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
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
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
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