5th Expanded Access Programmes World Congress 2023 Americas

Strategies and Considerations in Launching an Expanded Access Programmes

Revere Hotel Boston Common, Boston, MA, USA
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Expanded Access Programmes

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

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. 

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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

Eugean Jiwanmall

Sr Research Analyst, Med. Policy & Tech Evaluation

Independence Blue Cross

Jean Godin

Head, Global Access Programme

Sanofi

Christine Von Raesfeld

Founder & CEO

People with Empathy

Richard Klein

Director, Expanded Access Programs & Policy

GE2P2

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.

  • 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

  • In an era where ‘real-world data’ is increasing of interest, what are the limitations?
  • Data collection within early access programs in various regions
  • Ethical considerations and operational challenges in conducting data collection
  • Benefits of collecting RWD within early access programs
  • Country Specific Regulatory Pathways
  • Practical Challenges and Solutions in PTA
  • Unique Operational Considerations for PTA

Graham Sidorowicz, EVP Commercial, Bionical Emas

  • 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

CHALLENGES & POTENTIALS

  • 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
  • The main challenges in the execution of an EAP for cell and gene therapies are:
  • Treatment is generally available in a restricted number of specialist centres, so patients need to travel for
    treatment
  • The treatment is generally high-value, particularly in the case of rare indications.
  • The presentation will highlight lessons learnt and innovative solutions for EAPs and cross- border programs
    for cell and gene therapies 
  • 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 workingrelationship
  • 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

  • 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
  • What are the possible scientific and regulatory approaches to facilitate development?
  • How to create robust quality data packages to enable timely access to medicines for patients
  • Ways to guarantee that patient safety, efficacy, and product quality are not compromised
  • A discussion of experiences to date from early access approaches.

5:45 - 6:45 pm - Networking Drinks Reception - Sponsored by Bionical Emas

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

ACCESS & COMMERCIALIZATION

  • 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 healthcarestakeholders work together

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

  • 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/Founde, SYNGAP1 Foundation

  • Building access specific competencies in emerging Market Medical Affairs
  • Pivotal role of Early Access Programs

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

  • 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

Donna Cowan, Associate Director, EAP & Registry, Stealth Biotherapeutics*

  • 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 patientaccess to investigational medicine
  • Creating a state of the art payer engagement strategies to address challenges in pricing and reimbursement

Moderator:

Panelist:
Misty Gravelin, Project Lead, Expanded Access Support Unit, University of Michigan
Alan J. Balch, Chief Executive Officer, Patient Advocate Foundation
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross

MULTI-STAKEHOLDER APPROACH & COLLABORATION

  • 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
  • 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

  • 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 reducespolypharmacy 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 patientaccess to investigational medicines
  • Creating a state-of the art payer engagement strategies to address challenges in pricing and reimbursement

END OF CONFERENCE

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$799
  • Full Access - 2 Day Conference
  • All Day Refreshments & Lunches
  • Conference Documentations
  • Networking Drinks Reception

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Full Price
$1849
  • Full Access - 2 Day Conference
  • All Day Refreshments & Lunches
  • Conference Documentations
  • Networking Drinks Reception

Testimonials

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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