Gene Therapies for Rare Diseases | Access & Reimbursement | US | 2024
As the number of approved high-cost gene therapies increases, 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 spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), cerebral adrenoleukodystrophy (CALD), and beta thalassemia. The report examines the commercial plan coverage of and restrictions facing several marketed therapies (e.g., Novartis’s Zolgensma for SMA, Sarepta Therapeutics’ Elevidys for DMD, Bluebird Bio’s Skysona for CALD and Zynteglo for beta thalassemia, Vertex Pharmaceuticals / CRISPR Therapeutics’ Casgevy) and assesses the market access potential for gene replacement therapies in late-phase development.
Questions answered
- What restrictions do MCOs impose on gene therapies in their largest fully insured commercial plans?
- 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 factors are critical for payers to reimburse the first CRISPR-based therapy (Casgevy) for transfusion-dependent beta-thalassemia patients?
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 behaviour in the market access environment. Each report includes up-to-date analyses of drug coverage and restriction policies as well as payer and prescriber perspectives on key marketed drugs and their receptivity to emerging therapies.
Solution enhancement
Access & Reimbursement includes key analyses from Clarivate Real-World Data Product—comprehensive and timely data that provide clarity around the healthcare experiences and activities of hundreds of millions of patients, HCPs, and payers. Analyses include payer mix, percentage of claim rejections for key therapies, and reasons for claim rejection.
