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

  • No. of Pages: 2400
  • Publish Date: June-2022
  • Category: Pharmaceutical and Healthcare
Electronic Access - Single User License
CD-ROM Mail Delivery
Hard Copy Mail Delivery
Electronic Access - Multi-User License

Buy USD 20,000 Worth Of Reports (Single User/Multi User License) In USD 8000 Only

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


  • Global Orphan Designated Market Opportunity > USD 170 Billion by 2028
  • Global and Regional Market Size 2022 Till 2028
  • Market Size By Orphan Designated Cancer Type 2022 Till 2028
  • Top 50 Orphan Designated Cancer Drug Sales Forecast Till 2028
  • Top 50 Drugs Account For > 75% Of Global Orphan Cancer Drug Market
  • Top 50 Drugs Pricing, Dosage, Patent Insight
  • Orphan Designated Cancer Drug Clinical Trials Insight by Company, Country and Indication
  • Insight On More Than 450 Orphan Designated Cancer Drugs in Clinical Trials
  • Insight On More Than 200 Orphan Designated Cancer Drugs Commercially Available in Market



Orphan cancer drugs are novel pharmaceuticals which are indicated for the prevention, diagnosis, or treatment of rare cancers. These rare cancers are chronic and life-threatening which requires proper long-term medications. As these diseases are rare, therefore the drug development of these diseases requires considerable amount of investment, thus increasing the overall cost of the drug. However, the passage of orphan drug development act in 1983 has opened avenues for the development of novel orphan drugs which are intended for the management of these cancers. The passage of this act incentivizes the pharmaceutical companies to actively invest in this segment. Till date, more than 700 drugs have been granted orphan designation for the management of rare cancers.


This rapid increase in the designation also appears to have translated into an increase in approvals and commercialization of these drugs. As of now, more than 200 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. The sales of these drugs have been tremendously increasing from past few years, which have encouraged the further research and development activities in this domain.


The global orphan cancer drug market is highly concentrated with several key layers including Mersana Therapeutics, Bristol Myers Squibb, Immunocore, Amgen, Genmab, Roche, Lava Therapeutics, and others. The key players are actively receiving orphan drug designation by the regulatory bodies which are expediting their clinical trials and approval process. For instance in 2022, Mersana Therapeutics has received orphan drug designation from US FDA to XMT-2056 for the management of gastric cancer. XMT-2056 is an investigational STING-agonist antibody drug conjugate (ADC) which is developed using the company’s Immunosynthen platform and targeting a novel epitope of human epidermal growth factor receptor 2 (HER2). The company has planned to initiate phase-I trial of XMT-2056 in a range of HER2 expressing tumors, such as breast, gastric and non-small-cell lung cancers, in mid-2022.


In last few years, pharmaceutical companies have begun to embrace a more collaborative way of working to mitigate the obstacles of drug development. For instance, Immunocore entered into clinical trial collaboration and supply agreement with Sanofi. Under the terms of agreement, Sanofi will evaluate it’s precisely PEGylated, engineered version of IL-2, SAR444245, in combination with Kimmtrak, Immunocore’s novel bispecific protein targeting gp100, in HLA-A*02:01 positive patients with advanced unresectable or metastatic skin cancers as part of Sanofi’s ongoing Phase 1/2 study. The novel bispecific protein has been granted orphan drug designation by US FDA.


Despite several benefits associated with orphan cancer drug development, few factors restrain their growth in the market during the forecast period. Factors such as high cost of therapy, low prevalence of disease, difficulty in recruiting patients, lack of awareness among patients, and unavailability of diagnostic test for rare cancer hamper the growth of market. Although several challenges restrict the growth of market, but it is witnessed that the global orphan cancer drug market is continuously growing from past few years.


As per our report findings, the global orphan cancer drug market is expected to surpass US$ 150 Billion by 2028 which is mainly attributed to increase in prevalence of rare cancers associated with high demand for the orphan drugs in its management. The high spending on research and development of orphan cancer drugs, coupled with the presence of several large scale manufacturers are the major factoring driving the growth of market. Apart from this, several pharmaceutical companies like Amgen, Roche, Genetech have developed their assistance programs through which their potentially lower down the out-of pocket cost from the patients.


Need custom market research solution? We can help you with that too.