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 26th- 27th November 2025!

26 - 27

November

2025
  • Marriott Munich City West , Munich, Germany
  • Complimentary
  • Why attend?
  • Agenda
  • Speakers
  • Plan Your Visit
  • Event Gallery
  • 2025 Sponsors
  • Media Centre
  • Why partner?
  • Contact Us
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Sponsors include:

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Company Information

Euraxi is a project management specialist in clinical research and epidemiology, assisting clients with their projects.
Euraxi started Home Nursing activities on 2014 (over 180 projects awarded) with a dedicated nurses Team and Country Study Managers (France and affiliates in BEL/NED/DEU/ITA/SPA) working in various indication from children to elderly population.

Company Website

To learn more, please visit our website - https://www.euraxi.fr/en/

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Company Information

IDDI optimizes the clinical development of drugs, biomarkers and IVD using a unique combination of advanced biostatistics and innovative data collection technology.

IDDI covers the full spectrum of trial design, clinical data collection, management, analysis and reporting for Phase I- IV clinical trials.

– IDDI’s services include: Clinical Development Plans, Protocol Development, Randomization (IWRS/IRT) with Drug Supply Management, EDC, Medical Coding, Data Management, Biostatistics, IDMC Support, Biomarker/Diagnostics Validation, Medical Writing.

– IDDI’s therapeutic areas of expertise include oncology, ophthalmology, infectious diseases and cardiology.
– Experience: 27+ years – 980+ trials (630+ in oncology) – 20 FDA/EMA approvals to-date
– HQ in Belgium with offices in the USA ( Raleigh, NC, Boston, MA and San Francisco, CA).

Company Website

To learn more, please visit our website - http://www.iddi.com/

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    Why attend?

    This event will explore the challenges in clinical trials for orphan drug and rare disease trials in Europe, bringing together clinical operations professionals from across multiple therapeutic areas to discuss common obstacles and solutions. We will explore the benefits of early planning and engagement with the EMA and other regulatory authorities, the importance of working closely with patients and patient advocacy groups, recruiting and retaining patients for rare disease studies, and much more.

    We look forward to seeing you there!

    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.

    Testimonials

    See What It's All About

    Agenda

    • 26 Nov 2025
    • 27 Nov 2025
    Expand All

    8 AM

    Registration and refreshments

    8:50 AM

    Chair’s opening remarks

    Speakers

    Erela Dana
    Director of Neurology, GlobalData

    9 AM

    KEYNOTE: Funding, investment and budgeting for clinical trials: Sharing best practices to ensure trial success for small/ medium pharma and biotechs

    • Ensuring your trial is both cost-conscious and efficient without compromising on quality and patient safety
    • Why financial accruals for clinical and R&D processes are still so challenging despite years of work to alleviate these obstacles
    • Strategies for cost-effective budget allocation to maintain long-term financial health and positive ROI
    • Avoiding common mistakes to keep your clinical trial budget on track
    • Supporting startups: Tackling investor’s lack of willingness to invest in smaller biotechs

    Speakers

    Ivo Timmermans
    Co-Chief Executive Officer, Pleco Therapeutics

    9:30 AM

    Reserved for featured sponsor

    10 AM

    Designing appropriate study protocols with limited patient numbers and heterogeneity in rare diseases

    • Finding innovative ways to demonstrate efficacy and safety with small sample sizes
    • Strategies for embedding protocol flexibility in a rare disease environment
    • Collaborating with rare disease CROs to employ statistical methods and help select appropriate endpoints and enhance trial’s chance of success
    • Realistically assessing inclusion and exclusion criteria to ensure adequate enrolment

    Speakers

    Channa Debruyne
    Global Clinical Development Lead, Late stage & LCM Oncology, Servier

    10:30 AM

    Reserved for featured sponsor

    11 AM

    Morning refreshments and networking

    11:30 AM

    PANEL DISCUSSION: Overcoming sites’ reluctance to run trials in rare disease indications

    • When resources are tight, how can rare disease trials beat the coemption and win sites over
    • Incentivising sites to run orphan trials
    • Balancing patient, site, CRO and investor priorities in your trial design
    • How can CROs help the phenomenon, and enable rare disease patients to access the appropriate sites
    • Proving to sites the ROI in running rare disease trials

    Speakers

    Erela Dana
    Director for Neurology and Immunology, GlobalData
    Johannes de Munter
    CEO, Neuroplast
    Dr. Cornelia Dechant
    Patient Advocate, Co-Founder of International Porphyria Patient Network

    12:15 PM

    Reserved for event sponsor

    12:45 PM

    PATIENT PERSPECTIVE: How should pharma work with patient organisations and what are the best ways to engage with them?

    Speakers

    Dr. Cornelia Dechant
    Patient Advocate, Co-Founder of International Porphyria Patient Network

    1:15 PM

    Reserved for technology spotlight

    1:30 PM

    Lunch and networking

    2:30 PM

    How to optimize cost-efficiency in rare diseases clinical trials

    • Control costs in rare disease clinical trials by tackling complexity and uncertainty head-on
    • Optimize site and country selection to reduce startup delays and unnecessary costs
    • Streamline vendor management with clear oversight and performance-driven partnerships
    • Leverage operational efficiencies (monitoring strategies, data management, patient engagement)
    • Maintain quality and patient focus while optimizing financial sustainability

    Speakers

    Stéphane Wilzius
    Head of Clinical Operations a.i., Santhera Pharmaceuticals

    3 PM

    Reserved for event sponsor

    3:30 PM

    Alleviating data challenges in rare disease research when data sets are limited

    • Optimal ways to battle with data quality issues to ensure responsible data collection, analysis, and interpretation
    • Capturing data effectively to make the most of your limited data

    Speakers

    Clara Cali Mella
    Senior Analytics and Insights Manager, Bayer

    4 PM

    Afternoon refreshments and networking

    4:30 PM

    Analysis of Real World (Audit) Data in driving drug discovery

    • Use of RWD in driving drug discovery in rare diseases
    • Leveraging therapeutics for rare disease to larger, multiple global applications

    Speakers

    Myles Dakin
    CEO & Founder, Hypo-Stream, Cambridge UK

    5 PM

    CLOSING PANEL DISCUSSION: Strategies for minimizing participant burden to ensure on-time enrolment in rare disease research

    • Patient reimbursement: how to relieve financial pressures on patients participating in orphan trials
    • Where are the main hurdles patients face and how can these be removed?
    • Addressing low levels of health literacy in the patient population: how to improve communication
    • Engaging patients and advocacy groups early on in the clinical trial design process
    • Barriers to clinical trial participation from a patient perspective

    Speakers

    Erela Dana
    Director for Neurology and Immunology, GlobalData
    Nikolina Krajnovic
    Director Clinical Operational Execution, BioNTech
    Dr. Cornelia Dechant
    Patient Advocate, Co-Founder of International Porphyria Patient Network
    Stéphane Wilzius
    Head of Clinical Operations a.i., Santhera Pharmaceuticals

    5:45 PM

    Chair’s closing remarks

    END OF DAY 1 AND NETWORKING DRINKS

    8 AM

    Registration and refreshments

    8:50 AM

    Chair’s opening remarks

    Speakers

    Erela Dana
    Director for Neurology and Immunology, 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 diseases 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.

    1. Measuring success by assessing the effectiveness of your patient enrolment strategy

    2. Optimising the latest technology to drive innovation and drug development in orphan drug research

    3. Ensuring rare disease trials take an adaptive approach to be scientifically robust and operationally feasible

    10 AM

    Morning refreshments and networking

    10:45 AM

    KEYNOTE: The challenges of bringing your own stem cells back as a platform product (Neuro-Cells®) and complete both a Phase I and Phase II clinical trial in (sub)acute spinal cord injured patients

    • The heterogeneity of patients and the accuracy of the golden standards and what does that mean when studying rare diseases
    • Working with a living personalized ATMP product with a limited shelf life, the logistics and the two-step release certification
    • How to position your orphan treatment within standard care and what are the hurdles to overcome
    • Discussions with the rehabilitation experts and the patients: what impacts the quality of life of a patient suffering a spinal cord injury best.
    • A listing of the non-conformities and how to resolve this real time.

    Speakers

    Johannes de Munter
    CEO, Neuroplast

    11:15 AM

    Reserved for event sponsor

    11:45 AM

    A framework for inclusive and accessible clinical research in rare diseases

    Speakers

    Jenifer Waldrop
    Executive Director, Rare Disease Diversity Coalition

    12:15 PM

    Reserved for event sponsor

    12:45 PM

    Lunch, networking and prize draw

    2:45 PM

    PANEL DISCUSSION: How innovation will shape the future of clinical research in rare diseases

    • How AI tools are revolutionising rare disease patient enrolment and subsequently trial success?
    • Can we make rare disease trials, protocols and study design simpler with limited data and patient populations?
    • Training sites and staff on new systems to maximise the benefits of the latest technology and streamline processes
    • Assessing the efficiency and success of pragmatic trials for more patient-centred approaches: do they work for rare diseases?
    • Leveraging digital platforms and social media outreach to identify and recruit patients

    Seats available

    Speakers

    Erela Dana
    Director for Neurology and Immunology, GlobalData

    3:15 PM

    Session reserved for

    Speakers

    Asiyah Nawab, MSc
    Clinical Trial Analyst, GlobalData

    3:45 PM

    Chair’s closing remarks

    END OF CONFERENCE

    Speakers

    Select a speaker to learn more

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    Clara Cali Mella
    Senior Analytics and Insights Manager, Bayer

    Session Details:

    Alleviating data challenges in rare disease research when data sets are limited

    2025-11-26, 3:30 PM

    View In Agenda
    Next speaker
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    Asiyah Nawab, MSc
    Clinical Trial Analyst, GlobalData

    Asiyah Nawab, MSc, is an Analyst at GlobalData’s London office with the Immunology team. She is involved in the production of in-depth forecasting reports, opportunity assessments, and competitive landscape analysis reports for immunology-related areas. Additionally, Asiyah has extensive research experience in the field of Pharmaceutics, with her research in the encapsulation of drugs within microparticles using emulsion electrospraying, supplemented through in vitro bacterial studies. Asiyah holds a Master of Science in Pharmaceutics from University College London (UCL), and a Bachelor’s degree in Biochemistry from King’s College London.

    Session Details:

    Session reserved for

    2025-11-27, 3:15 PM

    View In Agenda
    Next speaker
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    Erela Dana
    Director for Neurology and Immunology, GlobalData

    Session Details:

    PANEL DISCUSSION: Overcoming sites’ reluctance to run trials in rare disease indications

    2025-11-26, 11:30 AM

    Session Details:

    Chair’s opening remarks

    2025-11-27, 8:50 AM

    Session Details:

    PANEL DISCUSSION: How innovation will shape the future of clinical research in rare diseases

    2025-11-27, 2:45 PM

    Session Details:

    CLOSING PANEL DISCUSSION: Strategies for minimizing participant burden to ensure on-time enrolment in rare disease research

    2025-11-26, 5:00 PM

    View In Agenda
    Next speaker
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    Myles Dakin
    CEO & Founder, Hypo-Stream, Cambridge UK

    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:

    Analysis of Real World (Audit) Data in driving drug discovery

    2025-11-26, 4:30 PM

    View In Agenda
    Next speaker
    Back
    Johannes de Munter
    CEO, Neuroplast

    Session Details:

    PANEL DISCUSSION: Overcoming sites’ reluctance to run trials in rare disease indications

    2025-11-26, 11:30 AM

    Session Details:

    KEYNOTE: The challenges of bringing your own stem cells back as a platform product (Neuro-Cells®) and complete both a Phase I and Phase II clinical trial in (sub)acute spinal cord injured patients

    2025-11-27, 10:45 AM

    View In Agenda
    Next speaker
    Back
    Dr. Cornelia Dechant
    Patient Advocate, Co-Founder of International Porphyria Patient Network

    Session Details:

    PANEL DISCUSSION: Overcoming sites’ reluctance to run trials in rare disease indications

    2025-11-26, 11:30 AM

    Session Details:

    PATIENT PERSPECTIVE: How should pharma work with patient organisations and what are the best ways to engage with them?

    2025-11-26, 12:45 PM

    Session Details:

    CLOSING PANEL DISCUSSION: Strategies for minimizing participant burden to ensure on-time enrolment in rare disease research

    2025-11-26, 5:00 PM

    View In Agenda
    Next speaker
    Back
    Ivo Timmermans
    Co-Chief Executive Officer, Pleco Therapeutics

    Session Details:

    KEYNOTE: Funding, investment and budgeting for clinical trials: Sharing best practices to ensure trial success for small/ medium pharma and biotechs

    2025-11-26, 9:00 AM

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

    Session Details:

    A framework for inclusive and accessible clinical research in rare diseases

    2025-11-27, 11:45 AM

    View In Agenda
    Next speaker
    Back
    Stéphane Wilzius
    Head of Clinical Operations a.i., Santhera Pharmaceuticals

    Session Details:

    How to optimize cost-efficiency in rare diseases clinical trials

    2025-11-26, 2:30 PM

    Session Details:

    CLOSING PANEL DISCUSSION: Strategies for minimizing participant burden to ensure on-time enrolment in rare disease research

    2025-11-26, 5:00 PM

    View In Agenda
    Next speaker
    Back
    Channa Debruyne
    Global Clinical Development Lead, Late stage & LCM Oncology, Servier

    Session Details:

    Designing appropriate study protocols with limited patient numbers and heterogeneity in rare diseases

    2025-11-26, 10:00 AM

    View In Agenda
    Next speaker

    Plan Your Visit

    Venue

    Marriott Munich City West , Munich, Germany

    Book Your Stay

    Accommodation

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    2025 Sponsors

    Select a sponsor to learn more

    FEATURED SPONSOR

    SESSION SPONSORS

    Coffee Break Sponsor

    EXHIBITORS

    CO-Sponsors

    Why partner?

     

    Contact us to become a partner

    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

    Become A Media Partner

    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

    Maya Hudson

    Deputy Head of Production

    +44 204540 7766

    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:

    Maya Hudson

    Deputy Head of Production


    +44 204540 7766