Day 1: ACCESS Programmes Congress

Conference Agenda

MUST ATTEND!

Expanded Access Programmes - Day 1

Day 1: Wednesday 1st December 2021

REGULATORY GUIDELINES & CURRENT TRENDS

  • Understanding the challenges in running a global EAP
  • The positive impact of EAP on patients with serious, immediately life-threatening, or rapidly debilitating diseases
  • Gain an understanding to increase access to treatment
  • The importance of cross collaborations between stakeholders
  • Overview of the US regulatory landscape
  • Understanding the Right to Try Act
  • Learning from FDA’s Project Facilitate
  • Background and Scope of early access approaches
  • Identifying challenges faced by Applicants of PRIME and Breakthrough therapies
  • Scientific elements and regulatory tools available to address the challenges and explore the flexibility within the current regulatory systems
  • Key considerations to ensuring a successful program
  • In an era where ‘real-world data’ is increasingly 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
                                                                          

                                ​                                                                               Anne B. Cropp. Pharm.D., BCAP
                                                                                                               Chief Scientific Officer 
                                                                                                          Early Access Care, LLC
 
  • Introduction to EAP and their differences
  • When does the implementation of Early access programme be considered?
  • What are the numerous challenges faced in the implementation of EAPs?
  • Key considerations for the successful implementation of early access program

CHALLENGES & POTENTIALS

  • 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
  • Implications of one-dose, potentially curative treatments
  • Compassionate use as research or treatment
    Regulatory, technical and logistical hurdles
  • Who bears the burden of paying for investigational therapies used in pre-approval non-trial settings?
  • 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.
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