Rare Disease & Orphan Indications

Rare Disease & Orphan Indications

Rare Disease and Orphan Indication Drug Experience

Medpace has conducted more than 40 global trials covering Phase I-V, involving 4,500 patients including these rare disease indications.

  • Acromegaly
  • Adrenocortical carcinoma (ACC)
  • Cushing’s Syndrome
  • Cystic Fibrosis
  • Eosinophilic Esophagitis
  • Familial Hypercholesterolemia
  • Fibrodysplasia
  • Gastroparesis
  • Glomerulonephritis
  • Growth Hormone Deficiency
  • Niemann Pick Type C disease
  • Nonsense Mutation Dystrophinopathy
  • Ossificans progressive (FOP or Stoneman’s Disease)
  • Renal vasculitis
  • Respiratory Syncytial Virus (RSV) in lung transplant patients
  • Tinnitus
  • TTR Amyloidosis

Focused Rare Disease Teams Deliver Results

  • Committed cross-functional team of Rare Disease experts comprised of doctors, project managers, regulatory consultants, and late phase specialists to orchestrate these complex studies
  • Board-certified Pediatricians with backgrounds treating pediatric patients with Rare Disease / Orphan Drug conditions
  • Global expertise in varied regulatory issues – critical for Rare Disease projects
  • ClinTrak, Medpace proprietary Study Management system, providing decision support for all aspects of a project including managing Late Phase Patient registries