5th Expanded Access Progammes World Congress 2023 Americas
Thursday 16th - Friday 17th November 2023
Paradigm Global Events is very pleased to welcome you to our upcoming 5th Expanded Access Programmes World Congress 2023 Americas.
Early access programmes offer ethical, compliant and controlled mechanisms of access to investigational medicinal products (IMP) outside of the clinical trial space and before marketing and authorisation.
Expanded Access also known by other terms such as compassionate use, early access, managed access, named-patient access, or pre-approval access is a potential pathway for a patient with an immediately life-threatening illness or severely debilitating disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) before they are reviewed and approved by a regulatory authority. When it is not possible for a patient to participate in a clinical trial and all other available medical options have been exhausted, the patient’s physician may seek special access to investigational medicine outside of the clinical trial setting. As investigational drugs have not yet received regulatory approval, it is very important to remember that their potential risks and benefits are not yet established. In general the person and his or her doctor must apply for access to the investigational product, the company has to choose to cooperate, and the medicines regulatory agency needs to agree that the risks and possible benefits of the drug or device are understood well enough to determine if putting the person at risk has sufficient potential benefit.
With patients thoroughly becoming knowledgeable, Pharmaceutical companies are bound to receive unsought request. However, there are challenges and obstacles such as diverse regulatory landscape, logistical differences, and complexities from planning an application to approval and implementation. Sponsors need to plan carefully before undertaking an Expanded Access Programme so that they have the resources, processes, and tools in place to support the inevitable demand.
EAPs promises huge potential to all stakeholders involved, from patients who obtained the medicines early to the pharmaceutical companies who provides it. Companies who invest in them may see considerable benefit in terms of relationship building with key stakeholders, such as patients, advocacy groups, scientists and regulators, as well as launch preparedness.
The two-day Congress will provide an interactive, cutting edge and comprehensive discussion and networking format led by key expert speakers with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies and concepts in obtaining the much needed access to these therapies for life changing treatment.
We look forward to meeting you at the Congress!
Sincerely yours,
Jocelyn Raguindin
Conference Director
Paradigm Global Events
GAIN THE LATEST INSIGHTS ON:
- Understanding the varying regulatory landscape, policies and guidelines to ensure a smooth execution of the programme
- The benefits and issues surrounding expanded access programme
- Criteria that must be met to authorize expanded access use, requirements for expanded access submissions
- The current compassionate use, or expanded access-related mechanisms
- Understanding operational complexities, best practices and strategies in running a global EAP
- Current trends and lessons learned from successful implementation
- Successfully navigating supply and logistical intricacies
- Incorporating patient voice from early planning to ensure a patient-centric access programme
- Robust data collection strategies for informed decision making
- Real-world data challenges and solutions
- Engaging representatives from all stakeholder groups to understand their perspective and
expectations of the programme
WHO SHOULD ATTEND?
This Congress will provide information to pharmaceutical and biotech companies, researchers, physicians, institutional review boards (IRBs), patient organizations and advocates, and regulatory agencies about the implementation of expanded access to investigational drugs.
Network with representatives involved in the following areas:
- Expanded Access
- Managed Access
- Early Access
- Market Access
- Medical Affairs
- Regulatory Affairs and Policy
- Patient Support and Patient Engagement
- Corporate Development
- RWE, and Data Management
- Quality and Compliance
- Clinical Development Programme
- Management
- Supply Chain Management
- Patient Access
- Clinical Research
- Sourcing and Logistics
- Consulting
- And much more…
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.
Wyndi Phillips, Vice President, Project Management, WEP Clinical
Annmarie Galli, Head, Global Medical Affairs Research and Compassionate Use / Managed Access Programs
Oncology, GSK
- 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
- Country Specific Regulatory Pathways
- Practical Challenges and Solutions in PTA
- Unique Operational Considerations for PTA
Graham Sidorowicz, EVP Commercial, Bionical Emas
- 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
Rachel Harisson, Associate Director, Pre-Approval Access Programs, argenx
CHALLENGES & POTENTIALS
- Setting up objectives of an EAP within the Asset Plan
- Importance of Early Conversations & cross-functional collaboration of internal stakeholders
- Managing the expectations of External Stakeholders
- Mitigating difficult conversations while setting up an EAP
Siddharth Jain, Medical Director –LSDs & HAE, Global Medical Affairs, Takeda
- Is an Open Label Extension feasible?
- Is Expanded Access/Compassionate Use the perfect plan?
- Regulatory Variations and Challenges
- Lessons Learned
Donna Cowan, Associate Director, EAP & Registry, Stealth Biotherapeutics Amber Bifolck Fisher, Clinical Research Manager, Kura Oncology, Inc.
- Advancing Equity
- Inclusive Communication
- Culturally Effective Decision Making
- Voices of Diversity
Chisa Nosamiefan, Co-founder, The Labalaba Foundation for Lupus
- Deep-dive into the reality of healthcare access around the world
- Streamlining the process and navigating early access regulations globally
- What does the future of health access look like? The interconnected ecosystem where all healthcare stakeholders work together
Moderator:
Panelists:
Jennifer E. Miller, PhD, Associate Professor , Yale School of Medicine Monica L Weldon, President/CEO/Founder, SYNGAP1 Foundation
5:45 - 6:45 pm - Networking Drinks Reception - Sponsored by Bionical Emas
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross
ACCESS & COMMERCIALIZATION
- What are the possible scientific and regulatory approaches to facilitate development?
- How to enable early access to treat Rare disease patients
- Ways to guarantee that patient safety, efficacy, and product quality are not compromised
- A discussion of experiences to date from early access approaches.
Moderator:
Panelist:
Emily Parks, Founder, POP!
Lee Sutton, Vice President, Business Development, EAP, WEP Clinical
- 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?
- Sustaining purposeful patient engagement across the lifecycle of medicines
Alan J. Balch, Chief Executive Officer, Patient Advocate Foundation
- Building your EAP with a ‘site-first’ approach
- Educating sites on their roles and responsibilities in an EAP
- Ensuring a clear communication plan to support sites in an expedited manner (people to people versus technology)
- Case study demonstrating the importance of site management
Wyndi Phillips, Vice President, Project Management, WEP Clinical
- The importance of patient advocacy in providing much-needed information on EAP’s available for patients
- 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 accelerate patient access to investigational medicine
- Creating a state of the art payer engagement strategies to address challenges in pricing and reimbursement
Moderator:
Panelist: Amber Bifolck Fisher, Clinical Research Manager, Kura Oncology, Inc. Alan J. Balch, Chief Executive Officer, Patient Advocate Foundation Chisa Nosamiefan, Co-founder, The Labalaba Foundation for Lupus
MULTI-STAKEHOLDER APPROACH & COLLABORATION
- Ideal Accelerated Pathway
- Problems of Excluding Some Disease
- Brainstorming: Removing the Barriers
- What’s the Long Game?: Rethinking the Regulatory Rules
Monica L Weldon, President/CEO/Founder, SYNGAP1 Foundation
TBC
Roberta Smith, President , Alagille Syndrome Alliance
- The importance of patient advocacy in providing much-needed information on EAP’s available for patients
- 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 accelerate patient access to investigational medicines
- Creating a state of the art payer engagement strategies to address challenges in pricing and reimbursement
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross
- 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
END OF CONFERENCE
