With the increasing number of approved innovative, high-cost gene therapies, value assessment and novel approaches to reimbursement are rapidly gaining importance. To assist developers of gene therapies in planning for market access challenges, this study analyzes MCO reimbursement policies and their effect on neurologists’ prescribing of gene therapies that treat the debilitating neuromuscular rare diseases spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). The report examines commercial plan coverage and restrictions for several marketed therapies—including Novartis’s Zolgensma and Biogen’s Spinraza for SMA, and Sarepta Therapeutics’ Exondys 51 and Vyondys 53 for DMD—and assesses the market access potential for late-phase gene replacement therapies for DMD.
Geography: United States.
Primary research: Survey of 100 U.S. neurologists who manage patients eligible for gene therapies. Survey of 30 U.S. managed care organization (MCO) pharmacy and medical directors (PDs/MDs).
Key drugs covered: Zolgensma, Spinraza, Exondys 51, Vyondys 53, Evrysdi, Viltepso, Amondys 45, key emerging gene therapies.
U.S. Access & Reimbursement provides integrated brand- and disease-level insight on reimbursement dynamics and the impact of U.S. payer policy on physician prescribing behavior in the market access environment, including up-to-date analysis of drug coverage and restriction policies and payer and prescriber perspectives on key marketed drugs and receptivity to emerging therapies.