5th Expanded Access Progammes World Congress 2023 Americas
Boston, MA, USA
Day 1 - Monday 11th September 2023
OVERVIEW OF US REGULATORY LANDSCAPE
- 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?
- Addressing the complex European regulatory framework
- Key considerations and current trends within expanded access programmes
- Strategies and lessons learned in launching several country-specific expanded access programmes in the US
- Key legislative changes to maximise the Scheme’s impact by accelerating availability of medicines for patients
- Facilitating collection of real- world data which may support regulatory decision making
- Implications to pharma industry and manufacturers supplying EAMS medicines
- Benefits to patients and physicians
- In an era where “real-world data” is increasingly 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
- Programme design strategies to optimise benefits and reduce risk
- State of the art real-world data collection and analysis
- Breaking down intricate global regulatory framework
- Accountable world class global distribution of investigational drugs
- 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
MULTI-STAKEHOLDER APPROACH & COLLABORATION
- Main challenges in the execution of an EAP for cell and gene therapies
- How common are expanded Access approvals in the field of advanced cell therapy?
- Are there any interesting parameters by which patient can participate?
- Who bears the burden of paying for investigational therapies used in pre-approval non-trial settings
- Lessons learnt and innovative solutions for EAPs and cross-border programs for cell and gene therapies.
- 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
- How should societies address a request for a single patient in need when a drug is not being made broadly available?
- Could sponsors or governments ethically say yes to one person and no to others?
- Who should be deciding which patients will live?
- Interaction between pharmaceutical companies and healthcare providers could foster the development of new medicines which will ultimately benefit patients.
- Would this enhance technological innovation, fosters knowledge creation, aids disease control, and reduces polypharmacy issues?
- Guidelines to help professionals navigate the opposing explanations of interactions with pharmaceutical companies
- The importance of patient advocacy in providing a 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 accelerates patient access to investigational medicines
- Creating a state-of the art payer engagement strategies to address challenges in pricing and reimbursement
5:30 - 6:30 pm - Networking Drinks Reception - Sponsored by Bionical Emmas
