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
close

Sponsors include:

Back

Company Information

ARENSIA Exploratory Medicine, a German-based company, is a leader in “first-in-patient” Phase IB and II/Proof-of-Concept clinical trials across a wide range of disease areas. With 14 proprietary research clinics and cutting-edge Phase I infrastructure, ARENSIA accelerates the early detection of efficacy signals for new therapies at an unparalleled speed. Its streamlined, highly efficient approach reduces patient recruitment time and costs by over 50% compared to conventional trial sites, which are often overcrowded and understaffed. This exceptional performance has resulted in a 93% repeat business rate and robust growth through collaborations with top pharmaceutical companies, biotechs, and global CROs. Headquartered in Düsseldorf, ARENSIA operates with a team of 600 professionals across 8 countries.

Company Website

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

Next Sponsor
Back

Company Information

Medidata is powering smarter treatments and healthier people through digital solutions to support clinical trials. Celebrating 25 years of ground-breaking technological innovation across more than 35,000 trials and 10 million patients, Medidata offers industry-leading expertise, analytics-powered insights, and the largest patient-level historical clinical trial data set in the world. More than 1 million registered users across approximately 2,300 customers trust Medidata’s seamless, end-to-end platform to improve patient experiences, accelerate clinical breakthroughs, and bring therapies to market faster. A Dassault Systèmes brand (Euronext Paris: FR0014003TT8, DSY.PA), Medidata is headquartered in New York City and has been recognized as a Leader by Everest Group and IDC. Discover more at www.medidata.com and follow us @Medidata.

Company Website

To learn more, please visit our website - https://www.medidata.com

Next Sponsor
Back

Company Information

Worldwide is changing how the world experiences CROs – in the best possible way. From early phase, bioanalytical sciences, late phase, post-approval and real-world evidence, Worldwide Clinical Trials provides full-service drug development services for central nervous system, cardiovascular, metabolic, general medicine, oncology and rare disease therapies.

Company Website

To learn more, please visit our website - https://www.worldwide.com

Next Sponsor
Back

Company Information

Suvoda is a global clinical trial technology company specializing in complex, life-sustaining studies in therapeutic areas like oncology, central nervous system (CNS), and rare disease. Founded in 2013 by experts in eClinical technologies, Suvoda empowers clinical trial professionals to manage the most urgent moments in the most urgent trials through advanced software solutions delivered on a single platform. Headquartered outside Philadelphia, Suvoda also maintains offices in Portland, OR, Barcelona, Spain, Bucharest and Iasi, Romania, and Tokyo, Japan. The company’s Net Promoter Score (NPS) consistently exceeds the technology industry average, contributing to the company being selected by trial sponsors and CROs to support more than 1,000 trials across 80 countries.

Company Website

To learn more, please visit our website - https://www.suvoda.com/

Next Sponsor
Back

Company Information

Syneos Health (Nasdaq:SYNH*) is the only fully integrated biopharmaceutical solutions organization. Our company, including a Contract Research Organization (CRO) and Contract Commercial Organization (CCO), is purpose-built to accelerate customer performance to address modern market realities. Created through the merger of two industry leading companies – INC Research and inVentiv Health – we bring together more than 21,000 clinical and commercial minds with the ability to support customers in more than 110 countries. Together, we share insights, use the latest technologies and apply advanced business practices to speed our customers’ delivery of important therapies to patients. To learn more about how we are shortening the distance from lab to lifeTM visit SyneosHealth.com. 

Company Website

To learn more, please visit our website - www.SyneosHealth.com

Next Sponsor
Back

Company Information

Scimega is a niche Canadian Oncology CRO with a singular focus on promoting and contributing to the successful conduct of cutting-edge oncology clinical trials in Canada. With a permanent, Lean-coached operations team our proven track record assisting mid-size and smaller US biotech/biopharma sponsors to undertake global oncology studies in Canada is bundled with 25 years of lessons learned!

Company Website

To learn more, please visit our website - www.scimega.com

Next Sponsor
Back

Company Information

Endpoint Clinical is a leading clinical trial technology and service partner, delivering adaptable RTSM solutions with a focus on security, stability, and trust. With decades of expertise and a proactive, innovation-driven approach, Endpoint minimizes friction, reduces risk, and enables your teams to focus where it matters most—on trial success.

Company Website

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

Next Sponsor
Back

Company Information

Translational Drug Development (TD2) is a world-class oncology drug development organization specializing in the development of new oncology medicines. TD2 offers fully integrated precision oncology clinical development solutions that combine the highest standards in clinical trials management, disruptive imaging, biomarker strategies, and unparalleled access to real-time patient data.

Company Website

To learn more, please visit our website - www.td2inc.com

Next Sponsor
Back

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/

Next Sponsor
Back

Company Information

Simply sourcing comparators – we offer a unique blend of small company flexibility and speed, backed with the large-scale buying power of the Orifarm Group, which is one of the largest pharmaceutical suppliers in Europe. With over €1.4B products sold in 2021 and more than 4,000 unique product lines sourced on a monthly basis, we have unparalleled access to “out of market” and manufacturer direct buying capability, both in the US and Europe.

Company website: www.Orifarm-CTS.com

Company Website

To learn more, please visit our website - www.Orifarm-CTS.com

Next Sponsor
Back

Company Information

PSI is a privately-owned, full-service contract research organization (CRO), operating globally. PSI’s reputation on the market place is that of a no-nonsense CRO, capable of saving pharmaceutical sponsors millions of development dollars by consistently meeting clinical trial timelines. PSI’s global reach supports the operations of clinical trials across multiple countries.

Company Website

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

Next Sponsor
Back

Company Information

Aixial Group is a well-established, oncology focused, global CRO. Aixial is recognized  for creative and proactive clinical expertise with an understanding and appreciation of the financial trust placed in their team by the sponsor to deliver results.

Next Sponsor
Back

Company Information

MMS Holdings (MMS) is an award-winning, data-focused clinical research organization (CRO) that supports the pharmaceutical and biotech industries with a proven, scientific approach to complex trial data and regulatory submission challenges. Strong industry experience, a 19-year track record, AI technology-enabled services, and a data-driven approach to drug development make MMS a valuable CRO partner. With a global footprint across four continents, MMS maintains an industry-leading customer satisfaction rating. For more information, visit www.mmsholdings.com

Company Website

To learn more, please visit our website - www.mmsholdings.com

Next Sponsor
Back

Company Information

iOMEDICO is a leading full-service CRO and Real-World Data provider focused on oncology and hematology with a recruitment rate of over 6,000 new patients each year. We conduct phase I-IV trials and the RWD platforms provide deep insight into patient journeys and PRO in the indication, support trial design, biomarker analyses and market access purposes.

Next Sponsor
Back

Company Information

Invisio supports sponsors, study sites and CROs in the implementation of international clinical study projects. Invisio provides, trains and coordinates clinical study personnel such as flying study nurses or physicians. Additionally, Invisio specializes in project management for multicenter clinical trials, as well as the parallel implementation of studies at multiple study sites across Europe.

Company Website

To learn more, please visit our website - www.invisio.info

Next Sponsor
Back

Company Information

QPS is a global contract research organization (CRO) providing discovery, preclinical and clinical drug development services since 1995. Our mission is to accelerate pharmaceutical breakthroughs across the globe by delivering custom-built research services. An award-winning leader focused on bioanalytics and clinical trials, QPS is known for proven quality standards, technical expertise, a flexible approach to research, client satisfaction and turnkey laboratories and facilities. We are passionate about our work, creating value for patients and customers while generating opportunities for employees to thrive.

Company Website

To learn more, please visit our website - www.qps.com

Next Sponsor
Back

Company Information

NextPharma expertise and skilled team will support your clinical program, locally or globally, in the following areas:

  • Primary and secondary packaging
  • Manufacturing of IMP and matching placebo
  • Randomisation
  • Worldwide distribution at controlled temperatures
  • IMPD and QP Service

Let NextPharma work with you to successfully and seamlessly take your compound to the next Phase

Company Website

To learn more, please visit our website - www.nextpharma.com

Next Sponsor
Back

Company Information

NeSPaT has been providing patient travel and reimbursement services since 2013 combining deep local expertise with wide regional coverage. With a multinational team and a focus on digital innovation combined with a human approach, NeSPaT streamlines patient logistics, offering tailored services, 24/7 support, and seamless documentation management.

Company Website

To learn more, please visit our website - https://nespat.net/

Next Sponsor
Back

Company Information

HEMEX is a European CRO supporting pharmaceutical, biotech, and MedTech SMEs with clinical development solutions. Headquartered in Basel and operating globally, we manage multinational trials. Our services include clinical operations, regulatory support, quality assurance, BA/BE and PK studies, patient recruitment, and advanced medical imaging

Company Website

To learn more, please visit our website - https://hemex.ch/

Next Sponsor
Back

Company Information

IROS, part of the M42 group, is a pioneering UAE-based Clinical Research Organization (CRO) dedicated to advancing excellence in clinical research. With comprehensive offerings across all therapeutic domains, a high-caliber team, and cutting-edge technologies, IROS delivers full-service and tailored solutions to drive innovation, improve patients’ lives, and shape the health landscape.

Company Website

To learn more, please visit our website - www.iros.ai

Next Sponsor

    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

    8:30 AM

    Registration and refreshments

    9:15 AM

    Chair’s opening remarks

    Speakers

    Erela Dana
    Director of Neurology, GlobalData

    9:30 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

    10 AM

    The AI-First Protocol in Rare Diseases: Accelerating Research for the Few Who Need It Most

    Rare-disease trials face unique challenges - small patient populations, dispersed sites, and limited data. This session explores how AI-first protocol design transforms feasibility and speed in rare-disease research, enabling smarter, patient-centric, and regulator-ready studies from day one. Key Takeaways:

    • How AI-first protocols reduce amendments and time-to-first-patient in small-population studies
    • Using real-world and registry data to simulate feasibility and optimize site selection
    • Building adaptive, patient-centric designs aligned with EMA and FDA expectations
    • Integrating AI into cross-functional workflows — from clinical to regulatory teams
    • Case examples showing measurable acceleration and cost savings in rare-disease trials

    Speakers

    Matteo Talotta
    Director of Clinical Solutions – Europe, Biorce

    10:30 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

    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
    Guido Wuerth
    Head of Corporate Development, Radiopharma, Affibody AB, Sweden

    12:15 PM

    Exploring GCC clinical research landscape

    • Infrastructure Strengths of the UAE & GCC for Clinical Research
    • UAE’s Precision Medicine and Genomics Ecosystem: A Global Benchmark
    • Why the GCC is Emerging as the Next Global Hub for Clinical Trials

    Speakers

    Islam Eltantawy
    General Manager, Iros

    12:45 PM

    Lunch and networking

    1:45 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, Santhera Pharmaceuticals

    2:15 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
    Data Strategy Lead Bayer

    2:45 PM

    PATIENT ADVOCATE PERSPECTIVE: The Impact of Patient Networks on EPP Care: Insights from the International Porphyria Patient Network and EPP Austria

    • Role of patient organisations in EPP and rare diseases
    • Why early and meaningful engagement matters
    • Common challenges (power imbalance, tokenistic involvement)
    • Best practice principles for collaboration (transparency, co-creation, fair compensation)
    • Case examples: IPPN and EPP Austria influencing clinical pathways and policy
    • Key recommendations for pharma partnerships

    Speakers

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

    3:15 PM

    Afternoon refreshments and networking

    3:45 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

    4:15 PM

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

    • Where are the main hurdles patients face and how can these be removed?
    • Barriers to clinical trial participation from a patient perspective
    • Informed consent and addressing various 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
    • Patient reimbursement: how to relieve financial pressures on patients participating in orphan trials

    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, Santhera Pharmaceuticals

    END OF DAY 1 AND NETWORKING DRINKS

    5 PM

    Chair’s closing remarks

    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

    THE ROUNDTABLES WILL TAKE PLACE IN THE ONCOLOGY CONFERENCE ROOM NEXT DOOR

    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 oncology and orphan drug 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. Discussing the increased role of China R&D in developing new products
      Hosted by Sezcan Ozturk, Vice President, Head of Europe, Luzsana Biotechnology
    2. AI in clinical operations
      Hosted by Pietro Belligoli, Researcher, Former Roche
    3. Running trials in low-cost countries
      Hosted by Martin Neumayer, Head Development Program Support and CRO-Vendor Management, Sandoz
    4. What is the best framework of strategies, processes and pathways for pharma and patient organisations to create positive, lasting partnerships?
      Hosted by Dr. Anupoma Haque, EUPATI Fellow, Patient-Centric Public & Digital Health Expert, HTA and Policy Professional, Inclusion & Equity Trainer

    Speakers

    Dr. Anupoma Haque
    EUPATI Fellow, Patient-Centric Public & Digital Health Expert, HTA and Policy Professional, Inclusion & Equity Trainer
    Martin Neumayer
    Head Development Program Support and CRO-Vendor Management, Sandoz
    Sezcan Ozturk
    Vice President, Head of Europe, Luzsana Biotechnology
    Pietro Belligoli
    Researcher, Former Roche

    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

    Are you listening? How to retain, not just engage, stakeholders in clinical trials and people as partners

    • Effective communication with target patients/families/care professionals and RETAINING them
    • How inclusion of AI looks like in this communication, trust building, meeting needs and retaining engagement, from:
    • Care researchers' perspective
    • Care Professionals' perspective, and most importantly,
    • Caregivers' perspective (lived experience and real-world-evidence) in the context of "rare diseases"

    Speakers

    Dr. Anupoma Haque
    EUPATI Fellow, Patient-Centric Public & Digital Health Expert, HTA and Policy Professional, Inclusion & Equity Trainer

    11:45 AM

    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
    Myles Dakin
    CEO & Founder, Hypo-Stream, Cambridge UK
    Tània Nadal
    Global Clinical Development Head, ESTEVE
    Clara Cali Mella
    Data Strategy Lead Bayer

    12:45 PM

    Lunch and networking

    1:45 PM

    Clinical trials in rare immunology disorders

    • Overview of rare diseases in Immunology
    • Pharma focus on rare diseases in Immunology for drug development - where do industry interests lie?
    • Challenges in rare disease trials in immunology
    • Case Study - Sarepta's Elevidys in Duchenne Muscular Dystrophy (DMD)

    Speakers

    Asiyah Nawab, MSc
    Clinical Trial Analyst, GlobalData

    2:15 PM

    A framework for inclusive and accessible clinical research in rare diseases

    • Uncovering the invisible: a 20-year analysis of medically underrepresented populations in clinical trials
    • Benchmarking against the US census reveals structural gaps and needs
    • Adopt a six-pillar architecture to embed equity from the ground up

    Speakers

    Jenifer Waldrop
    Executive Director, Rare Disease Diversity Coalition

    2:45 PM

    Afternoon refreshments, networking and prize draw

    3:30 PM

    CLOSING PANEL DISCUSSION: How AI is powering a new era of cancer drug discovery and development

    • Moving from basic functionalities to more meaningful impact and examples of this in practice
    • When AI is progressing so quickly, how will regulation evolve to match this speed?
    • When is AI not the answer?
    • How AI has revolutionised innovative ways of collecting data, biosimulation, and early disease diagnosis for oncology trials

    Moderator: Asiyah Nawab, Senior Healthcare Analyst (Immunology Team), GlobalData

    THIS SESSION WILL BE IN THE ONCOLOGY CONFERENCE ROOM

    Speakers

    Asiyah Nawab, MSc
    Clinical Trial Analyst, GlobalData
    Sezcan Ozturk
    Vice President, Head of Europe, Luzsana Biotechnology
    Stefano Ferrara
    Director, Clinical Science, BeiGene
    Dariusz Adamczewski
    Managing Director, Children’s Tumor Foundation Europe
    Pietro Belligoli
    Researcher, Former Roche

    END OF CONFERENCE

    4:30 PM

    Chair’s closing remarks

    Speakers

    Select a speaker to learn more

    Back
    Clara Cali Mella
    Data Strategy Lead Bayer

    I am dedicated to harnessing data to tackle complex challenges in the pharmaceutical sector, particularly within clinical operations to streamline drug development and enhance patient access. With a background in Data & Analytics Strategy, I specialize in solving problems and improving protocol design from an operational perspective. As an educator, I teach a modeling course at the university, mentoring aspiring data professionals. My focus is to leverage data-driven insights to optimize decision-making and contribute to more accessible treatments for patients.

    Session Details:

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

    2025-11-26, 2:15 PM

    Session Details:

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

    2025-11-27, 11:45 AM

    View In Agenda
    Next speaker
    Back
    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:

    Clinical trials in rare immunology disorders

    2025-11-27, 1:45 PM

    Session Details:

    CLOSING PANEL DISCUSSION: How AI is powering a new era of cancer drug discovery and development

    2025-11-27, 3:30 PM

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

    Detail oriented, highly motivated biomedical and healthcare professional with record of successful experience in device development and design; regulatory, clinical and strategic advisory services. Strong core financial modelling and analytical skill set, uniquely combined with advanced medical device and biotechnology background derived from academics.

    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, 11:45 AM

    Session Details:

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

    2025-11-26, 4:15 PM

    View In Agenda
    Next speaker
    Back
    Tània Nadal
    Global Clinical Development Head, ESTEVE

    My professional history is directly related to my 20+ years’ experience managing operations in healthcare sector across multiple locations, including Europe, US and Canada. What makes me stand-out is my record of optimising current processes to deliver quality services, establishing and growing businesses, and increasing staff performance and satisfaction. I am meticulous, creative, and focused professional with proven talent to foster productive relationships with top businesses / clients across all pharma segments. I excel at managing projects in all clinical phases, overseeing large budgets, as well as directing multidisciplinary global teams to exceed company KPIs while keeping the people development & care as main driver.

    Session Details:

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

    2025-11-27, 11:45 AM

    View In Agenda
    Next speaker
    Back
    Denise Lee
    Vice President Clinical Operations, Heidelberg Pharma

    Dynamic and passionate biopharma leader with over 25 years of global expertise spanning clinical practice and R&D, and CROs. A problem-solver and strategist at heart, I thrive on tackling challenges. As a Managing Director and servant leader, I have driven operational excellence and transformed business critical processes. Through empathy and empowerment, I have led diverse international teams with less than 10% employee turnover.

    In my current role, I saved over $2 million USD within six months through renegotiation and introduction of efficiencies, while doubling our satisfaction with vendors via introduction of governance structures and key performance, quality, and risk indicators.

    Deeply committed to ethical and sustainable practices, I leverage data-driven decision-making to align vision, mission, and objectives with impactful outcomes. I bring energy, focus, and leadership to every initiative, ensuring excellence and meaningful contributions to healthcare.

    Next speaker
    Back
    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, 3:45 PM

    Session Details:

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

    2025-11-27, 11:45 AM

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

    People management Strategic management Clinical Research Development for EU registration Clinical Research especially ATMP products Regulatory affairs especially ATMP products (EMA) Epidemiology Specialties: Autologous Stem Cell Research and Regulatory International research development market development in medical industry Change management and interim management

    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 ADVOCATE PERSPECTIVE: The Impact of Patient Networks on EPP Care: Insights from the International Porphyria Patient Network and EPP Austria

    2025-11-26, 2:45 PM

    Session Details:

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

    2025-11-26, 4:15 PM

    View In Agenda
    Next speaker
    Back
    Ivo Timmermans
    Co-Chief Executive Officer, Pleco Therapeutics
    • Strong record in clinical development, regulatory, medical affairs, medical information, pharmacovigilance
    • Worked at senior management level ranging from small biotech companies to major pharmaceutical companies, with strong cross functional and commercial alignment
    • Result-oriented, hands-on management style, entrepreneurial, team player
    • Experience in variety of international roles managing teams across the globe
    • Specialities: infectious diseases, virology, immunology, oncology, vaccines
    • Languages: proficient to fluent in English, German, French, Swedish, Dutch

    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, 9:30 AM

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

    I am a results-driven healthcare executive with a proven track record of fostering organizational excellence and advancing initiatives that strengthen communities. By promoting fairness, opportunity, and innovation, I create meaningful solutions that improve access to care for underserved populations while supporting the success of organizations and fostering a culture of collaboration and shared purpose. My niche is taking complex ideas, analyzing and communicating them effectively to the respective audiences. My key skills include leading teams, fostering global perspectives, facilitating, coaching, providing consultative services, and managing budgets. I thrive in a team-oriented organization where everyone feels welcome. I believe in having authentic and transparent relationships, in promoting an inclusive environment with a diverse group of multi-cultural thinkers to promote innovation and creativity in obtaining targets. I have been described as “the consummate professional who is warm and approachable." #ONO

    Session Details:

    A framework for inclusive and accessible clinical research in rare diseases

    2025-11-27, 2:15 PM

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

    With over 25 years of experience in global clinical operations, I’ve led the strategic execution of drug development programs across oncology, rare diseases, and cell & gene therapies—navigating complex regulatory environments while ensuring scientific and operational excellence.

    From feasibility to execution, I bring a sharp focus on quality, risk mitigation, and cross-functional alignment—ensuring trials are delivered on time, within budget, and with uncompromising integrity.

    Highlights of my expertise:
    - Strategic oversight of global clinical development and evidence generation
    - Vendor selection, negotiation, and long-term partnership management
    - Deep knowledge of ICH-GCP, FDA, EMA, PMDA, EU MDR/IVDR, and ENCePP
    - Strong track record in team building, process improvement, and change management
    - Hands-on experience with audits, quality systems, and cross-functional collaboration

    Session Details:

    How to optimize cost-efficiency in rare diseases clinical trials

    2025-11-26, 1:45 PM

    Session Details:

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

    2025-11-26, 4:15 PM

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

    Experienced Medical Director with a demonstrated history of working in the academic, regulatory and Pharma industry involved in all clinical aspects on drug development. Skilled in Oncology, Good Clinical Practice (GCP), Biotechnology, Drug Development, and Pharmaceutical Industry.

    Session Details:

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

    2025-11-26, 10:30 AM

    View In Agenda
    Next speaker
    Back
    Matteo Talotta
    Director of Clinical Solutions – Europe, Biorce

    Matteo brings a multidisciplinary perspective to clinical innovation, with experience across banking, academia, government, and the private sector. He has worked internationally in Canada, Italy, the U.S., Spain, and Romania, gaining firsthand insight into diverse healthcare challenges.
    His career spans start-ups, scale-ups, and multinational corporations, sharpening skills in leadership, strategy, and solution-building. At Biorce, an AI-driven organization, he focuses on transforming drug development and clinical trials, making life-changing treatments for rare diseases more accessible through advanced technology and human insight.

    Session Details:

    The AI-First Protocol in Rare Diseases: Accelerating Research for the Few Who Need It Most

    2025-11-26, 10:00 AM

    View In Agenda
    Next speaker
    Back
    Dr. Anupoma Haque
    EUPATI Fellow, Patient-Centric Public & Digital Health Expert, HTA and Policy Professional, Inclusion & Equity Trainer

    I am a Health and Technology (wearables, sensors, IoTs, hybrids) Management specialist with Business Development focus. Currently, I work with 2 Digital Health start-ups in Vienna, Austria and Dhaka, Bangladesh, as well as at the Ministry of Immigration and Diversity of Austria. I collaborate with different research initiatives on Diabetes & CVD Management, Mental Health Management of Researchers (COST Action EU) and remote monitoring. I support establishment & up-scaling health start-ups' technological, procedural and economic innovations to effect optimization of the Chain of Care. For me, Care is personalized, holistic, multidisciplinary involving individuals and organizations in an integrated, dynamic, and interactive system. I have a multidisciplinary background including Dentistry (Bachelor of Dental Surgery, Dhaka Dental College, Bangladesh), Public Health (Master with distinction, James P. Grant School of Public Health, Bangladesh), Health Care Management and Technology Management (Master, University of Twente, The Netherlands). Professionally, I gained competencies in Management (Health, technology, business, R&D, Policy), Business (Strategy, project management, main streaming), Networking and Advocacy. Result-oriented, problem-based, participatory, innovative, and holistic approach to analyzation and finding scalable, sustainable solutions are my strong points.

    Session Details:

    Interactive Speaker-Hosted Roundtable Discussions

    2025-11-27, 9:00 AM

    Session Details:

    Are you listening? How to retain, not just engage, stakeholders in clinical trials and people as partners

    2025-11-27, 11:15 AM

    View In Agenda
    Next speaker
    Back
    Nikolina Krajnovic
    Director Clinical Operational Execution, BioNTech

    Lean management / line management
    Main line management tasks: people development through trainings, coachings and sit-ins, performance assesments, resourcing
    Main process improvment tasks: structured problem solving sessions, interface coordination, SOP reviews, assesments and updates, quality systems development
    Main project delivery tasks: responsibility for project delivery within given timelines, budget and quality

    Session Details:

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

    2025-11-26, 4:15 PM

    View In Agenda
    Next speaker
    Back
    Islam Eltantawy
    General Manager, Iros

    Session Details:

    Exploring GCC clinical research landscape

    2025-11-26, 12:15 PM

    View In Agenda
    Next speaker

    Plan Your Visit

    Venue

    Marriott Munich City West , Munich, Germany

    Book Your Stay

    Accommodation

    Arena International Events Group “(Arena”) is aware of numerous third-party agencies which are contacting Arena’s customers and purporting to have access to hotel room bookings, events attendee lists and selling unsolicited services. It is unclear whether the agencies in question are in possession of such information, or whether they intend to scam unsuspecting customers. Whilst Arena International Events Group pursues such third-party companies for their potentially illicit activity, Arena encourages you to be cautious in engaging with such third-party agencies and Arena shall not accept any responsibility for any losses incurred by you should you choose to engage with third parties that have no affiliation to Arena International Events Group..

    Parking:

    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