Global Rare Cancer Drug Market Clinical Trials Report

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


  • Global Orphan Designated Market Opportunity > USD 150 Billion by 2028
  • Global & 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 > 70% Of Global Orphan Cancer Drug Market
  • Top 50 Drugs Pricing, Dosage, Patent Insight
  • Orphan Designated Cancer Drug Clinical Trials Insight by Company, Country & Indication
  • Insight On More Than 400 Orphan Designated Cancer Drugs in Clinical Trials
  • Insight On More Than 200 Orphan Designated Cancer Drugs Commercially Available in Market


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Rare cancers or orphan cancers are group of cancer which generally affect so few people that drug development for such diseases is considered unprofitable and neglected by the pharmaceutical companies. Most of these rare cancers are genetic and present throughout the patient's entire lifetime, even if the symptoms do not appear immediately. With the passage of orphan drug act, the government has created several economic incentives to stimulate orphan drug development. Grants, research design support, FDA fee waivers, tax incentives, orphan drug market exclusivity, and public diffusion of orphan innovation are main incentives for orphan cancer research and development.


Till date, more than 200 orphan designated cancer drugs have entered the global markets which have shown encouraging response in the pharmaceutical market. Some of the leading orphan cancer drugs are Revilimid, Opdivo, Yervoy, Keuytruda, Tecentriq, Imbruvica, and others. Since the awareness and understanding surrounding rare cancers has increased, a number of clinical stage biopharmaceutical companies and established market players have created strong pipeline candidates for orphan drugs, which are in various stages of clinical trials. The major key players in the market include Abbvie, Roche, Celgene Corporation, Bristol Myers Squibb, Novartis, Boehringer Ingelheim, AstraZeneca, and several others.


Several orphan designated drugs are also gaining rapid entry into the market which also suggests promising future of this segment. For instance, Incyte has recently announced that Japan MHLW has granted approval to Pemazyre (pemigatinib), a selective fibroblast growth factor receptor (FGFR) inhibitor, which is indicated for the management of patients with unresectable biliary tract cancer with FGFR2 gene fusion. Previously, the drug has also been granted orphan drug designation in Japan for the same therapeutic indication. Further in 2022, EMA has granted conditional marketing authorization to Carvykti (ciltacabtagene autoleucel) for the management of adult patients with relapsed and refractory multiple myeloma. The drug is developed from the joint collaboration between Janssen Pharmaceutical and Legend Biotech. In 2020, the drug has been designated orphan designation in Europe.


Biological therapy is currently holding the maximum share in the global market which is primarily due to their targeted mechanism of action and few associated adverse events. Monoclonal antibodies, antibody drug conjugates, bispecific antibodies, chimeric antigen receptor (CAR) T cell therapy and immune checkpoint inhibitors are various biological drug classes which have gained orphan designation for the management of cancer. However, their market is mainly restrained by the high cost which is not accessible to large population. The looming patent expiry of the biological drugs opens opportunities for the development of biosimilar drugs. Each biosimilar is highly similar to its approved reference product in terms of efficacy, safety, and quality without being identical to it. Several biosimilars of Avastin, Herceptin, and Rituximab have entered the market which has demonstrated encouraging sales.


As per our report findings, the global orphan cancer drug market is expected to surpass US$ 150 Billion by 2028. The market is mainly driven by the rising prevalence of various types of cancer such as breast, lung, colorectal, and other hematological tumors. In addition, huge investments by pharmaceutical companies in this segment and rising government initiatives to enhance awareness about rare cancers is also driving the growth of market For instance, Japan Medical and Research Development Agency (AMED) has launched The Initiative of Rare and Undiagnosed Disease (IRUD) which aims to support the diagnosis of patients with undiagnosed disease via data sharing and promote research into pathology that may lead to the development of new treatments and therapies. The rising trends among pharmaceutical companies and non-government organizations will also have a positive impact on the growth of market during the forecast period.




Neeraj Chawla

Kuick research

Research Head



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