The global orphan drugs market size was at USD 125,016.1 Million in 2017 and is projected to reach USD 294,037.8 Million by 2025, exhibiting at a CAGR of 11.4% during the forecast period (2018-2025).

According to the European Organization for Rare Diseases, orphan drugs are produced for diagnosis, prevention, and treatment of rare disorders. The oncology therapy area holds higher orphan drugs market share due to emergence of new cancer-related diseases. Additionally, FDA approvals to a series of cancer-related orphan drugs are expected to result into increased commercialization of orphan drugs, especially oncology therapy areas, during the forecast period.

The market is expected to witness higher demand for immunomodulators, as they regularize or amplify the immune system. Rising prevalence of infectious diseases in developed as well as emerging nations and higher prices of medications are projected to fuel demand for immunomodulators, enabling orphan drugs market growth during the forecast period. 

"Higher prevalence of cancer is projected to boost the global market"

Exponentially increasing incidence of cancer is boosting the major market players to develop oncology-related orphan drugs. Moreover, lucrative incentive policies by governments and approvals from FDA and EU regulations are paving way for the entry of new drugs into the orphan drugs Industry. In healthcare space, drugs manufactured for smaller number of patients hold the higher cost as compared to drugs manufactured for larger number of patients, due to which oncology therapy area is projected to accelerate with a considerable pace during the forecast period.

Market Segmentation

Based on the therapy area, the orphan drugs market segments include oncology, haematology, neurology, endocrinology, cardiovascular, respiratory, immunotherapy, and others. Oncology therapy area segment accounted for 49.8% share of market in 2017. In terms of drug type, the market is categorized as biologics and non-biologics. Based on distribution channel, the market segment includes hospital pharmacy, retail pharmacies, online sales, and others.

Regional Analysis

"High per capita expenditure followed by new FDA approvals to boost market in North America"

North America market generated a revenue of USD 67,144.6 Mn in 2017, and is anticipated to emerge dominant in the orphan drugs market share during the forecast period. North America is expected to witness considerable growth on account of rising government spending on research and development of orphan drugs, extensively accelerated FDA approvals for newly developed drugs, and rising emphasis on implementation of orphan drugs act.

Moreover, incentivisation of orphan drug development as well as enforcement of new marketing authorizations for orphan drugs are anticipated to drive the Europe market. The market growth in Asia Pacific region is likely to nurture at a considerable rate owing to higher prevalence of rare disorders followed by priority for execution of orphan drug legislations in Asian countries.

Key Market Players

"Pfizer Inc. & Eli Lily to Account for Considerable Market Share in Terms of Revenue"

The major players operating in the fragmented orphan drugs market are Biogen, Amgen Inc., Actelion Pharmaceuticals Ltd., Eli Lilly and Company, Pfizer Inc., Bayer AG and others. Eli Lily and Pfizer Inc. are expected to hold considerable share in the market. Huge infrastructure in terms of resources and robust distribution network of Pfizer Inc. is anticipated to offer significant share of the global orphan drug market to the company.

List of Key Companies Profiled:

• Amgen Inc.


• Bayer AG


• 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.

Report Coverage

Huge investment in research and innovation for new orphan drug development represents considerable opportunity for pharmaceutical manufacturers to register significant share in the global market. Factors such as lucrative incentive policies by governments and approvals from FDA and EU regulations are propelling the entry of new drugs into the orphan drugs industry.

The report provides qualitative and quantitative insights on orphan drugs industry, potential,  dynamics, trends and detailed analysis of market size and growth rate for all possible segments in the market. The market is segmented as per therapy area, drug type, distribution channel, and region. On the basis of therapy area, the global market is categorized into oncology, hematology, neurology, endocrinology, cardiovascular, respiratory, immunotherapy, and others.

On the basis of drug type, the market is segmented as biologics and non-biologics. Based on the distribution channel, the global orphan drugs Industry is segmented into hospital pharmacy, retail pharmacy, online sales, and others, Geographically, the market is segmented into four major regions, which are North America, Europe, Asia Pacific, and the rest of the world. The regions are further categorized into countries.

Along with this, the research report includes orphan drugs market potential, dynamics and competitive landscape. Additionally, the report also offers insights on the prevalence of major orphan disease in key countries, pipeline review for orphan drugs, technological developments, and healthcare spending by key countries - 2017.

SEGMENTATION

Key Industry Developments:

• In January 2019, Medunik Canada signed an agreement with HRA Pharma. The newly signed agreement between two companies give legal rights to Medunik Canada for marketing and distribution of Lysodren, an orphan drug which is prescribed to treat inoperable adrenal cortical carcinomas.


• In December 2017, Aprecia Pharmaceuticals and Cycle Pharmaceuticals agreed upon strategic partnership to develop and commercialize 3D printed orphan drugs in years to come.