Orphan Drugs Market Analysis and Reports | USA Conference Series

Market Analysis - Orphan Drugs 2017

Orphan drugs, also called niche busters, are intended to treat patients suffering from very serious diseases for which no treatment or at least a satisfactory one has so far been available. These diseases, often referred to as orphan diseases or rare diseases, affect only a small portion of the population .The number of diseases for which no treatment is currently available is estimated to be between 4,000 to 5,000, worldwide. Orphan drugs are medicinal products which are used for the treatment of diseases or conditions which affect a very small portion of the population which are known to be rare diseases Like Infectious diseases, Genetic Diseases and Etc.

The Global Orphan Drug Market was around US$97 Billion in 2014, and is anticipated to grow to around US$181.4 Billion by 2020.It is believed that, the market will continue the trend of its incessant growth owing to factors, such as inclining healthcare expenditure, increasing prevalence of chronic diseases, short timeline required for orphan drug development. Moreover, the Market Analysis report includes the revenue of top 10 orphan drugs in 2014. Worldwide, the orphan drug market reached $84.9 billion in 2009. The market is expected to grow at a compound annual growth rate (CAGR) of nearly 6% to reach $112.1 billion by 2014. The U.S. accounted for 51% of the market in 2009 and is expected to grow at a CAGR of 8.9% to reach $65.9 billion by 2014. Orphan drugs for the cancer sector generated the largest amount of revenues, $30.6 billion in 2009, and accounting for 36% of the market.  Revenues for cancer-related orphan drugs are expected to grow at a CAGR of 10% to reach $49.7 billion in 2014..According to the following market analysis report, Global Orphan Drugs Market Outlook 2020, in the wake of the above developments the Global Orphan Drug Industry is expected to increase at a CAGR of around 11% from 2014-2020.

Various drivers and challenges have been listed down that affect the Global Orphan Drug Industry. We have also deduced a list of top five companies which are ruling this industry. These companies include the likes of Novartis, Roche, Celgene, Pfizer and Sanofi.

Orphan Drugs Market: Overview:

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 Million 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

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. The global orphan drugs market is expected to see numerous developments including approval of several Ultra-Rare drugs, increasing scope of Gene therapy, development of drugs for rare blood disease, higher success prospects for Hematology compared to Solid Tumors, attractive pricing option for orphan drug competitors and striking opportunities in developing orphan drugs.

Top 10 Orphan Drugs by 2018:


Name of Drug



Previous Sales (2014) in $

Projected Sales(2018) in $

Rituxan ( Ritumab)


Chronic Lymphocytic Eukemia


$ 7.133  Bn

$ 6.988 Bn




$ 3.767 Bn

$ 6.598 Bn


Paroxysmal  Nacturnal Hemoglobinuria


$ 1.134 Bn

$ 3.404 Bn




$ 797 Million

$ 2.919 Bn


Chronic Myloid Leukemia


$ 998 Million

$ 2.584 Bn


Mutiple Myelome / Mantle Cell Lymphoma


$ 2.358 Bn

$ 2.381 Bn


Multiple Sclerosis

Biogen Idec

$ 2.913 Bn

$ 2.085 Bn

Alimta Pemetrexed

Advanced Nonsquamous Nonsmall Cell Lung cancer




$ 2.594 Bn

$ 2.017 Bn


Yervoy (Ipilimumab)



Bristol-Myers Squibb

$ 706 million

$ 2.005 Bn



Chronic Myeloid Leukemia / Acute Lymphoblastic Leukemia

Bristol-Myers Squibb

$ 1.019 Bn

$1.917 Bn

Major Research Associations related to Rare Diseases and Orphan Drugs

  • Alzheimer's disease Organizations
  • Ann & Robert H. Lurie Children’s Hospital
  • Boston Children’s Hospital
  • European Union Committee of Experts on Rare Diseases
  • EURORDIS Rare Diseases Europe
  • IRDR Intractable & Rare Diseases Research
  • Multiple Myeloma Research Foundation
  • National Alliances for Rare Diseases
  • National Institute of Health (NIH)
  • National Organization for Rare Diseases
  • Organization for Rare Diseases India (ORDI)
  • Orphan Europe
  • Philippine Society for Orphan Disorders
  • Rare Diseases or Syndromes and Clinical Societies
  • Rare Diseases Patient Association Funding
  • Rare Diseases South Africa
  • Rare Disorders Society Singapore
  • RARE Foundation Alliance
  • The Genetic and Rare Disorders Organization
  • The Manton Center for Orphan Disease Research
  • U.S. Food and Drug Administration.

There are more than 6,000 perceived uncommon diseases and it has been assessed that a further 5 are depicted every week.75% of rare diseases influence children.30% of rare disease patients die before 5 years old and, whilst each affects less than 0.1% of the population, the quantity of patients influenced by rare diseases is around 27-36 million in the EU, 25-30 million in the US, and 350 million people are affected around the world, but the true burden of rare diseases is difficult to estimate hard to evaluate subsequent to epidemiological information for most uncommon infections are not accessible. Among the rare diseases recognized and contemplated by EURORIDS it is found that 28.1% autosomal recessive inheritance,26.5% autosomal dominant inheritance, some are sporadic and few are with no etiology. There will be a steady increase in the cumulative number of diseases for which an orphan drug is approved, averaging just over 5 new diseases per year over the next 10 years. The annual per patient cost of global orphan drugs was varies between €1,251 and €407,631, with the median cost being €32,242 per year. The share of the total pharmaceutical market represented by orphan drugs is anticipated to increase from 3.3% in 2010 to a range of 4.6% in 2016 after which it is expected to level off through 2020.