Home » Agenda


6th Expanded Access Progammes World Congress 2024 Europe

Copthorne Tara Hotel London Kensington, London, United Kingdom

Thursday 11th - Friday 12th April 2024

Facilitate Live is very pleased to welcome you to our upcoming 6th Expanded Access Programmes World Congress 2024 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  


  • 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


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
  • Corporate Development
  • RWE, and Data Management
  • Quality and Compliance
  • Clinical Development Programme
  • Management
  • Supply Chain Management
  • Patient Access
  • Clinical Research
  • Sourcing and Logistics
  • Consulting
  • Marketing and commercialisation
  • Licensing and authorisation
  • Pricing and reimbursement
  • And much more…


  • 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

Nora Pöntynen, Global Medical Affairs, Boehringer Ingelheim International GmbH
Yasmina Tazir, Sr Director Clinical Science, Clinical Policy, Chief Medical Office, AstraZeneca

  • 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

Pedro Franco, Sr Director, Head, Europe Global Regulatory & Scientific Policy, Merck Healthcare

  • 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
  • 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

Cameron Miller, Director of Policy & Strategy, The Brain Tumour Charity


  • 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

Senior Representative, WEP Clinical

  • 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

Gordon Lundie, Executive Director, Market Access and Pricing, Gilead Sciences

  • 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


  • Internal stakeholder education and engagement
  • Setting global and local expectations
  • Cross functional consultations and considerations
  • Road map for development through implementation

Annie O’Keefe Martin, Global Medical Governance Lead for Managed Access Programs and Rare Humanitarian Programs, Sanofi

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

Lindsay Birrel, Co- CEO, Realise Advocacy, Chair of trustees, Medics 4 Rare Diseases

  • 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

Katja Berg, Innovative Value and Access Strategy, AstraZeneca

  • 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

Dr.Dinesh Kumar, International Medical Director, Medical Review & Compliance, Alnylam UK Ltd

  • 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
  • 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

Katja Berg, Innovative Value and Access Strategy, AstraZeneca
Paul Blakeley, Sr Policy Advisor, Health and Life Sciences, Tony Blair Institute for Global Change 

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


  • 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
  • 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

Sheela Upadhyaya, Chair Elect, Rare Disease Special Interest Group, ISPOR

  • 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?

Nora Pöntynen, Global Medical Affairs, Boehringer Ingelheim International GmbH

  • 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

Dr. Alberto Calabrò, Patient Access Program and Supply Leader, F. Hoffmann-La Roche Ltd

  • 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
  • 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.


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

Julie Powell, Marketing & Communications Director, Pleco Therapeutics

  • 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

Dr James Duboff, Strategic Partnerships Director, Genomics England

  • Operational aspects for global and local implementation
  • Safety Reporting Training for HCPs
  • Health Authority attention to and interest in safety reports
  • Inspection experience

Annie O’Keefe Martin, Global Medical Governance Lead for Managed Access Programs and Rare
Humanitarian Programs, Sanofi

  • 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
  • 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?
Scroll to Top