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Orphan Drugs Market 2015-2019

Orphan Drugs Market 2015-2019

About orphan drugs

An orphan drug is used to treat rare diseases or disorders. According to WHO, orphan or rare diseases are the pathological conditions that affect 0.65-1 out of 10,000 population worldwide. There are around 6,000 orphan diseases, of which 80% are genetic. Orphan drugs are developed by the pharmaceutical industry to serve the public health need. These are designated as Orphan Designations by the regulatory agencies to accelerate the approval process.

Technavio's analysts forecast the global orphan drugs market to grow at a CAGR of 10.05% over the period 2014-2019.

Covered in this report

The report covers the current scenario and the growth prospects of the global orphan drugs market for the period of 2015-2019. To calculate the market size, the report considers revenue generated from the sales orphan drugs.

Technavio's report, Global Orphan Drugs Market 2015-2019, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the Americas, APAC, and Europe; it also covers the landscape of the global orphan drugs market and its growth prospects in the coming years. The report includes a discussion of the key vendors operating in this market.

Key regions

  • Americas
  • APAC
  • Europe
Key vendors
  • Bristol-Mayer Squibb
  • Celgene
  • F. Hoffmann La Roche
  • Novartis
  • Pfizer
Other prominent vendors
  • AbbVie
  • Actelion Pharmaceuticals
  • Alexion Pharmaceuticals
  • Alnylam Pharmaceuticals
  • Amgen
  • AstraZeneca
  • Baxter International
  • Biogen Idec
  • Celldex Therapeutics
  • Eisai
  • Eli Lilly
  • GlaxoSmithKline
  • Intercept Pharmaceuticals
  • Johnson & Johnson
  • Merck
  • NewLink Genetics
  • Northwest Biotherapeutics
  • Novo Nordisk
  • Onyx Pharmaceuticals
  • Sanofi
  • Shire
  • Synageva BioPharma
  • Takeda
  • Vertex Pharmaceuticals
  • XOMA
Market driver
  • Shorter development timelines
  • For a full, detailed list, view our report
Market challenge
  • High cost of drug development
  • For a full, detailed list, view our report
Market trend
  • Increased focus on biological drugs
  • For a full, detailed list, view our report
Key questions answered in this report
  • What will the market size be in 2019 and what will the growth rate be?
  • What are the key market trends?
  • What is driving this market?
  • What are the challenges to market growth?
  • Who are the key vendors in this market space?
  • What are the market opportunities and threats faced by the key vendors?
  • What are the strengths and weaknesses of the key vendors?


Press Release

Technavio Announces the Publication of its Research Report – Global Orphan Drugs Market 2015-2019

Technavio recognizes the following companies as the key players in the Global Orphan Drugs Market: Bristol-Mayer Squibb, Celgene, F. Hoffmann La Roche, Novartis and Pfizer

Other Prominent Vendors in the market are: AbbVie, Actelion Pharmaceuticals, Alexion Pharmaceuticals, Alnylam Pharmaceuticals, Amgen, AstraZeneca, Baxter International, Biogen Idec, Celldex Therapeutics, GlaxoSmithKline, Eisai, Eli Lilly, Intercept Pharmaceuticals, Johnson & Johnson, Merck, Northwest Biotherapeutics, NewLink Genetics, Novo Nordisk, Onyx Pharmaceuticals, Sanofi, Shire, Takeda, Synageva BioPharma, Vertex Pharmaceuticals, and XOMA.

Commenting on the report, an analyst from Technavio’s team said: “The global orphan drugs market is expected to witness certain emerging trends. Increased focus on biologics is one such trend. Biologics are expected to be a preferred treatment option for the medical fraternity, patients, and the families of patients. This in turn will raise the number of treatment-seeking patients, which results in an increase in the overall consumption of medicines.”

According to the report, one of the main drivers in this market is the shorter development timelines. The clinical trials for orphan indication enrol fewer patients in the studies, which leads to a shorter and faster development process.

Further, the report states that one of the challenges in conducting clinical trials in rare diseases is the smaller number of patients available for clinical studies. Enrolling enough patients in clinical studies to generate significant comparative data is therefore a major challenge.

Companies Mentioned

Bristol-Mayer Squibb, Celgene, F. Hoffmann La Roche, Novartis, Pfizer, AbbVie, Actelion Pharmaceuticals, Alexion Pharmaceuticals, Alnylam Pharmaceuticals, Amgen, AstraZeneca, Baxter International, Biogen Idec, Celldex Therapeutics, GlaxoSmithKline, Eisai, Eli Lilly, Intercept Pharmaceuticals, Johnson & Johnson, Merck, Northwest Biotherapeutics, NewLink Genetics, Novo Nordisk, Onyx Pharmaceuticals, Sanofi, Shire, Takeda, Synageva BioPharma, Vertex Pharmaceuticals, XOMA.

  • Executive summary
    • Highlights
  • Scope of the report
    • Top-vendor offerings
      • Table Product offerings
  • Market research methodology
    • Research methodology
    • Economic indicators
  • Introduction
    • Key market highlights
  • Overview
    • Orphan medicines legislation
      • Table History of orphan medicines legislation
      • Table Incentives provided by US regulation
      • Table Incentives provided by Japanese regulation
      • Table Incentives provided by EU regulation
      • Table Incentives provided by Australian regulation
      • Table Incentives provided by South Korean regulation
      • Table Incentives provided by Singapore regulation
    • Orphan medicines centralized procedure in US/EU
      • Table Orphan drug regulatory process in EU/US
    • Product registration process for orphan drugs in Japan
      • Table Orphan drug registration process in Japan
      • Table Orphan drug development and regulatory challenges
    • Orphan drug designations and approvals
      • Table Orphan drug designations and approvals in US 2000-2014
      • Table Number of novel orphan molecular entities launched in US 2004-2013
      • Table Novel orphan molecular entities launched in US 2013
      • Table Number of orphan drug designations and approvals in Japan 2000-2012
      • Table Number of orphan medicinal products launched in Europe 2002-2014
      • Table Top orphan drug designation indications in EU 2014
      • Table Approved and designated orphan pharmaceutical products in US, EU, Japan, and Australia
      • Table Cumulative orphan designations in US, EU, and Japan per year 2000-2013
      • Table Source: Technavio, 2015Orphan drug designations by therapy areas 2013
    • Annual cost of orphan drugs
      • Table Average annual cost of orphan drugs per patient in US 2010-2014 ($)
      • Table Annual cost of several orphan drugs
      • Table Cost comparison of branded orphan drugs versus generic versions 2013 ($)
      • Table Orphan drug expenditure by therapeutic class 2007-2013
    • R&D costs for orphan and non-orphan drugs
      • Table R&D cost for Phase III trial for orphan and non-orphan drugs 2013 and 2014 ($ billions)
  • Pipeline snapshot
    • Table The worldwide top late-stage and filed orphan drugs are given below:
  • Market landscape
    • Market overview
    • Market size and forecast
      • Table Global orphan drugs market ($ billions)
    • Five forces analysis
      • Table Five forces analysis
  • Market segmentation by product
    • Table Global orphan drugs market by product
    • Table Global orphan drugs market by product 2014
    • Global biologic orphan drugs market
      • Table Global biologic orphan drugs market 2014-2019 ($ billions)
    • Global non-biologic orphan drugs market
      • Table Global non-biologic orphan drugs market 2014-2019 ($ billions)
  • Geographical segmentation
    • Table Global orphan drugs market by geographical segmentation 2014
    • Orphan drugs market in Americas
      • Table Orphan drugs market in Americas 2014-2019 ($ billions)
    • Orphan drugs market in EMEA
      • Table Orphan drugs market in EMEA 2014-2019 ($ billions)
    • Orphan drugs market in APAC
      • Table Orphan drugs market in APAC 2014-2019 ($ billions)
      • Table Global orphan drugs market by geographical segmentation 2014-2019 ($ billions)
  • Market drivers
    • Table Drivers of global orphan drugs market
    • Shorter development timelines
      • Table FDA filed to approved time: orphan versus non-orphan drugs (median time months)
      • Table Phase III trial length orphan versus non-orphan drugs (median length years)
    • Assistance from regulatory bodies
    • Incentives for developing orphan drugs
    • High return on investment
    • Exemption from pricing constraints
  • Impact of drivers
    • Table Impact of drivers
  • Market challenges
    • Table Challenges in global orphan drugs market
    • High cost of drug development
    • Decreased investment in marketing
    • Complications in conducting clinical trials
    • Unknown pathophysiology
    • Adverse policies of FDA
  • Impact of drivers and challenges
    • Table Impact of key drivers and challenges
  • Market trends
    • Table Trends in global orphan drugs market
    • Increased focus on biological drugs
    • Growing awareness of orphan drugs
    • Channel marketing strategy
    • Expansion of business
    • Entry into untapped market
  • Vendor landscape
    • Competitive scenario
    • Major vendors 2014
      • Table BMS: Key takeaways
      • Table Celgene: Global year-over-year growth and revenue of Vidaza 2012-2014 ($ millions)
      • Table Celgene: Global year-over-year growth and revenue of Revlimid 2012-2014 ($ billions)
      • Table Celgene: Key takeaways
      • Table Novartis: Key takeaways
      • Table Pfizer: Year-on-year growth rate and revenue of Inlyta 2012-2014 ($ millions)
      • Table Pfizer: Key takeaways
      • Table Global year-over-year growth and revenue of Rituxan/MabThera 2010-2014 ($ billions)
      • Table Avastin: Global revenue and year-over-year growth 2010-2014 ($ billions)
      • Table Roche: Key takeaways
    • Worldwide orphan drug sales revenue: Top 17 companies 2013
    • Top 10 orphan drugs in US 2014
      • Table Worldwide orphan drug sales 2007-2013 ($ billions)
    • Other prominent vendors
  • Key vendor analysis
    • BMS
      • Table BMS: Business segmentation by revenue 2014
      • Table BMS: Business segmentation by revenue 2013 and 2014 ($ billions)
      • Table BMS: Geographical segmentation by revenue 2014
    • Celgene
      • Table Celgene: Product segmentation by revenue 2014
      • Table Celgene: Product segmentation by revenue 2013 and 2014 ($ billions)
      • Table Celgene: Geographical segmentation by revenue 2014
      • Table Roche: Business segmentation by revenue 2014
      • Table Roche: Business segmentation by revenue 2013 and 2014 ($ billions)
      • Table Roche: Geographical segmentation by revenue 2014
    • Novartis
      • Table Novartis: Business segmentation by revenue 2014
      • Table Novartis: Business segmentation by revenue 2013 and 2014 ($ billions)
      • Table Novartis: Geographical segmentation by revenue 2014
    • Pfizer
      • Table Pfizer: Business segmentation by revenue 2014
      • Table Pfizer: Business segmentation by revenue 2014 (%)
      • Table Pfizer: Geographical segmentation by revenue 2014
  • Appendix
    • List of abbreviations
  • Explore Technavio

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