A rare disease, also called an “orphan” disease, is any condition affecting a small percentage of the population. While there is no single cutoff for what is considered a “small percentage,” the World Health Organization has suggested a condition affecting less than 1 in every 1,000 people, whereas the U.S. Rare Diseases Act of 2002 uses a stricter cutoff of 1 in every 1,500 people.
Recruiting patients with rare/orphan diseases can be challenging, but our team has developed successful outreach strategies in both adult and pediatric populations. We work closely with clinical sites, patient advocacy groups, and non-profit disease foundations to meet specific study goals. We have successfully conducted studies of such rare/orphan conditions as thalassemia, sickle cell anemia, hemophilia, Marfan Syndrome, and sudden infant death syndrome (SIDS).
Our Clinical Research experts have worked collaboratively in complex studies of rare/orphan diseases with multi-specialty teams and multiple stakeholders, e.g., industry sponsors, government agencies, professional societies, and foundations. Offerings to our clients include customized clinical trials, epidemiology, health and policy research, and patient registry services in areas as diverse as implantable pediatric medical devices and periventricular leukomalacia (a brain injury in premature infants).
