7th Expanded Access Programmes World Congress 2024 Americas

Strategies and Considerations in Launching an Expanded Access Programmes

Boston, MA, USA
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Expanded Access Programmes 2024

Strategies and Considerations in Launching an Expanded Access Programmes 

Dear Colleagues,

Expanded access or compassionate use is the use of an unauthorized drug or medical device under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a clinical trial, by the patient with an immediately life threatening illness or severely debilitating disease or condition who do not meet the enrollment criteria for the clinical trial in progress. These programs go under various names, including early access, special access, or managed access program , compassionate access, named-patient access, temporary authorization for use, cohort access, and pre-approval access.

When patient participation in clinical trials is not possible due to ineligibility or scarcity of ongoing trials, or if enrollment in clinical trials is not conceivable because the distance to the trial inhibits access, expanded access offers a possible route for gaining access to an investigational medical product. These are investigational drugs, biologics or medical devices that are being tested to see whether they are safe and effective. As investigational drugs have not yet received regulatory approval from FDA, the agency has not found these products to be safe or effective for their specific use. It is very important to remember that their potential risks and benefits are not yet established and their effectiveness to treat the condition is not yet known.

With patients thoroughly becoming knowledgeable, Pharmaceutical companies are bound to receive unsought requests. However, there are challenges and obstacles such as a 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.

The objective of this 2-day interactive congress is to provide the audience with imperative skills, best practices, innovations, technologies and concepts in obtaining the much-needed access to these therapies for life-changing treatment, led by key expert stakeholders with intimate knowledge in the industry.

We look forward to meeting you at the Congress!

Sincerely yours,

Jocelyn Raguindin
Conference Director
Paradigm Global Events / Facilitate Live

GAIN LATEST INSIGHTS ON:

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:

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Attendees

Key Industry Expert Speakers

Graham Sidorowicz

EVP Commercial

Bionical Emas

Jennifer M. Joe

Global Medical Strategy & Population Health Director

AstraZeneca

Donna Cowan

Associate Director, Expanded Access Programs

Stealth BioTherapeutics

Norah Xiao

Director, Digital Health Partnership

Astrazeneca

Emily M. Parks

Founder, CEO

POP!

Siddhart Jain

Medical Director, Global Medical Lead

Takeda

Eugean Jiwanmall

Senior Research Analyst for Medical Policy & Technology Evaluation

Independence Blue Cross

Amber Bifolck Fisher

Clinical Research Manager

Kura Oncology, Inc.

Monica L Weldon

President/CEO/Founder Bridge the Gap

SYNGAP Education and Research Foundation

Isabelle Lousada

Founder and CEO

Amyloidosis Research Consortium (ARC)

Rachel Harisson

Associate Director, Early Access Program

Apellis Pharmaceuticals

ALAN J. BALCH

Chief Executive Officer

Patient Advocate Foundation

Wyndi Phillips

Vice President, Project Management

WEP Clinical

Christine Von Raesfeld

Founder & CEO

People with Empathy

Chisa Nosamiefan

Co-founder

The Labalaba Foundation for Lupus

ANNMARIE GALLI

Head, Global Medical Affairs Research and Compassionate Use / Managed Access Programs Oncology

GSK

BHASKAR DUTTA

Head, Digital Health & Medical Affairs Technologies

Alexion

Roberta Smith

President

Alagille Syndrome Alliance

Robert Donnell

Executive Vice President, Medicines Access

Smartway Pharmaceuticals

Lee Sutton

Vice President, Business Development, EAP

WEP Clinical

Senior Representative

Coming Soon!

Available Shortly

Schedule

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

Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross

MACRO OUTLOOK & CURRENT TRENDS

  • 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?

Moderator:
Monica L Weldon, President/CEO/Founde, SYNGAP1 Foundation

Panelist:
Rachel Harrison, Associate Director, Early Access Program, Apellis Pharmaceuticals
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross

Amber Bifolck Fisher, Clinical Research Manager, Kura Oncology, Inc.

Wyndi Phillips, Vice President, Project Management, WEP Clinical 

Annmarie Galli, Head, Global Medical Affairs Research and Compassionate Use / Managed Access Programs
Oncology, GSK

  • Planning and designing for expanded access programs early can be difficult. This panel explores how pharma and biotech can work with Patient Advocacy Groups and Research Consortiums to ensure that the patient voice is part of early planning, and how digital communities can facilitate early planning and
    awareness of expanded access programs.

Jennifer M. Joe, Global Medical Strategy & Population Health Director, AstraZeneca
Bhaskar Dutta, Head, Digital Health & Medical Affairs Technologies, Alexion
Norah Xiao, Director, Digital Health Partnership, Astrazeneca
Isabelle Lousada, CEO and President, Amyloidosis Research Consortium

  • Country Specific Regulatory Pathways
  • Practical Challenges and Solutions in PTA
  • Unique Operational Considerations for PTA

Graham Sidorowicz, EVP Commercial, Bionical Emas

  • 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

Rachel Harisson, Associate Director, Pre-Approval Access Programs, argenx

CHALLENGES & POTENTIALS

  • Setting up objectives of an EAP within the Asset Plan
  • Importance of Early Conversations & cross-functional collaboration of internal stakeholders
  • Managing the expectations of External Stakeholders
  • Mitigating difficult conversations while setting up an EAP

Siddharth Jain, Medical Director –LSDs & HAE, Global Medical Affairs, Takeda

  • Is an Open Label Extension feasible?
  • Is Expanded Access/Compassionate Use the perfect plan?
  • Regulatory Variations and Challenges
  • Lessons Learned

Donna Cowan, Associate Director, EAP & Registry, Stealth Biotherapeutics
Amber Bifolck Fisher, Clinical Research Manager, Kura Oncology, Inc.

  • Advancing Equity
  • Inclusive Communication
  • Culturally Effective Decision Making
  • Voices of Diversity

Chisa Nosamiefan, Co-founder, The Labalaba Foundation for Lupus

  • Deep-dive into the reality of healthcare access around the world
  • Streamlining the process and navigating early access regulations globally
  • What does the future of health access look like? The interconnected ecosystem where all healthcare stakeholders work together

Moderator:

Panelists:

Jennifer E. Miller, PhD, Associate Professor , Yale School of Medicine
Monica L Weldon, President/CEO/Founder, SYNGAP1 Foundation

Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross

ACCESS & COMMERCIALIZATION

  • What are the possible scientific and regulatory approaches to facilitate development?
  • How to enable early access to treat Rare disease patients
  • Ways to guarantee that patient safety, efficacy, and product quality are not compromised
  • A discussion of experiences to date from early access approaches.

Moderator:

Panelist:
Emily Parks, Founder, POP!

Lee Sutton, Vice President, Business Development, EAP, WEP Clinical

  • How important is engaging patient early 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?
  • Sustaining purposeful patient engagement across the lifecycle of medicines

Alan J. Balch, Chief Executive Officer, Patient Advocate Foundation

  • Building your EAP with a ‘site-first’ approach
  • Educating sites on their roles and responsibilities in an EAP
  • Ensuring a clear communication plan to support sites in an expedited manner (people to people versus technology)
  • Case study demonstrating the importance of site management
  • The importance of patient advocacy in providing 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 accelerate patient access to investigational medicine
  • Creating a state of the art payer engagement strategies to address challenges in pricing and reimbursement

Moderator:

Panelist:
Amber Bifolck Fisher, Clinical Research Manager, Kura Oncology, Inc.
Alan J. Balch, Chief Executive Officer, Patient Advocate Foundation
Chisa Nosamiefan, Co-founder, The Labalaba Foundation for Lupus

MULTI-STAKEHOLDER APPROACH & COLLABORATION

  • Ideal Accelerated Pathway
  • Problems of Excluding Some Disease
  • Brainstorming: Removing the Barriers
  • What’s the Long Game?: Rethinking the Regulatory Rules

Monica L Weldon, President/CEO/Founder, SYNGAP1 Foundation

  • The importance of patient advocacy in providing 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 accelerate patient access to investigational medicines
  • Creating a state of the art payer engagement strategies to address challenges in pricing and reimbursement

Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross

  • Value of patient engagement in Early Access Programmes at the earliest stage in research and development
  • 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

END OF CONFERENCE

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