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Global Orphan Drugs Market Size, Share, Trends, COVID-19 impact, Growth Analysis Report – Segmented By Type (Biological Orphan Drugs & Non-Biological Orphan Drugs), Therapeutic Type and Region - Industry Forecast (2022 to 2027)

Published: January, 2022
ID: 1593
Pages: 175
Formats: report pdf report excel report power bi report ppt

Global Orphan Drugs Market Size (2022 to 2027)

As per our report, the size of the global orphan drugs market is estimated to be worth USD 208.8 billion by 2027 and USD 143 billion in 2022, growing at a CAGR of 7.86% during the forecast period. 

Global Orphan Drugs Market Share Insights:

  • Based on drugs, the biological segment dominated the orphan drugs market in 2021.
  • Based on the therapeutic area, the oncology segment led the market in 2021.
  • North America was the largest regional segment in the worldwide market in 2021.
  • The European region is expected to be valued at USD 55.65 billion by 2027.
  • The Asia-pacific region is anticipated to grow at a CAGR of 8.92% from 2022 to 2027.

Orphan drugs are designed with specific conditions to treat rare diseases. Most rare diseases come from genetic disorders. The other significant possibilities of getting affected by these diseases are bacteria, viruses, and allergies. For example, Coronavirus severely damages the world health system. This virus's pandemic situation caused by this virus has helped the pharmaceutical industries focus more on manufacturing drugs to fight against this virus. For instance, on March 25th, Remdesivir, an antiviral drug introduced by Gilead Sciences, Inc., received the Compassionate Use Program, an orphan drug status by the FDA. Currently, it is the top-ranked drug in the market to battle against Coronavirus.

According to an FDA report, it has approved more than 600 drugs since the Orphan Drug Act in 1983. Among those, 14% of orphan drugs are accepted with additional non-orphan indications. The ODA act has gained acceptance from many countries and boosted the manufacturing of orphan drugs to treat related diseases. This ODA act had come up with financial and non-financial considerations to assure availability and access to orphan drugs. Under this act, government agencies are offering tax credits and subsidies favoring the patient and pharmaceutical companies, which are positively impacting the market.


Increasing incidences of rare diseases are propelling the orphan drugs market's growth.

Over 30 million Americans are recognized with rare diseases, whereas 200,000 people have chronic or acute conditions such as cancer, autoimmune disorders, and digestive disorders, among others. In the United States, biopharmaceutical companies focus on introducing innovative vaccines or drugs for rare diseases as there are no treatment options for some unique conditions. There are approximately 7000 rare diseases, and only five have specific treatments. Advancements in genomic studies will help researchers to develop promising medicines. Studies found that 80% of the origin of the rare disease is genetic. In 2020, COVID-19 disease became a unique, challenging factor for researchers to determine effective treatment procedures.

The rising demand to increase the production rate of orphan drugs to make the drugs readily available in the stores drives the orphan drugs market.

Creating awareness of the availability of various treatment procedures and prevalence to improve healthcare services in hospitals and clinics with quality drugs is expanding the growth of the orphan drugs market. The growing tendency to develop novel drugs, the ever-increasing scale of healthcare centers and pharmaceutical companies, and the rise in disposable income in developed and developing countries further accelerate the market for orphan drugs.


Stringent rules and regulations by the government in approving new developments concerning people's safety remain a significant challenging factor for the orphan drugs market players. Furthermore, the lack of complete knowledge of the manufacturing process with the latest formula is slowly hindering the market's growth rate. Also, fluctuations in the availability of raw materials due to the increasing prices of final products are limiting the market's growth rate. Continuous changes in government bodies' economic strategies in various countries may also negatively impact the global orphan drugs market growth.

COVID-19 impact on the orphan drugs market:

According to the European organization for rare diseases, orphan drugs are produced to prevent and treat rare diseases. Many countries are in the fight against COVID-19. The pandemic created a negative impact on all industries except pharmaceuticals. The orphan drugs market has seen the fastest growth rate during the pandemic. The rise in the number of research and development activities by the market key players to find a vaccine or drug for the novel coronavirus disease has accelerated the market's growth. All the market key players have invested hugely in the development of vaccines. It is said that individuals suffering from rare diseases lack diagnostic and consultancy services by healthcare professionals. Therefore, an increasing number of people affected by COVID-19 greatly favors the growth of the orphan drugs market.




Market Size Available

2021 to 2027

Base Year


Forecast Period

2022 to 2027

Segments Covered

By Type, Therapeutic, and Region

Various Analyses Covered

Global, Regional & Country Level Analysis, Segment-Level Analysis, Drivers, Restraints, Opportunities, Challenges; PESTLE Analysis; Porter's Five Forces Analysis, Competitive Landscape, Analyst Overview of Investment Opportunities

Regions Covered

North America, Europe, Asia Pacific, Latin America, Middle East & Africa


This research report on the global orphan drugs market has been segmented & sub-segmented into the following categories.

Orphan Drugs Market – By Drug Type:

  • Biological
  • Non-Biological

Based on drug type, the biological orphan drugs segment had the largest share of the global orphan drugs market in 2021 and is predicted to continue dominating the segment during the forecast period. The rising incidence of various rare diseases curable with biological drugs and growing investments in developing drugs for rare diseases by private companies are leveraging this segment's growth.

The non-biological orphan drugs segment is predicted to showcase a healthy CAGR in the coming years. The rising prevalence of genomic studies and the rise in the number of people suffering from chronic diseases accelerate the segment's growth.

Orphan Drugs Market - By Therapeutic:

  • Hematology
  • Neurology
  • Oncology
  • Others

Based on therapeutic areas, the oncology segment held the major share of the global orphan drugs market in 2021 and is predicted to grow at a healthy pace during the forecast period owing to the number of people who have cancer worldwide. In addition, escalating healthcare expenditure is favoring segmental growth. As a result, companies such as Johnson & Johnson, Abbvie, and Bristol-Myers Squibb emphasize manufacturing orphan drugs related to the oncology segment. As a result, it has put oncology in the leading position in this segment.

The neurology segment will have the highest CGAR in the coming years, with the rise in neurological disorders worldwide. Also, growing support from the Food and Drug Administration is to boost the orphan drugs market's growth rate. The hematology category is to have significant shares. In addition, the increasing population is positively impacting the market's growth.

Orphan Drugs Market - By Region:

  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East and Africa

Regionally, the orphan drugs market in North America accounted for the most significant share of the global orphan drugs market in 2021. The region’s dominance is predicted to continue throughout the forecast period owing to the growing adoption of technological advancements. In addition, manufacturing innovative drugs in less time than regular drugs is leveraging the growth rate of the orphan drugs market in this region. Furthermore, the constant economic growth rate is fuelling the market size in North America. U.S accounted for the largest share of the global orphan drugs market in 2021, as it spends nearly 20% of its GDP on health. The global market outlook is projected to be positive as more governments adopt policies to encourage this sector, especially in developing countries.

The European orphan drugs market is anticipated to hold a substantial share of the global market during the forecast period and register a healthy CAGR during the forecast period owing to the growing prevalence of novel drugs. In addition, increasing incidences of rare diseases across the European region is another factor favoring the regional market growth.

The orphan drugs market in the Asia Pacific to hit the highest CAGR in the coming years due to the rising population is enhancing the orphan drugs market shares. India and China are significant countries contributing the largest share of the market growth. The launch of reimbursement schemes in favor of ordinary people also magnifies the market's growth.  Presently, the market for orphan drugs represents only about 6% of the entire pharmaceutical market. An extremely conducive environment currently prevails for the growth of the orphan drugs market, thereby encouraging more companies' entry into the market.

The Latin American orphan drugs market held a considerable share of the global market in 2021.

The orphan drugs market in MEA is expected to grow at a moderate CAGR in the coming years.


Johnson and Johnson, Novartis, Celgene, and AbbVie are the global market leaders for orphan drugs. In addition, companies like GlaxoSmithKline, Roche, Alexion, Sanofi, Bristol Myers Squibb, Pfizer, Vertex, and Merck are also playing a promising role in the orphan drugs market.


  • In February 2019, Roche bought Spark Therapeutics. This deal helped Roche to make its presence more robust in advanced therapies. On the other end, Spark Therapeutics was named the first company in the United States to receive gene therapy approval for their Luxturna. Luxturna is a therapy to treat rare inherited eye diseases.
  • In January 2019, an agreement between Medunik Canada and HRA pharma had legal rights for marketing and distributing an orphan drug named Lysodren, which helps treat inoperable adrenal cortical carcinomas.
  • In April 2018, AveXis was purchased by Novartis. AveXis is a Biotechnology Company based out of the United States that develops therapies for rare disorders related to neurological genetics. AVXS-101 is the lead candidate of AveXis, is a gene-replacement therapy to treat a rare disease called SMA (spinal muscular atrophy)
  • In December 2017, Aprecia Pharmaceuticals collaborated with Cycle Pharmaceuticals to introduce 3D-printed orphan drugs in foreseen years.
  • In November 2016, AbbVie, one of the leaders in manufacturing orphan drugs, received the Orphan Drug Designation from the FDA.  It was for their risankizumab, an investigational treatment for a rare disease called pediatric Crohn's disease.

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How big is the orphan drugs market?

The global orphan drugs market size was valued at USD 132.6 billion in 2021 and is estimated to grow to USD 208.8 billion by 2027.

Which region is growing the fastest in the global orphan drugs market?

The Asia-pacific region is expected to grow the fastest in the global market during the forecast period.

Does this report include the impact of COVID-19 on the orphan drugs market?

Yes, we have studied and included the COVID-19 impact on the global orphan drugs market in this report.

Which are the major players operating in the orphan drugs market?

Johnson and Johnson, Novartis, Celgene, AbbVie, GlaxoSmithKline, Roche, Alexion, Sanofi, Bristol Myers Squibb, Pfizer, Vertex, and Merck are some of the major companies operating in the global orphan drugs market.

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