Gene and cell therapies market access and funding / Eve Hanna and Mondher Toumi.

Contents Introduction to cell and gene therapies concepts and definitions in US and EU -- Cell and gene therapies : genuine products and potential for dramatic value -- Cell and gene therapies : regulatory aspects in US and EU -- The need for new HTA reference case for cell and gene therapies -- How to mitigate cell and gene therapies uncertainties and HTA risk adverse attitude? -- Cell and gene therapies funding : challenges and solutions for patients' access.
Abstract "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 health technology assessment (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), in addition to future trends to enhance patient access to these therapies"-- Provided by publisher.
Bibliography noteIncludes bibliographical references and index.
Access restrictionAvailable only to authorized users.
Technical detailsMode of access: World Wide Web
Genre/formElectronic books.
LCCN 2020009195
ISBN9780367408091 (hardback)
ISBN(ebook)

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