Know about Orphan Drugs

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The pharmaceutical agents that are administered to treat, diagnose, and prevent life-threatening diseases (rare diseases) are known as orphan drugs. The global orphan drugs market accounted for $106 billion in 2015, and is anticipated to reach $169 billion by 2022, registering a CAGR of 6.8% from 2016 to 2022.

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Orphan drugs are used to treat rare (orphaned) diseases prevalent in a very small patient pool. Hence, the pharmaceutical industry takes little interest in their development. Moreover, their development requires high cost and the chances of returns on investment are comparatively less when compared to non-orphan drugs. These drugs treat different rare diseases, such as non-Hodgkin lymphoma, acute myeloid leukemia, cystic fibrosis, and others.

Conducive government regulations and market exclusivity for orphan drugs drives the global orphan drugs market. In addition, surge in prevalence of rare diseases and rise in investment in research, drug development, and innovation supplement the market growth. However, limited patient pool for clinical trials & product marketing and high treatment costs per patient impede the market growth. Furthermore, growth in novel indications for known orphan drugs and untapped emerging markets offer lucrative opportunities for the market players.

Based on indication, this market is classified into non-Hodgkin lymphoma, acute myeloid leukemia, cystic fibrosis, glioma, pancreatic cancer, ovarian cancer, multiple myeloma, Duchenne muscular dystrophy, Graft vs host disease, renal cell carcinoma, and others. The non-Hodgkin lymphoma was the highest revenue contributor in 2015, while renal cell carcinoma is anticipated to grow at the highest rate. The growth for this segment is due to increase in investment in R&D throughout the world along with surge in awareness about kidney cancer.

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