Advanced Therapy Medicinal Product

Advanced Therapy Medicinal Products, or ATMPs, are advanced therapeutic drugs that are based on cell therapy or gene therapy (sometimes in combination with a medical device - where they are then called a tissue engineered product). The criteria to which a drug must conform to be classified as an ATMP, are defined in Article 17 of Regulation (EC) No 1394/2007 by the European Commission.^[1]

Development of ATMPs is typically highly experimental and often begins in academic research settings, such as universities and their hospitals. This, together with individualized manufacturing—often on a small scale for the treatment of serious, and frequently rare, diseases—has so far meant that the traditional (capital-rich) pharmaceutical industry has been almost entirely absent from this drug sector. Consequently, therapeutic concepts cannot reach market maturity due to lack of financial support for conducting clinical trials and limited regulatory experience (for example, complying with legal requirements to obtain marketing authorisation). A number of products from the heterogeneous group of ATMPs have now been approved, mainly for patients with rare diseases or for those for whom effective standard treatments are lacking. Because of the small number of cases, systematic collection of real-world clinical practice data in the post-approval phase is of particular importance for further study.

Authorization procedures for ATMPs differ between countries.^[2] Various protocols and regulatory frameworks are in place.

In the Member States of the European Union (EU) ATMPs must be authorised at the EU level (centralised) through the European Medicines Agency (EMA).^[3][4][5] For example, Czech Act No. 378/2007 Coll. on Pharmaceuticals recognises only medicinal products for cell and gene therapy. However, the EU directive is directly applicable.

In Saudi Arabia these matters are handled by the Saudi Food and Drug Authority (SFDA).^[6][7] Registration requires submission of specific clinical information, as well as information on chemical composition, manufacturing and quality control (CMC) to demonstrate compliance with the SFDA's requirements for drug approval.^[8]

In Brazil the regulatory framework includes the following provisions:

• IN 270/2023 establishes good manufacturing practices for products intended for advanced therapy;^[9]
• RDC 506/2021 - establishes the rules for conducting clinical trials with investigational advanced therapy products in Brazil^[10][11]
• RDC 505/2021 - provides for the registration of advanced therapy products.^[12]