Cover Image
市場調查報告書

新興市場孤兒藥的使用與價格

Orphan Drug Access and Pricing in Emerging Markets

出版商 Datamonitor Healthcare 商品編碼 603990
出版日期 內容資訊 英文 59 Pages
商品交期: 最快1-2個工作天內
價格
Back to Top
新興市場孤兒藥的使用與價格 Orphan Drug Access and Pricing in Emerging Markets
出版日期: 2018年01月12日 內容資訊: 英文 59 Pages
簡介

本報告針對新興市場孤兒藥市場調查、提供市場現況與價格動向、政策概要、地區分類市場使用動向、今後預測。

摘要整理

新興地區孤兒藥市場特徵

  • 孤兒藥與新興市場對產業成長的吸引力
  • 孤兒藥商業模式必須適應各地區
  • 相關情報

特殊疾病政策與能力

  • 特殊疾病罹患率的定義是重要步驟
  • 法律不一貫
  • 診斷與治療基本設施的變化
  • 政策無法保證使用
  • 相關情報

價格、補償、市場使用

  • 支付者沒有彈性是市場使用的課題
  • 一部分新興市場以例外方式使用
  • 支付者誤用孤兒藥國際價格
  • 新興市場發展登記制度
  • 相關情報

孤兒藥市場優先程度

  • 海灣國家的機會
  • 中東可獲得資金
  • 異文化促進地區需求
  • 新興市場尚未準備好新技術
  • 製造業者與使用資源不一致
  • 相關情報

附錄

  • 關於作者
  • 調查方法
  • 匯率
目錄
Product Code: DMKC0187268

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.

TABLE OF CONTENTS

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: Datamonitor Healthcare insights and recommendations
  • Figure 2: Key differences between emerging and advanced orphan drug markets
  • Figure 3: Datamonitor Healthcare 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: Datamonitor Healthcare 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: Datamonitor Healthcare 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
Back to Top