2nd Annual Orphan Drugs: Development and Commercialization Summit
14-15

March 2019

Austria

Vienna

About

We are pleased to invite you to the 2nd Annual Orphan Drugs: Development and Commercialization Summit, scheduled for March 14-15, 2019 in Vienna, Austria. This premier B2B event provides the appropriate platform to engage and discuss ideas with your fellow peers, while facilitating a professional atmosphere and environment for good company representation and development. The summit will shed light on orphan drugs for rare diseases development, market access, reimbursement models, and upcoming perspectives for research and investment. It is an...

We are pleased to invite you to the 2nd Annual Orphan Drugs: Development and Commercialization Summit, scheduled for March 14-15, 2019 in Vienna, Austria. This premier B2B event provides the appropriate platform to engage and discuss ideas with your fellow peers, while facilitating a professional atmosphere and environment for good company representation and development. The summit will shed light on orphan drugs for rare diseases development, market access, reimbursement models, and upcoming perspectives for research and investment. It is an honour and privilege to invite you to participate in this Summit. We look forward to welcoming you in Vienna in March!

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Who Should Attend

Chief Executives, Vice Presidents, Directors, Heads, Leaders, and Managers specialising in:

  • Acute Myeloid Leukemia
  • Big Data
  • Cancer/Oncology
  • Cell/Gene Therapy
  • Clinical Research/Trials/Development
  • Cystic Fibrosis
  • Diagnostic Development
  • Drug Discovery & Development
  • Endocrinology
  • Genetic Diseases
  • Graft vs. Host Disease
  • Health Economics/Outcomes Research (HEOR)
  • In-Licensing/Out-Licensing
  • Market Access
  • Molecular Diagnostics
  • Neurology, Mental Health, Neuromuscular, Musculoskeletal Disorder
  • Orphan Foundations/Associations
  • Pancreatic Cancer
  • Personalized Medicine
  • Rare & Ultra-Rare Diseases
  • Regulatory & Medical Affairs
  • Stratified Medicine
  • Translational Medicine
  • Alliance Management
  • Bioinformatics
  • Cardiovascular, Pulmonological Disorders
  • Cell/Molecular Biology
  • Commercial Development
  • Dermatology, Ophthalmology, Urology & Nephrology Immunology
  • Digital Health
  • Duchenne Muscular Dystrophy
  • Experimental Medicine
  • Glioma
  • Haematology
  • Immunology
  • Innovative Medicine
  • Metabolic & Gastrointestinal (GI) Disorders
  • Multiple Myeloma
  • Non-Hodgkin Lymphoma
  • Ovarian Cancer
  • Patient Advocacy
  • Pricing & Reimbursement
  • Regenerative Medicine
  • Renal Cell Carcinoma
  • Targeted Therapy
Key Practical Learning Points of the Summit

Novel practice to facilitate orphan drugs development: designing clinical trials, meeting safety requirements, etc.
Regulatory assessment and recent insights to fast track reviews
Emerging science and tech, RWD generation to reach cost-effectiveness and success with accurate diagnosis and orphan drug discovery and research
Partnering with patients and external innovations to accelerate orphan drugs development
Recent developments in HTA, innovative value-based price models and reimbursement decision-making for advanced therapies to optimise market access
Leveraging licensing and M&A priorities and overcoming IP barriers
Growing investment perspectives, and evaluations of early opportunities for orphan drugs development
Speakers
Dr. Joachim Fruebis, US

Sr Vice President, Clinical Development

Bioverativ – A Sanofi Company

Dr. Simona Parator, IT

Medical Head, Cell & Gene Therapy

Novartis

Dr. Hanspeter Rottensteiner, AT

Head, Gene Therapy EU Drug Discovery Austria

Shire

Dr. Lutz Müller, CH

Project Leader, Pharmaceutical Sciences

F. Hoffmann-La Roche Innovation Center

Dr. Olaf Ritzeler, DE

External Innovation Lead, Rare Diseases Strategy & Business Development

Sanofi

Dr. Jodie Gillon, US

Global Medical Lead, Patient Engagement Rare Diseases Pfizer Innovative Health

Pfizer, Inc

Nigel Nicholls, UK

Director

BioMarin Pharmaceuticals

Dr. Markus Peters, US

Former Chief Commercial Officer / Strategic Consultant

Agilis Biotherapeutics

Dr. Micah Rose, UK

Technical Adviser, Health Economic Modelling, Scientific Advice

NICE

Leung Ming Yu, NO

Scientific Officer, HTA & Reimbursement

Norwegian Medicines Agency

Dr. David Schwicker, CH

Principal

ORPHA Strategy Consulting

Dr. Leone Atkinson, US

Executive Director / Rare Disease & Pediatrics Team / Global Clinical Development

Covance

Dr. Mohamed Abou El-Enein, DE

Junior Professor for Clinical Development of Regenerative Therapies & Head of Clinical Development Platform

Charité Medical University Berlin

Dr. Lincoln Tsang, UK

Partner

Arnold & Porter

Dr. Raghuram Selvaraju, US

Managing Director / Senior Healthcare Equity Research Analyst

H.C. Wainwright & Co., LLC

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