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Latest Trends in Orphan Drug Pricing and Reimbursement

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  • 135 Pages
  • February 2018
  • Region: Global
  • Citeline
  • ID: 4489132
It is now some 35 years since the passage of the first law designed specifically to encourage investment in the discovery and development of rare disease treatments. Activity in the sector has been transformed since then. Once a neglected backwater, orphan drug development is now the subject of a research boom that is delivering innovative new treatments for rare diseases to the market in unprecedented numbers. Many carry premium price tags, and while overall pharmaceutical market growth rates have slowed, spending on orphan drugs is rising at double-digit rates.

Recent trends have sparked a debate on the structure of existing orphan drug laws, which critics say have been overtaken by scientific developments and are being exploited by the pharmaceutical industry. Regulators in both the US and Europe have announced plans to review current legislation, but opposition to substantive amendments on both sides of the Atlantic is strong, and major change appears unlikely in the foreseeable future. In the meantime, payers are being asked to fund orphan drug coverage bills that will reach new heights as the first wave of gene therapy products reaches the market. With their budgets under growing pressure, payers in most markets have begun to push back, implementing measures designed to limit their exposure to costs associated with orphan drug reimbursement.

This report provides a detailed analysis of orphan drug coverage, funding, and pricing systems in the US, Japan, and the EU's five biggest pharmaceutical markets (France, Germany, Italy, Spain and the UK). It also tracks recent developments in orphan drug pricing and reimbursement across these seven countries, and describes the impact of those changes on rare disease treatments in general, and on specific products in particular. Finally, it addresses some of the major issues facing payers and orphan drug manufacturers, highlighting the way these have been handled by both parties through individual case studies.

Table of Contents

  • Orphan drug coverage is beginning to test payer budgets
  • Some P&R frameworks are ill-equipped to deal with orphan drugs
  • Payers have begun to take evasive action
  • Some orphan manufacturers have learnt expensive lessons

  • Incentivizing investment in orphan drug R&D
  • From backwater to boom
  • Time for a rethink of orphan drug rules?
  • Payers are looking for ways to rein in orphan drug costs
  • Bibliography

  • Established systems reflect a benign approach to orphan coverage
  • Raising the orphan P&R bar
  • Bibliography

4 US
  • Still few constraints on orphan coverage or prices
  • Orphan-specific P&R rules are notable by their absence
  • Rising orphan costs begin to prompt closer scrutiny
  • Bibliography

5 UK
  • Orphan coverage reflects fragmented regulatory framework
  • Payers turn the screw as NHS funding crisis bites
  • Bibliography

  • New pressures on orphan drug prices emerge
  • Orphan-specific rules focus on pricing, not reimbursement
  • Payers seek increased leverage over orphan prices
  • Bibliography

  • Payers toughen stance on orphan prices
  • Orphans are reimbursed widely, but price is an issue
  • Calls for a tighter line on orphan P&R are growing
  • Bibliography

  • MEAs will remain the key to orphan access
  • Most orphans benefit from special provisions for "innovative" drugs
  • Payers may shift the focus of MEAs as orphan spend rises
  • Bibliography

  • Orphan coverage hit by healthcare funding crisis
  • National decisions determine orphan P&R status
  • Orphan P&R approval rates plunge as funding crisis bites
  • Bibliography

  • P&R overhaul will have implications for orphans
  • Reimbursement is comprehensive, but prices are tightly regulated
  • Spending surge prompts new P&R reform drive
  • Bibliography

  • Experience, and lessons learned
  • Data limitations pose a challenge for regulators, as well as payers
  • Payers crave certainty, where often there is none
  • Maximizing prospects for positive P&R outcomes
  • Case studies
  • Glybera and Strimvelis - salutory lessons for gene therapy pioneers
  • Orkambi - offering less to more proves a hard sell for Vertex
  • Exondys 51 - Sarepta works hard to win over skeptical payers
  • Imbruvica - highlighting the P&R gulf between Europe and the US
  • Kymriah and Yescarta - CAR-T pioneers break the US pricing mold

Table 1: Orphan drug definitions and development incentives
Table 2: Orphan drug indication approval numbers in the US, EU, and Japan
Table 3: Lenalidomide orphan designations, marketing authorizations, and indications in the US
Table 4: Global sales of Revlimid (lenalidomide)
Table 5: Orphan status of oncology drugs approved by the FDA in 2016
Table 6: Overview of approaches to orphan drug P&R in the US, Japan, and Europe
Table 7: Turning the screw on orphan drug reimbursement
Table 8: Potential solutions to the orphan drug funding challenge
Table 9: Leveraging pressure on orphan drug prices
Table 10: Relative exposure of marketplace plan holders to orphan drug costs
Table 11: Access to orphan drugs in the five major EU markets
Table 12: NICE appraisal outcomes
Table 13: Positive ultra-orphan drug appraisals by NICE
Table 14: Patient access schemes for orphan drugs recommended by NICE
Table 15: Ultra-orphan drugs in the NICE appraisal queue
Table 16: ASMR scores for new orphan drugs reviewed recently by HAS
Table 17: Change in ASMR rating following orphan drug reassessment
Table 18: Comparison of G-BA benefit assessment outcomes for orphan and non-orphan drugs*
Table 19: Orphan drug monitoring programs linked to performance-based MEAs
Table 20: Orphan drug spending and consumption trends in Italy
Table 21: Orphan drugs being bankrolled by AIFA's new innovative drug funds
Table 22: Benefits conferred on innovative drugs by AIFA's new algorithm
Table 23: Main contributors to hospital spending on orphan drugs in Spain
Table 24: Decline in P&R approval rates for non-cancer orphan drugs in Spain
Table 25: Flexible approach to the regulatory review of orphan drugs in the US
Table 26: Orkambi takes off in the US, but struggles elsewhere
Table 27: Global sales of ruxolitinib