Global Orphan Drug Market Size Clinical Trials Opportunity Insight

Release Date: 04-Feb-2022

Global Orphan Disease Drugs Clinical Trials Insight 2028 Report Highlights:



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Orphan diseases are those diseases which affects only a small population of the world. Although rare diseases affect about 2% of the global population, there are about 7000 orphan diseases with unmet need of medical treatment. Orphan drugs refer to pharmaceutical products which in diagnosis, prevention, or treatment of orphan diseases. These drugs are effective in targeting numerous indications including metabolic diseases, cancer, infections, neurological disorders, and other diseases. Since the passage of orphan drug act in 1983, there has been fierce competition among pharmaceutical companies to develop orphan drugs for rare therapeutic indications.


The increasing investment by the pharmaceutical giants has led to introduction of more than 500 orphan drugs in the market. Due to their specialized nature and niche applications coupled with small patient populations, these drugs have grown to become the priciest in the market. Despite several challenges including high price and low target population base, orphan drugs have gained considerable popularity among pharmaceutical companies which is mainly attributed to large benefits associated with their development including 50% research and development tax credit, clinical trial subsidies, waived or reduced regulatory fees, and for the first drug sponsor, eligibility for seven years of market exclusivity after receiving marketing approval.


With the presence of several major players, the global orphan drug market is highly competitive. The leading pharmaceutical companies in the market include Pfizer, Novartis, Roche, Eli Lilly, Bristol Myers Squibb, Johnson and Johnson, Takeda Pharmaceutical, Bayer, AstraZeneca, Sanofi, Biogen and several others. The companies have also entered into partnership or collaboration to maintain their position in the global market. For instance in 2020, Centogene and Molecular Health collaborated to initiate the Real-life data and Innovative Bioinformatic Algorithms (RIBA) project andndash; starting with Epilepsy as the first indication. RIBA aims to foster a unique novel precision medicine environment to accelerate, de-risk, and improve the development of new orphan drugs based on the combination of large real-life data sets in rare disease with innovative big data, innovative artificial intelligence, as well as computational algorithms and expertise.


Globally, US is occupying the majority of the share in the market which is mainly due to rapid approval of FDA orphan designation by the government. For instance in 2022, US FDA has granted orphan drug designation to AZ-3102 developed by Azafors in GM2 gangliosidosis including both Sandhoff and Tay-Sachs diseases. Similarly, Thesus Pharmaceutical has received orphan drug designation to THE-630 for the treatment of advanced gastrointestinal stromal tumors. In addition, ALX Oncology also received orphan drug designation to evorpacept, a next-generation CD47 blocker, for the treatment of patients with gastric cancer and gastroesophageal junction cancer. The increasing orphan drug designations by US FDA help in boosting the growth of the market in the region. Apart from this, the presence of large pharmaceutical sector which actively invest in research and development sector also boosts the growth of market in the region.


As per our report findings, the global orphan drug market is expected to surpass US$ 350 Billion by 2028. The market is driven by the increasing prevalence of rare diseases and rising awareness among individuals regarding the benefits of orphan drugs. In addition, increasing investment by pharmaceutical companies and rising initiatives by government bodies will also propel the growth of market in forthcoming years. Further, the potential advances in the field of immunotherapy and gene therapies will also progress the development of orphan drug during the forecast period.


The report aims to provide market sizes and forecast the values for different segments and countries till 2028. The study aims to include qualitative and quantitative perspectives about the industry within the regions and countries covered in the report. The report also outlines the significant factors, such as driving factors and challenges that will determine the market’s future growth.        

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