7th Expanded Access Programmes World Congress 2024 Americas
STEERING INTRICATE
REGULATORY REQUIREMENTS
AND CHALLENGES IN
SUCCESSFULLY IMPLEMENTING
EARLY ACCESS TO MEDICINES
Boston, MA, USA
Day 2 - Friday 15th November 2024
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
Carlos Izquierdo, Global Pricing & Access Lead – Early Pipeline Gastrointestinal, Takeda
- 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
- 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
Dan Lewi , Head, Business Development and Patient Advocacy, Pulse infoframe Inc., Co- founder, CATS
Foundation
- 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
