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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 worth USD 208.8 billion by 2027 and USD 143 billion in 2022, growing at a CAGR of 7.86% during the forecast period. 

Orphan drugs are designed with specific conditions to treat rare diseases. The majority of rare diseases come through genetic disorders. The other significant possibilities to get affected by these diseases are bacteria, viruses, and allergies. 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. has received the Compassionate Use Program, an orphan drug status by the FDA. As of now, 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 activities of orphan drugs to treat the diseases related. This ODA act had come up with financial and non-financial considerations to assure availability and access to orphan drugs. Under this act, the government agencies are offering tax credits and subsidies favoring the patient and pharmaceutical companies, which are showing a positive impact on the market.


Increasing incidences of rare diseases is a crucial driving factor for the market to grow eventually.

Over 30 million Americans are recognized with various rare diseases where 200,000 people affect chronic or acute conditions such as cancer, autoimmune disorders, and digestive disorders, among others. In the United States, biopharmaceutical companies focused on introducing innovative vaccines or drugs for rare diseases as there are no particular treatment options for some unique conditions. There are approximately 7000 rare diseases where only five diseases 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 genetically. In 2020, COVID-19 disease became a unique, challenging factor for the researchers to determine effective treatment procedures.

Additionally, the rising demand to increase the production rate of the orphan drugs to make the drugs readily available in the stores is propelling the growth rate of the market. Creating awareness towards the availability of various treatment procedures and prevalence to improve healthcare services in hospitals and clinics with quality drugs is expanding the size of the orphan drugs market. The growing tendency to develop novel drugs, the ever-increasing scale of health care 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 in concern towards people's safety remains 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 growth rate of the market. Also, fluctuations in the availability of raw materials due to the increasing prices of final products is limiting the growth rate of the market. Continuous changes in government bodies' economic strategies in various countries may also negatively impact the demand for the global orphan drugs market.

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 the 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 period. 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 is accelerated the demand of the market. All the market key players have invested hugely in the development of vaccines. It is said that individuals suffering from rare diseases are lack diagnostic and consultancy services by healthcare professionals. An increasing number of people affected by COVID 19 greatly influences the demand for 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 on 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 and analyzed market size & forecast for each segment until 2027.

Based on drug type, the orphan drugs market is segmented into:

  • Biological
  • Non-Biological

The biological orphan drugs segment is leading with the highest shares of the market in recent years. Rising different kinds of rare diseases that can be curable with biological drugs and growing investments in the development of drugs for rare diseases by private companies are leveraging this segment's demand.

The non-biological orphan drugs segment is to have a slight growth rate in the coming years. The rising prevalence of genomic studies and the rise in the number of people suffering from chronic diseases accelerates the segment's growth.

Orphan Drugs Market - By Therapeutic:

  • Hematology
  • Neurology
  • Oncology
  • Others

Oncology is ruling with dominant shares of the market with the increasing number of cancer patients. Escalating expenditure on healthcare is fuelling the demand for this segment. Companies like Johnson & Johnson, Abbvie, and Bristol-Myers Squibb emphasize manufacturing orphan drugs related to the oncology segment. It has put oncology in the leading position in this segment.

The neurology segment is to have the highest CGAR in coming years with the rise in the incidences of neurological disorders across the world. 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. Increasing population is also one of the factors impacting positively on the growth of the market.

Orphan Drugs Market - By Region:

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

Regionally, North America is holding the most significant shares of the market by adopting advanced technologies. Also, the manufacturing of innovative drugs in less time than regular drugs is leveraging the growth rate of the orphan drugs market in this region. The constant economic growth rate is fuelling the size of the market in North America.

U.S accounted for the largest share in the global Orphan Drugs market in 2019, as it spends nearly 20% of its GDP on health. The global market outlook is projected to be positive as more and more governments are adopting policies to encourage this sector, especially in developing countries.

Following North America, Europe is leading the market share with the growing prevalence of novel drugs. Increasing incidences of rare diseases across the region is also one factor leveling up the market's demand.

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 shares for the market growth. The launch of reimbursement schemes in favor of ordinary people is also magnifying the demand of the market. 

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.


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


  • On February 25th, 2019, Roche bought Spark Therapeutics. This deal helped Roche to make their presence more robust in the 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.
  • On January 16th, 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.
  • On April 9th, 2018, AveXis was purchased by Novartis. AveXis is a Biotechnology Company based out of the United States and 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)
  • On December 12th, 2017, Aprecia Pharmaceuticals collaborated with Cycle Pharmaceuticals to introduce 3D printed orphan drugs in foreseen years.
  • On November 30th, 2016, AbbVie, one of the leaders in manufacturing orphan drugs, has received the Orphan Drug Designation by the FDA.  It was for their risankizumab, an investigational treatment for a rare disease called pediatric Crohn's disease.

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