Expanded Access Programmes - Day 1
Day 1: 1st March 2022
CURRENT TRENDS & STRATEGIES
- Understanding the challenges in running a global EAP
- The positive impact of EAP on patients with serious, immediately life-threatening or
- rapidly debilitating diseases
- Gain an understanding to increase access to treatment
- The importance of cross collaborations between stakeholders
9:55 am - PLENARY KEYNOTE: Compassionate Use Programmes: Benefits, challenges, and key considerations for successful implementation
- Introduction to EAP and their differences
- When does the implementation of the Early Access programme be considered
- What are the numerous challenges faced in the implementation of EAPs?
- Key considerations for the successful implementation of early access program
11:10 am - Differences in the precise requirements and implementation between Europe and US pre-approval access
- Comparison between restriction in disease standard
- Limitation to the scope of access to drugs
- Application of use
- Applies to individual or cohorts
- Deep-dive into the reality of healthcare access around the world
- What does the future of health access look like?
- The interconnected ecosystem where all healthcare stakeholders (patients, carers, HCP’s, pharma, health advocates, politicians, policy makers, technologists, etc.) come together
- Balancing patient safety with potential therapeutic benefits
- Physicians perspective and experiences
- Risks and Rewards to Sponsors (Drug Manufacturer)
ACCESS & COLLABORATION
- Should a company avoid any risks to a development program to help a larger number of patientsgain access in the future?
- How to avoid a negative impact resulting from pre-approval access to investigational medicines
- Providing affordable access to those receiving the medication when compassionate use end
- Challenges & Opportunities of a company-wide roll-out of Early Access Programme
- Strategies & consideration for a successful implementation
- Established responsibilities from everyone involved to ensure smooth delivery to patients in need
- The importance of patient advocacy in providing much-needed information on EAP’s available forpatients
- Collaborating with one another to enable a much richer understanding of patient needs
- Patient groups’ importance in working with health authorities to advance policies that acceleratepatient access to investigational medicines
4:30 pm - Draft guidance to support quality data packages for PRIME marketing authorisation applications
- What are the possible scientific and regulatory approaches to facilitate development?
- How to create robust quality data packages to enable timely access to medicines for patients
- Ways to guarantee that patient safety, efficacy and product quality are not compromised
- A discussion of experiences to date from early access approaches.
5:05 pm - KEYNOTE PANEL DISCUSSION: The importance of patient engagement for successful early access programmes
- How important is engaging patient early 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