5th Expanded Access Programmes World Congress 2023 Americas

Strategies from planning an application to approval and implementation

Boston, MA, USA
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Expanded Access Programmes

Strategies from planning an application to approval and implementation

Dear Colleagues,

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

Graham Sidorowicz

EVP Commercial

Bionical Emas

Naomi Litchfield Director

Patient Advocacy

Bionical Emas

Donna Cowan

Associate Director, Expanded Access Programs

Stealth BioTherapeutics

Misty Gravelin, MPH, RAC

Project Lead, Expanded Access Support Unit

University of Michigan

Rachel Harrison

Associate Director, Early Access Program

Apellis Pharmaceuticals

Richard Klein

Director, Expanded Access Programs & Policy

GE2P2 Global Foundation

Eugean Jiwanmall

Senior Research Analyst for Medical Policy & Technology Evaluation

Independence Blue Cross

Neena Nizar

President & Founder

The Jansens Foundation

Monica L Weldon

President/CEO/Founder Bridge the Gap

SYNGAP Education and Research Foundation

Christine Von Raesfeld

Founder & CEO

People with Empathy

Magali Taiel, M.D.,

Chief Medical Officer


Jean Godin

Head, Global Access Programme


Eugean Jiwanmall

Sr Research Analyst, Med. Policy & Tech Evaluation

Independence Blue Cross

Lindsay Birrell

Co-Founder & CEO, Realise Advocacy, Trustee

Medics 4 Rare Diseases

Nicola Gillott

Head of Expanded Access

Amryt Pharma plc

Senior Representative

Available Shortly

Bionical Emmas

Richard Klein

Director, Expanded Access Programs & Policy


Senior Representative

Available Shortly

Bionical Emmas


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 Americas?
  • Addressing the complex European regulatory framework
  • Key considerations and current trends within expanded access programmes
  • Strategies and lessons learned in launching several country-specific expanded access programmes in the US
  • 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 physicians
  • In an era where “real-world data” is increasingly of interest, what limitations should exist?
  • Data collection guidelines within early access programmes in various regions
  • Ethical consideration and operational challenges in conducting data collection
  • Benefits of collecting RWD within early access programmes


  • Programme design strategies to optimise benefits and reduce risk
  • State of the art real-world data collection and analysis
  • Breaking down intricate global regulatory framework
  • Accountable world class global distribution of investigational drugs
  • 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


  • 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.
  • Challenges & Opportunities of a company-wide roll-out of Early Access Programme
  • Strategies & consideration for a successful implementation
  • Established responsibilities from everyone involved to ensure smooth delivery to patients in need
  • 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?
  • 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
  • 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

5:30 - 6:30 pm - Networking Drinks Reception - Sponsored by Bionical Emmas


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


  • Overcoming challenges brought by the pandemic
  • Patient-centred trial design to accommodate changes in patients need
  • Lessons learned and global distribution management
  • 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
  • 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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