Global Orphan Drug Market

The treatment of rare diseases requires the use of specialized drugs. Patient populations for rare diseases are most often quite small, consequently the products treating them earn the designation orphan drugs. These drugs must undergo the research, development, and clinical trials that all drugs do, yet the investment into the development is great, while the promise for recouping financial costs is not guaranteed. This study covers the spectrum of orphan diseases and highlights drugs already marketed as well as those in the development pipeline for them. Regional highlights, market opportunities, companies to watch, and legislative timelines are provided. This snapshot captures the global orphan drug landscape as of June 2016.
Published: 11 Nov 2016

Global Orphan Drug Market

High Pricing and Undefined Reimbursement Policies will Limit the Sales of Orphan Drugs

The treatment of rare diseases requires the use of specialized drugs. Patient populations for rare diseases are most often quite small, consequently the products treating them earn the designation orphan drugs. These drugs must undergo the research, development, and clinical trials that all drugs do, yet the investment into the development is great, while the promise for recouping financial costs is not guaranteed. This study covers the spectrum of orphan diseases and highlights drugs already marketed as well as those in the development pipeline for them. Regional highlights, market opportunities, companies to watch, and legislative timelines are provided. This snapshot captures the global orphan drug landscape as of June 2016.

Key Questions this Snapshot will Address

  • What therapeutic areas are currently the major focus for orphan drug development?
  • What key companies should be watched in the orphan drug development area?
  • What is the global timeline for the implementation of orphan drug legislation?
  • What are some of the cost implications for orphan drug therapeutics?
  • What are the challenges to development and commercialization of orphan drugs?
  • What challenges do payers face related to the high cost of orphan drug therapeutics?

Features of this Research

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