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2024-07-16| Special

Robust Regulatory Framework Paves the Way for Breakthrough in Regenerative Medicine

by Richard Chau
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With the aim of enhancing the legal compliance of Taiwan’s biomedical industry, under the guidance of the Department of Industrial Technology of the Ministry of Economic Affairs, the Science and Technology Law Institute (STLI) has commissioned GeneOnline to host a series of four thematic webinars covering regulatory issues related to regenerative medicine, nucleic acid drugs, AI and digital technologies in the biopharma industry, as well as health technology assessment. Through the sharing of updates on biotech regulations and their implementation by international experts, coupled with interactive discussions with local industry elites, it is hoped that participants will be able to grasp the key compliance issues of advanced medical products around the world, drawing on experiences of global players to promote the industry’s connection with the international market.

Related article: Taiwan Breakthrough: Next-Generation Sequencing Now Covered in Health Insurance, Benefitting 20,000 Cancer Patients Annually 

Enhancing Taiwan’s Biotech Compliance Through Regulatory Webinars

The first webinar took place on July 3 with more than 600 people joining the event. Dr. Shin Kawamata, CEO of Cyto-Facto, a Japanese cell and gene therapy CDMO company, and Dr. Christopher Tsai, Chairman of the BIONET Group, were invited as speakers for the event, titled “Cellular Revolution – Trends in Gene Therapy and Regenerative Medicine Regulations”.

In her welcoming speech, Huang Yu-Ying, Director of the Biomedical and Healthcare Division of STLI, mentioned that Taiwan has witnessed a remarkable development of advanced medical products in the post-COVID era. With the passage of the Regenerative Medicine Act and the Regenerative Pharmaceuticals Act (collectively referred to as the “Regenerative Medicine Acts”) in the Legislative Yuan in early June, Taiwan is now at the forefront of this biomedical revolution. Through the organization of international regulatory forums, the Institute expects to bring together experts from different countries to provide a platform for knowledge exchange and information sharing, to promote international biopharma partnerships, and to foster sustainable innovations in the industry.

Cell Therapy Expert Speaks on Regenerative Medicine Regulations in Japan

Dr. Shin Kawamata first shared the current status of the Japanese cell and gene therapy market and the evolution of its regulatory framework. As an international expert in this field, he has been committed to promoting the development of regenerative medicine in Japan and updating related regulations for many years. At the beginning of his speech, he pointed out that the Japanese cell and gene therapy market is multiplying. Some analysts estimate that the market will reach ¥260 billion (approx. $1.65 billion) by 2024 and is expected to exceed ¥1 trillion (approx. $6.34 billion) over the next 10 years. Many Japanese biotech companies have also invested funds in developing innovative therapies in this field in recent years, with cell transplantation, tissue transplantation and in vitro gene therapy being the most popular, and the main indications include neurological diseases, cardiovascular diseases, cancers, as well as endocrine and metabolic diseases.

As a leader in regenerative medicine in the Asia-Pacific (APAC) region, Japan has enacted three acts related to regenerative medicine since 2013, namely, the Regenerative Medicine Promotion Act, the Act on the Safety of Regenerative Medicine (Safety Act), and the Act on Pharmaceuticals and Medical Devices (PMD Act). These laws and regulations not only clearly define the types of regenerative medicine products, but also provide legal guidelines in various areas regarding these products, such as research and development, manufacturing, clinical trials, regulatory approvals, commercialization and applications, as well as safety and ethical considerations. In his speech, Dr. Kawamata explained the role of these regulations in the regenerative medicine industry in Japan.

Major Laws Governing Regenerative Medicine Development in Japan

The Regenerative Medicine Promotion Act is a comprehensive bill to promote policies on regenerative medicine from R&D to implementation and enable the public to access these innovative medical products quickly and safely. The other two laws constitute a dual-track regulatory system governing different practical aspects of regenerative medicine in Japan. 

The Safety Act regulates medical institutions and clinicians who perform cell and gene therapies. According to the Act, treatments are divided into three classes based on the level of risk. Those with the highest risk (e.g. embryonic stem cells, iPS cells, etc.) are in Class 1, those with moderate risk (e.g. somatic stem cells, mesenchymal stem cells, etc.) are in Class 2, while those with the lowest risk (e.g. autologous cell therapy for cancers, medical cosmetics, etc.) are in Class 3. In general, researchers, hospitals or doctors have to apply for approval by a Certified Committee for Regenerative Medicine before treatment implementation. The level of risk determines the stringency of the review process and places where the treatment can be performed. Class 3 treatments have relatively lenient criteria and can be performed in a clinic, while those in Class 1, the riskiest of all, require a two-pronged review by a Certified Special Committee and the Ministry of Health, Labor and Welfare before implementation in designated hospitals.

In response to the rise of regenerative medicine, the Japanese government amended the PMD Act by adding a chapter on the regulation of cell and gene therapies, which mainly targets biotech companies and pharmaceutical companies and covers the approval process of such products, their manufacturing and sales, insurance coverage, and post-marketing safety measures. Under the existing regulatory framework, pharma companies can apply for time-limited conditional approval for their regenerative medicine products without completing clinical trials. Following commercialization, companies have to continue to collect clinical data and adopt post-marketing safety measures to verify the efficacy and safety of their products, and then apply for formal approval before the conditional approval expires. This shortcut will enable cell and gene therapies to enter the market faster and benefit patients in need.

Dr. Shin Kawamata, CEO of Cyto-Facto, explains the current “Three Laws of Regenerative Medicine” in Japan – the Regenerative Medicine Promotion Act, the Act on the Safety of Regenerative Medicine, and the Act on Pharmaceuticals and Medical Devices (PMD Act). (Source: Screenshot from speaker’s presentation)

Transforming Cell Manufacturing Processes Through Digital Innovations

Next, Dr. Kawamata shared Cyto-Facto’s contributions to the development of regenerative medicine in Japan since its establishment in October 2022. Located in the Kobe Biomedical Innovation Cluster (KBIC), Japan’s largest biomedical industry cluster, Cyto-Facto managed to spin off from the Foundation for Biomedical Research and Innovation at Kobe (FBRI) to achieve full commercialization. The company also became the first Asian biotech company to manufacture Kymriah, a CAR-T cell therapy developed by Novartis, under the PIC/S GMP standard with a 95% success rate. In addition to R&D of innovative CAR-T cell therapies, Cyto-Facto also provides cell and gene therapy CMO/CDMO services.

Dr. Kawamata also mentioned the difficulties in quality control of cellular therapies compared to conventional drugs, given the considerable differences in the characteristics of cells in each batch. Therefore, by digitizing the cell manufacturing process, creating electronic batch production records and sharing the data with pharma and clinical trial sites, it is possible to alleviate the difficulties in quality control and boost the success rates. Also, these innovations ensure greater transparency and traceability of each part of the process, thereby earning the trust of pharma companies and regulatory authorities. 

With this in mind, Cyto-Facto is actively incorporating novel technologies including AI, the Internet of Things (IoT), automation, robotics and cloud data analytics into its manufacturing processes. In addition, the company has introduced an intelligent cell processing system called CellQualia to optimize the manufacturing and quality control of cell-based products. It includes a closed automated cell culture system, real-time imaging tools, in-process medium analysis, automated sampling and off-line analysis, and other cutting-edge technologies to realize the concept of “Quality by design (QbD)” and minimize the impact of variations in raw materials and cell culture on the manufacturing process.

As a concluding remark, Dr. Kawamata highlighted Taiwan’s unique geographic advantage, which makes it an important hub connecting major markets such as Japan, China, Singapore, Australia, Europe and the United States. Coupled with its enormous strength in the information and communications technology industry over the years, Taiwan not only holds immense potential for the development of regenerative medicine, but also for linking to the global market with cell manufacturers like Cyto-Facto, which are compliant with the PIC/S GMP standards. He envisioned a closer tie with Taiwan’s biotech and ICT companies to build a cross-border cell and gene therapy CMO/CDMO ecosystem based on the Taiwan-Japan partnership.

Related article: Advancing the Frontiers of Cell and Gene Therapy – An Interview with Dr. Shin Kawamata

BIONET’s Founder Explains Taiwan’s Regenerative Medicine Regulatory Framework

As the second speaker of the event, Dr. Christopher Tsai, Founder and Chairman of the BIONET Group, began his speech by stating how advanced economies such as Europe, the United States, Japan, and Korea have already established authorities for the regulation of regenerative medical products, leading to increasingly mature regulatory systems. Regarding R&D and clinical trials, the APAC region has been an active destination, second only to North America, with Japan and Korea taking the lead. 

Dr. Tsai agreed with Dr. Kawamata’s view that the closer ties between Taiwan and Japan will make them a global hotspot for regenerative medicine development for years to come. In terms of regulatory framework, he pointed out that in Japan and Korea, for example, the number of cell therapy products approved after the passage of related laws has grown significantly, allowing more patients with severe and rare diseases to receive new treatments. Referring to the successful experience of Japan and Korea, he opined that with the passage of Taiwan’s Regenerative Medicine Acts, the local regenerative medicine industry is also poised to thrive.

Regenerative Medicine Acts Hold Promise to Boost Taiwan’s Biotech Industry

Speaking about Taiwan’s Regenerative Medicine Acts, Dr. Tsai said that the new regulations are very similar to the existing ones in Japan, covering not only the regulatory system for pharmaceutical products and therapeutic modalities, but also practical issues including product safety, implementation guidelines, and patient recruitment in clinical trials. The combination of these elements will create a comprehensive legal framework for Taiwan that can bring benefits in five different ways. 

To begin with, the new legislation covers both extracellular vesicles and exosomes. Although this area is still in its infancy globally, Taiwan’s biotech industry and medical community are increasingly engaged in related R&D activities. With the government’s enactment of specific regulations on exosomes, Taiwan has the potential to take a leading role in exosome research.

Next, Dr. Tsai commented that the Regenerative Medicine Acts will facilitate collaborations between industry, academia, research and medical communities. Citing statistics from the Ministry of Health and Welfare (MOHW), he indicated that since the promulgation of the amended Regulations of Specific Medical Techniques or Examinations, or Medical Devices (collectively referred to as the “Regulations of Specific Medical Techniques”) in 2018, which loosened the regulations on cell therapies, as of the end of March 2024, over 500 applications for the implementation of cell therapies have been submitted to the MOHW, and among them 345 cases have been approved. Also, as many as 120 clinical trials related to regenerative medicine have been approved, with 87 of them involving different types of cell therapies, representing a marked increase compared to the days before the Regulations of Specific Medical Techniques came into force. This has laid a good foundation for cross-disciplinary collaborations in the field of regenerative medicine. 

Moreover, Dr. Tsai emphasized that the new regulations will attract more doctors, scholars and biopharma professionals to engage in research activities, thereby nurturing a new generation of talents in regenerative medicine. Meanwhile, with the increase in clinical R&D capacity, there will be a growing number of new pharmaceuticals utilizing stem cells, immune cells, gene therapy, or exosomes in the foreseeable future, and the cost of treatment is expected to be reduced accordingly, giving hope to patients plagued by various severe and rare diseases.

In closing, Dr. Tsai mentioned that the National Health Insurance Administration, MOHW included next-generation sequencing (NGS) testing for 19 types of solid or hematological tumors in Taiwan’s health insurance coverage starting from May 1, 2024, making it possible for cancer patients to receive more accurate diagnoses at affordable prices and for physicians to identify patients’ genetic mutations more effectively. Following the implementation of the new Regenerative Medicine Acts, the combination of these two policies will bring about a strong synergy that allows doctors and cancer patients to benefit from more personalized and precise therapeutic options, which will not only enhance the effectiveness of cancer prevention and treatment, but also improve patients’ survival and quality of life.

Dr. Christopher Tsai, Founder and Chairman of the BIONET Group, shared in his speech the five major areas of benefits brought about by Taiwan’s latest Regenerative Medicine Acts. (Source: Screenshot of speaker’s presentation)

Following the successful completion of the first webinar of the series, the other three sessions will be held on July 24, August 7 and August 28, 2024 respectively. Registration for each session will be open soon and is free of charge. All biopharma professionals are cordially invited to participate in these events. Please check the latest updates from the official website of STLI or GeneOnline. 

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