Each year, doctors diagnose about 1,000 Americans with cystic fibrosis, a rare hereditary disease that affects about 30,000 people in the United States. Thanks to advances in treatment, people with cystic fibrosis often live until their early 40s, but there is no cure for the disease.1
Life-threatening rare diseases like cystic fibrosis affect about one out of 15 people, or 400 million, worldwide according to International Federation of Pharmaceutical Manufacturers and Associations (IFPMA). Many of these rare diseases have therapies available to curb symptoms, but no effective treatments.2
Pharmaceutical manufacturers and biotechnology companies are working hard to address these unmet medical needs. However, difficulty understanding underlying disease mechanisms, as well as the challenge of finding adequate sample sizes, have made the process of bringing orphan drugs to market a frustrating process.
Patient registries assist orphan drug development by giving manufacturers access to clinical, genetic, and biological data from a large patient population. Using registries, sponsors can better understand the disease, its history, and patients’ needs, all of which benefit clinical trial design and effectiveness.