D. Renovaldi, M.Sc
International Ph.D Program in Medicine, Taipei Medical University

Introduction

Cell and Gene Therapy (CGT) represents a rapidly advancing field in biomedical science, offering innovative treatment strategies for diseases that were previously difficult or impossible to treat. These therapies involve the modification or administration of living cells or genetic material to restore normal biological functions, regenerate damaged tissues, or enhance immune responses against diseases such as cancer. Due to their complex biological nature and potential risks, CGT products require rigorous regulatory oversight to ensure their safety, quality, and efficacy.

In Indonesia, the regulation of CGT products is primarily overseen by the national drug regulatory authority, the Badan Pengawas Obat dan Makanan (BPOM). BPOM is responsible for the evaluation, approval, and monitoring of pharmaceutical and biological products before and after they are introduced into the market. Recognizing the rapid development of advanced therapies, BPOM issued the BPOM Regulation No. 8 of 2025 on Guidelines for the Evaluation of Advanced Therapy Products to establish a regulatory framework specifically addressing advanced therapeutic products, including cell and gene therapies. This regulation aims to ensure that innovative therapies are developed and implemented responsibly while maintaining patient safety.

Scope and Definition of Cell and Gene Therapy Products

Within the Indonesian regulatory system, CGT products fall under the broader category of Advanced Therapy Products. According to BPOM Regulation No. 8 of 2025, advanced therapy products include three main types: somatic cell therapy products, gene therapy products, and tissue-engineered products.

• Somatic cell therapy products consist of cells or tissues that have undergone significant manipulation or are used for purposes different from their original biological functions. These products exert therapeutic effects through pharmacological, immunological, or metabolic mechanisms. Examples include modified immune cells used in immunotherapy or expanded stem cells used for regenerative medicine.
• Gene therapy products involve the introduction, removal, or modification of genetic material within a patient’s cells. These products typically contain recombinant nucleic acids that regulate or modify gene expression to treat or prevent diseases. Gene therapy has shown promise in treating genetic disorders, cancers, and rare diseases.
• Tissue-engineered products, on the other hand, are designed to repair, regenerate, or replace damaged human tissues. These products may involve cells combined with scaffolds or biomaterials to support tissue growth and structural integrity.

However, the regulation does not include certain biological products such as vaccines for infectious diseases, conventional blood products, or products derived from embryonic stem cells. By defining these categories, BPOM establishes a clear regulatory scope for advanced therapies within Indonesia.

Regulatory Authority and Institutional Framework

The primary regulatory authority responsible for the oversight of CGT in Indonesia is BPOM. As the national agency responsible for drug and food control, BPOM has the mandate to regulate pharmaceutical products throughout their lifecycle. Its responsibilities include pre-market evaluation, authorization of clinical trials, approval of marketing applications, and post-market surveillance.

The authority of BPOM in regulating pharmaceutical and biological products is supported by national legal frameworks such as the Presidential Regulation No. 80 of 2017 on the National Agency of Drug and Food Control. Under this framework, BPOM ensures that all therapeutic products entering the Indonesian market meet established standards of safety, quality, and efficacy.

In addition to BPOM oversight, CGT development in Indonesia also involves collaboration with other institutions, including medical research institutions, hospitals, and ethics committees that evaluate the ethical aspects of clinical trials involving human subjects.

Regulatory Pathway for CGT Products

The development and approval of CGT products in Indonesia involve several stages of regulatory evaluation. These stages generally include preclinical research, manufacturing control, clinical trials, and marketing authorization.

Preclinical Development

Before a CGT product can enter clinical trials, developers must conduct extensive preclinical studies to evaluate its biological characteristics, safety, and therapeutic potential. These studies typically involve laboratory experiments and animal models. Developers must demonstrate that the product has an acceptable safety profile and a clear scientific rationale for its therapeutic use.

BPOM adopts a risk-based approach when evaluating advanced therapy products. Several risk factors are considered, including the origin of the cells, the degree of manipulation involved, the possibility of immune reactions, and the potential for uncontrolled cell growth or tumor formation.

Manufacturing and Quality Control

Manufacturing of CGT products must comply with Good Manufacturing Practices (GMP), which in Indonesia are referred to as Cara Pembuatan Obat yang Baik (CPOB). These standards ensure that biological products are consistently produced and controlled according to strict quality requirements.

For CGT products, manufacturing oversight is particularly important due to the variability of living cells and biological materials. Regulatory authorities require detailed documentation regarding the source of cells, methods of cell processing, storage conditions, sterility testing, and product characterization. Facilities involved in cell processing and manufacturing must meet specific regulatory standards and obtain appropriate certification.

Clinical Trial Authorization

Clinical trials are required to evaluate the safety and effectiveness of CGT products in human subjects. In Indonesia, all clinical trials involving investigational therapies must receive approval from BPOM before they can begin.

In addition, the research protocol must be reviewed by an institutional ethics committee to ensure compliance with ethical standards.

Clinical trials are typically conducted in multiple phases to evaluate different aspects of the therapy, including safety, optimal dosage, and therapeutic efficacy. Due to the complex nature of CGT products, long-term follow-up studies are often necessary to monitor potential delayed adverse effects.

Marketing Authorization

After successful clinical trials, developers may apply for marketing authorization from BPOM. The regulatory submission must include comprehensive data on product quality, preclinical research, and clinical trial results. BPOM evaluates the benefit-risk profile of the product before approving commercial use.

Because CGT products are highly innovative and may involve unique manufacturing processes, BPOM may conduct case-by-case evaluations based on the specific characteristics of the therapy.

Alignment with International Regulatory Standards

Indonesia’s regulatory framework for CGT is increasingly aligned with international guidelines. BPOM frequently refers to regulatory approaches used by global agencies such as the World Health Organization, the European Medicines Agency, and the U.S. Food and Drug Administration when evaluating advanced therapy products.

Adopting international standards helps ensure that CGT products developed or marketed in Indonesia meet globally recognized safety and quality requirements. This alignment also facilitates international collaboration in clinical research and biotechnology development.

Conclusion

Cell and Gene Therapy represents a transformative approach in modern medicine, but its complexity requires comprehensive regulatory oversight. In Indonesia, BPOM plays a central role in ensuring that CGT products are developed, evaluated, and monitored according to strict safety and quality standards. Through BPOM Regulation No. 8 of 2025, Indonesia has established a structured regulatory framework that addresses the unique challenges associated with advanced therapeutic products.

References

• Badan Pengawas Obat dan Makanan Republik Indonesia. 2025. Peraturan Badan Pengawas Obat dan Makanan Nomor 8 Tahun 2025 tentang Pedoman Penilaian Produk Terapi Advanced. Jakarta: BPOM.
• Badan Pengawas Obat dan Makanan Republik Indonesia. 2022. Peraturan Badan Pengawas Obat dan Makanan Nomor 13 Tahun 2022 tentang Perubahan atas Peraturan BPOM Nomor 21 Tahun 2020 tentang Organisasi dan Tata Kerja Badan Pengawas Obat dan Makanan. Jakarta: BPOM.