Advanced therapy products are mainly cell- or tissue-based products and gene products. Their therapeutic potential is promising but, at the same time, their complexity and rapid development also demand proper regulation. In the past 15 years, many jurisdictions started to develop regulatory regimen to properly regulate the development, production, provision and usage of advanced therapy products. In most cases, regulation of advanced therapy products is built upon the existing framework for pharmaceutical products.


Based on overseas experience, especially the regulations in the European Union, Hong Kong Government proposed a regulatory framework for advanced therapy products, which was discussed and endorsed by the Task Force on Regulation of Advanced Therapeutic Products in Hong Kong. The proposal was to classify advanced therapy products, including somatic cell therapy products, tissue engineered products and gene therapy products, as pharmaceutical products so that they are regulated under the Pharmacy and Poisons Ordinance (Cap. 138). Additional requirements on labeling, manufacturing and record keeping related to advanced therapy products are also provided. 


With supports from the medical profession, pharmaceutical field, academia and general public, the proposal of amending the Pharmacy and Poisons Ordinance was introduced to the Legislative Council for scrutiny. The Amendment Bill was eventually passed in July 2020 and the Pharmacy and Poisons (Amendment) Ordinance 2020 will come into force in 2021. 


Being a healthcare professional, understanding the development and regulation of advanced therapy products both in Hong Kong and overseas is important. This talk gives an overview on the global situation of advanced therapy products as well as the regulation of these products under the amended Pharmacy and Poisons Ordinance.

Chimeric antigen receptor T cell (Car-T) therapies are transformative therapeutics that are highly effective in treating relapsed and refractory B cell malignancies. Car-T products directed against CD19 are now approved internationally for B cell acute lymphoblastic leukaemia, Aggressive lymphoma (DLBCL, PMBL, tFL), mantle cell lymphoma and most recently follicular lymphoma, with emerging B cell maturation antigen (BCMA) directed therapies for multiple myeloma in late stage development. Peter MacCallum Cancer Centre has been an international leader in the development of cellular therapies for 20yrs and in 2019 was designated as a Centre of Excellence for Cellular Immunotherapies. I will review some of the data supporting the approval of Car-T therapies in haematologic malignancies and the management of the specific toxicities associated with Car-T therapies, cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). I will also outline our approach to the roll out of these products in the standard-of-care setting in Australia and the development of local manufacturing capacity.

Organs and tissue transplantations are the only curative therapy for major organ and tissue failures and loss, but are limited by the donor supply and often severe immune complications. Some of these obstacles may potentially be bypassed using cell-based regenerative therapy. Indeed, recent developments in the stem cell biology provided new hopes to heal or replace tissues and organs damaged due to congenital defects, ageing, disease, or trauma. The field of cell-based regenerative medicine encompasses numerous strategies, including the use of de-novo generated cell types from different stem cells as well as various combinations with other materials to repair or replace both structurally and functionally of the diseased organs. Despite the promising results from many pre-clinical studies, there are many major hurdles in the translation of cell-based regenerative therapy to the clinic. One of the major step to develop cell-based regenerative therapy is to achieve adequate safety and quality standards in manufacturing those products. In Hong Kong, current GMP guidelines and regulations are based on quality principles that have been established for pharmaceutical and healthcare manufacturers. Now the emergence of cell-based regenerative therapy raise new challenges, as manufacturing of cell-based products often deploys new biotechnological techniques like tissue engineering, genetic modification or sophisticated cell-culture techniques. Even more important is the fact that a product comprised of living cells or other fragile biological compounds cannot be standardised in the same way as a conventional pill. In this presentation, the future perspective to establish GMP cell-based therapy in Hong Kong will be discussed.