Advanced therapy medicinal products (ATMPs), i.e., cell and gene therapy products, is a rapidly evolving field of therapeutic development. A significant proportion of the products are being developed by academia or small/medium-sized enterprises (SMEs). The many challenges in translation posed by this class of products include aspects covering: manufacturing, non-clinical development plan as relevant to clinical trial, marketing authorization, and reimbursement. In this context, the term translation refers to the relevance of non-clinical data in relation to how it impacts on appropriate and efficient clinical development. In order to successfully overcome these challenges, a clear understanding of the requirements and expectations of all the stakeholders is critical. This article aims to cover the potential challenges related to such translation and suggested approaches to find solutions based on experience and learnings from the perspective of European Union. While commercial challenges have a significant impact on the ATMPs in general, it is considered outside the scope of this article. However, by adopting a strong scientific basis for translation as suggested in this article, it is likely such an approach would help rather than harm successful real world clinical use of ATMPs.
Cecile Rousseau, PhD, Director
With 10+ years in nonclinical and translational research and histotechnology, Dr Cécile F. Rousseau has considerable expertise in medical devices, engineered cellular therapies and companion diagnostics. Upon completing a Ph.D. program in Medical and Biological Engineering at the University Claude Bernard – Lyon 1 in 2002, Cécile proceeded with postdoctoral research in tissue engineering (including but not limited to cartilage, soft organs), toxicology and pharmacology at the Laboratory of Cartilage Biology and Engineering and at the Laboratory of Molecular Assembly of Biological Interest.
Dr Gopalan Narayanan, Vice President Disruptive Biologics
Within VCLS, Dr Narayanan provides leadership in the area of complex and disruptive biologics such as Cell and Gene therapies, called Advanced Therapies Medicinal Products (ATMPs) in Europe, including guidance on product development and regulatory strategy. In addition, he participates in the global effort of rationalising the development process, by enabling innovative regulatory mechanisms through which Advanced Therapies and other disruptive biologics can be developed and brought to patients faster and more efficiently.
Professor Romaldas Maciulaitis
Professor Romaldas Maciulaitis has worked at the Institute of Physiology and Pharmacology in the Department Nephrology at the Lithuanian University of Health Studies for more than 28 years. He has also been a CHMP and CAT member at the EMA for 14 years.The Professor completed his MD and PharmD studies more than 20 years ago and after PhD studies in Clinical Pharmacology, was involved into Drug Regulatory and Scientific Appraisals at the national (Lithuania) and international (European) levels. In 2009, he initiated new experimental Pharmacology research direction in his University in Kaunas on Regenerative Pharmacology applying cell preparations with research program focusing on PK/PD of cell therapies in renal and cartilage injuries.