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Research Pathways

Bringing gene and cell therapies from the lab bench to patients is a complex process and multiple pathways exists. The Research Pathways directory contains resources to help various stakeholders interested in therapy development navigate this process. Research Pathways is centred around the legal and regulatory aspects for cell and gene therapy development in Europe and further expanded to additional aspects. It is free to use and all resources in this database are categorised for improved searchability.

How to use Research Pathways  EuroGCT's Key Resources

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Showing 10 of 91

The EBMT/EHA CAR-T Cell Handbook

  • EU
  • 2022
  • EBMT, EHA and Springer
This open access CAR-T Cell Handbook published by Springer and co-promoted by the European Society for Blood and Marrow Transplantation (EBMT) and the European Hematology Association (EHA), provides an unparalleled overview of the CAR-T cell technology and covers several aspects of CAR-T cell treatments, including the underlying biology, indications, management of side-effects, access and manufacturing issues.

Theme

  • Show all 14
  • Fundamental Research
  • Clinical research
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Market access for ATMPs
  • Distribution
  • Pharmacovigilance
  • Pricing & reimbursement
  • Ethics

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

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

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
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