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?
  • 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

  • What mobile research nursing is all about.
  • Utilising mobile research nursing in paediatric trials.
  • Giving the child control over their participation in the trial.

Senior Representative, Illingworth Research


  • How to improve patient engagement during studies while collecting standardized, regulatory-grade
  • 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

  • How to close the gap between regulatory approval and funding agreement?
  • How do the key HTA and payers operate?
  • Samples of high profile reimbursement battles
  • How to overcome struggles to meet the strict cost-effectiveness threshold for new therapeutics?
  • The foundational value of building collaboration through trust, respect, transparency, and empathy
  • Not all advocacy organizations are alike: assessing the landscape
  • Real-world examples of innovative digital solutions driving collaboration among multiple

Tom Pulles, MD, VP, Head of Medical Affairs & Patient Advocacy, Ultragenyx Europe GmbH

  • 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.


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