Global Orphan Drugs Market 2023-2030

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List of Tables

Title: Global Orphan Drugs Market Size, Share & Trends Analysis Report by Drug Type (Biological, and Non-biological), by Disease Type (Oncology, Hematology, Neurology, Cardiovascular, and Others), by Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Online Pharmacy, and Others),Forecast Period (2023-2030).

The global orphan drugs market is anticipated to grow at a CAGR of 11.8% during the Forecast Period (2023-2030). The market's growth is attributed to significance and purpose of orphan drugs across the globe. Orphan pharmaceuticals are pharmacological compounds developed to treat particular rare medical conditions. A minimal number of patients normally suffer the aforementioned rare disorders. These medications can be financially unviable to develop without government assistance and incentives owing to their small patient bases. According to the BMJ, in May 2023, a pharmacological agent referred to as an orphan drug that has been developed to treat specific rare medical diseases. In addition to the tiny number of patients with the diseases, an orphan medicine cannot be made commercially viable to develop without government aid. Orphan medications are projected to account for 20% of all prescription drug sales globally (excluding generics), with an annual market growth rate of 11.7% vs 5.9% for non-orphan drugs. The use of increasingly limited disease terms to provide orphan status to a growing number of medications, rendered accessible by developments in genetics and precision medicine, raised concerns, though. Furthermore, 14 million new cases and 8 million fatalities from cancer are reported annually owing to its global prevalence. The demand for innovative and potent cancer treatments is increasing rapidly as estimates indicate that the number of cases can rise to 22 million by the year 2040. Ultimately, owing to their limited consumer base, treatments for uncommon cancers sometimes struggle to acquire the funding they demand.

Segmental Outlook

The global orphan drugs market is segmented on the drug type, disease type, and distribution channel. Based on the disease type, the market is sub-segmented into biological, and non-biological. Based on the disease type, the market is sub-segmented into oncology, hematology, neurology, cardiovascular, and others. Furthermore, on the basis of distribution channel, the market is sub-segmented into hospital pharmacy, retail pharmacy, online pharmacy, and others. The oncology subcategory is expected to capture a significant portion of the market share within the disease type segment. This is attributed to the increase in the prevalence of oncological diseases, increased awareness of cancer, early cancer screening, and the availability of oncological orphan medications for cancer treatment.

The Cardiovascular Sub-Segment is Anticipated to Hold a Considerable Share of the Global Orphan Drugs Market

Among the disease type, the cardiovascular sub-segment is expected to hold a considerable share of the global orphan drugs market. The segmental growth is attributed to the considerations for cardiovascular health. Increased sodium consumption has a direct correlation with several cardiovascular risk factors, particularly hypertension (high blood pressure). It is interesting that XywavTM has less sodium that coincides with broader health concerns related to consuming excessive sodium. For instance, in June 2021, Jazz Pharmaceuticals plc, launched the XywavTM (calcium, magnesium, potassium, and sodium oxybates) oral solution for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. The U.S. Food and Drug Administration (FDA) has recognized seven years of Orphan Drug Exclusivity. The sodium content of Xywav, an oxybate product, is 92% lower than that of sodium oxybate in the recommended dosage range of 6 to 9 grams, or roughly 1,000 to 1,500 mg/night.

Regional Outlook

The global orphan drugs market is further segmented based on geography including North America (the US, and Canada), Europe (UK, Italy, Spain, Germany, France, and the Rest of Europe), Asia-Pacific (India, China, Japan, South Korea, and Rest of Asia), and the Rest of the World (the Middle East & Africa, and Latin America). Among these, Asia-Pacific is anticipated to hold a prominent share of the market across the globe, owing to the increased government initiatives to boost the number of community health and oncology centers are anticipated to expand the market for orphan medications in emerging nations.

The Europe Region is Expected to Grow at a Significant CAGR in the Global Orphan Drugs Market

Among all regions, the Europe regions is anticipated to grow at a considerable CAGR over the forecast period. Regional growth is attributed to the rising wide range of orphan diseases across the region. The majority of the more than 7,000 orphan diseases that have been identified so far have genetic bases. There is an increased urgency for effective treatments owing to several of these diseases are thought to be harmful. According to Pharmaceutical Technology, in July 2022, More than 7,000 orphan diseases are believed to exist at the moment, the majority of that are genetic in origin and many of that are regarded as life-threatening. Orphan diseases, as defined by the European Union, afflict approximately in 2,000 individuals, despite their significant prevalence.

Market Players Outlook

The major companies serving the orphan drugs market include: AstraZeneca., At F. Hoffmann-La Roche Ltd, Bayer AG, Bristol-Myers Squibb India Pvt. Ltd., Daiichi Sankyo Co. Ltd., and others. The market players are considerably contributing to the market growth by the adoption of various strategies including mergers and acquisitions, partnerships, collaborations, funding, and new product launches, to stay competitive in the market. For instance in November 2021, Emmes launched its new center, Orphan Reach as a unique 'rare CRO'. The company renowned for proficiency in public health research across the public sector and biopharmaceutical industry has been included into the new rare diseases center.

The Report Covers:

Market value data analysis of 2022 and forecast to 2030.
Annualized market revenues ($ million) for each market segment.
Country-wise analysis of major geographical regions.
Key companies operating in the global orphan drugs market. Based on the availability of data, information related to new product launches, and relevant news is also available in the report.
Analysis of business strategies by identifying the key market segments positioned for strong growth in the future.
Analysis of market-entry and market expansion strategies.
Competitive strategies by identifying 'who-stands-where' in the market.

Product Code: OMR2027794

1. Report Summary

Current Industry Analysis and Growth Potential Outlook
1.1. Research Methods and Tools
1.2. Market Breakdown
- 1.2.1. By Segments
- 1.2.2. By Region

2. Market Overview and Insights

2.1. Scope of the Report
2.2. Analyst Insight & Current Market Trends
- 2.2.1. Key Findings
- 2.2.2. Recommendations
- 2.2.3. Conclusion

3. Competitive Landscape

3.1. Key Company Analysis
3.2. AbbVie Inc.
- 3.2.1. Overview
- 3.2.2. Financial Analysis
- 3.2.3. SWOT Analysis
- 3.2.4. Recent Developments
3.3. Alexion Pharmaceuticals, Inc.
- 3.3.1. Overview
- 3.3.2. Financial Analysis
- 3.3.3. SWOT Analysis
- 3.3.4. Recent Developments
3.4. Amgen Inc.
- 3.4.1. Overview
- 3.4.2. Financial Analysis
- 3.4.3. SWOT Analysis
- 3.4.4. Recent Developments
3.5. Key Strategy Analysis

4. Market Segmentation

4.1. Global Orphan Drugs Market by Drug Type
- 4.1.1. Biological
- 4.1.2. Non-biological
4.2. Global Orphan Drugs Market by Disease Type
- 4.2.1. Oncology
- 4.2.2. Hematology
- 4.2.3. Neurology
- 4.2.4. Cardiovascular
- 4.2.5. Others (Autoimmune Diseases, and Rare Blood Disorders)
4.3. Global Orphan Drugs Market by Distribution Channel
- 4.3.1. Hospital Pharmacy
- 4.3.2. Retail Pharmacy
- 4.3.3. Online Pharmacy
- 4.3.4. Others (Specialty Pharmacies, and Direct-to-Patient Distribution)

5. Regional Analysis

5.1. North America
- 5.1.1. United States
- 5.1.2. Canada
5.2. Europe
- 5.2.1. UK
- 5.2.2. Germany
- 5.2.3. Italy
- 5.2.4. Spain
- 5.2.5. France
- 5.2.6. Rest of Europe
5.3. Asia-Pacific
- 5.3.1. China
- 5.3.2. India
- 5.3.3. Japan
- 5.3.4. South Korea
- 5.3.5. Rest of Asia-Pacific
5.4. Rest of the World

6. Company Profiles

6.1. AstraZeneca.
6.2. At F. Hoffmann-La Roche Ltd
6.3. Bayer AG
6.4. Bristol-Myers Squibb India Pvt. Ltd.
6.5. Daiichi Sankyo Co. Ltd.
6.6. GlaxoSmithKline plc
6.7. Johnson & Johnson Pvt. Ltd.
6.8. Novartis AG
6.9. Pfizer Inc.
6.10. Sanofi
6.11. Takeda Pharmaceutical Co. Ltd.