International Conference on Orphan Drugs And Rare

Orphan Drugs 2020 Conference is delighted to welcome the participants from all over the world to attend the prestigious International Conference on Orphan Drugs & Rare Diseases scheduled during March 16-17, 2020 at Berlin, Germany which based on the theme "A Step Towards Advancement in The Research and Treatment of Rare Diseases".

Scientific sessions of Orphan Drugs 2020 includes orphan drugs, clinical research on orphan drugs, challenges in rare Diseases treatment, Immunological Rare Diseases, Rare Oncology, Rare Neurological Disorders.

Berlin is the capital and the leading city of Germany. The destinations which makes Berlin special are Alexander platz, Berlin Bei Nacht, Berlin Cathedral, Berlin TV Tower, Brandenburg Gate, Charlottenburg Palace and Park, Oberbaum Bridge, Museum Island, The Rebuilt Reichstag.

Scientific Sessions

Orphan Drugs

An orphan drug is a pharmaceutical agent developed to treat medical conditions. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy in many countries and has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development.

Clinical Research on Orphan Drugs

A clinical trial is a medical study performed to check the effects of a new or already existing drug, of a biological cure or of a clinical gadget that might deal with or curb a ailment already identified. The most important aim of a clinical trial is to evaluate two or various organizations of subjects, by means of using two or a number of remedies in order to determine the efficacy of a drug or of a biological treatment. Clinical trials are carefully and ethically performed in order to protect sufferers towards unwanted adverse reactions and to allow series and accurate analysis of the information concerning the disease.

Orphan Drugs and Ethical Issues

This track outlines the ethical dilemma of funding orphan drug lookup and development. To date, moral aspects of precedence placing for research funding have now not been an issue of dialogue in the bioethics debate. Conflicting ethical responsibilities of beneficence and distributive justice appear to demand very different degrees of funding for orphan drug research.

Orphan Drugs and Potentiality

An orphan drug is referred medicinal agent which has been developed to treat a extraordinary scientific ailment or situation, the state of affairs itself being described to as a uncommon disease. Rare disease, additionally recognized as an "orphan disease", is any kind of sickness which affects a small share of the population. Majority of distinguished ailments are genetic, and as a end result are present in the path of the individual's whole life, even if symptoms doesn’t exhibit up immediately. Many individual ailments exhibit up early in life, and about two 30 % of teenagers with uncommon diseases will die before getting 5 year old. two An health problem might also be considered distinct in one area of the world, or in a team of people, then again nevertheless be ordinary in any other use or crew of people.

Orphan Drugs strategies and developmental trends

As per the brand new researches, the orphan drug market is now opening to display traits viewed in typical pharma markets. The vary of orphan drug approvals have expanded in current years. Both novel entrepreneurs and new warning symptoms serve as the predominant drivers of this cutting-edge orphan drug growth. The new industrial agency model of orphan drugs may want to offer a cohesive healthcare decision that allows pharma groups to enhance extra current areas of therapeutics, analysis, treatment, monitoring, and affected person support. Additionally, the improvement of orphan tablets is faced with some challenges that are unique to wonderful diseases. In spite of the challenges, innovative Competitive Intelligence (CI) strategies can provide an attentive and increased approach for the worthwhile development of orphan drugs.

Challenges in Rare Diseases Treatment

An uncommon ailment is characterized as a condition that influences under 200,000 individuals. This clarification was shaped by Congress in the Orphan Drug Act of 1983. There might be upwards of 7,000 uncommon infections. Just a couple of classifications of uncommon infections are followed when an individual is analyzed. These incorporate certain irresistible infections, birth imperfections, and diseases.

Clinical Case Studies on Rare Diseases

A scientific take a look at is an experimentation supposed to figure out extraordinary logical and consequence parts of proposed treatment. It is indispensable to recollect that there is no certification a proposed therapy will be compelling. Families ought to be set up to regulate the desire for partaking in a scientific preliminary with the perception that the therapy might also now not be best and should end result in a authentic poor result.

Immunological Rare Diseases

The immune gadget is delegated to defend the body from assaults from backyard or inside. Many diseases can have an effect on immune system decreasing its capability to defend self or inducing an peculiar response against exterior or inner antigens. Rare diseases affecting immune system current some difficulty in frequent with different rare diseases and some peculiarities due to the large variability in the disease's expression.

Rare Oncology

The wide vary of types of extraordinary oncological illnesses incorporate tumors of the body and hematological problem of the blood. Albeit solely these malignancies are uncommon, as a gathering, they talk to a noteworthy large part of humans with cancer. Certain attributes of these malignancies separate them from step by step basic kinds of disease.

Rare Neurological Disorders

Many rare diseases currently have little to no effective measures of diagnoses and treatments, primarily due to lack of representative patients and funding towards researching rare disease pathogenesis. For instance, a large group of rare diseases that have ineffective diagnoses and treatments are the neurological disorders. These disorders target the nervous system, which include the brain, spinal cord, and all the nerves that run throughout the human body.

Treatment and Advanced Therapies for Rare Diseases

Determination and the remedy of an exclusive sickness end up complicated due to the prerequisites that a number of human offerings suppliers may have restricted inclusion with the recognizable proof of the illness circumstance with the finding of extraordinary infections. Moreover, investigation earlier than sign starting or conclusion commencing time in the contamination can be testing. A scientific take a look at is an experimentation meant to decide distinctive logical and outcome parts of proposed treatment. It is crucial to recollect that there is no certification a proposed remedy will be compelling. Families ought to be set up to alter the want for partaking in a scientific preliminary with the grasp that the remedy can also now not be best and may want to end result in a authentic terrible result.

Market Analysis


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. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy in many countries and has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development.

Why to organize this conference?

1. To share learning and best follow from thought leaders and specialists in the field of Orphan Drugs

2. To engage with compatible individuals with shared interests

3. To inspire and generate awarness to Rare diseases

4. To make a discussion about the diseases and betterment of its treatment

5. To form new partnerships all over the world

6. To get results and diffuse messages face to face in an exceedingly value effective manner

7. To provoke action and collectively bring the evolution in the field of Rare Diseases and its treatment

8. To network and meet new people and organisations who are professional in the field of Orphan Drugs

9. To encourage PR and media coverage

Importance & Scope

There are greater than 6000 Rare Diseases. Many rare diseases are identified at the age of childhood, making diagnostic awareness and understanding on treatment and care specifically necessary for pediatricians. The Rare Diseases are so rare that rarity can lead to various troubles including: difficulties in obtaining timely, correct diagnoses; lack of experienced healthcare supervisors; useful, dependable and well timed statistics may additionally be challenging to retrieve; lookup things to do are very less; growing new prescribed drugs can also not be economically feasible; treatments are on occasion very expensive; and in developing countries, the issues are compounded via different resource limitations.

Orphan Drug 2020 is emerging up with an exciting and excessive knowledge gaining convention application this yr also which includes plenary lectures, symposia, workshops on a range of topics, poster shows and a number packages for contributors from at some stage in the world. We welcome you to join us at the International Conference on Orphan Drugs and Rare Diseases in Berlin the place you will be sure to have a significant ride with scholars from around the world. The Rare Diseases Conference aims to extend the reach, impact and trade of scholarly thoughts via revolutionary technology, determined commitment and awesome service. It objectives to provide international collaboration and trade of complete and ultra-modern data on basic, translational, and utilized medical research on Rare iseases and Orphan Drugs. The field of rare disease products is continuously growing, expecting to reach $180 billion by 2020, with a CGR of 10.5% just for orphan drugs – this is twice the growth rate of the overall prescription drug market.

Top Global Universities


Columbia University Medical Center, Columbia

New York University School of Medicine, New York

Stanford University Medical Center, California,america

University of Massachusetts Medical School, Massachusetts,america

University of Texas Southwestern Medical Center, Texas,America

East Carolina University, North Carolina

East Tennessee State University, Tennessee

University of Pittsburgh Medical Center, Pittsburgh

Texas Medical Center, Texas

Columbia University Department of Middle Eastern, South Asian, and African Studies, New York


University of Birmingham, United Kingdom

Paracelsus Medical University, Austria

Grodno State Medical University, Belarus

Medical University of Varna, Bulgaria

University of Strasbourg, France

University of Patras, Greece

National University of Ireland, Galway

Biomedical University of Rome, Italy

Medical Sciences Division, University of Oxford, England

Charité – University Medicine Berlin, Germany

Freie Universitat Berlin, Germany

Humboldt University of Berlin, Germany

University of Eastern Piedmont, Italy

Poznan University of Medical Sciences, Poland

Rostov State Medical University, Russia

Albert Szent-Györgyi Medical University, Europe

Ternopil National Medical University, Ukraine

Bukovinian State Medical University, Ukraine

Ternopil National Medical University, Ukraine

Dnipro Medical Institute of Conventional and Alternative Medicine, Ukraine

Donetsk National Medical University, Ukraine


Asia University, Taiwan

International Medical University, Malaysia

Gulf Medical University, United Arab Emirates

Columbia University Medical Center, United Arab Emirates

University Malaya Medical Centre, Malaysia

Goa Medical College, India

Rajiv Gandhi University of Health Sciences, India

Tokyo Medical University, Japan

Yerevan State Medical University, Armenia

Wenzhou Medical University, China

King Edward Medical University, Pakistan

Capital University of Medical Sciences, China

Middle East

Middle East Technical University, Turkey

Biomedical research in the Middle East

Shandong University

Hacettepe University Medical School, Turkey

Global Research Centers


De La Salle Medical and Health Sciences Institute

South Texas Medical Center

Boston Medical Center

University of Texas Southwestern Medical Center

University of Cincinnati Academic Health Center

Stanford University Medical Center

UCSF Medical Center

Federal Medical Center, Carswell

Vydehi Institute of Medical Sciences and Research Centre

Texas Medical Center


U.S. Army Medical Materiel Center – Europe

City of Hope National Medical Center

VU University Medical Center

University of Texas MD Anderson Cancer Center

University Medical Center Freiburg

BaÅŸkent University Medical and Research Center of Alanya

United States Army Medical Research and Materiel Command

VCU Medical Center

United States Army Medical Research Institute of Infectious Diseases

Fitzsimons Army Medical Center

United States Army Medical Command


Naval Medical Research Center

Rajiv Gandhi Cancer Institute and Research Centre

University of Texas MD Anderson Cancer Center

California National Primate Research Center

West China Medical Center

Columbia University Medical Center

Capitol Medical Center

National Institute of Virology

Naval Medical Center San Diego

LAC+USC Medical Center

Middle East

Biomedical research in the Middle East

Medical centers in the United States

BaÅŸkent University Medical and Research Center of Alanya

Sheba Medical Center

University of Utah Middle East Center

Naval Medical Research Unit Three

Columbia University Medical Center

David Grant USAF Medical Center

National Center for Medical Intelligence

Middle East Cancer Consortium

Hospitals Associated with Rare Diseases Research Worldwide


The Manton Center for Orphan Disease Research

US hospital for rare disease research

Chicago Rare Disease Foundation

National Institute of Health (NIH) funds research consortia to study more than 200 rare diseases

Children’s Hospital of Pittsburgh (Center for Rare Disease Therapy)

The Children’s Hospital of Philadelphia

The Christ Hospital

Medical City Dallas Hospital

Barnes-Jewish Hospital

Middlesex Hospital (Connecticut)

Massachusetts General Hospital


Johns Hopkins Hospital

Massachusetts General Hospital


University College London Hospitals NHS Foundation Trust

Birmingham children’s Hospital (NHS Foundation Trust)

University Hospital of Wales

Medical Center Leeuwarden

NewYork–Presbyterian Hospital

Helsinki University Central Hospital

Yale New Haven Hospital

St. Olav's University Hospital

Aarhus University Hospital


Ahmedabad Civil Hospital

Beijing Hospital of Traditional Chinese Medicine

Dhaka Medical College Hospital

West China Hospital

Bumrungrad International Hospital

Fortis Hospital

Asan Medical Centre (AMC)

Buddhist Tzu Chi Medical Foundation

National Hospital Organisation Medical centre

Prince Court Medical Centre (PCMC)

Gleneagles Hospital

Hong Kong Sanatorium and Hospital (HKSH)

Luke’s Medical centre

Wooridul Spine Hospital

King’s College Hospital London – Dubai Hills

Middle East

Clinic for Health and Medical Care

Magrabi Hospitals and Centers

IHH Healthcare

Magrabi Hospitals and Centers

American Hospital

NMC Hospital Group

Mediclinic Middle East (EHL HealthCare Management Services)

Holistic Healing Medical Centre

Mediclinic Al Jowhara Hospital

Mediclinic Al Mamora

Fund Allotment to Rare Diseases Research

Physician-scientists at 22 consortia will collaborate with representatives of 98 patient advocacy groups to advance clinical research and investigate new treatments for patients with rare diseases. The collaborations are made possible through awards by the National Institutes of Health — totaling about $29 million in the fiscal year 2014 funding — to expand the Rare Diseases Clinical Research Network (RDCRN), which is led by NIH’s National Center for Advancing Translational Sciences (NCATS).


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