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Chile's Ministry of Health has approved the country's first dedicated technical standard for advanced therapy medicines, a category that includes gene therapies, cell therapies and tissue engineering products, bringing the country in line with international regulatory practice for some of the most complex and rapidly evolving treatments in modern medicine.

Technical Standard No. 257, establishes the quality, safety, manufacturing and registration requirements that apply to these products throughout their entire life cycle, from laboratory development through to clinical use.

Advanced therapy medicines are biological pharmaceutical products composed of cells or tissues that have been substantially manipulated or repurposed, used to treat, prevent or diagnose disease. The standard recognises four internationally accepted categories: somatic cell therapies, tissue engineering products, gene therapies, which include products containing DNA, non-viral vectors, viruses or genetically modified cells, and combined advanced therapy medicines that incorporate a medical device as part of their structure.

Officials cited the exceptional complexity of these products as the justification for a dedicated regulatory framework. Unlike conventional pharmaceuticals, advanced therapies must be manufactured, stored, transported and administered under highly controlled conditions, requiring specialist infrastructure, rigorous biosecurity protocols, qualified scientific personnel and a deep understanding of the biological behaviour of their therapeutic components.

The standard aligns Chilean requirements with those of leading international agencies including the European Medicines Agency, Spain's AEMPS, Japan's PMDA and Brazil's ANVISA, and will be administered by the Instituto de Salud Pública de Chile.

All advanced therapy medicines manufactured or imported into Chile must now obtain health registration with the institute before they can be used clinically. The standard also provides a pathway for provisional authorisation of products still under clinical investigation, recognising that some therapies, particularly those with very short efficacy windows, such as certain cell products, may need to be administered before all quality control processes can be fully completed.

The rules distinguish between industrial-scale manufacture and non-industrial production, the latter covering treatments prepared within hospitals, clinics or academic institutions for individual patients or small groups within the framework of a clinical study. Non-industrial facilities may outsource quality control testing to authorised external laboratories, but products prepared under this route may only be distributed within the clinical centre where they will be used.

Laboratories wishing to produce advanced therapies must demonstrate physical and operational independence from other pharmaceutical manufacturing areas, maintain dedicated cryogenic storage systems for biological starting materials, and appoint a designated biosafety officer independent from production and quality control staff. Technical directors of such laboratories may be pharmaceutical chemists, biotechnology engineers or specialist physicians.

The standard explicitly excludes from its scope conventional bone marrow transplants, blood and blood-derived products, organ transplantation and the use of autologous cells transplanted during the same surgical procedure without substantial manipulation.

Advanced therapy medicines such as CAR-T cell cancer treatments and gene therapies for rare diseases have transformed treatment options globally over the past decade, but their regulatory complexity has meant that many countries in Latin America have lacked a dedicated framework to govern them. Chile's new standard represents one of the region's most comprehensive approaches to the area.