Pre-Conference Workshop Day | Monday 7th October

8:00 Morning Coffee & Registration

Guiding Principles for Launching Effective Early Access Programmes

Advanced Strategies for Optimising Established Early Access Programmes

Workshop A

Workshop D

9:00 am EAPs 101: Understanding Diverse Terminology Programs & Establishing a Comprehensive Resource Guide for Effective Compassionate Use Implementation

  • Helen Dean Associate Director, Evidence Optimisation and Managed Access Team, Medical Affairs EUR / International, Jazz Pharmaceuticals Inc
  • Scott Siegert Executive Director - Strategy, Operations & Clinical Science and Development, Neurocrine Biosciences

Synopsis

  • Examining the nuances between Named Patient, Managed Access, Early Access, and Compassionate Use Programs and the implications of different terminology on programme structure
  • Delving into the essential regulatory elements necessary for opening an Early Access Program, ensuring compliance and clarity
  • Identifying the roles, responsibilities, and resources required to successfully launch and manage a program, including team structure and planning
  • Developing an educational program to familiarize internal stakeholders with different terminologies, best practices for establishing programs, and the key individuals and stages involved

9:00 am Innovative Approaches for Equitable Patient Access & Creating Effective Humanitarian Donation Programs

Synopsis

  • Learning how to design and organize these programs, including the key team structures required for successfulmanagement and execution
  • Gaining insights into budgeting and forecasting for humanitarian programs and long-term plans, emphasizing efficient resource allocation and strategic planning
  • Exploring critical regulatory implications when developing humanitarian donation plans, ensuring adherence to local laws and best practices for compliance
  • Navigating Free-of-Charge Donation Programs to overcome affordability challenges

11:00 Morning Break & Networking

Workshop B

Workshop E

12:00 pm Establishing a Successful & Comprehensive Standard Operating Procedure (SOP) to Streamline Processes & Improve Efficiency

  • Ann Watkins Associate Director, Evidence Optimisation and Managed Access Team, Medical Affairs EUR / International, Jazz Pharmaceuticals Inc
  • Nora Pöntynen Global Capability & Early Access Treatments, Boehringer Ingelheim

Synopsis

  • Examining best practises for creating a detailed SOP that serves as a reliable resource across various departments, ensuring consistent processes and clear communication
  • Explore factors that may not be covered in the SOP, such as exit strategies and their impact on program continuity and success
  • Understanding the importance of maintaining the program effectively, including supply chain considerations, timelines, and collaboration with cross-functional teams
  • Assessing the data needs beyond RWE and determine what essential information must be shared with internal teams

12:00 pm Access Strategies for Rare Disease Treatment

  • Eva Gallagher Vice President Global Medical Affairs, Agios Pharmaceuticals
  • Daniel O’connor Director - Regulatory Policy & Early Access, Association of the British Pharmaceutical Industry

Synopsis

  • Exploring initiatives that provide drugs to children with rare diseases, focusing on specialized approaches and strategies
  • Highlighting the steps and regulatory considerations involved in establishing managed access programs for rare conditions
  • Discussing potential loopholes and challenges in providing very early access to drugs for super rare diseases without robust clinical data
  • Suggesting practical measures that regulatory agencies can implement to facilitate drug development and access for patients with rare conditions
  • Engaging with patient communities groups

2:00 Lunch Break & Networking

Workshop C

Workshop F

3:00 pm Best Practices for Optimizing Stakeholder Engagement & Communication to Enhance Collaboration and Project Success

  • Simone Behre Lead Medical Advisor EMEA, Amylyx Pharmaceuticals Corp.
  • Danielle Rafferty Associate Director, Medical Operations, Global Access Mechanisms, Biogen

Synopsis

  • Learning how to create comprehensive internal policies and guidance for engaging with stakeholders effectively and receive internal buy-in
  • Understanding how to improve communication between programs and suppliers by enhancing supply chain forecasting and timelines
  • Exploring strategies for maintaining compliance and emphasizing the critical nature of programs as a last resort for patients
  • Discovering methods for enhancing communication among HCPs across different countries while adhering to local regulations

3:00 pm Establishing a Robust Forecasting Model for Anticipating Request Volume

Synopsis

  • Identifying the critical information needed to feed into the model for accurate forecasting
  • Exploring existing algorithms that can effectively predict patient request volume
  • Examining the model’s accuracy and the essential variables that contribute to its precision.
  • Determining which stakeholders can leverage the model for improved planning and decision-making

5:00 End of Workshop Day

FULL EVENT GUIDE
CONFERENCE DAY ONE