Rapid US & EU Opportunity Assessment of an Orphan Disease Therapeutic

Certara completed key opinion leader (KOL), payer, and patient research by conducting in-depth telephone interviews. This research provided key insights into development of a clinical program to maximize adoption and value of the treatment. These findings helped craft target product profiles, develop a payer landscape, and identify the price potential for each target product profile in the expected competitive environment.

A disease is considered rare when it affects fewer than 1 in 2,000 people. Although each rare disease does not affect many patients, there are estimated to be over 7,000 rare diseases. Individuals diagnosed with rare/orphan diseases are often left with no or limited treatment options. These conditions are often neglected because their treatments are not considered profitable due to their high costs and limited patient population.