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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

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 78

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.

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).

Theme

  • 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).

Theme

  • Show all 7
  • Fundamental Research
  • Clinical research
  • Incentives
  • Early interaction with regulators
  • ATMPs
  • Good Manufacturing Practice
  • Market access for ATMPs
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.

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
EuroGCT Resource

Conditional Marketing Authorisation of ATMPs

  • EU and UK
  • 2023
  • EuroGCT
To facilitate early patient access to medicinal products, marketing authorisation (MA) can be granted through specific regulatory mechanisms offered by European Union law. When an Advanced Therapy Medicinal Product (ATMP) addresses an unmet medical need of patients, a conditional marketing authorisation may be granted on the basis of less comprehensive data than usually required.

Theme

  • Show all 2
  • Commercialisation
  • Market access for ATMPs
EuroGCT Resource

Marketing Authorisation Application file for ATMPs

  • EU and UK
  • 2022
  • EuroGCT
Marketing authorisation application requires from the applicant the submission of a file containing documentation and data related to the medicinal product to be authorised. The file must comply with the Common Technical Document (CTD), a set of specifications for the marketing authorisation application dossier. Under the centralised procedure the application is submitted under a standardised electronic format, the electronic Common Technical Document (eCTD), through an online portal.

Theme

  • Show all 2
  • Commercialisation
  • Market access for ATMPs
EuroGCT Resource

Standard Marketing Authorisation Pathway: Centralised Procedure for ATMPs

  • EU and UK
  • 2022
  • EuroGCT
The centralised procedure allows an ATMP to be commercialised and made available to patients and healthcare professionals throughout the entire European Union on the basis of a single marketing authorisation at the European level.

Theme

  • Show all 2
  • Commercialisation
  • Market access for ATMPs
EuroGCT Resource

Support for innovative medicines’ development at the EMA level

  • EU and UK
  • 2023
  • EuroGCT
EMA's support for innovative medicines’ development aims to clarify the applicable regulatory requirements for the medicinal products in development. Both procedures and services support the developers through establishing best strategies as early as possible for obtaining the required data (robust quality, pre-clinical, and clinical data) for commercialising innovative medicinal products (including ATMPs) in the European Union (EU) through the applicable and most adapted regulatory pathway.

Theme

  • Show all 3
  • Research and Innovation
  • Early interaction with regulators
  • Market access for ATMPs
EuroGCT Resource

Packaging and labelling of ATMPs

  • EU and UK
  • 2022
  • EuroGCT
Packaging and labelling (including outer packaging, the immediate packaging and the package leaflet) provide the information on an advanced therapy medicinal product (ATMP) to patients.  

Theme

  • Show all 1
  • Packaging and labelling