Orphan Drugs for Rare Diseases Summit 2018

Topics

• Novel drug modalities, assays & models, therapeutic strategies
• Cutting-edge science and tech to enhance rare diseases research, diagnosis and therapeutics
• Emerging approaches for effective, fast, affordable and successful orphan drugs development
• Employee wellbeing & health in relation to office design
• Insights into regulatory, pricing, reimbursement, commercialization, market access for rare disorders drugs

Who should Attend

Senior attendees with responsibilities in:

• Personalized Medicine
• Drug Discovery & Development
• Experimental Medicine
• Translational Medicine
• Stratified Medicine
• Innovative Medicine
• Cell / Molecular Biology
• Regenerative Medicine
• Diagnostic Development
• Molecular Diagnostics
• Targeted Therapy
• Cell / Gene Therapy
• Genetic Diseases
• Rare & ultra-rare Diseases
• Endocrinology
• Metabolic & Gastrointestinal (GI) disorders
• Cancer / Oncology
• Cardiovascular, pulmonological disorders
• Dermatology, ophthalmology, urology and nephrology Immunology
• Neurology, mental health, neuromuscular and musculoskeletal disorder
• Immunology
• Haematology
• Acute Myeloid Leukemia
• Non-Hodgkin Lymphoma
• Glioma
• Cystic Fibrosis
• Ovarian Cancer
• Pancreatic Cancer
• Duchenne Muscular Dystrophy
• Multiple Myeloma
• Renal Cell Carcinoma
• Graft vs Host Disease
• Market Access
• Clinical Research / Trials / Development
• Alliance Management
• Pricing & Reimbursement
• N-Licensing/Out-Licensing
• Patient Advocacy
• Commercial Development
• Regulatory & Medical Affairs
• Orphan Foundations/Associations
• Health Economics/Outcomes Research (HEOR)
• Big Data
• Bioinformatics
• Digital Health