This partnership is rare.

Rare: adjective /rer/: harnessing the power that
comes from unity in a singular mission

Example: the rare disease research and patient

Personalized rare disease clinical development.

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

President of Rare Disease Business Unit

Joanna Reeder, Vice President of Project Management in the General Medicine Franchise for Worldwide Clinical Trials, 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 satisfy, if not exceed, customers’ expectations.

Michael Murphy, MD, PhD

Chief Medical and Scientific Officer

Amy Raymond, PhD, PMP

Senior Director, Therapeutic Strategy Lead, Rare Disease & Cellular and Genetic Medicines

Derek Ansel

Executive Director, Therapeutic Strategy Lead, Rare Disease

Juliane K. Mills, MS, MPH

Therapeutic Strategy Lead, Rare Disease

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.

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.

Rare Disease Experience

AutoImmune and Inflammation

  • Bullous Pemphigoid
  • IgA Nephropathy
  • Lupus Nephritis
  • Necrobiosis Lipoidica
  • Primary Sclerosing Cholangitis

Disorders of the Heart, Lungs, Kidneys

  • Focal Segmental Glomerulosclerosis
  • Idiopathic Pulmonary Fibrosis
  • Membranoproliferative Glomerulonephritis
  • Pulmonary Arterial Hypertension

Eye Disorders

  • Choroideremia
  • Fuchs Endothelial Corneal Dystrophy
  • Retinitis Pigmentosa

Musculoskeletal Disorders

  • Lambert–Eaton Myasthenic Syndrome
  • Multiple System Atrophy
  • Muscular Dystrophy
  • Pigmented Villonodular Synovitis

Blood Disorders

  • Acute Myeloid Leukemia
  • Idiopathic Thrombocytopenic Purpura
  • Sickle Cell Disease
  • Post-Essential Thrombocythemia Myelofibrosis

Genetic Disorders

  • Angelman Syndrome
  • Recessive X Chromosome Disorders

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

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

Rare Disease Resources