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PP09-67: Opportunities for Regional Convergence - Regulation of Advanced Therapy Medicinal Products in Asia Pacific

Poster Presenter

      Uttara Soumyanarayanan

      • Senior Education Associate
      • Centre of Regulatory Excellence, Duke-Nus Medical School


• Identify the similarities and differences in regulatory frameworks for ATMPs across the national regulatory authorities (NRA) in Asia-Pacific • Develop strategies to bridge the discrepancies among the NRAs to facilitate regulatory convergence for ATMPs.


• A survey was sent to selected NRAs to obtain information on different aspects comprising their ATMP regulatory frameworks & challenges faced by them in the review of ATMP dossiers. • The responses were presented during a workshop to obtain inputs and clarifications from subject matter experts.


• NRAs from the following 8 countries participated in the survey: European Union, Australia, Singapore, Japan, Malaysia, Korea, Thailand and Taiwan. • It is noted that EMA, MFDS and PMDA have an established regulatory framework specific to ATMPs, and TGA regulates these products under the biologics framework. Thai FDA as well as TFDA are in the process of formulating ATMP regulatory frameworks. • Three common terminologies have been used by the 8 agencies for advanced therapies, namely, advanced therapy medicinal products, cell and gene therapy products, and regenerative medicines. EMA, HSA, PMDA and Thai FDA have similar specific definitions for advanced therapies that describe the nature and purpose of these products. On the other hand, MFDS, TGA, TFDA and NPRA do not have a formal definition for ATMPs but indicate the use of cells and genes as medicinal products. Most NRAs categorize ATMPs based on the nature of the product: cell therapy, gene therapy, tissue-engineered products and combination products. While HSA, NPRA and TGA also have a risk-classification of the products based on the extent of manipulation: low-risk and high-risk products therapies. • The NRAs primarily follow ICH guidelines as relevant to ATMPs and mandate dossier submission in ICH CTD format. Apart from the common international guidelines, some of the NRAs refer to local guidelines that are specific to the region. HSA, NPRA and Thai FDA also refer to US FDA and EMA guidelines, which have been formulated using the global ICH guidance. • MFDS has the maximum marketing authorizations approvals (16) for ATMPs followed by the EU (10); PMDA and TGA have up to 5 products approved in this domain. NPRA, TFDA, HSA, Thai FDA do not have any ATMPs approved as yet. • Most regulatory agencies face challenges with required expertise for reviewing ATMP dossiers, specifically concerning manufacturing and quality information.


• ATMP is a nascent, yet rapidly evolving field, with many regulatory agencies still in the process of establishing frameworks, therefore offering potential opportunities for convergence right at the outset. There are several similarities as well as divergences in the regulatory structures across the NRAs; it is crucial to tap on the commonalities to achieve harmonization of the important components, namely definition and technical requirements. • The terminologies, definitions as well as the scope of regulation of ATMPs differ. A harmonized definition should ideally include the nature of the products as well as its intended use. It is essential to categorize the products based on their nature (cell, gene, tissue or combination); although risk-based classification can be included additionally. • Technical requirements for dossier evaluation is one of the critical components. Majority of the NRAs refer to international ICH guidelines and mandate CTD dossier format, both of which can be leveraged on to bridge the gaps. • While ICH forms the point of convergence among the NRAs, there are various barriers that can be encountered in their implementation resulting in significant differences in the developed frameworks. • With many regulatory agencies lacking expertise in ATMP, education and training workshops focusing on specific ICH guidelines for the evaluation of ATMP quality, efficacy and safety are valuable to train professionals reviewing ATMP dossiers. • Apart from workshops, roundtables also provide an opportunity for regulators to interact, share knowledge from their experiences thus providing learning opportunities for NRAs who are in the process of developing ATMP frameworks. • There is immense potential to exploit the commonalities in the regulatory structures governing ATMPs. Education, training as well as enhancing dialogues among regulators are the short term interventions to achieve the long term regulatory convergence goals.