The challenges of rare disease research, a global approach

Predictability in rare disease clinical trials

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.

PSI’s overall therapeutic expertise includes the management of pharmaceutical trials in a variety of rare disease indications. Treating rare diseases is one of the most rapidly expanding areas in clinical research yet there is no consensus on what constitutes an ideal approach. PSI’s methodology involves applying the lessons learned from pediatric and oncology drug development to rare disease programs. That means we can speak with you about project planning, protocol realities, logistical possibilities and the execution of all aspects of your clinical trial.

“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

The PSI Advantage

PSI CRO has received 2024 CRO Leadership Awards in the categories of Expertise, Quality, Compatibility, and Reliability in the Overall (combined Big and Small Pharma) respondent group. This is the seventh consecutive year that PSI has received leadership awards presented by Clinical Leader and Life Science Connect based on research conducted by ISR. The awards recognize CROs that are voted by sponsors to meet or exceed expectations.

PSI wins CRO Leadership Awards for Expertise, Compatibility, Quality, and Reliability

Therapeutic areas

Inflammatory Bowel Diseases (IBD)

Crohns’ Disease, Ulcerative Colitis and other IBDs.


Breast Cancer, Lung Cancer, Prostate Cancer, Colorectal Cancer, Ovarian Cancer and other Oncology indications.


Multiple Myeloma, Lymphomas, Leukemias and other oncohematological indications.

Infectious Diseases

Antimicrobial and Antifungal infections and other ID.

Multiple Sclerosis

Multiple Sclerosis, Spasticity and other neurological indications.

Rare Diseases

Hemophilia A & B, Von Willebrand Disease, Thrombocytopenia, Polycythemia Vera, Paroxysmal Nocturnal Hemoglobinuria (PNH).