About Conference

We are happy to announce that, following the success of previous Rare Diseases Congress series, we are delighted to invite participants from around the globe to attend the 14th World Congress on Rare Diseases and Orphan Drugs, which will be held on June 02-03, 2025, in Amsterdam, Netherlands. This congress will feature keynote presentations, special sessions, workshops, symposiums, oral talks, poster presentations, and exhibitions focused on innovative approaches to rare disease treatment.

Rare diseases refer to medical conditions that affect a very small number of individuals. They are characterized by a wide diversity of disorders and symptoms, which can vary not only from disease to disease but also among individuals suffering from the same condition. Approximately 80% of these disorders have identifiable genetic origins, while others may result from infections, allergies, neurological issues, or environmental factors. A medicinal product developed for the treatment of a rare disease is known as an orphan drug. In typical market environments, the pharmaceutical industry often pays little attention to developing and marketing orphan drugs aimed at a limited patient population.

The significance of this conference is underscored by the global burden of rare diseases. Approximately 800 million people worldwide are affected by various rare diseases, including around 100,000 children born with thalassemia each year, many of whom receive no treatment. This raises major ethical concerns regarding global health issues and the treatment of orphan and rare diseases.

Target Audience:

• Pathologists
• Infectious Diseases Specialists
• Pharmacists
• Epidemiologists
• Healthcare Professionals
• Microbiologists
• Bacteriologists
• Virologists
• Parasitologists
• Mycologists
• Research Scholars
• Training Institutes
• Pharmaceutical Companies
• Rare Diseases Associations
• Orphan Drugs Companies
• Students

Why Attend?

The 14th World Congress on Rare Diseases and Orphan Drugs will address the serious challenges posed by rare diseases, which have diverse impacts on individuals, public health, and economies. These conditions contribute to rising healthcare costs, lost productivity, and long-term disabilities. The conference will gather a global audience of scientific leaders, academia, healthcare professionals, and orphan drug experts to discuss emerging treatments and diagnostics. By uniting the rare diseases community, presenting the latest developments, and identifying unmet treatment needs, this congress aims to accelerate research and drug development in rare diseases, ultimately improving patient outcomes. The event is designed to provide an educational forum that fosters collaboration among clinical, research, government, and patient communities, promoting a coordinated approach to advancing research, clinical care, care delivery coordination, and other public health interventions critical to enhancing outcomes in rare diseases and orphan drugs research.

For abstract submission, please visit the link:https://rarediseases.insightconferences.com/abstract-submission.php

Sessions and Tracks

Market Analysis

The global orphan drugs market size was valued at USD 170.49 billion in 2023. The market is projected to grow from USD 189.17 billion in 2024 to USD 468.58 billion by 2032, exhibiting a CAGR of 12.0% during the forecast period. North America dominated the global market with a share of 53.94% in 2023.

Orphan drugs are developed by the pharmaceutical industry for economic reasons but respond to the public health needs. These drugs are developed to treat patients suffering from serious diseases for which no treatment, or at least a satisfactory one, has so far been available. For instance, an article published by the Orphanet in 2023 stated that the number of rare diseases for which no treatment is currently available is estimated to be between 4,000 and 5,000 globally. Moreover, 25 to 30 million people are reported to be affected by these rare diseases in Europe.

According to the European Organization for Rare Diseases, orphan drugs are produced for diagnosis, prevention, and treatment of rare disorders. The oncology therapy area holds a considerably higher share in the global market due to emergence of new cancer-related diseases. Additionally, FDA approvals to a series of cancer-related orphan drugs are expected to result in increased commercialization of these drugs, especially in the oncology therapy area, during the forecast period. The market will derive growth from higher demand for immunomodulators, as they regularize or amplify the immune system. Rising prevalence of infectious diseases in developed as well as emerging nations and higher prices of medications are projected to fuel the demand for immunomodulators, thus contributing to the global orphan drugs market growth during the forecast period.

The COVID-19 pandemic had a positive impact on the market growth due to an increase in regulatory approvals. In 2021, the growth of the market continued primarily due to the resumption of research and development activities of market players. This growth is expected to continue during the forecast period, owing to the launch of various pipeline candidates for various indications by key players. Such trends indicate that the market is poised for steady growth during the forecast period.

ORPHAN DRUGS MARKET TRENDS:

One of the prominent trends in the global market is strong product launches for these disease indications. Globally, patients suffering from rare diseases are seeking efficient and potent treatment for these disorders. Increasing R&D initiatives and rising advocacy by patients’ groups for effective treatment are propelling the introduction of newer and more effective product offerings. These trends have led to the development and marketing of several blockbuster drugs for the treatment of many types of cancers, immunological diseases, and other rare disorders.

As per the Orphan Drug Report 2022 by Evaluate Pharma, it is estimated that the rise of top 10 orphan drugs blockbusters will be worth between USD 3.0 billion and USD 13 billion by 2026. Furthermore, it estimated that 40% of Johnson & Johnson’s pharmaceutical sales will come from orphan drugs, especially through blood cancer drugs, such as Imbruvica and Darzalex, which is expected to be worth over USD 23.0 billion by 2026. Thus, the introduction of such sophisticated orphan therapeutics by established and emerging companies have been instrumental in boosting the adoption of these drugs, which is expected to aid the growth of the global market during the forecast period.