希少疾病用医薬品の世界市場：2023-2030年

世界の希少疾病用医薬品市場は予測期間（2023-2030年）にCAGR 11.8%で成長すると予測されます。同市場の成長は、全世界における希少医薬品の意義と目的に起因しています。希少医薬品は、特定の希少な病状を治療するために開発された薬理学的化合物です。前述のような希少疾患を患う患者は、通常ごく少数です。これらの医薬品は、患者数が少ないため、政府の援助や優遇措置がなければ、経済的に開発不可能な場合があります。BMJ誌によると、2023年5月、特定の希少疾患を治療するために開発されたオーファンドラッグと呼ばれる薬理学的製剤が承認されます。希少疾病の患者数が少ないことに加え、オーファンドラッグは政府の援助なしには商業的に開発することができないです。希少疾病用医薬品は、ジェネリック医薬品を除く全世界の医療用医薬品売上高の20％を占め、年間市場成長率は11.7％（非希少疾病用医薬品は5.9％）と予測されています。しかし、遺伝学と精密医療の発展によってアクセスしやすくなった希少疾病用医薬品の数を増やすために、ますます限定された疾病用語が使用されるようになり、懸念が高まっています。さらに、がんの世界の流行により、年間1,400万人の新規患者と800万人の死亡者が報告されています。革新的で強力ながん治療薬に対する需要は急速に高まっており、2040年までにその数は2,200万人にまで増加すると推定されています。結局のところ、消費者層が限られているため、珍しいがんの治療法は資金獲得に苦労することがあります。

セグメント別の展望

循環器領域は希少疾病用医薬品の世界市場で大きなシェアを占めると予想される

疾患タイプ別では、心血管サブセグメントが世界の希少疾病用医薬品市場でかなりのシェアを占めると予想されています。このセグメントの成長は、心血管の健康に対する配慮に起因しています。ナトリウム消費量の増加は、いくつかの心血管危険因子、特に高血圧（高血圧）と直接的な相関関係があります。XywavTMのナトリウム含有量が少ないことは、ナトリウム過剰摂取に関連する広範な健康上の懸念と一致していることは興味深いです。例えば、2021年6月、ジャズ・ファーマシューティカルズplc社は、7歳以上のナルコレプシー患者におけるカタプレキシーまたは過度の日中の眠気（EDS）の治療薬として、XywavTM（カルシウム、マグネシウム、カリウム、オキシベートナトリウム）内用液を発売しました。米国食品医薬品局（FDA）より7年間の希少疾病用医薬品としての独占的使用が認められています。オキシベート製剤であるXywavのナトリウム含有量は、オキシベートナトリウムの推奨用量範囲である6～9グラム（およそ1,000～1,500mg／夜）よりも92％低いです。

地域別展望

希少疾病用医薬品の世界市場は、北米（米国、カナダ）、欧州（英国、イタリア、スペイン、ドイツ、フランス、その他欧州地域）、アジア太平洋地域（インド、中国、日本、韓国、その他アジア地域）、世界のその他の地域（中東とアフリカ、ラテンアメリカ）を含む地域別にさらに細分化されます。なかでもアジア太平洋地域は、地域医療や腫瘍センターの数を増やすための政府の取り組みが活発化していることから、新興国における希少疾病用医薬品市場の拡大が期待され、世界市場で突出したシェアを占めると予想されます。

欧州地域は世界の希少疾病用医薬品市場において著しいCAGRで成長する見込み

全地域の中で、欧州地域は予測期間中にかなりのCAGRで成長すると予測されています。同地域の成長は、同地域全体で幅広い希少疾病が増加していることに起因しています。これまでに確認された7,000以上の希少疾病の大部分は遺伝的基盤を有しています。これらの疾患のいくつかは有害であると考えられているため、効果的な治療に対する緊急性が高まっています。ファーマシューティカル・テクノロジーによると、2022年7月現在、7,000以上の希少疾病が存在すると考えられており、その大部分は遺伝的なもので、その多くは生命を脅かすと考えられています。欧州連合（EU）が定義する希少疾病は、かなりの有病率にもかかわらず、約2,000人に1人が罹患しています。

目次

第1章 レポート概要

• 業界の現状分析と成長ポテンシャルの展望
• 調査方法とツール
• 市場内訳
  • セグメント別
  • 地域別

第2章 市場概要と洞察

• 調査範囲
• アナリストの洞察と現在の市場動向
  • 主な調査結果
  • 推奨事項
  • 結論

第3章 競合情勢

• 主要企業分析
• AbbVie Inc.
  • 概要
  • 財務分析
  • SWOT分析
  • 最近の動向
• Alexion Pharmaceuticals, Inc.
  • 会社概要
  • 財務分析
  • SWOT分析
  • 最近の動向
• Amgen Inc.
  • 概要
  • 財務分析
  • SWOT分析
  • 最近の動向
• 主要戦略分析

第4章 市場セグメンテーション

• 希少疾病用医薬品の世界市場：薬剤タイプ別
  • 生物学的製剤
  • 非生物学的製剤
• 希少疾病用医薬品の世界市場：疾患タイプ別
  • オンコロジー
  • 血液
  • 神経
  • 心血管
  • その他（自己免疫疾患、希少血液疾患）
• 希少疾病用医薬品の世界市場：流通チャネル別
  • 病院薬局
  • 小売薬局
  • オンライン薬局
  • その他（専門薬局、患者への直接販売）

第5章 地域別分析

• 北米
  • 米国
  • カナダ
• 欧州
  • 英国
  • ドイツ
  • イタリア
  • スペイン
  • フランス
  • その他欧州
• アジア太平洋
  • 中国
  • インド
  • 日本
  • 韓国
  • その他アジア太平洋地域
• 世界のその他の地域

第6章 企業プロファイル

• AstraZeneca.
• At F. Hoffmann-La Roche Ltd
• Bayer AG
• Bristol-Myers Squibb India Pvt. Ltd.
• Daiichi Sankyo Co. Ltd.
• GlaxoSmithKline plc
• Johnson & Johnson Pvt. Ltd.
• Novartis AG
• Pfizer Inc.
• Sanofi
• Takeda Pharmaceutical Co. Ltd.

LIST OF TABLES

• 1. GLOBAL ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DRUG TYPE, 2022-2030 ($ MILLION)
• 2. GLOBAL BIOLOGICAL ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 3. GLOBAL NON-BIOLOGICAL ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 4. GLOBAL ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DISEASE TYPE, 2022-2030 ($ MILLION)
• 5. GLOBAL ORPHAN DRUGS IN ONCOLOGY MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 6. GLOBAL ORPHAN DRUGS IN HEMATOLOGY MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 7. GLOBAL ORPHAN DRUGS IN NEUROLOGY MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 8. GLOBAL ORPHAN DRUGS IN CARDIOVASCULAR MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 9. GLOBAL ORPHAN DRUGS IN OTHER DISEASE TYPES MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 10. GLOBAL ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DISTRIBUTION CHANNEL, 2022-2030 ($ MILLION)
• 11. GLOBAL ORPHAN DRUGS BY HOSPITAL PHARMACY MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 12. GLOBAL ORPHAN DRUGS BY RETAIL PHARMACY MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 13. GLOBAL ORPHAN DRUGS BY ONLINE PHARMACY MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 14. GLOBAL ORPHAN DRUGS BY OTHER DISTRIBUTION CHANNELS MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 15. GLOBAL ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY REGION, 2022-2030 ($ MILLION)
• 16. NORTH AMERICAN ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY COUNTRY, 2022-2030 ($ MILLION)
• 17. NORTH AMERICAN ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DRUG TYPE, 2022-2030 ($ MILLION)
• 18. NORTH AMERICAN ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DISEASE TYPE, 2022-2030 ($ MILLION)
• 19. NORTH AMERICAN ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DISTRIBUTION CHANNEL, 2022-2030 ($ MILLION)
• 20. EUROPEAN ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY COUNTRY, 2022-2030 ($ MILLION)
• 21. EUROPEAN ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DRUG TYPE, 2022-2030 ($ MILLION)
• 22. EUROPEAN ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DISEASE TYPE , 2022-2030 ($ MILLION)
• 23. EUROPEAN ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DISTRIBUTION CHANNEL, 2022-2030 ($ MILLION)
• 24. ASIA- PACIFIC ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY COUNTRY, 2022-2030 ($ MILLION)
• 25. ASIA- PACIFIC ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DRUG TYPE, 2022-2030 ($ MILLION)
• 26. ASIA- PACIFIC ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DISEASE TYPE, 2022-2030 ($ MILLION)
• 27. ASIA- PACIFIC ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DISTRIBUTION CHANNEL, 2022-2030 ($ MILLION)
• 28. REST OF THE WORLD ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DRUG TYPE, 2022-2030 ($ MILLION)
• 29. REST OF THE WORLD ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DISEASE TYPE, 2022-2030 ($ MILLION)
• 30. REST OF THE WORLD ORPHAN DRUGS MARKET RESEARCH AND ANALYSIS BY DISTRIBUTION CHANNEL , 2022-2030 ($ MILLION)

LIST OF FIGURES

• 1. GLOBAL ORPHAN DRUGS MARKET SHAREBY DRUG TYPE, 2022 VS 2030 (%)
• 2. GLOBAL BIOLOGICAL ORPHAN DRUGS MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 3. GLOBAL NON-BIOLOGICAL ORPHAN DRUGS MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 4. GLOBAL ORPHAN DRUGS MARKET SHAREBY DISEASE TYPE, 2022 VS 2030 (%)
• 5. GLOBAL ORPHAN DRUGS IN ONCOLOGY MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 6. GLOBAL ORPHAN DRUGS IN HEMATOLOGY MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 7. GLOBAL ORPHAN DRUGS IN NEUROLOGY MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 8. GLOBAL ORPHAN DRUGS IN CARDIOVASCULAR MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 9. GLOBAL ORPHAN DRUGS IN OTHER DISEASE TYPES MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 10. GLOBAL ORPHAN DRUGS MARKET SHAREBY DISTRIBUTION CHANNEL, 2022 VS 2030 (%)
• 11. GLOBAL ORPHAN DRUGS BY HOSPITAL PHARMACY MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 12. GLOBAL ORPHAN DRUGS BY RETAIL PHARMACY MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 13. GLOBAL ORPHAN DRUGS BY ONLINE PHARMACY MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 14. GLOBAL ORPHAN DRUGS FOR OTHER DISTRIBUTION CHANNELS MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 15. GLOBAL ORPHAN DRUGS MARKET SHAREBY REGION, 2022 VS 2030 (%)
• 16. US ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 17. CANADA ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 18. UK ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 19. FRANCE ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 20. GERMANY ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 21. ITALY ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 22. SPAIN ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 23. REST OF EUROPE ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 24. INDIA ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 25. CHINA ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 26. JAPAN ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 27. SOUTH KOREA ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 28. REST OF ASIA-PACIFIC ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)
• 29. REST OF THE WORLD ORPHAN DRUGS MARKET SIZE, 2022-2030 ($ MILLION)

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.

Table of Contents

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.