8^th World congress on Rare Diseases & Orphan Drugs Nov 21-22, 2018 | Paris, France

                                                                         (Mattie Robinson, President)

On behalf of the organizing committee for the World Congress for Rare Diseases and Orphan Drugs, I am pleased to welcome you to the 8^th annual event convening here in Paris, France on November 21^st-22^nd, 2018.

The long-standing academic and creative history of Paris make it the ideal location for this Conference. May your observations and interactions during this two-day event further your progress toward improved quality of life for people living with rare diseases worldwide.

The 8^th World Congress on Rare Diseases & Orphan Drugs will highlight the latest innovations in understanding and treating Genetic Rare Diseases while providing a forum to examine challenges in Rare Disease Diagnosis. I invite you to use this time to forge purpose-driven collaborations with the regulatory authorities, researchers, and additional esteemed individuals in attendance from all over the world.

The organizing committee and I recognize information exchange as a vital function of international conferences. Throughout the two-day conference, there will be opportunities for networking and information exchange with leading scientists and researchers, friends and colleagues as well as sponsors and exhibitors.

I look forward to meeting you all during our time at the 8^th World Congress on Rare Diseases & Orphan Drugs. I hope that the experience leaves you with fresh ideas and renewed clarity for your continued success.

Respectfully yours,

Mattie Robinson, MS, MA
President
Micromattie Consulting Inc., USA

The Euro Orphan Drugs 2018 Conferences  bring together the senior level attendees and speakers from both academia, clinical research institutes, Clinical laboratories, Hospitals and from the top Biopharmaceutical companies from across the globe. Participants includes Directors, Deans, Research Professors, VPs, CTOs, Laboratory heads, Drug Developers, Clinical Trials Researchers, clinical trials sponsors, CROs, Medical doctors, & Researchers working in the sector of:

• Drug Discovery & Development
• Personalized, Translational, Experimental, & Regenerative Medicine
• Clinical Trials & Clinical Research
• Rare & ultra-rare Diseases
• Genetic Diseases
• Targeted Therapy, Cell & Gene Therapy
• Molecular Diagnostics & Diagnostic Product developers
• Patient Advocacy, Market Access, Pricing & Reimbursement
• In-Licensing/Out-Licensing
• Regulatory & Medical Affairs
• Commercial Development of Drugs
• Health Economics/Outcomes Research (HEOR)
• Orphan Foundations/Associations
• Experts working in Big Data & Digital Health
• Oncologist and Cancer experts
• Immunology & Inflammatory Disease
• Other Rare Disease & Therapy areas of: Neurology, mental health, neuromuscular disorders
• Patient Organizations
• HCP & Policy Makers
• Pharma Industry

Why Attend?

This two days of Orphan Drugs Congress will offer an interactive and most compressive sessions & networking with global experts working in the Rare Disease & Orphan Drugs segment. Orphan Drugs 2018 Paris Congress will help in gaining insights & to upgrade knowledge towards current activities growing globally in the Orphan Drugs sector starting from Laboratory research to field research, clinical trials to orphan drugs development, pricing and reimbursement, commercialization of orphan drugs & regulatory policies etc.

Orphan Drugs 2018 comprises of well organized scientific program stuffed with interactive sessions, one to one discussion rounds, industry academia interaction programs, industrials presentations, special regulatory sessions, and networking sessions.

Get Insights of:

• Key market trends of Orphan Drugs market globally
• Challenges the emerging markets facing
• Investment opportunities in the Orphan Drugs market
• Global regulatory landscape in the Rare Disease & Orphan Drug sector
• Barriers and opportunities in Rare Disease Diagnostics & Medical device market
• Pricing & reimbursement strategies in the Orphan Drugs sector
• Clinical trials details in Rare Disease & Orphan Drugs

• Overview of Rare Diseases & Orphan Drugs
• Types of Rare Diseases
• Genetic Rare Diseases
• FDA approval process of Orphan Drugs Products
• Challenges in Rare Diseases Diagnosis
• Rare Diseases Treatment Landscape
• Orphan Drug Pricing Reimbursement and Patient Access
• Ethical issues in Funding Orphan Drugs Research and Development
• Orphan Drugs Development Barriers
• Orphan Drugs Development: Current Trends & Future
• Clinical Trials & Clinical Research in Rare Diseases & Orphan Drugs
• Patient Concerns on Orphan Drugs
• Legislation & Regulatory policies in Orphan Drugs & Rare Disease Research
• Funding Opportunities in Rare Disease & Orphan Drugs Research
• Economical Perspective in Rare Diseases
• Designating Orphan Products: Drugs and Biologicals
• Developing Products for Rare Diseases & Conditions

List of Rare Disease Registries in Europe

• EUROFA
• EUROMAC
• EUROPAC
• European Porphyria Registry
• GPOH-registry sickle cell disease
• PROGNOSIS
• SCETIDE
• Thromboreductin-Registry
• EURO-WABB
• ECARUCA
• CEDATA-GPGE

For more detailsed list PS: European Rare Disease Meetings & Rregistries

List of research infrastructures useful to Rare diseases & orphan Drugs Research in Europe

• BBMRI-ERIC- Austria
• EORTC- Belgium
• APTEEUS- France
• ECRIN- France
• ERINHA- France
• GENETHON- France
• Orphanet- France
• OrphanDev- France
• PHENOMIN- France
• ChemBioNet- Germany
• CTSR- Germany
• EUMINAfab- Germany
• CIDSTEM- Italy
• EATRIS- Netherlands
• ECARUCA- Netherlands
• SEFALer- Spain
• INCF-DataSpace- Sweden
• COMET- UK

For more about the: List of Research Infrastructures in Orphan Drug & Rare Disease Conference & European Research Institutes

Updated List of Orphan Drug Products in Europe

• ARZERRA
• BAVENCIO
• BESPONSA
• BLINCYTO
• CARBAGLU
• CERDELGA
• CEPLENE
• DACOGEN
• DARZALEX
• DELTYBA
• FIRAZYR
• GAZYVARO
• HETLIOZ

For more details of Clinical trials in Orphan Drugs and Approved Orphan Drugs in Europe, PS: Euro Orphan Drug Conferences 2018 Paris

List of the latest marketing authorisations and Orphan Medicinal Products Designations

Overview of Orphan Medicinal Product Designation Procedure (2000-2018)