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ACCESS Programmes
Conference Agenda
Expanded Access Programmes - Day 2
Day 2: 2nd March 2022
Sjaak Vink, Chief Executive Officer, The SocialMedwork
CHALLENGES & POTENTIALS
- Background and scope of early access approaches
- Identifying challenges faced by Applicants of PRIME and Breakthrough therapies
- Scientific elements and regulatory tools are available to address the challenges and explore theflexibility within the current regulatory systems
- Key considerations to ensuring a successful program
- History and background about these new quality guidelines
- The new product life Cycle ICH Q12 guideline are step by step setting around the world (FDA JAPANANVISA CHINA ..)
- The impact of new regulations on innovation and flexibility, opportunity and challenges
Georges France, Quality Expert, Consultant for GSK
- Navigating the diverse regulatory differences between countries
- Understanding the different approaches to approval procedures for early access
- Important considerations before implementing EAP
The need for greater regulatory harmonisation to widen the early access
Katrin Schepp, General Counsel, The SocialMedwork
- What are the logistical bottlenecks encountered from the start of COVID-19
- How can you predict demand and guarantee product sustainability to set up and maintain EAP?
- How to effectively communicate with different stakeholders in preparation to EAP
- 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.
- Respond to unmet medical needs as early as possible
- Importance of EAP for acute diseases
- Specificities regarding Gene Therapy and Rare Diseases
Magali Taiel, Chief Medical Officer, Gensight Biologics
- In an era where “real-world data” is increasing of interest, what limitations should exist?
- Data collection guidelines within early access programmes in various regions
- Ethical consideration and operational challenges in conducting data collection
- Benefits of collecting RWD within early access programmes
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross
- Challenges facing the reimbursement system
- Understanding different payer strategies currently in place
- Alternative access schemes offer flexibility to
- policymakers but often lack transparency and clearrules.
- The uncertainties allow for dynamic responses to weaknesses in the main drug approval and reimbursement systems
- Strategic considerations and best practices to facilitate Expanded Access Programmes
- What are the comprehensive approaches that can support both clinical development and medical governance objectives
- Embracing patients need and physicians input to increase uptake and quality of EAP
- Various stakeholders engagement to leverage communication between pharma, clinician and patient organization
- Advantages of a collaborative patient-pharma relationship
Panellists:
Josie Godfrey, Co-Founder and CEO, Realise Advocacy
Lindsay Birrell, Co-Founder & CEO, Realise Advocacy, Trustee, Medics 4 Rare Diseases
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