The major advances in the field of biotechnology and molecular biology in the 21st century has led to a better understanding of pathophysiology of diseases. A new generation of biopharmaceuticals has emerged including a wide and heterogeneous range of innovative therapies. These aim to prevent or treat chronic or serious life-threatening diseases, previously considered incurable. Gene and Cell Therapies: Market Access and Funding shows how the regulatory environment has evolved to analyse and review those therapies while HTA agencies and payers remain resistant. It provides insight into current learning, how those products will be accessible and which policy changes will be required to permit patient access.
Chapter 1 Introduction to cell and gene therapies concepts and definitions in US and EU
Chapter 2 cell and gene therapies: genuine products and potential for dramatic value
Chapter 3 cell and gene therapies: Regulatory aspects in US and EU
Chapter 4 the need for new HTA reference case for cell and gene therapies
Chapter 5 How to mitigate cell and gene therapies uncertainties and HTA risk adverse attitude?
Chapter 6 Cell and gene therapies funding: challenges and solutions for patients’ access
Chapter 7 Conclusion