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Day 2

6th Expanded Access Programmes World Congress 2024 Europe

Copthorne Tara Hotel London Kensington, London, UK

Day 2 - Friday 12th April 2024


  • 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
  • Why do we need early access programs
  • What value do they bring and to who
  • What are the opportunities and challenges
  • Who should you engage with and when

Sheela Upadhyaya, Chair Elect, Rare Disease Special Interest Group, ISPOR

  • How should societies address a request for a single patient in need when a drug is not being made broadly available?
  • 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

  • Intro to the Expanded Access Data, Analytics, and Insights
  • What information are we still missing
  • What information is required to improve Expanded Access
  • Role of Real World Data in Expanded Access
  • From Data into the Decision Making

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
  • Main challenges in the execution of an EAP for cell and gene therapies
  • How common are expanded Access approvals in the field of advanced cell therapy?
  • Are there any interesting parameters by which patient can participate?
  • Who bears the burden of paying for investigational therapies used in pre-approval non-trial settings?
  • Lessons learnt and innovative solutions for EAPs and cross-border programs for cell and gene therapies.


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

Julie Powell, Marketing & Communications Director, Pleco Therapeutics

  • 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

  • Operational aspects for global and local implementation
  • Safety Reporting Training for HCPs
  • Health Authority attention to and interest in safety reports
  • Inspection experience

Annie O’Keefe Martin, Global Medical Governance Lead for Managed Access Programs and Rare
Humanitarian Programs, Sanofi

  • Alternative access schemes offer flexibility to policymakers but often lack transparency and clear rules.
  • The uncertainties allow for dynamic responses to weaknesses in the main drug approval and reimbursement systems
  • Challenges facing the reimbursement system
  • Understanding different payer strategies currently in place
  • Drivers of early access programmes
  • Setting up a clear perspective and expectations of the programme
  • Streamlining process and ensuring early access of medicinal products to patients
  • How regulators and reimbursement agencies can work together to help ensure that patients are able to access life changing medicines as soon as it is safe for them to do so?
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