Gene and Cell Therapies

The major advances in the field of biotechnology and molecular biology in the 21st century have led to a better understanding of the pathophysiology of several diseases. A new generation of biopharmaceuticals has emerged including a wide and heterogeneous range of innovative cell and gene therapies. These therapies aim to prevent or treat chronic or serious life-threatening diseases, previously considered incurable. This book describes the evolution and adaptation of the regulatory environment to assess these therapies in contrast with the resistance of HTA agencies and payers to acknowledge the specificity of cell and gene therapies and the need of adaptation of existing decision-making frameworks. This book provides insights on the learnings from current cell and gene therapies experience (regulatory approval, HTA and market access), policy changes required to enhance patient access to these therapies and future trends.

Key Features:

Describes the potential change of treatment paradigm and the specificity of cell and gene therapies including the gradual move from repeated treatment administration to one single administration with potential for a definite cure.

Highlights the challenges at HTA level.

Discusses the affordability of future cell and gene therapies and the possible challenges for health insurance systems.

Provides potential solutions to address these challenges and ensure patient access to innovation while maintaining the sustainability of health care systems.