Day 2: ACCESS Programmes Congress

Conference Agenda

MUST ATTEND!

Expanded Access Programmes - Day 2

Day 2: Wednesday 30th November 2022

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

ACCESS & COMMERCIALIZATION

  • 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 healthcare stakeholders work together

Moderator:
Robert Donnell, EVP, Medicines Access, Smartway Pharma

Panelists:
Jennifer E. Miller, PhD, Associate Professor , Yale School of Medicine
Dr Neena Nizar, Founder & Executive Director, The Jansen’s Foundation
Monica L Weldon, President/CEO/Founder, SYNGAP1 Foundation

  • 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/Founder, SYNGAP1 Foundation

  • What the institutional process at an academic medical center looks like, with examples
  • Available TEAMSS resources for physicians and academic medical centers
  • How to demonstrate value of Expanded Access support

Misty Gravelin, Project Lead, Expanded Access Support Unit, University of Michigan

  • 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

  • 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 medicine
  • Creating a state of the art payer engagement strategies to address challenges in pricing and reimbursement

Moderator:

Robert Donnell, EVP, Medicines Access, Smartway Pharma

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

PLEASE PROCEED TO THE CO-LOCATED MEETING ON ORPHAN DRUGS & RARE DISEASES.

  • A case study from a pioneering company that blazed a trail with a first-of-its-kind tissue-based regenerative
    medicine launch
  • Challenges and opportunities to consider when launching a commercial product for the rare disease
    community
  • Best practices from R&D to manufacturing to commercialization

Johanna Rossell, Chief Commerical Officer, Enzyvant

  • Contextual considerations for value evidence in Rare Diseases
  • HTA and patient access for orphan drugs – state of affairs
  • How can we build an ecosystem for rare diseases

Simu Thomas PhD, Vice President & Global Head HEOR, Alexion, AstraZeneca Rare Disease

  • Successful example of value-based pricing in Rare Disease
  • Potential changes in treatment paradigms to improve effectiveness and reduce cost of treatment
  • Legislative incentives to encourage development of orphan rare diseases therapies
  • Impact of orphan diseases treatment on healthcare payment system

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

  • Breaking down barriers for access
  • Strengthening health systems around the world and accelerating pathways to diagnosis
  • Stakeholders to address the rare diseases’ ecosystem gaps to collaboratively build a sustainable roadmap
    for better health and a brighter future for the patients suffering from rare diseases.
  • Challenges and opportunities in creating sustainable healthcare for all.

Moderator:

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

Panelists:

Tara J. Britt, Founder & President, Rare Disease Innovations Institute, Inc.
Frank Rivera, Co-Founder/President, Stronger Than Sarcoidosis
Kathleen Coolidge, Patient Advocacy, Pulse Infoframe