Orphan Drugs Market

Global Orphan Drugs Market: Global Size, Trends, Competitive, Historical & Forecast Analysis, 2022-2028: Increasing prevalence of rare diseases, rising approval of biological orphan drugs for multiple indications, and growing investment by the pharmaceutical giants are some of the major factors driving the growth of the global orphan drugs market.

The Global Orphan Drugs Market is valued at USD 153.67 Billion in 2021 and is expected to reach USD 367.39 Billion by 2028 with a CAGR of 13.26% over the forecast period.

Scope -

A pharmacological agent known as an orphan medicine was created to treat diseases that are too uncommon to be manufactured without assistance from the government. These illnesses are known as orphan diseases. Orphan drug products are pharmaceuticals used to treat serious or life-threatening illnesses or conditions that are unusual for treatment, diagnosis, or prevention. Pharmaceutical companies often have little interest in creating and selling medicines for rare diseases that only affect a tiny percentage of the population. Orphan drug products are pharmaceuticals used to treat serious or life-threatening illnesses or conditions that are unusual for treatment, diagnosis, or prevention. Under typical market circumstances, the pharmaceutical business has little interest in creating and promoting treatments that are intended for a limited population of patients. These medications are known as orphan medicines. Numerous oncological, hematologic, metabolic, immunologic, viral, and neurological illnesses can be successfully treated with them. Medical conditions like lymphoma, leukaemia, cystic fibrosis, glioma, pancreatic cancer, ovarian cancer, multiple myeloma, and renal cell carcinoma are frequently chronic, progressive, degenerative, and life-threatening and call for particular treatment options to be effective for particular symptoms.

The worldwide orphan drug industry has been negatively impacted by the COVID-19 epidemic. Many sectors, including a few in the healthcare sector, have shut down permanently or cut back their significant activities in response to the COVID-19 epidemic. As a result, even during the pandemic, the expansion of the orphan drug sector was significantly slowed down by inadequate screening services and limited access to specialists. The development of the orphan drug sector has been adversely impacted by other reasons, such as inadequate funding, restricted research and development activities, interrupted supply chains, and other difficulties.

The global orphan drugs market is segmented on the therapeutic area, drug components, route of administration, distribution channel, region & country level. Based on therapeutic area, the global orphan drugs market is segmented into respiratory disorders, autoimmune diseases, cancer, blood-related diseases, cardiovascular diseases, ophthalmic diseases, infectious diseases, genetic disorders, neurological disorders, and others. By drug components, the global orphan drugs market is segmented into non-biologics and biologics. By route of administration, the global orphan drugs market is segmented into parenteral, and oral. By distribution channel, the global orphan drugs market is segmented into specialty pharmacies, hospital pharmacies, and retail pharmacies.

The regions covered in the global orphan drugs market report are North America, Europe, Asia-Pacific, Latin America, and the rest of the World. Based on country level, the market of global orphan drugs is sub divided into U.S., Mexico, Canada, U.K., France, Germany, China, Italy, India, Japan, Southeast Asia, Middle East Asia (Saudi Arabia, UAE, Egypt) GCC, Africa, etc.

Key Players -

• F. Hoffmann-La Roche Ltd.
• Bristol-Myers Squibb Company
• Daiichi Sankyo Company Limited
• Novo Nordisk A/S
• Bayer AG
• AstraZeneca plc.
• Amgen Inc.
• Novartis AG
• GlaxoSmithKline plc.
• Others

Segmentation: -

By Therapeutic Area:

• Respiratory Disorders
• Autoimmune Diseases
• Cancer
• Blood-related Diseases
• Cardiovascular Diseases
• Ophthalmic Diseases
• Infectious Diseases
• Genetic Disorders
• Neurological Disorders
• Others

By Drug Components:

• Non-biologics
• Biologics

By Route of Administration:

• Parenteral
• Oral

By Distribution Channel:

• Specialty Pharmacy
• Hospital Pharmacy
• Retail Pharmacy

By Regional & Country Level:

• North America

• U.S.
• Canada

• Europe

• U.K.
• France
• Germany
• Italy

• Asia Pacific

• China
• Japan
• India
• Southeast Asia

• Latin America

• Brazil
• Mexico

• Middle East and Africa

• GCC
• Africa
• Rest of Middle East and Africa 

Increasing prevalence of rare diseases, rising approval of biological orphan drugs for multiple indications, and growing investment by the pharmaceutical giants are some of the major factors driving the growth of the global orphan drugs market.

One of the major factors in the growth of the orphan drug market is the increasing prevalence of rare diseases. An orphan disease or rare disease is any disease that affects a small percentage of the population. Rare disease treatments often require a "cold chain," a temperature-controlled supply chain that is not readily available in all countries. Orphan or rare diseases occur rarely in the population (i.e., 7 in 10,000). Orphan drugs refer to pharmaceutical products used in the diagnosis, prevention, or treatment of orphan diseases or rare diseases. These drugs are effective in targeting numerous indications, including cancer, metabolic diseases, infections, neurological disorders, and other diseases. For instance, according to the National Institutes of Health (NIH), on February 11th, 2020, there be approximately 7,000 rare diseases affecting between 25 and 30 million Americans. Therefore, the increasing prevalence of rare diseases is fueling the market growth.

In addition, the rising approval of biological orphan drugs for multiple indications is boosting the growth of the orphan drug market. Biologic drugs are used to treat rare diseases such as cancer that have fewer side effects and are more common in developed countries. Around the world, 12 people die every minute from different types of cancer. Various initiatives are being taken to address this growing figure. Currently, this includes biological orphan drugs. Also, the market is currently witnessing large-scale acquisitions, as well as other deals, by many players to expand the industry, which is driving huge investment in the market. For instance, as per the news published on June 13th, 2018, Cardax, Inc. announced that it had acquired industry veteran and orphan drug expert Frederick D. Sancilio, Ph.D., to initiate the company's orphan drug development program. Dr. Sancilio has more than 40 years of experience in the pharmaceutical industry, including establishing and operating a research institute that has contributed to more than 2,000 drug product registrations in the US, Asia, and Europe. Therefore, for commercial product development, companies focus on acquiring biological orphan drugs to increase their revenue, thus this factor is boosting the growth of the orphan drug market.

Moreover, the growing investment by the pharmaceutical giants is also supplementing the orphan drug market's growth. However, the high initial investment and high treatment costs associated with orphan drugs may hinder the growth of the global orphan drug market. Despite that, an increase in investment in research and development may create more opportunities in the global orphan drug market.

North America Holds a Major Share in the Global Orphan Drugs Market:

Geographically, North America is expected to have a significant market share for orphan drugs throughout the projected period because of the rising incidence of certain illnesses, expanding government efforts, and expanding patient population. These elements help the market. The market is expanding as a result of the rising incidence of different illnesses, including those that impact the endocrinology, cardiovascular, lymphatic, and respiratory systems. For instance, the Cleveland Clinic estimates that by May 5, 2022, almost 18 million individuals in the United States will pass away from cardiovascular disorders. As a result, the market for orphan drugs in the North American area has been driven by the rise in cardiovascular illness. Rising government activities are another element supporting industry expansion in this area. For instance, the FDA introduced a site for the submission of petitions for orphan medication designation on November 23, 2020, according to the US Food and Drug Administration. It might be challenging to develop medications for uncommon disorders. To support and facilitate effective processes for these kinds of vital therapies, collaboration—including the use of technology—is crucial. Governments in the American area are successfully boosting market share through such on-going investments and activities.

As of rising healthcare expenditure, increased medical investment, and expanding government programs to improve community health, Asia Pacific is the fastest-growing area in the worldwide orphan medication market. These elements help the market. According to the India Brand Equity Foundation, India's public spending on healthcare would be 2.1% of GDP in 2021–22, up from 1.8% in 2020–21 and 1.3% in 2019–20. Premiums sponsored by health insurance firms totaled Rs. 73,582.13 crores (US$ 9.21 billion) in FY22. As a result, rising healthcare spending is likely to fuel the expansion of the orphan medication market throughout the forecast period.

Recent Developments -

Neurocrine Biosciences Received Orphan Drug Designation for Valbenazine as a Treatment for Chorea Associated with Huntington's Disease

On May 12^th, 2022; Neurocrine Biosciences, Inc. announced that it has received US approval for valbenazine as a treatment for Huntington's disease (HD). It has received orphan drug designation from the Food and Drug Administration (FDA). The treatment of chorea associated with Huntington's disease (HD) is within the scope of this orphan drug designation. In December 2021, Neurocrine Biosciences reported top-line data from its Phase 3 KINECT-HD study evaluating the efficacy, safety, and tolerability of valbenazine, a once-daily selective vesicular monoamine transporter 2 (VMAT2) inhibitor in adults. Korea is associated with HD.

Agios Announced Food and Drug Administration (FDA) Orphan Drug Designation Granted to Mitapivat for Treatment of Thalassemia

On June 8^th, 2020; Agios Pharmaceuticals, Inc. A pioneer in the field of cellular metabolism who treats cancer and rare genetic diseases announced that the U.S. The Food and Drug Administration (FDA) has granted orphan drug designation to the company's first-in-class pyruvate kinase-R (PKR) activator, mitapivat, for the treatment of patients with sickle cell disease. Mitapivat is an investigational, oral, small-molecule allosteric activator of wild-type and various mutant PKR enzymes.

Report Analysis	Details
Historical data	2018 - 2021
Forecast Period	2022 - 2028
Market Size in 2021:	USD 153.67 Billion
Base year considered	2021
Forecast Period CAGR %:	13.26%
Market Size Expected in 2028:	USD 367.39 Billion
Tables, Charts & Figures:	175
Pages	200
Companies	F. Hoffmann-La Roche Ltd., Bristol-Myers Squibb Company, Daiichi Sankyo Company Limited, Novo Nordisk A/S, Bayer AG, AstraZeneca plc., Amgen Inc., Novartis AG, GlaxoSmithKline plc., and others
Segments Covered	By Therapeutic Area, By Drug Components, By Route of Administration, By Distribution Channel
Regional Analysis	North America, U.S., Mexico, Canada, Europe, UK, France, Germany, Italy, Asia Pacific, China, Japan, India, Southeast Asia, South America, Brazil, Argentina, Columbia, The Middle East and Africa, GCC, Africa, Rest of the Middle East and Africa

Key Benefits –

• Global Orphan Drugs Market report covers in-depth historical and forecast analysis
• Global Orphan Drugs Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.
• Global Orphan Drugs Market report helps to identify opportunities in marketplace.
• Global Orphan Drugs Market report covers extensive analysis of emerging trends and competitive landscape.