Skip to main content

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

Additional navigation options

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 84
EuroGCT Resource

Support specific to ATMPs’ development at EMA level

  • EU and UK
  • 2023
  • EuroGCT
Procedural support exclusively for Advanced Therapy Medicinal Products (ATMPs) at the EMA level offers incentives or an easier access to the procedures for developers of ATMPs, especially sponsors.

Theme

  • Show all 2
  • Research and Innovation
  • Early interaction with regulators

Orphanet: The portal for rare diseases and orphan drugs

  • Global
  • Orphanet
The Orphanet knowledgebase gathers and provides high-quality information on rare diseases for improving the diagnosis, care and treatment of patients with rare diseases.

Theme

  • Show all 7
  • Research and Innovation
  • Fundamental Research
  • Clinical research
  • ATMPs
  • Data storage
  • Data sharing / Open Data
  • Patient & Public Involvement

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.

Theme

  • Show all 6
  • Research and Innovation
  • Incentives
  • ATMPs
  • Capability
  • Market access for ATMPs
  • Pricing & reimbursement

EFPIA Intellectual Property

  • EU
  • EFPIA
Information compiled by European Federation of Pharmaceutical Industries and Associations (EFPIA) on Europe’s framework of incentives and rewards for discovering and developing new treatments. Topics covered include: Pharmaceutical Intellectual Property (IP), Supplementary Protection Certificates (SPCs), Regulatory Data Protection (RDP), Orphan Incentive, and Paediatric Incentives.

Theme

  • Show all 2
  • Incentives
  • 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