Orphan Drugs is vaccine, medicine, or in-vivo diagnostic agent that is intended to treat, diagnose, and prevent a rare disease (or) rare medical condition, the condition itself being as an orphan disease. According to U.S. Food and Drug Administration (U.S. FDA) a product that treats a rare disease affecting less than 2, 00,000 Americans. Orphan drugs are intended for the diagnosis, treatment or prevention of a life-threatening, chronic condition. Most of the rare diseases are genetic and thus are presenting throughout the persons entire life,and symptoms may not immediately appear. It is estimated that 5,000 - 8,000 rare diseases existed today, and affecting 6%- 8% of the population in total in European Union, approximately 27 Mn -36 Mn people are suffering from rare diseases in the European Union. The Symptoms of most of the rare diseases may appear at birth or in childhood, for instance, Lysosomal storage disorder, Cystic fibrosis, infantile spinal muscular atrophy, familial adenomatous polyposis (FAP), patent ductus arteriosus (PDA). However, some of the rare diseases symptoms are appear during adulthood, such as glioma, renal cell carcinoma, and acute myeloid leukemia. Maximum number of rare diseases has identified genetic origins. Some are the result of bacterial infections and viral and due to degenerative causes.
Orphan Drugs Market: Drivers and restraints
Global Orphan Drugs market can be driven by following drivers, Regulatory bodies are collaboration with the research institutes are promoting the research and development on rare disease. For instance, The European Union seventh frame work program for research and technical development will boost the research into rare diseases. National Organization for rare disease (NORD) enables to provide the scientific and technical information regarding rare diseases. These conducts patient assistance programs and provide information of medication, disease specific. Medical and scientific knowledge of rare disease is lacking. Treatment and medication are highly expensive.
Orphan Drugs Market: Overview
Increasing the demand for advanced technological treatments and novel drug products to treat various rare diseases are provides a robust market growth. According Global Genes Organization, Approximately 30 Mn patients in the United States are living with the rare diseases, 10% of U.S population suffering from rare diseases. European region also having 30 Million people are suffering from rare diseases. Globally there are approximately 350 Million people are living with rare diseases Increasing the research advancements and applications of these advancements to treat rare diseases propels Orphan Drugs market over the forecast period.
Orphan Drugs Market: Region-Wise Outlook
Depending upon the geographic regions Orphan Drugs market is segmented into seven key regions: Those are North America, Latin America, and Western Europe, Eastern Europe, and Asia pacific excluding japan, Middle East and Africa (MEA), Japan.
North America dominating the global Orphan Drugs market followed by Europe. Increasing awareness among the people towards the use of novel products and technologies to treat the rare diseases providing lucrative market. Asian Pacific region market is considered to rapidly evolving healthcare infrastructure and research and development, this region is anticipating providing a robust growth of global Orphan Drugs market over the forecast period.
Orphan Drugs Market: Key players
Some of the key players in Market are Hoffmann- La Roche, Celgene Corporation, Alexion Pharmaceuticals, Inc., Novartis AG, Takeda Pharmaceuticals Company Limited, Biogen Idec Limited, and Eli Lilly, Bristol-Myers Squibb, Vertex pharmaceuticals, Inc. Bayer AG, Sanofi, and Johnson & Johnson.
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Regional analysis includes
- North America (U.S., Canada)
- Latin America (Mexico, Brazil)
- Western Europe (Germany, Italy, U.K, Spain, France, Rest of Western Europe)
- Eastern Europe (Russia)
- Asia Pacific (China, India, ASEAN, Australia & New Zealand)
- Middle East and Africa (GCC, S. Africa)
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Orphan Drugs Market: Segmentation
Global Orphan Drugs Market can be segmented as following types
By Active Ingridients
- Brentuximab Vedotin
- Carglumic acid
- Histamine hydrochloride
By Disease/ Disorder
- Hodgkin lymphoma
- Paroxysmal nocturnal hemoglobinuria
- Chronic thromboembolic pulmonary hypertension (CTEPH)
- Chronic lymphocytic leukemia
- Chronic Myeloid Leukaemia
- T-cell acute lymphoblastic leukemia (T-ALL)
- Philadelphia chromosome positive chronic myelogenous leukaemia (Ph+ CML)
- Cystic fibrosis (CF)
- acute myeloid leukaemia
- Multiple Myeloma
- Isovaleric acidaemia
- Gaucher disease type 1 (GD1)
- Metastatic medullary thyroid carcinoma
- Advanced gastric cancer or gastro-esophageal junction adenocarcinoma
- Secondary Acute myeloid leukemia (AML)
- Severe hepatic venoocclusive disease (VOD)/ sinusoidal obstructive syndrome (SOS)
By End User
- Ambulatory Surgical Centres
- Detailed overview of parent market
- Changing market dynamics in the industry
- In-depth market segmentation
- Historical, current and projected market size in terms of volume and value
- Recent industry trends and developments
- Competitive landscape
- Strategies of key players and products offered
- Potential and niche segments, geographical regions exhibiting promising growth
- A neutral perspective on market performance
- Must-have information for market players to sustain and enhance their market footprint
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