Agenda at a Glance

Orphan Drugs & Rare Diseases - Conference Day 2

Day 2: Friday - 18th February 2022

  • Major challenges that each stakeholder encountered during the pandemic
  • Lessons learned from Covid-19 and how we can benefit from it?
  • How to build specific needs, support, and recommendations for sustainable healthcare systems that provide directions for better preparedness in the future?

Moderator:

Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck

Panellists:

Rebecca Stewart, CEO, RARE Revolution Magazine
David Ross, Patient Advocate, Rare Disease Mental Health
Peter MacLennan, CEO, Tailored Clinical Research Solutions Ltd

  • The aim is to provide an overview of the recent discussions and updates from the European Commission concerning the revision of the orphan drug legislation in Europe.

Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck

  • Every data point is precious
  • Optimal relationships with hospital sites are essential
  • Patient centricity must inform all stages of the development process

Peter MacLennan, CEO, Tailored Clinical Research Solutions Ltd

  • How to improve patient engagement during studies while collecting standardized, regulatory-grade data
  • Clinical and hybrid studies to improve real-world data collection for drug development

Dr Femida Gwadry-Sridhar, Founder & CEO, Pulse Infoframe

 

  • What is the state of play in rare and ultra-rare diseases and what needs to happen to have a
    sustainable future?
  • Input gathered from 18 advocacy organisations/healthcare professionals
  • Awareness / diagnosis / access to treatment / driving research and innovation / impact of covid /
    patient perspectives / health economist perspectives of EU drug policy / industry perspective
  • Public perception survey data around awareness of rare diseases and attitudes towards their
    prioritisation

Paul Greenland, President EMEA, Amryt Pharma

  • Rare Disease Overview
  • Framework for successful Rare Disease Drug Development
  • Ergomed Rare Disease Innovation Center Q&A

Zizi Imatorbhebhe, MBA, MS, PMP®,Senior Vice President, Global Strategy and Development, Rare Disease, Head Rare Disease Innovation Center, Ergomed

  • Clinical trial design and execution
  • Collaboration across key stakeholders through development and regulatory processes
  • Blueprint Medicines has demonstrated success in Europe

Andrew Kim, VP International Strategic Operations and & General Manager, Global Distributor Markets,
Blueprint Medicines

2:15 - 2:45, 2:50 - 3:20 pm - ROUNDTABLE DISCUSSIONS - divided between 2 sessions of 30 mins. (5 mins. to change table) - Attendees have 2 round table options to attend

Andrew Kim, VP International Strategic Operations and & General Manager, Global Distributor
Markets, Blueprint Medicines

Carole Scrafton, CEO & Co-Founder, FibroFlutters
David Ross, Patient Advocate, Rare Disease Mental Health
Paul Lim, Associate Director, Patient Advocacy, Blueprint Medicines

Karen Matthews, Business Development Manager, CRNCC, NIHR Clinical Research Network

Farhat Khanim MD, Director of Research, Institute of Clinical Sciences, University of Birmingham

  • History
  • Mechanism: how it works
  • The increasing number of RPDDs
  • Past, present, and future value of Priority Review Vouchers

Christian Girard, Strategic Consultant, Orphan Drugs Industry Advisory & Intelligence

  • Breaking down barriers for access
  • Strengthening health systems around the world and accelerate pathways to diagnosis
  • Stakeholders to address the rare diseases’ ecosystem gaps to collaboratively build a sustainable roadmap for better health and a brighter future for the patients suffering from rare diseases.
  • Challenges and opportunities in creating sustainable healthcare for all.

Moderator:
Daniel Lewi, Head of Business Development and Patient Advocacy, Pulse Infoframe Inc.

Panellists:

Farhat Khanim MD, Director of Research, Institute of Clinical Sciences, University of Birmingham
David Ross, Patient Advocate, Rare Disease Mental Health
Arvin M. Gouw, PhD, VP of Research & Development, Rare Genomics Institute

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