Orphan Drugs & Rare Diseases Global Congress

23rd Orphan Drugs & Rare Diseases Global Congress 2025

Innovative programmes to optimize access in a global healthcare market

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Orphan Drugs & Rare Diseases Global Congress 2025 Europe

The Fastest Growing Orphan Drugs & Rare Diseases Networking Event.

Introduction
Insight
Who Should Attend

Facilitate Live is proud to present our Orphan Drugs and Rare Diseases Global Congress 2025 Europe. It’s the 23rd 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:

Current Orphan Drugs landscape
Major drivers in the global rare disease market
Key developmental strategies carried out and Influencing factors that may affect market share to stand out in this industry
How is FDA managing orphan drugs during the pandemic?
Finding innovative and alternative ways in funding the development of Orphan Drugs
Patients perspective: what really matters to rare disease patients and caregivers?
Key authorities facilitating development and approval of diagnostic products/services
Coming Together in Developing Orphan Drugs and Crossing Borders
Trends and evolution of Advanced Therapy Medicinal Products
How Can the Developer and the Patient Assist in the Evolution and Development of Orphan Drugs to make it accessible to patients in a shorter period of time?
Strategies to improve Access and Affordability
What Do Developers Look for When Looking for an Outsourcing Partner?
What Do Insurance Companies Think About Orphan Drugs? Will They Make Modifications to Their Policies to Support Patients with Rare Diseases?
And much much more...

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:

Clinical Research Organizations
Clinical Operations
Medical Affairs, Global Patient Advocacy & Alliances
Research and Development
Rare and Ultra-Rare Diseases
Cell and Gene Therapy
Regenerative Medicine
Government Policy and Advocacy
Digital Patient Experience Lead
Clinical Insights and Experience
Head of Strategy, Access Services
Vice President, Site Collaborations and Patient Centricity
Head of Neuroscience
Personalised Medicine
External R&D Innovation
Innovative Medicine
Translational Science
Molecular Geneticist
Program Management
Patient Advocacy Groups
Public Affairs
Medical Affairs
Regulatory Affairs
Market Access
Pricing and Reimbursement
Health Economics Outcomes Research
Commercial Development
Investments and Funding
Product Specialist
Global Strategic Services
Business Planning & Operations

Head of Patient Engagement
Patient Services
Engagement Strategy
R&D Patient Engagement
Medical Affairs
Regulatory Affairs and Policy
Patient Support and Patient Engagement
RWE, and Data Management
Quality and Compliance
Clinical Development
Programme Management
Supply Chain Management
Patient Access
Clinical Research
Global Head, Digital Accelerator
Patient Engagement & Portfolio Strategy
Patient Support Strategy & Insights
Patient Experience
Clinical Research Organizations
Research and Development
Personalised Medicine
Pharmacies
Academia
And much much more..

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Key Industry Expert Speakers

2024 Speakers - 2025 Will Available Shortly

Comfirmed speakers Apply to speak

Schedule

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

Day 1 19/03/2025
Day 2 20/03/2025

Auditorium 1

8:00 am - Conference Registration and Welcome Coffee

8:30 am - Chairpersons Welcome and Opening Remarks

Dr. Femida Gwadry-Sridhar, Founder and CEO Pulse infoframe Inc.

MACRO OUTLOOK AND CURRENT TRENDS

8:40 am - KEYNOTE OPENING: Health Equity and Rare Disease: A New Frontier?

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

9:10 am - PATIENT OPENING KEYNOTE PANEL: Rare Diseases and Orphan Drugs: With focus on the patient, their family, and their quality of life

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?

Moderator:

Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse infoframe Inc.

Panelists:

David Pearce, President, Innovation & Research, Sanford Health

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

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

Dan Lewi, Head of Business Development and Patient Advocacy , Pulse infoframe Inc.

10:00 am - INDUSTRY KEYNOTE: Accelerating Orphan Medicine Commercialisation - Fostering Collaborative Speed-to-Patient Strategies

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

10:30 am - Morning Networking & Refreshments

11:10 am - PLENARY SESSION: An industry perspective – reflections on the review of the Pharmaceutical Package and implications for the Rare Disease Ecosystem

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

11:40 am - PLENARY SESSION: Prioritizing rare disease patient perspectives in drug development

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

12:10 pm - SPONSORED SPOTLIGHT: Evidence Generation using Real World Data

This presentation will focus on the role of RWD in natural history and patient registries.
How data can be utilized to help inform study design
The use of RWD as an external control and to find eligible patients for trials
Using RWD with prior clinical trial data to better understand patient outcomes.

Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse infoframe Inc.

1:10 pm - Networking Lunch Break

RESEARCH & DEVELOPMENT

2:10 pm - PLENARY SESSION: The importance of patient networks and incorporating diverse patient voice in diagnosis and clinical trial recruitment

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

2:30 pm - PLENARY SESSION: International Collaboration in Research and Development for Rare Diseases

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

3:00 pm - PLENARY SESSION: Ensuring Reliability in Quantifying Patient Populations for Orphan Drugs: Challenges and Solutions

Determining accurate patient population estimates for rare diseases is critical at all stages of drug development, from R&D through to securing orphan drug status and commercialisation.
Standard methods pose significant limitations due to the paucity of data, which presents risks to decision-making processes.
The availability of genetic databases has enabled a new “bottom-up” approach to establishing accurate data on patient populations, and mitigating such risks.
This presentation will discuss these new methods and how they are being applied.

Joshua Card-Gowers, Senior Evidence Lead, HealthLumen

3:30 pm - Afternoon Networking Break

4:10 pm - PLENARY SESSION: Our battle against a nano-rare, newly discovered and neurodegenerative DHDDS gene mutation

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

4:40 pm - PLENARY SESSION: The challenges of drug development by a patient group

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

5:10 pm - PANEL DISCUSSION: Strategies to promote research discoveries and development of orphan products to improve the health of people with rare disease

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

Moderator:

David Pearce, President, Innovation & Research, Sanford Health

Panelist:
Anthony Hall, Chief Medical Officer, Healx

Nick Sireau, PhD, Chair and CEO, AKU Society

Dr. Harriet Holme, Chair and Founder, PCDResearch.org

Surabhi Verma, Chief Medical Officer, Leadiant Biosciences

5:50 pm - Chairperson's Closing Remarks

Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse infoframe Inc.

Auditorium 1

8:00 am - Conference Registration and Welcome Coffee

9:30 am - Chairpersons Welcome and Opening Remarks

Sheela Upadhyaya – Life Sciences Consultant – specialising in Rare Disease

ACCESS & COLLABORATION

8:40 am - JOINT PRESENTATION: Public Private PAGs Payors Partnership – how to improve the European ecosystem (moonshot and together4RD initiatives) – role of data record and way of working

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

9:20 am - PLENARY SESSION: The Evolving Role of RWE in Health Technology Assessment

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

10:10 am - PLENARY SESSION: Collective efforts to catalyze the patient movement towards inclusion in health decision-making at all levels

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

10:40 am - Morning Networking & Refreshments

11:20 am - PLENARY SESSION: Promises and Pi�alls of using Real World Data and Evidence (RWD/E) in Orphan Drug Development.

Role of RWD/E in orphan disease drug development
Pitfalls in the use of RWD/E in orphan drug development
Repurposing and RWD/E

Surabhi Verma, Chief Medical Officer, Leadiant Biosciences

11:50 am - PLENARY SESSION: Critical role of patient organisations in the development of new therapies

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

12:20 pm - PLENARY SESSION: Systematic Serendipity - A Novel Approach for Finding New Treatments for Unaddressed Rare Diseases

Introduction of a novel methodology to tilt the cost-benefit function of rare disease treatment development
Leveraging both human and machine intelligence to identify treatment opportunities and build collaborative teams around relevant assets
Case study and preliminary outcomes from our Rare Neurology cohorts will be presented

Dr. Ron Jortner, Founder/CEO, Aspire Biosciences

12:50 pm - Networking Lunch Break

2:00 pm - PLENARY SESSION: The changing landscape In The Rare Diseases Eco System

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

2:30 pm - PLENARY SESSION: Importance of Patient Advocacy for Clinical Research

Why is patient advocacy beneficial to research?
Understanding of elements contributing to meaningful ways of involving patients in research
Advantages of involving patients early in research
What else needs to be done?

Carole Scrafton, Director & Co-Founder, Flutters and Strutters

3:00 pm - Afternoon Networking Break

3:30 pm - PANEL DISCUSSION: Improving Patient Access: Addressing Challenges from Innovation to Commercialization

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.

Moderator:

Dr. Ron Jortner, Founder/CEO, Aspire Biosciences

Panelist:

Julie Powell, Director, Patient Advocacy and Public Affairs, Pleco Therapeutics

Lindsay Randall, Founder, CEO, Mother, Arthur’s Quest

Hazel Dawson, Market Access Director, UK & Ireland, Alexion Pharma

Robert Donnell, EVP, Medicines Access, Smartway Pharma

4:10 pm - CLOSING PANEL DISCUSION: Global collaborations to improve diagnosis and care for persons living with a rare disease

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:

Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse infoframe Inc.

Panelists:
Anthony Hall, Chief Medical Officer, Healx

Juliet Hulse, Head of Clinical Operations, Pulse infoframe Inc.

Surabhi Verma, Chief Medical Officer, Leadiant Biosciences

Carole Scrafton, Director & Co-Founder, Flutters and Strutters

Dr. Ron Jortner, Founder/CEO, Aspire Biosciences

4:50pm - Session Chair's Closing Remarks

Sheela Upadhyaya – Life Sciences Consultant – specialising in Rare Disease

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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.