Agenda at a Glance

Orphan Drugs & Rare Diseases - Conference Day 1

Day 1: Thursday - 17th February 2022

Dr Femida Gwadry-Sridhar, Founder & CEO, Pulse Infoframe

  • Impact of new innovations to patients and caregivers
  • What difference do these recent innovations have made to their lives?
  • What must be done to accelerate innovations and broaden access at the same time
  • What challenges still remain to increased equity for people living with a rare disease and their
    families?

Moderator:

Josie Godfrey, Co-Founder and CEO, Realise Advocacy

Panellist:

  • David Edward Rose, Ultrarare Patient Speaker, BD Manager, RARE Revolution Magazine
  • Angela Columbano, Head Business Development & Partnership, Genethon
  • Rhian Kiely, RWE Commercial Director, Europe, Open Health Evidence & Access
  • What dynamic guideline recommendations will be put in the hands of the healthcare professionals
  • Collaborations and partnerships with life science industries
  • Will rare disease patients have quicker access to new treatments and innovations?

Sheela Upadhyaya, Associate Director – Highly Specialised Technologies, NICE

  • Changes in regulations affecting the European market
  • R&D strategies to be implemented to accelerate market access for orphan drugs
  • Different factors responsible for driving and restraining the growth of the market.
  • Potential growth opportunities in the global and regional markets.
  • Understand the ever-changing reimbursement environment
  • How can patients, governments, and orphan drug companies help support each other’s initiative to provide timely access to patients

Moderator:
Dr Femida Gwadry-Sridhar, Founder & CEO, Pulse Infoframe


Panellist:

Keith White, Head, Global Market Access, Amylyx Pharmaceuticals
Paul Beresford, Independent Consultant, former SVP, General Manager, International
Elisabetta Zanon, Director, EU Public Affairs & Advocacy, Alliance for Regenerative Medicine
Alexander Natz, Secretary-General, EUCOPE

While European regulatory agencies are adopting new pathways to embrace innovative therapies for
orphan diseases, it’s becoming increasingly clear that the reimbursement process and access pathways
haven’t caught up

  • Overview of challenges of recent orphan drugs from approval to market
  • Lessons learned
  • Considerations for biopharma companies looking to expand into this highly complex and nuanced market

Doug Danison, Head of Europe, bluebird bio*

  • Overview of current methods for their prevention, diagnosis, and treatment.
  • Strengths and limitation of current development pathways for new drugs, medical devices, and
    biologics for rare diseases
  • Special challenges that rare diseases create for research and product regulation
  • Current public policies relevant to product development for rare diseases

Robert Donnell, Executive Vice President, Medicines Access, Smartway Pharma

  • How is this advanced therapy fundamentally changing the way rare diseases patients are treated
  • Improving clinical development by new-age clinical trial design and recruitment
  • An effective framework for developing the delivery system for the next generation of medicine
  • Making manufacturing and commercialisation viable
  • Unconventional payment models to ensure innovative treatment can be accessed by rare disease patients

Moderator:
Elisabetta Zanon, Director, EU Public Affairs & Advocacy, Alliance for Regenerative Medicine

Panellist:

  • Alexander Natz, Secretary-General, EUCOPE
  • Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech. Evaluation, Independence Blue Cross
  • Zizi Imatorbhebhe, MBA, MS, PMP®,Senior Vice President, Global Strategy and Development, Rare
    Disease, Head Rare Disease Innovation Center, Ergomed

2:15 - 6:00 pm - Choose between 2 topic streams

ACCESS & COLLABORATION

Josie Godfrey, Co-Founder and CEO, Realise Advocacy

  • Introducing the Rare Disease Nurse Network
    (charity application in process), aiming to
    “become the Macmillan of Rare Disease”.
  • Overview of RDNN, aims, and objectives
  • Outline of our planned nurse service pilot
  • How people can help get involved

Helena Baker RGN, CEO, Rare Disease Nurse Network

  • Launch success is becoming increasingly
    challenging in a complex environment
  • Meeting a clinical trial endpoint and gaining a
    product licence is not always a guarantee of
    success
  • Early engagement to understand evidence
    needs reduces value uncertainty and facilitates
    a successful launch

Rhian Kiely, RWE Commercial Director, Europe, Open Health Evidence & Access

 

  • The dos and don’ts of negotiations
  • How to meet both payer and company expectations
  • The role of real-world evidence

Alexander Natz, Secretary-General, EUCOPE

  • Problem: a case study of a novel successful crowdfunding approach, called BeHEARD
  • Solution: the program allows rare disease researchers to apply for support in the form of
  • financial funding and cutting-edge experimental research tools and services.
  • Impact: As a result, the BeHEARD team has raised over USD $2 million, which will help accelerate cures for those with rare disorders

Arvin M. Gouw, PhD, VP of Research & Development, Rare Genomics Institute

  • Why does patient involvement matter in HTA and access?
  • What opportunities are there for patient engagement
  • When is the right time for patient organisations to get involved in HTA and access?
  • How can patient organisations and patients be supported to ensure they can maximise their impact?
  • How can the industry work with patent organisations?

Lindsay Birrell, Co-Founder & CEO, Realise Advocacy, Trustee, Medics 4 Rare Diseases

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

DISCOVERY & INNOVATION

Dr Femida Gwadry-Sridhar, Founder & CEO, Pulse Infoframe

  • Building a consortium
  • Preparing the clinical development plan Interacting with industry
  • Working with the regulators
  • Engaging with patients
  • Rolling out a successful set of clinical trials.

Nick Sireau, PhD, Chair and CEO, AKU Society

  • Update on cystic fibrosis gene therapy
  • Development of the NIHR ATMP Coordinating Group
  • Lessons learnt over three decades in the gene therapy field

Eric Alton FRCP, FMedSci, Professor of Gene Therapy & Respiratory Medicine, Imperial College

  • Patient impact through treatment development
  • Case studies of patient group involvement
  • Engaging the patient voice as an industry
  • The role of Findacure

Philippa Norman, Projects Coordinator, Findacure

  • Introduction to Genethon pipeline in rare diseases
  • Update on clinical programs
  • Next-generation gene therapy: focused on enhanced efficacy and improved manufacturing processes

Angela Columbano, Head Business Development & Partnership, Genethon

  • Choosing the correct design for your trail
  • Ensuring the right tools and infrastructure for sites and patients
  • Case study examples with learning points to take away

Juliet Hulse, Senior Director, Global RN Strategy and Patient Advocacy, Illingworth Research

  • Taking the example of lysosomal enzyme related diseases, I will present evidences on how a genetic, molecular and pathobiological understanding of rare and ultrarare diseases
    discovery and product development
  • I will also bring evidences of how an organelles centric discovery can identify and lead to the development of novel approaches for genetic defined neurodegenerative diseases and why patient-centric associations and foundations (such as the Michael J. Fox Foundation in GBA1 Parkinson) support those research avenues

Manolo Bellotto PhD, President & General Manager, Gain Therapeutics

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