Because of the rarity, heterogeneity and the complex patient management associated with rare conditions, creating and executing an orphan drug development program presents some of the highest hurdles in medicine. Worldwide Clinical Trials approaches rare diseases with therapeutic acumen and operational sophistication. We understand the science, medicine, potential regulatory constraints, and the operational hindrances.
Worldwide has participated in the design, execution, and/or analysis of >50 studies in orphan diseases. Therapies have included small-molecule enzyme inhibitors, immunotherapies (monoclonal antibodies), hormone therapy, and enzyme replacement therapy
Dependability and Sensitivity — Globally
Our experience proves that a delicate and empathetic approach to parents and to very-ill patients can be combined effectively with superior project-management skills to master the logistics of geographic dispersion (eg, different cultures, varying standards of care, transportation infrastructure).
Global reach is essential to assure access to patients, a process which can be elusive. Protocols must account for highly diverse clinical manifestations and disease progression, effectively introduce dose-ranging solutions and create design structures which are compatible with the demands placed upon families who must present patients to specialized centers for evaluations.
Clinical trials in rare disease disorders are necessarily small. Alternative trial designs and statistical techniques are needed. Worldwide is familiar with advances in the science of small clinical trials and associated analytic methods. With our practical experience, and knowledge of methodologically rigorous options, we can propose study designs and data-analysis strategies suited to the challenges of orphan diseases.
Rare Disease Portfolio
- Active systemic onset juvenile idiopathic arthritis
- Amyotrophic lateral sclerosis
- Cystic fibrosis
- Duchenne muscular dystrophy
- Fabry disease
- Gaucher’s disease
- Growth hormone insensitivity syndrome
- Hemophagocytic lymphohistiocytosis
- Idiopathic pulmonary fibrosis
- Idiopathic thrombocytopenic purpura
- Lambert-Eaton myasthenic syndrome
- Mucopolysaccharidosis I (Hurler syndrome)
- Mucopolysaccharidosis II (Hunter syndrome)
- Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
- Sickle cell disease
- Thrombotic microangiopathy
- Von Willebrand disease
- Velocardiofacial syndrome, psychosis