EMA Guideline on Quality, non-clinical and clinical Requirements for Investigational Medicinal Products for Advanced Therapies in Clinical Trials

The EMA's “Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials” provides comprehensive guidance on the structure and data requirements for clinical trials of ATMPs, including gene therapy products, cell-based therapies and tissue-engineered products. It covers various aspects including manufacturing, quality control, non-clinical and clinical development phases. A particular focus is on the requirements for exploratory studies, including first-in-human studies, as well as confirmatory studies.

Quality Documentation

The guideline emphasizes the need for detailed quality documentation for ATMPs. This includes information on the manufacture of the active substance, including the description of the manufacturing process, control of materials and process validation. Particular attention is paid to the characterization of the API in order to fully understand its structure and properties. Specifications and analytical methods for quality control of the API are also required.

Non-clinical Development

For the non-clinical development of ATMPs, the guideline provides recommendations for conducting studies to assess the safety and efficacy of the investigational product prior to the start of clinical trials. This includes pharmacodynamic and pharmacokinetic studies as well as toxicity testing. The aim is to identify potential risks and determine the safety profile of the product.

Clinical Development

With regard to clinical development, the guideline provides information on planning and conducting clinical trials with ATMPs. This includes the definition of study objectives, the selection of suitable endpoints and the consideration of specific aspects of ATMPs, such as the long-term follow-up of patients. The guideline emphasizes the importance of a robust study design in order to obtain meaningful and reliable data on the safety and efficacy of the product.

Special Considerations

The guideline also considers specific aspects such as the use of genome editing technologies and the associated risks. It emphasizes the need for careful evaluation of such technologies in the context of ATMPs and calls for additional safety studies to identify and minimize potential adverse effects.

The EMA guideline provides a critical framework for the development and evaluation of ATMPs in clinical trials. By adhering to these guidelines, developers can ensure that their products meet the required quality standards and that the safety of trial participants is guaranteed. This ultimately helps to bring innovative therapies to market safely and effectively. The guideline has already been approved by CAT and CHMP and will enter into force in July 2025. Read the full guideline “Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials” on the EMA website.