Featuring

Orphan Drugs & Rare Diseases Global Congress 2020

Virtual Live Event

Days
Hours
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20th - 21st October 2020 - 2 Days Conference
London, UK

Pharma Conference London UK

Orphan Drugs & Rare Diseases

Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases 2020 Europe. It’s the 12th in the series of our Flagship tri-annual Orphan Drugs and Rare Diseases event, this congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs.

The conference will provide a unique platform for the convergence of stakeholders in the orphan drugs industry to discuss and network with top tier government, pharmaceuticals, biotechs, non-profit organisations, orphan drugs developers, research organizations as well as patient groups and advocates. We are putting together an agenda that address the driving macroeconomic factors, policies, and issues that will steer the development of orphan drugs globally including access, commercialisation, collaborations, policies, reimbursement, pricing and more.

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Insights & Attendees

Featuring Key Industry Experts

David Gillen

Vice President, International Medical Affairs, Vertex

Thomas Kühler

Head Regulatory Science & Policy, EU/AMEE, Sanofi

Vu Truong

Chief Executive Officer, Aridis Pharmaceutical

Sheela Upadhyaya

Associate Director - Highly Specialised Technologies, NICE

All Expert Speakers

From a patient advocacy organization perspective, was a very informative meeting. Learned a lot from presentations and networking that will inform how we can add value to the advocacy-patient-drug developer dialogue.

aftd
Dr. Nadine Tutton

Scientific Director, Research, Association for Frontotemporal Degeneration

I was very impressed with the format, the content was interesting and well done. I felt I learned a lot and was glad to be able to attend.

pharmolam
Senior Director

Business Development at Pharm-Olam, LLC

Introducing

2020 CONFERENCE FORMAT

Orphan Drugs & Rare Diseases Global Congress 2020 – choose your focus streams in the afternoon of both conference days by attending keynotes, presentations and group discussions that have the most relevance to you. Choose from..

Commercialization, Current Trends and Collaborations

Commercialization, Current Trends and Collaborations

Access, Pricing and Reimbursement

Access, Pricing and Reimbursement

Innovations, Discovery and Clinical Development

Innovations, Discovery and Clinical Development

Advance Therapy Medicinal Products

Advance Therapy Medicinal Products

View Conference Agenda

Gold Sponsor

Gold Sponsor

Silver Sponsor

Lead Exhibitor

Associate Sponsor

Associate Sponsor

Sponsor & Exhibit
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Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases 2020 Europe. It’s the 12th in the series of our Flagship tri-annual Orphan Drugs and Rare Diseases event, this congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs.


The World Health Organization (WHO) defines a disease as rare when it affects 1.3 in 2000 people. There are more than 7,000 known rare diseases stemming from a multitude of factors; 80% are genetic in origin while the other 20% have environmental, immunological or other origins. Rare disease predominantly affect children and due to lack of diagnosis and treatment, sadly 3 out of 10 children born with rare disease does not get to live till 5.


Out of more than 7,000 rare diseases identified, only a fraction of them have approve treatment available. “Orphan drugs” are intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions. Currently, the regulatory climate is favourable with both FDA and European Medicines Agency, as they offer incentives, tax credits, user waivers and marketing exclusivity. As a result, the number of therapies approved for rare diseases for the past two decades has grown exponentially.


The conference will provide a unique platform for the convergence of stakeholders in the orphan drugs industry to discuss and network with top tier government, pharmaceuticals, biotechs, non-profit organisations, orphan drugs developers, research organizations as well as patient groups and advocates. We are putting together an agenda that address the driving macroeconomic factors, policies, and issues that will steer the development of orphan drugs globally including access, commercialisation, collaborations, policies, reimbursement, pricing and more.


The two-day Congress will provide an interactive, cutting edge and comprehensive discussion and networking format led by key experts with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies and concepts surrounding orphan drugs and rare diseases.


New format this year, we are putting together an agenda that addresses “Current Trends and Innovations driving access to meet unmet medical needs of rare disease patients”. We will gather together high calibre expert speakers to participate and share their knowledge and expertise through Panel discussions, keynote presentations and 4 major agenda streams addressing all aspect of the industry.


We look forward to welcoming you at the congress!

Jocelyn Raguindin
Jocelyn Raguindin

Jocelyn is Conference Director at Paradigm Global Events (PGE). She has over 15+ years experience in organizing pharma-industry conferences and has been at the helm of every PGE conference since 2013. Jocelyn is based in London, UK.

Gain Latest Insights on:

  • Current Orphan Drugs landscape and major drivers in global raredisease market
  • Key developmental strategies carried out and Influencing factorsthat may affect market share to stand out in this industry
  • Current FDA thinking regarding common issues encountered in raredisease drug development
  • Finding innovative and alternative ways in funding thedevelopment of Orphan Drugs
  • Patients perspective: what really matters to rare disease patientsand caregivers?
  • Key authorities facilitating development and approval of diagnosticproducts/services
  • Coming Together in Developing Orphan Drugs and CrossingBorders
  • Trends and evolution of Advance Therapy Medicinal Products
  • How Can the Developer and the Patient Assist in the Evolution andDevelopment of Orphan Drugs to make it accessible to patient inshorter period of time?
  • Strategies to improve Access and Affordability
  • What Do Developers Look for When Looking for an OutsourcingPartner?
  • What Do Insurance Companies Think About Orphan Drugs? WillThey Make Modifications to Their Policies to Support Patients withRare Diseases?
  • Opportunity to network with Peers, potential Partners and Investors

You Will Meet:

  • Presidents, Heads/Chiefs, Directors, VPs and Managers in the are of:
  • Research and Development
  • Personalised Medicine
  • Regenerative Medicine
  • External R&D Innovation
  • Innovative Medicine
  • Rare and Ultra-Rare Diseases
  • Cell and Gene Therapy
  • Translational Science
  • Molecular Geneticist
  • Program Management
  • Patient Advocacy Groups
  • Public Affairs
  • Medical Affairs
  • Regulatory Affairs
  • Clinical Research Organizations
  • Market Access
  • Pricing and Reimbursement
  • Health Economics Outcomes Research
  • Commercial Development
  • Investments and Funding
  • Product Specialists
  • Global Strategic Services
  • Business Planning and Operations
  • Pharmacies
  • Academia

David Gillen

Vice President, International Medical Affairs, Vertex

David is the Vice President of International Medical Affairs at Vertex and has been in this role since March 2016. David qualified in Medicine, Physiology and Clinical Pharmacology from St. Mary’s Hospital (Imperial College of Science, Technology and Medicine) in 1992. He retained this interest in Pharmaceutical Medicine during his postgraduate training in Cardiology and General Medicine and joined Pfizer Global Research and Development in Sandwich in 1998. At Pfizer, David worked in Clinical Development and in Medical Affairs in the UK and also spent time working at a Global level in New York for 4 years. He rejoined Pfizer UK in April 2007 as UK Medical Director. Prior to leaving Pfizer, David was Vice President for Medical Affairs in Pfizer’s Primary Care Business Unit covering Europe, Canada, Australia and New Zealand where he established a Regional Medical Affairs group covering Pfizer’s primary care portfolio of medicines.

 

After leaving Pfizer David held the role of Head of International Medical Affairs for Gilead Sciences. In this role he managed and led a group of Medical Directors across Europe and Asia – Pacific. In 2011 he then moved to work at Celgene where he was Head of Drug Safety and Risk Management for EMEA / APAC for Celgene Ltd. In addition David acted as the QPPV for Celgene’s Pharmacovigilance System in Europe.

 

Outside Industry David has been active in the UK Clinical Research community for some time. He has represented Industry on the 2009 Academy of Medical Sciences Rawlin’s review looking into UK Clinical Research competitiveness and Governance. Beyond Clinical Research, David has served on the Innovation sub- committee of the ABPI (Initiating the ABPI Real – World data project), represents Industry on the NICE Appeal Panel was a member of the 2009 RCP working party looking at the relationship between Industry, Academic Medicine and the NHS

Thomas Kühler

Head Regulatory Science & Policy, EU/AMEE, Sanofi

Thomas Kühler is a PhD chemist by training with a post-doctoral degree obtained with late Nobel Prize Laureate Donald J. Cram. In recognition of his contributions to the field of Medicinal Chemistry and longstanding experience in drug discovery he was appointed Associate Professor in Medicinal Chemistry at Uppsala University in Sweden.
Thomas has garnered his work experience from Hässle AB in Sweden (now AstraZeneca), the Medical Products Agency (the Swedish drug regulator), Novo Nordisk A/S in Denmark, and most recently Sanofi in France.
Thomas has served on the Board of the Drug Information Association in the US. He also was a member and chair of the Board of The Organisation for Professionals in Regulatory Affairs in the UK. He is currently serving on the Board of the MedTech & Pharma Platform headquartered in Switzerland.

Vu L. Truong

Ph.D. CEO & CSO, Aridis Pharmaceuticals

Dr. Truong is a founder of Aridis and elected to the CEO position in 2014 after having served as the company’s Chief Scientific Officer for the past 9 years. He has over 20 years of experience in biopharmaceutical drug development, and a life-long interest and research experience in infectious diseases, focusing on the development of innovative human monoclonal antibodies and vaccines designed to address life threatening infections. His product development experience includes FluMist™, Synagis™ mAb, and a number of other monoclonal antibody-based therapeutics. Dr. Truong is the principal architect of the Aridis technologies, which includes a range of anti-infective products, APEXTM human mAb discovery and production platform technology, and pharmaceutical processing technologies. He received his Ph.D. in Pharmacology and Molecular Sciences at the Johns Hopkins University School of Medicine.

Sheela Upadhyaya

Associate Director - Highly Specialised Technologies, NICE

Sheela Upadhyaya is currently the Associate Director of the Highly Specialised Technology program at NICE and is responsible for running the program to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS.

She has extensive experience in understanding the issues that face companies and other stakeholders when trying to secure access for medicines for the orphan and ultra-orphan conditions. These include developing innovative access arrangements in liaison with industry, clinicians, patients and the NHS.


Sheela has co-authored several papers that discuss HTA methods for assessing value of orphan medicines and presented at many conference issue panels on the subject.
Prior to joining NICE, she commissioned rare and ultra-orphan disease services in the NHS delivering improvements through collaborating with industry, clinicians and patient groups. During that time, she successfully decommissioned failing services and seamlessly transferred the care of patients to better quality services.

Sheela has a passion for partnership working and believes that collaboration across the sector is the key to delivering high quality outcomes for all.