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Learn details of the orphan drugs market | boom comes with a steady stream of outsourcing

Medical Market Research

Global Orphan Drugs Market is expected to garner $169 billion from $106 billion and register a CAGR of 6.8% during the forecast period, 2016-2022.

Orphan drugs have positively impacted diverse fields of therapeutics and treatment. Diseases that were otherwise debilitating and life-threatening can be managed through orphan drug administration.

These drugs have also contributed significantly in diagnosis and prevention of oncologic, metabolic, immunologic, neurologic, and other rare diseases. Orphan drugs are expected to witness high adoption rate with increase in distinct indications and interventions, such as lymphomas, carcinomas, leukemia, and others.

Orphan drug market has gained momentum in the healthcare industry and pharmaceutical sector owing to the favorable government policies, conducive regulations abiding orphan drugs, and market exclusivity for these drugs. In addition, surge in prevalence of rare diseases also drives the market growth.

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Orphan drugs are specialized pharmaceutical agents that are administered for treatment of rare (orphan) diseases. These diseases have a very low prevalence rate, hence, pharmaceutical companies do not readily invest in these drugs as the returns on investment in orphan drugs is risky when compared with 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.

In addition, the increase in indications of orphan drugs to treat an array of different diseases such as lymphoma, leukemia, myeloma, and others boost the market growth.

Orphan drugs form a niche yet considerably profitable segment of the pharmaceutical industry. Furthermore, the market exclusivity periods enjoyed by these drugs owing to favorable government legislations especially in the U.S.

and Europe add to their value and profitability. 

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Disease Type

  • Oncologic Diseases
  • Metabolic Diseases
  • Hematologic and Immunologic Diseases
  • Infectious Diseases
  • Neurologic Diseases
  • Other Rare Diseases


  • 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

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