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 97 studies in Orphan Diseases, we understand those challenges and have the expertise to help you successfully develop orphan drugs.
- Our regulatory and scientific expertise help to ensure the best designs for orphan diseases studies
- We can assist you with site selection, logistics, data management and operational strategy
- We bring together deep therapeutic experience, extensive knowledge of historical data registry and market access, and regulatory consulting
- Our statistical experts are able to implement methods to analyze clinical trials involving small patient numbers
Our experience includes 95+ rare diseases clinical studies in a broad range of indications:
| · ADA-Deficient SCID | · Mesothelioma | |
| · Anemia, Sickle Cell Disease | · Midgut Carcinoid Tumors | |
| · Chronic Idiopathic Thrombocytopenia Purpura (ITP) | · Multifocal motor neuropathy (MMN) | |
| · Crigler-Najjar syndrome | · Myasthenia Gravis (MG) | |
| · Cutaneous T-cell Lymphomas (CTCL) | · Niemann Pick Type C disease | |
| · Dermatomyositis | · Non-alcoholic Steatohepatitis | |
| · Friedreich’s Ataxia | · Prev. of acute graft vs host disease (aGvHD) | |
| · Gastric Cancer, Stomach, GIST | · Retinitis pigmentosa (RP) | |
| · Hailey-Hailey disease (HHD) | · Scleroderma | |
| · Hereditary Angioedema (HAE) | · Sickle Cell Disease | |
| · Lamellar Ichthyosis | · Spinal Muscular Atrophy | |
| · Leukemia (AML)/Myelodysplastic Syndrome (MDS) | · Stargardt disease | |
| · Liver/Biliary/Pancreas cancer | · Urea Cycle Disorders | |
| · LOHN: Leber Hereditary Optic Neuropathy | · Wilson’s Disease | |
| · Lymphoma, Non Hodgkin Lymphoma (NHL) | · Wiskott-Aldrich Syndrome | |