Dublin, Jan. 22, 2025 (GLOBE NEWSWIRE) — The “Orphan Drugs – Global Strategic Business Report” report has been added to the ResearchAndMarkets.com’s offering.
The global market for orphan drugs was valued at USD 179.5 billion in 2023 and is projected to reach USD 394.7 billion by 2030, growing at a CAGR of 11.9% from 2023 to 2030. This comprehensive report provides an in-depth analysis of market trends. drivers and forecasts, helping you make informed business decisions.
Orphan drugs are becoming an important focus in the pharmaceutical industry, driven primarily by the need to treat rare diseases that affect a small percentage of the population but have historically been neglected due to limited market potential. These drugs have been developed specifically to treat, prevent, or diagnose rare medical conditions, often called orphan or rare diseases, that affect fewer than 200,000 individuals a year in the United States.
Growing awareness of rare diseases, coupled with advocacy efforts by patient groups and nonprofit organizations, has put pressure on pharmaceutical companies to focus on developing treatments for these underserved conditions. Government incentives, such as market exclusivity, tax credits and accelerated approvals, have played a crucial role in encouraging pharmaceutical companies to invest in the development of orphan drugs.
Growth in the orphan drug market is driven by several factors, including government incentives such as extended market exclusivity, grants, and reduced fees that make orphan drug development financially viable for pharmaceutical companies. Advances in biotechnology and genomics have also enabled the development of targeted therapies for rare diseases, increasing the number of possible treatments. Increasing prevalence and diagnosis rates of rare diseases, due in part to improved medical knowledge and awareness, are also contributing to the expansion of the orphan drug market.
Additionally, favorable regulatory frameworks and expedited approval processes from agencies such as the FDA and EMA are helping to bring orphan drugs to market faster. The role of patient advocacy groups, which actively fundraise, participate in research and advocate for patients with rare diseases, is another key driver of growth, as they contribute to an environment that supports innovation in drug development orphanage.
How is innovation shaping the orphan drug market?
Technological and scientific advances have significantly fueled the development of orphan drugs, particularly through advances in genomics, personalized medicine, and biotechnology. Innovations such as CRISPR gene editing, targeted therapies and monoclonal antibodies have revolutionized the ability to understand the underlying genetic and molecular causes of rare diseases, making it possible to create more effective and targeted treatments.
Additionally, the increasing use of biomarkers for accurate diagnosis and drug matching is paving the way for personalized orphan drug therapies that are tailored to patients’ individual genetic makeup. This has not only increased the efficacy of treatments, but also minimized side effects, making therapies more acceptable to patients suffering from chronic and rare diseases.
How do changes in health care dynamics and patient needs affect orphan drugs?
Changing dynamics in healthcare and evolving patient needs are impacting the orphan drug market in multiple ways. There is a growing recognition of the significant unmet medical needs faced by patients suffering from rare diseases, leading to a growing number of regulatory frameworks aimed at fast-tracking the approval process for these treatments.
As a result, regulatory bodies such as the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are increasingly adopting measures to support faster access to orphan drugs. Additionally, patient advocacy groups have played a key role in driving policy changes and raising awareness, which, in turn, has encouraged more clinical trials and funding in the orphan drug space. This growing awareness is also empowering patients to seek specialized care and treatments, leading to increased diagnosis rates for rare diseases and thereby expanding the potential market for orphan drugs.
Key insights:
- Market Growth: Understand the significant growth trajectory of the Biologics segment, which is expected to reach USD 286.3 billion by 2030 at a CAGR of 12.7%. The non-biological drugs segment is also set to grow at 10.0% CAGR during the analysis period.
- Regional Analysis: Gain insights into the US market, estimated at $72.3 billion in 2023 and China, projected to grow at an impressive CAGR of 13.1% to reach $30.2 billion by 2030. Discover growth trends by region other key markets, including Japan, Canada, Germany, and Asia-Pacific.
Report Features:
- Comprehensive Market Data: Independent analysis of annual sales and market forecasts in USD million from 2023 to 2030.
- In-depth regional analysis: Detailed insights into key markets, including the US, China, Japan, Canada, Europe, Asia-Pacific, Latin America, the Middle East and Africa.
- Company Profiles: Coverage of major players in the global orphan drug market such as AbbVie Inc., Actelion Pharmaceuticals Ltd., Alexion Pharmaceuticals Inc., Amgen Inc., AstraZeneca Plc. and more.
- Complementary updates: Get free report updates for a year to keep you informed of the latest market developments.
Main attributes:
| The report attribute | The details |
| Number of pages | 409 |
| Forecast period | 2023 – 2030 |
| Estimated market value (USD) in 2023 | 179.5 billion dollars |
| Projected market value (USD) by 2030 | 394.7 billion dollars |
| Compound Annual Growth Rate | 11.9% |
| Regions covered | global |
Main topics covered:
MARKET SUMMARY
- Orphan drugs: A beacon of hope for patients with rare diseases
- The slow growth of the orphan drug market: A side effect of COVID-19
- Competitive landscape
- Orphan Drugs – Market Share of Top Global Competitors in 2024 (E)
- Competitive market presence – strong/active/country/irrelevant for worldwide players in 2024 (E)
- Global market overview and analysis
- The global orphan drug market will continue to show signs of strong health
- Top 10 orphan drugs worldwide
- Biologics, Oncology and Hospital Pharma: Pulsating Segments of the Global Orphan Drug Market
- North America holds the commanding share of the global orphan drug market
- Recent market activity
- Influencer/Product/Tech information
TRENDS AND INDICATORS
- Increasing prevalence of rare diseases to drive the global orphan drug market
- Estimated prevalence of selected rare diseases (per 100,000 population): 2023
- The growth of the biopharmaceutical industry and the rise of orphan drugs
- List of Orphan Drugs Approved by the US FDA (2022-2024)
- List of orphan drugs approved in Europe between 2022 and 2024
- Advances in genetic and molecular research are transforming the treatment of rare diseases
- Oncology’s dominance of the orphan drug landscape: expanding horizons in rare and common cancer treatments
- Total number of new cancer cases by type: 2022
- The high cost associated with the development of orphan drugs – a major challenge
- Serious focus on research and development activity to drive market demand
- Global R&D Pipeline Value Composition: Orphan Drugs and Non-Orphan Drugs (2022-2026)
- Major orphan drug companies: Percentage of orphan drug sales as a percentage of total sales for major drug companies (2023)
- Select Recent Advances
- Favorable government policies promote market growth
- Technologies and strategies to stay ahead of the orphan drug development game
- Incentives & Guidelines for Injecting Adrenaline in Orphan Drug Development Efforts
- The rising trend in health care spending creates significant opportunities
- Global Healthcare Expenditure (In trillions of dollars) for 2017, 2019, 2021 and 2023
FOCUS ON SELECTED PLAYERS:Some of the 111 companies featured in this global orphan drug market include
- AbbVie Inc.
- Actelion Pharmaceuticals Ltd.
- Alexion Pharmaceuticals Inc.
- Amgen Inc.
- AstraZeneca Plc.
- Bayer AG
- Biogen Inc.
- BioMarin Pharmaceutical Inc.
- Boehringer Ingelheim GmbH
- Bristol-Myers Squibb Company
- Celgene Corp.
- CEL-SCI Corporation
- Concordia International Corp.
- Eli Lilly and Company
- F. Hoffmann-La Roche Ltd.
- Genzyme Corp.,
- Johnson & Johnson
- Merck & Co Inc.
- Merck Serono SA
- Novartis AG
- Pfizer Inc.
- Recordati SpA
- Sanofi SA
- Shire Plc.
- Vertex Pharmaceuticals
For more information about this report, visit https://www.researchandmarkets.com/r/ezu6d3
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