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?
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
- 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
- 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
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.