Featuring

12th Orphan Drugs & Rare Diseases Global Congress 2020 Europe

Days
Hours
Minutes
Seconds

Monday 16th March 2020 - Pre-Conference Workshop
17th - 18th March 2020 - 2 Days Conference
London, UK

Venue

Millennium Gloucester Hotel

Address

4-18 Harrington Gardens, South Kensington, London, SW7 4LH

Phone

020 7373 6030

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.

Read Full Introduction
Insights & Attendees

Featuring Key Industry Experts

Bob Stevens

Group CEO, MPS Society

Edny Unui

Director, Business Development Caribous Biosciences

Didier Caizergues

Head, Regulatory Affairs Department, GENETHON

Joanne M.Hackett

Chief Commercial Officer, Genomics England

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

2020 Associate Sponsor

2020 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

Bob Stevens

Group CEO, MPS Society

Bob Stevens is the Group CEO of MPS Society & MPS Commercial, he is also Co-Chair of the International MPS Network and Vice-Chair of the LSD Collaborative. Bob’s previous roles include being the Managing Director of a charity supporting people of all ages with learning disabilities and complex needs and indeed he was a Trustee for the MPS Society for over 10 years.

Prior to being involved within the not for profit sector Bob had a successful career in the commercial sector within construction. Bob spent over 10 years operating at Board level alongside his private property development portfolio.


Bob has two sons with MPSII (Hunter’s Syndrome) which is one of the rare genetic diseases that is supported by the MPS Society, he says of his role that it is not a job but ‘a way of life’.

Edny Inui

Director, Business Development, Caribou Biosciences

Edny Inui, PhD, MBA is the Director of Business Development at Caribou Biosciences, a biotech company founded out of UC Berkeley by CRISPR pioneers Jennifer Doudna, Martin Jinek, and Rachel Haurwitz. Edny leads the out-licensing of Caribou’s CRISPR genome engineering technologies in addition to strategic in-licensing and partnering of new technologies in support of the Caribou portfolio of allogeneic CAR-T cell therapies. She has spent over a decade in the biotechnology industry with prior roles as Senior Manager, Business Development and Strategy at Acadia Pharmaceuticals and as a Senior Biotechnology Analyst for Biomedtracker, Informa Pharma Intelligence. She holds a BS in Biology from the Massachusetts Institute of Technology, a PhD in Neuroscience from the University of Michigan, and an MBA from the University of Chicago Booth School of Business.

Didier Caizergues

Head, Regulatory Affairs Department, GENETHON

A Doctor in Pharmacy, who holds a degree in Health law. He enjoyed a long career in the field of Regulatory Affairs.


He began his career as a consultant with International Drug Development (IDD), but soon joined the company SYNTHELABO in its Research and Development center where he worked for nine years. In this office Dr Didier CAIZERGUES oversaw the registration of new molecules that had evolved from cardiovascular, urological and anti‐depressive research.

He later headed up the international registration department for the Pierre Fabre group and for the next six years he practices to the fields of anti‐cancer drugs, anti‐depressants and orphan diseases. In 2001 he joined the GENETHON as Director of International Regulatory Affairs where he set up a regulatory affairs department specialized in gene and cell therapies in the fields of rare diseases. He obtained several clinical trials authorization in European countries and US for gene therapy products with lentivirus and AAV in the fields of muscular dystrophies and paediatric immunodeficiencies. He also manages scientific advice with the European Medicines Agency. He is also in charge of the assessment of new molecules or molecules already marketed still in the field of rare diseases in different working groups in European projects (Treat‐NMD).

Joanne M. Hackett

Chief Commercial Officer, Genomics England

Joanne M. Hackett is the Chief Commercial Officer at Genomics England, where she is responsible for their industry engagement strategy by developing, managing and accelerating relationships with commercial organisations − creating opportunities for collaboration both nationally and globally.

Professor Hackett is a clinical academic, an investor, and a strategic, creative visionair with global experience spanning successful start-ups to Fortune 500 companies. Aside from her curious passion for life and positivity, Joanne is known for building innovation, driving personalised medicine and leading through fast paced, complex changing ecosystems and integrations. Joanne’s goal is to contribute in bringing the world novel, cost effective and simple health care solutions, and she is particularly keen on building the case for prevention, open science and citizen genomics. She has extensive global experience across academic, business and clinical institutions, and enjoys sharing her experiences with the Boards she sits on as well as companies she provides strategic advice to.

Joanne has been publicly recognised for her relentless pursuit of revolutionising healthcare and has been named the Accenture Life Science Leader of the year 2019, Freshfields Top 100 Most Influential Women 2019, One HealthTech 2018 Top 70 Women in the NHS, Pharmaceutical Market Europe’s  2018 30 women leaders in UK healthcare and BioBeat 2017 Top 50 Women in Biotech Award. Joanne believes in human courage and perseverance against the odds, and demonstrates that positive change, whether in a company or in one’s personal life, can be carved out from even the greatest of trials. As a believer of ‘health = wealth’, Joanne is an internationally known yoga instructor.