Research Pathways

Bringing gene and cell therapies from the lab bench to patients is a complex process.The Research Pathways directory contains resources to help guide various stakeholders 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, guidelines and recommendations, then further expanded to additional aspects including ethics, scientific developments, commercialisation, health economics and more. 

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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

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Showing 10 of 35

Manufacturing process development of ATMPs within a regulatory framework for EU clinical trial & marketing authorisation applications

  • 2016
  • Cell And Gene Therapy Insights
In the European Union, the regulatory approval of ATMPs for use in patients requires that data demonstrating their quality, safety and efficacy are submitted in dossiers to regulatory agencies for review. Indeed, such dossiers have a specific format that, in the case of quality data in particular, is informative for the manufacturing process development strategy. This manuscript describes how dossier requirements can be implemented into the design of industrialized ATMP manufacturing processes and fulfilled to enable effective regulatory submissions.
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Theme

  • Show all 10
  • Clinical research
  • ATMPs
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • 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.
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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
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.
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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

Taking ATMPs to Market

  • UK
  • 2018
  • CGT Catapult
A presentation by Cell and Gene Catapult (CGT Catapult) on topics including: ATMP regulatory framework in EU, therapy classification, early development support by EMA (EU) and MHRA (UK), and market access pathways.
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Theme

  • Show all 4
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Market access for ATMPs
EuroGCT Resource

Marketing Authorisation under exceptional circumstances for 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. A marketing authorisation under exceptional circumstances for medicinal products may be granted when the applicant is unable to provide comprehensive data on the efficacy and safety under normal conditions of use, because the indication to be treated is too rare, the present state of scientific knowledge is insufficient to provide comprehensive information, or it would be unethical to collect this information.
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Theme

  • Show all 2
  • Commercialisation
  • Market access for ATMPs

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
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

Regulatory Framework for Advanced Therapy Medicinal Products in Europe and United States

  • 2019
  • Frontiers in Pharmacology
This article reviews the legal frameworks in the EU and US for Advanced Therapy Medicinal Products (ATMPs), as well as the criteria to be met to define a product as such. The similarities and differences that exist between both regions are discussed in order to identify those nuances that may affect the development of an ATMP.
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Theme

  • Show all 3
  • Legal classification
  • ATMPs
  • Market access for ATMPs

EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation

  • 2019
  • Molecular Therapy - Methods & Clinical Development
Advanced therapy medicinal products (ATMPs) require evaluation by the European Medicines Agency’s Committee for Advanced Therapies prior to being placed on the European market, subject to a Marketing Authorisation granted by the European Commission. This review discusses how the regulatory pathway for any particular ATMP, with or without PRIME designation, is determined and navigated.
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Theme

  • Show all 7
  • Clinical research
  • Translational Science
  • Legal classification
  • ATMPs
  • Good Manufacturing Practice
  • Market access for ATMPs
  • Pharmacovigilance
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