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

Report Highlights:

• Global Orphan Designated Market Opportunity > USD 170 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

Orphan cancer drugs are novel pharmaceuticals that are indicated for the prevention, diagnosis, or treatment of rare cancers. These rare cancers are chronic and life-threatening which require proper long-term medications. As these diseases are rare, therefore the drug development of these diseases requires a considerable amount of investment, thus increasing the overall cost of the drug. However, the passage of the 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 pharmaceutical companies to actively invest in this segment. To date, more than 700 drugs have been granted orphan designation for the management of orphan-designated 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 over the past few years, which has encouraged 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 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) that 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 the 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 a clinical trial collaboration and supply agreement with Sanofi. Under the terms of the agreement, Sanofi will evaluate its 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 the high cost of therapy, low prevalence of disease, difficulty in recruiting patients, lack of awareness among patients, and unavailability of diagnostic tests for rare cancer hamper the growth of the market. Although several challenges restrict the growth of the market, it is witnessed that the global orphan cancer drug market is continuously growing over the past few years. 

As per report findings, the global orphan cancer drug market is expected to surpass US$ 150 Billion by 2028 which is mainly attributed to the increase in the prevalence of rare cancers associated with high demand for 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 the market. Apart from this, several pharmaceutical companies like Amgen, Roche, Genetech have developed assistance programs through which their potentially lower the out-of-pocket cost for the patients.