Day 1: ACCESS Programmes Congress

Conference Agenda

MUST ATTEND!

Expanded Access Programmes - Day 1

Day 1: Tuesday 29th November 2022

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

MACRO OUTLOOK & CURRENT TRENDS

  • Understanding the challenges in running a global EAP
  • The positive impact of EAP on patients with serious, immediately life-threatening, or rapidly debilitating
    diseases
  • Gain an understanding to increase access to treatment
  • The importance of cross collaborations between stakeholders

Moderator: 

Monica L Weldon, President/CEO/Founder, SYNGAP1 Foundation

Panelist:

Rachel Harrison, Associate Director, Early Access Program, Apellis Pharmaceuticals
John Lagus, Executive VP Business Development, Tanner Pharma Group
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross

  • 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

  • Understanding the importance of providing an early access program for acute diseases
  • Using an early access program to provide patients treatments between clinical trials and commercial
    approval
  • Considering the additional requirements when running an expanded access program for gene therapies

Magali Taiel, M.D., Chief Medical Officer, GenSight-Biologics

  • Where Machine Learning works best
  • How non-technical pharmaceutical executives can critically assess new applications of the technology
  • Case study

Jeffrey Thibeault, Co-founder & Chief Technology Officer, MedaSystems

  • Overview of how named patient supply aligns with a company’s broader market access strategy
  • When a withdrawal of a marketing authorization might make sense
  • Use of named patient supply to allow continued patient access when market withdrawals occur
  • Potential implications of international reference pricing and how named patient supply can support
  • Case studies

John Lagus, Executive Vice President, Business Development, Tanner Pharma Group

CHALLENGES & POTENTIALS

  • What are the requirements from each stakeholder?
  • Best practice in developing this into a coherent program across numerous jurisdictions
  • The ethical and commercial connectivity and implications
  • How can you provide long term access and where is this suitable.

Robert Donnell, EVP, Medicines Access, Smartway Pharma

  • Patients often take on the role of research partner or Citizen Scientist. The benefits of such a partnership include
    increased public access and involvement of patients in research, immersion of patient community values relevant
    to research, outreach and educational potential with the community, and ultimately, the democratization of
    science

Dr Neena Nizar, Founder & Executive Director, The Jansen’s Foundation

  • 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 working
    relationship
  • 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

  • TBC

Nina Liu, Director of Clinical Product Management, Pulse Infoframe

  • 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.

Moderator:
Dr Neena Nizar, Founder & Executive Director, The Jansen’s Foundation

Panelist:
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross
Monica L Weldon, President/CEO/Founder, SYNGAP1 Foundation
Robert Donnell, EVP, Medicines Access, Smartway Pharma

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