Gene Therapy – Access & Reimbursement – Access & Reimbursement – Gene Therapies for Rare Diseases (US)

With the increasing number of approved innovative, high-cost gene therapies, value assessment and novel reimbursement approaches are rapidly gaining importance. To assist developers of gene therapies in planning for market access challenges, this study analyzes MCO reimbursement policies and their impact on physicians’ prescribing of gene therapies for neuromuscular diseases, including SMA, DMD, and CALD, as well as the hematological disorder beta thalassemia. In this report, we examine commercial plan coverage and restrictions on several marketed therapies—including Novartis’s Zolgensma for SMA, Sarepta Therapeutics’ Elevidys for DMD, Bluebird Bio’s Skysona for CALD and Zynteglo for beta thalassemia, as well as Vertex Pharmaceuticals / CRISPR Therapeutics’ Casgevy—and assess the market access potential for late-stage gene replacement therapies.

Questions answered

• What restrictions do MCOs impose on gene therapies in their largest fully insured commercial plans?
• What are the coverage criteria large insurers are placing on these therapies?
• What is the expected duration of effect payers believe is needed to justify reimbursement of gene therapies?
• What are the average rebates that payers collect for the approved gene therapies under study?
• How likely are neurologists to prescribe the recently approved gene therapies, such as Itvisma (intrathecal Zolgensma), for SMA?

Product description

Leveraging Clarivate’s proprietary data sources and surveys with physicians and payers, Access & Reimbursement provides integrated brand- and disease-level insight on reimbursement dynamics and the impact of payer policy on physician prescribing behavior in the United States. Through detailed analyses of drug coverage, restriction policies, and contracting, as well as payer and prescriber perspectives on key marketed drugs and their receptivity to emerging therapies, this report enables you to:

• Identify and learn how to overcome roadblocks to market access to best position your brand.
• Identify key stakeholders and reimbursement drivers and assess the impact that reimbursement decisions have on treatment selection.
• Evaluate competitor strategies for securing favorable market access terms.