Global Orphan Drug Rare Disease Clinical Trials Market USD 350 Billion Opportunity

Release Date: 23-Nov-2021

Global Orphan Drugs Clinical Trials Insight Report Highlights:



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Orphan drugs refer to the medications which are intended for diagnosing, preventing and treating rare, life-threatening diseases or disorders. The rare diseases are specific conditions which generally affect less than 200,000 people. The most frequently occurring rare diseases are Primary immunodeficiency disorders, Lysosomal storage disorders (Gaucher’s disease, Mucopolysaccharidoses, Pompe disease, fabry disease etc.) small molecule inborn errors of metabolism (Maple Syrup urine disease, organic acidemias etc.), Cystic Fibrosis, osteogenesis imperfecta, certain forms of muscular dystrophies and spinal muscular atrophy, etc.


Till now, a cocktail of orphan drugs have entered the market, whose adoption rates are continuously expanding. At present, incentives for drug development by government authorities in various countries are encouraging pharmaceutical companies to develop orphan drugs. For instance, National Institute of Health (NIH) supports research to improve orphan drugs including fast track approval, waiver of fees, protocol assistance and marketing exclusivity. Furthermore in Japan, government incentives include financial subsides for up to 50% of expenses for clinical and non-clinical research, 10 year marketing rights, and reduction in tax.


Currently, the global orphan drug market is mainly dominated by cancer drugs which are mainly attributed to the large number of product approvals and their robust sales in the market. Moreover, in last few years the prevalence of cancer has increased drastically which is mainly due to increase in geriatric population, smoking, alcohol consumption, exposure to carcinogens, limited physical activity, and poor diet. The increase in prevalence of rare cancers eventually boosts up the growth of market. Apart from this, neurology and cardiovascular segment also have a significant share in the global market.


The advent of biotechnology has for sure aided in the research and development of rare diseases. However, the development of orphan drug possesses several challenges including low disease prevalence, disease severity, small and heterogeneous population, difficulties in patient recruitment, and limited knowledge of the history of disease.  Apart from this, the orphan drugs are generally priced high which are unaffordable to majority of patients. However, to overcome this pharmaceutical companies and research institutions have developed favorable reimbursement policies which ensure the availability of drugs to all patients.


Despite several challenges, various pharmaceutical companies including Novartis, Pfizer, Sanofi, Takeda Pharmaceutical, Amgen, Biogen, Alexion, Merck, and others are continuously investing large amount in the development of orphan drugs. Regionally, US are holding the most significant share in the market by adopting advanced technologies. The presence of major key players in the region and adoption of strategies including collaboration or partnerships also drive the growth of market in the region. In addition to this, pharmaceutical companies have also developed several co-pay programs like Amgen 360, to further reduce the financial toxicity on patient. Following US, Europe is also leading market with the growing approval of orphan drugs.


Although, Asia-Pacific region is currently present at nascent stage but it is expected to exhibit highest CAGR during the forecast period due to the rising population and the increase in prevalence of rare diseases. India and China are the major countries contributing the largest shares of market growth. To compete with US companies, India origin companies including Sun Pharma, Biocon, ReGrow Bioscience, Pirmal, Natco and Cadila Pharma are continuously investing a large amount for the development of orphan drugs in India. Recently, Pirmal Pharma Solutions entered into partnership with Epirium Bio on an exclusive manufacturing relationship for new orphan drugs targeting rare diseases with high unmet needs. Apart from this, China have initiated several government and private funded programs including patient assistance program (PAP), crowd funding platforms, commercial health insurances, critical disease insurances which help in reducing the cost of management of rare diseases in China.




Neeraj Chawla

Research head

Kuick Research



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