USD
GBP 
CNY 
EUR 
INR 
JPY 
MYR 
ZAR 
KRW 
THB 
High Pricing and Undefined Reimbursement Policies will Limit the Sales of Orphan Drugs
11-Nov-2016
North America
Market Research
NF58-01-00-00-00
LS00270-NA-MR_19375
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.
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)
The Frost & Sullivan Story
Value Proposition: Future of Your Company & Career
Global Perspective
Industry Convergence
360º Research Perspective
Implementation Excellence
Our Blue Ocean Strategy
Speak directly with our analytics experts for tailored recommendations.
Recent related Life Sciences research
13 Jan 2025 | Global | Market Outlook
Top 6 Growth Opportunities for the Digital Health Industry, 2025
Driven by digital advancements, the healthcare industry is transforming rapidly. Current trends and challenges create constant change, and industry stakeholders must adapt to remain attractive, competitive, and unique as robust organizations. Each year, competitors must gain a fresh, comprehensive v...
17 Dec 2024 | Global | Market Research
Cybersecurity in Healthcare, United States, 2024
Healthcare is the industry most affected by cyberattacks, which have targets like hospitals, healthcare organizations, patient databases, and connected devices. These attacks not only destabilize healthcare systems but also threaten care delivery and patient safety. The global healthcare infrastruct...
12 Dec 2024 | Global | Market Outlook
Top 6 Growth Opportunities for Clinical Diagnostics, Global, 2025
Accounting for 2%-3% of total healthcare expenditure and influencing 70% of clinical decision-making, diagnostics is the silent champion supporting all stages of precision health, including risk stratification, screening, early diagnosis, prognosis, monitoring, and relapse. The discovery and trans...
05 Dec 2024 | Global | Frost Radar
Frost Radar™: Enterprise Conversational AI in Healthcare, 2024
This Frost Radar™ benchmarks the companies and their solutions that allow healthcare payers and providers to create next-generation virtual assistants. Conversational AI is primarily used to streamline patient and provider processes and interactions across the care continuum—the pre-care, at-care, a...
27 Nov 2024 | Global | Market Research
Generative AI in Payer and Provider IT Transformation, Global, 2024-2029
The healthcare payer and provider industry is gradually adopting generative artificial intelligence (Gen AI) solutions across its value chain. Almost all organizations have shown an intent to implement Gen AI-based solutions in their products or processes, with a few already embarking on this journe...
Purchase includes:
- Report download
- Growth Dialog™ with our experts
Growth Dialog™
A tailored session with you where we identify the:- Strategic Imperatives
- Growth Opportunities
- Best Practices
- Companies to Action
Impacting your company's future growth potential.
| Deliverable Type | Market Research |
|---|---|
| No Index | No |
| Podcast | No |
| Author | Barbara Gilmore |
| WIP Number | NF58-01-00-00-00 |
| Is Prebook | No |
Global Orphan Drug Market
$6,000.00
Special Price $4,500.00 save 25 %