Research Pathways

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The development of innovative therapies starts with translating discoveries from basic research. This section explores the steps from fundamental to pre-clinical and clinical research underpinning the development of innovative gene and cell therapies.

Therapy classification determines the legal mechanisms that govern the regulation of gene and cell therapies to ensure the quality, safety, and efficacy of these therapies on the EU market.

Advanced therapy medicinal products (ATMP) manufacturing concerns the industry-scale production of these therapies for supporting clinical development and commercialisation. This section provides an overview of the ATMP manufacturing processes, key terms, and related resources.

Enabling timely patient access to Advanced Therapy Medicinal Products (ATMPs), commercialisation covers marketing authorisation, and post-authorisation activities such as pharmacovigilance, pricing and reimbursement.

Health data discussed in this theme includes all information that is created, collected, received, maintained, processed, distributed and analysed along the gene and cell therapy development pathway. It starts from the data derived from biological samples used in research, and ends with the data collected for post-marketing safety and efficacy studies.

Patient and Public Involvement (PPI) is defined as “research being carried out ‘with’ or ‘by’ members of the public rather than ‘to’,‘about’ or ‘for’ them.” Partnering with the researchers, patients and public are involved throughout all stages of clinical research, including identifying, prioritising, designing, managing, undertaking, evaluating and communicating research. 

Ethical issues along the lab to patient pathway of gene and cell therapy development.