Orphan Drugs Market, by Product Type (Biologics and Non-Biologics), by Disease Type (Oncology Disease, Neurological Disease, Metabolic Disease, Hematologic and Immunologic Disease and Others) – Global Industry Insights, Trends and Forecast, 2017 - 2025

Created On: Jun-2018
Report ID: IR54
Format: PDF

Market Introduction

Orphan drugs refers to the pharmaceutical agent that are developed specifically to treat rare medical condition known as orphan disease such as Aagenaes syndrome, oncological disease, leukemia among others. According to World Health Organization such disease affects 0.65-1 out of every 1000 population. The Orphan Drug Act was passed on 1983 by USA to stimulate the development, research and the approval of the products that are required to treat such diseases. There are approximately 6000 orphan diseases out of which eighty percent are caused due to genetic disorders and some are caused due to bacterial or viral infections. These diseases are considered as the rarest ones and the symptoms mostly appear at birth or during the childhood. For instance, cystic fibrosis, lysosomal storage disorder and familial adenomatous polyposis among others. However, some disease symptoms also appear during adulthood such as renal cell carcinoma, acute leukemia and glioma among others.

Growing awareness toward chronic disease among the population and increasing advanced technological treatments and drug products has resulted to the growth of the market of orphan drugs. According to American Cancer Statistics in 2018 it is estimated that there will be about 63,340 new case of kidney cancer cases which requires proper treatment. Globally there is approximately 350 million population who are suffering from the rare disease. Increasing research and development among the manufacturers to proper provide drugs for the treatment is triggering the global orphan drugs market during the forecast period.

Market Dynamics

Regular product development of the orphan drugs, government regulations and initiatives taken by the regulatory bodies to treat such diseases are the major factors driving the market. Ovid Therapeutics a biopharma company who are focused toward developing medicines that transform the lives of people with rare neurological diseases, announced that the FDA has granted permission to orphan drug TAK-935/OV935 for the treatment of the syndrome known as Lennox-Gastaut. In 2017 Takeda Pharmaceutical Company Limited and Ovid formed a collaboration to commercialize and develop TAK-935/OV935 for the treatment of epileptic encephalopathies, which are group of rare epilepsies that results to morbidities and can worsen over time. Several agencies such as FDA of United States and other health care department are taking initiatives toward biotechnology and healthcare industries to enhance research and development on orphan drugs. However, manufacturing orphan drugs to treat the rare diseases do not generate proper profit margin for the manufacturers due to the prolonged timeline of the manufacturing, product development and expenses thus are restraining the market growth. In addition to this number of patients suffering from the orphan diseases are low and pharmaceutical companies are given rights to manufacture and distribute the drugs for a particular set of years. Moreover, untapped emerging economies and development of products in recent years are generating opportunity to the market during the forecast period.

Segmentation Analysis

The market has been segmented on the basis of different product type such as biologics and non-biologics. In terms of product segmentation, biologic segment remained the leading market in terms of revenue in 2017 and is expected to remain the dominant segment during the forecast period. This is mainly due to the high efficacy of biologic products for the treatment of the chronic diseases, fewer side effects and the development of the generic versions of such drugs. According to Insights and Reports it has been recorded there are more than 50% of orphan drugs that are considered to be biologics.

On the basis of different disease type the market has been segmented into oncological disease, neurological disease, metabolic disease hematologic and immunologic disease and others which includes cardiovascular and infectious diseases. In terms of diseases oncology disease segmented dominated the market in 2017. Increasing number of patients over the years has resulted to the dominance of the oncological disease segment. According to National Cancer Institute, in 2018 an estimated of 1,735,350 new cases of cancer patients will be diagnosed in United States. The number of new cases associated with cancer is 439.2 per 100,000 men and women per year.

Regional Outlook

In terms of regional segmentation, North America stands to be the largest market in 2017 followed by Europe owing to the increasing adoption of such drugs for the treatment of the rare diseases. According to Global Genes Organization, there are approximately 30 million patients in the United States who are living with the rare diseases and 10% of the U.S population are suffering from such rare diseases. Asia Pacific is expected to show the fastest growth rate during the forecast period owing to the increasing regulations towards orphan drugs. For instance, Asian countries has developed orphan drug legislation such as Singapore Orphan Drug Exemption to the Medical Act and Japan’s Orphan Drug Amendment to develop guidelines and regulatory requirement for the products to enter the market of Asia Pacific.

Competitive Landscape

The market for orphan drugs has players who emphasizes upon product innovation, launch, merger and acquisition and geographic expansion in order to grab a larger market share. For instance, in 2016, Intercept Pharmaceuticals received approval from U.S FDA for their first orphan drug product, obeticholic acid that are marketed in U.S as Ocaliva.

Our Research Approach Includes
  •  Market Outlining
  •  Framing discussion guide
  •  Data Validation
  • Data Analysis
  •  Re-Validation and Finalization of Data
  •  Report Insights and Publishing
Our Research Approach Includes
  •  Market Outlining
  •  Framing discussion guide
  •  Data Validation
  • Data Analysis
  •  Re-Validation and Finalization of Data
  •  Report Insights and Publishing
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