RARE DISEASES
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THE PSI STORY
Rare Diseases Clinical Trials
Approved
www.psi-cro.com
PSI’s strength is in its people and their attitude. The PSI management team that founded the company 18+ years ago is still here and fully engaged in day-to-day project life. “Navy seals of the CRO industry” was how one of our clients described PSI. In our industry more than 90% of clinical trials fall behind deadlines, while 95% of PSI studies finish on-time or ahead of schedule. We subscribe to the belief that “it’s kind of fun to do the impossible”. We maintain our practice of investing in talent because we know that only devoted and accountable people can deliver the kind of service that will ensure outstanding results for our clients. Our mission is to be the best CRO in the world as measured by our clients and our employees. With 1,400 folks in 50+ countries we have a lot of talent to look after. Dozens of customers in a variety of therapeutic areas have come to PSI and stayed. Maybe that is why after 18+ years in the industry PSI still enjoys a solid reputation and grows mainly through repeat and referral business. When combined, these characteristics yield tangible outcomes for our customers. Studies run on time and on budget and save hundreds of millions of development dollars.
MYELODYSPLASTIC SYNDROME REGULATORY CONSULTANCY FEASIBILITY STUDY VON WILLEBRAND DISEASE HUNTER SYNDROME CREATE CRF SET UP STUDY DATABASE STATS ANALYSIS PLAN SITE SELECTION SET UP STUDY LOGISTICS STUDY FILING WITH COMPETENT AUTHORITIES
AHEAD ON TIME ON TIME AHEAD ON TIME AHEAD AHEAD ON TIME ON TIME ON TIME
SITE CONTRACTS HEMOPHILIA A AND B
ON TIME ON TIME AHEAD ON TIME AHEAD ON TIME ON TIME AHEAD AHEAD AHEAD ON TIME AHEAD ON TIME
EWING FAMILY OF TUMORS AND RHABDOMYOSARCOMA
SITES ENROLLMENT READY POLYCYTHEMIA VERA FIRST PATIENT IN SHORT BOWEL SYNDROME NSCLC CARRYING KNOWN HER2 ACTIVATING MUTATIONS
LAST PATIENT IN LAST PATIENT OUT SECONDARY ACUTE MYELOID LEUKEMIA LOCK DATABASE FINALIZE INTEGRATED REPORT
ON TIME
ACCOUNTABILITY: DOING THE RIGHT THING PSI’s reputation is that of a no-nonsense CRO that delivers by taking the right actions even when that is the harder path. However well one plans a study, the unexpected will happen. What differentiates a good vendor from everyone else is the manner in which difficulties are managed. At PSI, studies enroll on time not only because they are well-planned, but also because the project teams assume an attitude of “get it done or die trying”. Most of the projects we undertake are “tough”. PSI has won many unwinnable battles by sheer persistence in an effort to keep our promises to our clients. To us, doing the right thing means: • We do our best to keep your expectations in line with reality because we are accountable for every promise we make. • We speak our truth even if it is not what you expect to hear. We wish to guide you on the right path, not let you believe something just because it is what you want to hear. • We show you how we can meet your study timelines by telling you which countries can enroll and how to realistically integrate the contributions of strategically important regions and KOLs.
Feasibility Completed: Ahead of schedule
Protocol Completed: On-Time
TICAL LOG7I.0S6.2013 1
MEDICAL
FEASIBILITY PSI is an expert at conducting regional or global feasibility that provides the best choice of sites and geography with accurate recruitment forecasts. In recent years, our specialists located in 11 countries of North America, Europe, Asia and Latin America have conducted hundreds of feasibility assessments for Rare Disease programs studies in 50+ countries. These assessments cover, but are not restricted to, evaluation of the target population availability and factors that limit it, standards of care, competitive studies, regulatory climates and the need to engage particular Key Opinion Leaders into the study. We weigh all of these factors before making recommendations on the best choice of sites and geography for your program. 95% of our studies are finished on time or ahead of schedule.
.2013
25.10
CAL
I CLIN
Comprehensive Project Plan: On-Time
Database Lock: Ahead of Schedule
PSI HAS CONDUCTED CLINICAL TRIALS IN THE FOLLOWING RARE & ORPHAN INDICATION DISEASES: Making the Impossible POSSIBLE 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.
Acute Peripheral Arterial Occlusion Cervical Dystonia Cystic Fibrosis Ewing Family of Tumors and Rhabdomyosarcoma † Gastrointestinal Stromal Tumors Hemolytic Paroxysmal Nocturnal Hemoglobinuria Hemophilia A and B † Hunter Syndrome † Leber Congenital Amaurosis Myelodysplastic Syndrome †
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: 1) assess the clinical reality of the protocol 2) identify the investigators 3) 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.
First Patient In: On-Time
Last Patient In: Ahead of schedule
pediatric indications
NSCLC carrying known HER2 Activating Mutations Polycythemia Vera Retinitis Pigmentosa Secondary Acute Myeloid Leukemia Short Bowel Syndrome † Systemic Lupus Erythematosus Type 2 Diabetes Mellitus in Adolescents † Thrombotic Thrombocytopenic Purpura Von Willebrand Disease
PSI HAS CONDUCTED FEASIBILITY IN THE FOLLOWING RARE DISEASE INDICATIONS: Acromegaly Actinic Keratosis Congenital Fibrinogen Deficiency Ependymoma Growth Hormone Deficiency Idiopathic Growth Hormone Deficiency Idiopathic Pulmonary Fibrosis Hemophagocytic Lymphohistiocytosis Huntington’s Disease
Last Patient Out: Ahead of schedule
First Patient In: On-Time
Idiopathic Thrombocytopenic Purpura Lysosomal Acid Lipase Deficiency Mastocytosis Merkel Cell Carcinoma Mucopolysaccharidosis Type I Pilonidal Sinus Pompe Disease Sanfilippo Syndrome Scleroderma
Last Patient In: Ahead of schedule
ENROLLMENT GRAPHS
Hunter’s syndrome – Phase I/II 35 30 25 20 15 10
Planned Actual
5 0
Dec
Jan
Feb
Mar
Apr
Safety Database Set-up: On-Time
May
Jun
Jul
Aug
Sep
Oct
Nov
Dec
Jan
Final Statistical Analysis: Ahead of Schedule
Hemophilia B – Phase III
80
70 60
50 40
Planned
30
Actual
20
Jun
Apr
May
Mar
Jan
Feb
Dec
Oct
Nov
Sep
Jul
Aug
Jun
Apr
May
Mar
Jan
Feb
Dec
Oct
Nov
Sep
Jul
Aug
Jun
Apr
0
May
10
Secondary Acute Myeloid Leukemia – Phase III
On-Time Enrollment
250 Physicians On-Board
On-Time Project Delivery
The geographical span for our Rare Disease experience
The geographical span for our Rare Disease Feasibility Experience
Study Filings with Competent Authorities: On-Time
Sites Enrollment Ready: On-Time
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