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Day 2

8th Expanded Access Programmes World Congress 2025 Europe

Comprehensive Analysis on Executing Expanded Access Programmes

Copthorne Tara Hotel London Kensington, London, UK

Day 2 - Thursday 20th March 2025

BREAKING DOWN BARRIERS TO PATIENT ACCESS

  • 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
  • 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?
  • 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
  • Different source for gathering data from EAPs
  • Inherent collection of patient data
  • EAP vs RWE
  • Continue to focus providing access
  • Optimize contact with HCPs
  • 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
  • EAP Essentials for Small Pharma
  • Planning and Compliance
  • EAP Design Principles
  • Vendor Selection Criteria

CHALLENGES AND POTENTIAL OF EAP

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