A virtual event from BresMed and Cello Health

Rare Diseases: Access All Areas

Opportunities and Implications for Rare Disease Patients

Tuesday, 9th November 2021

3-hour virtual event 0800 (EST) / 1300 (GMT) / 1400 (CET)

  • Materials are available for professionals in the biotech and pharma industries
  • Register below to receive post-event content
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Pharma Progress pattern

“It’s all about collaboration. By itself, a rare disease patient group can’t really accomplish that much, but when you work with scientists, industry, other patient groups, clinicians and regulators, that’s when we can make a really significant difference.”

Nick Sireau

CEO and Chair of Trustees, AKU Society

Event overview

Starting with the patients' perspective, we explored the challenges of living with a rare disease, and highlighted new research and synergies across rare disease communities, as well as the implications for pharma. We also examined the appraisal landscape for rare diseases, and looked at best practices for value assessment and patient engagement. Finally, we delved into patient advocacy's success in raising awareness of unmet needs and unique ability to source potential early-stage therapeutic solutions.

Attendees heard about

  • 'Patient challenge' new research, synergies and unmet need across the community of rare disease patients; implications for pharma
  • How rare diseases are appraised internationally and the implications and opportunities for patients
  • Developing breakthrough therapies in partnership with the patient and preparing for the transition to commercialization


Welcome and Introduction

Jon Williams, Group CEO

Understanding patients with rare diseases

PART 1: A personal perspective on rare diseases

Nick Sireau

CEO and Chair of Trustees, AKU Society

"I am very pleased and excited to be part of this event and to showcase the struggles of rare disease patients and their supporters in the quest to bring treatments to their communities. I look forward to exploring solutions together to bring innovation and urgency to the need for better market access, policy, and stakeholder engagement. By definition, rare disease means fewer patients, but our unmet needs are significant and our vision is to help to bring treatments to rare disease patients that are also sustainable for health services."

Damien Eade

PART 2: New Social Media Listening (SML) insights: Patients' perspectives in rare diseases

Damian Eade

Managing Director, Cello Health Logic

Isaac Batley

Chairman, Cello Health Communications

We explored the challenges of living with a rare disease and highlighted synergies across rare disease communities, as well as the implications for pharma.

Rare disease appraisal landscape and bringing novel technologies to market

Matthias Bischof

Senior Director, Global Health Economics and Outcomes Research, Novartis Gene Therapies

Peter Jackson

PhD FRCP, Chair, NICE HST Committee

Jane Moorhouse

Principal Insight Analyst, BresMed

Moderator: James Horscroft

Principal Insight Analyst, BresMed

Matthias Bischof
Peter Jackson

Partnering with the Patient to Innovate and Prepare for the Transition to Commercialization

Sharon Hesterlee

PhD, Executive Vice President & Chief Research Officer,
Muscular Dystrophy Association

Kristin Phillips,

MPH, Senior Director, Community Engagement,
Taysha Gene Therapies

Nick Sireau

CEO and Chair of Trustees,
AKU Society

Moderator: Michael Rice,

VP, Head of Advanced Therapeutics
Cello Health BioConsulting

Sharon HesterLee
Kristin Phillips
Damien Eade


Jane Ayton

Senior Vice President,
Cello Health Consulting

Phara Progress pattern Phara Progress pattern
Jon Williams, CEO

"BresMed and Cello Health are excited to host the second Pharma Progress conference, one of a series of virtually enabled, short and focused events. The conference will focus on the implications of rare diseases for patients and pharma and how we as an industry can work with those affected to support the development of treatments and care pathways which meet the needs of those living with rare diseases."

Jon Williams, Group CEO

Pharma Progress pattern

Download Event Summary

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