So far more than 1000 trials are currently ongoing with gene and cell therapies. Cure is no longer a dream for several monogenic disorders. However there is still uncertainty on how those products will be accessed by patients and at what price. This book aim to show how the regulatory environment has evolved to analyze and review those therapies. Inhaltsverzeichnis Chapter 1 Introduction to cell and gene therapies concepts and definitions in the US and EU .................................................... 1 Chapter 2 Cell and gene therapies: genuine products and potential for dramatic value ........................................... 19 Chapter 3 Cell and gene therapies: regulatory aspects in the US and EU ............................................................................ 31 Chapter 4 The need for new HTA reference case for cell and gene therapies ............................................................................ 59 Chapter 5 How to mitigate uncertainties and HTA risk-averse attitude? ............................................... 91 Chapter 6 Cell and gene therapies funding: challenges and solutions for patients' access ........................................ 101 Chapter 7 Conclusion ................................................................................ 129
