Gene Therapies for Rare Diseases | Access & Reimbursement | US | 2022

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.

Questions answered

• What restrictions do MCOs impose on gene therapies in their largest fully insured commercial plans, and to what extent do they limit neurologists’ prescribing of these therapies?
• What kind of contractual arrangements, including rebate contracts and risk-based contracts, do MCOs have with marketers of gene therapies?
• What kinds of price concessions and clinical data do payers want to see to favorably cover emerging gene therapies, and what pharmacoeconomic models are they most likely to use in their decision-making?
• How likely are neurologists to prescribe emerging gene replacement therapies for DMD? What payer controls would constrain their prescribing of these therapies?

Content highlights

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.

Product description

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.