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A disease is defined as “rare” in the U.S. when it affects fewer than 200,000 at any time; in Europe, when it affects fewer than 1 in 2,000. Because rare diseases are misdiagnosed or take years to diagnose, many patients receive insufficient treatment and support. Rare Disease Day was established to raise awareness about the impact on patients’ lives.

Progress is being made, through the sharing of scientific knowledge and international cooperation in clinical research, leading to the development of new treatments. Enacted in 1983, the U.S. Orphan Drug Act is helping expedite the development of “orphan drugs,” or treatments for rare diseases.

How Worldwide Clinical Trials Addresses Rare Diseases

Rare disease clinical trials have unique challenges, ranging from access to a patient population to regulatory hurdles. Worldwide Clinical Trials has risen to that challenge, participating in the design, execution, and/or analysis of more than 60 studies for rare diseases since 2008. With over 1,400 employees in 60+ countries, Worldwide has the global reach and therapeutic expertise to meet the complex rare disease trial needs for a wide range of indications, including:

  • Duchenne muscular dystrophy
  • Sickle cell disease
  • Idiopathic pulmonary fibrosis
  • Cystic fibrosis
  • And more