19th Orphan Drugs and Rare Diseases Global Congress 2023 Europe
Collaborations to Accelerate Rare Disease Drug Development and Access
Hilton London Kensington, United Kingdom
3rd - 4th April 2023
Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases Global Congress 2023 Europe. It’s the 19th in the series of our Flagship tri-annual Orphan Drugs & Rare Diseases event. This congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs.
The COVID-19 Pandemic has impacted the lives of just about everyone, patients receiving continuous treatment and care included. The Rare Disease community is already experiencing considerable challenges in achieving early and detailed diagnosis and having access to care and life-saving treatment, to chronic, highly complex, progressive, and severely disabling nature of their diseases, which generate specific care needs. Undoubtedly, during this crisis, the rare diseases community is experiencing unprecedented overall disruption even more…
According to a report by Fortune Business Insights, the Global Orphan Drugs Market size will emerge from the COVID-19 crisis at a moderate growth during the forecast period of 2020 to 2026. The global orphan drugs market size is projected to reach USD 340.84 billion by the end of 2027. As stated, the market was worth USD 151.00 billion in 2019 and will exhibit a CAGR of 10.5% during the forecast period, 2020-2027.
The current regulatory climate increasing the number of regulatory approvals, expanding numbers of successful clinical trials related to these drugs, tremendous investments in R & D, and the interest and existence of several large-scale manufacturers, will create opportunities and contribute exponentially to the overall market growth.
The two-day Congress will provide an interactive, cutting edge, and comprehensive discussion and networking format led by key experts with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies, and concepts surrounding orphan drugs and rare diseases.
We look forward to meeting you at the Congress!
Sincerely yours,
Jocelyn Raguindin
Conference Director
Paradigm Global Events
Robert Mitchell-Thain, CEO, PBC Foundation
CURRENT LANDSCAPE AND EAP
- Understanding the challenges in planning and implementing a global EAP
- Country specific framework and requirements to access investigational drugs
- Scientific elements and regulatory tools available to address the challenges and explore the flexibility within the current regulatory systems
- Significant degree of administrative effort required to initiate the programme for a timely access
- How far are we from establishing harmonised guidelines across Europe?
Nicola Gillott, Head of Expanded Access, Amryt Pharma
Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck
Graham Sidorowicz, EVP Commercial, Bionical Emas
- Setbacks happen to every organization. How do some leaders turn them into big success while others stay stuck?
- The key questions to ask to move from frustration to innovation and transform your organization’s journey.
- The world is open to new ways of thinking – how will we capitalize on this to accelerate rare disease research and advocacy??
Kristin Smedley, CEO, Curing Retinal Blindness Foundation
Regulatory Challenges on Early Access Programmes
Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck
- Understanding the disease area and impact on the patient and family
- When to engage with patient advocacy organisations and the value they can bring
- Best practices to ensure patients are partners in your clinical development process
Graham Sidorowicz, EVP Commercial, Bionical Emas
Naomi Litchfield, Director, Patient Advocacy, Bionical Emas
- Learn more about the nuances of Real-world studies
- Learn about principles for designing RWE studies: protocol, IC, Ethics
- Key considerations for using digital technology
- Managing RW studies for successful outcomes
- Case studies
Florence Barkats, Chief Commercial Officer, CISIV Ltd
- Before considering Early Access Patient Organisations & Patients need to be engaged much earlier in the
process - Think Natural History
- Think Reimbursement not Approval
- Understand what it is that you want to demonstrate and how (Biomarkers – science fiction not real world)
- Don’t just think data – Think about telling a story
Bob Stevens, Group CEO, MPS Society/Rare Disease Research Partners
- The market access perspective – when and how expanded access is helpful
- Expanded access in the context of the overall access strategy
- Going beyond the large countries to a global strategy
Robert Palmer, Head of Value & Access Oncology, APAC, MEA, Novartis
- Overcoming challenges to participant recruitment to rare disease research
- How patient recruitment and recall into the Rare Diseases BioResource take place
- Working with academia, industry and patient groups to facilitate rare disease research
- Case studies from previous 15 years of rare disease research
Hannah Stark, Operations Lead, NIHR BioResource
- Oligonucleotides allowing the targeting of a specific gene mRNA represents a promising and increasing class of recently approved genetic drugs.
- The thee main strategies will be detailed: the antisense approach uses simple strand olidodeoxynucleotides and aims at either exon skipping or mRNA degradation; and the interfering RNA silencing strategie.
- The different chemistries used for each strategy will be illustrated, as well as the pros and cons
- Recent examples of therapeutic success and applications in clinical development phase will be displayed.
Pr Daniel Scherman, Head of Medicine, Life Sciences Division, European Academy of Sciences EURASC
- 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 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, CSO & General Manager, Gain Therapeutics
- 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
Hannah Stark, Operations Lead, NIHR BioResource
Anthony Hall, Chief Medical Officer, Healx Manolo Bellotto, CSO & General Manager, Gain Therapeutics Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck
Robert Mitchell-Thain, CEO, PBC Foundation
Sponsored by:
Josie Godfrey, Director, JG Zebra Consulting, Joint CEO, Realise Advocacy
CHALLENGES AND OPPORTUNITIES
- The National Disease Registration Service (NDRS, NHS England) collects patient data on cancer, congenital anomalies and rare diseases, and provides expert, timely analysis to support clinical teams, academics, charities and policy makers to help plan and improve treatments and healthcare in England.
- The talk will illustrate how collaboration with NDRS can provide whole-population actionable insights into rare disease occurrence, treatment and outcomes.
- This can inform health policy and deliver pioneering rare disease research
Peter Lanyon, Rare Diseases Clinical Lead, National Disease Registration Service, NHS England
- International Collaboration and partnerships
- Access to diagnostics
- Access to treatments
- Diverse populations
- Future directions of RD research
David Pearce, President of Innovation, Research & World Clinic, Sanford Health
- The key challenges for Rare Disease drug development and access
what is the EU HTA - Description of EU HTA principles and timeline
- A SWOT analysis of the situation
- What has been done so far and what next
Sheela Upadhyaya, Chair Elect, Rare Disease Special Interest Group, ISPOR
- Challenges for commercialization in small markets
- Changes in the market access and regulatory environment
- How to priorotize markets – and how not to
- Success stories
Rüdiger Schulze, VP & GM, Germany & Central/Eastern Europe, Ultragenyx
- The impact rare disease patient organisations can have on access decisions
- Challenging conventional approaches to patient engagement in access
- Planning for success
Josie Godfrey, Director, JG Zebra Consulting, Joint CEO, Realise Advocacy
- Developing trend and obstacles faced by rare disease clinical trials
- The foundational value of building collaboration through trust, respect, transparency, and empathy
- Not all advocacy organizations are alike: assessing the landscape
- Real-world examples of innovative digital solutions driving collaboration among multiple stakeholders
- Value of partnering with advocacy organizations to create innovative patient-driven solutions
Moderator:
Florence Barkats, Patient Advocate, NHCollab
Panelists:
Lara Bloom, President and CEO, The Ehlers-Danlos Society Anthony Hall, Chief Medical Officer, Healx Bob Stevens, Group CEO, MPS Society/Rare Disease Research Partners
ACCESS AND COLLABORATION
- The value of expert patients and why they should be paid
- Case studies demonstrating the benefits patient groups can bring, and issues that arise when they are not
consulted - How industry can engage the patient voice and move from patient centricity to productive patient
partnerships
Hannah Harvey, Senior Projects Officer, Beacon for Rare Diseases
- Good partnership
- Patient involvement
- Using the App to collect patient data, where the data has been used, how it has helped to inform decisions
- Examples of abstracts, and their outcomes
Robert Mitchell-Thain, CEO, PBC Foundation
- Strategy
- Team
- Indications
- Scouting
- Funding
- Walk the talk
Christian Girard, Co-founder, OrphaPedia
- The origins of nitisinone as a herbicide
- AKU as the iconic rare genetic disease
- How a patient group, academics and industry worked together to develop nitisinone for AKU
Nick Sireau, Chair & CEO, AKU Society
- 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: Josie Godfrey, Director, JG Zebra Consulting, Joint CEO, Realise Advocacy
Panelists: Anthony Hall, Chief Medical Officer, HealxNick Sireau, Chair & CEO, AKU Society Robert Mitchell-Thain, CEO, PBC Foundation
Josie Godfrey, Director, JG Zebra Consulting, Joint CEO, Realise Advocacy