The global orphan drugs market was worth US$ 234.12 Bn in 2021, according to a new report published by Absolute Markets Insights. The market is expected to expand at a CAGR of 10.7% from 2022 to 2030. Orphan drugs are medications that have been designed expressly to treat a rare medical condition referred to as an orphan disease. It might be defined as medications that fulfil public health requirements but are not created by the pharmaceutical sector because of financial restrictions. A doctor might only see one case of an orphan disease per year or less because they are frequently so uncommon. By creating orphan pharmaceuticals, academic physician-scientists have attempted to fill this therapeutic gap. However, there are a lot of obstacles to overcome, such as professional barriers, a lack of finance, and the need for several specialties. The creation of the National Organization for Rare Diseases, the Orphan Drug Act, the creation of a grant programme to finance the development of orphan drugs, the establishment of the National Institutes of Health Office of Rare Diseases, and the adoption of orphan drug legislations by other nations are all positive developments which are being recorded over the last few years. Although there have been improvements, there are still a tremendous number of orphan diseases and 300 orphan medications and technologies that have been authorized in the last 25 years.

Get PDF sample report with related graphs & charts (Pre & post COVID-19 impact analysis):

Different regions and countries have defined orphan drugs differently. This might appear to be a small figure at first, but there could be up to 30 million people living in the European Union who are affected by uncommon diseases. On the other hand, in Japan for a drug to be considered for orphan drug designation in Japan, it must satisfy the three requirements listed below. Japan defines an illness as uncommon if there are fewer than 50,000 prevalent cases (0.4%) of it. The proposed drug is either clinically superior to medications already on the Japanese market or it addresses a disease or condition for which there are no other treatments accessible in Japan. The applicant should have a well-defined product development strategy and scientific evidence to back up the drug’s requirement in Japan. A new application development (NDA) can be submitted following the conclusion of clinical studies.

The COVID-19 pandemic has supported the growth of global orphan drugs market. In March 2022, 6 million individuals would have died as a result of the COVID-19 pandemic, which has been determined to have affected more than 460 million people worldwide. For instance, the company Gilead had begun its process to streamline COVID-19 patients to receive its experimental antiviral remdesivir on a wider basis. Moreover, the company is looking forward to obtaining an orphan drug designation for the potential of COVID-19 treatment.  Despite various challenges the average number of marketing-authorization applications to the EMA for orphan medicinal products during COVID-19 (2020 – 2021) were higher as compared to before the pandemic.

Speak to our analyst in case of queries before buying this report:

Global Orphan Drugs Market Report Highlights

  • Orphan drugs for the oncology sector has generated the largest amount of revenue. According to the most current study, since the program’s start, oncology treatments have obtained much more orphan drug designations than treatments for other diseases. Only central nervous system (CNS) illnesses have achieved the need of at least 100 annual orphan drug designations in a specific therapy area, except from oncology. Oncology leads the orphan drugs market, and cancer medicines have consistently received more orphan drug designations than therapies in other therapy areas, according to trends seen in the first 20 years following the passage of the Act.
  • Clinical trial funds for orphan products are a tried-and-true way to successfully cultivate and encourage the creation of novel, safe, and efficient medical solutions for uncommon illnesses/conditions. Leading players in the global orphan drugs market are heavily investing into clinical trials. Moreover, clinical trials for orphan drugs are now being accelerated through the development of patient registries. Patient registries are databases that save medical data and allow for real-time post-marketing surveillance, including monitoring of off-label drug usage, as well as patient recruitment in clinical studies. Thus, with the above stated factors the global orphan drugs market is expected to flourish in the upcoming years.
  • Asia Pacific region is anticipated to be the fastest growing region in the orphan drugs market during the forecast period 2022-2030. In the last few years with the rising prelevance of rare diseases countries like Japan, South Korea, and Taiwan have established systematic economic and regulatory incentives to encourage the development of drugs for rare diseases. A conditional approval for Orphan drugs that have already received approval abroad is now being offered by China’s FDA, despite in the absence of data from local trials. China is also actively promoting the regulation of rare diseases and orphan drugs. The general standards for accelerating the registration and licensing of medications for the treatment of rare diseases are now being set under these new Asian frameworks which is paving the path for the growth of the global orphan drugs market.

View our exclusive press releases on Industry Global News24

  1. Global Skin Microbiome Market 2022 – 2030
  2. Global Black Masterbatches Market 2022 – 2030

List of Key Players of Global Orphan Drugs Market

  • Alexion Pharmaceuticals, Inc.
  • Axcan Pharma International
  • Bristol-Myers Squibb
  • Cephalon, Inc.
  • Eli Lilly and Company.
  • F. Hoffmann-La Roche Ltd
  • Lipomed AG
  • Merck KGaA
  • Novartis AG
  • Pfizer Inc.
  • Pierre Fabre Group
  • Recordati Rare Diseases
  • Sanofi (Genzyme Ltd.)
  • Takeda Pharmaceutical
  • Other Industry Participants

Purchase the latest in-depth Orphan Drugs Market Report @

Global Orphan Drugs Market Segmentation

Absolute Markets Insights has segmented the global orphan drugs market on the basis of type, therapy area, clinical phase, end user, and region further into countries.

Global Orphan Drugs Market Type Outlook (Revenue, USD Million, 2015 – 2030)

  • Biologic
  • Non-biologic

Global Orphan Drugs Market Therapy Area Outlook (Revenue, USD Million, 2015 – 2030)

  • Oncology
  • Neurology
  • Cardiology
  • Hematology
  • Respiratory
  • Immunology
  • Endocrinology
  • Ophthalmology
  • Infectious Disease
  • Others

Global Orphan Drugs Market Clinical Phase Outlook (Revenue, USD Million, 2015 – 2030)

  • Pre-clinical
  • Phase 1
  • Phase 2
  • Phase 3
  • Approval

Global Orphan Drugs Market End-User Outlook (Revenue, USD Million, 2015 – 2030)

  • Hospitals and clinics
  • Specialty clinics
  • Research centers
  • Others

Request for customization to meet your precise research requirements:

Global Orphan Drugs Market Regional Outlook (Revenue, USD Million, 2015 – 2030)

  • North America (U.S., Canada, Mexico, Rest of North America)
  • Europe (France, The UK, Spain, Germany, Italy, Nordic Countries (Denmark, Finland, Iceland, Sweden, Norway), Benelux Union (Belgium, The Netherlands, Luxembourg), Rest of Europe
  • Asia Pacific (China, Japan, India, New Zealand, Australia, South Korea, Southeast Asia (Indonesia, Thailand, Malaysia, Singapore, Rest of Southeast Asia), Rest of Asia Pacific
  • Middle East & Africa (Saudi Arabia, UAE, Egypt, Kuwait, South Africa, Rest of Middle East & Africa)
  • Latin America (Brazil, Argentina, Rest of Latin America)

Top Reports:

Global Serological Transplant Diagnostics Market –

Global Non-Alcoholic Steatohepatitis Diagnostics Market –

About Us:

Absolute Markets Insights assists in providing accurate and latest trends related to consumer demand, consumer behavior, sales, and growth opportunities, for the better understanding of the market, thus helping in product designing, featuring, and demanding forecasts. Our experts provide you the end-products that can provide transparency, actionable data, cross-channel deployment program, performance, accurate testing capabilities and the ability to promote ongoing optimization. From the in-depth analysis and segregation, we serve our clients to fulfill their immediate as well as ongoing research requirements. Minute analysis impact large decisions and thereby the source of business intelligence (BI) plays an important role, which keeps us upgraded with current and upcoming market scenarios.

Contact Us:

Contact Name: Shreyas Tanna

Company: Absolute Markets Insights

Email Id:

Phone: IN +91-7400-24-24-24, US +1-510-420-1213


Leave a Reply

Your email address will not be published. Required fields are marked *