Meet Inspiring Speakers and Experts at our 3000+ Global Events with over 1000+ Conferences, 1000+ Symposiums and 1000+ Workshops on Medical, Pharma, Engineering, Science, Technology and Business.

Explore and learn more about Conference Series : World’s leading Event Organizer

Conference Series Conferences gaining more Readers and Visitors

Conference Series Web Metrics at a Glance

  • 3000+ Global Events
  • 100 Million+ Visitors
  • 75000+ Unique visitors per conference
  • 100000+ Page views for every individual conference

Unique Opportunity! Online visibility to the Speakers and Experts

Renowned Speakers

Amrita Dosanjh

Amrita Dosanjh

Rady Children’s Hospital, Scripps Hospital USA

M Khalid Ijaz

M Khalid Ijaz

City University of New York USA

Khalid Mubarak Bindayna

Khalid Mubarak Bindayna

Arabian Gulf University Bahrain

Thomas Licker

Thomas Licker

Infection Control Technologies USA

Grażyna Rydzewska

Grażyna Rydzewska

UJK, Kielce Portugal

Rashmi Gopal-Srivastava

Rashmi Gopal-Srivastava

National Institutes of Health USA

Leena Bhattacharya Mithal

Leena Bhattacharya Mithal

Northwestern University USA

Recommended Global Pharmaceutical Sciences Webinars & Conferences

Asia Pacific & Middle East


About Conference

The Orphan Drugs Conferences Committee is pleased to announce the "4th World Congress on Rare Diseases & Orphan Drugs," which will be held in Paris, France from September 25-26, 2024.

Orphan Drugs 2024 represents an international conference dedicated to rare diseases, encompassing genetically caused rare conditions and cutting-edge research and development in various fields. This event serves as a global platform that addresses rare conditions worldwide and focuses on the exploration and development of new treatments.

The theme of the conference is "Pioneering Innovation for Rare Diseases: Bridging Science, Hope, and Care."

A rare disease, as defined by the World Health Organization, is a condition that affects one in every 1000 people, but in today's context, these rare diseases are not as uncommon as the name suggests. They have garnered significant attention from the pharmaceutical industry and scientific community over the past three decades.

This global congress on Orphan Drugs and rare diseases offers an interdisciplinary platform for experts to present their latest research findings and discuss emerging advancements and trends in the field of rare conditions.

The Congress will bring together experts from all segments of the value chain, facilitating interactions with decision-makers in your area of interest and fostering collaboration for future research endeavors. The conference's objective is to bridge the gap between industry and academia by uniting biotechnologists, scientists, researchers, medical professionals, innovators in orphan medicine, professors, research organizations, and experts from the pharmaceutical industry, as well as startup companies and key players in the industry.


Why to Attend?

Join us and connect with a global audience focused on Orphan Medicines and related advancements. This exclusive event provides a unique opportunity to engage with the largest gathering of stakeholders in the Orphan Medicines community. You can showcase your innovations, share valuable insights, network with current and potential professionals, host a cutting-edge research workshop, and elevate your brand visibility during this two-day conference. With renowned speakers, the latest research findings, breakthroughs, and the most recent updates in Orphan Medicines, this conference is an exceptional platform.

Target Audience:

Professors and Academic Faculty
CEOs and Founders of Companies
Research Groups
Representatives from Regulatory Authorities
Patent Attorneys
Business Development Managers
Directors, Presidents, and Vice Presidents
Industry Research Scientists and Chief Scientific Officers

Sessions & Tracks

Track 01: Orphan Drug Therapeutics

Orphan therapeutics is pharmaceuticals used to diagnose, help, or treat life-changing or life-altering conditions or diseases that are rare. Because of the small case groups needed for clinical trials, orphan medicine development is particularly delicate. By description, rare conditions affect smaller than Americans, with some affecting only a many hundred. There are about 7000 circumstances like this. Because the pharmaceutical business has little interest in developing and promoting treatments for a bitsy number of cases, these medicines are appertained to as "orphan" medicines. The exceptionally high cost of bringing a remedial product to the request would not be covered by prognosticated deals, according to medicine makers. As a result, the prospective request for new pharmacological treatments is minimal, and the pharmaceutical assiduity would suffer a financial loss as a result. The FDA has handed several gratuities and impulses to medicinal and exploration businesses to help with drug development and treatment choices for orphan diseases.

  •  Challenges in Orphan Drug Development
  •  Drug Repurposing/Repositioning
  •  Regulatory Considerations for Drug Repurposing
  •  Future Perspectives

Track 02: Rare Genetic Diseases/Disorders

A genetic complaint is a medical condition caused by one or further gene mutations. A chromosomal abnormality or a mutation in a single gene (monogenic) or multitudinous genes (polygenic) might beget it. Because of the high number of inheritable abnormalities, one out of every twenty-one people is affected with a" rare" inheritable complaint (generally defined as affecting lower than 1 in people). The maturity of inheritable conditions is rare in and of itself. The prevalence of rare disorders is used to define them rather than unifying pathological or clinical criteria. Rare conditions cover a wide range of pathologies and pathogenesis mechanisms since they're distinguished by their low frequency. Rare inheritable conditions serve as a springboard for uncovering new biology that has far-reaching counteraccusations for common mortal diseases. Changes in DNA are intertwined in the cause of around 80 rare diseases. Indeed if the symptoms are not constantly apparent, numerous conditions are present throughout a person's life. Rare Endocrine Genetic Diseases

  •   Paediatric Rare Genetic Disorders
  •   Rare Disease Genomics
  •   Detection of Rare Genetic Diseases

Track 03: Challenges in Rare Diseases

With strict rules executed for unprecedented infirmities, it's to a great degree delicate to share in phenomenal sickness ask about on a subject without the backing of a clinician also constraining the clinical trials limited keeping in mind the end thing to have a trust of filling them. Generally, there's an insufficiency of benefits for exploration on exceptional conditions of disseminates included with a little request degree and nonattendance of being written work and endured specialists are some huge challenges. 452 medicines and inoculations being produced for remarkable conditions use stimulating new coherent and particular data. A strong bit of the specifics, which offer look for after those anguish from one of the rights around unknown conditions, address imaginative better ways to deal with target affliction.

Track 04: Orphan Drugs Clinical Research

Clinical trials funding for orphan products have been shown to be a viable technique of fostering and promoting the development of novel, safe, and effective medical products for rare diseases and conditions. Orphan drug clinical development is both challenging and exciting. There is no single aspect that is truly unique to it; rather, it encompasses the majority of the challenges: design, outcomes, recruiting, ethics, cost, probability, and predictability. To solve these challenges, academic institutions, small and major pharmaceutical firms, patient representatives, and health authorities must work together to give assistance and creative ideas. The ultimate goal is to provide medications with a good benefit-risk ratio to individuals with unmet medical needs. FDA is focusing their efforts with a new funding opportunity to facilitate and move new therapies along in drug development in a safe and efficient manner by encouraging innovative clinical trial methods such as adaptive and seamless trial designs, modelling and simulations, and basket and umbrella trials to address the remaining unmet need and lack of treatments for the majority of rare diseases. These techniques are critical for effective trials and data analysis, which can help speed up drug development.

Track 05: Global Rare Diseases and Orphan Drug Market

A rare complaint, occasionally known as an orphan complaint, is one that affects a small proportion of the world's population. Indeed if symptoms may not develop incontinently, the maturity of these rare conditions is inherited and present throughout a case's continuance. There has been an increase in the circumstance of rare conditions each across the world. An orphan medicine is a pharmaceutical product used for the opinion, forestalment, and treatment of colorful rare conditions or conditions, according to the European Organization for Rare Conditions. These conditions are distinct from other conditions in that they've a veritably low frequencies rate relative to other conditions, and so are bought by a veritably limited patient population. The global orphan medicine request was valued atUS$147.56 billion in 2019 and is prognosticated to grow at a CAGR of 10 toUS$413.36 billion by 2030.

  •  Business Strategies
  •  Research & Development Activities
  •  Supply Chain Analysis
  •  Competitive Landscape
  •  Market Composition Analysis
  •  Analysis and Forecasts

Track 06: Advancing Research for rare diseases

Rare disease research is difficult due to the scarcity of patient information on the conditions. Because there are so few people affected, patient recruitment for clinical studies might be very difficult. To address these difficulties, a number of charities, patient advocacy organizations, and government agencies have developed online registries to collect patient data for researchers, doctors, and public health experts to use. It's crucial to remember that study into the aetiology, processes, and treatment approaches of rare diseases can help to advance advancements in common diseases. Rare diseases are biologically complex, and scientists are working hard to figure out what causes them and how they progress clinically. As researchers identify the molecular and genetic drivers of many diseases, we've witnessed amazing progress in the creation of medicines to treat patients with rare diseases.

Track 07: Living with a Rare Disease

Uncommon Dis temperatures are a different miscellaneous gathering of affections with a little in like way beside of their abnormality impacting with affecting the people. 80 of uncommon ails have honored inheritable origins and others may have some natural factors, there are wide and a many which are yet to be analyzed. Some uncommon ails are gained while some impacted people go about as Conditions audited. Notwithstanding anomaly, they speak to a critical medicinal and medical issue due to their event. For different uncommon distemperatures have no treatment, still in the event that it exists and if began on time as conveniently accessible to cases, there's a decent conjecture for them to be able for typical life. The issues of cases reflected and affected by uncommon distemperatures are related to the nonappearance of conclusion and accessible passing and their treatment or balancing exertion.

Track 08: Genomics and Proteomics of Rare Disease

Despite our understanding of the inheritable mutations that uphold uncommon conditions, little is known about their cellular impacts. Because developments in genome sequencing have far surpassed the throughout in functional follow-up examinations, functional studies on rare conditions have fallen behind gene identification. Likewise, pharmaceutical and biotech enterprises have many impulses to invest in medicine exploration and development for conditions that only impact a small number of people. Rare conditions have always been delved at the cellular position on an individual base. A study published lately phenotyped a library of rare complaint-causing variations and their wild- type alleles in detail. Whole-genome sequencing can give sapience into the genetics of rare diseases; it can miss up to half of the cases that are screened. The experimenters discovered that integrating proteomics and genomics increases rare complaint individual delicacy.

  • Protein Modification
  • Protein Translation
  • Genomic Studies
  • Transcriptomics

Track 09: Overview on Rare Diseases

An orphan drug is a pharmaceutical specialist that has been produced particularly to treat an uncommon remedial condition, the condition itself being indicated to as a fugitive sickness or uncommon infection. Fugitive specifics for the utmost part take after an indistinguishable executive advancement way from whatever other pharmaceutical item, in which testing concentrates on pharmacokinetics and pharmacodynamics, dosing, security, good and acceptability. In any case, some factual weights are dropped with an end thing to keep up enhancement force. The enhancement of fugitive specifics has been fiscally boosted through US law by means of the Orphan Drug Act of 1983.

Track 10: Patient Concerns for Orphan Drugs

Orphan medicines are vaccines or antibodies proposed to treat, avoid or assay an orphan complaint. Cases of rare affections incorporate inheritable conditions, uncommon tumours, infectious tropic affections and degenerative infections. The meaning of uncommon affections shifts cornerwise over wards yet generally considers disease commonness, soberness and presence of indispensable remedial options

Track 11: Gaming and Abuse of Orphan Drugs

The 1983 Orphan Drug Act was planned to give compose associations liberal motivators to produce particulars that may not generally appear to be salutary gambles. Organizations regularly apply for a fugitive drug assignment ahead of schedule in the drug advancement handle. On the off chance that a fugitive drug is at last championed for the request, the FDA postpones the US$2.17-million customer charge that associations must pay to the FDA for new specifics. The associations also get put credits for caused clinical- trial costs, alongside seven times of request restrictiveness, amid which time signatures for relative specifics are blocked.

Track 12: Pharmacology and Toxicology

Due to the unique characteristics of rare diseases, developing specifics for them might be delicate. The oddity of a condition makes medicine development delicate. Due to a deficit of people, well- designed clinical trials to demonstrate efficacy can be delicate to conduct, and studies substantial enough to determine major hazards are nearly insolvable to do. When no other options are available, cases may be ready to risk side goods in exchange for implicit benefits, but the benefit-to- detriment balance is delicate to quantify and may be unfavourable in some situations. Clinical pharmacology, a translational discipline, is a critical element of medicine development, including remedial development for rare conditions. Health directors have fiscal challenges when it comes to innovative medicinally, particularly orphan medicines. Individual rare diseases impact a small number of people, yet they inclusively affect a significant portion of the population. They bear our assistance.

  •  Drug Repurposing
  •  Safety Assessment
  •  Clinical Trials
  •  Patient Involvement
  •  Translational Research
  •  Clinical Pharmacology
  •  Orphan Drug Development

Track 13: Revolutionary Changes

Regrettably," rare diseases" has historically been low on the public health agenda, a fact that the 350 million individuals worldwide who suffer from these conditions are well apprehensive of. This is unsurprising since, while each of these rare conditions has too little a health burden to make a simple health-economic justification, rare conditions are allowed to be just as common as other diseases. Rare complaint discovery and operation is an illustration of an area that has served from technological advancements. The integration of new drugs for rare conditions into being care systems, on the other hand, calls into question a translational drug premise that there's a direct pathway from bench to bedside. In mindfulness of the impact of patterns of clinical interventions and associated health services on cases and their families' quality of life, a range of case-acquainted issues may be most applicable to examine for incremental curatives.

Track 14: Entrepreneurs Investment Meet

A stage meant to associate Entrepreneurs, Proposers, and Investors around the world. It's anticipated to make and encourage the most advanced and reasonable meeting place for drawing in individualities in worldwide business addresses, assessment, and prosecution of promising business studies. A fiscal specialist might discover the most noteworthy implicit enterprise openings widely, which give a great degree of profitability. For business people, this would be a perfect place to discover reasonable bookmakers and cohorts to begin as well as grow their business. In this way, it's an indefectible place to associate Entrepreneurs, Business Owners, Early Stage Companies, and Established Corporates with National or International Investors, Corporate Investors, and Implicit Business Mates.

Track 15: Rare Diseases and Ultra Rare Diseases

Interest in rare diseases has expanded in recent times, as substantiated by politicians and health authorities' dockets, yet ultra-rare conditions still admit far too little attention. Although no legal bracket of an “ultra-rare" complaint has been created, the National Institute for Health and Care Excellence has informally designated this subcategory for specifics having suggestions for diseases with a frequency of lower than one in per 50 000 people. The approach, by which ultra-rare complaint exploration should be conducted, as well as the question of quality, is both critical considerations. Although lower strict criteria may be used for orphan medicinal than for medicines treating more current conditions, this shouldn't be used as a reason to deny people with rare conditions the stylish available treatment. When a case is diagnosed with a rare or ultra-rare condition, having the correct croakers and treatment plan is just as vital as having a support system.

Abstract Submission Guidelines:

The 3rd World Congress on Rare Diseases & Orphan Drugs has officially initiated the abstract submission process and extends a warm invitation to prospective speakers. Abstracts can be conveniently submitted online through the Speaker abstract portal or via email at

It is essential to adhere to specific conditions: abstracts should not have been previously presented elsewhere, except as part of a thesis, and must not be under consideration by any other conference. The primary speaker is responsible for ensuring that all co-authors grant approval for abstract publication. All abstracts, whether intended for oral or poster presentation, must be exclusively in English, encompassing the title, text, author names, and affiliations.

Upon submission, provide essential information, including the presenting author's contact details, co-authors' names, and affiliation details for all authors.

The abstract submission process is a pivotal aspect of the congress selection procedure. We strongly encourage potential speakers to seize this opportunity, share their research findings with a global audience, and contribute to the discourse on rare diseases and orphan drugs. We eagerly anticipate your submissions and your presence at the 4rd World Congress on Rare Diseases & Orphan Drugs.

Market Analysis:

Rare diseases, often referred to as orphan diseases, are medical conditions that impact a small fraction of the population. In the United States, a rare disease is defined as a disorder affecting fewer than 200,000 individuals, while in Europe, it's considered rare if it affects fewer than 1 in 2,000 people. Orphan drugs are pharmaceuticals designed to treat these rare diseases. Developing drugs for such conditions can be intricate and costly due to the limited number of patients. Consequently, governments worldwide have introduced various incentives, including tax credits, grants, and regulatory support, to foster orphan drug development.

According to a report by Evaluate Pharma, the global orphan drug market is projected to reach $262 billion by 2026, with a compound annual growth rate of 12.1% from 2019 to 2026. This growth is attributed to several factors, including the increasing prevalence of rare diseases, an aging population, and advancements in technology and research.

While North America and Europe currently dominate the orphan drug market, accounting for more than 80% of global sales, other regions such as Asia-Pacific and Latin America are anticipated to witness substantial growth. This is due to improvements in healthcare infrastructure and heightened awareness of rare diseases in these regions.

The orphan drug market is expected to continue expanding, driven by various factors. However, the development of drugs for rare diseases remains a complex and challenging process, necessitating ongoing research and development efforts to meet the needs of individuals with rare diseases.

To Collaborate Scientific Professionals around the World

Conference Date September 25-26, 2024

For Sponsors & Exhibitors

Speaker Opportunity

Past Conference Report

Supported By

Journal of Infectious Diseases & Therapy Journal of Heavy Metal Toxicity and Diseases

All accepted abstracts will be published in respective Conference Series International Journals.

Abstracts will be provided with Digital Object Identifier by


  • Adult Rare Diseases
  • Advocacy Organizations
  • Awareness
  • Biotechnology
  • Clinical Management
  • Clinical Trials
  • Community Support
  • Data Sharing
  • Diagnosis
  • Diagnosis Challenges
  • Drug Development
  • Epidemiological Studies
  • Epidemiology
  • Ethical Considerations
  • Funding
  • Gene Therapy
  • Genetic Disorders
  • Genetic Testing
  • Genomics
  • Global Impact
  • Health Equity
  • Healthcare Access
  • Healthcare Equity
  • Healthcare Policy
  • Healthcare Systems
  • Innovations
  • International Collaboration
  • Multidisciplinary Collaboration
  • Orphan Drugs
  • Patient Advocacy
  • Patient Empowerment
  • Patient-Centered Care
  • Pediatric Rare Diseases
  • Pharmaceutical Industry
  • Policy
  • Precision Medicine
  • Public Health
  • Quality Of Life
  • Rare Disease Education
  • Rare Disease Guidelines
  • Rare Disease Networks
  • Rare Disease Research Centers
  • Rare Diseases
  • Regulatory Agencies
  • Regulatory Framework
  • Research
  • Telemedicine
  • Therapeutic Advances
  • Therapies
  • Treatment