European Union

Benefits: European Reference Networks (ERNs) connect specialist centers across the EU for rare or complex diseases. These networks help identify trial participants, share data, and align best practices. Clinical trials in the EU are accessible to patients across 26 EU countries.

Select Leading Clinical Centers:

Charité – Universitätsmedizin Berlin (Berlin, Germany)

1. • Specializations: Rare neurological disorders, gene therapy, rare cancers

   • Trial Focus: 500+ trials/year | Early-phase industry trials

   • Key Advantage: Strong EMA ties and industry partnerships

Necker-Enfants Malades Hospital (Paris, France)

1. • Specializations: Pediatric genetic diseases, metabolic disorders, CAR-T therapy

   • Trial Focus: Pioneering gene therapies | EMA-fast-track site

   • Key Advantage: Home to Imagine Institute (rare disease research)

University Medical Center Utrecht (Netherlands)

1. • Specializations: Neuromuscular diseases, mitochondrial disorders

   • Trial Focus: Innovative trial designs | High recruitment speed

   • Key Advantage: Streamlined ethics approvals

• How to access a rare disease clinical trial if you’re a non-EU national? 

  • Non-EU nationals living in the EU

    • Foreigners legally residing in the EU (e.g., on work visas, student visas, asylum, or long-term permits) are fully eligible.

  • Non-EU residents (Living Outside the EU)

    • May participate in specific cases, especially in rare disease trials where:

      • Patient populations are scarce

      • Cross-border recruitment is necessary

      • The trial site approves enrollment 

      • Local laws and insurance coverage allow it

• How to apply? 

  • Visas: If you are a non-EU national and not living in an EU country, you may need to apply for a Schengen visa, an entry permit for non-EU nationals to make a short, temporary visit of up to 90 days within any 180-day period to a country in the Schengen area. 

    • The visa application must demonstrate the medical purpose and logistics of the trial. Required documents include:

      • Official invitation letter from the clinical trial sponsor or site.

      • Proof of trial enrollment (e.g. patient information sheet or informed consent form)

      • Details of medical insurance, accommodation, and travel. Sometimes, proof of expenses covered by the sponsor or a third party may be required. 

  • The clinical trial site can also help you learn more about the visa requirements.

  • Check for the Schengen visa application details here. 

Quick Links on rare disease clinical trials here:

• • Orphanet

  • EU clinical trials

  • Interactive clinical trials map

  • ClinicalTrials.gov