Prevalence of Pericarditis for Orphan Drug Designation Application

Analysis Group conducted analyses that supported Kiniksa in its successful Food and Drug Administration (FDA) application for orphan drug designation (ODD) for rilonacept for the treatment of pericarditis, a debilitating autoinflammation of tissue around the heart that often causes sharp chest pain. ODD is granted to products intended for the treatment of a rare disease, which the FDA defines as one that affects less than 200,000 persons in the US.

An Analysis Group team led by Managing Principal Mei Sheng Duh and Vice President Maral DerSarkissian conducted a national surveillance study using data from the National Ambulatory Medical Care Survey (NAMCS), National Hospital Ambulatory Medical Care Survey (NHAMCS), and National (Nationwide) Inpatient Sample (NIS) to estimate the annualized prevalence of pericarditis. A pooled, stabilized, annual prevalence was calculated and imputed to the size of the US population to arrive at a total number of US prevalent cases of pericarditis in 2020 of 168,205, which is below the FDA’s threshold of 200,000.

Based in large part on these analyses, FDA granted an ODD to Kiniksa’s rilonacept for the treatment of pericarditis on July 14, 2020.