Rare Diseases Market Analysis and Reports | USA Conference Series

Market Analysis - Rare Diseases 2018

Importance and Scope

A rare/orphan disease is outlined as a condition that affects fewer than 200,000 people. This definition was created by Congress within the Orphan Drug Act of 1983. There may be as many as 7,000 rare diseases and 70% of them have no form of treatment. Regulatory edges like longer market exclusivity, breakthrough designations, reduced fees and tax incentives are all encouraging investment. However, the marketing process and lifecycle for a rare disease drug are very different to a mass market product and require particular skills and knowledge.

Rare Diseases 2018 will be the best platform for all the doctors, researchers, renowned Scientists, research scholars, students who are working in this field across the globe under a single roof to exchange their knowledge related to Rare Diseases and Orphan Drugs. This international event is an effort to find an alternative for invasive imaging technique against rare diseases like Zellweger syndrome, Muscular Dystrophy, Alkaptonuria, Angelman syndrome, Prader-Willi syndrome, Tay Sachs disease, Fragile X syndrome, Gaucher disease as well as many others.

Why Boston?

Boston is placed among the top 30 most economically powerful cities in the world. Encompassing $363 billion, the Greater Boston metropolitan area has the sixth-largest economy in the country and 12th-largest in the world. Boston's colleges and universities exert a significant impact on the regional economy. Boston attracts more than 350,000 college students from around the world, who contribute more than US$4.8 billion annually to the city's economy. The city is home to a number of technology companies and is a hub for biotechnology, with the Milken Institute rating Boston as the top life sciences cluster in the country. Boston receives the highest absolute amount of annual funding from the National Institutes of Health of all cities in the United States. The city is considered highly innovative for a variety of reasons, including the presence of academia, access to venture capital, and the presence of many high-tech companies.

Conference Highlights

  • Different types of Rare Diseases
  • Clinical Research and Public Awareness
  • Mystery Diagnosis of Rare Diseases
  • Challenges in Rare Diseases Treatment
  • Rare Infectious Diseases and Immune Deficiencies
  • Rare Diseases in Cancer
  • Rare Diseases in Aging
  • Orphan Drugs- development trends and strategies
  • Clinical Research on Orphan Drugs
  • Orphan Drugs and Ethical Issues
  • Future Hereditary of Rare Diseases and Orphan Drugs
  • Patient organizations and their role in drug development or clinical research

Why attend?

In today's economic climate your business selections are as crucial as ever. 5th Annual Congress on Rare Diseases and Orphan Drugs (Rare Diseases 2018) allow you to maximise your time and marketing dollars whereas receiving immediate feedback on your new merchandises and services. Rare Diseases 2018 is organizing an outstanding Scientific Exhibition/Program and expects the world's leading specialists involved Rare Diseases and Orphan Drugs. Your company will benefit with outstanding exposure to the leaders in Rare Diseases and Orphan Drugs. Rare Diseases 2018 is a stimulating opportunity to showcase the new technology, the new products of your company, and/or the service your industry may offer to a wide-ranging international audience.

Target Audience

Join the Rare Diseases and Orphan Drugs community to keep up to date with the industry and to learn from our expert speaker panel, bringing you important new case studies and reports on this year’s relevant topics.

  • Directors, Board Members, Presidents, Vice Presidents, Deans and Head of the Departments
  • Researchers, Scientists, Faculties, Students
  • Rare Diseases Associations and Societies
  • Medical Colleges
  • Industrial delegates from Academia and Research
  • Industrial professionals from biomedical companies and healthcare sectors
  • Medical Devices Manufacturing Companies
  • Drug Manufacturing Companies and Industries
  • Laboratory Technicians and Diagnostic Companies
  • Business Entrepreneurs and Industrialists

A Unique Opportunity for Advertisers, exhibitors, and Sponsors at 5th Annual Congress o Rare Diseases and Orphan Drugs (Rare Diseases 2018): http://rarediseases.conferenceseries.com/sponsors.php

 

Statistics of Researchers, Academicians, and Industrial

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

·         EveryLife 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 Organisation 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)

·         Boston Children’s Hospital (The Manton Center for Orphan Disease Research) (Boston, 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

·         Alexion

·         Amgen

·         Amicus Therapeutics

·         AstraZeneca

·         Baxter

·         Bayer

·         Biogen

·         BioMarin

·         Bioxcel

·         Bristol-Myers Squibb

·         Celgene

·         cydan

·         Eli Lilly

·         Genentech

·         Gilead Sciences

·         GlaxoSmithKline

·         Johnson & Johnson

·         Leadiant Biosciences

·         Lumos Pharma

·         Medivir

·         Merck

·         Novartis

·         Novo Nordisk

·         OrPha Swiss

·         Orphan Europe

·         orphan reach

·         Pfizer

·         Pharmacyclics

·         PharmaMar

·         PhRMA

·         ProMetic Life Sciences

·         Recordati S.p.A.

·         Roche

·         Sanofi Genzyme

·         Sarepta Therapeutics

·         Shire

·         Spectrum Pharmaceuticals

·         Swedish Orphan Biovitrum

·         Takeda

·         Teva

·         Ultragenyx

·         Vertex Pharmaceuticals

·         QuintilesIMS

 

Glance at Market analysis report

Summary GBI Research, the leading business intelligence provider, has released its latest research “Orphan Disease Therapeutics Market to 2018 – Improved Understanding of Rare Diseases’ Heterogeneity and Novel New Clinical Trial Designs to Foster Innovation”, which provides insights into the orphan disease therapeutics market until 2018. It includes the geographical distribution of Fabry, Pompe, Mucopolysaccharidosis VI, Idiopathic Thrombocytopenic Purpura, Huntington’s disease and Ovarian cancer markets across the US, the top five countries of Europe and in Japan. The report provides competitive benchmarking for the leading companies and also analyzes the mergers, acquisitions and licensing agreements that shape the global markets. GBI Research’s analysis shows that the overall global orphan disease therapeutics market is expected to grow at a significant compound annual growth rate (CAGR) of 13.1% from $2.3 billion 2010 to $6 billion in 2018 in the US, the top five countries of Europe and Japan. Increasing awareness of the disease and drugs among patients and physicians, patent protection and exclusivity of Nplate and Promacta for ITP market, anticipated launch of new molecules such as ACR-16, AMR-101 and HD-02 for the treatment of HD and the approval of Avastin for the treatment of advanced ovarian cancer in Europe will drive the global orphan disease therapeutics market in the forecast period.

Annualized market data for the orphan diseases therapeutics market from 2004 to 2010, forecast forward to 2018.- Analysis of the leading therapeutic segments. These include Fabry Disease, Pompe Disease and Mucopolysaccharidosis VI, Idiopathic Thrombocytopenic Purpura, Huntington’s disease and Ovarian Cancer.- Analysis of the orphan diseases therapeutics market in the leading geographies of the world, which include the US, the UK, Germany, France, Italy, Spain, and Japan – Market characterization of the orphan diseases therapeutics market including market size, annual cost of therapy, and treatment usage patterns – Key drivers and barriers that have a significant impact on the market – Coverage of pipeline molecules in various phases of drug development – Competitive benchmarking of leading companies. The key companies studied in this report are Genzyme Corporation, Shire Plc, and BioMarin - Key M&A activities, licensing agreements that have taken place between 2010 and 2011 in the global orphan diseases therapeutics market.

The 2013 Orphan Drug Report from Evaluate, which launched today at the 2013 BIO international Convention, sheds light on the market dynamics of orphan drugs — pharmaceutical products aimed at rare diseases or disorders — projecting that sales will experience a compound annual growth rate of 7.4 percent between 2012 and 2018, nearly double that of the prescription drug market, excluding generics.  The report based on EvaluatePharma® data found that the worldwide orphan drug market is set to reach $127 billion by 2018, double that of the overall prescription drug market.

Market Growth of Rare Diseases Research

GBI Research’s analysis shows that the overall global orphan disease therapeutics market is expected to grow at a significant compound annual growth rate (CAGR) of 13.1% from $2.3 billion 2010 to $6 billion in 2018 in the US, the top five countries of Europe and Japan.

The 2013 Orphan Drug Report from Evaluate, which launched today at the 2013 BIO international Convention, sheds light on the market dynamics of orphan drugs — pharmaceutical products aimed at rare diseases or disorders — projecting that sales will experience a compound annual growth rate of 7.4 percent between 2012 and 2018, nearly double that of the prescription drug market, excluding generics.  The report based on Evaluate Pharma  data found that the worldwide orphan drug market is set to reach $127 billion by 2018, doubling that of the overall prescription drug market

The global orphan drugs market reached $84.9 billion in 2009 growing from $58.7 billion in 2006 from $54.5 billion in 2005.The market is expected to grow at a compound annual growth rate (CAGR) of nearly 6% to reach $112.1 billion by 2014.The U.S. accounted for 51% of the market in 2009 and is expected to grow at a CAGR of 8.9% to reach $65.9 billion by 2014.By 2020, orphan drugs will own 19% of the total share of prescription drug sale excluding generics, reaching a whopping $176 billion in annual sales, according to Andreas Hadjivasiliou, an analyst with Evaluatepharma.

Fund Allotment to Rare Diseases Research

Since 1989, NORD’s research grants have resulted in at least two FDA-approved products and the publication of numerous significant journal articles. Our more than 100 disease-specific research funds are supported primarily by patients and patient organizations.  Industry support is welcome and would demonstrate a commitment to the study of diseases for which there are few if any- other sources of funding.

NORD's Research Program provides seed money grants to academic scientists for clinical studies related to the development of diagnostics or treatments of rare diseases. Requests for proposals are posted once a year in the late winter or early spring. NORD's Research Program also includes the NORD/Roscoe Brady Lysosomal Storage Diseases Fellowships.

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).

On behalf of the Rare Diseases 2018 organizing committee, we are pleased to announce that the 5th Annual Congress on Rare Diseases and Orphan Drugs (Rare Diseases 2018) will be held from August 29-30, 2018 in Boston, USA. Rare Diseases 2018 provides a premier interdisciplinary platform for researchers to present the latest research findings and describe emerging technologies, and directions in rare diseases and orphan drugs issues. The conference seeks to contribute to presenting novel research results in all aspects of Rare Diseases and Orphan Drugs.

The conference's goal will to provide a scientific forum for all international prestige scholars around the world and enable the interactive exchange of state-of-the-art knowledge. The conference will focus on evidence-based benefits proven in clinical trials and scientific experiments.

The scientific program will focus on current advances in the clinical research, clinical trials, drug discovery, drug development, and drug treatment and orphan drug clinical trials with a particular focus on their role in maintaining the academic level in rare diseases and orphan drugs. In addition, mentoring sessions for poster presenters will provide guidance from world leaders for our experts of the future.