Rare Diseases and Orphan Drugs – Access & Reimbursement – Access & Reimbursement – Gene Therapies for Rare Diseases (US)
Given the availability of an 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 physicians’ prescribing of gene therapies that treat the neuromuscular diseases spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and cerebral adrenoleukodystrophy (CALD) and the hematological disorder beta thalassemia. In this report, we examine commercial plan coverage of 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-phase gene replacement therapies.
Questions answered
- What restrictions do MCOs impose on gene therapies in their largest fully insured commercial plans?
- What are the average rebates that payers collect for the approved gene therapies under study?
- How likely are neurologists to prescribe an emerging gene therapy such as intrathecal Zolgensma for SMA?
- What are the reasons why payers reject claims for gene therapies?
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.
