Rare Diseases and Orphan Drugs – Access & Reimbursement – Rare Diseases and Orphan Drugs – Top Five European Markets


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.


  • Focusing on SMA and beta thalassemia, what factors influenced HTA and P&R outcomes for current orphan agents?
  • What impact do gene therapies and other orphan drugs have on payer budgets? What measures will payers implement to rein in costs?
  • What pharmacoeconomic models will payers use to assess the value of orphan drugs and gene therapies? How will curative therapies be valued?
  • What funding mechanisms are used for orphan drugs, and what measures are used for controlling and monitoring prescribing across the top five European markets?
  • How will prescribing barriers for current orphan drugs evolve, what challenges will emerging orphan drugs likely face in the near future? What can manufacturers do to encourage optimal pricing and reimbursement terms, and to drive uptake?


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).


Spinraza, Zolgensma, risdiplam, branaplam, Zynteglo, luspatercept


  • Actionable recommendations to optimize market access
  • Market access success and stumbles
  • Market access roadblocks
  • Pricing and reimbursement dynamics
  • Impact of payer policy and coverage on prescribing
  • Market access outlook for emerging therapies