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What is gene and cell therapy?
What are ATMPs, and where do they fit into gene and cell therapy?
Why are animal models used when testing the safety of a new therapy?
What role do preclinical and clinical trials play in ensuring patient safety?
Why are some conditions more suitable for gene and cell therapies than others?
There is an approved treatment available for my condition - why can't I receive it?
How to look for approved medicines?
How are medicines developed and approved?

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Accelerated assessment
Adeno-associated virus (AAV)
Adenovirus
Adult stem cells
Advanced Therapy Investigational Medicinal Product (ATIMP)
Advanced Therapy Medicinal Product (ATMP)
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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 21

ATMP guidebook

  • Netherlands
  • 2025
  • FAST
The ATMP guidebook published by the Centre for Future Affordable & Sustainable Therapy Development (FAST) in Netherlands delivers essential guidance on European and Dutch regulations and provides a roadmap to navigate the complex ATMP development process.
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Theme

  • Show all 17
  • Research and Innovation
  • Fundamental Research
  • Clinical research
  • Incentives
  • Early interaction with regulators
  • Therapy classification
  • Legal classification
  • ATMPs
  • Good Manufacturing Practice
  • Commercialisation
  • Market access for ATMPs
  • Distribution
  • Pharmacovigilance
  • Pricing & reimbursement
  • Data
  • Data protection
  • Patient & Public Involvement

Academic development of ATMPS in Belgium

  • Belgium
  • 2024
  • Belgian Health Care Knowledge Centre (KCE)
A Report published by the Belgian Health Care Knowledge Centre (KCE) on academically developed ATMPs in Belgium.
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Theme

  • Show all 13
  • Research and Innovation
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Funding
  • Incentives
  • ATMPs
  • Manufacturing
  • Good Manufacturing Practice
  • Scalability
  • Commercialisation
  • Market access for ATMPs
  • Pricing & reimbursement
EuroGCT Resource

Commercialisation

  • EU and UK
  • 2022
  • EuroGCT
Enabling timely patient access to Advanced Therapy Medicinal Products (ATMPs), commercialisation covers marketing authorisation, and post-authorisation activities such as pharmacovigilance, pricing and reimbursement.
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Theme

  • Show all 6
  • Commercialisation
  • Market access for ATMPs
  • Distribution
  • Advertising
  • Pharmacovigilance
  • Pricing & reimbursement
EuroGCT Resource

Case Study: Strimvelis

  • EU and UK
  • 2023
  • EuroGCT
Case Study on Strimvelis: Strimvelis is the first ex vivo gene therapy to be licensed, and its success is the result of a joint effort among different stakeholders. Here we collated existing information about each stage of its development as a guide for the research community to learn about its development process.
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Theme

  • Show all 13
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Funding
  • Market access for ATMPs
  • Pharmacovigilance
  • Pricing & reimbursement
  • Data
  • Data protection
  • Data storage
  • Data collection, processing, controlling
  • Data sharing / Open Data
  • Mission creep / data misuse

Creating EU market access roadmaps for cell and gene therapies

  • UK
  • 2016
  • CGT Catapult
Market access roadmaps for cell and gene therapies published by Cell and Gene Therapy Catapult (CGT Catapult). Contains individual reimbursement roadmaps for UK (England and Wales), and EU countries (Spain, Italy, France, and Germany).
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Theme

  • Show all 9
  • Funding
  • Early interaction with regulators
  • Therapy classification
  • Legal classification
  • ATMPs
  • Not ATMPs: Human body elements for therapeutic applications
  • Not ATMPs: medical devices containing tissues and cells
  • Market access for ATMPs
  • Pricing & reimbursement

New guidance on the development and marketing of ATMPs in the UK and EU in the post-BREXIT landscape

  • UK
  • 2022
  • CGT Catapult
A guidance by the Cell and Gene Therapy Catapult (CGT Catapult) on the development and marketing of ATMPs in the UK and EU in the post-BREXIT landscape.
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Theme

  • Show all 15
  • Clinical research
  • Translational Science
  • Early interaction with regulators
  • Therapy classification
  • Legal classification
  • ATMPs
  • Not ATMPs: Human body elements for therapeutic applications
  • Not ATMPs: medical devices containing tissues and cells
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Packaging and labelling
  • Market access for ATMPs
  • Distribution
  • Pharmacovigilance
  • Pricing & reimbursement

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

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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Theme

  • 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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Theme

  • 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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Theme

  • Show all 16
  • 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
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