Orphan Drug Access and Pricing in Emerging Markets

  • ID: 4462208
  • Report
  • 59 pages
  • Datamonitor Healthcare
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This analysis covers the four major emerging regions of the Middle East and Africa, Latin America, Eastern Europe, and Asia-Pacific. Within these regions, relatively wealthier markets with a proven marketplace for orphan medicines have been prioritized. The topic focus is on rare-disease policy, access, and pricing, as well as the stakeholder landscape for low-prevalence disorders and orphan drugs. For the purposes of this analysis, local definitions for rare disease were used where available, as well as conventional definitions of both “ultra-orphan” disorders and rare tumor types.

The insights in this analysis are aimed at a general audience within the market access and pricing field, with a limited amount of disease-specific information. In this review, key market access customers for rare diseases in 20 emerging markets have been analyzed, as well as pricing and funding pathways where these specifically relate to orphan drugs.
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EXECUTIVE SUMMARY
Budget limitations are a major hurdle but opportunities still exist for some rare diseases
Each market will require customized access strategy to maximize chance of success
Lack of diagnosis and treatment infrastructure, coupled with payer rigidity present key access challenges
Gulf region presents the most lucrative opportunity, with well-funded niches in other regions

KEY FEATURES OF EMERGING ORPHAN DRUG MARKETS
Orphan drugs and emerging markets are industry growth engines
The orphan drug business model requires local adaptation
Bibliography

RARE DISEASE POLICY AND CAPABILITY
Prevalence-based definitions of rare diseases are a critical step
Legal frameworks are often incoherent
Challenges in diagnostic and treatment infrastructure
Policy does not guarantee access
Bibliography

PRICING, REIMBURSEMENT, AND MARKET ACCESS
Payers’ rigidity is the key market access challenge
Exceptional access mechanisms exist in some leading emerging markets
Payers misuse international price referencing for orphan drugs
Registries are evolving in emerging markets
Bibliography

PRIORITY EMERGING ORPHAN DRUG MARKETS
The Gulf states are the stand-out opportunity
Only the Middle East matches prevalence with funding
Heterogeneity drives specific local needs
Emerging markets are not ready for new technology
Manufacturers should match resources to access
Bibliography

APPENDIX
About the author
Methodology
Exchange rates

List of Figures
Figure 1: The author's insights and recommendations
Figure 2: Key differences between emerging and advanced orphan drug markets
Figure 3: Insights and recommendations
Figure 4: Funded gold-standard treatments for selected rare diseases in key emerging markets
Figure 5: Case study: Vyndaqel in Brazil
Figure 6: Case study: 2017 reimbursement list update in China
Figure 7: Insights and recommendations
Figure 8: Market access requirements for orphan drugs in selected emerging markets
Figure 9: Case study: Soliris in Mexico
Figure 10: Case study: Spinraza and Ofev in Turkey
Figure 11: Case study: hemophilia in Colombia
Figure 12: Insights and recommendations
Figure 13: Key emerging orphan drug markets - positive and negative attributes
Figure 14: Case study: lysosomal storage disorders in South Africa
Figure 15: Inborn errors of metabolism - key subtypes and treatments

List of Tables
Table 1: Legal framework of orphan drugs in key emerging markets
Table 2: Exceptional access mechanisms in key emerging markets
Table 3: Rare disease populations in the Middle East
Table 4: Local currency to US dollar exchange rates
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