The global rare disease drug market is expected to grow at a significant CAGR during the forecast period. Any disease that affects a small fraction of the population is considered rare. An orphan disease is a rare ailment that lacks a large enough market in some parts of the world to obtain support and resources for identifying cures for it, unless the government grants economically beneficial conditions for developing and selling such treatments. Orphan medications are those that have been developed or sold for medical reasons. The FDA approved 32 new medicines and biologics with orphan drug designation in 2020. Clinical trials for orphan medicine therapies are substantially less expensive than those for other diseases, owing to the fact that trial sizes are naturally smaller than those for more common diseases with a bigger patient population.
The key companies operating in the industry are highly inclined towards the adoption of different growth strategies including partnerships and collaborations, mergers and acquisitions, geographical expansion, new product launches to remain competitive in the marketplace. For instance, in November 2021, for rare disease patients, MedGenome, South Asia’s leading genomics research and diagnostics company, partnered with Emmes, a global, full-service Clinical Research Organization (CRO) dedicated to support the advancement of public health and biopharmaceutical innovation through human genomics. The two firms would work together to establish unique disease registries that would include patients’ epidemiologic, phenotypic, and genetic data to improve trial design, promote patient recruitment, and create standard control arms for advanced statistical models of treatment effect. Hemophilia, Duchenne muscular dystrophy, muscular atrophies, and retinitis pigmentosa are among the first rare disorders to be addressed by the Emmes/MedGenome collaboration. In addition to this, Emmes opened a new rare disease centre in November 2021, combining its biostatistics, data management, and clinical research skills with Orphan Reach’s rare disease patient and clinical trial experience. In May 2021, Emmes purchased Orphan Reach, a company established in the UK.
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Market Coverage
- The market number available for – 2022-2030
- Base year- 2022
- Forecast period- 2023-2030
Segment Covered-
- By Drug Type
- By Therapeutic Area
Regions covered-
- North America
- Europe
- Asia-Pacific
- Rest of the World
Competitive Landscape: Novartis International AG, Pfizer Inc., and F. Hoffmann-La Roche Ltd. among others.
Rare Disease Drug Market Report by Segment
By Drug Type
- Biologics
- Non-Biologics
By Therapeutic Area
- Cancer
- Blood Related Disorders
- Central Nervous System (CNS)
- Respiratory Disorders
- Musculoskeletal Disorders
- Cardiovascular Disorders
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Rare Disease Drug Market Report by Region
North America
- United States
- Canada
Europe
- UK
- Germany
- Spain
- France
- Italy
- Rest of Europe
Asia-Pacific
- India
- China
- Japan
- South Korea
- Rest of APAC
Rest of the World
- Latin America
- Middle East & Africa
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