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Day 1

5th Expanded Access Progammes World Congress 2023 Americas

Boston, MA, USA

Day 1 - Monday 11th September 2023


  • 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


  • 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

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