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PUBLISHER: Stratistics Market Research Consulting | PRODUCT CODE: 1383503

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PUBLISHER: Stratistics Market Research Consulting | PRODUCT CODE: 1383503

Orphan Drugs Market Forecasts to 2030 - Global Analysis By Disease Type, Drug Type, Sale, Drug, Distribution Channel and By Geography

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According to Stratistics MRC, the Global Orphan Drugs Market is accounted for $175.8 billion in 2023 and is expected to reach $445.5 billion by 2030 growing at a CAGR of 14.2% during the forecast period. Drugs used to identify and treat uncommon medical conditions are known as orphan drugs. Numerous oncological, metabolic, hematologic, chronic, progressive, viral, and neurological illnesses can be effectively treated with them. Orphan medications often employ a targeted approach, concentrating on the particular genetic or molecular abnormalities that underlie a rare disease. This results in more precise and effective treatment options, leading to improved patient outcomes, including increased survival rates, symptom management, and overall quality of life.

According to the Genetic and Rare Diseases (GARD) Information Center, there are more than 10,000 known rare diseases in the U.S. that affect about 1 in 10 people or 30 million people in the U.S.

Market Dynamics:


Rise in disease awareness

Growing knowledge of diseases is a major factor driving the market for orphan medications since it makes rare diseases better recognized, which encourages advocacy, funding, and research. Moreover, an increase in patient, healthcare professional and advocacy group awareness drives up the need for efficient therapies, which in turn motivates pharmaceutical corporations to fund research and development of orphan drugs. In the end, it expands the market and enhances the lives of people with rare diseases by making specialized therapies more accessible. Therefore, it also results in increased support for funding, regulatory incentives, and partnerships.


High initial costs

The process of creating medications for uncommon diseases can be costly due to the extensive research, clinical trials, and regulatory procedures involved. Additionally, orphan medications are generally costly since these expenses must be covered by drug prices. It can be difficult to deliver rare disease therapies that are both widely available and reasonably priced due to the high expenses, which can also pose access challenges for insurers, healthcare systems, and patients. Hence, this can leave patients with limited treatment options and hinder progress in addressing the unmet medical needs of individuals with rare diseases.


Developments in regulatory incentives

Extended market exclusivity, tax rebates, and accelerated approval procedures are all included in these incentives. They incentivize pharmaceutical companies to spend money on the development of orphan drugs by lowering financial risks and accelerating time to market. Furthermore, these kinds of incentives make these ventures more financially viable, which in turn encourage innovation and make more orphan medications available to meet the unmet medical requirements of people with rare diseases. Pharmaceutical businesses and those with rare illnesses benefit from this in a win-win scenario. Hence, government regulations that provide favourable incentives to encourage the development of therapies for rare diseases are a major factor driving the orphan drug industry.


Risks associated with limited potential

The limited market potential can discourage companies from pursuing orphan drug development, resulting in fewer treatment options for patients with rare diseases and potential delays in accessing innovative therapies. Moreover, drugs known as orphans are created to treat uncommon conditions that only a tiny number of people have. The market for these medications is less than that of medications for more prevalent disorders because of the small number of patients. Furthermore, pharmaceutical corporations may find it financially undesirable to invest in the research and development of orphan medications as a result, which is a drawback of the market's expansion.

COVID-19 Impact

The outbreak of COVID-19 has disrupted workflows in the health care sector around the world. Numerous industries, including some healthcare-related sub-sectors, have been forced to temporarily close their doors due to the disease. The market for orphan drugs has also experienced negative growth, which can be linked to a number of issues, including a drop in screening services, a decrease in access to specialists, treatment interruptions, restricted operations in the majority of industries, insufficient funding for academic institutions and research, the temporary closure of significant academic institutions, supply chain disruptions, and difficulties delivering necessary or post-sales services.

The oncological diseases segment is expected to be the largest during the forecast period

The oncological diseases segment is estimated to hold the largest share. Orphan drugs play a crucial role in the treatment of oncological diseases. A medication created especially to treat uncommon diseases that only affect a small number of patients is known as an orphan drug. Certain subtypes or mutations within a cancer type can be classified as rare diseases, even though individual cancer types might not be. These orphan medications target particular genetic defects or molecular pathways that contribute to the development and spread of cancer. Due to favourable regulatory incentives, a shift towards precision medicine, and the rising prevalence of rare cancer subtypes, the market for orphan drugs for oncological diseases has experienced significant growth.

The biologics segment is expected to have the highest CAGR during the forecast period

The biologics segment is anticipated to have lucrative growth during the forecast period. Biologics can be created to target the underlying genetic abnormalities or molecular pathways that cause the disease in the case of orphan diseases, where the patient population is small. This focused approach lowers possible side effects and improves treatment efficacy. One sort of biologic that has been developed is the monoclonal antibody. It binds specifically to certain proteins on cancer cells, causing the immune system to attack the tumor. The orphan drug market has witnessed a growing number of biologic drugs being developed and approved for rare diseases, including rare forms of cancer.

Region with largest share:

Asia Pacific commanded the largest market share during the extrapolated period owing to the growing infrastructure of industries, rising disposable incomes, and favourable government policies in the region. Furthermore, clinical trial growth in Asia-Pacific is anticipated to accelerate beyond that of the United States and Europe, contributing to the region's market expansion. Moreover, it is anticipated that an increase in government initiatives to expand the number of oncology and community health centers will broaden the availability of orphan medications in developing nations.

Region with highest CAGR:

North America is expected to witness profitable growth over the projection period, due to the presence of major market players and a large population of patients with orphan diseases. Additionally, a growing number of orphan product approvals, a sizable number of orphan drugs in the development stage, and rising orphan drug spending in the area are the other factors driving the orphan drug market in the North American region.

Key players in the market

Some of the key players in the Orphan Drugs Market include: Bayer AG, Novartis AG, F. Hoffmann-La Roche Ltd, Bristol-Myers Squibb Company, Amgen Inc., Novo Nordisk A/S, Alexion Pharmaceuticals Inc., GlaxoSmithKline plc, AstraZeneca, Celgene Corporation, Mylan, Biogen, Merck KGaA, Takeda Pharmaceutical Company Limited, Eli Lilly and Company and Shire Pharmaceutical.

Key Developments:

In March 2023, F. Hoffmann-La Roche Ltd: Roche announces collaboration with Lilly to enhance early diagnosis of Alzheimer's disease.

In January 2022, Amgen and Generate Biomedicines announced a research collaboration agreement to discover and create protein therapeutics for five clinical targets across several therapeutic areas and multiple modalities.

Disease Types Covered:

  • Oncological Diseases
  • Metabolic Diseases
  • Hematologic & Immunologic Diseases
  • Infectious diseases
  • Neurological diseases

Drug Types Covered:

  • Non-Biologics
  • Biologics

Sales Covered:

  • Prescribed
  • Generics

Drugs Covered:

  • Rituxan
  • Revlimid
  • Imbruvica
  • Opdivo
  • Darzalex
  • Pomalyst
  • Other Drugs

Distribution Channels Covered:

  • Online Pharmacies
  • Hospital Pharmacies
  • Retail Pharmacies
  • Other Distribution Channels

Regions Covered:

  • North America
    • US
    • Canada
    • Mexico
  • Europe
    • Germany
    • UK
    • Italy
    • France
    • Spain
    • Rest of Europe
  • Asia Pacific
    • Japan
    • China
    • India
    • Australia
    • New Zealand
    • South Korea
    • Rest of Asia Pacific
  • South America
    • Argentina
    • Brazil
    • Chile
    • Rest of South America
  • Middle East & Africa
    • Saudi Arabia
    • UAE
    • Qatar
    • South Africa
    • Rest of Middle East & Africa

What our report offers:

  • Market share assessments for the regional and country-level segments
  • Strategic recommendations for the new entrants
  • Covers Market data for the years 2021, 2022, 2023, 2026, and 2030
  • Market Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, and recommendations)
  • Strategic recommendations in key business segments based on the market estimations
  • Competitive landscaping mapping the key common trends
  • Company profiling with detailed strategies, financials, and recent developments
  • Supply chain trends mapping the latest technological advancements

Free Customization Offerings:

All the customers of this report will be entitled to receive one of the following free customization options:

  • Company Profiling
    • Comprehensive profiling of additional market players (up to 3)
    • SWOT Analysis of key players (up to 3)
  • Regional Segmentation
    • Market estimations, Forecasts and CAGR of any prominent country as per the client's interest (Note: Depends on feasibility check)
  • Competitive Benchmarking
    • Benchmarking of key players based on product portfolio, geographical presence, and strategic alliances
Product Code: SMRC24351

Table of Contents

1 Executive Summary

2 Preface

  • 2.1 Abstract
  • 2.2 Stake Holders
  • 2.3 Research Scope
  • 2.4 Research Methodology
    • 2.4.1 Data Mining
    • 2.4.2 Data Analysis
    • 2.4.3 Data Validation
    • 2.4.4 Research Approach
  • 2.5 Research Sources
    • 2.5.1 Primary Research Sources
    • 2.5.2 Secondary Research Sources
    • 2.5.3 Assumptions

3 Market Trend Analysis

  • 3.1 Introduction
  • 3.2 Drivers
  • 3.3 Restraints
  • 3.4 Opportunities
  • 3.5 Threats
  • 3.6 Emerging Markets
  • 3.7 Impact of Covid-19

4 Porters Five Force Analysis

  • 4.1 Bargaining power of suppliers
  • 4.2 Bargaining power of buyers
  • 4.3 Threat of substitutes
  • 4.4 Threat of new entrants
  • 4.5 Competitive rivalry

5 Global Orphan Drugs Market, By Disease Type

  • 5.1 Introduction
  • 5.2 Oncological Diseases
    • 5.2.1 Pancreatic Cancer
    • 5.2.2 Acute Myeloid Leukemia
    • 5.2.3 Multiple Myeloma
    • 5.2.4 Ovarian Cancer
    • 5.2.5 Renal Cell Carcinoma
  • 5.3 Metabolic Diseases
    • 5.3.1 Hunter Syndrome
    • 5.3.2 Gaucher Disease
    • 5.3.3 Fabry Disease
    • 5.3.4 Hypoparathyroidism
  • 5.4 Hematologic & Immunologic Diseases
    • 5.4.1 Hemophilia
    • 5.4.2 Hereditary Angioedema
  • 5.5 Infectious diseases
  • 5.6 Neurological diseases
    • 5.6.1 Huntington's Disease
    • 5.6.2 Alzheimer's Disease

6 Global Orphan Drugs Market, By Drug Type

  • 6.1 Introduction
  • 6.2 Non-Biologics
  • 6.3 Biologics

7 Global Orphan Drugs Market, By Sale

  • 7.1 Introduction
  • 7.2 Prescribed
  • 7.3 Generics

8 Global Orphan Drugs Market, By Drug

  • 8.1 Introduction
  • 8.2 Rituxan
  • 8.3 Revlimid
  • 8.4 Imbruvica
  • 8.5 Opdivo
  • 8.6 Darzalex
  • 8.7 Pomalyst
  • 8.8 Other Drugs

9 Global Orphan Drugs Market, By Distribution Channel

  • 9.1 Introduction
  • 9.2 Online Pharmacies
  • 9.3 Hospital Pharmacies
  • 9.4 Retail Pharmacies
  • 9.5 Other Distribution Channels

10 Global Orphan Drugs Market, By Geography

  • 10.1 Introduction
  • 10.2 North America
    • 10.2.1 US
    • 10.2.2 Canada
    • 10.2.3 Mexico
  • 10.3 Europe
    • 10.3.1 Germany
    • 10.3.2 UK
    • 10.3.3 Italy
    • 10.3.4 France
    • 10.3.5 Spain
    • 10.3.6 Rest of Europe
  • 10.4 Asia Pacific
    • 10.4.1 Japan
    • 10.4.2 China
    • 10.4.3 India
    • 10.4.4 Australia
    • 10.4.5 New Zealand
    • 10.4.6 South Korea
    • 10.4.7 Rest of Asia Pacific
  • 10.5 South America
    • 10.5.1 Argentina
    • 10.5.2 Brazil
    • 10.5.3 Chile
    • 10.5.4 Rest of South America
  • 10.6 Middle East & Africa
    • 10.6.1 Saudi Arabia
    • 10.6.2 UAE
    • 10.6.3 Qatar
    • 10.6.4 South Africa
    • 10.6.5 Rest of Middle East & Africa

11 Key Developments

  • 11.1 Agreements, Partnerships, Collaborations and Joint Ventures
  • 11.2 Acquisitions & Mergers
  • 11.3 New Product Launch
  • 11.4 Expansions
  • 11.5 Other Key Strategies

12 Company Profiling

  • 12.1 Bayer AG
  • 12.2 Novartis AG
  • 12.3 F. Hoffmann-La Roche Ltd
  • 12.4 Bristol-Myers Squibb Company
  • 12.5 Amgen Inc.
  • 12.6 Novo Nordisk A/S
  • 12.7 Alexion Pharmaceuticals Inc.
  • 12.8 GlaxoSmithKline plc
  • 12.9 AstraZeneca
  • 12.10 Celgene Corporation
  • 12.11 Mylan
  • 12.12 Biogen
  • 12.13 Merck KGaA
  • 12.14 Takeda Pharmaceutical Company Limited
  • 12.15 Eli Lilly and Company
  • 12.16 Shire Pharmaceutical
Product Code: SMRC24351

List of Tables

  • Table 1 Global Orphan Drugs Market Outlook, By Region (2021-2030) ($MN)
  • Table 2 Global Orphan Drugs Market Outlook, By Disease Type (2021-2030) ($MN)
  • Table 3 Global Orphan Drugs Market Outlook, By Oncological Diseases (2021-2030) ($MN)
  • Table 4 Global Orphan Drugs Market Outlook, By Pancreatic Cancer (2021-2030) ($MN)
  • Table 5 Global Orphan Drugs Market Outlook, By Acute Myeloid Leukemia (2021-2030) ($MN)
  • Table 6 Global Orphan Drugs Market Outlook, By Multiple Myeloma (2021-2030) ($MN)
  • Table 7 Global Orphan Drugs Market Outlook, By Ovarian Cancer (2021-2030) ($MN)
  • Table 8 Global Orphan Drugs Market Outlook, By Renal Cell Carcinoma (2021-2030) ($MN)
  • Table 9 Global Orphan Drugs Market Outlook, By Metabolic Diseases (2021-2030) ($MN)
  • Table 10 Global Orphan Drugs Market Outlook, By Hunter Syndrome (2021-2030) ($MN)
  • Table 11 Global Orphan Drugs Market Outlook, By Gaucher Disease (2021-2030) ($MN)
  • Table 12 Global Orphan Drugs Market Outlook, By Fabry Disease (2021-2030) ($MN)
  • Table 13 Global Orphan Drugs Market Outlook, By Hypoparathyroidism (2021-2030) ($MN)
  • Table 14 Global Orphan Drugs Market Outlook, By Hematologic & Immunologic Diseases (2021-2030) ($MN)
  • Table 15 Global Orphan Drugs Market Outlook, By Hemophilia (2021-2030) ($MN)
  • Table 16 Global Orphan Drugs Market Outlook, By Hereditary Angioedema (2021-2030) ($MN)
  • Table 17 Global Orphan Drugs Market Outlook, By Infectious diseases (2021-2030) ($MN)
  • Table 18 Global Orphan Drugs Market Outlook, By Neurological diseases (2021-2030) ($MN)
  • Table 19 Global Orphan Drugs Market Outlook, By Huntington's Disease (2021-2030) ($MN)
  • Table 20 Global Orphan Drugs Market Outlook, By Alzheimer's Disease (2021-2030) ($MN)
  • Table 21 Global Orphan Drugs Market Outlook, By Drug Type (2021-2030) ($MN)
  • Table 22 Global Orphan Drugs Market Outlook, By Non-Biologics (2021-2030) ($MN)
  • Table 23 Global Orphan Drugs Market Outlook, By Biologics (2021-2030) ($MN)
  • Table 24 Global Orphan Drugs Market Outlook, By Sale (2021-2030) ($MN)
  • Table 25 Global Orphan Drugs Market Outlook, By Prescribed (2021-2030) ($MN)
  • Table 26 Global Orphan Drugs Market Outlook, By Generics (2021-2030) ($MN)
  • Table 27 Global Orphan Drugs Market Outlook, By Drug (2021-2030) ($MN)
  • Table 28 Global Orphan Drugs Market Outlook, By Rituxan (2021-2030) ($MN)
  • Table 29 Global Orphan Drugs Market Outlook, By Revlimid (2021-2030) ($MN)
  • Table 30 Global Orphan Drugs Market Outlook, By Imbruvica (2021-2030) ($MN)
  • Table 31 Global Orphan Drugs Market Outlook, By Opdivo (2021-2030) ($MN)
  • Table 32 Global Orphan Drugs Market Outlook, By Darzalex (2021-2030) ($MN)
  • Table 33 Global Orphan Drugs Market Outlook, By Pomalyst (2021-2030) ($MN)
  • Table 34 Global Orphan Drugs Market Outlook, By Other Drugs (2021-2030) ($MN)
  • Table 35 Global Orphan Drugs Market Outlook, By Distribution Channel (2021-2030) ($MN)
  • Table 36 Global Orphan Drugs Market Outlook, By Online Pharmacies (2021-2030) ($MN)
  • Table 37 Global Orphan Drugs Market Outlook, By Hospital Pharmacies (2021-2030) ($MN)
  • Table 38 Global Orphan Drugs Market Outlook, By Retail Pharmacies (2021-2030) ($MN)
  • Table 39 Global Orphan Drugs Market Outlook, By Other Distribution Channels (2021-2030) ($MN)

Note: Tables for North America, Europe, APAC, South America, and Middle East & Africa Regions are also represented in the same manner as above.

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Jeroen Van Heghe

Manager - EMEA



Christine Sirois

Manager - Americas


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