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Research Pathways

Bringing therapies from the lab bench to patients is a complex process. We've compiled resources and signposting to help guide through the development process - from legal regulations to best practices.

Find here information and resources for researchers, regulators, and those interested in therapy development.

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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 54

EMA Guidelines relevant for advanced therapy medicinal products

  • EU
  • EMA
Scientific guidelines developed by The European Medicines Agency (EMA) relevant for Advanced Therapy Medicinal Products (ATMPs).

Theme

  • Show all 14
  • Fundamental Research
  • Clinical research
  • Translational Science
  • ATMPS
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • European Pharmacopeia
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Market access for ATMPs
  • Pharmacovigilance

ELSIBI: Ethical, Legal and Social Implications of Biomedical Innovations

  • France
  • ELSIBI
The ELSIBI research blog aims to centralize research in law at the crossroads of other scientific disciplines in the field of ethical, legal and social implications of biomedical innovations and to disseminate them to the scientific community at large. Its content is developed at three levels, in French and/or in English.

Theme

  • Show all 4
  • Therapy classification
  • Commercialisation
  • Data
  • 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

MHRA Innovative Licensing and Access Pathway

  • UK
  • 2021
  • MHRA
The Innovative Licensing and Access Pathway (ILAP) by Medicines and Healthcare products Regulatory Agency (MHRA) provides enhanced regulatory and other expert input for supporting innovative approaches to the safe, and timely development of medicines.

Theme

  • Show all 3
  • Early interaction with regulators
  • Market access for ATMPs
  • Patient & Public Involvement

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

IGI CRISPR Genome Engineering Tools & Reagents

  • USA
  • IGI
CRISPR Genome Engineering Tools & Reagents published by the Innovative Genomics Institute (IGI). The resources include protocols, reagents, computational tools for setting up CRISPR experiments, and analyzing data.

Theme

  • Show all 1
  • Fundamental Research

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.

Theme

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

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 10
  • Fundamental Research
  • Clinical research
  • Translational Science
  • ATMPS
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • European Pharmacopeia
  • Investigational medicinal products dossier
  • Capability
  • Scalability

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

UKRI Health Funding Map

  • UK
  • UKRI and BIA
Health Funding Map developed by UK Research and Innovation (UKRI) Innovate UK KTN, the UK BioIndustry Association (BIA) and more funders. This interactive map helps the innovators to navigate the complicated world of health innovation funding across UK and EU.

Theme

  • Show all 1
  • Funding