2nd Annual Clinical Trials in Rare Diseases Europe 2025

We are pleased to announce our event 2nd Annual Clinical Trials in Rare Diseases will take place on 25th- 26th November 2025!

25 - 26

November

2025
  • Munich, Germany
  • Complimentary
  • Why attend?
  • 2024 Agenda
  • 2024
  • Plan Your Visit
  • 2024 Sponsors
  • Media Centre
  • Event Gallery
  • Why partner?
  • Contact Us

Why attend?

A FANTASTIC NETWORK OF YOUR PEERS AND COLLEAGUES

With over 40 solution providers this will be a perfect platform to reconnect with current business partners, meet new vendors and discover which new technologies are being employed to improve your Rare Disease clinical trials.

See What It's All About

2024 Agenda

  • 19 Nov 2024
  • 20 Nov 2024
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8 AM

Registration and refreshments

8:50 AM

Chair’s opening remarks

Speakers

Erela Dana
Director of Neurology, GlobalData

9 AM

Transforming patient participation to make a meaningful impact on life-saving research

  • A focus on the prospective approach to improve trial readiness: long-term preparation for a successful trial
  • Growing the 4 components of trial readiness: Community, Site, Science, Regulator
  • Engaging with the rare disease community and advocacy groups to grow involvement of well-informed patients
  • Leveraging investigator expertise for pragmatic trial designs and patient identification
  • Building the evidence, from natural history to endpoint development, to support informed decisions by health authorities
  • Key patient recruitment considerations: Diversity Action Plans, Paediatrics et al: enrolling specific populations in rare disease studies

Speakers

Eugénie Suter, MD, PhD
Medical Director in Neuroscience & Rare Diseases, Roche

9:30 AM

Shaping the future of rare disease clinical trials: policy innovations and industry needs in Europe

  • Examining how the ACT EU initiative enhances collaboration, transparency, and efficiency in clinical trials.
  • Understanding the pivotal role of ERNs in enabling data sharing and streamlining trial logistics across Member States and challenges for industry integration.
  • Identifying the policy adjustments, infrastructure support, and regulatory flexibility needed from a pharmaceutical industry perspective to foster a more effective and sustainable clinical trial ecosystem in Europe under the Clinical Trials Regulation

Speakers

Dr. Seán Byrne
Senior Manager, Legal & Regulatory Affairs, EUCOPE (European Confederation of Pharmaceutical Entrepreneurs)

10 AM

Evidence: what is desirable, what is required, what is feasible?

  • What make evidence generation challenging in rare diseases?
  • Rare diseases have a series of characteristics that make them not fitting the mould of more prevalent diseases when it comes to clinical trials.
  • Strategies exists to address these complexities but are not always recognized by regulators or payers
  • Uncertainties in data package might only be resolved over time
  • Biomarkers, surrogate endpoints, PROs, natural history, real world data, comparators, single arm trials all have a role to play
  • Explaining the trade-offs faced during a development program is important to give context to the persisting uncertainties
  • We should seek to have an understanding of what is the best possible evidence for any given rare diseases at a point in time

Speakers

Vinciane Pirard
Senior Director of Public Affairs - Rare Diseases , Sanofi

10:30 AM

Morning refreshment and networking

11:15 AM

INTERACTIVE PANEL DISCUSSION: Do you really need clinical trials for rare disease drug research and development?

  • Implementing alternative methodology like RWD or AI to obtain and model data when clearly defined clinical trial roadmaps and regulatory pathways seldom exist
  • Discussing the necessity and ethics of placebos for orphan drug trials: Can we do something different?
  • How can you run studies with limited populations, does the clinical trial model work in this area of research?

Speakers

Eugénie Suter, MD, PhD
Medical Director in Neuroscience & Rare Diseases, Roche
Vinciane Pirard
Senior Director of Public Affairs - Rare Diseases , Sanofi
Erela Dana
Director of Neurology, GlobalData
Danny Liaw
Senior Director, MSD

12:15 PM

Truly acknowledging the value that patients can bring to every stage of the pharmaceutical development pathway

  • Turning to patients to help reduce waste, shorten development times and ensure the right outcomes are selected and measured effectively
  • Shedding light on the ACT-EU Multistakeholder partnership advisory board: an overview from a patient and policy perspective

Speakers

Russell Wheeler
Patient Advocate & Trustee , Leber's Hereditary Optic Neuropathy Society

12:45 PM

Lunch and networking

2 PM

Placing fear reduction at the core of the patient experience strategy: assessing the impact of diminished anxiety and increased sense of control for rare disease clinical trials

  • Improving the sense of security to reduce unnecessary suffering and distress for patients
  • Understanding the different types of patients and their individual response behaviours
  • Being hyper aware of cultural differences and biases to ensure sensitivity and effective outcomes for all
  • Sharing best practice on effective strategies with case studies and examples to help reduce patient drop out rate

Speakers

Roel van der Heijde
Facilitator & Trainer, Roel Rotterdam & Patient Experience Association

2:30 PM

Miransertib—an interesting path to an interesting disease

  • Developmental history of miransertib
  • Past clinical trial history in rare diseases
  • Current research
  • Future directions

Speakers

Danny Liaw
Senior Director, MSD

3 PM

Afternoon refreshments and networking

3:45 PM

Real World Data/ Evidence in translational medicine and rare disease clinical trials

  • AMR, Evolution and history of the natural world
  • The innate immune system – a billion years Proof of concept
  • RWD RWE – surgical sites and improved outcomes in oral surgery
  • RWD RWE – Serious burns and a survival benefit
  • COVID pandemic RWD RWE pulmonary administration
  • Safety studies and tolerability
  • The evolutionary successor to antibiotics is a multi-modal anti-inflammatory via TNF-a, Il6 and Il-10
  • Key milestones and next steps: CHMP and Orphan designation

Speakers

Myles Dakin
Chairman and Founder, Hypo-Stream

4:30 PM

Clinical trials in rare CNS disorders

  • Overview of rare diseases in neurology
  • Pharma focus on rare diseases in CNS for drug development – Where do industry interests lie?
  • Challenges in rare disease trials in neurology – biomarkers, endpoints, patient populations
  • Case study – R&D strategies in amyotrophic lateral sclerosis (ALS)

Speakers

Erela Dana
Director of Neurology, GlobalData

5 PM

Chair’s closing remarks

Speakers

Erela Dana
Director of Neurology, GlobalData

END OF DAY 1 AND NETWORKING DRINKS

8 AM

Registration and refreshments

8:50 AM

Chairperson’s opening remarks

Speakers

Manasi Vaidya
Editor, Insights and Pharmaceutical Technology, GlobalData

9 AM

Interactive Speaker-Hosted Roundtable Discussions

Interactive roundtable sessions offer a unique opportunity to come together with your peers to share best practice and develop solutions to critical challenges facing the industry as a whole. Each discussion will be led by a table moderator and will focus on a different challenge within rare disease clinical trials. Roundtables are an exciting, interactive way to build your personal network and learn from the experience and expertise of others.

After 30 minutes, delegates will have the opportunity to swap and choose a different table, and each roundtable will run twice.

RT 1 Providing start-up focused strategic input on the global study plan

Hosted by Elizabeth Thomson, Rare Disease Clinical Trial Expert

RT 2 Strategies for strengthening rare disease trial diversity to reduce healthcare disparities
RT 3 Improving support given to caregivers to reduce burden on patients’ families

10:30 AM

Morning refreshments and networking

11:15 AM

Challenges and opportunities in rare disease clinical development

  • How to approach the design and conduct of a clinical development plan in the rare disease space from a biotech perspective
  • Maximizing organizational and operational efficiency, and key considerations for regulatory adherence
  • How should pharma work with patient organisations and what are the best ways to engage with them?

Speakers

Carl Bjartmar
Chief Medical Officer, Augustine Therapeutics

11:45 AM

Insights into barriers to care in the rare disease community

  • Insights on the differences between represented and underrepresents populations on their barriers to care
  • Challenges and nuances of rare disease patients’ journey to receive a diagnosis
  • Mental health and well-being: the emotional toll rare diseases take on patients and caregivers
  • Exploring overall experiences individuals have had within the healthcare system: strengths and areas for improvement

Speakers

Jenifer Waldrop
Executive Director, Rare Disease Diversity Coalition

12:15 PM

Lunch, networking and prize draw

1:30 PM

Rare Disease studies can’t only recruit in US and EU5

  • What is a rare disease where?
  • Finding patients and rare disease recruitment
  • Are there locations that replace Russia, Ukraine and Israel where sadly we cannot work? A look at Asia Pacific

Speakers

Elizabeth Thomson
Rare Disease Clinical Trial Expert

2 PM

INTERACTIVE PANEL DISCUSSION: Navigating the complexities of insufficient resources to allow your rare disease trial to succeed in an increasingly competitive landscape

  • Battling site competitiveness to reduce delays on study start-up: what can you do differently to tackle these operational challenges?
  • When there are immense pressures on the trial ecosystem, how can we build and ensure quality in an innovative way
  • Developing an optimized strategy as a small to medium-sized biotech with limited resources to stay afloat
  • Discussing the importance of protocol optimization and specialized statistical support to reduce the pressure on sites and relieve bottlenecks
  • Global health inequalities: Are rare diseases rare everywhere?

Speakers

Manasi Vaidya
Editor, Insights and Pharmaceutical Technology, GlobalData
Carl Bjartmar
Chief Medical Officer, Augustine Therapeutics
Russell Wheeler
Patient Advocate & Trustee , Leber's Hereditary Optic Neuropathy Society
Myles Dakin
Chairman and Founder, Hypo-Stream

3 PM

HEAR FROM ACORSS THE POND, VIRTUAL SESSION Innovation and digital health for rare diseases

  • Challenges and limitations of rare disease research: how new solutions can help to overcome this
  • Embracing a patient centric approach in rare disease trials through digital health
  • Opportunities for innovation for orphan drug trials in the US

Speakers

Taiba Quraishi
Innovation Accelerator Lead, Alexion

3:30 PM

Chair’s closing remarks

END OF CONFERENCE

2024

Select a speaker to learn more

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Eugénie Suter, MD, PhD
Medical Director in Neuroscience & Rare Diseases, Roche

As a physician scientist, Eugenie Suter, MD, PhD, is dedicated to improving life for people with neurologic disorders. Her commitment
to drug development is founded on experience in research (Ph.D. in Neuroscience at Northwestern University; Immunology at Harvard's Boston Children's Hospital) as well as clinical medicine (Northwestern University; UCLA). Dr. Suter enjoys fostering innovation by drawing on her own experience and training in business, clinical trials, public policy and linguistics,. Driven by the need she witnesses clinically, she aims to bring new treatment options to individuals living with neurologic and other life-altering disorders through her work in Neuroscience & Rare Diseases as well as the Cardiovascular & Metabolic group at Roche.

Session Details:

Transforming patient participation to make a meaningful impact on life-saving research

2024-11-19, 9:00 AM

Session Details:

INTERACTIVE PANEL DISCUSSION: Do you really need clinical trials for rare disease drug research and development?

2024-11-19, 11:15 AM

View In Agenda
Next speaker
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Vinciane Pirard
Senior Director of Public Affairs - Rare Diseases , Sanofi

Global pharmaceutical, OMP and pediatric EU legislation. Rare diseases policies . Gene Therapy-ATMP . HTA and access frameworks. Cross border access. Patient engagement. Incentives for medicine development. Repurposing.
- All aspects of medical affairs across the product life cycle - Compassionate Use and Medical Need programs. Diagnostics. Real world evidence and registries
- Multistakeholder engagement

I’m a generalist in a highly specialised environment and a Medical Doctor with over 25 years experience primarily in biotechnology and innovative pharmaceutical companies.

Strong international experience in medical and policy departments collaborate across functions and across stakeholders.

Session Details:

Evidence: what is desirable, what is required, what is feasible?

2024-11-19, 10:00 AM

Session Details:

INTERACTIVE PANEL DISCUSSION: Do you really need clinical trials for rare disease drug research and development?

2024-11-19, 11:15 AM

View In Agenda
Next speaker
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Russell Wheeler
Patient Advocate & Trustee , Leber's Hereditary Optic Neuropathy Society

Experienced Patient Advocate active as a volunteer in the non-profit sector in Europe focused on rare diseases and especially rare eye disease. Background in finance and business strategy. Healthcare is in crisis and we need to collaborate to make things better - researchers, clinicians, regulators, payers, industry and patients, we all need to play our part. And across borders too - separatist nonsense like Brexit does not make anything better and we have to expend additional scarce resource to try and overcome the problems it creates

Session Details:

Truly acknowledging the value that patients can bring to every stage of the pharmaceutical development pathway

2024-11-19, 12:15 PM

Session Details:

INTERACTIVE PANEL DISCUSSION: Navigating the complexities of insufficient resources to allow your rare disease trial to succeed in an increasingly competitive landscape

2024-11-20, 2:00 PM

View In Agenda
Next speaker
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Roel van der Heijde
Facilitator & Trainer, Roel Rotterdam & Patient Experience Association

Roels inspiration: “Be the change you wish to see in the world” - Ghandi

Roel van der Heijde lives in Rotterdam, The Netherlands. He has a MA in International Business, is an international acknowledged NLP trainer, executive team coach and a certified death and grief counsellor.

He is a driven and experienced trainer and facilitator in:

  • - Patient Fear Reduction (for all caregivers);
  • - Team Collaboration;
  • - Vulnerability & Leadership;
  • - Discrimination & Inclusion.

Roels motivation: “Acknowledgement and recognition of the fears and emotions of our patients are the basis for excellent patient experience and patient safety”.

“Working with Groups I experience as thoroughly fascinating and inspiring. In interaction with groups I am every time amazed by the enormous potential of practical knowledge and direct experience that lies hidden in groups and that is so easily overlooked. Herein lies the basis for excellent vulnerable leadership.

Session Details:

Placing fear reduction at the core of the patient experience strategy: assessing the impact of diminished anxiety and increased sense of control for rare disease clinical trials

2024-11-19, 2:00 PM

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Next speaker
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Luca Faoro
Global Clinical Operations and Portfolio Management Team Leader , Novartis

A data driven, high performing and results orientated Team Leader, with 20+ years of experience in Strategic Planning and Programs Execution and with 15+ years of experience in direct management and development of teams and individuals, with a proven track record for delivering and optimizing clinical studies, leading high-performing teams in global, matrix-based environment across multiple therapeutic areas and compounds.

Expert in data analysis and interpretation, proficient in tools and technologies associated to project execution and recruitment strategies, and confident to work across different stakeholder groups to develop tactical/operational plans aligned with regulatory and commercial objectives.

Passionate about science, project management and people mentoring and improvement, with also a solid expertise in the regulation of clinical studies and data protection, risk assessment and root cause analysis, audits and implementation of CAPAs.

Next speaker
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Elizabeth Thomson
Rare Disease Clinical Trial Expert

Accomplished Clinical Operations and Development Executive with an impressive track record, boasting hands-on experience in Clinical Development, (25 years in consultancy) Across all phases (I to IV), from translational medicine to lifecycle management.

Held pivotal roles including Program Management, Clinical Operations, Site Selection and Patient Recruitment, Regulatory Affairs, Safety Oversight, Medical Writing, and Clinical Quality Assurance.

Spearheaded and successfully navigated asset licensure initiatives.

Worked with a diverse client base ranging from medium-sized pharma companies to venture capital-backed startups.

Managed a workforce of up to 32 individuals, overseeing operations both domestically and internationally.

Possesses a global perspective, having lived and worked in diverse regions including the UK, Germany, Japan, and Australia. Proven ability to excel in culturally complex and logistically challenging geographies, as well as managing single protocol global studies.

Led strategic transformation initiatives by identifying opportunities for efficiency improvement, enhancing business performance, driving innovation through strategic thinking, cross-company leadership, change management expertise, and a profound knowledge of industry trends and best practices.

Actively served as a Corporate and Departmental Diversity, Equity, and Inclusion (DE&I) representative, emphasizing the importance of diversity and inclusion within the workplace.

Held the role of a guest lecturer at King's College London, contributing to the Masters in Clinical Research program.

Session Details:

Rare Disease studies can’t only recruit in US and EU5

2024-11-20, 1:30 PM

View In Agenda
Next speaker
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Carl Bjartmar
Chief Medical Officer, Augustine Therapeutics

Carl Bjartmar, M.D., Ph.D. CEO and co-founder of BjartmarMed AB. Experienced global pharma and biotech executive providing medical, scientific, and operational consulting expertise to private equity and small/medium companies in drug development, business development and due diligence. Key expertise clinical development within orphan, neuromuscular, neurology and oncology indications. Solid record of global leadership, including two IPOs on Nasdaq OMX, agreed clinical plans with FDA, EMA and PMDA, successful completion of phase I-IV studies, and new drug approvals. Between 2015 and 2018 Chief Medical Officer at Wilson Therapeutics AB, Stockholm, Sweden (acquisition by Alexion Pharmaceuticals in 2018 at 70% premium - USD 850m). Chief Medical Officer at Augustine Therapeutics, Leuven, Belgium. Board member of iCoat Medical AB. More than 30 international publications (peer-reviewed articles, review articles, book chapters).

Session Details:

Challenges and opportunities in rare disease clinical development

2024-11-20, 11:15 AM

Session Details:

INTERACTIVE PANEL DISCUSSION: Navigating the complexities of insufficient resources to allow your rare disease trial to succeed in an increasingly competitive landscape

2024-11-20, 2:00 PM

View In Agenda
Next speaker
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Erela Dana
Director of Neurology, GlobalData

Erela Dana BSc, MPhil, is the Director of Neurology at GlobalData based in London. Erela has extensive disease area analysis and forecasting experience coupled with a wide breadth of therapy area expertise drawing from her previous roles. Erela has over 10 years’ experience in pharma, biotech and medical device industries both in-house and in consulting. Her industry experience includes EMA product launching as well as integration of strategic multichannel marketing plans, specializing particularly on novel biologic products including immunotherapies. As part of her prior consultancy engagements Erela has worked on numerous projects at GlobalData as a principal consultant as well as during her time at IQVIA Financial Institution Consulting, Align Strategy and Informa Pharma Consulting focusing on commercial due diligences, market opportunity assessments and new product planning, and investment strategies, including transactional advisory provided to investment professionals. Erela holds a master’s degree (MPhil) in Bioscience Enterprise from University of Cambridge and a BSc in Biomedical Engineering from Johns Hopkins University.

Session Details:

Chair’s opening remarks

2024-11-19, 8:50 AM

Session Details:

INTERACTIVE PANEL DISCUSSION: Do you really need clinical trials for rare disease drug research and development?

2024-11-19, 11:15 AM

Session Details:

Clinical trials in rare CNS disorders

2024-11-19, 4:30 PM

Session Details:

Chair’s closing remarks

2024-11-19, 5:00 PM

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Myles Dakin
Chairman and Founder, Hypo-Stream

Myles Dakin founded the research company, Hypo-Stream, to address anticipated needs in burns treatment and nosocomial infection with AMR pathogens.  His R&D journey has led to discovery of a multi-modal anti-inflammatory agent that neutralises both Il-6 and TNF-a alongside altering the ratio of Il-10 to Il-6 in a favourable way.  Hypo-Stream has progressed to an emerging pharma company with CHMP supported Ph III ready asset and orphan designation in a rare disease alongside a pipeline with potential global impact.

Session Details:

Real World Data/ Evidence in translational medicine and rare disease clinical trials

2024-11-19, 3:45 PM

Session Details:

INTERACTIVE PANEL DISCUSSION: Navigating the complexities of insufficient resources to allow your rare disease trial to succeed in an increasingly competitive landscape

2024-11-20, 2:00 PM

View In Agenda
Next speaker
Back
Danny Liaw
Senior Director, MSD

Session Details:

INTERACTIVE PANEL DISCUSSION: Do you really need clinical trials for rare disease drug research and development?

2024-11-19, 11:15 AM

Session Details:

Miransertib—an interesting path to an interesting disease

2024-11-19, 2:30 PM

View In Agenda
Next speaker
Back
Jenifer Waldrop
Executive Director, Rare Disease Diversity Coalition

Jenifer Ngo Waldrop joined the Black Women’s Health Imperative (BWHI) as Executive Director of the Rare Disease Diversity Coalition (RDDC) in October 2022. Before joining the leadership team of RDDC, she developed models and programs for diversity, equity, and inclusion initiatives to support multiple business units with Fortune 500 companies and organizations in Asia, Canada, and the US.

Jenifer has led initiatives at non-profit organizations where she recruited local community members to participate in numerous health-related fundraising events and developed programs to target disparities and underrepresented groups. In addition to facilitating development and fundraising, Jenifer developed a reputation for successful coalition building.

Jenifer received her undergraduate degree from Colby College and her Master of Human Resources Development from Villanova. As the Professional Development Director of the National Association of Asian-American Professionals (NAAAP) of Colorado, Jenifer continues her outreach in society. Through this organization, she mentors formally and informally.

Session Details:

Insights into barriers to care in the rare disease community

2024-11-20, 11:45 AM

View In Agenda
Next speaker
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Taiba Quraishi
Innovation Accelerator Lead, Alexion

Session Details:

HEAR FROM ACORSS THE POND, VIRTUAL SESSION Innovation and digital health for rare diseases

2024-11-20, 3:00 PM

View In Agenda
Next speaker
Back
Manasi Vaidya
Editor, Insights and Pharmaceutical Technology, GlobalData

Session Details:

Chairperson’s opening remarks

2024-11-20, 8:50 AM

Session Details:

INTERACTIVE PANEL DISCUSSION: Navigating the complexities of insufficient resources to allow your rare disease trial to succeed in an increasingly competitive landscape

2024-11-20, 2:00 PM

View In Agenda
Next speaker
Back
Dr. Seán Byrne
Senior Manager, Legal & Regulatory Affairs, EUCOPE (European Confederation of Pharmaceutical Entrepreneurs)

Session Details:

Shaping the future of rare disease clinical trials: policy innovations and industry needs in Europe

2024-11-19, 9:30 AM

View In Agenda
Next speaker

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2024 Sponsors

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SESSION SPONSORS

EXHIBITORS

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Partnership Opportunities

We work with you to design a bespoke package to meet your business needs, where you could:

  • Demonstrate your expertise on the agenda – have the attention of the entire audience. For greatest impact, why not position your talk before a networking break and immediately conduct follow up conversations with highly engaged attendees
  • Showcase your business and its services at an exhibition booth, which provides a perfect meeting place for prospective new clients
    Present the value proposition of your offering over your competitors, in front of an audience of active, engaged potential customers
  • Speak with key decision makers as part of our intimate, interactive roundtable style workshop sessions with over 8 hours allowed for networking

Event reach

  • 100+ attendees each year
  • 80% attendees C-suite level

 

Take a look at our current sponsors

Media Centre

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A FANTASTIC NETWORK OF YOUR PEERS AND COLLEAGUES!

With over 40 solution providers, this conference will be a perfect platform to reconnect with current business partners, meet new vendors and discover which new technologies are being employed to improve your Rare Disease clinical trials.

 

See What It's All About

Enquiry

Contact Us

SPONSORSHIP ENQUIRIES

Conor Taylor

Sponsorship Manager

+44 (0) 20 7936 6870

SPEAKING OPPORTUNITIES

Niki Khoshkbary

Senior Conference Producer

MARKETING ENQUIRIES

Moona Popal

Marketing Manager

DELEGATE ENQUIRIES

Sunny Saikia

VIP Delegate Manager

SPONSORSHIP ENQUIRIES

To enquire about sponsorship opportunities for the conference, please contact:

Conor Taylor

Sponsorship Manager


+44 (0) 20 7936 6870

SPEAKER ENQUIRIES

To enquire about speaking opportunities for the conference, please contact:

Niki Khoshkbary

Senior Conference Producer