REGENERATIVE MEDICINE UNLOCKING PATIENT ACCESS AND COMMERCIAL POTENTIAL (PB 2023) (Pharmaceuticals, Health Economics and Market Access)

A comprehensive review of the challenges that exist in patient accessibility to regenerative medicines (RMs), presenting clinical trials, marketing authorization, HTA, pricing, reimbursement, affordability, payment and partnership agreements of RMs and commercialization. Specfically, we investigated how COVID-19 has impacted the RM industry by elaborating on the disruptions it caused but also the new opportunities it brought. The ultimate goal of this work is to make strategic recommendations for manufacturers and decisions-makers on effective strategies to address the above obstacles and facilitate patient access to promising regenerative medicines.

FEATURES

Regenerative medicine (RM) is an emerging interdisciplinary field aiming to replace or regenerate human cells, tissues, or organs in order to restore normal function.        

1. RM holds the promise of revolutionizing treatment in the 21st century.       
2. RMs bring new hope for some previously untreatable diseases, as well as holding promise for the treatment of common chronic diseases.        
3. Rapid advancements in biotechnology and improved understanding of disease pathophysiology have attracted tremendous interests in the development of RMs.        
4. Discusses the high cost of RMs which may challenge the sustainability of healthcare insurers (public and private).

Tingting Qiu holds a PhD degree in public health and a master's degree in pharmacology. Her area of expertise includes regulatory affairs, health policy, health technology assessment, economic analysis, and price and reimbursement of innovative therapies, such as orphan drugs and regenerative medicines. She has authored over 30 publications and conference presentations focused on the market access of cell and gene therapies at the European and global level. She worked as a price and market access analyst at Creativ-Ceutical (Paris, France) for 4 years from 2018, during which she was deeply involved in addressing the challenges in meeting the expectations of relevant stakeholders to facilitate the patient access to lifesaving treatments. Since 2021, she has been a research associate to EA 3279 CERESS Department of Public Health at Aix-Marseille University. Currently, she is a researcher in the Beijing Institute of Clinical Pharmacy, Beijing Friendship Hospital, Capital Medical University (Beijing, China). Apart from endeavoring to advance the market access of cell and gene therapies in China and globally, she is also engaged in projects focusing on the impact of implementing diagnosis-related groups reform on the healthcare system, the methodology of health economic analysis in China, and evidence-based assessment of Chinese and western medicines.

Mondher Toumi is an MD by training and has an MSc in biostatistics and in biological sciences (option pharmacology) and a PhD in economic sciences. Toumi is a 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 embraced a career 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 Journal of Market Access and Health Policy (JMAHP) which was just granted PubMed indexation. Toumi is also a 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.