Global Orphan Drug Market

Global Orphan Drug Market

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

RELEASE DATE
11-Nov-2016
REGION
North America
Research Code: NF58-01-00-00-00
SKU: LS00270-NA-MR_19375

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LS00270-NA-MR_19375

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$4,500.00 save 25 %

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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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Related Research
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.
More Information
No Index No
Podcast No
Author Barbara Gilmore
WIP Number NF58-01-00-00-00
Is Prebook No