By Cléa Stémitsiotis, MSc and James A. Levine, MD, PhD, Professor
A Race against time
How medicines are made. A guide for the rare disease community.
Age: All ages
Pagination: 56 pages
Size: 230 x 163 mm
Language: English
Publication: June 2024
ISBN: 978-2-38427-135-1 (printed book)/ 978-2-38427-136-8 (ePub)
Free
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.
How to use this guide
The purpose of this guide is to explore the steps involved during the rare disease medicine development process. It is not a comprehensive science textbook but rather a ‘quick read’. Each chapter is short and designed to be read in 5-10 minutes.
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 the impact evaluation of Fondation Ipsen and innovative finance for rare diseases.
James A. Levine, MD, PhD, Professor, is President of Fondation Ipsen. He is a serial entrepreneur, scientist and physician, trained in business at Harvard Business School.