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Glossary

Adeno-associated virus (AAV)
Adenovirus
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Antibody
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Research Pathways

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.

How to use Research Pathways  EuroGCT's Key Resources

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Showing 10 of 37

EFPIA The root cause of unavailability and delay to innovative medicines

  • EU
  • 2023
  • EFPIA
Analysis by the European Federation of Pharmaceutical Industries and Associations (EFPIA). EFPIA's Root Causes Analysis identified 10 interrelated factors that were causing access to medicines to be delayed.
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Theme

  • Show all 2
  • Market access for ATMPs
  • Pricing & reimbursement

EMA Guidelines relevant for advanced therapy medicinal products

  • EU
  • European Medicines Agency (EMA)
Scientific guidelines developed by The European Medicines Agency (EMA) relevant for Advanced Therapy Medicinal Products (ATMPs).
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  • Show all 13
  • Fundamental Research
  • Clinical research
  • Translational Science
  • ATMPs
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Market access for ATMPs
  • Pharmacovigilance

EMA Support for advanced therapy developers

  • EU
  • European Medicines Agency (EMA)
Advisory services and incentives the European Medicines Agency (EMA) offers for supporting the development of advanced therapy medicinal products (ATMPs).
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  • Show all 7
  • Fundamental Research
  • Clinical research
  • Incentives
  • Early interaction with regulators
  • ATMPs
  • Good Manufacturing Practice
  • Market access for ATMPs

The EBMT/EHA CAR-T Cell Handbook

  • EU
  • 2022
  • EBMT, EHA and Springer
This open access CAR-T Cell Handbook published by Springer and co-promoted by the European Society for Blood and Marrow Transplantation (EBMT) and the European Hematology Association (EHA), provides an unparalleled overview of the CAR-T cell technology and covers several aspects of CAR-T cell treatments, including the underlying biology, indications, management of side-effects, access and manufacturing issues.
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  • Show all 14
  • Fundamental Research
  • Clinical research
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Market access for ATMPs
  • Distribution
  • Pharmacovigilance
  • Pricing & reimbursement
  • Ethics

EFPIA Challenges and facilitators in the development of orphan and paediatric medicines

  • EU
  • EFPIA
A report developed collaboratively by The European Federation of Pharmaceutical Industries and Associations (EFPIA) and the Office of Health Economics (OHE). It presents a conceptual model for understanding how to best ensure sustained innovation in areas of need, and provides illustrative case studies on five different therapies to highlight specific challenges and facilitators around developing medicines for rare and paediatric conditions.
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  • Show all 6
  • Research and Innovation
  • Incentives
  • ATMPs
  • Capability
  • Market access for ATMPs
  • Pricing & reimbursement

IRDiRC Orphan Drug Development Guide

  • Global
  • IRDiRC
The Orphan Drug Development Guidebook (ODDG) by the International Rare Diseases Research Consortium (IRDiRC) is a patient focused guidebook that describes the available tools, incentives, resources and practices specific for developing traditional and innovative drugs/therapies for rare disease indications and how to best use them.
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  • Research and Innovation
  • Fundamental Research
  • Clinical research
  • Funding
  • Incentives
  • Early interaction with regulators
  • ATMPs
  • Market access for ATMPs
  • Pricing & reimbursement
  • Data
  • Data protection
  • Data storage
  • Data collection, processing, controlling
  • Data sharing / Open Data
  • Patient & Public Involvement
  • Ethics

Responsible personalised medicine: Exploring the ethical, legal, social, political and economic issues of manufacturing, distribution, access and reimbursement.

  • UK
  • 2022
  • UCL Future Targeted Healthcare Manufacturing Hub
A report published by University College London (UCL) Future Targeted Healthcare Manufacturing Hub provides an overview of the ethical, legal, social, political and economic issues underpinning the “manufacturing, business and regulatory challenges” that confront the development and delivery of affordable and accessible personalised medicines.
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Theme

  • Show all 6
  • Manufacturing
  • Market access for ATMPs
  • Distribution
  • Pricing & reimbursement
  • Data protection
  • Ethics

ABPI ATMP Roadmap

  • UK
  • 2021
  • ABPI
The Association of the British Pharmaceutical Industry (ABPI) has developed an ATMP roadmap which sets out the key steps in the end-to-end pathway for ATMPs in UK, from non-clinical research through patient treatment and monitoring.
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Theme

  • Show all 15
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Commercialisation
  • Market access for ATMPs
  • Distribution
  • Pharmacovigilance
  • Pricing & reimbursement
  • Data storage

EMA pilot programme

  • EU
  • 2022
  • EMA
European Medicines Agency (EMA) pilot program on enhanced regulatory support for academic and non-profit ATMP developers
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Theme

  • Show all 5
  • Incentives
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Market access for ATMPs

ARM Project A-Gene

  • Global
  • 2021
  • ARM
The Alliance for Regenerative Medicine (ARM) and the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) produced A-Gene, a case study-based guide to integrating Quality by design (QbD) principles in gene therapy Chemistry Manufacturing & Control (CMC) programs.
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Theme

  • Show all 12
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Early interaction with regulators
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Market access for ATMPs
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