Gene and Cell Therapies: Market Access and Funding

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.

Key Features:

• Discusses the affordability of future therapy and the possible challenges for health insurance systems
• Highlights the gradual move from repeated treatment administration to one single administration with potential for a definite cure
• Describes the potential change of paradigm which will challenge all payers and may question the sustainability of our health care 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.