Cell and Gene Therapy Products are modern treatments to treat diseases. Treatment of these products is typically aimed at the underlying cause of the problem, rather than the symptoms.
CGTs are applied, for example, in the field of cancer, rare genetic diseases, and regenerative medicine. These products are used in living cells and with modified genes, so there are certain regulations applicable to these products throughout the world. Everything from clinical trials, manufacturing, product approval, to patient safety is regulated for these products. Interested in more specifics of the rules? Read the blog below.
What are Cell and Gene Therapy Products?
Cell and gene therapy products include the usage of human cells or genetic material that are used to treat, prevent, and potentially cure diseases.
- Cell therapy is a part of using living cells to repair or replace damaged cells.
- Gene therapy includes adding, changing, or replacing genes. It helps improve the functioning of the body.
These include, for instance, CAR-T cell therapy, stem cells, and therapies for inherited genetic disorders.
Types of CGT Products
The types of CGT products differ in how they work and what they contain. The types include:
- Cell Therapy Products
Cell therapy products use living cells to repair or replace damaged cells or tissues. Some of these are stem cell therapies or CAR-T therapies.
- Gene Therapy Products
Alter or fix genes to cure disease, particularly inherited diseases and some cancers.
- Tissue-Engineered Products
Play a role in the restoration, repair, or regeneration of skin, cartilage, or bone.
- Combined Products
Mix cells or tissues with medical devices or biomaterials to be used for treatment.
How CGT Products are Classified Globally
CGT products are subject to different special categories of regulation in various countries.
| Region | Classification | Categories Included |
| United States | Human cells, tissues, and human cellular and tissue-based products (HCT/Ps) and biologics | Cell therapies, gene therapies, tissue products |
| European Union | Medicinal products for Advanced Therapy (ATMPs) | Gene therapies, somatic cell therapies, tissue-engineered products, combined ATMPs |
| United Kingdom | Advanced Therapy Medicinal Products (ATMPs) | Cell and drug therapies, tissue-engineered products, Gene Therapies |
| Japan | Regenerative Medical Products | Cell therapies and gene therapies |
| India | Cell & Gene Therapy Products (CGTPs) | New therapies are based on cells and genes. |
Why CGT Products Require Specialized Regulations
CGT products are more complicated than conventional medicines and need special regulations in this respect since they are based on living cells or modified genes. They also need to have tighter regulations for their production, holding, and long-term safety monitoring procedures.
Regulatory Authorities for CGT Products
Various countries have specific health regulatory bodies and regulations for CGT products in their jurisdiction. The table below provides an overview of the regulatory landscape in key markets.
Current Status of CGT Regulations in Major Markets
Below are the global cell and gene therapy regulations status:
| Country/Region | Regulatory Authority | Product Terminology | Current Regulatory Status |
| European Union | EMA | Advanced Therapy Medicinal Products (ATHMPs) | Demystified the complex pathway for approval into the Established ATMP pathway. |
| United States | FDA (CBER – OTP) | Immune cell or Biopharma Gene products (cell and gene therapy products / HCT/Ps) | Still developing framework with streamlined approval processes |
| Singapore | HSA | Cell, Tissue, and Gene Therapy Products (CTGTPs) | A proper, dedicated framework under the Health Products Act focusing on human health and safety. |
| China | NMPA | Cell and Gene Therapy Products | An advanced and evolving structure in this area, in which there is increased supervision. A rapidly changing framework with increased oversight. |
| Japan | PMDA & MHLW | Regenerative Medical Products | Conditional and time-limited approval system |
| Thailand | Thai FDA | Cell and Gene Therapy Products | Developing a regulatory framework |
| Taiwan | TFDA | Cell and Gene Therapy Products | The existing framework and guidance for formal CGT |
| India | CDSCO & ICMR | The first course on the Cell and Gene Therapy Products (CGTPs) is planned for late September/early October. | One functional design – backed up by national guidance – is appearing. |
| Brazil | ANVISA | Advanced Therapy Products | There are special advanced therapy regulations. |
| Mexico | COFEPRIS | Advanced Therapies / Biologics | Moving from case-law to regulatory control. Development of case law regulatory elements. |
| South Korea | MFDS | Advanced Biopharmaceuticals | Integrated framework, Advanced Regenerative-Bio Act. |
| Australia | TGA | Biologicals and Gene Therapies | Established risk-based framework |
| Canada | Health Canada | Cell and Gene Therapies | The Biologics Framework regulates them. |
| United Kingdom | MHRA | Preparing advanced therapy medicinal products (ATMPs) | ATMP independent UK framework |
| Saudi Arabia | SFDA | Advanced Therapy Medicinal Products | Developing regulatory pathways |
| United Arab Emirates | MOHAP | Cell and Gene Therapies | Emerging regulatory oversight |
| Malaysia | NPRA | Cell and Gene Therapy Products | Ensuring that a technical guidance framework is developed |
| Indonesia | BPOM | Advanced Therapy Products | Early-stage regulatory framework |
| Argentina | ANMAT | Pharmaceutical products for Advanced Therapy. | Expanding regulatory oversight |
| Switzerland | Swissmedic | Advanced Therapy Medicinal Products (ATMPs). | Modern equipment system, conformant with the European standards. |
Regulatory Checklist for CGT Products
Typical regulatory specifications for CGT products typically involve:
- Determine the classification of the product and the evaluation of the pathway through which it will access the market.
- Preclinical safety studies
- Donor screening and Raw material controls
- Ethical and clinical trial approvals.
- Compliance with GMP requirements
- The identification of products, their purity, and potency.
- Specific testing of virus safety and sterility.
- They are subjected to a process of validation of stability and storage.
- Long-term safety monitoring
- Pharmacovigilance and Adverse Event Reporting System.
- Submissions for marketing authorization or product approval.
Clinical Trial Requirements for CGT Products
Clinical trials for CGT products are different from those for traditional drug products because they are composed of living cells or genetic modifications that can have long-term effects.
Typically, they must do the following:
- The pre-clinical safety and biodistribution studies.
- This barometer-related indicator is measured prior to clinical use in the process of manufacturing a product that has GMP Compliance.
- Testing for product identity, purity, and potency.
- Ethics committee and regulatory approvals
- Long-term patient follow-up
- Traceability of products and materials from donors
There are some regional variations, such as:
| Region | Key Difference |
| United States | For some gene therapies, the FDA could ask for as long as 15 years of long-term monitoring. |
| European Union | Risk-based Assessment & Centralized EMA review. |
| Japan | Time or conditionally approved medicines for regeneration |
| China | Increasingly tough manufacturing controls and clinical monitoring |
| India | Guidance for Development provided by CDSCO & ICMR, following the guidelines |
Expedited Approval Programs for CGT Products
Countries have alternative processes to approve CGT products to treat rare, severe, or life-threatening conditions.
| Region | Expedited Program |
| United States | Rate marks, alternatives, Breakthrough Therapy, Priority Review (R/F/B/T) |
| European Union | PRIME |
| Japan | Conditional and Time-Limited Approval |
| United Kingdom | ILAP |
| South Korea | Easy appointment for Advanced Biopharmaceuticals review. |
There are still studies to be confirmed and pathways to undergo monitoring following their approval.
Manufacturing and GMP Requirements for CGT Products
The manufacturing and GMP Requirements for CGT Products are as follows.
Products of CGT are subject to more stringent GMP controls due to the presence of living cells, tissues, or genetic material in the product.
General GMP Standards are:
- GMP-certified manufacturing facilities
- Analysis of the risks and hazards of food in everyone’s role in food safety.
- Sterile processing and contamination control.
- The screening of donors and raw materials is carried out.
- Identity, purity, and potency testing
- Chain of identity/custody tracking
- The control of cold-chain storage and transport
- Batch traceability and quality documentation, tracking of work batches, and the record of work quality.
There are also CGT-specific GMP guidelines in other countries, including the US, EU, Japan, and South Korea.
Conclusion
General regulatory frameworks for clinical trials, manufacturing, GMP compliance, and long-term safety monitoring apply to cell and gene therapy (CGT) products. While global regulatory systems are converging, there is still some regional variation in product classification, how products are approved, and post-market requirements.
Artixio provides regulatory strategy, clinical trial support, GMP compliance, pharmacovigilance, and market access services to biotech and life sciences companies for CGT products in some of the largest markets.
Want to make your cross-border CGT regulatory technique and approval easier? Call Artixio, phone number: info@artixio.com.
FAQs
What are ATMPs?
A category of EU regulations, Advanced Therapy Medicinal Products (ATMPs), refers to the terms used for advanced therapies comprising gene or cell therapies, tissue-engineered products, and combined products.
Who regulates the products of CGT?
It can be different based on the country. Examples include FDA (US), EMA (EU), PMDA (Japan), MHRA (UK), CDSCO (India), and NMPA (China).
Why do we need long-term follow-up of the patients in the CGT?
The delayed effects or long-term risks of some CGT products may necessitate the need for patient monitoring for several years after administration by the regulators.
