What we do

Rare Diseases Expertise and Experience

Rare diseases, also known as orphan diseases, are medical conditions that affect a relatively small number of people within a population, often making them challenging to diagnose and treat due to their limited prevalence.

Rare Diseases CRO Expert

Optimapharm has significant expertise in Rare Diseases studies, having conducted over 90 studies, with 38% of them dedicated to paediatric populations.

Our extensive portfolio encompasses clinical studies in more than 45 Rare Diseases indications, including conditions such as Cystic fibrosis, Niemann-Pick type C, Hemophilia A, Crohn’s Disease, Idiopathic Pulmonary Fibrosis, AA Amyloidosis, Cushing’s syndrome, Epidermolysis Bullosa, and many others.

90+ Studies conducted

73% of Phase II and Phase III
Optimapharm Rare Disease studies by trial phase

Close collaboration with

170+ sites

 

385 patients enrolled

45 Rare Diseases indications

Optimapharm’s top 8 indications in Rare Diseases clinical studies
Optimapharm's top Rare Disease clinical studies indications

Staff experience

99 staff members with Rare Diseases projects experience
Optimapharm Rare Disease projects staff experience

Factsheet

Rare Disease clinical trials

Case Study

Case study_Pediatric Rare Disease Clinical Trials

Brochure

Optimapharm brochure