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Research Pathways

Bringing therapies from the lab bench to patients is a complex process. We are developing our own as well as signposting to existing resources to help guide researchers and those interested in therapy development through this process. 

The Research Pathways is centred around the legal and regulatory aspects that are required for cell and gene therapy development in Europe, from binding law, non-binding guidelines and recommendations, then further expanded to additional aspects including scientific developments, health economics and more. 

Learn more about Research Pathways

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More ways to navigate Research Pathways

Key Pathways Resources

Signposting to key resources on ATMP development

Case Studies

Learn from specific development cases and relevant resources

Patient and Public Involvement

ATMP Engage Patient and Public Involvement Directory

Actors & Networks

Mapping the stakeholders in Europe and beyond
Showing 10 of 75
EuroGCT Resource

Case Study: Holoclar

  • EU and UK
  • 2023
  • EuroGCT
Case Study on Holoclar: Holoclar is one of the few ATMPs that have been granted marketing authorisation in Europe. We collated existing information about each stage of development for Holoclar, from early-stage research to approval, for the research community to learn about its development process.

Theme

  • Show all 18
  • Research and Innovation
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Funding
  • Incentives
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Commercialisation
  • Market access for ATMPs
EuroGCT Resource

Market Access for ATMPs

  • EU and UK
  • 2023
  • EuroGCT
Market Access for Advanced Therapy Medicinal Products (ATMPs): centralised authorisation procedure, expediting marketing authorisation pathways, and European Medicines Agency (EMA) regulatory support schemes

Theme

  • Show all 1
  • Market access for ATMPs

EATRIS Transmed Academy

  • EU
  • EATRIS
EATRIS Transmed Academy is the EATRIS online learning environment for translational scientists. Find here self-paced online courses, live courses, workshops, recorded webinars, guidance documents, resources and more.

Theme

  • Show all 6
  • Research and Innovation
  • Translational Science
  • Funding
  • Legal classification
  • Manufacturing
  • Commercialisation

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.

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
EuroGCT Resource

Human body elements for therapeutic applications

  • EU and UK
  • 2023
  • EuroGCT
Not Advanced therapy medicinal products (ATMPs): Human body elements, including tissues, cells, blood and blood components can be used for therapeutic purposes.

Theme

  • Show all 2
  • Therapy classification
  • Not ATMPs: Human body elements for therapeutic applications
EuroGCT Resource

Data sharing / Open data

  • EU and UK
  • 2023
  • Inserm, University of Toulouse Paul Sabatier and EuroGCT
Data sharing is the act of providing data or access to data from a data holder to a data user, for a defined processing purpose, subject to applicable technical, financial, legal, or organisational use requirements. Open data refers to making available research data or publications without restrictions regarding the reuses.

Theme

  • Show all 2
  • Data sharing / Open Data
  • Ethics
EuroGCT Resource

Good Manufacturing Practice

  • EU and UK
  • 2023
  • EuroGCT
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.

Theme

  • Show all 2
  • Manufacturing
  • Good Manufacturing Practice

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.

Theme

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

Cost and availability of novel cell and gene therapies

  • 2023
  • EMBO Reports
Advanced gene and cellular therapies risk a second “valley of death” due to their high costs and low patient population. As these are life-saving therapies, measures are urgently needed to prevent their withdrawal from the market.

Theme

  • Show all 2
  • ATMPs
  • Pricing & reimbursement

WHO Human Genome Editing (HGE) Registry

  • Global
  • WHO
The World Health Organization (WHO) Human Genome Editing (HGE) Registry is a central database that collects information of clinical trials using human genome editing technologies.

Theme

  • Show all 2
  • Clinical research
  • Data storage