Global Orphan Drug Market
Global Orphan Drug Market
High Pricing and Undefined Reimbursement Policies will Limit the Sales of Orphan Drugs
11-Nov-2016
North America
Description
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.
Table of Contents
Scope and Segmentation
Key Questions this Snapshot will Address
Key Takeaways—Orphan Drug Market
Executive Summary—Market Snapshot
Executive Summary—Market Snapshot (continued)
8 Big Market Themes for Orphan Drugs
A Vibrant Ecosystem
Cancer Therapies Remain the Largest Orphan Drug Segment
Global Overview and Analysis
Regional Highlights
Game-changing Companies
Companies to Watch
New Market Opportunities—Orphan Drugs
Key 2016 Predictions—Orphan Drug Market
Timeline of Orphan Drug Legislation
Disruptive Technologies and Advances in this Field
Overall Environmental Impact Factors
Orphan Drug Payer Environment
A Snapshot of Global Drug Price Control Methods
Key Conclusions
The Last Word—3 Big Predictions
Legal Disclaimer
Orphan Diseases with FDA-approved Therapies
Orphan Diseases with FDA-approved Therapies (continued)
Orphan Diseases with FDA-approved Therapies (continued)
Orphan Diseases with FDA-approved Therapies (continued)
Orphan Diseases with FDA-approved Therapies (continued)
Orphan Diseases with FDA-approved Therapies (continued)
Orphan Diseases with FDA-approved Therapies (continued)
Top Indications by Activity and Disease Overview—Blood and Lymphatic System Diseases
SCD—Orphan Drugs on the Market
SCD—Orphan Drugs in Development
SCD—Orphan Drugs in Development (continued)
SCD—Orphan Drugs in Development (continued)
MDS—Orphan Drugs on the Market
MDS—Orphan Drugs in Development
TTP—Orphan Drugs in Development
Top Indications by Activity and Disease Overview
PAH—Orphan Drugs on the Market
PAH—Orphan Drugs in Development
PAH—Orphan Drugs in Development (continued)
Calciphylaxis—Orphan Drugs in Development and on the Market
Top Indications by Activity and Disease Overview
CF—Orphan Drugs on the Market
CF—Orphan Drugs in Development
CF—Orphan Drugs in Development (continued)
CF—Orphan Drugs in Development (continued)
CF—Orphan Drugs in Development (continued)
IPF—Orphan Drugs on the Market
IPF—Orphan Drugs in Development
IPF—Orphan Drugs in Development (continued)
Top Indications by Activity and Disease Overview
Fragile X Syndrome—Orphan Drugs in Development
HIBM—Orphan Drugs in Development
Top Indications by Activity and Disease Overview
SSc—Orphan Drugs in Development
EB—Orphan Drugs in Development
Top Indications by Activity and Disease Overview
Ped CD—Orphan Drugs on the Market
Ped CD—Orphan Drugs in Development
Ped CD—Orphan Drugs in Development (continued)
Ped UC—Orphan Drugs on the Market
Ped UC—Orphan Drugs in Development
Ped UC—Orphan Drugs in Development (continued)
Pouchitis—Orphan Drugs in Development
Top Indications by Activity and Disease Overview
T1D with Residual Beta-cell Function—Orphan Drugs in Development
T1D with Residual Beta-cell Function—Orphan Drugs in Development (continued)
Top Indications by Activity and Disease Overview
ARS—Orphan Drugs in Development
ARS—Orphan Drugs in Development (continued)
Top Indications by Activity and Disease Overview
RP—Orphan Drugs in Development
RP—Orphan Drugs in Development (continued)
Stargardt Disease—Orphan Drugs in Development
Uveitis—Orphan Drugs on the Market
Uveitis—Orphan Drugs in Development
Top Indications by Activity and Disease Overview
Behcet's Disease—Orphan Drugs in Development
Top Indications by Activity and Disease Overview
CMV Infection—Orphan Drugs on the Market
CMV Infection—Orphan Drugs in Development
Malaria—Orphan Drugs on the Market
Malaria—Orphan Drugs in Development
Leishmaniasis—Orphan Drugs on the Market
Leishmaniasis—Orphan Drugs in Development
Top Indications by Activity and Disease Overview
Top Indications by Activity and Disease Overview (continued)
MPS—Orphan Drugs on the Market
MPS—Orphan Drugs in Development
MPS—Orphan Drugs in Development (continued)
Acromegaly—Orphan Drugs on the Market
Acromegaly—Orphan Drugs in Development
Urea Cycle Disorders—Orphan Drugs on the Market
Urea Cycle Disorders—Orphan Drugs in Development
NPC Disease—Orphan Drugs on the Market
NPC Disease—Orphan Drugs in Development
Pompe Disease—Orphan Drugs on the Market
Pompe Disease—Orphan Drugs in Development
Top Indications by Activity and Disease Overview
DMD—Orphan Drugs in Development
DMD—Orphan Drugs in Development (continued)
DMD—Orphan Drugs in Development (continued)
DMD—Orphan Drugs in Development (continued)
SMA—Orphan Drugs in Development
Top Indications by Activity and Disease Overview
ALS—Orphan Drugs in Development
ALS—Orphan Drugs in Development (continued)
ALS—Orphan Drugs in Development (continued)
ALS—Orphan Drugs in Development (continued)
HD—Orphan Drugs on the Market
HD—Orphan Drugs in Development
FA—Orphan Drugs in Development
Top Indications by Activity and Disease Overview
Pancreatic Cancer—Orphan Drugs on the Market
Pancreatic Cancer—Orphan Drugs in Development
Pancreatic Cancer—Orphan Drugs in Development (continued)
Pancreatic Cancer—Orphan Drugs in Development (continued)
Pancreatic Cancer—Orphan Drugs in Development (continued)
Pancreatic Cancer—Orphan Drugs in Development (continued)
Pancreatic Cancer—Orphan Drugs in Development (continued)
Glioma—Orphan Drugs on the Market
Glioma—Orphan Drugs in Development
Glioma—Orphan Drugs in Development (continued)
Glioma—Orphan Drugs in Development (continued)
Glioma—Orphan Drugs in Development (continued)
Glioma—Orphan Drugs in Development (continued)
AML—Orphan Drugs on the Market
AML—Orphan Drugs in Development
AML—Orphan Drugs in Development (continued)
AML—Orphan Drugs in Development (continued)
AML—Orphan Drugs in Development (continued)
AML—Orphan Drugs in Development (continued)
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Author | Barbara Gilmore |
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