Agenda at a Glance
Orphan Drugs & Rare Diseases - Conference Day 1
Day 1: Monday - 3rd April 2023
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