Orphan Drugs Market

Orphan Drugs Market: Global Industry Analysis and Opportunity Assessment 2016-2026

  • Ongoing
  • June 2020
  • REP-GB-1225
  • PDF PPT EXCEL
Orphan Drugs Market
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This Orphan Drugs market study offers a comprehensive analysis of the business models, key strategies, and respective market shares of some of the most prominent players in this landscape. Along with an in-depth commentary on the key influencing factors, market statistics in terms of revenues, segment-wise data, region-wise data, and country-wise data are offered in the full study. This study is one of the most comprehensive documentation that captures all the facets of the evolving Orphan Drugs market.


Orphan Drugs is vaccine, medicine, or in-vivo diagnostic agent that is intended to treat, diagnose, and prevent a rare disease (or) rare medical condition, the condition itself being as an orphan disease. According to U.S. Food and Drug Administration (U.S. FDA) a product that treats a rare disease affecting less than 2, 00,000 Americans. Orphan drugs are intended for the diagnosis, treatment or prevention of a life-threatening, chronic condition. Most of the rare diseases are genetic and thus are presenting throughout the persons entire life,and symptoms may not immediately appear. It is estimated that 5,000 - 8,000 rare diseases existed today, and affecting 6%- 8% of the population in total in European Union, approximately 27 Mn -36 Mn people are suffering from rare diseases in the European Union. The Symptoms of most of the rare diseases may appear at birth or in childhood, for instance, Lysosomal storage disorder,  Cystic fibrosis, infantile spinal muscular atrophy, familial adenomatous polyposis (FAP), patent ductus arteriosus (PDA). However, some of the rare diseases symptoms are appear during adulthood, such as glioma, renal cell carcinoma, and acute myeloid leukemia. Maximum number of rare diseases has identified genetic origins. Some are the result of bacterial infections and viral and due to degenerative causes.

Orphan Drugs Market: Drivers and restraints

Market Growth Analysis

Disclaimer: This data is only a representation. Actual data may vary and will be available in the report.

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Orphan Drugs Market: Segmentation

Global Orphan Drugs Market can be segmented as following types

Orphan Drugs Market: Overview

Orphan Drugs Market: Region-Wise Outlook

Depending upon the geographic regions Orphan Drugs market is segmented into seven key regions: Those are North America, Latin America, and Western Europe, Eastern Europe, and Asia pacific excluding japan, Middle East and Africa (MEA), Japan.

North America dominating the global Orphan Drugs market followed by Europe. Increasing awareness among the people towards the use of novel products and technologies to treat the rare diseases providing lucrative market. Asian Pacific region market is considered to rapidly evolving healthcare infrastructure and research and development, this region is anticipating providing a robust growth of global Orphan Drugs market over the forecast period.

Orphan Drugs Market: Key players

Some of the key players in Market are Hoffmann- La Roche, Celgene Corporation, Alexion Pharmaceuticals, Inc., Novartis AG, Takeda Pharmaceuticals Company Limited, Biogen Idec Limited, and Eli Lilly, Bristol-Myers Squibb, Vertex pharmaceuticals, Inc. Bayer AG, Sanofi, and Johnson & Johnson.

The Research report presents a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, and statistically supported and industry-validated market data. It also contains projections using a suitable set of assumptions and methodologies. The research report provides analysis and information according to categories such as market segments, geographies, types, technology and applications.

The report is a compilation of first-hand information, qualitative and quantitative assessment by industry analysts, inputs from industry experts and industry participants across the value chain. The report provides in-depth analysis of parent market trends, macro-economic indicators and governing factors along with market attractiveness as per segments. The report also maps the qualitative impact of various market factors on market segments and geographies.

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Global Orphan Drugs market can be driven by following drivers, Regulatory bodies are collaboration with the research institutes are promoting the research and development on rare disease. For instance, The European Union seventh frame work program for research and technical development will boost the research into rare diseases. National Organization for rare disease (NORD) enables to provide the scientific and technical information regarding rare diseases. These conducts patient assistance programs and provide information of medication, disease specific. Medical and scientific knowledge of rare disease is lacking. Treatment and medication are highly expensive.

Orphan Drugs Market: Segmentation

Global Orphan Drugs by Products:

  • By Active Ingridients
    • Obinutuzumab
    • Lenalidomide
    • Brentuximab Vedotin
    • Riociguat
    • Ofatumumab
    • Nelarabine
    • Bosutinib
    • Mannitol
    • Carglumic acid
    • Aztreonam
    • Histamine hydrochloride
    • Eliglustat
    • Cabozantinib
    • Ramucirumab
    • Decitabine
    • Defibrotide
  • By Disease/ Disorder
    • Hodgkin lymphoma
    • Paroxysmal nocturnal hemoglobinuria
    • Chronic thromboembolic pulmonary hypertension (CTEPH)
    • Chronic lymphocytic leukemia
    • Chronic Myeloid Leukaemia
    • T-cell acute lymphoblastic leukemia (T-ALL)
    • Philadelphia chromosome positive chronic myelogenous leukaemia (Ph+ CML)
    • Cystic fibrosis (CF)
    • acute myeloid leukaemia
    • Multiple Myeloma
    • Isovaleric acidaemia
    • Gaucher disease type 1 (GD1)
    • Metastatic medullary thyroid carcinoma
    • Advanced gastric cancer or gastro-esophageal junction adenocarcinoma
    • Secondary Acute myeloid leukemia (AML)
    • Severe hepatic venoocclusive disease (VOD)/ sinusoidal obstructive syndrome (SOS)

Increasing the demand for advanced technological treatments and novel drug products to treat various rare diseases are provides a robust market growth.  According Global Genes Organization, Approximately 30 Mn patients in the United States are living with the rare diseases, 10% of U.S population suffering from rare diseases. European region also having 30 Million people are suffering from rare diseases. Globally there are approximately 350 Million people are living with rare diseases Increasing the research advancements and applications of these advancements to treat rare diseases propels Orphan Drugs market over the forecast period.

Orphan Drugs Market: Region-Wise Outlook

The report covers exhaustive analysis on:

  • Market Segments
  • Market Dynamics
  • Market Size
  • Supply & Demand
  • Current Trends/Issues/Challenges
  • Competition & Companies involved
  • Technology
  • Value Chain

Regional analysis includes

  • North America (U.S., Canada)
  • Latin America (Mexico, Brazil)
  • Western Europe (Germany, Italy, U.K, Spain, France, Rest of Western Europe)
  • Eastern Europe (Russia)
  • Asia Pacific (China, India, ASEAN, Australia & New Zealand)
  • Japan
  • Middle East and Africa (GCC, S. Africa)

Report Highlights:

  • Detailed overview of parent market
  • Changing market dynamics in the industry
  • In-depth market segmentation
  • Historical, current and projected market size in terms of volume and value
  • Recent industry trends and developments
  • Competitive landscape
  • Strategies of key players and products offered
  • Potential and niche segments, geographical regions exhibiting promising growth
  • A neutral perspective on market performance
  • Must-have information for market players to sustain and enhance their market footprint.
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  • Extensive database and key dynamics elevating the market with varied verticals associated
  • Underlying Orphan Drugs Market forces that are compelling consumers or businesses to purchase the product
  • How novel technologies and consumer-centric innovations will create new value-adding opportunities for companies active in the Orphan Drugs Market
  • What will be environment and regulatory impacts on the market over the next decade
  • Investment Feasibility Matrix - It shows returns on investment, level of competition, success rate of investment, and potential risks associated with the market. The analysis shows that the competition is high in the established market, while the returns for short-term investment are also taken into consideration
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