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
11:40 - Plenary Keynote: Real-World Data collection in Early Access Programs: Challenges and Opportunities
- 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
12:15 pm - KEYNOTE PANEL DISCUSSION: Expanded Access Programmes: Key considerations for successful implementation
- 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
5:05 pm - KEYNOTE PANEL DISCUSSION: Challenges faced by applicants to complete quality, manufacturing development and data requirements during the development of products in early access approaches.
- 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.