Orphan Drugs Market Size to Grow by USD 477 Bn by 2032 | Oncology Segment Accounted to Hold a Significant Revenue Share

According to Market.us, The presence of a large patient pool suffering from rare diseases and the availability of advanced treatment modalities are important catalysts propelling the expansion of the orphan drugs market.

New York, April 24, 2023 (GLOBE NEWSWIRE) — The Orphan Drugs Market size accounted for USD 168 billion in 2022 and it will expand to USD 477 billion by 2032, growing at a CAGR of 11.3% during the forecast period from 2023 to 2032. Pharmaceutical products used to diagnose, prevent, and treat rare medical conditions are referred to as orphan drugs. Due to the fact that they are produced for a selective group of patients, these medications typically have a limited market because they are made to meet a specific public health requirement.

Orphan Drugs Market Value

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Key Takeaway:

  • By therapy in 2022, the oncology segment is accounted to hold a significant revenue share and dominate the orphan drugs market.
  • By drug type, the biologics segment dominated the market in 2022.
  • By distribution channel, the hospital pharmacies segment dominated the market in 2022 and is expected to remain dominant over the forecast period.
  • In 2022, North America dominated the market with the highest revenue share of 46.0%.
  • Asia-Pacific will grow at a significant CAGR from 2023-2032.

Orphan drug market expansion is anticipated to be driven by favorable government regulations and reimbursement rules. Additionally, it is anticipated that the market’s expansion will also be aided by an increase in healthcare spending and the prevalence of rare diseases.

Factors affecting the Growth of the Orphan Drugs Market

There are several factors that can affect the growth of the orphan drugs market. Some of these factors include:

  • Rising incidence of rare diseases: The market growth of orphan drugs is mainly attributed to the rising incidence of rare diseases.
  • Favorable regulation: The global orphan drugs market is likely to expand significantly as a result of favorable regulations and reimbursement policies.
  • Large patient population: The market expansion is further influenced by the large patient pool and advanced treatment for rare diseases.
  • High cost: The higher cost of medications for rare diseases is likely to hamper the market growth during the forecast period.

Top Trends in Global Orphan Drugs Market

Rising demand for immunomodulators and favorable government regulations are expected to fuel market growth. The result of the rising incidence of infectious diseases in developing and developed countries and also the rising drug costs are anticipated to support market growth. Moreover, the industry’s growth is also aided by the possibility of orphan medication manufacturers holding exclusive commercial rights.

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Market Growth

The market for orphan drugs is anticipated to expand as a result of the rising prevalence of rare diseases, favorable regulations, and reimbursement policies. Additionally, market expansion is influenced by rising healthcare expenditure. However, the higher costs of drugs for rare disease is likely to impede market growth in coming years.

Regional Analysis

North America accounted for the highest revenue share of the global orphan drugs market in 2022. The market growth is mainly influenced by regional development that is being aided by favorable government funding and policies. Moreover, promoting laws that enable the rapid approval of such drugs, drug status exclusivity for such drugs, and a robust health system all contribute to the industry’s expansion. The European market is anticipated to expand quickly owing to the region’s large patient population and wide-ranging acceptance of advanced treatments for rare diseases. Furthermore, the Asia-Pacific orphan drugs market is expected to expand significantly as the prevalence of rare diseases rises and people become more aware of them.

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Scope of the Report

Report Attribute Details
Market Value (2022) USD 168 Billion
Market Size (2032) USD 477 Billion
CAGR (from 2023 to 2032) 11.3%
North America Revenue Share 46.0%
Historic Period 2016 to 2022
Base Year 2022
Forecast Year 2023 to 2032

Market Drivers

Rare or orphan diseases only affect 7 out of every 10,000 people. However, rare diseases’ global prevalence has been rising in recent years. Treatments for rare diseases frequently require a cold chain, which is a temperature-controlled supply chain that is not readily accessible in all nations. In January 2022, there will be approximately 54,500 new cases of oropharyngeal or oral cancer in the United States, according to an article that was shared by the American Cancer Society. 11,250 people are affected by oral (oropharyngeal) cancer. These cancers can strike at any age, but the average age at diagnosis is 60-65. One in five of all cases involves patients under the age of 55 or slightly more than 20%. As a result, the market for orphan drugs is expanding in response to the rising incidence of diseases.

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Market Restraints

Lack of awareness among patients and physicians regarding the treatment of orphan diseases and the high costs associated with drug development is likely to hamper the market growth during the forecast period. Moreover, high per-patient treatment costs and regulatory restrictions on profitability may also pose a challenge to the market’s growth during the forecast period.

Market Opportunities

The increasing prevalence of rare diseases is anticipated to create numerous growth opportunities during the forecast period. Medicines for rare diseases like yellow fever, Waardenburg syndrome, von Willebrand disease, abetalipoproteinemia, diabetic macular edema, yellow nail syndrome, autism spectrum disorder (ASD), and von Hippel-Lindau syndrome, among others, are in high demand, which helps in the expansion of the market. Around 300 million people across the globe are anticipated to be affected by recurrent, rare, and chronic illnesses, as well as increased demand for significant amounts of orphan drug production. In addition, the FDA provides free exemptions, favorable reimbursement policies, public R&D grants, and premium pricing to help businesses in expansion.

Report Segmentation of the Orphan Drugs Market

Therapy Insight

The oncology segment is anticipated to hold the largest revenue share during the forecast period. The drug division that deals with oncology treats an increasing number of cancer patients with various diseases each year. The development of effective medications to treat or maintain control of chronic conditions is necessary due to the rising geriatric rate of the elderly population. In the field of drug development, there is a growing demand and interest in powerful medications to combat the situation. As a consequence of this, it is anticipated that the global market for orphan drugs will experience development opportunities in the coming years.

Drug Type Insight

The biologics segment is accounted as the most lucrative and holds the largest market share in the global market. Rare diseases have long been treated with biological medicines. Despite its higher prevalence in developed nations, cancer, a most orphan disease, has been successfully treated by biological therapies with fewer side effects. In addition, the current trend of biological orphan drug approval for a variety of applications has led to expansion and has inspired both existing and new market players to arrive in this sector.

Distribution Channel Insight

The hospital pharmacies held the largest revenue share and are expected to dominate the global orphan drugs market over the forecast period. This is because skilled healthcare providers in hospitals are required to administer a significant amount of medications intravenously. As a result, hospital pharmacies need to ensure the availability of rare diseases, which in turn contributes to segment growth.

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Market Segmentation

By Therapy

  • Oncology
  • Neurology
  • Endocrinology
  • Hematology
  • Cardiovascular
  • Immunotherapy
  • Respiratory
  • Other Therapies

By Drug Type

  • Biologics
  • Non-Biologics

By Distribution Channel

  • Hospital Pharmacy
  • Retail Pharmacy
  • Online Pharmacy

By Geography

North America

  • The US
  • Canada

Western Europe

  • Germany
  • France
  • The UK
  • Spain
  • Italy
  • Portugal
  • Ireland
  • Austria
  • Switzerland
  • Benelux
  • Nordic
  • Rest of Western Europe

Eastern Europe

  • Russia
  • Poland
  • The Czech Republic
  • Greece
  • Rest of Eastern Europe


  • China
  • Japan
  • South Korea
  • India
  • Australia & New Zealand
  • Indonesia
  • Malaysia
  • Philippines
  • Singapore
  • Thailand
  • Vietnam
  • Rest of APAC

Latin America

  • Brazil
  • Colombia
  • Chile
  • Argentina
  • Mexico
  • Costa Rica
  • Rest of Latin America

Middle East & Africa

  • Algeria
  • Egypt
  • Israel
  • Kuwait
  • Nigeria
  • Saudi Arabia
  • South Africa
  • Turkey
  • United Arab Emirates
  • Rest of MEA

Competitive Landscape

The competitive landscape of the market has also been examined in this report. Major market players include

  • Hoffmann-La Roche Ltd. (Switzerland)
  • Novartis AG (Switzerland)
  • Johnson & Johnson Services Inc. (U.S.)
  • Merck KGaA (Germany)
  • Bristol-Myers Squibb Company (U.S.)
  • Pfizer Inc. (U.S.)
  • Eli Lilly and Company (U.S.)
  • Novo Nordisk A/S (Denmark)
  • Takeda Pharmaceutical Company Limited (Japan)
  • Alexion Pharmaceuticals, Inc. (U.S.)
  • Kyowa Kirin Co., Ltd. (Japan)
  • GlaxoSmithKline Plc
  • AbbVie Inc. Company Profile
  • Sanofi S.A
  • Amgen Inc. (U.S.)
  • Biogen Inc. (U.S.)
  • Celldex Therapeutics (U.S.)
  • GSK plc. (U.K.)
  • Eisai Co., Ltd. (Japan)
  • Vertex Pharmaceuticals Incorporated (U.S.)
  • Alexion Pharmaceutical Inc.
  • ALX Oncology Holdings Inc.
  • BioMarin Pharmaceutical Inc.
  • Agios Pharmaceuticals Inc.
  • DAIICHI SANKYO Company Ltd.
  • Other Key Players

Recent Development of the Orphan Drugs Market

  • In March 2022, CER-001, which was developed by ABIONYX Pharma, was granted FDA approval for Orphan Drugs Designation (ODD) for the diagnosis of LCAT deficiency that manifests as kidney dysfunction or ophthalmologic illness. Both partial LCAT insufficiency, which causes Fisheye Disease, and comprehensive LCAT deficiency, which causes renal symptoms and corneal opacities, are referred to by this term. The progression of LCAT deficiency, for which there is no treatment, can eventually result in kidney disease that calls for kidney transplantation or dialysis and/or corneal opacification that calls for a transplant.
  • In April 2022, Shanghai Henlius Biotech revealed in a press release stating that the Food and Drug Administration has granted serplulimab, also known as Hansizhuang, orphan drug status for the treatment of SCLC, or small cell lung cancer.
  • In February 2022, The European Commission granted AZP-3601 Orphan Drug Designation (ODD) for the treatment of hypoparathyroidism, according to Amolyt Pharma, a multinational corporation that focuses on developing therapeutic peptides for rare endocrine and associated conditions. ODD grants sponsors access to centralized marketing authorization, funding for the proposed, reduced regulatory costs, and ten years of market exclusiveness if granted.

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