The challenges of rare disease research, a global approach
Rare disease trials come with inherent challenges
arising from two elements; a sparse patient population and a wide geographical spread.
And the challenges just cascade from there. When the cost per patient is considered, it becomes readily apparent that conducting through feasibilty is a key critical success factor for orphan drug trials. PSI-managed sites show consistently stellar performance in rare disease trials, and every last one have completed recruitment on time or ahead of trials.
With new innovations in gene therapy, and the unraveling of rare disease medical mysteries closer and closer to reality, rare disease clinical research trials are more important now than ever before.
PSI’s key differentiator is our ability to plan and execute complex clinical trials on time, saving our customers millions of development dollars.
“I'm always impressed with how PSI employees conducted themselves, and I frequently hear them say 'That is not a problem' no matter how big or small the request may be.”
Director, Clinical Operations USA
Therapeutic areas
Gastroenterology (GI)
Inflammatory bowel diseases (Crohn's disease, ulcerative colitis) and other GI indications.
Oncology
Breast cancer, lung cancer, prostate cancer, colorectal cancer, ovarian cancer, and other oncology indications
Radiopharmaceuticals
Hematology
Multiple myeloma, lymphomas, leukemias, hemophilia and other bleeding disorders
Infectious Diseases (ID)
UTI, CAP/VAP/HAP, skin infections, bacteremia, fungal diseases, sepsis, hepatitis, and other ID
Neurology
Multiple sclerosis, spasticity, stroke, Alzheimer's, Parkinson's, and other neurological indications
Rare Diseases
Hemophilia A & B, von Willebrand disease, thrombocytopenia, polycythemia vera, PNH, and other rare diseases
