Global Orphan Drugs Market – Industry Trends and Forecast to 2030

Global Orphan Drugs Market, By Drug Type (Biological, Non-Biological), Disease Type (Oncologic Diseases, Metabolic Disease, Hematologic And Immunologic Diseases, Infectious Diseases, Neurological Diseases, Others), Indication Type (Non-Hodgkin Lymphoma, Acute Myeloid Leukemia, Cystic Fibrosis, Glioma, Pancreatic Cancer, Ovarian Cancer, Multiple Myeloma, Duchenne Muscular Dystrophy, Renal Cell Carcinoma, Graft Vs Host Disease, Others), Drug Type (Revlimid, Rituxan, Opdivo, Keytruda, Imbruvica, Soliris, Jakaf, Pomalyst, Darzalex, Spinraza, Adcetris), Sale (Generics, Prescribed), Therapy Class (Oncology, Blood, Central Nervous Systems, Endocrine, Cardiovascular, Respiratory), Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Online Sales, Others) – Industry Trends and Forecast to 2030.

 Orphan Drugs Market Analysis and Size

The rapid rise in the prevalence of hypertension, diabetes, and cardiovascular diseases is expected to accelerate market growth from 2023 to 2030. Similarly, an increase in the prevalence of rare diseases and an increase in healthcare expenditure are expected to boost the growth of the orphan drugs market. Additionally, favorable reimbursement policies and favourable government policies are expected to drive market growth. Additionally, the expansion of the product pipeline through the launch of new drugs and an increase in the rate of R&D initiatives are expected to influence the growth of the orphan drugs market.

Data Bridge Market Research analyses that the orphan drugs market which is USD 174.11 billion in 2022, is expected to reach USD 359.86 billion by 2030, at a CAGR of 9.5% during the forecast period 2023 to 2030. In addition to the insights on market scenarios such as market value, growth rate, segmentation, geographical coverage, and major players, the market reports curated by the Data Bridge Market Research also include depth expert analysis, patient epidemiology, pipeline analysis, pricing analysis, and regulatory framework.

Orphan Drugs Market Scope and Segmentation  

Market Definition

Orphan drugs are medical products intended to prevent, diagnose, or treat rare, life-threatening disorders. These are known as orphan drugs because the pharmaceutical industry has little interest in developing and marketing these products for a small number of patients under normal market conditions.

Orphan Drugs Market Dynamics

Drivers

• Rising cases of oral cancer

Rare disease treatments frequently necessitate a cold chain, a temperature-controlled supply chain that is not readily available in all countries. People are rarely affected by orphan or rare diseases (i.e. 7 out of 10,000). However, the global prevalence of rare diseases has been increasing in recent years. According to an article shared by the American Cancer Society, in the United States in January 2022, there are approximately 54,000 new cases of oropharyngeal or oral cancer each year. Oropharyngeal or oral cancer claims the lives of 11,230 people. The average age of people diagnosed with these cancers is 63, but they can strike at any age. Patients under the age of 55 account for slightly more than 20% (1 in 5) of all cases. Thus, the rising prevalence of diseases is driving the growth of the orphan drugs market.

Opportunities

• Rising prevalence of rare diseases

Recurrent, chronic, and rare illnesses, as well as increased demand for significant amounts of orphan drug production, are expected to affect approximately 300 million people worldwide. Rare disease medicines, such as Yellow Fever, von Willebrand Disease, Waardenburg Syndrome, Diabetes Macular Edema, Abetalypoproteinemia, ASD, Yellow Nail Syndrome or Von Hippel Lindau Syndrome and others, are increasing in demand for medicinal products that help increase the market. Furthermore, FDA free exemptions, public R&D grants, favourable reimbursement policies, and premium pricing are available to help businesses grow.

Restraints/Challenges

• High cost associated with drug development

A lack of awareness among patients and physicians about orphan disease treatment, as well as high prices associated with these drug development, are expected to be major restraints on the growth of the orphan drugs market, while high per patient treatment costs and regulatory restrictions on profitability may pose a challenge to the market's growth during the forecast period.

This orphan drugs market report provides details of new recent developments, trade regulations, import-export analysis, production analysis, value chain optimization, market share, impact of domestic and localized market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographic expansions, technological innovations in the market. To gain more info on the orphan drugs market contact Data Bridge Market Research for an Analyst Brief, our team will help you take an informed market decision to achieve market growth.

Recent Developments

• In 2020, Retrophin Inc., a biopharmaceutical company based in the United States, paid $90 million for Orphan Technologies. This agreement includes OT-58, a novel enzyme replacement therapy for treating classic homocystinuria currently in Phase 1/2 trials. The acquisition expands the pipeline of potential first-in-class treatments for rare diseases. Orphan Technologies is a clinical-stage biopharmaceutical company based in Switzerland that is focused on the development of OT-58.

Global Orphan Drugs Market Scope

The orphan drugs market is segmented on the basis of drug type, disease type, indication type, drug type, sale, therapy class and distribution channel. The growth amongst these segments will help you analyze meagre growth segments in the industries and provide the users with a valuable market overview and market insights to help them make strategic decisions for identifying core market applications.

Drug Type

• Biological
• Non-Biological

 Disease Type

• Oncologic Diseases
• Metabolic Disease
• Hematologic and Immunologic Diseases
• Infectious Diseases
• Neurological Diseases
• Others

 Indication Type

• Non-Hodgkin Lymphoma
• Acute Myeloid Leukemia
• Cystic Fibrosis
• Glioma
• Pancreatic Cancer
• Ovarian Cancer
• Multiple Myeloma
• Duchenne Muscular Dystrophy
• Renal Cell Carcinoma
• Graft Vs Host Disease
• Others

 Drug Type

• Revlimid
• Rituxan
• Opdivo
• Keytruda
• Imbruvica
• Soliris
• Jakaf
• Pomalyst
• Darzalex
• Spinraza
• Adcetris

 Sale

• Generics
• Prescribed

 Therapy Class

• Oncology
• Blood
• Central Nervous Systems
• Endocrine
• Cardiovascular
• Respiratory

Distribution Channel

• Hospital Pharmacy
• Retail Pharmacy
• Online Sales
• Others

Orphan Drugs Market Regional Analysis/Insights

The orphan drugs market is analysed and market size insights and trends are provided by country, drug type, disease type, indication type, drug type, sale, therapy class and distribution channel as referenced above.

The countries covered in the orphan drugs market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

North America dominates the orphan drugs market due to high prevalence of rare disorders.

Asia-Pacific is expected to grow at the highest growth rate in the forecast period of 2023 to 2030 because of the developing healthcare infrastructure, as well as the high prevalence of other diseases such as endocrinology, cardiovascular and lymphatic systems, and respiratory disorders.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points like down-stream and upstream value chain analysis, technical trends and porter's five forces analysis, case studies are some of the pointers used to forecast the market scenario for individual countries. Also, the presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

Competitive Landscape and Orphan Drugs Market Share Analysis

The orphan drugs market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies' focus related to orphan drugs market.

Some of the major players operating in the orphan drugs market are:

• F. Hoffmann-La Roche Ltd. (Switzerland)
• Novartis AG (Switzerland)
• Pfizer Inc. (U.S.)
• Eli Lilly and Company (U.S.)
• Johnson & Johnson Services Inc. (U.S.)
• Merck KGaA (Germany)
• Novo Nordisk A/S (Denmark)
• Bristol-Myers Squibb Company (U.S.)
• Alexion Pharmaceuticals, Inc. (U.S.)
• Kyowa Kirin Co., Ltd. (Japan)
• Amgen Inc. (U.S.)
• Biogen (U.S.)
• Celldex Therapeutics (U.S.)
• GSK plc. (U.K.)
• Eisai Co., Ltd. (Japan)
• Takeda Pharmaceutical Company Limited (Japan)
• Vertex Pharmaceuticals Incorporated (U.S.)