Rare Diseases

Experience & Services

Making a difference, where the patient is key.

Ergomed and PSR joined forces together to create a leading orphan drug development expert team that will allow our clients to implement patient driven solutions in their clinical trials. Patients will define our future landscape, including the rise of both precision and preventative medicine.

What sets us apart:

  • Unique insight into orphan drug programs to accurately address the specific challenges and project needs that rare disease trials require in order to provide an innovative and solid infrastructure
  • Extensive experience in innovative regulatory & clinical development (Pharma, biotech, CRO, Academia)
  • Global access to hard-to-find patients in under-served regions
  • A regulatory consultancy service to provide complete support for orphan drug related interactions with the EMA and FDA (e.g. Orphan Drug designations, PIP, Protocol Assistance/Scientific Advise)
  • A unique site management model that allows for faster recruitment of quality patients and site coordination
  • Complete staffing solutions specialising in placing team members with orphan drug experience

Rare disease clinical trials are not just smaller versions of conventional clinical trials and therefore require an innovative approach. By combining Ergomed’s global reach and site management model with PSR’s specialized focus on orphan drug development, we will continue to improve the lives of patients and their families impacted by rare diseases across the world.

Ergomed RARE DISEASES has experience of conducting multiple trials in designated rare indications. Through its Site Management model and Study Physician Team Support, Ergomed has shown that it can locate and support sites in studies of ultra-rare diseases like infantile hypophsophatemia, paroxyzmal nocturnal haemoglobinuria and primary hyperoxaluria, diseases for which recruitment can be highly problematic. Ergomed has successfully prepared Orphan Designation applications and have established links with physician networks, key opinion leaders, government bodies and patient groups. Ergomed provides regulatory and scientific expertise which helps to ensure the best designs for rare diseases clinical programs and registries. We offer our specialised Site Management Model that allows smooth interaction of the sites and patient organisations and advocacy groups. Combined, Ergomed and PSR have managed over 65 rare disease projects and trials.  Our experience list follows:

  • Abdominal Aortic Anerusm
  • Acanthamoeba Keratitis
  • Acute Myeloid Leukemia
  • Alkaptonuria
  • Alport Syndrome
  • ALS
  • AML
  • Burns
  • Chemotherapy-Induced Neutropenia (CIN)
  • CML
  • Cystic Fibrosis
  • Cystinosis
  • Duchenne Muscular Dystrophy
  • Eosinophilic Esophagitis
  • Gaucher’s Disease
  • GH Deficiency
  • GvH Disease
  • Haemophilia
  • Haemophilia A
  • Head & Neck Carcinoma
  • Hereditary Antithrombin III Deficiency
  • Hereditary Tyrosinemia Type 1
  • Heriditary Angioedema
  • Idiopathic Thrombocytopenic Purpura (ITP)
  • Mitochondrial OXPHOS Disease
  • Mucopolysaccharidosis
  • Neuroblastoma
  • Non-Hodgkin’s Lymphoma
  • Pancreatic Cancer
  • Paroxyzmal Noctural Hemoglobinuria (PNH)
  • Primary Hypeoxraluria
  • Pulmonary Hypertension
  • Relapsing Remitting Multiple Sclerosis
  • Renal Cell Carcinoma
  • Scleroderma
  • Sickle Cell Disease
  • Spinal Muscular Atrophy
  • Thalassemia
  • Wilson Disease
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