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This guideline provides specific considerations for the design, conduct, oversight, and long-term follow-up of clinical trials involving Advanced Therapy Medicinal Products (ATMPs) in the Kingdom of Saudi Arabia.
It supplements the SFDA Guideline for Good Clinical Practice (GCP) and shall be read in conjunction with the Regulations and Requirements for Conducting Clinical Trials on Drugs, ICH E6(R3), and other applicable SFDA guidance documents.
While this document does not have the force of law, compliance with its principles is expected for all ATMP clinical trials submitted to or conducted under SFDA oversight. Alternative approaches are acceptable if supported by adequate scientific justification and prior discussion with SFDA.
As a corollary to the above, it is equally important to note that SFDA reserves the right to request information or material, or define conditions not specifically described in this guidance, in order to allow the department to adequately assess the safety, efficacy or quality of a therapeutic product. SFDA is committed to ensuring that such requests are justifiable and that decisions are clearly documented.
Advanced Therapy Medicinal Products (ATMPs) represent a rapidly evolving class of medicinal products offering transformative therapeutic potential. ATMPs are classified into the following: Gene therapy medicinal product, Cell based medicinal product (includes both somatic cell therapy medicinal products and tissue engineered products) and Combined ATMP products contain as an integral part of the product also a medical device.
Due to their unique biological nature, complex manufacturing, short shelf life, potential for lifelong effects, and donor-recipient traceability requirements, ATMP clinical trials require oversight tailored to their specific characteristics beyond general Good Clinical Practice (GCP) principles.
The Kingdom of Saudi Arabia, through Vision 2030 and the National Biotechnology Strategy, is committed to becoming a regional leader in regenerative medicine. To ensure a consistent, science-based regulatory approach aligned with international standards, the Saudi Food and Drug Authority has developed this guideline to provide additional requirements for the conduct, monitoring, and oversight of ATMP clinical trials, complementing the existing general requirements.
1.1. Purpose and Scope
This guideline provides additional requirements for the conduct, monitoring, and oversight of clinical trials involving ATMPs, including gene therapy medicinal products (GTMPs), cell based medicinal products (CBMPs), and combined ATMPs, including in vivo and ex vivo gene-editing products (e.g. CRISPR-based therapies).
It aims to ensure participant safety, data integrity, and regulatory consistency while recognizing the unique characteristics of ATMPs such as short shelf life, biological variability, donor-related variability, and long-term effects.
1.2. Related guidelines
This document should be read in conjunction with the following guidelines:
- Regulations and Requirements for Conducting Clinical Trials on Drugs
- ICH E6(R3) Good Clinical Practice
- ICH E8(R1) General Considerations for Clinical Studies
- Guidance on Submission of Chemistry, Manufacturing, and Control (CMC) Information for Cell-based Clinical Trial Applications.
- Guidance on Submission of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Clinical Trial Application.
- Classification of Advanced Therapy Medicinal Products
- ICH Q9 (R1) Quality Risk Management
Where differences exist, this guideline prevails for ATMP trials conducted under SFDA oversight.
1.3. Definitions and Classification
Classification shall follow SFDA’s Guideline on Classification of Advanced Therapy Medicinal Products and harmonize with internationally accepted definitions and its amendments.
Sponsors and investigators shall ensure end-to-end chain of identity and custody from donor collection (if applicable) to recipient administration. Unique identifiers (e.g. ISBT-128 labelling) should be used whenever feasible. Storage and transport must maintain product viability and biological integrity, using validated and controlled conditions (e.g., cryogenic or temperature-controlled systems with continuous monitoring and alarm systems). Deviation management procedures shall address temperature excursions, timing deviations, or on-site preparation failures and shall include predefined contingency actions.
Complete traceability from donor to recipient and vice versa shall be ensured for all human-derived materials. Records must enable retrieval of donor information, processing details, batch identifiers, and recipient data. Sponsors shall ensure data systems comply with the Saudi Personal Data Protection Law. Where human cell or tissue donors are used, full traceability is required from starting materials and raw materials, including all substances coming into contact with the cells or tissues through to confirmation of the receipt of the products at the point of use whilst maintaining the privacy of individuals and confidentiality of health-related information. Traceability records must be retained for a minimum of 30 years after the expiry date of the product. Particular care should be taken to maintain the traceability of products for special use cases, such as donor-matched cells. National requirements apply to blood components when they are used as supportive or raw material in the manufacturing process of medicinal products. For ATMPs, traceability requirement regarding human cells including haematopoietic cells must comply with the principles laid down in applicable national legislation. The arrangements necessary to achieve the traceability and retention period should be incorporated into technical agreements between the responsible parties.
Where appropriate, information should be provided in the Protocol and the Investigator's Brochure on the measures that should be put in place to protect clinical trial subjects from identified risks. The following are some non-exhaustive examples:
- If the results of the sterility test of the product are not available at release, appropriate mitigation measures should be described, including liaison with clinical staff where out of specification test results (for sterility) are obtained after the release of the product.
- If there is a risk that a subject that has received an investigational ATMP develops cytokine release syndrome, the investigator should be informed about measures that should be in place before treating the patient (e.g. availability of IL-6 inhibitors and Intensive care capabilities).
Sponsors shall design and implement long-term safety follow-up (LTFU) plans proportionate to the risk profile of the ATMP.
For gene therapy and most cell-based products, follow-up should generally extend from 5 to 15 years post-administration, for products containing integrating vectors or genome-editing components, follow-up of at least 15 years or for the lifetime of the patient is recommended. The plan shall include: endpoints for delayed events (e.g., tumorigenicity, vector persistence, off-target editing, new malignancy or autoimmunity), data collection mechanisms, and investigator responsibilities for late adverse event reporting. For gene therapy medicinal products and genome-editing ATMPs, a stand-alone LTFU protocol shall be submitted. For other ATMPs it is encouraged.
ATMP manufacturing must comply with GMP standards applicable to ATMPs. Process changes or site transfers during development require a comparability assessment demonstrating maintenance of quality, safety and clinical relevance. The Investigational Medicinal Product Dossier (IMPD) shall describe donor source, manufacturing steps, stability, release criteria, viral safety evaluation, and comparability studies.
The sponsor shall perform an ATMP-specific risk assessment covering manufacturing, supply chain, and clinical use. Monitoring and quality management activities must be proportionate to the identified risks and include chain-of-custody verification and control of in-process handling. Critical-to-quality factors unique to ATMPs should be addressed through a risk-based quality system aligned with ICH E8(R1) and ICH Q9(R1).
In addition to standard GCP requirements, the informed consent process shall ensure that participants are informed about:
- The novel nature and mechanism of the ATMP.
- Potential long-term or unknown risks including the possibility of lifelong consequences.
- Extended follow-up obligations and potential re-contact.
- Future scientific use of remaining tissues or cells.
- Genetic modification or vector-related risks (if applicable) and implications for the patient.
For ATMPs containing or consisting of genetically modified organisms (GMOs), the sponsor shall provide an environmental risk assessment and biosafety management plan approved by SFDA prior to trial initiation. Sites must apply containment, waste disposal, and staff protection measures suitable for the risk class of the product.
When an ATMP is combined with a medical device or scaffold, the sponsor must describe the device's classification, source, and role in accordance with SFDA medical device regulations. Clinical data must demonstrate safety and performance of the combination. Device–product interactions shall be evaluated in the IMPD.
Sponsors remain responsible for post-trial monitoring, long-term safety reporting, and communication of new risk information to participants even after trial completion or marketing authorization. Participants should be informed of clinically relevant results or follow-up findings. SFDA may require establishment of or participation in national registries to track long-term ATMP outcomes.
- ICH E6(R3): Good Clinical Practice
- ICH E8(R1): General Considerations for Clinical Studies
- EMA Guideline on Good Clinical Practice for ATMPs (2019)
- EMA Guideline on Quality, Non-Clinical and Clinical Requirements for ATMPs (2025)
- WHO TRS No. 1016 (2023): Cell and Gene Therapy Considerations
- ICH Q5E Comparability of Biotechnological/Biological Products Subject to Changes in their Manufacturing Process
Last update: 10 August 2026
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