Orphan Drugs Market Expected to Reach $435.68 Billion by 2030 | CAGR of 11.8%

According to a new report published by Allied Market Research, titled, “Orphan Drugs Market, by Disease Type: Global Opportunity Analysis and Industry Forecast, 2021–2030," the global orphan drugs market size was valued at $140.00 billion in 2020 and is projected to reach $435.68 billion by 2030 registering a CAGR of 11.8% from 2021 to 2030.

Orphan drugs are specialized pharmaceutical agents administered for the treatment of rare (orphan) diseases. These diseases have a very low prevalence rate; thus, pharmaceutical companies do not readily invest in these drugs as the returns on investment in orphan drugs are risky as compared to non-orphan drugs. Moreover, multiple clinical trials for drug testing cannot be voluntarily performed due to the small patient population. However, orphan drugs have shown tremendous potential in diagnosis and treatment of cancer; this trend is expected to continue throughout the forecast period.

Favorable government policies and rise in cases of rare diseases boost the orphan drugs market growth. In addition, availability of market exclusivity for orphan drugs developers, and increase in indications of orphan drugs to treat an array of different diseases such as lymphoma, leukemia, myeloma, and others, also boost the growth of the market. However, limited patient pool for clinical trial & product marketing and high treatment costs per patient, restrain the market growth. On the other hand, growth in novel indications designated for known orphan drugs and untapped emerging markets are expected to offer profitable opportunities for the growth of the orphan drugs market during the forecast period.

COVID-19 is a large family of viruses that causes illness ranging from common cold to more severe respiratory diseases. The global orphan drugs market also declined in 2020 due to factors such as a decline in screening services, reduced access to specialists, treatment interruption, limited operations in most industries, inadequate funding to research & academic institutes, temporary closure of major academic institutes, and disrupted supply chain and challenges. However, the market is anticipated to witness recovery in 2021, and show stable growth for orphan drugs market in the coming future.

The global orphan drugs market is segmented on the basis of disease type, and region. By disease type, it is categorized into oncological diseases, metabolic diseases, hematologic & immunologic diseases, infectious diseases, neurological diseases, and other rare diseases that include transplantation diseases, gastrointestinal diseases, dermatologic diseases, perinatal & congenital abnormalities, and others.

Oncological diseases segment is again divided into acute myeloid leukemia (AML), pancreatic cancer, ovarian cancer, multiple myeloma, renal cell carcinoma, and others. In addition, metabolic diseases segment is further classified into hunter syndrome, Fabry disease, Gaucher disease, hypoparathyroidism, and others. Furthermore, hematologic & immunologic diseases segment is segmented into hereditary angioedema (HAE), hemophilia, and others. Moreover, neurological diseases segment is further segmented into Duchenne muscular dystrophy (DMD), Alzheimer's disease, Huntington's disease, and others. The oncological diseases segment was the major shareholder in 2020, owing to increase in prevalence of oncological diseases, rise in cancer awareness among population, early screening of cancer and availability of oncological orphan drugs for treatment of cancer.

Key Findings Of The Study

• Based on disease type, the oncological diseases segment held the largest share in the global orphan drugs market in 2020.
• Based on region, North America is expected to experience growth at the highest rate, registering a CAGR of 11.0% during the orphan drugs market forecast period.

North America accounted for the largest share of the global orphan drugs market in 2020, and is expected to remain dominant throughout the forecast period. This was attributed to presence of large patient population and early adoption of advance drugs, strong presence of key players, availability of drugs, early diagnosis, and favorable reimbursement policies.