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Day 2

5th Expanded Access Programmes World Congress 2023 Americas

Boston, MA, USA

Day 2 - Tuesday 12th September 2023


  • Value of patient engagement in Early Access Programmes
  • How important is engaging patient 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?
  • Knowing what motivates the stakeholders within early access
  • Sustaining purposeful patient engagement across the lifecycle of medicines
  • Compassionate use programmes are instrumental in ensuring continuity of access to drugs until approval and reimbursement decisions are finalised.
  • The importance of a registry of drugs offered on a compassionate use basis
  • Further transparency to allow such programmes to be evaluated and direct patients to sources of treatment.
  • Understanding what EAP are for
  • Physicians responsibility in applying for EAP
  • Meeting criteria for EAP
  • Learning how patients will be transitioned to commercial supply
  • 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
  • Why is patient forcasting important in planning early access programmes
  • Factors that could influence patient uptake in early access programmes
  • Overcoming difficulties that affects patient uptake
  • Should a company avoid any risks to a development program to help a larger number of patients gain access in the future?
  • How to avoid a negative impact resulting from pre-approval access to investigational medicines
  • Providing affordable access to those receiving the medication when compassionate use end


  • Overcoming challenges brought by the pandemic
  • Patient-centred trial design to accommodate changes in patients need
  • Lessons learned and global distribution management
  • Companies should anticipate the need for EA and should have a written policy
  • The policy should be available to the person entitled to expanded access
  • Companies must be accountable to the patients sending expanded access requests
  • Sharing of data analysis is crucial for improvement
  • Alternative access schemes offer flexibility to policymakers but often lack transparency and clear rules.
  • The uncertainties allow for dynamic responses to weaknesses in the main drug approval and reimbursement systems
  • Challenges facing the reimbursement system
  • Understanding different payer strategies currently in place
  • Drivers of early access programmes
  • Setting up a clear perspective and expectations of the programme
  • Streamlining process and ensuring early access of medicinal products to patients
  • How regulators and reimbursement agencies can work together to help ensure that patients are able to access life changing medicines as soon as it is safe for them to do so?
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