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Rare diseases archive

Explore rare diseases studied in clinical trials

619 rare diseases in the catalogue3 138 linked trials

Top 10 most studied rare diseases

Rare diseases with the highest number of active trials right now.

Browse all rare diseases ↓

Colours show each disease's body system – lighter shades rank lower.

  • Blood and lymphatic system disorders58%
  • Gastrointestinal disorders20%
  • Neoplasms benign, malignant and unspecified11%
  • Hepatobiliary disorders11%

No. 1 most studied

Plasma cell myeloma

Blood and lymphatic system disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
164Active trials · 20% of the top 10
recruiting now
50
countries
25
open to children
0
substances tested
141
View disease
20%Plasma cell myeloma

Shares are of these ten diseases' active trials together.

All rare diseases

Browse every rare disease currently studied across European trials.

The diseases archive

619 rare diseases

About rare diseases in clinical trials

A disease is called rare in the EU when it affects fewer than 5 in 10 000 people. Individually uncommon, together they affect millions – and most still have no approved treatment.

Because the number of patients is small, trials in rare diseases often recruit across several countries at once. That is why a trial listed here may be worth looking at even when it runs nowhere near you.

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Clinical Trials Concierge

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