With the aim of harmonizing the regulatory framework in Ukraine regarding the content and scope of the Investigational Medicinal Product Dossier for advanced therapy medicinal products (hereinafter – ATMP) and on the basis of international guidelines:
- Committee for Advanced Therapies (CAT) EMA/CAT/852602/2018 "Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials";
- Swedish Medical Products Agency (MPA) 2019-05-09 Guide: Investigational medicinal product dossier for ATMP;
- Committee for Advanced Therapies (CAT) EMA/CAT/CPWP/686637/2011 Guideline on the risk-based approach according to annex I, part IV of Directive 2001/83/EC applied to Advanced therapy medicinal products (Guideline on the risk-based approach according to annex I, part IV of Directive 2001/83/EC for ATMPs);
- Committee for Advanced Therapies (CAT) EMA/CAT/499821/2019 "Questions and answers. Comparability considerations for Advanced Therapy Medicinal Products (ATMP)";
- of the Committee for Medicinal Products for Human Use (CHMP) EMA/CHMP/BWP/271475/2006 rev.1 "Guideline on potency testing of cell based immunotherapy medicinal products for the treatment of cancer",
specialists of the State Expert Center of the Ministry of Health of Ukraine have developed the guideline ST-N MoHU 42 – 9.1:2024 "Medicinal products. Requirements for the dossier of investigational advanced therapy medicinal products within clinical trials", approved by order of the Ministry of Health of Ukraine dated 12.11.2024 No. 1895.
ATMPs are a new category of medicines with broad therapeutic potential for the treatment of various types of diseases, such as cancer, neurodegenerative and autoimmune diseases. The manufacturing processes of ATMPs are notable for the complexity of the production process. Some ATMP technologies are borrowed from the principles of manufacturing monoclonal antibodies presented in established regulatory documentation; others, more recent ATMPs, are manufactured using related processes that encompass the manufacturing processes of genetically modified products and transplants.
The purpose of this document is to provide guidance on the structure and requirements for the Dossier submitted with an application for a clinical trial for the approval of exploratory and confirmatory clinical trials in Ukraine with ATMPs.
The guideline is multidisciplinary and addresses:
- specifics of the development, manufacture and quality control of ATMP products,
- specifics of preclinical and clinical development of ATMPs;
- considerations regarding genome editing tools;
- determination of the efficacy of ATMPs;
- development of a comparability program for ATMP products;
- the benefit-risk assessment during the development and use of these products in humans;
- requirements for research trials (including First in Human studies) and confirmatory trials.
This guideline applies together with the provisions of the Procedure for Conducting Clinical Trials of Medicinal Products and Expert Evaluation of Clinical Trial Materials and the Model Regulation on Ethics Commissions, approved by order of the Ministry of Health of Ukraine dated 23 September 2009 No. 690, as amended.
You can familiarize yourself with this guideline at the link.
