5th Expanded Access Progammes World Congress 2023 Americas
Strategies and Considerations in Launching an Expanded Access Programmes
Revere Hotel Boston Common, Boston, MA, USA
Day 1 - Thursday 16th November 2023
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross
MACRO OUTLOOK & CURRENT TRENDS
- Understanding the challenges in planning and implementing a global EAP
- Country specific framework and requirements to access investigational drugs
- Scientific elements and regulatory tools available to address the challenges and explore the flexibility within the current regulatory systems
- Significant degree of administrative effort required to initiate the programme for a timely access
- How far are we from establishing a harmonised guidelines across Americas?
Moderator:
Monica L Weldon, President/CEO/Founde, SYNGAP1 Foundation
Panelist:
Rachel Harrison, Associate Director, Early Access Program, Apellis Pharmaceuticals Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross
Amber Bifolck Fisher, Clinical Research Manager, Kura Oncology, Inc.
- Planning and designing for expanded access programs early can be difficult. This panel explores how pharma and biotech can work with Patient Advocacy Groups and Research Consortiums to ensure that the patient voice is part of early planning, and how digital communities can facilitate early planning and
awareness of expanded access programs.
Jennifer M. Joe, Global Medical Strategy & Population Health Director, AstraZeneca
Bhaskar Dutta, Head, Digital Health & Medical Affairs Technologies, Alexion
Norah Xiao, Director, Digital Health Partnership, Astrazeneca
Isabelle Lousada, CEO and President, Amyloidosis Research Consortium
- In an era where ‘real-world data’ is increasing of interest, what are the limitations?
- Data collection within early access programs in various regions
- Ethical considerations and operational challenges in conducting data collection
- Benefits of collecting RWD within early access programs
- Country Specific Regulatory Pathways
- Practical Challenges and Solutions in PTA
- Unique Operational Considerations for PTA
Graham Sidorowicz, EVP Commercial, Bionical Emas
- Considerations in planning EAP’s with patient in mind
- Engaging with patient and advocacy groups early in development for well informed decision
- Strategies to ensure patients voice is incorporated in development discussions
- When to consider EAP and when it might not be the right thing to do for the community
CHALLENGES & POTENTIALS
- Value of patient engagement in Early Access Programmes at the earliest stage in research and development
- 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
- The main challenges in the execution of an EAP for cell and gene therapies are:
- Treatment is generally available in a restricted number of specialist centres, so patients need to travel for
treatment - 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- border programs
for cell and gene therapies
- 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 workingrelationship
- What are the best practices and ethics behind a successful early access programme?
- Sustaining purposeful patient engagement across the lifecycle of medicines
Alan J. Balch, Chief Executive Officer, Patient Advocate Foundation
- Intro to the Expanded Access Data, Analytics, and Insights
- What information are we still missing
- What information is required to improve Expanded Access
- Role of Real World Data in Expanded Access
- From Data into the Decision Making
- 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.
5:45 - 6:45 pm - Networking Drinks Reception - Sponsored by Bionical Emas
