Orphan Drugs Market Outlook 2024-2032:
The orphan drugs market size reached a value of USD 214.8 Billion in 2023. Looking forward, the market is expected to reach USD 541.3 Billion by 2032, exhibiting a growth rate (CAGR) of 10.5% during 2024-2032.
The market is driven by the imposition of supportive regulations and policies, rapid advances in genomics and personalized medicine, increasing awareness about rare diseases and patient advocacy, growing financial incentives and market exclusivity, and the heightened investment by pharmaceutical companies in rare disease treatments.
Increasing Prevalence of Rare Diseases
The rising prevalence of rare diseases across the globe is a primary driver of the orphan drugs market. Rare diseases are genetic in nature and affect a small percentage of the population but collectively impact millions of people worldwide. With over 7,000 identified rare diseases, many patients face limited treatment options. However, the increasing awareness and diagnosis of these conditions, facilitated by advances in medical technology and genetic testing, have highlighted the urgent need for effective treatments. In line with this, the increasing investment by governments and healthcare organizations in research to better understand these diseases is driving the demand for orphan drugs. For instance, in the United States alone, approximately 30 million people suffer from rare diseases. This growing patient pool necessitates the development of specialized medications, prompting pharmaceutical companies to invest in orphan drug research and development (R&D).
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Furthermore, the increasing prevalence of rare diseases, leading to the establishment of rare disease registries and patient databases, which provide valuable data for researchers and pharmaceutical companies, is creating a positive outlook for the market. These databases facilitate patient identification for clinical trials, enhancing the development and approval process for orphan drugs. Additionally, the emergence of patient advocacy groups that play a crucial role in raising awareness and funding for rare disease research is anticipated to drive the market growth.
Imposition of Favorable Regulatory Frameworks
The imposition of favorable regulatory frameworks across major countries is positively impacting the orphan drugs market growth. They offer incentives to pharmaceutical companies who are developing orphan drugs that include market exclusivity for a certain period, tax credits, fee waivers, and grants, which lower the financial risks associated with R&D. For example, the Orphan Drug Act (ODA) of 1983 in the United States has been instrumental in encouraging the development of treatments for rare diseases by providing seven years of market exclusivity. Similar regulations in Europe and other regions offer ten years of market exclusivity. These policies have created a more attractive environment for investment in orphan drugs, leading to an increase in the number of approved orphan drug designations and subsequent market growth.
Additionally, regulatory agencies have streamlined the approval process for orphan drugs, offering fast-track designations, priority reviews, and accelerated approvals. This expedited process reduces the time and cost associated with bringing orphan drugs to market, making it more feasible for pharmaceutical companies to invest in rare disease treatments. Along with this, the heightened collaborative efforts between regulatory agencies, pharmaceutical companies, and patient advocacy groups that contribute to the development of comprehensive regulatory frameworks are supporting the market growth. In addition, regulatory bodies also offer support throughout the drug development process by providing protocol assistance and scientific advice to companies developing orphan drugs. This support helps streamline the development process, ensuring that clinical trials are designed effectively and meet regulatory requirements, thereby reducing the time and cost associated with bringing an orphan drug to market.
Rapid Technological Advancements in Biotechnology
The rising innovations in gene therapy, personalized medicine, and advanced biologics that are revolutionizing the treatment landscape for rare diseases are creating a positive outlook for the market. These technologies enable the development of targeted therapies that address the underlying genetic causes of diseases, thus offering more effective and personalized treatment options. For example, gene therapy has been effective in treating rare genetic disorders like spinal muscular atrophy (SMA) and certain types of hemophilia. As a result, the ability to design treatments that can specifically target defective genes or proteins has opened new avenues for drug development, making it possible to address previously untreatable conditions.
Consequently, biotechnological advancements are expanding the therapeutic possibilities within the orphan drugs market, driving further research and development (R&D) investments and innovation. Moreover, rapid advancements in molecular diagnostics and next-generation sequencing (NGS) that improve the identification of genetic mutations and biomarkers associated with rare diseases are providing a thrust to the market growth. This has facilitated the development of precision medicines tailored to individual patients’ genetic profiles, enhancing treatment efficacy and reducing adverse effects.
Apart from this, the burgeoning integration of artificial intelligence (AI) and machine learning (ML) in drug discovery processes to accelerate the identification of potential orphan drug candidates, thereby optimizing the development pipeline, is catalyzing the market growth.
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Leading Companies in the Orphan Drugs Market:
The market research report by IMARC encompasses a comprehensive analysis of the competitive landscape in the market. Some of the companies in the orphan drug industry are AbbVie Inc., Alexion Pharmaceuticals Inc., Amgen Inc., Biogen Inc., Bristol-Myers Squibb Company, F. Hoffmann-La Roche AG (Roche Holding AG), Jazz Pharmaceuticals Plc, Johnson & Johnson, Merck & Co. Inc., Novartis AG, Pfizer Inc., Sanofi S.A., Takeda Pharmaceutical Company Limited and Teva Pharmaceutical Industries Ltd.
In April 2024, Alexion’s Voydeya (danicopan) was approved in the U.S. as an add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care Ultomiris (ravulizumab) or Soliris (eculizumab) to address the needs of approximately 10-20% of patients with PNH who experience clinically significant EVH while treated with a C5 inhibitor.
In May 2024, Biogen agreed to buy privately held Human Immunology Biosciences for up to $1.8 billion, bulking up on rare disease medicines. Human Immunology’s (HI-Bio) felzartamab has completed mid-stage studies for a type of kidney disease called primary membranous nephropathy and in patients with a disease where the immune system makes antibodies that damage their transplanted organ. The San Francisco-based company is also testing the therapy in patients with another chronic kidney disease known as IgA nephropathy. The therapy developer plans to conduct late-stage trials.
In February 2024, Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) accepted the supplemental new drug application (sNDA) for Augtyro (repotrectinib). This application is for the treatment of adult and pediatric patients who are 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion and are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity. The filing acceptance is based on results from the registrational Phase 1/2 TRIDENT-1 trial (adult patients with NTRK-positivesolid tumors) and CARE study (pediatric patients with NTRK-positivesolid tumors).
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Regional Analysis:
The major markets for orphan drugs include the North America (United States and Canada), Asia-Pacific (China, Japan, India, South Korea, Australia, Indonesia, and others), Europe (Germany, France, United Kingdom, Italy, Spain, Russia, and others), Latin America (Brazil, Mexico, and others), and the Middle East and Africa. According to projections by IMARC, North America has the largest patient pool for cancer/tumor profiling while also representing the biggest market for its treatment. According to projections by IMARC, North America has the largest patient pool for orphan drugs while also representing the biggest market for its treatment.
North America’s orphan drug market is driven by a confluence of factors. The imposition of the supportive regulatory environment that is fostering the development of treatments for rare diseases through incentives, tax credits, and assistance with the drug approval process is catalyzing the market growth. Moreover, the prevalence of rare diseases in North America is another critical factor driving the market growth. Along with this, the increasing awareness and diagnosis of these conditions, which underscore the urgent need for effective treatments, is fueling the market growth.
Besides this, the increasing availability of advanced medical facilities, cutting-edge technology, and a highly skilled healthcare workforce that are essential for the diagnosis, treatment, and management of rare diseases is fueling the market growth. Moreover, the presence of leading research institutions and universities that foster innovation and collaboration in rare disease research is strengthening the market growth.
Key information covered in the report.
Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2032
Breakup by Drug Type:
- Biological
- Non-Biological
Breakup by Disease Type:
- Oncology
- Hematology
- Neurology
- Cardiovascular
- Others
Breakup by Phase:
- Phase I
- Phase II
- Phase III
- Phase IV
Breakup by Top Selling Drugs:
- Revlimid
- Rituxan
- Copaxone
- Opdivo
- Keytruda
- Imbruvica
- Avonex
- Sensipar
- Soliris
- Others
Breakup by Distribution Channel:
- Hospital Pharmacies
- Retail Pharmacies
- Online Stores
- Others
Breakup by Region:
- North America
- United States
- Canada
- Asia Pacific
- China
- Japan
- India
- South Korea
- Australia
- Indonesia
- Others
- Europe
- Germany
- France
- United Kingdom
- Italy
- Spain
- Russia
- Others
- Latin America
- Brazil
- Mexico
- Others
- Middle East and Africa
Competitive Landscape:
The report has also analysed the competitive landscape of the market with some of the key players.
· AbbVie Inc.
· Alexion Pharmaceuticals Inc.
· Amgen Inc., Biogen Inc.
· Bristol-Myers Squibb Company
· Johnson & Johnson
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