Orphan Drugs Market Trends, Growth, CAGR and Global Industry Forecast to 2027
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 Orphan Drugs Market Research Report: Information by Drug Type (Biologics & Non-Biologics), Sale (Generics & Prescribed), Drug (Revlimid, Rituxan, Opdivo, & Others), Therapy Class (Oncology, Blood, Endocrine, & Others), Region- Forecast till 2027

Orphan Drugs Market Overview

The orphan drugs market is expected to register a CAGR of 10% to reach USD 340.50 Billion by 2027.

Orphan drugs are medical products intended for diagnosis, prevention, or treatment of serious, life-threatening disorders that are rare. These are called orphan drugs because, under normal market conditions, the pharmaceutical industry has little interest in developing and marketing these products intended for only a small number of patients. Orphan drugs are developed to treat patients suffering from the serious rare disease for which no treatment or at least one treatment is available. This disease affects a very small portion of the population. Extended market exclusivity and government incentives for orphan medicines as well as the rising prevalence of rare diseases and increasing healthcare expenditure drive the growth of the market. However, high per patient treatment cost and regulatory restriction on profitability may hamper the growth of the market

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Orphan drugs are drugs that are neglected by the pharmaceutical industry due to their low volume of demand. Orphan drugs are often used to treat rare diseases that don’t affect large chunks of the population. As a result, the overall demand for these drugs remains low. This results in pharmaceutical companies staying away from these drugs or not prioritizing their development, as the commercial returns are not adequate. However, the field of orphan drug development has received strong support from the U.S. government in recent years. Other countries in North America and Western Europe have also enacted policies to support the development of orphan drugs, in order to improve the overall population’s health. This is likely to remain a major driver for the global Orphan Drugs Market over the forecast period, as government support is essential in mobilizing strong development avenues in the orphan drugs market. The increasing healthcare expenditure around the world is thus likely to be a major driver for the global orphan drugs market.


 The increasing prevalence of rare diseases is also likely to be a major driver for the global orphan drugs market over the forecast period. Awareness about rare diseases has grown rapidly over the last few years, leading to increasing diagnosis numbers and increasing research into these diseases. This is also likely to remain a key driver for the global orphan drugs market over the forecast period.

Competitive Analysis:

Leading players in the global orphan drugs market include Bristol Myers Squibb, Pfizer Inc., Vertex Pharmaceuticals Inc., Alexion Pharmaceuticals Inc., Janssen Global Services LLC, Sanofi, Shire Pharmaceutical, Eli Lilly and Company, Merck KGaA, Takeda Pharmaceutical Company Limited, Biogen, Novartis, Celgene Corporation, Mylan, and F. Hoffman-La Roche AG.

In January 2020, the U.S. FDA granted orphan drug status to avatrombopag, an oral thrombopoietin receptor agonist used to treat various kinds of thrombocytopenia.

The same month, UK-based EUSA Pharma and Chinese company BeiGene announced a collaboration for the development of orphan biologic products Sylvant (siltuximab) and Qarziba (dinutuximab beta).


The global orphan drugs market is segmented on the basis of drug type, sale, drug, therapy class, and region.

By drug type, the global orphan drugs market is segmented into biologics and non-biologics. The biologics segment accounted for a dominant share in the market in 2017.

By sale, the global market is segmented into generic and prescribed.

By drug, the global orphan drugs market is segmented into revlimid, rituxan, opdivo, keytruda, imbruvica, soliris, jakaf, pomalyst, darzalex, spinraza, and adcetris.

By therapy class, the global orphan drugs market is segmented into oncology, hematology, central nervous system, endocrine, cardiovascular, and respiratory.

Regional Analysis:

The Americas segment holds a dominant share of close to 40% in the global orphan drugs market and is likely to remain the leading revenue generator over the forecast period due to the growing number of North American companies operating in the market. Government assistance has also been a major driver for the orphan drugs market in North America.

Europe holds the second largest share in the market, followed by Asia Pacific. The Asia Pacific market is expected to exhibit robust growth over the forecast period due to the increasing investment in the healthcare sector in the region.


About Market Research Future:

At Market Research Future (MRFR), we enable our customers to unravel the complexity of various industries through our Cooked Research Report (CRR), Half-Cooked Research Reports (HCRR), Raw Research Reports (3R), Continuous-Feed Research (CFR), and Market Research & Consulting Services.


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