A rare disease, also known as an orphan disease, is a medical condition that affects only a small percentage of the population. These diseases are often genetic in nature, meaning they are present throughout a person's entire life, even if symptoms do not appear immediately. The global prevalence of rare diseases is on the rise, with 6,084 rare diseases reported in 2016, according to a survey by the QuintilesIMS Institute. While some rare conditions may affect only a handful of individuals, others, like cystic fibrosis, impact hundreds or thousands of people, with approximately 30,000 individuals affected in the United States alone. Collectively, rare disease patients make up about 6% to 8% of the population.
Several factors contribute to the increasing prevalence of rare diseases, including the growing trend of delayed parenthood and increased use of fertility therapies. As a result, there is a broad diversity of approximately 6,000 rare diseases, each characterized by unique disorders. The symptoms not only vary from disease to disease but can also differ among patients with the same condition. This variability in symptoms can lead to misdiagnosis and delays in receiving proper treatment.
The causes of rare diseases are diverse, with the majority being genetic in origin, directly resulting from changes in genes or chromosomes. The rarity and complexity of these diseases pose challenges for accurate diagnosis and effective treatment. Additionally, the limited understanding of many rare diseases further complicates the development of targeted therapies.
It is crucial to address the unique challenges posed by rare diseases, including the need for specialized medical care, research, and awareness. Advances in genetic testing and research technologies are providing new opportunities to better understand and diagnose rare diseases. Collaborative efforts among healthcare professionals, researchers, and patient advocacy groups are essential in improving the lives of individuals affected by rare diseases. Increased awareness, research funding, and a focus on innovative therapies are key components in addressing the unique needs of this population and working towards better outcomes for those living with rare diseases.
Report Attribute/Metric | Details |
---|---|
Market Opportunities | Development of new technologies for an orphan drug |
Market Dynamics | Rising prevalence of rare diseases Growing investment in research and development of orphan drugs Increasing awareness regarding rare disorders |
Orphan drugs market size was valued at USD 196.60 billion in 2023 and is projected to grow from USD 219.54 Billion in 2024 to USD 480.27 billion by 2032, exhibiting a compound annual growth rate (CAGR) of 9.08% during the forecast period (2024-2032). The rising prevalence of rare diseases, growing investment in research and development of orphan drugs, and increasing awareness regarding rare disorders are driving the market growth.
Orphan Drugs Market Trends
The growing R&D investment efforts made by major companies for the orphan drug creation of fresh product offerings is one of the key driving factors influencing the worldwide market. A number of significant clinical-stage biopharmaceutical businesses and well-established market competitors have excellent pipeline candidates for orphan pharmaceuticals in various phases of clinical trials as a result of the growing awareness and knowledge of rare diseases. For instance, the pharmaceutical industry invested 238 billion US dollars worldwide in research and development in 2021. In contrast, R&D spending reached a total of USD 137 billion in 2012.
Orphan Drugs Drug Type Insightsorphan drugs market segmentation, based on drug type, includes biologics and non-biologics. The biologics segment is to hold the majority share in 2021 in the orphan drugs market revenue. This is due to the rising incidence of various rare diseases curable with biological drugs and growing investment in developing drugs for rare diseases by private companies. Furthermore, major market companies are increasing the number of biological products they are supplying, which is causing them to dominate the biological orphan medication industry.
December 2018: Merck KGaA(Germany), Darmstadt, Announces FDA orphan drug designation for Bifunctional Immunotherapy M7824 in biliary tract cancer.
The orphan drugs market segmentation is based on a sale that includes, generics and prescribed. The generics segment has dominated the market in 2021 and is projected to be the faster-growing segment during the forecast period, 2022-2030. This is due to the low cost of generics as an alternative to branded drugs and a large number of patents expired branded drugs. In addition, Generic firms are having a harder time introducing new treatments as it gets more difficult to develop, produce, and advertise original pharmaceuticals that have lost their patent protection. Original patents worth approximately USD 217 billion that cover a wide range of products, including biologics, oral solids, injectables, inhalers, and over the counter (OTC) drugs, are about to expire. Therefore, approval is necessary for generic medications due to strict regulatory rules, which is anticipated to impede segment expansion.
ORPHAN DRUGS MARKET, BY SALE, 2021 & 2030 (USD BILLION)
Source: Secondary Research, Primary Research, MRFR Database, and Analyst Review
The orphan drugs market data has been bifurcated into revlimid, rituxan, opdivo, keytruda, imbruvica, soliris, jakaf, pomalyst, darzalex, and spinraza. The opdivo segment dominated the market in 2021 and is projected to be the faster-growing segment during the forecast period, 2024-2032. The growth of the opdivo segment in the market is mainly due to the drug works by helping the body's own immune system eliminate cancer cells. Unlike chemotherapy, Opdivo specifically targets cancer cells and often has fewer side effects than traditional chemotherapy drugs.
The orphan drugs market segmentation is based on the therapy class that includes Oncology, blood, central nervous systems, endocrine, cardiovascular, and respiratory. The contract research organizations segment has dominated the market in 2021 and is projected to be the faster-growing segment during the forecast period, 2022-2030. This is due to the emergence of new cancer-related diseases and increasing demand for the treatment of oncology disorders worldwide. For instance, In June 2022, ALX Oncology Holdings Inc.(US) received U.S. FDA grant as orphan drug designation (ODD) to Evorpacept, for the treatment of patients with Acute Myeloid Leukemia. As a result, the oncology section of the orphan drug industry is increasingly dominated by investors who are investing huge sums of money in oncology in order to develop solutions with uncommon medicines for patients suffering from unusual cancer conditions.
August 2022:FDA Approved of Mylan's (US)APOKYN NDA; A Novel treatment for Parkinson's Disease. The FDA approval clears the way for Bertek to market APOKYN and provides a new therapy for "off" episodes, one of the most debilitating aspects suffered by patients with Parkinson's disease.
By Region, the study segments the market into North America, Europe, Asia-Pacific, and the Rest of the World. The North America orphan drugs market is expected to account for USD XX billion in 2021 and is expected to exhibit an XX% CAGR during the study period. This is attributed to the North America. Therefore, favorable regulatory policies and increasing investment by pharmaceutical companies can also be attributed to this regional growth. In addition, The United States has the greatest market share in North America. The fact that a drug with orphan drug classification in the US obtains seven years of marketing exclusivity after FDA clearance for a particular indication, tax credits, and user fee waivers is one of the factors driving market expansion.
Further, the major countries studied are the U.S., Canada, Germany, France, the UK, Italy, Spain, China, Japan, India, Australia, South Korea, and Brazil.
Figure 3: ORPHAN DRUGS MARKET SHARE BY REGION 2021 (%)
Source: Secondary Research, Primary Research, MRFR Database, and Analyst Review
Europe orphan drugs market is expected to account for the second-largest market share due to the increase as a result of numerous pharmaceutical businesses exporting their goods outside of or inside Europe. Further, the Germany orphan drugs market is expected to hold third place for the market share, and the UK orphan drugs market is expected to fastest-growing market, and which is in the 4th place in the European region. Furthermore, France orphan drugs market will hold the 5th place for the market share.
The Asia-Pacific orphan drugs market is expected to grow at a CAGR of XX% from 2022 to 2030. This is due to the due to the presence of established market players across the region and the faster development of medical infrastructure, and higher disposable incomes across the region. Moreover, China orphan drugs market is expected to hold the largest market share, and India orphan drugs market is expected fastest-growing market in the Asia-Pacific region.
For instance, India has reportedly higher rare diseases population than the world average, but initiatives from the government side are still less, and in fact, India lacks national legislation for orphan medicines and rare diseases, in spite that these are most populated countries, making it to be the world leader in the market for the orphan drugs market.
The Rest of the World includes the Middle East, Africa, and Latin America. Nevertheless, due to a lack of awareness and low income, particularly in Africa, the Middle East and Africa held the smallest proportion of the orphan drugs market.
Orphan Drugs Key Market Players & Competitive Insights
The orphan drugs market is characterized by the presence of many global, regional, and local vendors catering to the demand created by research scientists and pharmaceutical and biotechnology companies operating in the industry. The market comprises tier-1, tier-2, and local players. The tier-1 and tier-2 players have reach across the globe with diverse product portfolios. Companies such as F. Hoffmann-La Roche AG (Switzerland), Mylan (US), Celgene Corporation (US), and Novartis AG (Switzerland) dominate the orphan drugs market due to product differentiation, financial stability, strategic developments, and diversified regional presence. The players are focused on investing in research and development. Furthermore, they adopt strategic growth initiatives, such as expansion, product launches, joint ventures, and partnerships, to strengthen their market position and capture a large customer base.
One of the primary business strategies adopted by manufacturers in the Orphan Drugs industry to benefit clients and expand the orphan drugs market sector is to manufacture locally to reduce operating costs.F. Hoffmann-La Roche Ltd (Roche) is one of the leading companies in the field of diagnostics and pharmaceuticals. It develops and markets products for the diagnosis and treatment of anemia, cancer, anticoagulation therapy, cardiovascular diseases, diabetes, central nervous system, chlamydia, dermatology, gonorrhea, gout, hepatitis B and C, hemostasis disorders, HIV/AIDS, HPV, infectious diseases, and autoimmune diseases. The firm operates in two major business segments, namely, pharmaceuticals and diagnostics. The pharmaceuticals segment offers a wide range of drugs for therapeutic areas, such as oncology, inflammation, neuroscience, infectious diseases, and ophthalmology. Furthermore, the diagnostics segment offers a wide range of tests for cancer and companion diagnostic tests. The company has a strong presence in North America, Europe, Asia, and the Middle East. For instance, In December 2019, F. Hoffmann-La Roche Ltd (Switzerland) entered licensing agreement with Sarepta Therapeutics to advance the treatment for a rare disease named Duchenne Muscular Dystrophy (DMD).
Also, Novartis AG (Novartis) is a pharmaceutical company that researches, develops, manufactures, and distributes a wide range of healthcare products globally. The company provides products that find applications in cancer, cardio-metabolic, immunology & dermatology, ophthalmology, neuroscience, and respiratory disease areas. Its Sandoz segment offers active ingredients and finished dosage forms of pharmaceuticals for cardiovascular, dermatology, central nervous system (CNS), gastrointestinal and hormonal therapy, metabolism, oncology, ophthalmic, pain, and respiratory disorders and diseases; and finished dosage forms anti-infective. It operates in the Americas, Europe, the Middle East, and Asia-Pacific.
Key Companies in the Orphan Drugs Market include
Orphan Drugs Industry Developments
July 2021:Novartis Ag (Switzerland) received a U.S. FDA grant as Orphan Drug Designation (ODD) for NIS793 (which is a first-class novel antibody, specific for Transforming Growth Factor, Beta) in combination with standard care of chemotherapy, for the treatment of pancreatic chemotherapy.
November 2019:Celgene Corporation (US) received approval for an orphan medicine named REBLOZYL for the treatment of anemia in adults with beta-thalassemia who require regular red blood cell (RBC) transfusion.
July 2021:Novartis AG (Switzerland) received FDA orphan drug designation for NIS793 in pancreatic cancer. NIS793 is a potential first-in-class novel antibody specific for Transforming Growth Factor Beta (TGFฮฒ), which is known to have an important role in metastatic pancreatic ductal carcinoma (mPDAC) and other solid tumors.
February 2021:FDA approved for TEPMETKO Merck KGaA's (Germany)oral MET inhibitor, the first and only once-daily treatment for patients with metastatic non-small cell lung cancer with METex14 skipping mutations.
May 2020:Eli Lilly and Company (US) received U.S. FDA Approval for Retevmo (selpercatinib), the First Therapy Specifically for Patients with Advanced RET-Driven Lung and Thyroid Cancers.
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