Even Small Studies Can Make a Major Impact in Rare Diseases
Unique logistical challenges and lack of precedence can make rare disease trials intimidating. Worldwide offers customized insight, deep expertise and novel solutions to get the most out of your efforts.
OUR EXPERTS IN RARE DISEASE
The standard approaches used in large-scale studies often don’t apply to the world of rare disease research, so intelligent innovation and careful consideration can help maximize the impact of all data, even from a small number of patients. You need a team that combines deep scientific expertise with fresh perspectives on design and patient outreach—experts with decades of experience in the basics and the willingness to innovate to find a unique approach that will work for your program.
JOANNA REEDER
VICE PRESIDENT, PROJECT MANAGEMENT, OTHER AND RARE DISEASES
Joanna has more than 30 years’ experience in directing global projects in many therapeutic areas across all phases of clinical trials. As a key member of Worldwide’s leadership team, her personal aim is to motivate and empower project teams to achieve project goals and surpass customer expectations. Her career has focused on managing portfolios of work in the endocrinology and inflammatory therapeutic areas, and she has significant experience in autoimmune diseases, rare disease indications, biosimilars, and rheumatology.
DEREK ANSEL
SENIOR DIRECTOR, THERAPEUTIC STRATEGY LEAD, RARE DISEASES
Derek has worked in clinical research for more than ten years with a focus almost exclusively in rare and pediatric diseases, including non-malignant hematology, autoimmune diseases, metabolic disorders, movement disorders, and other genetic conditions. At Worldwide, he leads and supports corporate initiatives within rare and pediatric diseases and maintains relationships with over 45 patient-focused advocacy organizations. Derek holds a master’s degree in Pharmacology and Toxicology from Michigan State University and is pursuing a master’s degree in Genetic Counseling from Bay Path University.
IAN BRAITHWAITE, PHD
SENIOR VICE PRESIDENT, PROJECT MANAGEMENT, GENERAL MEDICINE AND RARE DISEASE
Ian’s career in clinical research spans three decades and includes experience in both CROs and pharmaceutical companies. At Worldwide, he works to improve the lives of patients by shepherding novel treatments from the lab to the marketplace. Ian oversees strategic development, project management, and operational delivery across immunology/inflammatory diseases (including rheumatoid arthritis, lupus, psoriasis, IBD, asthma, COPD, and more), rare diseases (such as sickle cell disease, IPF, bullous pemphigoid, cutaneous T-cell lymphoma), and oncology.
Novel endpoint development for diverse diseases
Our expertise in biomarker development and custom-validated clinical assessments takes the guess work out of selecting the proper endpoints for your clinical trial.
True passion for the patient experience
Finding patients remains a critical piece in rare disease research. We have decades of experience working with patients, families, advocacy groups, and key opinion leaders in rare conditions to help recruit and retain our most vulnerable patient populations.
Knowledgeable regulatory collaboration
Worldwide collaborates early and often with regulatory agencies to improve the selection of outcomes measures and the appropriateness of endpoints. Our nimble global network helps ease the way toward submission—without last-minute surprises—for approval by regulatory agencies around the world.
Agility and experience for smooth trial execution
Rare disease trials are increasingly more complex. Worldwide navigates these immense logistical challenges using smart operations planning, our vetted global network, and our experienced team to ensure that even the most complex rare disease trials run smoothly toward a new drug application.
You Need a CRO to Go the Extra Mile
Rare and orphan disease clinical research presents a unique set of obstacles. That’s why your drug development program deserves a partner with the right tools to help you navigate the journey.
Rare Disease Experience
- AutoImmune and Inflammation
- Bullous Pemphigoid
- IgA Nephropathy
- Lupus Nephritis
- Necrobiosis Lipoidica
- Primary Sclerosing Cholangitis
- Blood Disorders
- Acute Myeloid Leukemia
- Idiopathic Thrombocytopenic Purpura
- Sickle Cell Disease
- Post-Essential Thrombocythemia Myelofibrosis
- Disorders of the Heart, Lungs, Kidneys
- Focal Segmental Glomerulosclerosis
- Idiopathic Pulmonary Fibrosis
- Membranoproliferative Glomerulonephritis
- Pulmonary Arterial Hypertension
- Endocrine and Metabolic Disorders
- Congenital Adrenal Hyperplasia
- Fabry Disease
- Familial Chylomicronemia Syndrome
- Fatty Acid Biosynthesis Disorders
- Growth Hormone Deficiency
- Homozygous Familial Hypercholesterolemia
- Lysosomal Storage Disorder
- Mucopolysaccharidosis
- Niemann-Pick Disease
- Phenylketonuria
- Tyrosinemia
- Adrenal Insufficiency
- Eye Disorders
- Choroideremia
- Fuchs Endothelial Corneal Dystrophy
- Retinitis Pigmentosa
- Genetic Disorders
- Angelman Syndrome
- Recessive X Chromosome Disorders
- Musculoskeletal Disorders
- Lambert–Eaton Myasthenic Syndrome
- Multiple System Atrophy
- Muscular Dystrophy
- Pigmented Villonodular Synovitis
- Nervous System Disorders
- Amyotrophic Lateral Sclerosis
- Batten Disease
- Charcot-Marie-Tooth Disease
- Huntington Disease
- Neuromyelitis Optica
- Vanishing White Matter Disease
- Rare Cancers
- Cutaneous T-Cell Lymphoma
- Gastrointestinal Stromal Tumor
- Glioblastoma Multiforme
- Hepatocellular Carcinoma
- Triple-Negative Breast Cancer
- B-Cell Non-Hodgkin Lymphoma
Our Dedicated Team
Eteri Tsetskhladze, MD, PhD, FESC, FACC
Vice President, General Medicine Medical Lead, Medical Affairs
Barry Dussault
Executive Director, Project Management, Franchise Area Lead, Analgesia, Addiction, and Neurology Rare Disorders
