Clinical Trials in Rare Diseases Europe 2024

We are pleased to announce the launch of a brand new event: Clinical Trials in Rare Diseases will take place on 19th-20th November 2024!

19 - 20

November

2024
  • Sheraton Brussels Airport Hotel ,Brussels, Belgium
  • Complimentary
  • Why attend?
  • Agenda
  • Speakers
  • Plan Your Visit
  • Sponsors
  • Media Centre
  • Event Gallery
  • Why partner?
  • Contact Us
close

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

Agenda

  • 19 Nov 2024
  • 20 Nov 2024
Expand All

8 AM

Registration and refreshments

8:50 AM

Chair’s opening remarks

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

Session reserved for event sponsor

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

Session reserved for event sponsor

11 AM

Morning refreshment and networking

11:30 AM

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

12 PM

Session reserved for event sponsor

12:30 PM

Addressing regulatory considerations to rare disease populations to ensure patient safety and compliance

  • How the latest changes in European clinical trial requirements and regulation have affected companies’ willingness to invest in these markets
  • Ensuring regulatory harmonisation of clinical trials in the EU for global brands: What are the challenges for central labs and what are they telling sponsors?
  • Gaining clarity on the requirements for clinical evidence and post-market performance follow-up
  • Solutions to promote orphan drug trial collaboration between regulators and healthcare systems

Speakers

Maria Fernanda Di Tata
Global Medical Affairs Director for Renal - Rare Diseases , Novartis

1 PM

Session reserved for Technology Spotlight

1:15 PM

Lunch and networking

2:15 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 Multistakeholder partnership advisory board: an overview from a patient and policy perspective

Speakers

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

2:45 PM

Session reserved for event sponsor

3:15 PM

Geopolitical considerations for the progress of rare disease clinical trials in Europe

  • Addressing cultural and socioeconomic diversity when adapting the evolving role of patient advocates and engagement strategies to respond to the varied needs and cultural contexts within Europe
  • What the global conflicts mean for the future of rare disease trials in European markets, is the outlook promising?
  • Discussion of global financial markets and how to adapt to them for rare disease trials

Speakers

Andrea Cusac
Fractional CCO , Hypo-Stream

3:45 PM

Afternoon refreshments and networking

4:15 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 maximize 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

4:45 PM

Session reserved for event sponsor

5:15 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

5:45 PM

Chair’s closing remarks

END OF DAY 1 AND NETWORKING DRINKS

8 AM

Registration and refreshments

8:50 AM

Chairperson’s opening remarks

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 How can we establish trust within less-represented groups?

10 AM

Morning refreshments and networking

10:30 AM

Insights to drive smarter decisions: What is strategically needed to achieve trial results in rare disease studies?

  • Navigating the complexities of clinical trial budget forecasting and payment
  • Assessing study feasibility to achieve registrational and commercial objectives
  • Validate your data: Integrating data visibility and generating the curve to determine your starting points
  • A case study on PNH: Exploring this collaborating with Alexion Pharmaceuticals

Speakers

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

11 AM

Session reserved for event sponsor

11:30 AM

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

12:15 PM

Session reserved for event sponsor

12:45 PM

Lunch and networking

1:45 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

2:15 PM

Session reserved for event sponsor

2:45 PM

Afternoon break and prize draw

3:15 PM

Improving support given to caregivers to reduce burden on patients’ families

  • Implementing support services for caregivers so they can provide information and early warnings of declining patient health
  • Educating caregivers so they are able to provide more compassionate care and more prepared to financially support
  • Maximizing efficiency and patient safety in rare disease trials through caregiver engagement
  • Prioritising caregiver involvement and embracing new ways to ensure they are fully supported

3:45 PM

Clinical trials in rare neuroimmunology diseases

Speakers

Erela Dana
Director of Neurology, GlobalData

4:15 PM

Chair’s closing remarks

Speakers

Select a speaker to learn more

Back
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:

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

2024-11-19, 11:30 AM

View In Agenda
Next speaker
Back
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:

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

2024-11-19, 11:30 AM

View In Agenda
Next speaker
Back
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, 2:15 PM

View In Agenda
Next speaker
Back
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, 5:15 PM

View In Agenda
Next speaker
Back
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.

Session Details:

Insights to drive smarter decisions: What is strategically needed to achieve trial results in rare disease studies?

2024-11-20, 10:30 AM

View In Agenda
Next speaker
Back
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, 1:45 PM

View In Agenda
Next speaker
Back
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).

Next speaker
Back
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:

Clinical trials in rare neuroimmunology diseases

2024-11-20, 3:45 PM

View In Agenda
Next speaker
Back
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.

Next speaker

Plan Your Visit

Venue

Sheraton Brussels Airport Hotel Brussels National Airport Brussels Belgium 1930

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 and events attendee lists. For the avoidance of doubt, Arena will only share attendee lists with carefully selected third party partners for event registration purposes. Whilst Arena International Events Group pursues third party agencies 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:

The Brussels National Airport train station is located right underneath the airport, with trains departing to Brussels Central Station 6 times per hour from 5:30am to 11:30pm. These trains also stop at North Station and continue to South Station, while connections to the European District leave twice per hour. Guests can park their cars in the secured and covered parking of the hotel, which is accessible from the 5th floor of Front Park 2 at a Special Rate of 32 €. OR There is the Brussels Airport Interparking which is located next to the hotel. The price would be 24 € per day. The same P2 parking must be used in order to access the airport parking. Click here for directions: google.com/maps

Sponsors

Select a sponsor to learn more

SESSION SPONSORS

EXHIBITORS

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

MEDIA PARTNERS

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

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