Expanded Access Programmes

6th Expanded Access Programmes World Congress 2024 Europe

Navigating intricate regulatory requirements and addressing operational complexities

Copthorne Tara Hotel London Kensington, London, United Kingdom

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Expanded Access Programmes 2024 Europe

Navigating intricate regulatory requirements and addressing operational complexities

Introduction
Insight
Who Should Attend

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 11/04/2024
Day 2 12/04/2024

Auditorium 1

8:00 am - Conference Registration and Welcome Coffee

9:00 am - Chairpersons Welcome and Opening Remarks

OVERVIEW OF EUROPEAN REGULATORY LANDSCAPE

9:10 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

9:50 am - OPENING KEYNOTE: MHRA International Recognition Procedure (IRP): A New Licensing Route for medicines

Understanding the new MHRA’s IRP framework
How does it compare to other regulatory guidelines currently available
The significance of IRP for marketing authorization and patient access

10:20 am - KEYNOTE SESSION: Update on the UK's Early Access to Medicine Scheme (EAMS)

Key legislative changes to maximise the Scheme’s impact by accelerating availability of medicines for patients
Facilitating collection of real-world data which may support regulatory decision making
Implications to pharma industry and manufacturers supplying EAMS medicines
Benefits to patients and physician

10:50 - MORNING NETWORKING REFRESHMENTS

STRATEGIES AND CURRENT TRENDS

11:30 am - SPONSORED SPOTLIGHT: Patient Engagement: How can Positive Patient Engagement be Optimized Throughout the EAP Process?

Understanding the disease area and impact on the patient and family
When to engage with patient advocacy organisations and the value they can bring
Best practices to ensure patients are partners in your clinical development process

12:00 am - PLENARY SESSION: Patient-centric approach to Expanded Access Programmes

Considerations in planning EAP’s with patient in mind
Engaging with patient and advocacy groups early in development for well informed decision
Strategies to ensure patients voice is incorporated in development discussions
When to consider EAP and when it might not be the right thing to do for the community

12:30 pm - SPONSORED SPOTLIGHT: Real-world data from expanded access programmes – Where are we now?

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

1:00 pm - NETWORKING LUNCH

MULTI-STAKEHOLDER APPROACH & COLLABORATION

2:00 pm - Early Access to Medicines - A patients view

Before considering Early Access Patient Organisations & Patients need to be engaged much earlier in the process
Think Natural History
Think Reimbursement not Approval
Understand what it is that you want to demonstrate and how (Biomarkers – science fiction not real world)
Don’t just think data – Think about telling a story

2:30 pm - Patients considerations for Early Access Programmes

Understanding what EAP are for
Physicians responsibility in applying for EAP
Meeting criteria for EAP
Learning how patients will be transitioned to commercial supply

3:00 pm - 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

3:30 pm - AFTERNOON NETWORKING REFRESHMENT

4:10 pm - Collaborations between healthcare providers and life science companies for greater access

Interaction between pharmaceutical companies and healthcare providers could foster the development of new medicines which will ultimately benefit patients.
Would this enhance technological innovation, fosters knowledge creation, aids disease control, and reduces polypharmacy issues?
Guidelines to help professionals navigate the opposing explanations of interactions with pharmaceutical companies

4:40 pm - Increasing health equity with Early Access

Importance of early access to medicine in deploying treatments to the patients who need it
Sustainable approaches to use pathways in low and middle income countries
Addressing logistical barriers to Access
Ethical issues and considerations

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

6:00 - 7:00 pm - Networking Drinks Reception

Auditorium 1

8:30 am - WELCOME COFFEE & REGISTRATION

9:00 am - CHAIRPERSON’S OPENING REMARKS

BREAKING DOWN BARRIERS TO PATIENT ACCESS

9:10 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

9:50 am - Strategic 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?

10:20 am - Early Access and Rare Diseases Innovations

Compassionate use programmes are instrumental in ensuring continuity of access to drugs until approval and reimbursement decisions are finalised.
The importance of a registry of drugs offered on a compassionate use basis
Further transparency to allow such programmes to be evaluated and direct patients to sources of treatment.

10:50 - MORNING NETWORKING REFRESHMENTS

11:30 am - From Expanded Access Data, Analytics, and Insights to the Decision Making

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

12:00 pm - SPONSORED SPOTLIGHT: 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 uptak

12:30 pm - SPONSORED SPOTLIGHT: Expanded Access Programmes in Advanced Cell Therapies

Main challenges in the execution of an EAP for cell and gene therapies
How common are expanded Access approvals in the field of advanced cell therapy?
Are there any interesting parameters by which patient can participate?
Who bears the burden of paying for investigational therapies used in pre-approval non-trial settings?
Lessons learnt and innovative solutions for EAPs and cross-border programs for cell and gene therapies.

1:00 pm - NETWORKING LUNCH

CHALLENGES AND POTENTIAL OF EAP

2:00 pm - Expanded Access: Organizational strategies drug companies should adopt

Companies should anticipate the need for EA and should have a written policy
The policy should be available to the person entitled to expanded access
Companies must be accountable to the patients sending expanded access requests
Sharing of data analysis is crucial for improvement

2:30 pm - The Impact of Pre-approval Access on the developers of new medicines

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

3:00 pm - AFTERNOON NETWORKING REFRESHMENT

3:30 pm - Patterns of alternative access: Unpacking extraordinary drug reimbursement system and engaging with payers

Alternative access schemes offer flexibility to policymakers but often lack transparency and clear rules.
The uncertainties allow for dynamic responses to weaknesses in the main drug approval and reimbursement systems
Challenges facing the reimbursement system
Understanding different payer strategies currently in place

4:00 pm - KEYNOTE PANEL DISCUSSION: Reaching the potential of Expanded Access Programmes: This decade and beyond

Drivers of early access programmes
Setting up a clear perspective and expectations of the programme
Streamlining process and ensuring early access of medicinal products to patients
How regulators and reimbursement agencies can work together to help ensure that patients are able to access life changing medicines as soon as it is safe for them to do so?

4:40 pm - CHAIRPERSON’S CLOSING REMARKS

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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.