Orphan drugs are pharmaceutical products developed specifically to treat rare diseases or conditions, often referred to as orphan diseases. These conditions typically affect a small percentage of the population, making the development of treatments less financially attractive for pharmaceutical companies. To encourage the development of orphan drugs, governments often provide incentives such as tax credits, market exclusivity, and grants. These drugs play a crucial role in addressing unmet medical needs for patients with rare disorders.
Orphan Drugs Market Overview
The Global Orphan Drugs Market is estimated to reach a CAGR of 11.2% during the forecast period (2024-2031).
Orphan drugs are used to treat, prevent, or diagnose life-threatening or chronically debilitating diseases, particularly rare disorders. These rare conditions affect fewer than 200,000 people in the U.S. and fewer than 5 in 10,000 individuals in the European Union.
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Orphan Drugs Market Dynamics
The growing awareness of rare diseases is expected to fuel market growth in the coming years. According to a 2022 article in Perspectives in Clinical Research, regulatory bodies like the FDA have recently launched an online platform, the "FDA Rare Disease Photo and Video Project," to document and share staff experiences and real-time patient stories. The FDA's Office of Patient Affairs promotes patient-centric initiatives by organizing meetings with patients, caregivers, and FDA staff. Additionally, nonprofit organizations such as the National Organization for Rare Disorders in the U.S. and the European Organisation for Rare Diseases in Europe actively support regulatory efforts and research, while also advocating for patients through assistance programs, information sharing, and fostering connections among patient groups.
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The Orphan Drugs Market is segmented
by Product Type: Biological, Non-Biological
by Therapy Area: Oncology, Hematology Diseases, Central Nervous System, Cardiovascular, Endocrine, Respiratory, Immunomodulators, Gastro-Intestinal, Musculoskeletal, Systemic Anti-Infectives, Dermatology, Others
by Region: North America, Latin America, Europe, Asia Pacific, Middle East, and Africa
Orphan Drugs Companies
· AbbVie Inc.
· Alexion Pharmaceuticals, Inc.
· Bristol-Myers Squibb Company
· Celgene Corporation
· Johnson & Johnson
· Merck & Co., Inc.
· Novartis AG
· Roche Holding AG
· Sanofi SA
· Shire plc
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