Conference Agenda
MUST ATTEND!
Expanded Access Programmes - Day 1
Day 1: 1st March 2022
Sjaak Vink, Chief Executive Officer, The SocialMedwork
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
- 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
Joint Presentation:
Martine Zimmermann, PharmD, SVP, Head Regulatory Affairs, R&D & Commercial Quality, Alexion
Wendy Erler, VP, Global Head Patient Experience, STAR and Patient Advocacy, Alexion
- Frameworks for RWD collection in EAP
- Regional variability in major global markets
- Recent regulatory updates and practical examples
Joint Presentation:
Graham Sidorowicz, EVP Commercial, Bionical Emas
Rakesh Davda, Head of Real World Data & Market Access, Bionical Emas
- 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
Robert Donnell, Executive Vice President, Medicines Access, Smartway Pharma
- Balancing patient safety with potential therapeutic benefits
- Physicians perspective and experiences
- Risks and Rewards to Sponsors (Drug Manufacturer)
ACCESS & COLLABORATION
- Main challenges in the execution of an EAP for cell and gene therapies are:
1. Treatment is generally available in a restricted number of specialist centres, so patients need to travel for treatment
2. The treatment is generally high-value, particularly in the case of rare indications.
- The presentation will highlight lessons learnt and innovative solutions for EAPs and cross-borderprograms for cell and gene therapies.
Tom Smith, Program Management Director, Expanded Access, Durbin PLC – Uniphar Group
- 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
Nicola Gillott, Head of Expanded Access, Amryt Pharma plc
- 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
- Learn about a first of its kind tool to support patients, caregivers and providers
- Discuss co-creation as a standard for patient tools
Christine MacCracken, Head, Patient Strategy & Solution, Janssen Pharmaceuticals, J&J Company
- 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
Moderator:
Paolo Morgese, EU Director Market Access & Member Relations, Alliance for Regenerative Medicine
Panellists:
Josie Godfrey, Co-Founder and CEO, Realise Advocacy
Lindsay Birrell, Co-Founder & CEO, Realise Advocacy, Trustee, Medics 4 Rare Diseases
