Rare Disease Treatment Market Analysis

Rare Disease Treatment Market Analysis

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Based on the drug type, the global market is classified into biologics and non-biologics. The biologics segment dominated the market with revenue of USD 166.9 billion in 2023.

• The demand for biologics in rare disease treatment increases due to several benefits it possesses such as high specificity, low toxicity, extreme sensitivity to physical conditions, etc. Also, technological advancements in pharmaceutical and biotechnology industry allow large-scale syntheses of biologics in a cost-effective manner. In conditions of safety, biologics are identified to cause less severe adverse events.

   

• Furthermore, the cost of several biologic drugs is covered by the medical insurance, thus enabling patients to access effective treatments.

Based on the therapeutic area, the global rare disease treatment market is classified into cancer, blood-related disorders, central nervous system, respiratory disorders, musculoskeletal disorders, cardiovascular disorders, and other therapeutic area. The cancer segment dominated the market with market share of 48.9% in 2023.

• Growing adoption of biological therapy including gene therapy, cytokine therapy, monoclonal antibodies, targeted drug therapy, immunoconjugates for treating the cancer will boost market growth in the future. Also, the growing emphasis on biologics to treat the rare diseases with high compatibility, specificity and low toxicity will witness steady growth in the future.

   

• Furthermore, various market players are focusing on development of novel products for treating these rare diseases that will propel the growth significantly.

   

Based on the patient, the global rare disease treatment market is classified into adult and pediatric. The adult segment is expected to exhibit CAGR of 8.7% during the analysis period.

• The adult segment contributed maximum share owing to increasing adult and geriatric population pool suffering from rare disorders. As per the report published by National Institute of Health in November 2023, it has been reported that an estimated 25 to 30 million Americans are affected with rare disease. In addition, Undiagnosed Disease Program (UDP) has received nearly 10,000 inquiries, reviewed more than 3,000 applications, and admitted about 900 patients to the NIH Clinical Center for comprehensive weeklong evaluations. Thus, the increase in the number of people diagnosed with rare disease and the growing demand to develop therapeutic drugs for adult patient population is substantially increasing in the recent past years that will further propel the market growth positively.

   

Based on route of administration, the rare disease treatment market is classified into oral and injectable. The oral segment is expected to reach USD 235.1 billion by end of 2032.

• The high market revenue is attributed to various advantages of the oral route of administration such as non-invasiveness, the convenience of drug administration, and patient compliance among others.  According to the research article published in 2021, around 60% of approved small-molecule drugs available commercially are administered through the oral route.  Furthermore, oral therapies offer a higher rate of bioavailability at an affordable cost. Thus, the easy availability of oral dosage in store is another factor that will contribute the market growth for this segment.

In 2023, North America secured a substantial market share of 48.8% in the global rare disease treatment market and is expected to dominate throughout the forecast period.

• This is attributable to numerous variables including the presence of key market players, an increase in the number of rare diseases, and rising awareness regarding the benefits of precision medicine among others.

   

• Also, federal agencies such as the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), allocate funding for rare disease research and development to support basic and translational research, clinical trials, and infrastructure development, thereby driving innovation in rare disease treatments.

   

• Furthermore, reimbursement policies in the U.S., including coverage by Medicare and private insurance companies, influence the commercial viability of rare disease treatments. Thus, the aforementioned factors contributed to North America's dominance in the market.

   

Germany to experience the highest growth rate in the market, outstripping other countries in Europe in the assessment period.

• Germany has a relatively high prevalence of rare diseases in the Europe region, with an estimated 4 million people affected. This large patient population creates a significant market opportunity for pharmaceutical companies developing rare disease treatments.

   

• Further, Germany has a well-developed clinical research infrastructure, including academic medical centers, research hospitals, and clinical trial networks. Thus, the availability of research infrastructure facilitates clinical trials for rare disease treatments, thereby accelerating their development and commercialization which in turn help in the market growth.

   

The India rare disease treatment market is expected to grow with a significant CAGR of 11.5% during the forecast period.

• The increase in awareness pertaining to rare diseases among healthcare providers, patients, and the public in India will leads to higher demand for treatment options, thereby supplementing the market growth.

   

• Additionally, Indian pharmaceutical companies often collaborate with international partners, including academic institutions, research organizations, and multinational pharmaceutical companies, to conduct clinical trials and develop rare disease treatments. These partnerships facilitate knowledge sharing, technology transfer, and resource pooling, thus fostering the country level market growth.