A Race against Time.

How medicines are made. A guide for the rare disease community.

Cléa Stémitsiotis, James A. Levine

• Publisher: Fondation Ipsen Press
• Year: 2025
• eBook ISBN: 9782384271368
• Edition: 0
• Topic: Medicine
• Subtopic: Biotechnology in Medicine

Making medicines is complex and, requires meticulous research, innovative thinking, and unwavering dedication. In the realm of rare diseases, this journey is even more complex because the illnesses are so rare. Even rare disease experts often do not know how to help their patients. We need more treatments because 90% of people living with rare disease do not have an effective treatment. This book explores the steps involved in making medicines. It is our response to requests from the rare disease community. There is an urgent need to understand how medicines are created so that patients and those who care for them can advocate with greater clarity and louder voices. We hope you find this book useful.ABOUT THE AUTHORS James A. Levine, PhD, MD, MBA, Professor 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 founda- tion 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. His business training, focused on entrepreneurship, was from Harvard Business School. With a background in wearable technologies, data gathering and data analytics, and with more than 100 patents and trademarks, Levine helped found 35 companies. He was the Innovator of the Year in the state of Minnesota, the World Trade Fair and NASA. Under the Mayo Clinic NEAT Trademarks, Levine's team delivered scalable health solutions to 72 US corporations. A great deal of James' work focusses on biotech development, scalable health solutions and business opportunities in underserved regions in the United States, France and low/middle countries such as the Democratic Republic of Congo, Cote d'Ivoire, Afghanistan, Jamaica, Asia, Kenya and India. Consulting to the President of the United States, the US State De- partment, US Army and internationally, James in 2018 was awarded the President's Medal for promoting social embeddedness. 300 million people live with rare diseases without effective cures. Sus- tainable solutions require the efficient use of capital to optimize biotech- nology companies, maximize impact and minimize suffering.
Cléa Stémitsiotis holds a Bachelor's degree in Management from the London School of Economics and a Master's degree in Economics and Business from Sciences Po Paris. She works on impact assessment and innovative financing for rare diseases.

Table of contents

• Cover
• Title
• Contents
• Introduction
• Summary
• Understanding Rare Diseases
• Target Identification and Medicine Discovery
• Medicine Design and Development
• Preclinical testing
• Focus on the Orphan Drug Designation
• Clinical trials
• Regulatory Approval and Post-Marketing Considerations
• Patient Access and Support
• Focus on Milestones for Rare Disease Companies, Relationships with Regulators, and Investors
• Conclusion
• Acknowledgements
• References and additional resources
• Copyright
• Backcover