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North America Orphan Drugs Market Research Report – Segmented By Type, Therapeutic, Country (United States, Canada and Rest of North America) - Industry Analysis on Size, Share, Trends, COVID-19 Impact & Growth Forecast (2022 to 2027)

Published: January, 2022
ID: 1594
Pages: 145

North America Orphan Drugs Market Size (2022 to 2027)

The size of the North American Orphan Drugs Market is estimated to be worth USD 61.23 billion by 2027. The market was valued at USD 42.63 billion in 2022 and is growing at a CAGR of 7.51% during the forecast period.

Orphan drugs are medical products utilized to treat ailments or conditions that concern a tiny percentage of the population. Diseases that have low occurrence rates are no less important than ones that have a higher frequency.

Orphan drugs are a comparative niche segment because they are utilized for the treatment of rare diseases. Finding the right equilibrium between cost and profit can be a tightrope walk for an ailment with a low frequency. The number of illnesses considered orphans stands at about 7,000 currently, with just about 250 new diseases being added to this list yearly.

Factors such as an increasing the number of health disorders among people due to the adoption of sedentary lifestyles is a major factor driving the demand of the market. Rare diseases are increasing day by day where the manufacturers have to focus on the development of new drugs or vaccines, which is straight accelerating the market. Changes in the schemes and policies by the government favoring the end-users are lavishing the market's growth. Increasing investments in the healthcare centers and pharmaceutical industries to ensure patients' safety is expanding the size of the market.

However, the dearth of skilled people in manufacturing drugs with the latest formula is slowly hindering the market's growth. Also, fluctuations in the prices of final products due to decreased availability of raw materials have remained a challenging factor for the market developers. In addition, stringent rules and regulations by the government in approving new products are inhibiting the market's demand. Moreover, increasing side effects with long-term use of these drugs, especially in cancer patients, are limiting the market's growth rate.

This research report on the North American orphan drugs market is segmented and sub-segmented into the following categories:

By Type: 

  • Biological
  • Non-biological

By Therapeutic: 

  • Hematology
  • Neurology
  • Oncology
  • Others

By Country: 

  • The United States
  • Canada
  • Rest of North America

Regional Analysis:

Regionally, the United States orphan drugs market is predicted to lead the North American orphan drugs market followed by Canada during the forecast period. Although, benchmarks for the designation of orphan disease differ from country to country. In the United States, if the disease is prevalent in fewer than 200,000 people, then it’s termed a rare disease. The market's growth in this region is powered by factors such as prolonged market exclusivity and government enticements for orphan medicine.

The U.S. has the largest market share of orphan drugs. It dominates the development of orphan drugs with more than 300 clinical trials. There is a lot of research and development going on in the field of orphan drugs. There are around 200000 patients in the United States who need orphan drugs for rare diseases. The US FDA approved 80 new drugs in 2017. The orphan drug act in 1983 has encouraged the pharmaceutical company to invest in such a small market. In the US, orphan drugs cover pharmaceutical, biological, medical devices, and dietary products. The office of orphan product development (OOPD) with the US Food and Drug Administration promotes the availability of a safe and efficient product for the treatment of rare diseases. The government gives tax credits for clinical research of orphan drugs.

The Canadian orphan drugs market is estimated to showcase a healthy CAGR from 2022 to 2027. There is no regulatory framework for orphan drugs in Canada. The rare diseases are collectively estimated to affect 1 in 12 Canadians. The Canadian government is collaborating with US government organizations for research on new drugs. An increasing number of COVID-19 cases is leveling up the growth rate of the market. 


The major companies dominating the market in this region are Novartis, GlaxoSmithKline, Roche, Alexion, Sanofi, Bristol Myers Squibb, Pfizer, Vertex, Celgene, and Merck.


                1.1 Market Definition                    

                1.2 Study Deliverables                  

                1.3 Base Currency, Base Year and Forecast Periods                           

                1.4 General Study Assumptions                

2. Research Methodology                                           

                2.1 Introduction                              

                2.2 Research Phases                      

                                2.2.1 Secondary Research            

                                2.2.2 Primary Research 

                                2.2.3 Econometric Modelling     

                                2.2.4 Expert Validation  

                2.3 Analysis Design                         

                2.4 Study Timeline                          

3. Overview                                      

                3.1 Executive Summary                

                3.2 Key Inferences                         

                3.3 Epidemology                             

4. Drivers, Restraints, Opportunities, and Challenges Analysis (DROC)                                      

                4.1 Market Drivers                          

                4.2 Market Restraints                    

                4.3 Key Challenges                         

                4.4 Current Opportunities in the Market               

5. Market Segmentation                                              

                5.1 Type                              

                                5.1.1 Introduction           

                                5.1.2 Biological

                                5.1.3 Non Biological        

                                5.1.4  Y-o-Y Growth Analysis, By Type     

                                5.1.5  Market Attractiveness Analysis, By Type   

                                5.1.6  Market Share Analysis, By Type    

                5.2 Therapeutic                                

                                5.2.1 Haematology         

                                5.2.2 Neurology

                                5.2.3 Oncology 

                                5.2.4 Others      

                                5.2.5  Y-o-Y Growth Analysis, By Therapeutic       

                                5.2.6  Market Attractiveness Analysis, By Therapeutic     

                                5.2.7  Market Share Analysis, By Therapeutic      

6. Geographical Analysis                                              

                6.1 Introduction                              

                                6.1.1 Regional Trends    

                                6.1.2 Impact Analysis     

                                6.1.3 Y-o-Y Growth Analysis        

                                       By Geographical Area

                                       By Type

                                       By Therapeutic

                                6.1.4  Market Attractiveness Analysis     

                                       By Geographical Area

                                       By Type

                                       By Therapeutic

                                6.1.5  Market Share Analysis      

                                       By Geographical Area

                                       By Type

                                       By Therapeutic

                6.2 United States                            

                6.3 Canada                         

7.Strategic Analysis                                        

                7.1 PESTLE analysis                         

                                7.1.1 Political     

                                7.1.2 Economic 

                                7.1.3 Social         

                                7.1.4 Technological         

                                7.1.5 Legal          

                                7.1.6 Environmental      

                7.2 Porter’s Five analysis                              

                                7.2.1 Bargaining Power of Suppliers        

                                7.2.2 Bargaining Power of Consumers    

                                7.2.3 Threat of New Entrants     

                                7.2.4 Threat of Substitute Products and Services

                                7.2.5 Competitive Rivalry within the Industry      

8.Market Leaders' Analysis                                          

                8.1 Novartis                       

                                8.1.1 Overview

                                8.1.2 Product Analysis   

                                8.1.3 Financial analysis  

                                8.1.4 Recent Developments       

                                8.1.5 SWOT analysis       

                                8.1.6 Analyst View          

                8.2 GlaxoSmithKline                      

                8.3 Roche                           

                8.4 Alexion                        

                8.5 Sanofi                           

                8.6 Bristol Myers Squibb                              

                8.7 Pfizer                            

                8.8 Vertex                          

                8.9 Celgene                       

                8.10 Merck                        

9.Competitive Landscape                                            

                9.1 Market share analysis                            

                9.2 Merger and Acquisition Analysis                        

                9.3 Agreements, collaborations and Joint Ventures                          

                9.4 New Product Launches                          

10.Market Outlook and Investment Opportunities                                           


                a) List of Tables                

                b) List of Figures                              

  1. North America Orphan Drugs Market By Type, From 2022 - 2027 (USD Million)
  2. North America Biological Market By Region, From 2022 - 2027 (USD Million)
  3. North America Non Biological Market By Region, From 2022 - 2027 (USD Million)
  4. North America Orphan Drugs Market By Therapeutic, From 2022 - 2027 (USD Million)
  5. North America Haematology Market By Region, From 2022 - 2027 (USD Million)
  6. North America Neurology Market By Region, From 2022 - 2027 (USD Million)
  7. North America Oncology Market By Region, From 2022 - 2027 (USD Million)
  8. North America Other therapeutics Market By Region, From 2022 - 2027 (USD Million)
  9. United States Orphan Drugs Market By Type, From 2022 - 2027 (USD Million)
  10. United States Orphan Drugs Market By Therapeutic, From 2022 - 2027 (USD Million)
  11. Canada Orphan Drugs Market By Type, From 2022 - 2027 (USD Million)
  12. Canada Orphan Drugs Market By Therapeutic, From 2022 - 2027 (USD Million)

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