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.


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. 



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.

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