- 14th - 15th November 2024
- Boston, MA, USA
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:
- 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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Key Industry Expert Speakers
Eugean Jiwanmall
Senior Research Analyst for Medical Policy & Technology Evaluation
Independence Blue Cross
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
- 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
- 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.
- 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?
- 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
- 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
CHALLENGES & POTENTIALS
- Opening an EAP without Vendor Support
- Considering Stakeholder Expectations
- Managing Resource Limitations
Amber Bifolck Fisher, Senior Clinical Research Manager, Expanded Access, Kura Oncology
- 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
- 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
Christine Istanboulian, Global Medical Operations , Early Access Programs, Sanofi
- 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
- 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?
- 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?
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross
BREAKING DOWN BARRIERS TO PATIENT ACCESS
- 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:
- 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
- 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
- Advancing Equity
- Inclusive Communication
- Culturally Effective
- Decision Making
- Voices of Diversity
- 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
Senior Representative, Pulse Inforframe
- 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
MULTI-STAKEHOLDER APPROACH & COLLABORATION
- 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
- 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
- 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?
- 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:
END OF CONFERENCE
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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.
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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
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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.
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PhD, Managing Director, Atheln, Inc
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- Boston, MA, USA
- USA
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