Global Orphan Drugs Market: Overview 

Orphan drug products are medicines for the therapy of life-threating or extremely severe illnesses or disorders which are uncommon for diagnosis, prevention or therapy. The pharmaceutical industry is little interested in the development and marketing of products designed for a tiny amount of patients under ordinary market circumstances. These medicines are called orphans. In 1983, the Orphan Drug Act was enacted to encourage drug companies to create rare disease therapy. The FDA Office for the Development of Orphan Products determines whether a drug is an orphan. 

The incredibly high costs of placing a medicinal product on the market in drug companies would not be recovered from anticipated product revenues. This also leads to a small market potential for fresh drug use and an economic loss for the drug companies sector. 

orphan drugs market

The worldwide orphan medicinal products market is expected to swell during the forecast period to give increasing significance to research and development operations. Over the forecast period 2017 to 2025 the international market for orphan drugs is expected to improve by 12.10 percent at CAGR. By the end of 2025, the worldwide market for orphan drugs should reach a net estimate of 318,5 billion US dollars. 

The global orphan drugs market is segmented by the disease type, by indicator, and by technology for study. Further, the market is considered in terms of different geographical regions in the report. 

Rising Prevalence of Rare Diseases to Strengthen Orphan Drugs Market 

Recurring, chronic and rare illnesses and the elevated demand for significant amounts of orphan drug production are currently forecast to affect around 300 million people worldwide. Rare disease medicines, such as Yellow Fever, von Willebrand Disease, Waardenburg Syndrome, Diabetes Macular Edema, Abetalypoproteinemia, ASD, Yellow Nail Syndrome or Von Hippel Lindau Syndrome and others, are increasing in demand for medicinal products that help increase the market. In addition, FDA free exemptions, public R&D grants, favourable reimbursement policies and premium pricing for business development. 

In 2019, it is likely that the U.S. will see around 1,762,450 new cancer cases, according to the National Center for Health Statistics. The demand for orphan medicines is likely to rise in the coming years as these figures are motivated. As a result, there have been a total of 4,171 drugs in the US as orphan drugs. Such Government initiatives encourage drug manufacturers to invest more in the said industry. 

Although the market for orphan medicines is developing at a decent rate, certain variables can impede its development. The elevated price of orphan medicines, inadequate data on rare illnesses and the absence of competent health experts are factors that reduce development in the worldwide market for orphan medicines. Furthermore, increasing competition from generic sectors can seriously threaten market growth. 

Global Orphan Drugs Market: Regional Outlook 

The global orphan drugs market is segmented for study into North America, Europe, and Rest of the World. Among these, North America is expected to register for high growth in the global market. 

There are several factors contributing to the development of the industry, including promoting laws that enable rapid approval of such drugs, drug status exclusivity for such drugs and a solid medical infrastructure. 

Global Orphan Drugs Market: Companies Mentioned 

Key vendors profiled in our report on the global orphan drugs market include Novartis AG, F. Hoffmann-La Roche Ltd., Bristol-Myers Squibb Company, GlaxoSmithKline plc., Daiichi Sankyo Company Limited, Novo Nordisk A/S, Bayer AG, AstraZeneca plc., and Amgen Inc., among several 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
.