8:00 am
Coffee & Registration

8:50 am Chair’s Opening Remarks

Reviewing the European Regulatory Environment

9:00 am Panel Discussion: Unpacking Regulations to Successfully Navigate the European Landscape

Synopsis

  • Assessing the key differences between FDA and EMA regulations
  • Addressing the differing early access programme regulations in Europe and how to implement them
  • Effectively conducting programmes in European countries with regulation gaps

9:45 am Examining Early Access Regulations in Emerging European Markets

Synopsis

  • Exploring which countries in Europe have begun to adopt early access programmes
  • Discussing which forms of access are feasible under current regulations
  • Assessing who who is eligible for an early access programme, and who makes this decision

10:15 am
Morning Break & Speed Networking

Reducing Barriers to Entry for Early Access Programmes to Support More Patient Populations

11:15 am How to Launch & Execute an Expanded Access Programme With RWD Collection

Synopsis

  • Assess what is required of a Biopharmaceutical company to launch an EAP with RWD collection
  • Examine the key phases before launching an EA programme
  • Discuss the reporting metrics and insights required to assess an EA programme’s performance
  • Reflect on lessons learned to streamline effective workflows between a service provider and a biopharmaceutical company

11:45 am Outlining Disparities in Compassionate Use Access in Unauthorised Medicines

Synopsis

  • Outlining observed disparity in compassionate use access across countries
  • Analysing the existence and public availability of compassionate use regulations and local clinical trial activity, and their association with compassionate use requests
  • Understanding the association between macroeconomic factors and compassionate use request activity

12:15 pm How To Create a Positive Patient Experience in Early Access Programmes

Synopsis

  • When to engage with the patient community, and the value this engagement will bring to the EAP
  • Educational materials to support patients and families
  • Managing expectations for exit strategy planning for different regulatory outcomes

1:00 pm
Lunch Break

1:45 pm Panel Discussion: Engaging Different Stakeholders to Maximise Early Access Programme Success

  • Erin Lambet Senior Director Expanded Access Program Lead, Argenx
  • Ana Guersoni Medical Affairs Brazil Lead, CSL Behring Biotherapies
  • Anne Raimond-Thibaut Global Oncology Data Generation Director, Global Medical & Patient Affairs, Servier

Synopsis

  • Overcome internal resistance to early access programmes by enhancing stakeholders’ understanding of the protection pharmaceutical companies are granted by European regulations
  • Discussing strategies to convey the value proposition and benefits of EAPs to stakeholders
  • Educating key stakeholders on the strengths and limitations of early access programmes

2:30 pm Ensuring People with Rare Diseases Receive Early Access to Treatments: A Case Study with Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy

Synopsis

  • Running a Gene Therapy Early Access Programme for a rare disease
  • Outlining requirements and global strategies required
  • Understanding the timings and urgency for acute diseases

3:00 pm The Value of Real-World data From Early Access Programmes in Ultra Rare Diseases

Synopsis

  • Preparing for a global venue
  • Managing individual requests when there is no active or ongoing development program
  • Essential elements for closure

 

3:30 pm
Afternoon Break

4:00 pm Working with Physicians & Patients to Increase Access to Early Access Programmes

  • François Houÿez Treatment Information & Access Director & Health Policy Advisor , EURORDIS

Synopsis

  • Building relationships with physicians to increase early access programme awareness
  • Improving your organisation’s transparency to enable doctors and patients to remain informed of which programmes are running in each country
  • Increasing patient involvement while planning an early access programme, to create a programme that is fully patient-centric

4:30 pm Panel Discussion: Involving the Patient Voice in Early Access Programmes

  • François Houÿez Treatment Information & Access Director & Health Policy Advisor , EURORDIS
  • Carole Scrafton Director & Co-Founder, Patient Advocate & Patient Speaker, Flutters and Strutters (FibroFlutters and ZebraStrutters)
  • Sjaak Vink Founder & CEO , Everyone.org

Synopsis

  • Outlining requests from patient advocates for the industry, and the methods to meet them
  • Understanding patient position on data collection through early access programmes
  • Questioning the patient warp speed

5:15 pm Chair’s Closing Remarks & End of Conference Day One