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希少疾病用医薬品の世界市場-2022-2029

Global Orphan Drugs Market - 2022-2029
出版日: | 発行: DataM Intelligence | ページ情報: 英文 200 Pages | 納期: 約2営業日

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希少疾病用医薬品の世界市場-2022-2029
出版日: 2022年10月13日
発行: DataM Intelligence
ページ情報: 英文 200 Pages
納期: 約2営業日
ご注意事項 :
本レポートは最新情報反映のため適宜更新し、内容構成変更を行う場合があります。ご検討の際はお問い合わせください。
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  • 概要
  • 目次
概要

市場力学

研究開発活動の活発化、希少疾病用医薬品の治療への採用の増加、老年人口の増加が希少疾病用医薬品市場の成長を牽引します。

希少疾患に対する認知度の向上が市場成長の原動力となる見込み

希少疾患とその治療法に関する認知度の高まりが、予測期間中に市場を押し上げると予想されます。Perspective in Clinical Research 2022に掲載された記事によると、FDAなどの規制当局は最近、スタッフの経験やリアルタイムの患者の証言を記録・共有するための「FDA Rare Disease Photo and Video Project」というオンラインプラットフォームを開発したとのことです。患者、介護者、FDA職員との様々な会議を設定することで、患者事務局はこの目標に沿った患者中心主義を支援しています。全米希少疾患機関や欧州希少疾患機関など複数の非営利団体が、それぞれ欧州と米国の規制・研究活動を支援し、患者支援、情報共有、各種患者団体間のネットワーキングなどを通じて患者アドボカシーを推進しています。

さらに、Institute for Clinical and Economic Reviewに掲載された論文によると、2020年時点で開発されている細胞・遺伝子治療は希少疾患向けであり、希少疾患の70％以上が遺伝性疾患であると予測されています。希少疾病用医薬品法が成立して以来、2020年半ばの時点で、FDAは599品目の希少疾病用医薬品を認可しています。ファースト・イン・クラスの希少疾病用医薬品の割合は、非希少疾病用医薬品よりも大幅に高く、米国で認可された希少疾病用医薬品は全体の50％に達しています。パンデミックにもかかわらず、臨床試験活動全体が継続しているのは、業界が混乱に適応し、進行中の研究をサポートするための新しい戦略を構築しているためです。2021年に新たに計画された臨床試験の開始は、2020年から14％、2019年から19％増加し、5,500件が報告されました。

十分な有効性と安全性のデータ収集の難しさが市場成長の足かせに

しかし、少人数で十分な有効性と安全性のデータを収集することの難しさ、開発者とヘルスケアシステムの両方が財政的支援を受けるリスク、ほとんどの疾患に対する理解の浅さなどが、希少疾患の治療法や治療薬の開発における障壁となっています。政府の活動だけでは、希少疾患の商業的可能性が一般的に低いことから生じる市場のギャップを縮めるには十分ではありません。さらに、「超希少疾患」ではなく「希少疾患」を定義する作業定義を決定することは、超希少医薬品の開発を刺激する政策基盤を構築する上で大きな障害のひとつとなるでしょう。これらの要素は、さらなる市場の拡大を制限しています。

COVID-19の影響度分析

COVID-19の登場は、世界の希少疾病用医薬品市場にかなりの影響を与えました。Frontiers in Public Health 2021に掲載された論文によると、特に遺伝子を基盤とする希少疾患は約5～8千種類あり、世界中で約4億人が罹患しています。希少疾病の患者さんやその介護者は、重大なケアの不備やアンメットニーズを報告しています。研究者にとっては、罹患者の大規模な同胞を集めて研究資金を調達することの難しさと費用がすでに問題になっています。誰もが抱える健康への一般的な不安と合わせて、希少疾患の人々はCOVID-19による疑問という二重の負担を抱えているのです。彼らはまた、薬の供給や、定期的に必要な必須作業療法へのアクセスにも直面しています。

一方、2020年にCOVID-19の大流行が予測される中、FDAは32のオーファン指定医薬品と生物学的製品を承認し、希少疾病用医薬品研究開発の進歩を著しく持続させた。医薬品評価研究センター事務局は、特定の希少疾患の販売申請を評価するために、希少疾患ハブを設立しました。希少疾病チームと協力し、希少疾病政策を可能にするための支援と援助を受けた。FDAは、その後の申請件数の増加に対応するため、希少疾病用医薬品近代化パイロットプログラムを開始しました。FDAは、申請の量や複雑さに関係なく、すべての申請を90日の規定時間内に審査することになっています。

世界の希少疾病用医薬品市場レポートでは、約65以上の市場データ表、65以上の図表、200ページ以上（概算）の範囲でのアクセスを提供することになります。

目次

第1章 市場調査手法とスコープ

  • 調査手法
  • 調査目的および調査範囲

第2章 市場の定義と概要

第3章 エグゼクティブサマリー

  • 製品タイプ別市場内訳
  • 治療領域別市場内訳
  • 流通チャネル別市場内訳
  • 地域別市場内訳

第4章 市場力学

  • 市場影響要因
    • 促進要因
    • 糖尿病罹患率の高さ
    • 研究活動の活発化
    • 抑制要因
    • 薬剤費の高騰
    • 影響分析

第5章 産業分析

  • ポーターのファイブフォース分析
  • サプライチェーン分析
  • 規制分析
  • プライシング分析
  • アンメットニーズ分析

第6章 COVID-19分析

  • COVID-19の市場分析
    • COVID-19以前の市場シナリオ
    • COVID-19の現在の市場シナリオ
    • COVID-19の後、あるいは将来のシナリオ
  • COVID-19の中での価格ダイナミクス
  • 需要-供給スペクトラム
  • パンデミック時の市場に関連する政府の取り組み
  • メーカーの戦略的取り組み
  • まとめ

第7章 製品タイプ別

  • 生物学的製剤
  • 非生物学的

第8章 治療領域別

  • オンコロジー
  • 血液疾患
  • 中枢神経系
  • 循環器領域
  • 内分泌
  • 呼吸器系
  • 免疫調整剤
  • 消化器系
  • 筋骨格系
  • 全身性抗感染症薬
  • 皮膚科領域
  • その他

第9章 流通チャネル別

  • 病院内薬局
  • 小売薬局
  • オンライン薬局

第10章 地域別

  • 北米
    • 米国
    • カナダ
    • メキシコ
  • 欧州
    • ドイツ
    • 英国
    • フランス
    • イタリア
    • スペイン
    • その他欧州
  • 南米
    • ブラジル
    • アルゼンチン
    • その他の南米地域
  • アジア太平洋地域
    • 中国
    • インド
    • 日本
    • オーストラリア
    • その他アジア太平洋地域
  • 中東・アフリカ地域

第11章 競合情勢

  • 主な展開と戦略
  • 企業シェア分析
  • 治療タイプ別ベンチマーク

第12章 企業プロファイル

  • AbbVie Inc.
    • 企業概要
    • 治療タイプのポートフォリオと説明
    • 主なハイライト
    • 財務概要
  • Alexion Pharmaceuticals, Inc.
  • Bristol-Myers Squibb Company
  • Celgene Corporation
  • Johnson & Johnson
  • Merck & Co., Inc.
  • Novartis AG
  • Roche Holding AG
  • Sanofi SA
  • Shire plc

第13章 希少疾病用医薬品の世界市場-データM

目次
Product Code: DMPH667

Market Overview

Orphan Drugs Market was valued at US$ xx million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of 11.2% during the forecast period (2022-2029).

Life-threatening or persistently disabling diseases are treated, prevented from occurring, or diagnosed with an orphan drug. It is mostly used to treat rare disorders. Less than 200,000 Americans and five out of every 10,000 people in the European Union are affected by this rare sickness or condition.

Market Dynamics

The rise in research and development activities, rising adoption of orphan drugs for the treatment of diseases, and an increase in the geriatric population will drive the orphan drugs market growth.

The increasing awareness of rare diseases is expected to drive the market growth

The rising awareness of rare diseases and treatment for them is expected to boost the market over the forecast period. As per the article published in Perspective in Clinical Research 2022, regulators like the FDA have recently developed an online platform called the "FDA Rare Disease Photo and Video Project" to record and share staff experiences and real-time patient accounts. By setting up various meetings with patients, caregivers, and FDA employees, the office of patient affairs supports patient-centricity with this goal. Several nonprofit organizations, such as the National Organization for Rare Disorders and the European Organisation for Rare Diseases, support regulatory and research activities in Europe and the United States, respectively, and promote patient advocacy through patient assistance, information sharing, and networking between various patient groups.

Furthermore, per the article published in Institute for Clinical and Economic Review, cell and gene therapies being developed as of 2020 are for rare diseases, and it is predicted that more than 70% of rare diseases are genetic. As of the middle of 2020, the FDA had authorized 599 orphan products since the Orphan Drug Act was passed. The percentage of first-in-class orphan pharmaceuticals is substantially higher than that of nonorphan drugs, with 50% of all orphan drugs authorized in the U.S. Despite the pandemic, overall clinical trial activity has continued because the industry has adjusted to the disruption and created new strategies to support ongoing research. Five thousand five hundred new planned clinical trial launches were reported in 2021, up 14% from 2020 and 19% from 2019.

The difficulty of gathering sufficient efficacy and safety data will hamper the growth of the market

However, the difficulty of gathering sufficient efficacy and safety data in small groups, the risk of financial support for both developers and healthcare systems, and the limited understanding of most diseases are barriers to developing medical treatments or cures for rare diseases. Governments' actions alone are insufficient to narrow the market gap that arises from each rare disease's generally low commercial potential. Additionally, deciding on a working definition of what defines a "rare" as opposed to an "ultra-rare" illness would be one of the major obstacles in building a policy platform to stimulate the development of ultra-rare drugs. These elements are limiting further market expansion.

COVID-19 Impact Analysis

The appearance of COVID-19 considerably impacted the global orphan drugs market. As per the article published in Frontiers in Public Health 2021, there are about five to eight thousand rare diseases, particularly with a genetic basis, affecting approximately 400 million people worldwide. People with rare diseases and their caretakers report significant care insufficiencies and unmet clinical needs. The difficulty and expense of assembling large compatriots of affected individuals for study and conducting research funding is already a problem for researchers. Together with the general anxieties about health concerns everyone else has, people with rare diseases have a double burden of the question due to COVID-19. They also face a supply of medications and the accessibility of essential occupational therapies they need regularly.

On the other hand, with the prevailing COVID-19 pandemic in 2020, the FDA approved 32 orphan-designated drugs and biological products to significantly sustain progress in orphan drug research and development. The Center for Drug Evaluation and Research office created a rare disease hub to assess marketing applications for certain rare diseases. It collaborated with a rare disease team to receive support and assistance for enabling a rare disease policy. FDA launched the orphan drug modernization pilot program to keep pace with the increase in application in subsequent years. The FDA is to review all applications within a stipulated time of 90 days, irrespective of the volume and complexity of the application.

Segment Analysis

The oncology segment is expected to grow at the fastest CAGR during the forecast period (2022-2029)

The oncology segment is expected to boost the market throughout the forecast. In July 2022, The FDA granted orphan drug designation to several therapies in development for oncology indications. MB-106 (Mustang Bio) is a CD20-targeted, autologous chimeric antigen receptor T-cell therapy. The orphan drug designation applies to the use of the agent for the treatment of Waldenstrom macroglobulinemia. Paxalisib (Kazia Therapeutics Limited) is a phosphatidylinositol 3-kinase inhibitor. The designation applies to the agent's treatment of atypical rhabdoid/teratoid tumors, an aggressive and rare childhood brain cancer. VBI-1901 (VBI Vaccines) is a bivalent gB/pp65 immunotherapeutic vaccine candidate. The designation applies to the use of this agent for the treatment of glioblastoma.

In addition, in January 2021, the U.S. Food and Medication Administration (FDA) granted PVSRIPO (stage IIB-IV) as an orphan drug for the treatment of advanced melanoma by Istari Oncology, Inc. . A patient's adaptive and innate immune systems are activated by PVSRIPO, a novel viral immunotherapy based on the Sabin type 1 polio vaccine, to promote an anti-tumor response and establish long-term immunologic memory to the prevention of cancer recurrence.

Geographical Analysis

North America region holds the largest market share of the global orphan drugs market

North America dominates the market for orphan drugs and is expected to show a similar trend over the forecast period. It is anticipated to hold a significant market size over the forecast period (2022-2029) owing to the advanced healthcare system, government initiatives and the presence of many market players. The FDA Office of Orphan Products Development approved orphan drug designation for novel drugs and biologics that are used for the safe and effective treatment, diagnosis and prevention of rare diseases or disorders that suffer fewer than 200,000 people in the United States region. The designation allows key players to qualify for various incentives, including tax credits for qualified clinical trials and, upon regulatory approval, 7 years of market exclusivity. In addition, the U.S. remains the country with the earliest and highest number of launches and among the 72 NASs launched in 2021, a record 44 (over 60%) were characterized by the FDA as first-in-class, and more than half (40) carried an orphan drug designation indicating their use for patients with rare diseases.

Moreover, in September 2020, AbbVie's experimental therapy for patients with spinal cord injury, elezanumab (ABT-555), was given Orphan drugs and Fast Track designations by the USFDA. A monoclonal antibody of the human immunoglobulin (Ig)G1 isotype called elezanumab binds only to the repellent guidance molecule A (RGMa).

Competitive Landscape

The orphan drugs market is a moderately competitive presence of local and global companies. Some of the key players which are contributing to the growth of the market are AbbVie Inc., Alexion Pharmaceuticals, Inc., Bristol-Myers Squibb Company, Celgene Corporation, Johnson & Johnson, Merck & Co., Inc., Novartis AG, Roche Holding AG, Sanofi SA, and Shire plc among others. The major players are adopting several growth strategies, such as product launches, acquisitions, and collaborations, contributing to the expansion of the orphan drugs market globally.

For instance,

  • In June 2020, Chiasma, Inc. announced that the U.S. Food and Drug Administration (FDA) approved MYCAPSSA (octreotide) capsules for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide.

AbbVie Inc

Overview:

AbbVie is a research-based biopharmaceutical organization founded in 2013 and headquartered in the United States. It is developing innovative and advanced therapies to meet the requirement of complex and serious diseases.

Product Portfolio:

HUMIRA (adalimumab): It is used to reduce symptoms of Moderate to severe rheumatoid arthritis (RA) in adults, Psoriatic arthritis (PsA) in adults, and ulcerative colitis in adults.

The global orphan drugs market report would provide access to approximately 65+ market data tables, 65+ figures, and in the range of 200+ (approximate) pages.

Table of Contents

1. Market Methodology and Scope

  • 1.1. Research Methodology
  • 1.2. Research Objective and Scope of the Report

2. Market Definition and Overview

3. Executive Summary

  • 3.1. Market Snippet By Product Type
  • 3.2. Market Snippet By Therapy Area
  • 3.3. Market Snippet By Distribution Channel
  • 3.4. Market Snippet by Region

4. Market Dynamics

  • 4.1. Market Impacting Factors
    • 4.1.1. Drivers
    • 4.1.2. High prevalence of diabetes disease
    • 4.1.3. Rising research activities
    • 4.1.4. Restraints:
    • 4.1.5. High cost of drugs
    • 4.1.6. Impact Analysis

5. Industry Analysis

  • 5.1. Porter's Five Forces Analysis
  • 5.2. Supply Chain Analysis
  • 5.3. Regulatory Analysis
  • 5.4. Pricing Analysis
  • 5.5. Unmet Needs

6. COVID-19 Analysis

  • 6.1. Analysis of Covid-19 on the Market
    • 6.1.1. Before COVID-19 Market Scenario
    • 6.1.2. Present COVID-19 Market Scenario
    • 6.1.3. After COVID-19 or Future Scenario
  • 6.2. Pricing Dynamics Amid Covid-19
  • 6.3. Demand-Supply Spectrum
  • 6.4. Government Initiatives Related to the Market During Pandemic
  • 6.5. Manufacturer's Strategic Initiatives
  • 6.6. Conclusion

7. By Product Type

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Product Type
    • 7.1.2. Market Attractiveness Index, By Product Type
  • 7.2. Biological*
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 7.3. Non-biological

8. By Therapy Area

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Area
    • 8.1.2. Market Attractiveness Index, By Therapy Area
  • 8.2. Oncology*
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 8.3. Hematology Diseases
  • 8.4. Central Nervous System
  • 8.5. Cardiovascular
  • 8.6. Endocrine
  • 8.7. Respiratory
  • 8.8. Immunomodulators
  • 8.9. Gastro-Intestinal
  • 8.10. Musculoskeletal
  • 8.11. Systemic Anti-infectives
  • 8.12. Dermatology
  • 8.13. Others

9. By Distribution Channel

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 9.1.2. Market Attractiveness Index, By Distribution Channel
  • 9.2. Hospital Pharmacies*
    • 9.2.1. Introduction
    • 9.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 9.3. Retail Pharmacies
  • 9.4. Online Pharmacies

10. By Region

  • 10.1. Introduction
    • 10.1.1. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029, By Region
    • 10.1.2. Market Attractiveness Index, By Region
  • 10.2. North America
    • 10.2.1. Introduction
    • 10.2.2. Key Region-Specific Dynamics
    • 10.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Product Type
    • 10.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Area
    • 10.2.5.
    • 10.2.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 10.2.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 10.2.7.1. The U.S.
      • 10.2.7.2. Canada
      • 10.2.7.3. Mexico
  • 10.3. Europe
    • 10.3.1. Introduction
    • 10.3.2. Key Region-Specific Dynamics
    • 10.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Product Type
    • 10.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Area
    • 10.3.5.
    • 10.3.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 10.3.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 10.3.7.1. Germany
      • 10.3.7.2. U.K.
      • 10.3.7.3. France
      • 10.3.7.4. Italy
      • 10.3.7.5. Spain
      • 10.3.7.6. Rest of Europe
  • 10.4. South America
    • 10.4.1. Introduction
    • 10.4.2. Key Region-Specific Dynamics
    • 10.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Product Type
    • 10.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Area
    • 10.4.5.
    • 10.4.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 10.4.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 10.4.7.1. Brazil
      • 10.4.7.2. Argentina
      • 10.4.7.3. Rest of South America
  • 10.5. Asia Pacific
    • 10.5.1. Introduction
    • 10.5.2. Key Region-Specific Dynamics
    • 10.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Product Type
    • 10.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Area
    • 10.5.5.
    • 10.5.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 10.5.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 10.5.7.1. China
      • 10.5.7.2. India
      • 10.5.7.3. Japan
      • 10.5.7.4. Australia
      • 10.5.7.5. Rest of Asia Pacific
  • 10.6. Middle East and Africa
    • 10.6.1. Introduction
    • 10.6.2. Key Region-Specific Dynamics
    • 10.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Product Type
    • 10.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Area
    • 10.6.5.
    • 10.6.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel

11. Competitive Landscape

  • 11.1. Key Developments and Strategies
  • 11.2. Company Share Analysis
  • 11.3. Treatment type Benchmarking

12. Company Profiles

  • 12.1. AbbVie Inc.*
    • 12.1.1. Company Overview
    • 12.1.2. Treatment type Portfolio and Description
    • 12.1.3. Key Highlights
    • 12.1.4. Financial Overview
  • 12.2. Alexion Pharmaceuticals, Inc.
  • 12.3. Bristol-Myers Squibb Company
  • 12.4. Celgene Corporation
  • 12.5. Johnson & Johnson
  • 12.6. Merck & Co., Inc.
  • 12.7. Novartis AG
  • 12.8. Roche Holding AG
  • 12.9. Sanofi SA
  • 12.10. Shire plc

LIST NOT EXHAUSTIVE

13. Global Orphan Drugs Market- DataM

  • 13.1. Appendix
  • 13.2. About Us and Services
  • 13.3. Contact Us