Progress in R&D and technology, regulatory and monetary incentives for advancing the development of rare diseases treatment, and ongoing industry focus are key drivers of growth in the number of approved and emerging orphan drugs. As the number of rare disease approvals climbs, the discussion around orphan drug value assessment, particularly for curative gene therapies, is rapidly evolving. A key concern for developers is how payers will control the growing cost of orphan cell and gene therapies, which often command high prices, and how payer policy will affect prescribing of these essential treatments. This study analyzes the health technology assessment (HTA) and pricing and reimbursement (P&R) policies that regulate market access for orphan drugs, specifically exploring the evolving dynamics affecting reimbursement and uptake of current and emerging orphan drugs for existing therapies in spinal muscular atrophy (SMA) and beta thalassemia. The secret of market access success for gene therapies launched in these indications will serve as insightful case studies for emerging curative gene therapies in other rare diseases.
European Access & Reimbursement provides integrated brand-level and country-specific analysis of primary market research conducted with payers and prescribers to examine reimbursement dynamics and evaluate the impact of payer policy on prescribing.
France, Germany, Italy, Spain, and United Kingdom
Survey of 250 medical oncologists across the top 5 European markets (50 per country)Interviews with 10 payers top 5 European markets (2 per country).
KEY DRUGS COVERED
Spinraza, Zolgensma, risdiplam, branaplam, Zynteglo, luspatercept