book❤️[READ]✔️ Inside The Orphan Drug Revolution: The Promise of Patient-Centere

Inside The Orphan Drug Revolution: The Promise of Patient-
Centered Biotechnology

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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&#8213aleader 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.