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

Conditional Marketing Authorisation 

  • 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.
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Theme

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  • Commercialisation
  • Market access for ATMPs

Expediting marketing authorisation pathways

  • EU and UK
  • 2023
  • EuroGCT
Within the European Union, three legal procedures have been developed to expedite access to new medicines, including ATMPs: conditional marketing authorisation, marketing authorisation under exceptional circumstances, and accelerated assessment.
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Theme

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  • Commercialisation
  • Market access for ATMPs

Specificities of the marketing authorisation dossier for biosimilars

  • EU and UK
  • 2023
  • EuroGCT
EU's biosimilar pathway increases patients’ access to safe and effective Advanced Therapy Medicinal Products (ATMPs) at a lower cost compared to the existing reference medicinal products which have lost their exclusivity rights. The applicant for a biosimilar marketing authorisation has to demonstrate in a simplified dossier high similarity of the biosimilar to the reference medicinal product in terms of quality and clinical aspects.
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Theme

  • Show all 2
  • Commercialisation
  • Market access for ATMPs

ATMPs and Orphan medicinal products

  • EU and UK
  • 2023
  • EuroGCT
Advanced therapy medicinal products (ATMPs) and orphan medicinal products. Obtaining the orphan designation gives access to financial and regulatory incentives aimed at improving the attractivity of research and development of innovative medicinal products for rare diseases. 
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Theme

  • Show all 2
  • Therapy classification
  • ATMPS

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
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Theme

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  • Research and Innovation
  • Clinical research

Good Manufacturing Practice

  • EU and UK
  • 2023
  • EuroGCT
Good Manufacturing Practice (GMP) is a code of quality standards concerning the manufacture, processing, packing, release and holding of a medicinal product placed within the European Economic Area (EEA). GMP describes the minimum standard that a medicines manufacturer must meet in their production and quality control processes, including those involving medicinal products intended for export only and medicines and active substances imported into the EU.
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Theme

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  • Manufacturing
  • Good Manufacturing Practice

Packaging and labelling

  • 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.  
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Theme

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  • Packaging and labelling

Manufacturing Authorisation

  • EU and UK
  • 2023
  • EuroGCT
Any company wishing to manufacture a medicinal product must hold a manufacturing authorisation issued by the national competent authority of the Member State where they carry out these activities. To obtain a manufacturing authorisation, all medicinal products for human use intended for the European Union market must be produced in accordance with EU quality standards: Good Manufacturing Practice (GMP) principles and guidelines, and the European Pharmacopeia.
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Theme

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  • Manufacturing Authorisation
  • Good Manufacturing Practice

Marketing Authorisation Application file

  • 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.
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Theme

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  • Commercialisation
  • Market access for ATMPs

Case Study: Strimvelis

  • EU and UK
  • 2023
  • EuroGCT
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 collection and storage
  • Data acquisition, processing, controlling
  • Data sharing, Open Data
  • Mission creep, data misuse
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