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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


  • 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?
  • 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

  • Changes in regulations affecting the European market
  • How to find the right patient and design a collaborative clinical trial?
  • Developing strong economic models in demonstrating the value of subsidizing patient healthcare cost
  • Establishing a well-informed sales and marketing plan to ensure a successful market access
  • Engaging and empowering patients
  • Perspective of when, and how to engage patients, advocacy organizations and caregivers
  • Benefits of early and consistent engagement on recruitment, retention, regulatory and access issues.
  • Ways patients and patient representatives can be involved in fundamental and translational research into
    their disease.
  • Success factors toward building a good multi-stakeholder network
  • How to maximize the positive impact of patients and advocates on research.


  • 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


  • How are this advance therapy fundamentally changing the way rare diseases patients are treated?
  • Improving clinical development by new age clinical trial design and recruitment
  • Effective framework for developing the delivery system for the next generation of medicine.
  • Making manufacturing and commercialization viable
  • Gene therapies in the pipeline


Eric Crombez, Chief Medical Officer, Gene Therapy and Inborn Errors of Metabolism, Ultragenyx

  • 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


  • 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
  • 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

  • Recognising key areas to ensure patients’ experiences, perspectives, needs, and priorities are captured and
    meaningfully incorporated into drug development and evaluation.
  • Patient’s input to improve quality, relevance, safety and efficacy of drug development
  • The role of respective stakeholders and the way they interact, from the early steps of drug development to
    access in real life?

Adrien Lemoine, Co-Founder & CEO, Bloomsbury Genetic Therapies

  • 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


  • Identify how the rapidly changing COVID-19 environment impacted the lives of the Rare Disease community.
  • The effect of the pandemic on the ‘diagnostic odyssey’ in rare diseases
  • Facilitating the identification of new frameworks to structure day-to-day lives
  • Focus on mental well-being and coping with isolation, while consistent communication relating to health
    and social care throughout was essential.


David Pearce, President of Innovation, Research & World Clinic, Sanford Health

  • 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

  • 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?

Josie Godfrey, Co-Founder and 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

Panel Discussion: 

Lara Bloom, President and CEO, The Ehlers-Danlos Society


  • 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
  • How industry can engage the patient voice and move from patient centricity to productive patient

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

  • Payers are becoming more aggressive in scrutinizing price and implementing restrictions to patient access
    that have traditionally been reserved for competitive, high budget impact drug classes
  • Recent development in orphan drugs pricing and reimbursement and the impact of those changes
  • Addressing some of the major issues facing payers and orphan drug manufacturers, highlighting the way
    these have been handled by both parties. Implementing measures designed to limit exposure to costs
    associated with orphan drug reimbursement.
  • 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.
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