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孤兒藥

市場調查報告書

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1527236

孤兒藥市場 - 全球產業規模、佔有率、趨勢、機會和預測，按藥物類型、疾病類型、配銷通路、地區和競爭細分，2019-2029F

Orphan Drugs Market - Global Industry Size, Share, Trends, Opportunity and Forecast, Segmented By Drug Type, By Disease Type, By Distribution Channel, By Region and Competition, 2019-2029F

出版日期: 2024年08月02日 | 出版商:

TechSci Research | 英文 184 Pages | 商品交期: 2-3個工作天內

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簡介目錄

2023 年全球孤兒藥市場估值為 1872.4 億美元，預計到 2029 年預測期內將強勁成長，複合年成長率為 12.31%。相對少數的人。這些疾病通常被稱為“孤兒疾病”，因為由於患者人數有限，它們可能不會引起製藥公司的高度關注。孤兒藥物旨在解決患有罕見疾病的患者未得到滿足的醫療需求，否則這些患者可能沒有可行的治療選擇。對於製藥公司來說，開發罕見疾病藥物可能具有挑戰性且有財務風險，因為與更常見的疾病相比，潛在市場很小。在美國，1983 年的《孤兒藥法案》是一項具有里程碑意義的立法，它引入了這些激勵措施，並為許多孤兒藥的開發鋪平了道路。世界其他國家也存在類似的孤兒藥法規和誘因。

市場概況
預測期	2025-2029
2023 年市場規模	1872.4億美元
2029 年市場規模	3723.1億美元
2024-2029 年複合年成長率	12.31%
成長最快的細分市場	生物
最大的市場	北美洲

主要市場促進因素

罕見疾病盛行率上升推動市場成長

生物技術的進步推動市場成長

主要市場挑戰

患者群體較少

對疾病缺乏了解

患者識別和診斷

主要市場趨勢

患者人口結構的變化推動市場成長

對個人化醫療的日益關注

細分市場洞察

疾病類型見解

區域洞察

Global Orphan Drugs Market was valued at USD 187.24 billion in 2023 and is anticipated to project robust growth in the forecast period with a CAGR of 12.31% through 2029. Orphan Drugs or orphan medicinal products, are pharmaceutical agents developed to treat rare medical conditions or diseases that affect a relatively small number of people. These conditions are often referred to as "orphan diseases" because they may not attract significant attention from pharmaceutical companies due to their limited patient populations. Orphan drugs are intended to address unmet medical needs for patients with rare diseases that might not have viable treatment options otherwise. Developing drugs for rare diseases can be challenging and financially risky for pharmaceutical companies, as the potential market is small compared to more common conditions. In the United States, the Orphan Drug Act of 1983 was a landmark piece of legislation that introduced these incentives and paved the way for the development of many orphan drugs. Similar orphan drug regulations and incentives exist in other countries around the world.

Market Overview
Forecast Period	2025-2029
Market Size 2023	USD 187.24 Billion
Market Size 2029	USD 372.31 Billion
CAGR 2024-2029	12.31%
Fastest Growing Segment	Biological
Largest Market	North America

Key Market Drivers

Rising Prevalence of Rare Diseases Drives the Market Growth

The rising prevalence of rare diseases plays a significant role in driving the growth of the global orphan drugs market. The prevalence of rare diseases is increasing due to factors such as aging populations, environmental pollution, and genetic mutations. As medical knowledge and diagnostic capabilities improve, more rare diseases are being accurately diagnosed. This has led to an increase in the reported prevalence of these conditions. Greater awareness among healthcare professionals, patients, and advocacy groups has also contributed to improved diagnosis and recognition of rare diseases. The rising prevalence of rare diseases means that there is a larger patient population in need of treatment. This increased patient demand drives the development and commercialization of orphan drugs. For instance, According to data from the U.S. Food and Drug Administration stats, more than 7,000 rare diseases impact over 30 million individuals in the United States. Numerous rare conditions pose life-threatening risks, with the majority lacking effective treatments.

The larger patient population with rare diseases presents a market opportunity for pharmaceutical companies. While each individual rare disease may have a small patient population, the collective number of patients across various rare diseases becomes significant, making the development of orphan drugs economically viable.

Advances in genetics and genomics have allowed for better understanding of the underlying causes of many rare diseases. This knowledge has facilitated the development of targeted therapies and personalized medicine approaches, driving the demand for orphan drugs tailored to specific genetic mutations. Regulatory incentives and support from government agencies and healthcare systems have encouraged pharmaceutical companies to invest in research and development of orphan drugs for rare diseases. This support includes expedited regulatory review processes, extended market exclusivity, and reduced fees. Strong advocacy from patient groups and rare disease organizations has raised awareness about the challenges faced by individuals with rare diseases. These advocacy efforts put pressure on governments and industry stakeholders to invest in research and development for orphan drugs.

Advancements in Biotechnology Drives the Market Growth

Advancements in biotechnology play a crucial role in driving the growth of the global orphan drugs market by enabling the development of innovative treatments for rare diseases. Biotechnology has enabled a deeper understanding of the underlying genetic and molecular mechanisms of rare diseases. This knowledge has led to the development of targeted therapies that specifically address the root causes of these diseases. These therapies are often more effective and have fewer side effects than traditional treatments, making them attractive options for rare disease patients.

Biotechnological tools, such as genomics and proteomics, allow for personalized medicine approaches. Physicians can analyze a patient's genetic makeup to determine the most suitable treatment, optimizing therapeutic outcomes and minimizing adverse effects. This precision medicine approach is particularly relevant for rare diseases, where each patient's condition can be unique. Biotechnology has revolutionized the drug discovery process. Techniques like high-throughput screening, computational modeling, and structural biology have accelerated the identification and development of potential drug candidates for rare diseases. These speeds up the research and development pipeline for orphan drugs. Biotechnology has paved the way for innovative gene and cell therapies, which hold great promise for treating rare genetic disorders. Gene therapy involves introducing healthy genes into a patient's cells to correct genetic mutations, while cell therapy uses engineered cells to replace or repair damaged tissues. These approaches offer potential cures or long-term disease management for certain rare diseases.

Key Market Challenges

Small Patient Populations

Pharmaceutical companies invest substantial resources in research and development, clinical trials, and regulatory processes to bring a drug to market. However, with a small patient population, the potential revenue from sales of the orphan drug may be limited, making it challenging to recoup these high development costs. The small market size can make orphan drug development economically unviable, especially for diseases with extremely low prevalence. Companies may be hesitant to invest in research and development if they anticipate that the financial returns will not justify the investment. Developing drugs is inherently expensive, involving preclinical testing, clinical trials, and regulatory approval processes. The limited patient pool of rare diseases means that these costs need to be spread across fewer potential users, resulting in higher costs per patient for orphan drugs.

Lack of Disease Understanding

Without a thorough understanding of the underlying mechanisms and causes of a rare disease, it can be difficult to identify suitable drug targets. This hampers the development of effective treatments that address the root causes of the condition. Validating potential drug targets is essential for drug development. Lack of disease understanding may lead to uncertainty about the relevance and significance of proposed targets, potentially resulting in a higher risk of failure in clinical trials. Developing a new drug requires a deep understanding of the disease's molecular pathways and mechanisms. Lack of insight into these factors can lead to delays in drug discovery efforts, as researchers may need to invest additional time and resources to uncover crucial information. Biomarkers are important for diagnosing diseases, monitoring treatment response, and predicting disease progression. The lack of disease understanding can hinder the identification of reliable biomarkers, making it challenging to track disease status and treatment efficacy. Clinical trials for orphan drugs often require specialized designs due to the small patient populations. A lack of disease understanding can complicate the design of these trials, potentially leading to difficulties in recruiting patients and interpreting results.

Patient Identification and Diagnosis

Rare diseases often have nonspecific symptoms that overlap with more common conditions. This can lead to underdiagnosis or misdiagnosis, delaying appropriate treatment and potentially excluding patients from clinical trials or access to orphan drugs. Healthcare professionals, including primary care physicians, may lack awareness or knowledge about rare diseases. This can result in delayed or missed diagnoses, preventing timely treatment initiation. Rare diseases may require specialized medical expertise for accurate diagnosis. However, the scarcity of experts in these conditions can lead to delayed or inaccurate diagnoses. The diagnostic journey for individuals with rare diseases can be long and frustrating. Multiple visits to various healthcare providers and misdiagnoses can lead to delays in receiving appropriate care and treatment. Some rare diseases require specialized and often expensive diagnostic tests, which may not be readily available or covered by insurance. Limited access to these tests can delay diagnosis and treatment. Access to healthcare resources and expertise can vary by region or country. Patients in underserved areas may face additional challenges in accessing accurate diagnosis and appropriate care.

Key Market Trends

Changing Patient Demographics Propels the Market Growth

Changing patient demographics, particularly an aging population, have a significant impact on driving the growth of the global orphan drugs market. As the global population ages, there is a higher prevalence of age-related diseases, many of which fall under the category of rare diseases. Conditions such as Alzheimer's disease, Parkinson's disease, and certain types of cancer become more common with advancing age. This creates a greater demand for orphan drugs that target these specific rare conditions. The risk of developing genetic and rare diseases often increases with age. As people live longer, the chances of developing these conditions also rise, leading to a larger patient population in need of orphan drug treatments. Elderly individuals often have complex health needs due to the presence of multiple chronic conditions and comorbidities. Orphan drugs that can address these unique and intricate health challenges are particularly valuable in an aging population.

With improving diagnostic technologies and methods, more rare diseases are being accurately identified in elderly patients. This contributes to a better understanding of the prevalence and diversity of rare diseases within the aging demographic. The aging population requires long-term care and management of chronic conditions. Orphan drugs that offer disease-modifying effects or improve quality of life become essential components of geriatric care. Older individuals often experience age-related changes in drug metabolism and response. Orphan drugs that offer personalized treatment approaches based on genetic and molecular profiles align well with the needs of the aging population. Developed countries with aging populations tend to have well-established healthcare systems and higher healthcare spending. This can lead to better access to orphan drugs and greater willingness to invest in research and development for rare diseases.

The Increasing Focus on Personalized Medicine

The increasing focus on personalized medicine is a significant trend in the global orphan drugs market, transforming the way rare diseases are treated and driving innovation in drug development. Personalized medicine involves tailoring medical care, including drug treatments, to the individual characteristics of each patient. Personalized medicine allows for the development of targeted therapies that address the specific genetic, molecular, and cellular characteristics of a patient's disease. For rare diseases, which are often caused by specific genetic mutations, this approach can lead to more effective treatments with fewer side effects.

Advances in genomics and molecular profiling have enabled the identification of genetic mutations and biomarkers associated with rare diseases. This information helps in patient stratification, treatment selection, and clinical trial design. Personalized medicine allows healthcare providers to create individualized treatment plans based on a patient's unique genetic makeup and disease characteristics. This approach maximizes treatment efficacy while minimizing potential harm. Pharmaceutical companies can use personalized medicine approaches to design clinical trials with patient populations most likely to benefit from a specific treatment. This can lead to more efficient drug development processes and faster approval timelines. Personalized medicine often involves the development of companion diagnostics-tests that identify whether a patient is likely to respond to a particular treatment. This ensures that patients receive the most appropriate therapy, enhancing treatment outcomes.

Segmental Insights

Disease Type Insights

In 2023, the Orphan Drugs market was dominated by oncology disease segment and is predicted to continue expanding over the coming years. Rare cancers may have low incidence, the cumulative prevalence of all rare cancers is significant. This contributes to a substantial patient population in need of orphan drug treatments. Many rare cancers have limited treatment options available, creating a strong demand for new and innovative therapies. The rarity of these cancers often results in a lack of established standard treatments. Oncology has been at the forefront of precision medicine, with genetic and molecular profiling guiding treatment decisions. This aligns well with the orphan drugs market's trend towards personalized therapies. Many orphan drugs in oncology are targeted therapies that specifically address molecular abnormalities driving the growth of cancer cells. This approach offers the potential for higher treatment efficacy and fewer side effects.

Regional Insights

The North America region has established itself as the leader in the Global Orphan Drugs Market. North America, particularly the United States and Canada, boasts advanced healthcare infrastructure, including well-established healthcare systems, medical institutions, and research centers. This infrastructure supports the development, approval, and distribution of orphan drugs. North America is a hub for pharmaceutical research and development, with numerous biotechnology and pharmaceutical companies headquartered in the region. These companies invest heavily in orphan drug research, driving innovation and new treatment options. Regulatory agencies in North America, such as the U.S. Food and Drug Administration (FDA) and Health Canada, provide incentives and expedited pathways for orphan drug development and approval. This encourages companies to focus on rare diseases.

Key Market Players

Novartis AG

Pfizer Inc.

Sanofi SA

Bristol-Myers Squibb Company

GSK plc

Alexion Pharmaceuticals, Inc.

AbbVie Inc.

Merck KgaA

Johnson & Johnson

F. Hoffmann-La Roche Ltd.

Report Scope:

In this report, the Global Orphan Drugs Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

Orphan Drugs Market, By Drug Type:

Biological Non-biological

Orphan Drugs Market, By Disease Type:

Oncology Haematology Neurology Cardiovascular Others

Orphan Drugs Market, By Distribution Channel:

Hospital Pharmacy Online sales Others

Global Orphan Drugs Market, By Region:

United States
Canada
Mexico

China
India
South Korea
Australia
Japan

Germany
France
United Kingdom
Spain
Italy

Brazil
Argentina
Colombia

South Africa
Saudi Arabia
UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Orphan Drugs Market.

Available Customizations:

Global Orphan Drugs Market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

1. Product Overview

1.1. Market Definition
1.2. Scope of the Market
- 1.2.1. Markets Covered
- 1.2.2. Years Considered for Study
- 1.2.3. Key Market Segmentations

2. Research Methodology

2.1. Objective of the Study
2.2. Baseline Methodology
2.3. Key Industry Partners
2.4. Major Association and Secondary Sources
2.5. Forecasting Methodology
2.6. Data Triangulation & Validation
2.7. Assumptions and Limitations

3. Executive Summary

3.1. Overview of the Market
3.2. Overview of Key Market Segmentations
3.3. Overview of Key Market Players
3.4. Overview of Key Regions/Countries
3.5. Overview of Market Drivers, Challenges, Trends

4. Voice of Customer

5. Global Orphan Drugs Market Outlook

5.1. Market Size & Forecast
- 5.1.1. By Value
5.2. Market Share & Forecast
- 5.2.1. By Drug Type (Biological, Non-biological)
- 5.2.2. By Disease Type (Oncology, Haematology, Neurology, Cardiovascular, Others)
- 5.2.3. By Distribution Channel (Hospital, Pharmacy, Online sales, Others)
- 5.2.4. By Company (2023)
- 5.2.5. By Region
5.3. Market Map

6. North America Orphan Drugs Market Outlook

6.1. Market Size & Forecast
- 6.1.1. By Value
6.2. Market Share & Forecast
- 6.2.1. By Drug Type
- 6.2.2. By Disease Type
- 6.2.3. By Distribution Channel
- 6.2.4. By Country
6.3. North America: Country Analysis
- 6.3.1. United States Orphan Drugs Market Outlook
  - 6.3.1.1. Market Size & Forecast
    - 6.3.1.1.1. By Value
  - 6.3.1.2. Market Share & Forecast
    - 6.3.1.2.1. By Drug Type
    - 6.3.1.2.2. By Disease Type
    - 6.3.1.2.3. By Distribution Channel
- 6.3.2. Mexico Orphan Drugs Market Outlook
  - 6.3.2.1. Market Size & Forecast
    - 6.3.2.1.1. By Value
  - 6.3.2.2. Market Share & Forecast
    - 6.3.2.2.1. By Drug Type
    - 6.3.2.2.2. By Disease Type
    - 6.3.2.2.3. By Distribution Channel
- 6.3.3. Canada Orphan Drugs Market Outlook
  - 6.3.3.1. Market Size & Forecast
    - 6.3.3.1.1. By Value
  - 6.3.3.2. Market Share & Forecast
    - 6.3.3.2.1. By Drug Type
    - 6.3.3.2.2. By Disease Type
    - 6.3.3.2.3. By Distribution Channel

7. Europe Orphan Drugs Market Outlook

7.1. Market Size & Forecast
- 7.1.1. By Value
7.2. Market Share & Forecast
- 7.2.1. By Drug Type
- 7.2.2. By Disease Type
- 7.2.3. By Distribution Channel
- 7.2.4. By Country
7.3. Europe: Country Analysis
- 7.3.1. France Orphan Drugs Market Outlook
  - 7.3.1.1. Market Size & Forecast
    - 7.3.1.1.1. By Value
  - 7.3.1.2. Market Share & Forecast
    - 7.3.1.2.1. By Drug Type
    - 7.3.1.2.2. By Disease Type
    - 7.3.1.2.3. By Distribution Channel
- 7.3.2. Germany Orphan Drugs Market Outlook
  - 7.3.2.1. Market Size & Forecast
    - 7.3.2.1.1. By Value
  - 7.3.2.2. Market Share & Forecast
    - 7.3.2.2.1. By Drug Type
    - 7.3.2.2.2. By Disease Type
    - 7.3.2.2.3. By Distribution Channel
- 7.3.3. United Kingdom Orphan Drugs Market Outlook
  - 7.3.3.1. Market Size & Forecast
    - 7.3.3.1.1. By Value
  - 7.3.3.2. Market Share & Forecast
    - 7.3.3.2.1. By Drug Type
    - 7.3.3.2.2. By Disease Type
    - 7.3.3.2.3. By Distribution Channel
- 7.3.4. Italy Orphan Drugs Market Outlook
  - 7.3.4.1. Market Size & Forecast
    - 7.3.4.1.1. By Value
  - 7.3.4.2. Market Share & Forecast
    - 7.3.4.2.1. By Drug Type
    - 7.3.4.2.2. By Disease Type
    - 7.3.4.2.3. By Distribution Channel
- 7.3.5. Spain Orphan Drugs Market Outlook
  - 7.3.5.1. Market Size & Forecast
    - 7.3.5.1.1. By Value
  - 7.3.5.2. Market Share & Forecast
    - 7.3.5.2.1. By Drug Type
    - 7.3.5.2.2. By Disease Type
    - 7.3.5.2.3. By Distribution Channel

8. Asia-Pacific Orphan Drugs Market Outlook

8.1. Market Size & Forecast
- 8.1.1. By Value
8.2. Market Share & Forecast
- 8.2.1. By Drug Type
- 8.2.2. By Disease Type
- 8.2.3. By Distribution Channel
- 8.2.4. By Country
8.3. Asia-Pacific: Country Analysis
- 8.3.1. China Orphan Drugs Market Outlook
  - 8.3.1.1. Market Size & Forecast
    - 8.3.1.1.1. By Value
  - 8.3.1.2. Market Share & Forecast
    - 8.3.1.2.1. By Drug Type
    - 8.3.1.2.2. By Disease Type
    - 8.3.1.2.3. By Distribution Channel
- 8.3.2. India Orphan Drugs Market Outlook
  - 8.3.2.1. Market Size & Forecast
    - 8.3.2.1.1. By Value
  - 8.3.2.2. Market Share & Forecast
    - 8.3.2.2.1. By Drug Type
    - 8.3.2.2.2. By Disease Type
    - 8.3.2.2.3. By Distribution Channel
- 8.3.3. South Korea Orphan Drugs Market Outlook
  - 8.3.3.1. Market Size & Forecast
    - 8.3.3.1.1. By Value
  - 8.3.3.2. Market Share & Forecast
    - 8.3.3.2.1. By Drug Type
    - 8.3.3.2.2. By Disease Type
    - 8.3.3.2.3. By Distribution Channel
- 8.3.4. Japan Orphan Drugs Market Outlook
  - 8.3.4.1. Market Size & Forecast
    - 8.3.4.1.1. By Value
  - 8.3.4.2. Market Share & Forecast
    - 8.3.4.2.1. By Drug Type
    - 8.3.4.2.2. By Disease Type
    - 8.3.4.2.3. By Distribution Channel
- 8.3.5. Australia Orphan Drugs Market Outlook
  - 8.3.5.1. Market Size & Forecast
    - 8.3.5.1.1. By Value
  - 8.3.5.2. Market Share & Forecast
    - 8.3.5.2.1. By Drug Type
    - 8.3.5.2.2. By Disease Type
    - 8.3.5.2.3. By Distribution Channel

9. South America Orphan Drugs Market Outlook

9.1. Market Size & Forecast
- 9.1.1. By Value
9.2. Market Share & Forecast
- 9.2.1. By Drug Type
- 9.2.2. By Disease Type
- 9.2.3. By Distribution Channel
- 9.2.4. By Country
9.3. South America: Country Analysis
- 9.3.1. Brazil Orphan Drugs Market Outlook
  - 9.3.1.1. Market Size & Forecast
    - 9.3.1.1.1. By Value
  - 9.3.1.2. Market Share & Forecast
    - 9.3.1.2.1. By Drug Type
    - 9.3.1.2.2. By Disease Type
    - 9.3.1.2.3. By Distribution Channel
- 9.3.2. Argentina Orphan Drugs Market Outlook
  - 9.3.2.1. Market Size & Forecast
    - 9.3.2.1.1. By Value
  - 9.3.2.2. Market Share & Forecast
    - 9.3.2.2.1. By Drug Type
    - 9.3.2.2.2. By Disease Type
    - 9.3.2.2.3. By Distribution Channel
- 9.3.3. Colombia Orphan Drugs Market Outlook
  - 9.3.3.1. Market Size & Forecast
    - 9.3.3.1.1. By Value
  - 9.3.3.2. Market Share & Forecast
    - 9.3.3.2.1. By Drug Type
    - 9.3.3.2.2. By Disease Type
    - 9.3.3.2.3. By Distribution Channel

10. Middle East and Africa Orphan Drugs Market Outlook

10.1. Market Size & Forecast
- 10.1.1. By Value
10.2. Market Share & Forecast
- 10.2.1. By Drug Type
- 10.2.2. By Disease Type
- 10.2.3. By Distribution Channel
- 10.2.4. By Country
10.3. MEA: Country Analysis
- 10.3.1. South Africa Orphan Drugs Market Outlook
  - 10.3.1.1. Market Size & Forecast
    - 10.3.1.1.1. By Value
  - 10.3.1.2. Market Share & Forecast
    - 10.3.1.2.1. By Drug Type
    - 10.3.1.2.2. By Disease Type
    - 10.3.1.2.3. By Distribution Channel
- 10.3.2. Saudi Arabia Orphan Drugs Market Outlook
  - 10.3.2.1. Market Size & Forecast
    - 10.3.2.1.1. By Value
  - 10.3.2.2. Market Share & Forecast
    - 10.3.2.2.1. By Drug Type
    - 10.3.2.2.2. By Disease Type
    - 10.3.2.2.3. By Distribution Channel
- 10.3.3. UAE Orphan Drugs Market Outlook
  - 10.3.3.1. Market Size & Forecast
    - 10.3.3.1.1. By Value
  - 10.3.3.2. Market Share & Forecast
    - 10.3.3.2.1. By Drug Type
    - 10.3.3.2.2. By Disease Type
    - 10.3.3.2.3. By Distribution Channel

11. Market Dynamics

11.1. Drivers
11.2. Challenges

12. Market Trends & Developments

12.1. Recent Developments
12.2. Product Launches
12.3. Mergers & Acquisitions

13. PESTLE Analysis

14. Porter's Five Forces Analysis

14.1. Competition in the Industry
14.2. Potential of New Entrants
14.3. Power of Suppliers
14.4. Power of Customers
14.5. Threat of Substitute Product

15. Competitive Landscape

15.1. Novartis AG
- 15.1.1. Business Overview
- 15.1.2. Company Snapshot
- 15.1.3. Products & Services
- 15.1.4. Financials (As Reported)
- 15.1.5. Recent Developments
- 15.1.6. Key Personnel Details
- 15.1.7. SWOT Analysis
15.2. Pfizer Inc.
15.3. Sanofi SA
15.4. Bristol-Myers Squibb Company
15.5. GSK plc
15.6. Alexion Pharmaceuticals, Inc.
15.7. AbbVie Inc.
15.8. Merck KgaA
15.9. Johnson & Johnson
15.10. F. Hoffmann-La Roche Ltd