About Conference
Welcome Message
With immense pride and pleasure, we cordially welcome participants from all over the globe to our “4th World Congress on Rare Diseases and Orphan Drugs” which is scheduled on August 19-20, 2020 in Frankfurt, Germany. Rare Diseases Meetings 2020 plans to assemble professors, scientists and researchers related to diagnosis of Rare or Orphan diseases, formulation of orphan drugs and research, particularly to share their research experiences and bring awareness regarding these diseases to the outside world especially young researchers and student delegates.
About conference
We are delighted to invite all the participants over the globe to attend the 4th World Congress on Rare Disease and Orphan Drugs, which will be held during August 19-20, 2020 at Frankfurt, Germany. “Rare Diseases Meetings 2020 conference is to assemble professors, scientists and researchers related to diagnosis of Rare or Orphan diseases,
Rare Diseases Meetings 2020 provides an excellent platform for researchers to enunciate their advances in Rare Diseases & Orphan Drugs related sessions ranging from basic research to diagnosis, preparation of disease related biomarkers and clinical development. This is a prestigious conference which brings together; about the speech provoking topics and recent research in the field of infectious diseases related issues related to novel therapies on rare diseases and orphan drugs.
Target Audience
- Pathologists
- Infectious Diseases Specialists
- Pharmacists
- Epidemiologists
- Health Care Professionals
- Microbiologists
- Bacteriologists
- Virologists
- Parasitologists
- Mycologists
- Researcher Scholars
- Training Institutes
- Pharmaceutical Companies
- Rare Diseases Associations
- Orphan Drugs Companies
- Students
For Scientific Sessions please go through the link:
For Abstract Submission please go through the link:
Market Analysis
Market Research Growth on Rare Diseases and Orphan Drugs
The Rare Diseases are distributed in such a way that four fifth of the cases accounted by some 350 Rare Diseases. About only 5% of rare diseases are having approved drug treatment with only 326 new drugs being approved by the FDA and brought in to the market. Rare diseases are rare and often debilitating or even life-threatening diseases or conditions with a prevalence of 0.65%-1%, the cause of rare diseases is mostly genetic in nature and sometimes it may be as a result of infections or degenerative causes.
In 2018, Orphan Drug sales were of the order of 93 billion. Orphan Drugs represented 35% of the industry’s new drug approvals. The genetic diseases are subdivided by the therapeutic area, which is leading the global market in the past will show similar traction in the coming next eight years. This segment is anticipated to be valued at US$ 56,241 by the end of 2025. According to Statistics of MRC, the Global Orphan Drug market is estimated at $145.89 million in 2018 and is expected to reach $265.63 million by 2022 growing at a CAGR of 10.5% from 2018 to 2022.
Market Report of Orphan Drugs in European region
Between 5,000 and 8,000 distinct rare diseases exist, affecting between 6% and 8% of the population in total – in other words, between around 27 million and 36 million people in the EU. Most people suffer from diseases affecting fewer than 1 in 100,000 people.80% of rare diseases have identified genetic origins, and affect between 3% and 4% of births. Other rare diseases are due to degenerative and proliferative causes. Symptoms of some rare diseases may appear at birth or in childhood, including spinal muscular atrophy, lysosomes storage disorders, patent duct us arteriosus (PDA), familial adenomatous polyposis (FAP) and cystic fibrosis. More than half of rare diseases appear during adulthood, such as renal-cell carcinoma and acute myeloid leukemia.
Target Audience
- Professors
- Specialists
- Researchers
- Students
- Industrial delegates
- Academia and Research
- Scientists
- Biomedical companies
- Healthcare Professionals
- Infectious Diseases Specialist
- Directors, Board Members, Presidents, Vice Presidents, Deans and Head of the Departments Scientists
- Rare Diseases Associations and Societies
- Patient Group
- Business Entrepreneurs and Industrialists
Why to attend?
Join the Rare Diseases and Orphan Drugs Congress to keep up to date with the industry and to learn from our speaker panel, bringing you important new case studies, people sharing their research views and reports on these relevant topics.
- Gain knowledge of different rare diseases and their unique challenges, as well as how treatment methods can be transferred to other rare diseases
- Introducing cell and gene therapies to rare disease treatment - learn how gene therapy methods can improve the treatment of rare diseases and why it is becoming more commercially successful
- Learn about how the regulatory landscape for drug approvals is different between countries, and why it is important to keep informed about the regulations and guidelines of each region
- These diseases are responsible for rising healthcare costs, lost productivity, and long-term disability.
- It is a chance to communicate with individuals over the globe concentrated on finding out about Rare diseases and orphan drugs.
Scope and Importance of Rare Diseases and Orphan Drugs
Rare diseases
A rare disease is any disease that affects a small percentage of the population. In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discovering treatments for it, except by the government granting economically advantageous conditions to creating and selling such treatments.
Most rare diseases are genetic and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30% of children with rare diseases will die before reaching their fifth birthday
Orphan drugs
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.“Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare.
Major Associations Worldwide
· National Organization for Rare Diseases
· Canadian Organization for Rare Diseases
· Organization for Rare Diseases India
· The Boler-Parseghian Center for Rare & Neglected Diseases
· Cystic Fibrosis Foundation
·European Union Committee of Experts on Rare Diseases
· Multiple Myeloma Research Foundation
· U.S. Food and Drug Administration
·Birmingham children’s Hospital
·EURORDIS
·Every Life Foundation for Rare Diseases
·Global Genes
·Rare Diseases South Africa
. Short Bowel Syndrome Foundation
. Rare Disorders Society Singapore
· Genetic and Rare Diseases Information Center (GARD)
· Students4RareDiseases
· Malaysian Rare Disorders Society
· New Zealand Organization for Rare Disorders (NZORD)
·Rare Disease United Foundation
·Rare Diseases Denmark
· Genetic Alliance UK
·Rare Voices Australia
·BioPontis Alliance for Rare Diseases
. Rare Diseases Malta
· The International Rare Diseases Research Consortium (IRDIRC)
· Genetic Diseases Association - UAE
· National Tay-Sachs and Allied Diseases (NTSAD)
Top Universities Worldwide
· Harvard University
· University of Oxford
· University of Cambridge
· Rare Genomics Institute
· GMEC, The Global Medical Excellence Cluster
· University of Zurich
· Stanford University
· Cambridge University
· Yale University
· Emory University
· Karolinska University
· John Hopkins University
· Newcastle University
· University of Valencia
· Osaka University
·McMaster University
Hospitals Associated with Rare Diseases Research Worldwide
·The Manton Center for Orphan Disease Research (Boston, USA)
·US hospital for rare disease research (USA)
·NORD- National Organization for Rare Disorders (USA)
. Chicago Rare Disease Foundation (Chicago, USA)
· National Institute of Health (NIH) funds research consortia to study more than 200 rare diseases (USA)
. Children’s Hospital of Pittsburgh (Center for Rare Disease Therapy) (USA)
·Birmingham children’s Hospital (NHS Foundation Trust) (UK)
·The Children’s Hospital of Philadelphia (USA)
Industries Associated with Rare Diseases Research Worldwide
. Abbvie
· Actelion
· AFM-Telethon
· Alexon
· Amgen
· Amicus Therapeutics
. AstraZeneca
· Baxter
· Bayer
· Biogen
· Bio Marin
· Bioxcel
· Bristol-Myers Squibb
· Celgene
· Cyan
· Eli Lilly
·Genentech
· Gilead Sciences
·GlaxoSmithKline
·Johnson & Johnson
· Leadiant Biosciences
· Lumos Pharm
· Medivir
· Merck
· Novartis
· Novo Nordisk
. Orphan Swiss
·Orphan Europe
·orphan reach
·Pfizer
· Pharmacyclics
· Pharm Amar
· Pharm
·Prom etic Life Sciences
· Recordation Spa.
· Roche
· Sanofi Genzyme
. Sarepta Therapeutics
· Shire
· Spectrum Pharmaceuticals
· Swedish Orphan Biovitrum
· Takeda
· Teva
· Ultragenyx
· Vertex Pharmaceuticals
· Quintile Sims
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