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Day 2

6th Expanded Access Programmes World Congress 2024 Europe

Holiday Inn London - Bloomsbury, an IHG Hotel, London, UK

Day 2 - Friday 12th April 2024

Dan Lewi, Head of Business Development and Patient Advocacy , Pulse infoframe Inc., co- founder CATS


  • Value of patient engagement in Early Access Programmes
  • How important is engaging patient at the earliest stage in research and development
  • The need for a continuous meaningful dialogue between stakeholders to establish an effective working relationship
  • What are the best practices and ethics behind a successful early access programme?
  • Knowing what motivates the stakeholders within early access
  • Sustaining purposeful patient engagement across the lifecycle of medicines

Carole Scrafton, Director & Co-Founder, Flutters and Strutters
Dr Lorna Pender, Global Patient Engagement Lead, Clinigen Group

Hannah Plowman, Real World Data Specialist, Clinigen Group

  • How should societies address a request for a single patient in need when a drug is not being made broadly
  • Could sponsors or governments ethically say yes to one person and no to others?
  • Who should be deciding which patients will live?

Nora Pöntynen, Global Medical Affairs, Boehringer Ingelheim International GmbH

  • Value of real-world data in EAP
  • The use of real-world data for Market access and reimbursement
  • Regulatory considerations and country differences for collecting real-word data
  • Case studies

Dr David Gillen, Chief Medical Officer, Norgine

  • Different source for gathering data from EAPs
  • Inherent collection of patient data
  • EAP vs RWE
  • Continue to focus providing access
  • Optimize contact with HCPs

Dr. Alberto Calabrò, Patient Access Program and Supply Leader, F. Hoffmann-La Roche Ltd

  • Why is patient forcasting important in planning early access programmes
  • Factors that could influence patient uptake in early access programmes
  • Overcoming difficulties that affects patient uptake

Sara Radenovic, Director, Managed Access, GSK
Ana Tediosi, Head of Expanded Access Strategy, WEP Clinical

Robert Donnell, EVP, Medicines Access, Smartway Pharma

  • EAP Essentials for Small Pharma
  • Planning and Compliance
  • EAP Design Principles
  • Vendor Selection Criteria

Julie Powell, Director, Patient Advocacy and Public Affairs, Pleco Therapeutics


  • Open Label Trial
  • 20 children, 2 rare disease
  • Encouraging results lead to off  label prescribing
  • Inequitable access and reimbursement
  • The fight goes on

Lindsay Randall, Founder, CEO, Mother, Arthur’s Quest

  • National genomic sequencing programme for cancer and rare disease
  • Clinical genomic data enhances target selection and validation
  • Using genomics to improve efficacy and safety of therapeutic candidates
  • Incorporation of genomics into patient selection / enrolment criteria
  • Early intervention enablement using genomics

Dr James Duboff, Strategic Partnerships Director, Genomics England 

  • What to do if a drug is ultimately not approved for funding
  • How can patient families help advance the funding and approval process with HTAs
  • What role does world data and observational studies play in supporting the pricing reimbursement decision
  • What does one do if the funding process fails.

Dan Lewi, Head of Business Development and Patient Advocacy , Pulse infoframe Inc., co- founder CATS

Juliet Hulse, Head of Clinical Operations, Pulse infoframe Inc.

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