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

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



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

Previous 2023 Speakers 


President of Innovation, Research, & World Clinic

Sanford Health

Graham Sidorowicz

EVP Commercial

Bionical Emas

Naomi Litchfield Director

Patient Advocacy

Bionical Emas

Senior Representative

Available Shortly

Coming Soon!


Head of Value & Access Oncology, APAC, MEA



Chief Medical Officer, Gene Therapy and Inborn Errors of Metabolism



Director for Global Regulatory & Scientific Policy (GRASP)




PBC Foundation


Operations Lead

NIHR BioResource


Senior Projects Officer

Beacon for Rare Diseases





Chair Elect, Rare Disease Special Interest Group


Josie Godfrey

Co-Founder and CEO

Realise Advocacy


Chair and CEO

AKU Society


Head of Medicine, Life Sciences Division

European Academy of Sciences EURASC


Chief Medical Officer



Group CEO

MPS Society


Head of Expanded Access

Amryt Pharma


Rare Diseases Clinical Lead, National Disease Registration Service

NHS England

Nicola Gillott

Head of Expanded Access

Amryt Pharma plc


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
  • 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
  • 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


  • 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


  • 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
  • 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


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

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
  • 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?
  • 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.
  • 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
  • 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
  • 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.


  • 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
  • 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
  • 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?

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Group Discounts Are Also Available

Advocates // Non Profit

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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