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市场调查报告书
商品编码
1136911

全球孤儿药市场-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: 约2个工作天内

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简介目录

市场动态

研发活动的增加、孤儿药治疗采用的增加以及老年人口的增加正在推动孤儿药市场的增长。

罕见病意识的提高有望推动市场增长

在预测期内，人们对罕见病及其治疗的认识不断提高，预计将提振市场。根据发表在 Perspective in Clinical Research 2022 上的一篇文章，FDA 等监管机构最近推出了一个名为 FDA 罕见病照片和视频项目的在线平台，用于记录和分享员工经验和实时患者证词。发达。通过安排与患者、护理人员和 FDA 工作人员的各种会议，患者事务办公室支持以患者为中心以实现这一目标。包括国家罕见病局和欧洲罕见病局在内的几个非营利组织分别支持欧洲和美国的监管和研究活动，并通过患者宣传、信息共享和不同患者群体之间的网络来促进患者宣传. 增加。

此外，据临床与经济评论研究所发表的一篇论文显示，截至2020年正在开发的细胞和基因疗法是针对罕见病的，预计70%以上的罕见病是遗传病。自《孤儿药法案》颁布以来，截至 2020 年年中，FDA 已批准了 599 种孤儿药。 First-in-class孤儿药的比例明显高于非孤儿药，50%的孤儿药在美国获得许可。儘管大流行，但随着行业适应中断并製定新策略以支持正在进行的研究，总体临床试验活动仍在继续。计划于 2021 年开始的新临床试验比 2020 年增加 14%，比 2019 年增加 19%，报告了 5,500 项。

难以收集足够的疗效和安全性数据阻碍市场增长

但是，难以收集到足够数量的疗效和安全性数据，开发者和医疗保健系统面临资金支持的风险，以及对大多数疾病的了解不足。这些都阻碍了罕见病治疗方法和药物的开发疾病。仅靠政府的行动不足以弥补罕见病商业潜力普遍较低导致的市场缺口。此外，确定一个定义“罕见疾病”而不是“超罕见疾病”的工作定义将是建立政策基础以刺激超罕见药物开发的主要障碍之一。这些因素限制了进一步的市场扩张。

COVID-19 影响分析

COVID-19 的出现对全球孤儿药市场产生了相当大的影响。根据发表在《2021 年公共卫生前沿》上的一篇论文，大约有 5,000 到 8,000 种罕见疾病，尤其是那些基于基因的疾病，影响着全球约 4 亿人。患有罕见疾病的患者及其护理人员报告了显着的护理差距和未满足的需求。对于研究人员来说，招募大量受影响个体的同胞资助研究的难度和费用已经是一个问题。由于 COVID-19 以及每个人都存在的一般健康问题，罕见病患者承担着双重问题。他们还面临着药物供应和获得他们需要的基本职业治疗的问题。

同时，FDA 已批准 32 种孤儿药和生物製剂，在预计 2020 年 COVID-19 大流行期间显着维持孤儿药研发的进展。药物评价与研究中心秘书处成立了罕见病中心，以评估特定罕见病的上市申请。与罕见病团队合作并获得支持和帮助以实施罕见病政策。 FDA 启动了孤儿药现代化试点计划，以满足随后申请量的增加。预计 FDA 将在 90 天内审查所有申请，无论申请的数量或复杂程度如何。

全球孤儿药市场报告将提供对大约 65 多个市场数据表、65 多个图表和 200 多页（大约）的访问。

内容

第一章市场研究方法和范围

  • 调查方法
  • 调查目的和范围

第 2 章市场定义和概述

第 3 章执行摘要

  • 按产品类型划分的市场细分
  • 按治疗区域划分的市场细分
  • 按分销渠道划分的市场细分
  • 按地区划分的市场细分

第 4 章市场动态

  • 市场影响因素
    • 驱动程序
    • 糖尿病高发
    • 启动研究活动
    • 约束因素
    • 飙升的药品费用
    • 影响分析

第五章行业分析

  • 波特五力分析
  • 供应链分析
  • 监管分析
  • 定价分析
  • 未满足的需求分析

第 6 章 COVID-19 分析

  • COVID-19 的市场分析
    • COVID-19 之前的市场情景
    • COVID-19 的当前市场情景
    • COVID-19 后或未来情景
  • COVID-19 期间的价格动态
  • 供需范围
  • 大流行期间与市场相关的政府举措
  • 製造商的战略举措
  • 总结

第 7 章按产品类型

  • 生物製品
  • 非生物

第 8 章按治疗领域

  • 肿瘤学
  • 血液病
  • 中枢神经系统
  • 循环区
  • 内分泌
  • 呼吸系统
  • 免疫调节剂
  • 消化系统
  • 肌肉骨骼系统
  • 全身性抗感染药
  • 皮肤科
  • 其他

第 9 章分销渠道

  • 医院药房
  • 零售药房
  • 在线药店

第 10 章按地区划分

  • 北美
    • 美国
    • 加拿大
    • 墨西哥
  • 欧洲
    • 德国
    • 英国
    • 法国
    • 意大利
    • 西班牙
    • 其他欧洲
  • 南美洲
    • 巴西
    • 阿根廷
    • 其他南美洲
  • 亚太地区
    • 中国
    • 印度
    • 日本
    • 澳大利亚
    • 其他亚太地区
  • 中东和非洲

第 11 章竞争格局

  • 主要发展和战略
  • 公司份额分析
  • 按治疗类型划分的基准

第 12 章公司简介

  • 艾伯维公司
    • 公司简介
    • 治疗类型组合和描述
    • 主要亮点
    • 财务摘要
  • 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 章全球孤儿药市场-DATAM

简介目录
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