Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Development of Tissue Engineered Products in the EU - Will the European Commission’s and EMA's Action Plan on Advanced Therapy Medicinal Products make the difference? ***

Dr. Sabine Küppers (Abschlußjahr: 2024)

Summary
Language: English
Advanced Therapy Medicinal Products (ATMPs), which are divided into the three subtypes Tissue Engineered Products (TEPs), Gene Therapy Medicinal Products (GTMPs) and somatic Cell Therapy Medicinal Products (sCTMPs), are developed since approximately forty years and offer a great clinical perspectives for unmet medical needs and rare diseases. But nevertheless, currently only 18 ATMPs have got a marketing authorisation valid in the European Union (EU), including two TEPs. In order to identify the key issues in ATMP development, the European Medicines Agency (EMA) hosted a workshop for stakeholders with diverse backgrounds in May 2016. Based on the issues identified, the European Commission and the EMA published an Action Plan on ATMPs in October 2017 in order to initiate certain follow-up actions to improve and to facilitate the development and the marketing authorisation of innovative ATMP in the EU. The proposed actions target challenges at all stages of development and include several services and activities as well as guidance documents specific on ATMPs.
This thesis provides a comparison of the European legislative landscape concerning ATMPs, especially TEPs, before and six years after the European Commission’s and EMA’s Action Plan on ATMPs has been published. In addition, it is assessed which points of the action plan have already been implemented. Seven new or revised guidance documents are further reviewed to determine whether the previously identified stakeholders’ issues have been sufficiently addressed. Overall, it can be stated that the assessed guidelines reflect many suggestions and introduces highly needed adaptations and exceptions. Of these guidance documents, the “Eudralex Volume 4 - Guidelines on Good Manufacturing Practice specific to Advanced Therapy Medicinal Products” are expected to have the greatest positive impact on the development of ATMPs in the EU, because it is the most comprehensive one and addresses the most stakeholders’ issues, e.g. more detailed guidance on the risk-based approach for the manufacturing process. But in summary, it can be said that some of the biggest hurdles in the development of ATMPs, e.g. the missing harmonisation of requirements for genetically modified organisms, cells and tissues used as starting materials, raw materials and excipients across the Member States, are still unaddressed. Therefore, it is questionable whether the measures implemented so far will have a major impact on the number of further marketing authorisations for ATMPs in the EU.
Pages: 74
Annexes: 0, Pages: 0