Rare Diseases

IDDI, Your Partner to Accelerate Your Clinical Trials in Rare Diseases

Clinical studies in rare diseases need a CRO with specific strategies regarding site selection, patient enrollment, and retention.  They require special expertise in designing protocols, executing clinical trial design for a sparse and often widely distributed population as well as the understanding of the regulatory constraints of orphan status. When conducting clinical trials in orphan diseases, sponsors face medical, scientific and operational challenges.

Experience in Rare Diseases

Having conducted 54 studies in Orphan Diseases, we understand those challenges and have the expertise to help you successfully develop orphan drugs.

Our experience includes 50+ rare diseases clinical studies in a broad range of indications: 

  • Acute myeloid leukemia (AML)
  • ADA-Deficient SCID
  • Biliary Tract Cancer
  • Crigler-Najjar syndrome
  • Cutaneous T-Cell Lymphoma
  • Lamellar Ichthyosis
  • Leber hereditary optic neuropathy
  • Leukemia/Myelodysplastic Syndrome (MDS)
  • Liver/Biliary/Pancreas cancer
  • Lymphoma, Non Hodgkin Lymphoma (NHL)
  • Mesothelioma
  • Midgut Carcinoid Tumors
  • Niemann Pick Type C disease
  • Positive Chronic Myelogenous Leukemia
  • Prev. of acute graft vs host disease (aGvHD)
  • Retinitis pigmentosa (RP)
  • Scleroderma
  • Spinal Muscular Atrophy
  • Urea Cycle Disorders
  • Wilson’s Disease
  • Wiskott-Aldrich Syndrome