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.
- 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 cure
- Highlights the challenges at the 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 healthcare systems
Mondher Toumi is M.D. by training, M.Sc. in Biostatistics, and in Biological Sciences (option pharmacology) and Ph.D. in Economic Sciences. Mondher Toumi is Professor of Public Health at Aix-Marseille University. After working for 12 years as Research Manager in the department of pharmacology at the University of Marseille, he joined the Public Health Department in 1993. In 1995 he embraces a carrier in the pharmaceutical industry for 13 years. Toumi was appointed Global Vice President at Lundbeck A/S in charge of health economics, outcome research, pricing, market access, epidemiology, risk management, governmental affairs and competitive intelligence. In 2008, he founded Creativ-Ceutical, an international consulting firm dedicated to support health industries and authorities in strategic decision-making. In February 2009 he was appointed Professor at Lyon I University in the Department of Decision Sciences and Health Policies. The same year, he was appointed Director of the Chair of Public Health and Market Access. He launched the first European University Diploma of Market Access (EMAUD) an international course already followed by almost 350 students. Additionally, he recently created the Market Access Society to promote education, research and scientific activities at the interface of market access, HTA, public health and health economic assessment. He is editor in Chief of the Journal of Market Access and Health Policy (JMAHP) which was just granted PubMed indexation. Toumi is also visiting Professor at Beijing University (Third Hospital). He is a recognized expert in health economics and an authority on market access and risk management. He has more than 250 scientific publications and communications, and has contributed to several books.
Eve Hanna is global pricing and market access consultant at Creativ-ceutical, where she engages in several types of market access research. Eve is PhD in Health economics, pharmD, and holds a master's degree in pharmaco-epidemiology. She has successfully completed the European Market Access University Diploma (EMAUD) and is currently senior lecturer in health technology assessment (HTA) and Market Access at EMAUD. Her area of expertise is cell and gene therapies HTA, funding and market access; she has several publications in peer-reviewed journals and major conferences all focused on gene and cell therapies. Eve has contributed to three books mainly on orphan drugs topics. During her five years of experience in the field of market access, Eve has acquired proficient knowledge on HTA, pricing and reimbursement processes and health policy at global level.
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