Global Orphan Drugs Market Analysis


  • Type: Therapeutics Area
  • Published : March 2021
  • Region: Worldwide
  • Country: Other
  • Report ID : 107

  • Format: PPT, PDF

Global Orphan Drugs Market Size and Share Analysis, By Therapy Area (Oncology, Haematology, Neurology, Endocrinology, Cardiovascular, Respiratory, Immunotherapy, and Others), By Drug Type (Biologics, and Non-Biologics), By Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Online Sales, and Others), and Regional Forecast, (2019-2028)


Report Overview

The global orphan drugs market size stood at around USD 151 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

According to the European Organization for Rare Diseases, these drugs are produced for diagnosis, prevention, and treatment of rare disorders. The oncology therapy area holds a considerably higher share in the global market due to emergence of new cancer-related diseases. Additionally, FDA approvals to a series of cancer-related orphan drugs are expected to result in increased commercialization of these drugs, especially the oncology therapy area, during the forecast period. The market is will derive growth from higher demand for immunomodulators, as they regularize or amplify the immune system.

Major Orphan Disease

The major disease includes oncology, hematology, neurology, cardiovascular and other disease types. The oncology segment held a dominant orphan drugs market share in 2019. The dominance is due to the presence of several oncology drugs in the product development pipelines of key players, and also the presence of a large number of orphan drugs dedicated for the treatment of cancers. The hematology segment is projected to be the second most dominant segment owing to a number of new product launches and increasing positive regulatory approvals. The neurology segment is also anticipated to register a comparatively strong CAGR due to positive developments in the product offerings for key diseases such as multiple sclerosis. Cardiovascular drugs segments is anticipated to register lower CAGR in the forecast period.

1. Global Orphan Drugs Market Overview……………………………………………………..
A. Market Size
2. Market Growth Drivers and Restraints…………………………………..……………….
A. Market Drivers
I. Favourable government policies
II. Surge in prevalence of rare diseases
B. Market Restraints
I. Limited patient pool for clinical trials and product marketing
II. High treatment costs per patient
3. Orphan Drugs Market by Therapy Area..………………………………………………………………….
A. Oncology
B. Hematology
C. Neurology
D. Cardiovascular
E. Other Disease Types
4. Orphan Drugs Market Segmentation……………..……………………………………….
A. By Drug Type
I. Biologics
II. Non-Biologics
B. By Distribution Channel
I. Hospital Pharmacy
II. Retail Pharmacy
III. Online Sales
IV. Others
C. By Geography
I. North America Market Analysis
II. Europe Market Analysis
III. Asia-Pacific Market Analysis
IV. Middle-East and Africa Market Analysis
V. South America Market Analysis
5. Orphan Major Drugs Market Share……………………………………………………..
A. Market Analysis, Insights and Forecast by Revenue
6. Competitive Landscape………………………………………………………….………..
A. Major Players
B. Products in Pipeline
7. Key Company Profiles…………………………………………………………………….
A. Amgen Company overview, Product & Services, Strategies & Financials
B. Bayer Company overview, Product & Services, Strategies & Financials
C. Alexion Company, Product & Services, Strategies & Financials
8. Healthcare Policies………………………………….…………………………..…………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………………..
A. Key Industry Developments of Orphan drugs market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Drug Type:

The drug type segment is further sub-segmented in biologics and non-biologics. The overwhelming presence of product offerings classified as biologics are some of the major factors responsible for the dominance of the segment in 2019. According to an estimate by the U.S. Food and Drug Administration (U.S. FDA), the Orphan Products Development (OOPD) of the organization have led to the development and marketing of over 600 drugs and biologic products for rare disorders since 1983. The non-biologics segment accounted for a lower share of the global market and also registered a comparatively low CAGR.

By Distribution Channel:

The distribution channel is further sub-segmented in hospital pharmacy, retail pharmacy, online sales and others. Online sales to register a higher CAGR during the forecast period. The hospital pharmacy is expected to hold the highest share in the market during the forecast period. The primary cause of this dominance is because a significantly large number of drugs have to be administered at hospitals intravenously by trained healthcare professionals. The retail pharmacy segment is expected to grow at a comparatively low CAGR during the forecast period. The online pharmacy segment is expected to register a high CAGR especially due to the ease given to the consumers in terms of the purchase of drugs.

By Region:

North America currently dominates the market for orphan drugs and is expected to continue its stronghold for a few more years. In the North American region, the United States holds the largest market share. One of the reasons for market growth is that in the United States, a drug designated orphan drug status receives seven years of marketing exclusivity upon FDA approval for a specific indication, tax credits, and user fee waiver. Since 2013, the new indication approvals have increased greatly. Hence, the United States orphan drugs market is expected to grow during the forecast period.​

Top Major Players

The orphan drugs market is moderately competitive and consists of quite a few major players. In terms of market share, a few of the major players currently dominate the market. With the rising number of diseases and an increasing number of cases each year, a few smaller players are entering the market and holding a substantial share. Currently, Bristol-Myers Squibb Company, and F. Homann-La Roche Ltd. dominate the market, accounting for a significant share of the global market in 2019. This is primarily due to the strong sales and product portfolio of the oncology related drugs of these companies. However, several upcoming key players such as Biogen Inc., and BioMarin Pharmaceutical Inc. with their innovative product pipelines for various orphan diseases are emerging. Other prominent players include Amgen, Alexion, Novartis, AstraZeneca, Daiichi Sankyo Company, Limited and Agios Pharmaceuticals., who also have significant presence in the global market with novel drugs.

Research Methodology

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  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

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