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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 84

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

ECRIN CAMPUS for Regulatory and Ethical Requirements

  • EU
  • ECRIN
The Regulatory and Ethical Tool (CAMPUS) by the European Clinical Research Infrastructure Network (ECRIN) is a central resource for information about clinical trial regulatory and ethical requirements covering 22 European countries and multiple study types.

Theme

  • Show all 2
  • Clinical research
  • Ethics

ATMP Sweden Regulatory Guide

  • Sweden
  • ATMP Sweden
ATMP Sweden's regulatory guide for the development of the Advanced Therapy Medicinal Products (ATMPs)

Theme

  • Show all 12
  • Fundamental Research
  • Clinical research
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling

ABPI ATMP Roadmap

  • UK
  • 2021
  • ABPI
The Association of the British Pharmaceutical Industry (ABPI) has developed an ATMP roadmap which sets out the key steps in the end-to-end pathway for ATMPs in UK, from non-clinical research through patient treatment and monitoring.

Theme

  • Show all 15
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Commercialisation
  • Market access for ATMPs
  • Distribution
  • Pharmacovigilance
  • Pricing & reimbursement
  • Data storage

NCATS PaVe-GT: Paving the Way for Rare Disease Gene Therapies

  • USA
  • NCATS
The Platform Vector Gene Therapy (PaVe-GT) pilot project lead by National Center for Advancing Translational Sciences (NCATS) at National Institutes of Health (NIH) seeks to increase the efficiency of clinical trial startup by using the same gene delivery system and manufacturing methods for multiple rare disease gene therapies.

Theme

  • Show all 9
  • Fundamental Research
  • Clinical research
  • Translational Science
  • ATMPs
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability

ABPI Bridging between pre- and clinical boundary

  • United Kingdom
  • 2018
  • The Association of the British Pharmaceutical Industry (ABPI)
A report for a joint workshop held by the Academy of Medical Sciences and the Association of the British Pharmaceutical Industry (ABPI). The meeting looked to identify challenges, gaps, and opportunities for greater integration of pre-clinical and clinical science, and the vision for the future of translational research in the UK.

Theme

  • Show all 4
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Funding
EuroGCT Resource

Clinical trials

  • EU and UK
  • 2023
  • EuroGCT
Clinical trials are a type of clinical studies performed to investigate the safety and/or the efficacy of a medicinal product

Theme

  • Show all 2
  • Research and Innovation
  • Clinical research

Improving Patient Involvement in Medicines Research and Development: A Practical Roadmap

  • 2017
  • Therapeutic Innovation & Regulatory Science
A practical roadmap for Patient Involvement (PI) opportunities along the 4 key stages of the medicines R&D life cycle.

Theme

  • Show all 1
  • Patient & Public Involvement