Expanded Access Programmes

7th Expanded Access Programmes World Congress 2024 Americas

STEERING INTRICATE REGULATORY REQUIREMENTS AND CHALLENGES IN SUCCESSFULLY IMPLEMENTING EARLY ACCESS TO MEDICINES

Boston, MA, USA

00Days
00Hours
00Minutes
00Seconds

Book now Apply To Speak

Exhibit & sponsorship opportunities

Expanded Access Programmes 2024

STEERING INTRICATE REGULATORY REQUIREMENTS AND CHALLENGES IN SUCCESSFULLY IMPLEMENTING EARLY ACCESS TO MEDICINES

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 / Facilitate Live

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...

0 +

speakers

days Congress

0 +

Presentation

0 +

Attendees

Key Industry Expert Speakers

Comfirmed speakers Apply to speak

Schedule

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

Day 1 14/11/2024
Day 2 15/11/2024

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

MACRO OUTLOOK & CURRENT TRENDS

9:10 am - OPENING PANEL DISCUSSION: Global Expanded Access Programmes: Challenges, Benefits and Solutions

When should companies consider the idea of Early Access Programmes?
Understanding the challenges in running a global EAP
Navigating country specific framework and requirements
Addressing challenges on patients demands and supply
Positive impact of EAP on patients with serious, immediately life-threatening, or rapidly debilitating diseases

Moderator:

Panelist:

Elsa Luna, Director, Expanded Access Operations, Pfizer

9:50 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.

10:30 am - Expanded Access – The Who, the What, the When, and the How

What is expanded access and when do we use it?
How do we plan for expanded access?
Who needs to be involved with expanded access and what is the key to its success?
What are the means to get the medicine to the patient?

11:00 am - MORNING NETWORKING REFRESHMENTS

11:30 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

Jennifer Grigonis, Sr. Director Business Development EAP, Bionical Emas

Tori Rizk, Associate Portfolio Director, Bionical Emas

Patrick Raletz, Project Manager EAP, Bionical Emas

Juan Rosario, Project Manager EAP, Bionical Emas

12:30 pm - 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

1:00 pm - NETWORKING LUNCH

CHALLENGES & POTENTIALS

2:00 pm - Compassionate Use Challenges for Small Biotech Companies

Opening an EAP without Vendor Support
Considering Stakeholder Expectations
Managing Resource Limitations

Amber Bifolck Fisher, Senior Clinical Research Manager, Expanded Access, Kura Oncology

2:30 pm - Effective site management – the key to executing a successful EAP

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
Case study demonstrating the importance of site management

Melanie Wolf, Associate Director, Global Early Access Programs, Sarepta Therapeutics

3:00 pm - Expanded Access Programmes: Key considerations for successful implementation

Introduction to EAP and their differences
When does the implementation of the Early Access programme be considered?
What are the numerous challenges faced in the implementation of EAPs?
Key considerations for the successful implementation of early access program

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

3:30 pm - AFTERNOON NETWORKING REFRESHMENT

4:10 pm - Role of Empathy: The Patients Perspective of Drug Development and Access

Recognizing the importance of patient-centered approaches.
Advocating for equitable access to medications
Prioritizing empathy and understanding in healthcare decision-making.

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

4:40 pm - Patient/Caregiver perspectives on expanded access programs.

Expanded access is often access to treatment where no other options exist
How to build a patient centric EAP
Will include case study on a rare pediatric programs life saving EAP

Jennifer McNary, Founder, One Rare

5:10 pm - STAKEHOLDERS KEYNOTE PANEL: Reaching the potential of Expanded Access Programmes

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?

5:50 pm - CHAIRPERSON’S CLOSING REMARKS

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

BREAKING DOWN BARRIERS TO PATIENT ACCESS

9:10 am - KEYNOTE PANEL: The importance of patient engagement to drive success in early access programmes

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

Moderator:

Panelist:

9:50 am - Partnerships that work: How to engage and educate patient communities in the expanded access arena.

The need for expanded access in the rare disease community
How increase awareness can accelerate access
Incorporating patient stories and lived experience to drive change

Carlos Izquierdo, Global Pricing & Access Lead – Early Pipeline Gastrointestinal, Takeda

10:20 am - Ethical Obligations Regarding Expanded Access (Compassionate Use)

The ethical imperative for gene therapy companies to proactively develop expanded access policies
Obstacles developers face in providing expanded access to gene therapy products
Conditions with limited windows for treatment deserve special consideration in expanded access

10:50 am - MORNING NETWORKING REFRESHMENTS

11:30 am - Patient Engagement: How can Positive Patient Engagement be Optimized Throughout the EAP Process?

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

12:00 pm - SPONSORED SPOTLIGHT: Planning for success to support an EAP

Dan Lewi , Head, Business Development and Patient Advocacy, Pulse infoframe Inc., Co- founder, CATS
Foundation

12:30 pm - SPONSORED SPOTLIGHT: 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

MULTI-STAKEHOLDER APPROACH & COLLABORATION

2:00 pm - Managing the different stakeholders for EAP for success

Determining if an EAP is right for your therapy and company
Internal stakeholders – all internal functions
External stakeholders – expansion from Physicians and Regulators to all stakeholders (Payors, Reviewers, Patients and others)
Consistent management through concept, initiation, management and closure
Different approaches needed for Gene and highly specialist therapies
Transferring the patient to a treatment site in a different country

2:30 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 reduces polypharmacy issues?
Guidelines to help professionals navigate the opposing explanations of interactions with pharmaceutical companies

3:00 pm - AFTERNOON NETWORKING REFRESHMENT

3:30 pm - STAKEHOLDERS KEYNOTE PANEL: Reaching the potential of Expanded Access Programmes

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?

4:10 pm - CLOSING PANEL DISCUSSION: 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
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?
What does the future of health access look like?

Panelists:

4:10 pm - CHAIRPERSON'S CLOSING REMARKS

END OF CONFERENCE

Expanded Access Programmes 2024 sponsors

Become a sponsor/exhibitor

Newsletter Sign Up

No Spam - You will only receive relevant information on Expanded Access Programmes, and event updates

Delegate Tickets

Group Discounts Are Also Available

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.