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RARE DISEASE 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. Conducting a rare disease trial comes with inherent challenges that arise from two elements; a sparse patient population and a wide geographical spread. The challenges cascade from there: assess the clinical reality of the protocol, identify the investigators, determine an enrollment rate and calculate an overall timeline based on various regulatory requirements in each country. When the cost per patient is considered, it becomes readily apparent that conducting thorough feasibility is a key critical success factor for orphan drug trials. PSI’s dedicated, global Feasibility Department, comprised of medical specialists, laboratory experts, imaging specialists, logisticians and regulatory experts, has conducted feasibility across a wide range of rare diseases. Each protocol has received a thorough medical, clinical, logistical and regulatory assessment. When we apply this process to your study and combine it with our knowledge of the nuances and complexities associated with conducting orphan drug trials, we can provide recommendations for the best choice of sites, geography and realistic timelines for your study. These elements allow our clients to determine the true price of a study.

For more information about PSI, please visit our website WWW.PSI-CRO.COM or reach us at CONTACT@PSI-CRO.COM

It has been a great pleasure to work with each of you. I truly admire your ability to work so effectively with the sites- I’m sure our study would not have been such a great success if it weren’t for the relationships you have built with our investigators. Clinical Trial Manger, USA

PSI has conducted clinical trials in the following rare & orphan indication diseases: Acute Peripheral Arterial Occlusion

Polycythemia Vera

Atypical Hemolytic Uremic Syndrome

Retinitis Pigmentosa

Cervical Dystonia

Sanfilippo Syndrome †

Cystic Fibrosis

Secondary Acute Myeloid Leukemia

Ewing Family of Tumors and Rhabdomyosarcoma †

Short Bowel Syndrome †

Gastrointestinal Stromal Tumors

Systemic Lupus Erythematosus

Hemolytic Paroxysmal Nocturnal Hemoglobinuria

Type 2 Diabetes Mellitus in Adolescents †

Hemophilia A and B †

Thrombotic Thrombocytopenic Purpura

Hunter Syndrome †

Von Willebrand Disease

Leber Congenital Amaurosis


NSCLC carrying known HER2 Activating Mutations

PSI has conducted feasibility in the following rare disease indications: Acromegaly

Idiopathic Thrombocytopenic Purpura

Actinic Keratosis

Lysosomal Acid Lipase Deficiency

Congenital Fibrinogen Deficiency



Merkel Cell Carcinoma

Growth Hormone Deficiency

Mucopolysaccharidosis Type I

Idiopathic Growth Hormone Deficiency

Myelodysplastic Syndrome

Idiopathic Pulmonary Fibrosis

Pilonidal Sinus

Hemophagocytic Lymphohistiocytosis

Pompe Disease

Huntington’s Disease


WWW.PSI-CRO.COM PSI CRO AG Corporate Headquarters Baarerstrasse 113a, 6300 Zug, Switzerland • Tel: +41 41 228 10 00

Global Office Locations North America: Ft. Washington, PA / Burlingame, CA / San Francisco, CA Latin America: Buenos Aires, Argentina Europe: Oxford, UK / Munich, Germany / Neusiedl am See, Austria Minsk, Belarus / Sofia, Bulgaria / Prague, Czech Republic / Tallinn, Estonia Budapest, Hungary / Milan, Italy / Warsaw, Poland / Bucharest, Romania Belgrade, Serbia / Kiev, Ukraine / Russian Federation: St. Petersburg, Moscow, Novosibirsk

Operational Presence by Region North America / Europe / Latin America Asia-Pacific / South Africa

Us new rarediseas  
Us new rarediseas