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Discover the North American orphan drugs market to reach $48.96 billion by 2021

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North America Orphan Drugs Market by Type (Biological and Non Biological), by Therapeutic (Haematology, Neurology, Oncology, and Others) and by Region - Industry Analysis, Size, Share, Growth, Trends, and Forecasts (2016–2021)

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The North America Orphan Drugs Market was worth $34.08 billion in 2016 and estimated to be growing at a CAGR of 7.51%, to reach $48.96 billion by 2021.

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Orphan drugs are medical products which are utilized for the treatment of ailments or conditions which concern a very small 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 comparatively niche segment because they are utilized for the treatment of rare diseases.

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For an ailment that has low frequency, finding the right equilibrium between cost and profit can be a tightrope walk.

The total number of illnesses considered as orphan stands at about 7,000 currently, with just about 250 new diseases being added to this list yearly.

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The growth of the North America Orphan Drugs market is mainly due to factors such as an increasing healthcare expenditure, favourable government initiatives, growing prevalence of chronic diseases, and small timeline required for orphan drug development. However, factors such as high initial investment, clinical study impediments, and regulatory obstacles are hindering the growth of the market in this region.

The North America Orphan Drugs market is segmented based on Type and Therapeutic. The market for Orphan Drugs, on the basis of Type is segmented into Biological and Non Biological.

Non Biological Orphan Drugs lead the market, on the basis of type with more than 60% of the market share. On the basis of Therapeutic, the Orphan Drugs market is segmented into Haematology, Neurology, Oncology, and Others.

On the basis of Therapeutics, Neurology accounts for almost 40% of the entire market share.

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The North America market has been geographically segmented into US and Canada. Benchmarks for designation of orphan disease differs country to country, in US if the disease is prevalent in fewer than 200,000 people, then it’s termed as a rare disease.

The growth of the market in this region is powered by factors such as prolonged market exclusivity and government enticements for orphan medicine.

Presently, the market for orphan drugs represents only about 6% of the complete pharmaceutical market. An extremely conducive environment currently prevails for the growth of the orphan drugs market, thereby encouraging the entry of more companies into the market.

Market Segmentation                                              


  • Introduction           
  • Biological
  • Non Biological        
  • Y-o-Y Growth Analysis, By Type     


  • Haematology         
  • Neurology
  • Oncology 
  • Others      

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The scope of the report:

  • Regional and country-level analysis and forecasts of the study market; providing Insights on the major countries/regions in which this industry is blooming and to also identify the regions that are still untapped
  • Segment-level analysis in terms of technology, component, and type along with market size forecasts and estimations to detect key areas of industry growth in detail
  • Identification of key drivers, restraints, opportunities, and challenges (DROC) in the market and their impact on shifting market dynamics
  • Study of the effect of exogenous and endogenous factors that affect the global market; which includes broadly demographic, economics, and political, among other macro-environmental factors presented in an extensive PESTLE Analysis
  • Study the micro environment factors that determine the overall profitability of an Industry, using Porter’s five forces analysis for analysing the level of competition and business strategy development
  • A comprehensive list of key market players along with their product portfolio, current strategic interests, key financial information, legal issues, SWOT analysis and analyst overview to study and sustain the market environment
  • Competitive landscape analysis listing out the mergers, acquisitions, collaborations in the field along with new product launches, comparative financial studies and recent developments in the market by the major companies
  • An executive summary, abridging the entire report in such a way that decision-making personnel can rapidly become acquainted with background information, concise analysis and main conclusions
  • Expertly devised analyst overview along with Investment opportunities to provide both individuals and organizations a strong financial foothold in the market

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