Rare Diseases and Orphan  Products

Is payer scrutiny impacting access to your orphan drug? 

Your dedication and investment to help patients facing rare diseases is one we are especially proud to support. Your commitment to creating unique therapies is inspiration for our equally unique insights and capabilities to address the complex educational, access and reimbursement challenges orphan drugs face.  Take a moment to learn more about the ways we may be able to partner with you.

Helping your most specialized populations – an Xcenda analysis

Duchenne muscular dystrophy (DMD) is a devastating, muscle-wasting disease  affecting only a small number of mostly adolescent males. However, discussions on evidentiary requirements, pricing, and access of pharmaceuticals are making international headlines. While there is no cure for DMD, a few treatments have reached advanced clinical development. Eteplirsen, ataluren, and deflazacort have garnered extensive media coverage related to appraisal and market access. Join our experts as they examine ways to sustain investment to ensure affordability for all stakeholders including patients, prescribers, payers, and society.
Young man's hand controlling motorized wheelchair

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