6th Expanded Access Programmes World Congress 2024 Europe

Navigating intricate regulatory requirements and addressing operational complexities

Holiday Inn London - Bloomsbury, an IHG Hotel, London, United Kingdom
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Expanded Access Programmes 2024 Europe

Navigating intricate regulatory requirements and addressing operational complexities

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



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:

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Key Industry Expert Speakers

2024 Speakers 

Annie O’Keefe Martin

Global Medical Governance Lead for Managed Access Programs and Rare Humanitarian Programs


Nora Pöntynen

Global Medical Affairs

Boehringer Ingelheim International GmbH

Gordon Lundie

Executive Director, Market Access and Pricing

Gilead Sciences


Sr Director, Head, Europe Global Regulatory & Scientific Policy

Merck Healthcare

Sara Radenovic


Managed Access, GSK

Katja Berg,

Innovative Value and Access Strategy


Jennifer Grigonis

Sr. Director Business Development, EAP

Bionical Emas

Dr David Gillen

Chief Medical Officer


Dr. Alberto Calabrò

Patient Access Program and Supply Leader

F. Hoffmann-La Roche Ltd

Ana Tediosi

Head of Expanded Access Strategy

WEP Clinical

Cameron Miller

Director of Policy & Strategy

The Brain Tumour Charity

Sheela Upadhyaya

Chair Elect, Rare Disease Special Interest Group


Julie Powell

Marketing & Communications Director

Pleco Therapeutics

Dr James Duboff

Strategic Partnerships Director

Genomics England

Robert Donnell

Executive Vice President – Medicines Access

Smartway Pharma

Paul Blakeley

Sr Policy Advisor, Health and Life Sciences

Tony Blair Institute for Global Change

Bob Stevens

Group CEO

MPS Society

Lindsay Randall

Founder, CEO

Mother, Arthur’s Quest

Carole Scrafton

Director & Co-Founder

Flutters and Strutters

Menno A. Kiel

former Global Medical Leader, Research Physician & Health Economist


Lorna Pender

Patient Group Lead


Hannah Plowman

Real World Data Specialist


Juliet Hulse

Head of Clinical Operations

Pulse infoframe Inc.

Dan Lewi

Head of Business Development and Patient Advocacy

Pulse infoframe Inc., Co- founder, CATS Foundation


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


  • 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
Robert Donnell, EVP, Medicines Access, Smartway Pharma

Dr.Dinesh Kumar, International Medical Director, Alnylam UK

  • 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

  • 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


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

Ana Tediosi, Head of Expanded Access Strategy, WEP Clinical

  • 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

Robert Donnell, EVP, Medicines Access, Smartway Pharma, a leading provider of Early / Expanded Access Programs (EAP).

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

Bob Stevens, Group CEO, MPS Society


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

Jennifer Grigonis, Sr. Director Business Development, EAP, Bionical Emas

  • 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

  • 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

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

Menno A. Kiel, Global Medical Leader, Research Physician & Health Economist, Roche


  • 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 

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

Dr James Duboff, Strategic Partnerships Director, Genomics England

Robert Donnell, EVP, Medicines Access, Smartway Pharma

Dan Lewi, Head of Business Development and Patient Advocacy , Pulse infoframe Inc., co- founder CATS


  • 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

Carole Scrafton, Director & Co-Founder, Flutters and Strutters
Dr Lorna Pender, Global Patient Engagement Lead, Clinigen Group

Hannah Plowman, Real World Data Specialist, Clinigen Group

  • How should societies address a request for a single patient in need when a drug is not being made broadly
  • 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

  • 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

Dr David Gillen, Chief Medical Officer, Norgine

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

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

Sara Radenovic, Director, Managed Access, GSK
Ana Tediosi, Head of Expanded Access Strategy, WEP Clinical

Robert Donnell, EVP, Medicines Access, Smartway Pharma

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

Julie Powell, Director, Patient Advocacy and Public Affairs, Pleco Therapeutics


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

Lindsay Randall, Founder, CEO, Mother, Arthur’s Quest

  • 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 

  • 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.

Dan Lewi, Head of Business Development and Patient Advocacy , Pulse infoframe Inc., co- founder CATS

Juliet Hulse, Head of Clinical Operations, Pulse infoframe Inc.

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

Standard Registration Rate

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


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.
Adriana E. Manzi
PhD, Managing Director, Atheln, Inc

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