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.
