Emergence of Orphan Drugs in Cancer Therapy

Release Date: 30-Aug-2022

Global Orphan Cancer Drug Market, Drug Sales, Price and Clinical Trials Insight 2028 Report Highlights:



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Over the past few years, it is observed that the incidence rate of rare cancers is increasing rapidly and hence the demand of novel drugs targeting these conditions is rising year by year. Orphan cancer drugs are a special category of drugs, which are intended to treat rare cancers. Till date, about 200 rare cancers have been identified which represents about 22% of the overall cancer incidences. The most frequently occurring rare cancers are esophageal cancer, chronic myeloid leukemia, merkel cell carcinoma, glioblastoma, hepatocellular carcinoma, and others.


Because of the low incidences of rare cancers, their management and diagnosis possess a major challenge. Despite this, more than 200 drugs have been granted orphan designation for the management of rare cancers till now. As of now, more than 50 drugs have been commercially available for the management of rare cancers. Several biological drugs such as Herceptin, Rituximab, Blincyto, Kymriah, Opdivo, Keytruda, Yervoy, and others have been granted orphan designations which have shown encouraging response in the market owing to their targeted mechanism of action.


Since the awareness and understanding of rare cancers have increased, several pharmaceutical companies have actively invested in this segment. Apart from this, the benefits associated with the development of orphan cancer drugs also propels the investment in this segment. For instance in US once the orphan cancer designation is granted, sponsors receive 25% federal tax credit tax for the costs of clinical testing and are also eligible to pay for FDA grants to further defray these costs. In addition, if the orphan drug receives FDA approval for orphan indication, the Orphan Drug Act provides with a 7 year period of “orphan drug exclusivity” during which competitors are prohibited from marketing the same drug to treat the same disease.


The global orphan drug market is highly consolidated and consists of several major key players including Novartis, Pfizer, Merck, Sanofi, Amgen, Biogen, Takeda Pharmaceutical, and several others. Rapid launch of new products is one of the major factors which are adopted by key players in the market. For instance in 2022, US FDA has granted both an orphan drug designation and a fast track designation to a novel sonodynamic therapy platform as a potential treatment for patients with recurrent glioblastoma and other malignant gliomas. Another, Phanes Therapeutics also received orphan drug designation to PT886 for the treatment of pancreatic cancer. PT886 is a first-in-class bispecific antibody targeting claudin 18.2 (CLDN18.2) and cluster of differentiation 47 (CD47) being developed for patients with pancreatic cancer as well as gastric and gastroesophageal cancers.


One of the biggest challenges faced by the physicain for the management of orphan disease is related to the difficulty in diagnosis and medical condition of patients. This is mainly due to lack of investigation of the disease and hence physician try to provide symptomatic relife to the patient without targeting the major cause. Research studies are now focused on developing novel diagnostic test for rare cancer such as Galleri Test. This novel test present a far more efficient way of detecting cancer. Instead of searching for any one type of cancer, it screens an individual for multiple cancers. Its potential is to change the current screening process from screening for individual cancers to one where individuals are screened for multiple cancers with a single blood test. The test is highly accurate and has a 0.5% false-positive rate.


As per our analysis, the global orphan cancer drug market is expected to surpass US$ 150 Billion by 2028. The market is driven by several factors including increase in prevalence of rare cancer and rising availability of early diagnostic tests. Apart from this, large number of orphan designated cancer drugs which are expected to gain market authorization in forecast period will also drive the growth of market.  

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