Bringing gene and cell therapies from the lab bench to patients is a complex process and multiple pathways exists. The Research Pathways directory contains resources to help various stakeholders interested in therapy development navigate this process. Research Pathways is centred around the legal and regulatory aspects for cell and gene therapy development in Europe and further expanded to additional aspects. It is free to use and all resources in this database are categorised for improved searchability.
La fabbricazione di medicinali per terapie avanzate (MTA) riguarda la fabbricazione su scala industriale di queste terapie per supportare lo sviluppo clinico e la commercializzazione. Questa sezione fornisce una panoramica dei processi di produzione dei MTA, dei termini chiave e delle risorse correlate.
Enabling timely patient access to Advanced Therapy Medicinal Products (ATMPs), commercialisation covers marketing authorisation, and post-authorisation activities such as pharmacovigilance, pricing and reimbursement.
After a marketing authorisation is granted for a medicine, whether patients can access it is highly dependent on its pricing and on its reimbursement by national health insurance. Pricing and reimbursement schemes are determined at the national level, not at the EU level
Good Manufacturing Practice (GMP) is a code of quality standards concerning the manufacture, processing, packing, release and holding of a medicinal product placed within the European Economic Area (EEA). GMP describes the minimum standard that a medicines manufacturer must meet in their production and quality control processes, including those involving medicinal products intended for export only and medicines and active substances imported into the EU.
Advanced therapy medicinal products (ATMPs) and orphan medicinal products. Obtaining the orphan designation gives access to financial and regulatory incentives aimed at improving the attractivity of research and development of innovative medicinal products for rare diseases.
La classificazione delle terapie determina i meccanismi giuridici che regolano la disciplina delle terapie geniche e cellulari, al fine di garantire la qualità, la sicurezza e l'efficacia di tali terapie sul mercato dell'Unione Europea.
Lo sviluppo di terapie innovative inizia con l'applicazione delle scoperte fatte nella ricerca di base. Questa sezione descrive le varie fasi, dalla ricerca di base alla ricerca preclinica e clinica, all'origine dello sviluppo di terapie geniche e cellulari innovative.