Expanded Access Programmes

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

Introduction
Insight
Who Should Attend

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:

The current compassionate use, or expanded access-related mechanisms
The benefits and issues surrounding expanded access programme
Understanding the varying regulatory landscape, policies and guidelines to ensure a smooth execution of the programme
Criteria that must be met to authorize expanded access use, requirements for expanded access submissions
Understanding operational complexities, best practices and strategies in running a global EAP
Successfully navigating logistical intricacies
Incorporating patient voice from early planning to ensure a patient-centric access programme
Engaging representatives from all stakeholder groups to understand their perspective and expectations of the programme
Robust data collection strategies for informed decision making
Real-world data challenges and solutions
And much more...

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:

Expanded Access
Managed Access
Early Access
Market Access
Medical Affairs
Regulatory Affairs and Policy
Patient Support and Patient Engagement
RWE, and Data Management
Quality and Compliance
Clinical Development
Programme Management
Supply Chain Management
Patient Access
Clinical Research
Global Head, Digital Accelerator
Patient Engagement & Portfolio Strategy
Patient Support Strategy & Insights
Patient Experience

Medical Affairs, Global Patient Advocacy & Alliances
Government Policy and Advocacy
Digital Patient Experience Lead
Clinical Operations
Clinical Insights and Experience
Head of Strategy, Access Services
Vice President, Site Collaborations and Patient Centricity
Head of Neuroscience
Sourcing and Logistics
Consulting
And much more...

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Schedule

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

Day 1 16/11/2023
Day 2 17/11/2023

8:00 am - WELCOME COFFEE & REGISTRATION

8:50 am - CHAIRPERSON’S OPENING REMARKS

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

MACRO OUTLOOK & CURRENT TRENDS

9:00 am - OPENING PANEL DISCUSSION: Realizing a Global Expanded Access Programme

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.

9:45 am - KEYNOTE PANEL: Digital Patient Communities, Patient Advocacy Groups, and Expanded Access

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

10:30 - MORNING NETWORKING REFRESHMENTS

11:10 am - SPONSORED SPOTLIGHT: Real-World Data collection in Early Access Programs: Challenges and Opportunities

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

11:45 am - SPONSORED SPOTLIGHT: Challenges and Solutions Utilizing EAP Pathways for Post Trial Access

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

Graham Sidorowicz, EVP Commercial, Bionical Emas

12:20 pm - PLENARY SESSION: Patient-centric approach to Expanded Access Programmes

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

1:00 pm - NETWORKING LUNCH

CHALLENGES & POTENTIALS

2:00 pm - The importance of patient engagement to drive success in early access programmes

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

2:35 pm - SPONSORED SPOTLIGHT: Practical considerations when planning and implementing cell and gene therapy Expanded Access programs

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

3:10 pm - The importance of patient engagement for successful early access programmes

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

3:45 pm - AFTERNOON NETWORKING REFRESHMENT

4:15 pm - From Expanded Access Data, Analytics, and Insights to the Decision Making

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

4:50 pm - KEYNOTE PANEL DISCUSSION: Challenges faced by applicants to complete quality,manufacturing development and data requirements during the development of products in early accessapproaches.

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.

5:35 pm - CHAIRPERSON’S CLOSING REMARKS

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

8:00 am - WELCOME COFFEE & REGISTRATION

9:00 am - CHAIRPERSON’S OPENING REMARKS

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

ACCESS & COMMERCIALIZATION

9 :10 am - STAKEHOLDERS KEYNOTE PANEL: Global Health Access for Everyone

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

9:55 am - Things to Consider: Expanded Access for Rare Disease

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

10:30 am - MORNING NETWORKING REFRESHMENTS

11:00 am - Shaping the Future of Rare Diseases in Emerging Markets: A Medical Affairs Perspective

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

11:35 am - Expanded Access: Organizational strategies drug companies should adopt

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*

12:10 pm - PANEL DISCUSSION: Multi-Stakeholder Approaches to accelerating access to therapies – withemphasis to collaboration between patient advocacy groups, health professionals, manufacturers & Payers

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

1:00 pm - NETWORKING LUNCH

MULTI-STAKEHOLDER APPROACH & COLLABORATION

2:00 pm - Designing innovative models for Manage access programmes internally

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

2:30 pm - Patterns of alternative access: Unpacking extraordinary drug reimbursement system and engaging with payers

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

3:00 pm - Collaborations between healthcare providers and life science companies for greater access

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

3:30 pm - AFTERNOON NETWORKING REFRESHMENT

4:00 pm - CLOSING PANEL DISCUSSION: Multi-Stakeholder Approaches to accelerate access to therapies– with emphasis to collaboration between patient advocacy groups, health professionals, manufacturers &Payers

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

4:45 pm - CHAIRPERSON'S CLOSING REMARKS

END OF CONFERENCE

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

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

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

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.