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 been named a 2022 CRO Leadership Award Champion in the categories of Compatibility, Quality, and Reliability across two respondent groups (Overall and Small Pharma). This is also the fourth year in a row in which PSI CRO received CRO Leadership Awards in all five categories, also including Expertise and Capabilities. The CRO Leadership Awards are presented by Life Sciences Leader and Clinical Leader based on research conducted by ISR reports. The awards recognize CROs that are voted by sponsors to meet or exceed expectations.

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).