查看購物車
  • Simplified Chinese
  • English
  • Japanese
  • Korean
封面
市場調查報告書
商品編碼
1136911

全球孤兒藥市場-2022-2029

Global Orphan Drugs Market - 2022-2029
出版日期: | 出版商: DataM Intelligence | 英文 200 Pages | 商品交期: 約2個工作天內

價格

※ 本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

簡介目錄

市場動態

研發活動的增加、孤兒藥治療採用的增加以及老年人口的增加正在推動孤兒藥市場的增長。

罕見病意識的提高有望推動市場增長

在預測期內，人們對罕見病及其治療的認識不斷提高，預計將提振市場。根據發表在 Perspective in Clinical Research 2022 上的一篇文章，FDA 等監管機構最近推出了一個名為 FDA 罕見病照片和視頻項目的在線平台，用於記錄和分享員工經驗和實時患者證詞。發達。通過安排與患者、護理人員和 FDA 工作人員的各種會議，患者事務辦公室支持以患者為中心以實現這一目標。包括國家罕見病局和歐洲罕見病局在內的幾個非營利組織分別支持歐洲和美國的監管和研究活動，並通過患者宣傳、信息共享和不同患者群體之間的網絡來促進患者宣傳. 增加。

此外，據臨床與經濟評論研究所發表的一篇論文顯示，截至2020年正在開發的細胞和基因療法是針對罕見病的，預計70%以上的罕見病是遺傳病。自《孤兒藥法案》頒布以來，截至 2020 年年中，FDA 已批准了 599 種孤兒藥。 First-in-class孤兒藥的比例明顯高於非孤兒藥，50%的孤兒藥在美國獲得許可。儘管大流行，但隨著行業適應中斷並製定新策略以支持正在進行的研究，總體臨床試驗活動仍在繼續。計劃於 2021 年開始的新臨床試驗比 2020 年增加 14%，比 2019 年增加 19%，報告了 5,500 項。

難以收集足夠的療效和安全性數據阻礙市場增長

但是，難以收集到足夠數量的療效和安全性數據，開發者和醫療保健系統面臨資金支持的風險，以及對大多數疾病的了解不足。這些都阻礙了罕見病治療方法和藥物的開發疾病。僅靠政府的行動不足以彌補罕見病商業潛力普遍較低導致的市場缺口。此外，確定一個定義“罕見疾病”而不是“超罕見疾病”的工作定義將是建立政策基礎以刺激超罕見藥物開發的主要障礙之一。這些因素限制了進一步的市場擴張。

COVID-19 影響分析

COVID-19 的出現對全球孤兒藥市場產生了相當大的影響。根據發表在《2021 年公共衛生前沿》上的一篇論文，大約有 5,000 到 8,000 種罕見疾病，尤其是那些基於基因的疾病，影響著全球約 4 億人。患有罕見疾病的患者及其護理人員報告了顯著的護理差距和未滿足的需求。對於研究人員來說，招募大量受影響個體的同胞資助研究的難度和費用已經是一個問題。由於 COVID-19 以及每個人都存在的一般健康問題，罕見病患者承擔著雙重問題。他們還面臨著藥物供應和獲得他們需要的基本職業治療的問題。

同時，FDA 已批准 32 種孤兒藥和生物製劑，在預計 2020 年 COVID-19 大流行期間顯著維持孤兒藥研發的進展。藥物評價與研究中心秘書處成立了罕見病中心，以評估特定罕見病的上市申請。與罕見病團隊合作並獲得支持和幫助以實施罕見病政策。 FDA 啟動了孤兒藥現代化試點計劃，以滿足隨後申請量的增加。預計 FDA 將在 90 天內審查所有申請，無論申請的數量或複雜程度如何。

全球孤兒藥市場報告將提供對大約 65 多個市場數據表、65 多個圖表和 200 多頁（大約）的訪問。

內容

第一章市場研究方法和範圍

  • 調查方法
  • 調查目的和範圍

第 2 章市場定義和概述

第 3 章執行摘要

  • 按產品類型劃分的市場細分
  • 按治療區域劃分的市場細分
  • 按分銷渠道劃分的市場細分
  • 按地區劃分的市場細分

第 4 章市場動態

  • 市場影響因素
    • 驅動程序
    • 糖尿病高發
    • 啟動研究活動
    • 約束因素
    • 飆升的藥品費用
    • 影響分析

第五章行業分析

  • 波特五力分析
  • 供應鏈分析
  • 監管分析
  • 定價分析
  • 未滿足的需求分析

第 6 章 COVID-19 分析

  • COVID-19 的市場分析
    • COVID-19 之前的市場情景
    • COVID-19 的當前市場情景
    • COVID-19 後或未來情景
  • COVID-19 期間的價格動態
  • 供需範圍
  • 大流行期間與市場相關的政府舉措
  • 製造商的戰略舉措
  • 總結

第 7 章按產品類型

  • 生物製品
  • 非生物

第 8 章按治療領域

  • 腫瘤學
  • 血液病
  • 中樞神經系統
  • 循環區
  • 內分泌
  • 呼吸系統
  • 免疫調節劑
  • 消化系統
  • 肌肉骨骼系統
  • 全身性抗感染藥
  • 皮膚科
  • 其他

第 9 章分銷渠道

  • 醫院藥房
  • 零售藥房
  • 在線藥店

第 10 章按地區劃分

  • 北美
    • 美國
    • 加拿大
    • 墨西哥
  • 歐洲
    • 德國
    • 英國
    • 法國
    • 意大利
    • 西班牙
    • 其他歐洲
  • 南美洲
    • 巴西
    • 阿根廷
    • 其他南美洲
  • 亞太地區
    • 中國
    • 印度
    • 日本
    • 澳大利亞
    • 其他亞太地區
  • 中東和非洲

第 11 章競爭格局

  • 主要發展和戰略
  • 公司份額分析
  • 按治療類型劃分的基準

第 12 章公司簡介

  • 艾伯維公司
    • 公司簡介
    • 治療類型組合和描述
    • 主要亮點
    • 財務摘要
  • 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 章全球孤兒藥市場-DATAM

簡介目錄
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