Age: All ages
Pagination: 256 pages
Size: 210 x 148 mm
Language: English
Publication: January 2026
ISBN: 978-2-38427-356-0 (print)/ 978-2-38427-357-7 (ePub)
Free
Every rare disease begins with a patient and a question: why isn’t there a treatment?
Too often, the answer isn’t scientific, it is financial. The will does exist. The does will exist. But the funding pathways don’t.
For decades, rare disease research has been caught between two worlds: public grants that are too short-term to sustain discovery, and private investment that sees too little market potential to engage. Thousands of promising ideas never leave the lab, not because they failed, but because the funding system did. This book is written to change that. Across the world, funders, patient organizations, and scientists are experimenting with bold, hybrid approaches that blend the discipline of finance with the purpose of medicine. Venture philanthropy, public–private partnerships, social impact bonds, and collaborative consortia are not financial curiosities, they are survival strategies for rare disease innovation.
The aim of Alternate Funding Mechanisms in Rare Disease is simple: to make these approaches understandable, usable, and actionable. Whether you lead a foundation, manage a research program, or simply care deeply about someone living with a rare condition, this book shows how to turn financial creativity into measurable impact. It is not an academic text. It is a manual for people who are tired of hearing “there is no funding” and ready to ask, “what if we built our own?”
Dr. Levine, President Fondation Ipsen, has three decades of experience in the healthcare sector principally at Mayo Clinic. For the last 6 years he has been President of Fondation Ipsen, an international science foundation focused on biotech innovation in Rare Diseases. A physician and scientist, James has published more than 200 articles, six papers in Science and Nature plus articles in journals such as, the New England Journal of Medicine, Lancet and JAMA. He has written four books published in 19 languages in 37 countries.
