21st Orphan Drugs & Rare Diseases Global Congress 2024

Innovative programmes to optimize access in a global healthcare market

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Orphan Drugs & Rare Diseases Global Congress 2024 Europe

The Fastest Growing Orphan Drugs & Rare Diseases Networking Event. 

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

GAIN LATEST INSIGHTS ON:

WHO SHOULD ATTEND?

This congress is specially created for valued stakeholders in the Rare Disease community:

Network with Presidents, Heads/Chiefs, VPs, Directors, and Managers in the area of:

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Attendees

Key Industry Expert Speakers

David Pearce

President of Innovation, Research, & World Clinic

Sanford Health

Anne-Sophie Chalandon

Head of Global Rare Diseases and CGT Policy

Sanofi

Pedro Franco

Director for Global Regulatory & Scientific Policy (GRASP)

Merck

Amanda Bok

Chief Strategy Officer

The Synergist

Owen Marks

Principal, OMGEN, Head of Rare Diseases and Diversity and Inclusion Co-Chair

Pfizer

Manuela Maronati

Head of Rare Disease Europe

UCB

Rüdiger Schulze

VP & GM, Germany & Central/Eastern Europe

Ultragenyx

Nicholas Brooke

Founder & Executive Director

Synergist

Arti Patel

Genomics Associate, Great Ormond Street Hospital

Trustee, Treacher Collins

Alexander Natz

Secretary General

EUCOPE

Nigel Nicholls

General Manager UK/Nordics & Baltics

Egetis Therapeutics AB

Hannah Harvey

Senior Projects Officer

Beacon for Rare Diseases

Kerry Leeson-Beevers

Chief Executive

Alström Syndrome UK

Sheela Upadhyaya,

Chair Elect, Rare Disease Special Interest Group

ISPOR

Josie Godfrey

Co-Founder and CEO,

Realise Advocacy

Nick Sireau

Chair & CEO

AKU Society

Dr Usman Khan

Chair

Motor Neurone Disease (MND) Association

Bob Stevens

Group CEO

MPS Society

Melanie Dixon

Trustee

Cure DHDDS

Dr. Ron Jortner

Founder/CEO

Aspire Biosciences

Carole Scrafton

Director & Co-Founder,

Flutters and Strutters

Rohan Simon

Associate Director - Business Development

Insights Research Organization & Solutions

ANTHONY HALL

Chief Medical Officer

Healx

Lara Bloom,

President and CEO

The Ehlers-Danlos Society

Kristin Smedley

CEO

Curing Retinal Blindness Foundation

Hayley Chapman

Senior Program Director

The Synergist

Nick Sireau PhD

Chair and CEO

AKU Society

Robert Palmer

Head of Value & Access Oncology, APAC, MEA

Novartis

Schedule

Content Rich Program Agenda!  Featuring Keynote Presentations, Stream Sessions, Panel, and Round Table Discussions.

MACRO OUTLOOK AND CURRENT TRENDS

  • Scene setting on rare disease – definitions and unmet need
  • Progress in last decades – melting the iceberg of need?
  • Introducing the Health Equity agenda in life sciences
  • Evolution of Health Equity space – patient representation, expert patients, patient organisations, engagement platforms, differentiation, and articulation within the protected characteristic space
  • Health Equity and Rare Disease – aspects of a Ven diagram?
  • Thought/discussion points for conference.

Dr Usman Khan, Chair, Motor Neurone Disease (MND) Association

  • 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?

Panelists:

Melanie Dixon, Trustee, Cure DHDDS
Carole Scrafton, Director & Co-Founder, Flutters and Strutters

  • 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

  • Beyond Data and Strategic HTA Submission Tactics
  • Harnessing Community Insights for Better Launch Planning
  • Developing Sustainable Go-to-Market Approaches for Orphan Medicines
  • Building an Ecosystem of Collaboration to Drive Health-System Change

Owen Marks, Head of Rare Diseases and Diversity and Inclusion Co-Chair, Pfizer

  • Understanding the necessity of partnering with the patient community from the start
  • Elevating the patient voice – the UCB experience in gMG
  • The role of real-life experience in changing the treatment paradigm
  • Innovation approaches in evaluating new medicines for rare diseases
  • The importance of building rare capabilities within a company to support launch

Manuela Maronati, Head of Europe, Rare Disease Organization, UCB

  • Overview of UAE’s Unique Research Landscape Positioning
  • Deep dive into UAE’s rare disease prevalence
  • Significant opportunities for clinical research
  • Quality standards for conducting clinical research in the region

Rohan Simon, Associate Director – Business Development, IROS

  • 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 
  • The role that patient networks can play in improving diagnosis, clinical trial recruitment and retention.
  • The need to stop thinking of communities as being ‘hard to reach’ and appreciate that some services are ‘hard to access’.
  • Overcoming barriers and striving for equity.
  • Allowing ourselves to be vulnerable and reflecting on our own practices and organisational culture.
  • Developing inclusive and accessible services and support.
  • Including the voices of people who often go unheard – involving and engaging with people from diverse and marginalised communities.
  • Learning from people with lived experience.

Kerry Leeson-Beevers, Chief Executive, Alström Syndrome 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, & World Clinic Sanford Health

  • 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
  • Receiving a diagnosis – the start of our journey.
  • Reaching out to the scientific community and searching for patients.
  • Planning our strategy in how best to tackle this ultra rare disease.
  • Problems encountered along the way.
  • Our hopes for the future.

Melanie Dixon, Trustee, Cure DHDDS

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

Panelist:
Anthony Hall, Chief Medical Officer, Healx

Robert Mitchell-Thain, CEO, PBC Foundation

Sheela Upadhyaya – Life Sciences Consultant – specialising in Rare Disease

ACCESS & COLLABORATION

  • This session reports on the collective vision and efforts of global patient leaders towards empowering and enabling the patient movement to ‘level’ and ‘leverage’ up towards delivering its full potential for the benefit of patients, health systems, and society

Amanda Bok, Chief Strategy Officer, The Synergist

  • 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

Sheela Upadhyaya – Life Sciences Consultant – specialising in Rare Disease 

  • The difference between real world data and real world evidence
  • The utility of RWE within a drug development pathway
  • RWE frameworks across the world
  • Use of RWE in HTA

Sheela Upadhyaya – Life Sciences Consultant – specialising in Rare Disease

  • 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

  • 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

  • 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 

To Follow

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

  • Today’s Rare Disease Landscape
  • Challenges & Opportunities
  • The importance of patient networks and incorporating diverse patient voice in diagnosis and clinical trial recruitment
  • Patients before Profits – How to improve the clinical trial experience for patients and retention for sponsors
  • The Importance of Early Access
  • Understanding end points and emerging science
  • Collaboration, Collaboration, Collaboration !!!

Bob Stevens, Group CEO, MPS Society

  • 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.

Moderator:


Panelists:
Anthony Hall, Chief Medical Officer, Healx

Event sponsors

Orphan Drugs and Rare Diseases Global Congress 2023 / 2024 Europe

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Testimonials

what people say

Thank you for a good experience with the PARADIGM Global Events… very high level, very good Organization, we overpassed our expectatives, we will be pending for another congress related to pharmaceutical and biotech… we learned a lot, and i think that the content of agenda was state of the art, thank you again.
Salvador Berrios
ERP/ CRM / Product Configurator Manager
The consistently high quality of international faculty was most impressive. It was also obvious that they were carefully chosen not only for their particular expertise but also for their ability to communicate the subject matter to clinical and non-clinical participants alike. As market access consultant for pharma clients of various sizes, I now feel much better prepared to explain the nuances & dynamics involved in bringing their products to market. In addition, I am in a much better position to intelligently advice my clients & help them set realistic expectations
Thomas Martin
Associate Vice President, VCGA - Quintiles
I was impressed with the organization of this event. I attend many conferences per year and find the organizers on site to be detached and not focus on good service to the delegates. This team was even providing weather forecast warning to ensure everyone was aware and made appropriate plans. Well Done.
Adriana E. Manzi
PhD, Managing Director, Atheln, Inc

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