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Rare Cancer Orphan Drug Clinical Trials Insight 2028

Rare Cancers Accounts For More Than 30% Of Global Orphan Drug Clinical Trials Says Kuick Research

/EIN News/ -- Singapore, May 19, 2022 (GLOBE NEWSWIRE) -- Global Orphan Drugs Market & Clinical Trials Insight Report 2028 Highlights:

  • Clinical Insight On More Than 600 Marketed Orphan Drugs
  • Clinical Insight On More Than 1300 Orphan Drugs in Clinical Trials
  • Orphan Designated Drug Clinical Status by Indication & Country
  • Global Market Opportunity More Than USD 350 Billion
  • Market Exclusivity & Patent Protection Criteria for Orphan Drugs
  • Global & Regional Orphan Drug Market Sales Opportunity
  • Orphan Drug Reimbursement Policy
  • Orphan Clinical Pipeline Overview Company, Drug Class, Formulation, Indication, Region, Priority Status, Patient Segment, Phase

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Over the past few years, it is observed that the incidence rate of rare disorders is increasing rapidly and hence the demand of novel drugs targeting these conditions is rising year by year. Orphan drugs are a special category of drugs, which are intended to treat rare disease. Till date, about 500 drugs have been granted orphan designation by regulatory bodies and about 200 of them are currently available in the global market. Although the target population size is small, these drugs have shown robust sales in the market which indicates promising future of orphan drug in the global market.

One of the major factor responsible for rapid increase in market size of orphan drug is that the orphan drugs have a market exclusivity of 7 years and hence provides high cost saving for the development of niche drugs. The 1983 orphan drug act also encourages the pharmaceutical giants for developing orphan drugs and provides several benefits including tax and incentive savings. In additrion to this, the orphan drugs require less amount of funds for advertising and marketing of drug as the target population is small as well as the price of orphan drug is also less as compared to niche drugs. All these benefits of orphan drugs are indicating the huge market potential of this segment which attract pharmaceutical giants to invest in this segment.

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, EDI-301 developed by Edita Medicine has received orphan drug designation by US FDA for the treatment of beta thalassemia. EDIT-301 is an experimental gene editing therapy which consists of CD34+ hematopoietic stem and progenitor cells obtained from patients and are edited at the gamma globin gene (HBG1 and HBG2) promoters by a AsCas12a nuclease and is present in phase-I/II clinical trials.    

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.   For overcoming this challenge, researchers are employing several advanced technologies. The entrance of artificial intelligence can act as gamechanger as it can help the physician to understand the patient’s condition as well as to develop novel drug candidate in minimum time period.

It is believed that several rare genetic diseases are associated with the appearance or gradual development of distinctive facial appearances in affected individuals. FDNA has developed Face2Gene, which is a novel technology that analyzes facial photos to identify facial morphology associated with rare diseases. This is a free tool used by hundreds of geneticists worldwide and the technology converts facial photos into a de-identified mathematical algorithm that describes the characteristics of each facial feature. FDNA is collecting data from images of individuals with confirmed diagnosis of many different genetic diseases to further improve this technology. These rising trends in the market will boost the growth of market during the forecast period.

As per our report findings, the global orphan drug market is expected to surpass US$ 350 Billion by 2028. The increase in prevalence of cancers and other rare genetic disorders is one of the key factors driving the growth of orphan drugs market The market is also driven by the increasing awareness among the population regarding the benefits of orphan drugs. In addition, implementations of new laws and rising government initiatives are expected to boost the growth of market in coming few years.

For More Information Related To Report Contact:

Neeraj Chawla
Research Head
Kuick Research


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