Day 1: ACCESS Programmes Congress

Conference Agenda

MUST ATTEND!

Expanded Access Programmes - Day 1

Day 1: Monday 20th September 2022

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
  • What is expanded access and when do we use it?
  • How do we plan for expanded access?
  • Who needs to be involved with expanded access and what is the key to its success?
  • What are means to get the medicine to the patient?

Kimberly Buchanan,
Associate Director, MRL Global Development Quality, Global Clinical Supplies,
Merck & Co

  • Overview of the US regulatory landscape
  • Understanding the Right to Try Act
  • Learning from FDA’s Project Facilitate
  • 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
 
  • 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

RESERVED

  • Components required for timely execution
  • So easy even a scientist can do it
  • Setting expectations for the family
  • Potential for data

William Richard Light, Chief Scientific Officer, VirTech Bio

CHALLENGES & POTENTIALS

  • Overcoming challenges brought by the pandemic
  • Patient-centred trial design to accommodate changes in patients need
  • Lessons learned and global distribution management

Christine Von Raesfeld, Founder & CEO, People with Empathy

  • Introduction to EAP and their differences
  • When does the implementation of the 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

John Lagus, Executive Vice President Business Development, Tanner Pharma Group


Dana Dunne, Expanded Access Program Director, Tanner Pharma Group

  • EAP doesn’t jeopardize the risk of approval
  • Post-approval perspective on the EAPs

-Spinraza
-Zolgensma
-Evrysdi

Khrystal Davis, Founder, Texas Rare Alliance

  • What is the level of awareness and knowledge about the expanded access and Right To Tryprograms among community oncologists across the United States
  • How often do community oncologists use the two programs, and how successful are they inobtaining investigational drugs for their patients?
  • What can we take away from the results of this study?

Marjorie Zettler, Director of Clinical Science, Regor Pharmaceuticals, Inc.

  • 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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