Orphan Drugs Market Size 2024-2028

The orphan drugs market size is forecast to increase by USD 146.1 billion, at a CAGR of 11.62% between 2023 and 2028. Market growth hinges on various factors such as a strong pipeline and recent drug approvals, a surge in rare disease occurrences, and incentives for developing orphan drugs. The robust pipeline and recent approvals signify a proactive approach to addressing medical needs, especially for niche conditions. The increasing prevalence of rare diseases highlights the urgency for innovative treatments and therapies. Additionally, incentives for orphan drug development, such as tax credits and market exclusivity, encourage pharmaceutical companies to invest in research and development for rare conditions. These combined factors drive the growth trajectory of the market, fostering advancements in healthcare and catering to unmet medical needs in specialized areas.

What will be the size of the Market During the Forecast Period?

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Market Dynamics and Customer Landscape

The market is driven by the urgent need to address rare medical conditions and orphan diseases, including oncologic diseases, metabolic diseases, immunologic diseases, and neurological diseases. With an increasing focus on personalized medicine, there's a rising demand for clinical trials and research to develop effective treatments. Challenges lie in navigating regulatory frameworks for orphan drug approvals, ensuring biological and non-biological drug safety, and managing outpatient hospital visits for administration. Despite these hurdles, innovations like immunomodulators offer promise in improving outcomes for individuals with non-life-threatening diseases. The Market is a specialized sector within the pharmaceutical industry dedicated to addressing rare medical conditions and orphan diseases. These conditions, which include oncological diseases, metabolic diseases, hematologic disease, and various other rare diseases, often affect a small portion of the population. As a result, developing drugs for these conditions requires extensive clinical trials and clinical research to ensure safety and efficacy. Within the oncology therapy area, orphan drugs are crucial for treating conditions such as Hodgkin lymphoma, acute myeloid leukemia, and cystic fibrosis. These drugs may be biological or non-biological and can target specific aspects of the disease, such as immunomodulators for enhancing immune responses.

Key Market Driver

The rising incidence of rare diseases is notably driving market growth. Rare diseases and a subset of non-orphan diseases are specifically treated with orphan drugs. Inherited genetic disorders, uncommon cancers, tropical infectious illnesses, and neurodegenerative illnesses are examples of rare diseases. In spite of their low incidence, rare diseases paradoxically affect a sizable portion of the population worldwide. According to the National Institutes of Health (NIH), 400 million people worldwide suffer from rare illnesses. One out of every two patients in the US with a rare disease is a child, according to the NIH. Monogenic or single-gene disorders are very common and frequently passed down through the germline.

However, many inherited rare illnesses have no diagnostic tools available. There are about 350 rare diseases that affect 80% of all patients with rare diseases. The 350 rare illnesses are therefore the subject of extensive research by numerous diagnostic and pharmaceutical companies. These studies have increased public awareness of the use of orphan products, which will fuel the market's expansion. Therefore, the high incidence of rare illness will increase the requirement for orphan drugs, which, in turn, will drive market growth during the forecast period.

Significant Market Trends

Strategic focus on alliances, partnerships, and M&A is an emerging trend in market growth. Strategic alliances and partnerships allow companies to co-develop and market products, increase their product portfolios, and reach a wider geographic audience. As a result, it is anticipated that the market's companies will keep looking for strategic opportunities to seize, which will fuel the market's expansion during the forecast period. In order to deal with impending patent expiration and the requirement to restock the strong pipeline, the market is currently embracing the M&A trend. A concentration on various therapeutics fueled by rising M&A activities and the adoption of cutting-edge, economical manufacturing technologies will pave the way for the development of novel orphan drugs.

Furthermore, the market players are using a variety of strategies for productive strategic alliances to overcome the difficulties posed by manufacturing procedures and translational biology. There have been several cross-disciplinary gene therapy collaborations that have been partnership-focused, including those with patient and advocacy groups, small and large biopharmaceutical companies, and data-driven technology companies. Hence, such collaborations will boost the growth of the market during the forecast period. 

Major Market Challenge

Delays in diagnosis are major challenges impeding market growth. A precise and timely diagnosis is the initial step to improving care for people with rare diseases. Due to inaccurate, expensive testing, general ignorance, and the asymptomatic nature of many rare diseases, the diagnosis of these conditions has historically been hampered. A delayed diagnosis can postpone the start of an effective course of treatment, which could cause serious, irreversible, and crippling disease to progress. The delayed diagnosis often results in premature deaths and delays in patient care. Numerous rare diseases can be misdiagnosed and take longer to diagnose because of the asymptomatic nature of many relatively common symptoms.

Further, the longer it takes to diagnose a rare disease, the more physician-patient consultations take place, which may make patients and caregivers feel frustrated and scared when the diagnosis is delayed. The average wait time for patients in developing and underdeveloped nations is 5–30 years between the onset of the first symptoms and the official diagnosis of their illness. Only 30% of cases around the world have access to genetic counseling because rare diseases have genetic roots.  Furthermore, the vast majority of patients around the world are initially given a false diagnosis. This results in the use of therapeutic treatments being restricted and causes medical interventions to be based on incorrect diagnoses. Hence, these limitations will negatively impact the global market during the forecast period.

Key Market Customer Landscape

The market forecasting report includes the adoption lifecycle of the market, covering from the innovator’s stage to the laggard’s stage. It focuses on adoption rates in different regions based on penetration. Furthermore, the market research and growth report also includes key purchase criteria and drivers of price sensitivity to help companies evaluate and develop their growth strategies.

Market Customer Landscape

Who are the Major Market Players?

Companies are implementing various strategies, such as strategic alliances, partnerships, mergers and acquisitions, geographical expansion, and product/service launches, to enhance their presence in the market.

• AbbVie Inc.: The company offers orphan drugs such as Humira and Venetoclax.

The market report also includes detailed analyses of the competitive landscape of the market and information about 15 market companies, including:

• Alnylam Pharmaceuticals Inc.
• Astellas Pharma Inc.
• AstraZeneca Plc
• bluebird bio Inc.
• Bristol Myers Squibb Co.
• CSL Ltd.
• F. Hoffmann La Roche Ltd.
• Gilead Sciences Inc.
• GlaxoSmithKline Plc
• Ipsen Pharma
• Johnson and Johnson Services Inc.
• Merck and Co. Inc.
• Novartis AG
• Novo Nordisk AS
• Pfizer Inc.

Qualitative and quantitative market growth analysis of companies has been conducted to help clients understand the wider business environment as well as the strengths and weaknesses of key market players. Data is qualitatively analyzed to categorize companies as pure play, category-focused, industry-focused, and diversified; it is quantitatively analyzed to categorize companies as dominant, leading, strong, tentative, and weak.

Market Segmentation

The market share growth by the hospital pharmacy segment will be significant during the forecast period. Inpatient medications are prepared, made, stored, and distributed by the hospital pharmacy department. Orphan diseases are rare diseases that require the quickest and easiest care and treatment. In general, the easiest place to meet these needs is a hospital pharmacy.

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The hospital pharmacy segment was valued at USD 58.80 billion in 2018. This segment has several benefits, including the ability to tailor each patient's dosing schedule. assisting medical professionals in making drug-related decisions and assisting patients in understanding and taking their medications. These uses are helping this market segment grow. Additionally, studies reveal that orphan drugs that were approved in the US, Europe, and Japan had respective market availability rates of 37.8%, 24.6%, and 52.4%. At the 24 tertiary public hospitals, the median availability of 31 orphan drugs was 20.8%. Thus, the hospital pharmacy segment in the market is expected to witness growth during the forecast period.

Which are the Key Regions for the Market?

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North America is estimated to contribute 39% to the growth of the global market during the forecast period. Technavio’s analysts have elaborately explained the regional trends and drivers that shape the market during the forecast period. The market growth in North America is mainly attributed to six driving factors, namely, new drug approvals, growing diagnosis of rare diseases, availability of reimbursement schemes, the high premium price of orphan drugs, the strong presence of key companies of orphan drugs, and incentives or encouragement from government regulatory agencies and non-profit organizations. Novel drugs have been flooding the US market in North America over the past ten years. To aid in the evaluation and development of products that show promise for the diagnosis and/or treatment of rare diseases, the US FDA established the Office of Orphan Products Development (OOPD). A record number of orphan drugs were approved and introduced in the US in 2022, providing patients with new treatment options. The market in North America is anticipated to experience growth during the forecast period as a result of such a favorable environment for the approval of new drugs.

Further, another key factor influencing the growth of the market in North America is the increasing prevalence of rare diseases. Due to the development of sophisticated and affordable genetic profiling, the diagnosis rate for rare diseases has been rising in the US and Canada over time. Approximately 7,000 rare diseases affect 25–30 million people in the US today, according to the National Institutes of Health (NIH) and the US Food and Drug Administration, with 250–280 new rare diseases being described each year. Additionally, about 350 well-known rare diseases affect 80% of all patients who receive a diagnosis of a rare disease. Thus, the high prevalence of rare diseases will help propel the market in North America during the forecast period.

Segment Overview

The market research report provides comprehensive data (region-wise segment analysis), with forecasts and estimates in "USD Billion " for the period 2024-2028, as well as historical data from 2018-2022 for the following segments.

• Distribution Channel Outlook
  • Hospital pharmacy
  • Specialty pharmacy
  • Retail pharmacy
  • Others

• Product Outlook 
  • Biologics
  • Non-biologics

• Region Outlook 
  • North America
    • The U.S.
    • Canada
  • Europe
    • U.K.
    • Germany
    • France
    • Rest of Europe
  • Asia
    • China
    • India
    • Others
  • Rest of World
    • Saudi Arabia
    • South Africa
    • Others

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Market Analyst Overview

The Market focuses on pharmaceutical agents developed for rare medical conditions, necessitating rigorous clinical trials and specialized screening services. These drugs cater to various diseases like oncological, metabolic, hematologic, immunologic, neurologic, and gastrointestinal conditions, including rare congenital abnormalities. Specific examples include treatments for Acute Myeloid Leukemia (AML), pancreatic cancer, and Alzheimer's disease. With advancements in biopharmaceutical companies, therapies for non-life-threatening diseases like hypertension and diabetes are also gaining attention. The market dynamics involve collaborations with oncology centers, adherence to FDA regulations, and considerations for temperature-controlled supply chains. Despite challenges such as healthcare expenditure and reimbursement policies, the market continues to innovate, especially in rare disease treatments, supported by organizations like the American Cancer Society.

Moreover, the Market encompasses pharmaceutical agents designed to address a range of rare diseases across various medical domains such as oncology, metabolism, hematology, immunology, neurology, transplantation, gastrointestinal, and dermatology. Conditions like ovarian cancer, multiple myeloma, Huntington's disease, and hemophilia fall under this category. Clinical trials play a pivotal role in testing these drugs, often involving community health centers. These drugs can be biological or non-biological and are specifically tailored for diseases like Hodgkin lymphoma, cystic fibrosis, and Duchenne muscular dystrophy.

Furthermore, leading drugs in this segment include Revlimid, Rituxan, Opdivo, Keytruda, Imbruvica, Soliris, Jakaf, Pomalyst, Darzalex, and Spinraza, targeting critical ailments like cardiovascular, respiratory, and central nervous system disorders. The market also explores online sales channels, especially for cardiovascular diseases and conditions like oral cancer. Key challenges in this market include maintaining a temperature-controlled supply chain, navigating FDA regulations, and ensuring access to enzyme replacement therapy and immunomodulators. Despite these challenges, ongoing clinical research in hematology, neurology, endocrinology, and immunotherapy continues to drive innovation in the orphan drugs space, making them increasingly accessible via online pharmacies and outpatient hospital visits.

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What are the Key Data Covered in this Market Research Report?

• CAGR of the market during the forecast period
• Detailed information on factors that will drive the growth of the market between 2023 and 2027
• Precise estimation of the size of the market size and its contribution to the market in focus on the parent market
• Accurate predictions about upcoming trends and changes in consumer behavior
• Growth of the market industry across North America, Europe, Asia, and Rest of World (ROW)
• Thorough market forecast analysis of the market’s competitive landscape and detailed information about companies
• Comprehensive market growth and forecasting analysis of factors that will challenge the growth of market companies

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