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21st Orphan Drugs and Rare Diseases Global Congress 2024 Europe

Collaborations to Accelerate Rare Disease Drug Development and Access

Hilton London Kensington, United Kingdom

11th - 12th April 2024

Facilitate Live is proud to present our Orphan Drugs and Rare Diseases Global Congress 2024 Europe. It’s the 21st 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
Facilitate Live


  • The total burden of rare disease, which affects not only patients, but their caregivers, family members, and society as a whole.
  • Addressing major logistical and regulatory issues due to minimal clinical research centers and sufficient expertise.
  • Special consideration and flexibility to ensure that vulnerable patients with rare diseases have a sustained pathway to access
  • What challenges still remain to increased equity for people living with a rare disease and their families?
  • 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
  • The European Parliament is currently reviewing the Pharmaceutical Package, which will potentially be voted on in April – this new text will outline the incentive and regulatory ecosystem for OMP developers. This will be a major milestone in the legislative review.
  • The outcomes of these discussions will shape the landscape for OMP developers in the EU for the next 20 years.
  • Reflect on the changes taking place outside the scope of the legislative framework, including the role of PPP and ERNs

Alexander Natz, Secretary General, EUCOPE

  • Implications of the new EU Pharma package
  • Joint EU HTA and impact on local HTA procedures
  • Trends in Orphan Drug pricing
  • New regulations in selected EU countries

Rüdiger Schulze, VP & GM, Germany & Central/Eastern Europe, Ultragenyx

  • Establishing Trust and partnership
  • Using 3rd party agencies as a networking, capacity and bridge building partners
  • Building joint successful advocacy campaigns
  • Exploring ethical issues and barriers in joint working
  • What does best practice look like now and in the future.

Nigel Nicholls, General Manager UK/Nordics & Baltics, Egetis Therapeutics AB

  • 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


  • Every data point is precious
  • Optimal relationships with hospital sites are essential
  • Patient centricity must inform all stages of the development process
  • Clinical trial issues most important to individuals living with rare disease
  • Initiating clinical trials that better meet the needs of affected individuals and their families.
  • Building community networks to reach underserved and underrepresented communities
  • The importance of trust building in improving diagnosis and clinical trial participation

Kerry Leeson-Beevers, Chief Executive, Alström Syndrome UK

  • Challenges of Development: The Regulatory and Developmental Environment
  • Prospective Strategies to Overcome the Challenges
  • Role of Big Pharma: Recent Trends
  • Progress that has been made in the development of orphan drugs by pharma industries


  • Clinical breakthroughs
  • Review of the therapeutic means of rare diseases and drug development of rare diseases
  • Progress of treatment of rare diseases in order to provide a reference for clinical use and new drug development of rare diseases
  • 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

The AKU Society successfully led the 10 year development programme of the drug nitisinone for the ultra rare genetic disease Alkaptonuria (AKU, or Black Bone Disease). But we are now learning that the disease is even more serious than originally thought and that limiting the drug’s side effects requires a strict low protein diet.
That’s why the AKU Society has launched its own drug discovery and development programme that it is funding at the University of Liverpool in order to develop new drugs that will resolve these issues. Find out at our talk what challenges face patient groups carrying out their own drug discovery programme and how to overcome these.

Nick Sireau, PhD, Chair and CEO, AKU Society

  • 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 Mitchell-Thain, CEO, PBC Foundation


  • 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?
  • How are public private partnerships advancing?
  • How could they add value? the power of PPP
  • How the futures PPP looks like? What is really exciting?
  • Conclusion, look to the future

Anne-Sophie Chalandon, Head of Global Public Affairs, Rare Diseases & CGT Policy, Specialty Care, Sanofi

  • Why support groups are important & helpful for patients and their families.
  • Examples of accomplishments of patients’ groups, e.g., TCFSG IVG PGD, Unique – Information Guides, GA UK – SWAN.
  • The future of patient support groups
  • Working relationships between patient support groups and pharma, healthcare professionals.

Arti Patel, Genomics Associate, Great Ormond Street Hospital, Trustee, Treacher Collins Family Support and
Gene People UK

  • How to collaborate with academic, industry, and patient advocate stakeholders
  • Hot topics in rare diseases
  • Future of therapeutic development for rare diseases

David Pearce, President, Innovation & Research, Sanford Health

  • There are a number of resources and tools that exist at EU and national level that can improve access to OMPs without requiring new legislation, but rather require better implementation or expansion of existing tools, such as the Transparency Directive, early access discussions like MoCA, the Cross-border healthcare ecosystem, and ensuring an appropriate EU HTA methodology.

Alexander Natz, Secretary General, EUCOPE

  • Contextual considerations for value evidence in Rare Diseases
  • HTA and patient access for orphan drugs – state of affairs
  • How can we build an ecosystem for rare diseases?

To Follow

Fleur Chandler, Head of Market Access UK and Ireland, Sanofi

  • Patients as Key Partners in Drug Development
  • Role of Real-World Evidence in Rare Disease
  • Value of Registries in Rare Disease – pros and cons
  • Rare disease programs have historically leveraged creative methods to deal with difficulties inherent in orphan disorders
  • The industry is evolving to address new roles in sustainability, pricing legislation, and multi-stakeholder management in addition to the patient
  • Partnerships can make rare disease programs more successful in meeting and surpassing new industry standards
  • Successful example of value-based pricing in Rare Disease
  • Potential changes in treatment paradigms to improve effectiveness and reduce cost of treatment
  • Legislative incentives to encourage development of orphan rare diseases therapies
  • Impact of orphan diseases treatment on healthcare payment system


  • In rare disease, patients look ‘upstream’ with hope for development of new treatments and access to trials and ’downstream’ for better, faster diagnoses and access to treatments. No other disease category faces the same sorts of challenges all along the continuum. Recent market conditions have halted development of a number of promising potential therapies, and new policies, coverage decisions, financial pressures and other factors have generated additional gaps, delays and difficulties in access to newly approved medicines.
  • New approaches that are emerging from discovery to treatment in response to these challenges.
  • 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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