Cristina Damatarca
Agility Clinical, USA
Title: Unique challenges and opportunities in conducting Pharmacovigilance for orphan drug
Biography
Biography: Cristina Damatarca
Abstract
Diseases that manifest in patient populations representing at the maximum 6–8% of the world population are defined as rare diseases or orphan diseases. These are pathologies whose incidence at birth is less than 1 in 2000. Orphan diseases are often so rare that a physician may observe only 1 case a year or less, and proper treatment may only be a personalized encounter between doctor and patient. One way to fill this therapy vacuum is by developing orphan drugs. Orphan drugs are a significant part of personalized medicine. In order to optimize the benefit-risk of these medicines, innovative pharmacovigilance methods need to be put in place that take into consideration the unique challenges of drug development for orphan indications as well as the realities of the patient population. The presentation will provide a summary of the existing methodologies for performing targeted pharmacovigilance for orphan drugs, as well as new innovative methods developed to address some of these challenges.