Orphan Drugs Market Is Expected To Increase at a 12.48% of CAGR by 2030

Published Date : 25 May 2023

The orphan drugs market was projected to more than double in size from USD 171.31 billion in 2022 to roughly USD 438.9 billion in 2030, progressing with a CAGR of 12.48 percent from 2022 to 2030.

Pharmaceutical products utilized in the diagnosis, prevention, and treatment of uncommon medical conditions are referred to as orphan medications. These medicines are designed to fulfill a particular public health need and typically have a small market because they are made for a select set of patients. Numerous neurological, immunologic, viral, metabolic, hematologic, oncological, and metabolic illnesses can be successfully treated with them. Health conditions like lymphoma, leukemia, cystic fibrosis, glioma, pancreatic cancer, ovarian cancer, multiple myeloma, and renal cell carcinoma are frequently life-threatening, chronic, progressive, degenerative, and disabling and call for particular treatment options that are efficient for their unique symptoms.

Market Growth:

Growing R&D efforts in the development of orphan pharmaceuticals are one of the key factors propelling the global market. The market for orphan pharmaceuticals is expanding as a result of leading industry participants' ongoing efforts to provide unique product offerings. The expanding patient population associated with orphan diseases is attracting pharmaceutical companies and other stakeholders to engage in this industry.

For instance, the U.S. FDA sponsored a virtual public conference in March 2021 to celebrate Rare Disease Day and bring together stakeholders to discuss methods that can support the development of rare disease products. There has been a noticeable increase in uncommon disease awareness and consciousness among the general community. The demand for effective and promising treatments is further increased by the fact that patients with rare illness conditions are looking for them.

Regional Analysis:

According to regional analysis, North America has the greatest market share because there are many important market players and a sizable number of people who suffer from rare diseases. Additionally, a large number of orphan pharmaceuticals are in the development phase, as well as more orphan drugs are being spent in the region, all of which are contributing to the North American region's dominance in the orphan drug industry.

As a result of enhanced government initiatives and supportive policies for research & development in orphan pharmaceuticals, the European region, followed by the Asia Pacific market, is predicted to see a higher CAGR over the projected period. Additionally, these regions are making inroads into the world market for orphan pharmaceuticals due to growing product uptake and robust healthcare infrastructure.

Orphan Drugs Market Report Scope:

Report Coverage Details
Market Revenue in 2023 USD 192.68 Billion
Projected Forecast Revenue in 2030 USD 438.9 Billion
Growth Rate from 2022 to 2030 CAGR of 12.48%
Largest Market North America
Base Year 2022
Forecast Period 2022 to 2030
Regions Covered North America, Europe, Asia-Pacific, Latin America, and Middle East & Africa

Market Dynamics:

Market Drivers:

One of the main factors propelling the market's expansion is the rising incidence of cancer and other uncommon genetic illnesses. Innovative orphan medications are being created by pharmaceutical companies to offer patients individualized treatments. The market is also being stimulated by rising public awareness of the advantages of orphan medications. The development of new pharmaceutical drug producers and the adoption of advantageous government regulations to stop the spread of infectious diseases are other factors driving the market's expansion. Other growth-promoting elements include a variety of product developments, like the creation of biological orphan medications. Due to the fact that these medications can restore stem cell damage and treat diseases like cancer, their demand is rising significantly. Additional factors, including advancements in healthcare infrastructure and intense research and development (R&D) efforts, are anticipated to fuel the market even more.

Market Restraints

The market for orphan pharmaceuticals may be constrained by the high cost of these medications. Orphan medications are only made available to certain patients due to the rarity of orphan diseases. Orphan medications are expensive due to the limited supply and significant investments in research and development. Globally speaking, the COVID-19 pandemic has had a significant negative impact on business operations. Additionally, the dissemination of COVID-19 affected various groups in both favorable and harmful ways. A few industries that provide healthcare services were among those forced to close their doors. Additionally, the COVID-19 outbreak had a detrimental effect on the market for orphan pharmaceuticals because of a shortage of funding, challenges with operational research efforts, and a disrupted supply chain in most industries.

Market Opportunities

Although there is a significant opportunity for market participants in the global orphan drug market due to the rising prevalence of orphan illnesses. Additionally, despite the fact that orphan illnesses are uncommon, more than 400 million people worldwide are afflicted by one of the 7000 distinct forms that have been identified. For market participants in the orphan medication sector, this opens up tremendous possibilities.

Drug Type Insights:

The market for orphan medications can be further divided into biological and non-biological orphan products depending on the drug type. Biologics, also known as biological products, is a broad category of medications that include vaccines, growth factors, monoclonal antibodies, immune modulators, as well as goods made from human blood and plasma, according to the World Health Organization (WHO). Major market companies are increasing the number of biological products they are supplying, which is causing them to dominate the biological orphan medication industry. The biological segment is anticipated to grow more rapidly than the non-biological segment throughout the projection period as a result of the increase in cutting-edge biomedical research. The biological orphan drug market is also dominated by the growing number of biological orphan products that are undergoing various stages of clinical studies.

Therapy Insights

The market for orphan pharmaceuticals is further divided into cancer, hematology, neurology, infectious illnesses, metabolic disorders, endocrinology, immunology, and other uncommon diseases based on the therapy they are used for. The market for orphan pharmaceuticals worldwide is currently dominated by the oncology category, which is anticipated to expand at a faster CAGR throughout the forecast period. The category is dominated by the increasing number of oncology clinical advancements and the presence of potentially accessible orphan medication therapies for cancer patients. For instance, the U.S. FDA granted ALX Oncology Holdings Inc.'s Evorpacept the orphan drug designation (ODD) in June 2022 to treat patients with Acute Myeloid Leukemia.

Distribution Channel Insights

According to the distribution channel, it is anticipated that the hospital pharmacies sector will hold the biggest market share in the orphan medication industry. Patients with orphan diseases must only be managed and treated with IV drug doses while being observed and monitored by qualified healthcare personnel. As a result, orphan pharmaceuticals are made available, ideally through hospital pharmacies, further dominating this market niche for orphan drugs. Due to the very small number of orphan medications that have been approved, retail pharmacies, internet pharmacies, and other pharmacy stores are expected to see a relatively low CAGR during the forecast period.

Recent Development:

  • In July 2021, For the treatment of pancreatic cancer, Novartis' NIS793 (a first-class monoclonal antibody that is specific for Transforming Growth Factor, Beta) earned an Orphan Drug Designation (ODD) award from the U.S. Food and Drug Administration.
  • In Nov 2019, REBLOZYL, an orphan drug from Celgene Corporation and Acceleron Pharma Inc., has been approved to treat anemia in adults with beta-thalassemia who need regular red blood cell (RBC) transfusions.
  • In Dec 2019, F. Hoffmann-La Roche Ltd. and Sarepta Therapeutics have a license agreement to develop the treatment of the rare disease Duchenne muscular dystrophy (DMD)

Major Key Players:

  • F. Hoffmann-La Roche Ltd
  • Celgene Corporation
  • Merk & Co., Inc.
  • Novartis AG, Sanofi
  • Takeda Pharmaceutical Company Limited
  • AstraZeneca
  • Alexion Pharmaceuticals Inc.
  • Eli Lilly and Company
  • Amgen Inc.

Market Segmentation:

By Drug Type

  • Biologicals
  • Non-Biologicals

By Therapy

  • Oncology
    • Ovarian cancer
    • Multiple Myeloma
    • Pancreatic Cancer
    • Renal Cell Carcinoma
    • Others
  • Haematology
    • Hereditary Angioedema
    • Hemophilia
    • Others
  • Neurology
    • Alzheimer’s Disease
    • Huntington’s Disease
    • Duchenne Muscular Dystrophy
    • Others
  • Infectious Diseases
  • Metabolic Disorders
    • Gaucher Disease
    • Hypoparathyroidism
    • Hunter Syndrome
    • Fabry Disease
    • Others
  • Endocrinology
  • Immunology
  • Others

By Distribution Channel

  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies
  • Others

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