Table of Contents

5 EXECUTIVE SUMMARY
5 Budget limitations are a major hurdle but opportunities still exist for some rare diseases
5 Each market will require customized access strategy to maximize chance of success
5 Lack of diagnosis and treatment infrastructure, coupled with payer rigidity present key access challenges
5 Gulf region presents the most lucrative opportunity, with well-funded niches in other regions

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

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

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

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

57 APPENDIX
57 About the author
57 Methodology
57 Exchange rates

LIST OF FIGURES
7 Figure 1: Datamonitor Healthcare insights and recommendations
9 Figure 2: Key differences between emerging and advanced orphan drug markets
13 Figure 3: Datamonitor Healthcare insights and recommendations
18 Figure 4: Funded gold-standard treatments for selected rare diseases in key emerging markets
21 Figure 5: Case study: Vyndaqel in Brazil
24 Figure 6: Case study: 2017 reimbursement list update in China
28 Figure 7: Datamonitor Healthcare insights and recommendations
33 Figure 8: Market access requirements for orphan drugs in selected emerging markets
35 Figure 9: Case study: Soliris in Mexico
37 Figure 10: Case study: Spinraza and Ofev in Turkey
40 Figure 11: Case study: hemophilia in Colombia
47 Figure 12: Datamonitor Healthcare insights and recommendations
50 Figure 13: Key emerging orphan drug markets – positive and negative attributes
51 Figure 14: Case study: lysosomal storage disorders in South Africa
53 Figure 15: Inborn errors of metabolism – key subtypes and treatments

LIST OF TABLES
15 Table 1: Legal framework of orphan drugs in key emerging markets
30 Table 2: Exceptional access mechanisms in key emerging markets
48 Table 3: Rare disease populations in the Middle East
58 Table 4: Local currency to US dollar exchange rates