Global Orphan Drugs Market: Snapshot

During the 2017-2025 forecast period, the global orphan drugs market is expected to display a healthy growth rate, backed by a number of factors such as high return on investments for the development of orphan drugs, strong research and development initiatives for orphan drugs in developed countries, and increasing number of rare diseases related to genetic mutations that are treated using orphan drugs.

In matters of competition, players in the moderately fragmented global orphan drugs market are focused on strategic acquisitions, and are focused to leverage incentives offered by governments for efforts for R&D of orphan drugs.

A report by Transparency Market Research estimates the global orphan drugs market will expand at a healthy 12.1% CAGR between 2017 and 2025, for the market to be valued at US$318.5 bn by the end of 2025.


orphan drugs market


Increasing Number of Rare Cancer Cases Stokes Growth of Oncology Disease Type Segment

The report studies the global orphan drugs market on the basis of disease type, product type, distribution channel, and geography. The segments of this market depending upon disease type are oncology, gastrointestinal, pulmonary, neurology, hematology, cardiovascular, metabolic disorders, endocrinology, infectious diseases, and others. Of them, in 2016, oncology led the market vis-à-vis revenue, and the segment is expected to hold on to its dominant position until the end of 2025. The strong growth of oncology disease type segment is mainly because of increasing drug approvals for treatment of rare cancers. In the European Union, for instance, the burden of cancer has been growing significantly, with 22% of all diagnosed cancer cases identified as rare cancers. The segment of metabolic disorders is expected to expand at a significant CAGR over the forecast period. The increasing number of urea cycle disorders that form a significant part of metabolic disorders account for the high growth of metabolic disorders segment.

In terms of product type, the report divides the global orphan drugs market into biologic and non-biologic products. Vis-à-vis revenue, biologic products led the market in 2016 and is anticipated to stay dominant over the report’s forecast period. Further, the segment of biologic products is expected to display a significant CAGR over the forecast period mainly because of high efficacy of biologic products for treatment of diseases, fewer side effects, and difficulty to develop generic versions of these drugs.

On the basis of distribution channel, the global orphan drugs market has been segmented into hospital pharmacies, retail pharmacies, specialty pharmacies, and others. Hospital pharmacies led the overall market in 2016 on the basis of revenue, and the trend is anticipated to continue over the forecast period. Moreover, increasing hospitalization for the treatment of rare diseases, ease of availability of products in hospital pharmacies, and increasing referrals from physicians for hospitalization are aiding growth of the hospital distribution channel.

Meticulous Efforts for R&D of Orphan Drugs Makes North America Leader

Geography-wise, the global orphan drugs market is segregated into North America, Europe, and Rest of the World. In 2016, North America held the leading share of the market due to various factors that include a well-established healthcare infrastructure, favorable regulations for the approval of drugs for rare diseases, drug exclusivity status received by orphan drugs, and initiatives by governmental and non-governmental agencies to raise awareness about use of orphan drugs for treatment of rare diseases. Within North America, the U.S. contributes substantially to the orphan drugs market as more than 30 million people are reported to suffer from rare diseases in the country. This calls for strong R&D for development of orphan drugs.

Prominent names in the orphan drugs market include Novartis AG, Celgene Corporation, Pfizer Inc., Alexion Pharmaceuticals Inc., Novo Nordisk A/S, Eisai Co. Ltd., Bayer AG, Merck & Co. Inc., Biogen Inc., Amgen Inc., Bristol-Myers Squibb Company, F. Hoffmann-La Roche Ltd., Sanofi S.A., Eli Lilly and Company, AstraZeneca plc., Daiichi Sankyo Company Limited, GlaxoSmithKline plc., Johnson & Johnson, and Shire plc. among others.

Orphan Drugs Market: Overview

The underlying reasons that are fuelling demand for orphan drugs are the increasing occurrences of rare diseases. During forecast timeframe of 2017 to 2025, the world market for orphan drugs is prognosticated to register a robust rate of growth, and the same is supported by several factors like sound research and development for orphan drugs especially in the developed economies, growing incidences of rare diseases, and high return on investments (ROI) that can be obtained for the invention and development of these drugs.

Considering the vendor landscape, the market for orphan drugs is considered to be reasonably fragmented and the market players emphasize on strategically important acquisitions. The companies are also more focused on taking advantage of the various incentives that are been provided by the governments to encourage research and development work on orphan drugs.

Orphan Drugs Market: Trends and Opportunities

In many developed economies such as the United States, the United Kingdom (the U.K.) intense focus on research and development work on orphan drugs begets fee waivers, government benefits, and tax credits and are as such contributing towards market growth. According to Research Office Legislative Council Secretariat, around 30 million people are suffering from some form of rare diseases or the other in the United States. As such, a total number of 4171 drugs have been given the status of orphan drug in the United States. Such initiatives by the government are encouraging the manufacturers of such drugs to make more investments in the said market.

In addition to that, soaring incidences of many types of unusual illness that are associated with various genetic reasons result in investments for drug development for these uncommon diseases. Such investments are proving to be advantageous for the market for orphan drugs. As per the data of a non-profit organization, Global Genes, more than 7,000 kinds of uncommon diseases have been detected worldwide. Presently, around 300 million individuals across the globe are forecasted to be attacked by some rare diseases and such high figures demand for substantial amount of manufacturing of orphan drugs.

Orphan Drugs Market: Geography

Taking geographical segmentations into consideration, the international market for orphan drugs is divided into Europe, North America, and Rest of the World. In the year 2016, the region of North America accounted for lion’s share of the said market owing to several factors that such as encouraging regulations that facilitate speedy approval of such drugs, exclusivity of the drug status that are meant for such drugs, and prevailing robust medical infrastructure all contribute towards the growth of the market.

The country of United States, North America, makes substantial contribution towards this market since around 30 million American people are reportedly affected with some form rare disease of the other. Such high occurrences of rare diseases demand sound research and development work for the purpose of development of better orphan drugs.

Orphan Drugs Market: Company Profiling

Amgen Inc., Bayer AG, F. Hoffmann-La Roche Ltd., Alexion Pharmaceuticals Inc., Novo Nordisk A/S, Novartis AG, Bristol-Myers Squibb Company, AstraZeneca plc., Daiichi Sankyo Company Limited, GlaxoSmithKline plc. are some of the topnotch players of this market. The leading companies account a major share of the market, making it very difficult for new players to enter the market.

For the purpose of this study, the market has been segmented as follows:

  • Global Orphan Drugs Market, by Disease Type
    • Oncology
    • Gastrointestinal
    • Pulmonary
    • Neurology
    • Hematology
    • Cardio-vascular
    • Metabolic disorders
    • Endocrinology
    • Infectious diseases
    • Others
  • Global Orphan Drugs Market, by Indicator
    • Biologic
    • Non-biologic
  • Global Orphan Drugs Market, by Technology
    • Hospital Pharmacies
    • Specialty Pharmacies
    • Retail pharmacies
    • Others
  • Global Orphan Drugs Market, by Geography
  • North America
    • U.S.
    • Canada
  • Europe
    • U.K.
    • Germany
    • Spain
    • France
    • Italy
    • Rest of Europe
  • Rest of the World
    • Japan
    • Australia
    • China
    • Brazil
    • South Korea
    • Others