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.
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The major advances in the field of biotechnology and molecular biology in the twenty-first century have led to a better understanding of the pathophysiology of 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 and 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 health technology assessment (HTA) agencies and payers to acknowledge the specificity of cell and gene therapies and the need to adapt existing decision-making frameworks. This book provides insights on the learnings from the experience of current cell and gene therapies (regulatory approval, HTA, and market access), in addition to future trends to enhance patient access to these therapies.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-time single administration with the potential to be definite cureHighlights the challenges at the HTA levelDiscusses the affordability of future cell and gene therapies and the possible challenges for health insurance systemsProvides potential solutions to address these challenges and ensure patient access to innovation while maintaining the sustainability of healthcare systems
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