Expanded Access Programmes

8th Expanded Access Programmes World Congress 2025 Europe

Comprehensive Analysis on Executing Expanded Access Programmes

Copthorne Tara Hotel London Kensington, London, United Kingdom

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Co-located with Orphan Drugs & Rare Diseases Global Congress 2025 Europe

Expanded Access Programmes 2025 Europe

Comprehensive Analysis on Executing Expanded Access Programmes

Introduction
Insight
Who Should Attend

Facilitate Live is very pleased to welcome you to our upcoming 8th Expanded Access Programmes World Congress 2025 Europe.

Early access programmes offer ethical, compliant and controlled mechanisms of access to
investigational medicinal products (IMP) outside of the clinical trial space and before marketing and authorisation.

Expanded Access also known by other terms such as compassionate use, early access, managed access, named-patient access, or pre-approval access is a potential pathway for a patient with an immediately life-threatening illness or severely debilitating disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) before they are reviewed and approved by a regulatory authority. When it is not possible for a patient to participate in a clinical trial and all other available medical options have been exhausted, the patient’s physician may seek special access to investigational medicine outside of the clinical trial setting. As investigational drugs have not yet received regulatory approval, it is very important to remember that their potential risks and benefits are not yet established. In general the person and his or her doctor must apply for access to the investigational product, the company has to choose to cooperate, and the medicines regulatory agency needs to agree that the risks and possible benefits of the drug or device are understood well enough to determine if putting the person at risk has sufficient potential benefit.

With patients thoroughly becoming knowledgeable, Pharmaceutical companies are bound to receive unsought request. However, there are challenges and obstacles such as diverse regulatory landscape, logistical differences, and complexities from planning an application to approval and implementation. Sponsors need to plan carefully before undertaking an Expanded Access Programme so that they have the resources, processes, and tools in place to support the inevitable demand.

EAPs promises huge potential to all stakeholders involved, from patients who obtained the medicines early to the pharmaceutical companies who provides it. Companies who invest in them may see considerable benefit in terms of relationship building with key stakeholders, such as patients, advocacy groups, scientists and regulators, as well as launch preparedness.

The two-day Congress will provide an interactive, cutting edge and comprehensive discussion and networking format led by key expert speakers with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies and concepts in obtaining the much needed access to these therapies for life changing treatment.

We look forward to meeting you at the Congress!

Sincerely yours,

Jocelyn Raguindin
Conference Director
Paradigm Global Events / Facilitate Live

GAIN LATEST INSIGHTS ON:

Understanding the varying regulatory landscape, policies and guidelines to ensure a smooth execution of the programme
The benefits and issues surrounding expanded access programme
Criteria that must be met to authorize expanded access use, requirements for expanded access submissions
The current compassionate use, or expanded access-related mechanisms
Understanding operational complexities, best practices and strategies in running a global EAP
Current trends and lessons learned from successful implementation
Successfully navigating supply and logistical intricacies
Incorporating patient voice from early planning to ensure a patient-centric access programme
Robust data collection strategies for informed decision making
Real-world data challenges and solutions
Engaging representatives from all stakeholder groups to understand their perspective and expectations of the programme
And much more...

WHO SHOULD ATTEND?

This Congress will provide information to
pharmaceutical and biotech companies, researchers, physicians, institutional review boards (IRBs), patient organizations and advocates, and regulatory agencies about the implementation of expanded access to investigational drugs.

Network with representatives involved in the following areas:

Expanded Access
Managed Access
Early Access
Market Access
Medical Affairs
Regulatory Affairs and Policy
Patient Support and Patient Engagement
RWE, and Data Management
Quality and Compliance
Clinical Development
Programme Management
Supply Chain Management
Patient Access
Clinical Research
Global Head, Digital Accelerator
Patient Engagement & Portfolio Strategy
Patient Support Strategy & Insights
Patient Experience

Medical Affairs, Global Patient Advocacy & Alliances
Government Policy and Advocacy
Digital Patient Experience Lead
Clinical Operations
Clinical Insights and Experience
Head of Strategy, Access Services
Vice President, Site Collaborations and Patient Centricity
Pricing and reimbursement
Licensing and authorisation
Head of Neuroscience
Sourcing and Logistics
Consulting
And much more...

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Schedule

Content Rich Program Agenda! Featuring Keynote Presentations, Stream Sessions, Panel, and Round Table Discussions.

Day 1 19/03/2025
Day 2 20/03/2025

Auditorium 1

8:00 am - Conference Registration and Welcome Coffee

8:30 am - CHAIRPERSON’S OPENING REMARKS

OVERVIEW OF EUROPEAN REGULATORY LANDSCAPE

08:40 am - OPENING KEYNOTE PANEL: Navigating the complex regulatory framework that defines the Expanded Access Programme

Understanding the challenges in planning and implementing a global EAP
Country specific framework and requirements to access investigational drugs
Scientific elements and regulatory tools available to address the challenges and explore the flexibility within the current regulatory systems
Significant degree of administrative effort required to initiate the programme for a timely access
How far are we from establishing a harmonised guidelines across Europe

Panelist:

9:20 am - OPENING KEYNOTE: Missing the point of early treatment access – the UK must do more

Overview of the access journey in the UK
Investigation of the benefits and challenges of Brexit for early and expanded access
How early access can benefit patients
Gaps in the UK against other countries
Where VPAS 2024 dropped the ball
Where access could go in future

09:55 am - PLENARY SESSION: Varying regulatory landscape, and the impact on a global patient community

Patients are well informed and capable of understanding where in the world treatments are available through expanded access
Not every regulatory framework is favourable and this can lead to inequality and a frustrated patient community

10:30 - MORNING NETWORKING REFRESHMENTS

STRATEGIES AND CURRENT TRENDS

11:10 am - SPONSORED SPOTLIGHT: Navigating the Transition of Assets with an Expanded Access Program

Defining key milestones and timelines effectively
Selecting the appropriate stakeholders for involvement
How to effectively manage communication and leadership when multiple companies are involved

11:50 pm - SPONSORED SPOTLIGHT: Initiating and managing EAP’s which meet the needs of Patient, HCP and Manufacturer

Integrating an EAP with the therapy clinical and commercialization plan
– Internal stakeholders
– Alignment on objectives
– Optimal timings for planning and initaition
Working with external stakeholders
– Stakeholder mapping
– Engaging with patient and advocacy groups early in development for well informed decision
– Considerations in planning EAP’s with patient in mind
Practical guide to initiation
The link to Market Access

12:30 pm - PLENARY SESSION: Expanded Access Programmes – The Patient Impact

The Landscape
A UK perspective
The Good, The Bad & The Ugly
What does this mean for patients and their future
Next Step

1:05 pm - NETWORKING LUNCH

MULTI-STAKEHOLDER APPROACH & COLLABORATION

2:05 pm - SPONSORED SPOTLIGHT: Ethical Decision-Making for Early Access to Investigational Medicines in Rare Disease

Defining guiding principles
Determining the key stakeholders
Considerations for a decision-making framework

2:45 pm - PLENARY SESSION: Benefits of Early Access Programmes

Why do we need early access programs
What value do they bring and to who
What are the opportunities and challenges
Who should you engage with and when

3:20 pm - AFTERNOON NETWORKING REFRESHMENT

4:00 pm - PLENARY SESSION: Navigating the link between early access and true access

The market access perspective – when and how expanded access is helpful
Expanded access in the context of the overall access strategy
Going beyond the large countries to a global strategy

4:35 pm - A physician’s perspective on advancing EAPs

Being inclusive to HIV+ patients
When the patient disagrees – Dealing with online petition
The right to a second-opinion following a rejection

5:10 pm - KEYNOTE PANEL DISCUSSION: Multi-Stakeholder Approaches to accelerate access to therapies – with emphasis to collaboration between patient advocacy groups, health professionals, manufacturers & Payers

The importance of patient advocacy in providing a much-needed information on EAP’s available for patients
Collaborating with one another to enable a much richer understanding of patient needs
Patient groups importance in working with health authorities to advance policies that accelerates patient access to investigational medicines
Creating a state-of the art payer engagement strategies to address challenges in pricing and reimbursement

5:50 pm - CHAIRPERSON’S CLOSING REMARKS

Auditorium 1

8:30 am - WELCOME COFFEE & REGISTRATION

8:30 am - CHAIRPERSON’S OPENING REMARKS

BREAKING DOWN BARRIERS TO PATIENT ACCESS

08:40 am - OPENING PANEL DISCUSSION: The importance of patient engagement to drive success in early access programmes

Value of patient engagement in Early Access Programmes
How important is engaging patient at the earliest stage in research and development
The need for a continuous meaningful dialogue between stakeholders to establish an effective working relationship
What are the best practices and ethics behind a successful early access programme?
Knowing what motivates the stakeholders within early access
Sustaining purposeful patient engagement across the lifecycle of medicines

9:20 am - PLENARY SESSION: Ethical issues that need to be addressed in making decisions regarding pre- approval access

How should societies address a request for a single patient in need when a drug is not being made broadly
available?
Could sponsors or governments ethically say yes to one person and no to others?
Who should be deciding which patients will live?

9:55 am - PLENARY SESSION: Real-world data from expanded access programmes – Where are we now?

Value of real-world data in EAP
The use of real-world data for Market access and reimbursement
Regulatory considerations and country differences for collecting real-word data
Case studies

10:30 - MORNING NETWORKING REFRESHMENTS

11:10 am - PLENARY SESSION: The meaning of collecting and using data from patients enrolled in EAPs

Different source for gathering data from EAPs
Inherent collection of patient data
EAP vs RWE
Continue to focus providing access
Optimize contact with HCPs

11:45 pm - PANEL DISCUSSION: The importance of patient forecasting in Early Access Programmes

Why is patient forcasting important in planning early access programmes
Factors that could influence patient uptake in early access programmes
Overcoming difficulties that affects patient uptake

12:25 pm - PLENARY SESSION: Strategic Planning for Effective EAP in Small Pharma: From Conceptualization to Choosing the Right Vendor

EAP Essentials for Small Pharma
Planning and Compliance
EAP Design Principles
Vendor Selection Criteria

1:00 pm - NETWORKING LUNCH

CHALLENGES AND POTENTIAL OF EAP

2:00 pm - PLENARY SESSION: Translating science for patients: Patient Organisation Funded Trial Results Are Life Changing, But The Fight For Access Goes On.

Open Label Trial
20 children, 2 rare disease
Encouraging results lead to off label prescribing
Inequitable access and reimbursement
The fight goes on

2:35 pm - PLENARY SESSION: How genomic sequencing improves drug development

National genomic sequencing programme for cancer and rare disease
Clinical genomic data enhances target selection and validation
Using genomics to improve efficacy and safety of therapeutic candidates
Incorporation of genomics into patient selection / enrolment criteria
Early intervention enablement using genomics

3:10 pm - The role of EAP in establishing a pathway for drugs to be made available to families.

What to do if a drug is ultimately not approved for funding
How can patient families help advance the funding and approval process with HTAs
What role does world data and observational studies play in supporting the pricing reimbursement decision
What does one do if the funding process fails.

3:45 - pm - CHAIRPERSON’S CLOSING REMARKS

End of Conference

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Full Access - 2 Day Conference
All Day Refreshments & Lunches
Conference Documentations
Networking Drinks Reception

Full Access - 2 Day Conference
All Day Refreshments & Lunches
Conference Documentations
Networking Drinks Reception

Full Access - 2 Day Conference
All Day Refreshments & Lunches
Conference Documentations
Networking Drinks Reception

Full Access - 2 Day Conference
All Day Refreshments & Lunches
Conference Documentations
Networking Drinks Reception

Testimonials

what people say

Thank you for a good experience with the PARADIGM Global Events… very high level, very good Organization, we overpassed our expectatives, we will be pending for another congress related to pharmaceutical and biotech… we learned a lot, and i think that the content of agenda was state of the art, thank you again.

Salvador Berrios

ERP/ CRM / Product Configurator Manager

The consistently high quality of international faculty was most impressive. It was also obvious that they were carefully chosen not only for their particular expertise but also for their ability to communicate the subject matter to clinical and non-clinical participants alike. As market access consultant for pharma clients of various sizes, I now feel much better prepared to explain the nuances & dynamics involved in bringing their products to market. In addition, I am in a much better position to intelligently advice my clients & help them set realistic expectations

Thomas Martin

Associate Vice President, VCGA - Quintiles

I was impressed with the organization of this event. I attend many conferences per year and find the organizers on site to be detached and not focus on good service to the delegates. This team was even providing weather forecast warning to ensure everyone was aware and made appropriate plans. Well Done.