pharmaceutical development. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry. Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception―a leader in the field as a strategy consultant, biotechnology executive, and venture entrepreneur. 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. He concludes with thoughtful consideration of important questions. Why do drugs to treat orphan diseases cost so much? How can we ensure they are affordable? How can their effectiveness be responsibly assessed? And how can access to them be expanded internationally? This book graphically and poignantly illustrates how far an important healthcare revolution has come and reminds us that if not nurtured, it could end before its immense promise has been fulfilled."

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"""A great service. Well organized and tells a forty-year-long tale with a series of poignant chapters that bring the humanity of the patients, scientists, and others at the companies into the narrative."" -- ""Jim Greenwood, six-term US Congressman; retired President and CEO of Biotechnology Innovation Organization "" ""A one-of-a-kind book by a one-of-a-kind author. 'One-of-a-kind' book because it's written from the ground real-time, not from twenty thousand feet. 'One-of-a-kind' author because it's the voice of not only an observer but, more importantly, an active participant over forty years."" -- ""John Howe, former President and CEO, Project HOPE; recipient of public service awards from the American Medical Association, the US Army, the US Surgeon General, and many others"" ""Both unusually informative and interesting, Inside the Orphan Drug Revolution should get strong readership as a biography of the Orphan Drug Act and the progress it stimulated. It is an important contribution to the public discourse."" -- ""Phil Sharp, winner, 1993 Nobel Prize in Medicine; MIT Institute Professor; Biogen Co-Founder"" ""Patient-centered biotech companies have brought hope to families facing rare genetic diseases. Jim Geraghty has long been at the center of that revolution at Genzyme and elsewhere. His book alerts readers to policy issues of importance to every prospective parent or grandparent or anyone who cares about the health of humankind."" -- ""Deval Patrick, former two-term Governor of Massachusetts "" ""The Orphan Drug Act has been one of the most successful pieces of health-related legislation of the last fifty years. It truly has wrought a revolution for the lives of countless families. This book brings to life the forgotten story of the patient advocates who championed it, the resistance overcome to pass it, and the dramatic advances it has enabled for countless people afflicted by devastating genetic diseases."" -- ""Henry Waxman, twenty-term US Congressman; principal sponsor of the Orphan Drug Act"""

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