3rd Annual Clinical Trials in Rare Diseases 2025

Clinical Trials in Rare Diseases will take place on 17th-18th September 2025!

17 - 18

September

2025
  • Marriott Princeton at Forrestal, Princeton, USA
  • Complimentary
  • Why attend?
  • Agenda
  • Speakers
  • Plan Your Visit
  • Event Gallery
  • Sponsors
  • Media Centre
  • Why partner?
  • Contact Us
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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.

100+

Attendees

30+

Exhibitors

100+

Attendees

30+

Exhibitors

See What It's All About

Agenda

  • 17 Sep 2025
  • 18 Sep 2025
Expand All

8 AM

Registration and refreshments

8:50 AM

Chairperson’s opening remarks

Speakers

John Seman
Chief Executive Officer, REVITALE Pharma

9 AM

Case Study: Cutting edge technology – using AI in rare diseases

  • Sharing insights on how to use AI to bring drugs to rare disease patients
  • Running through step by step processes including pre IND, FDA work, organising and conducting trial
  • Discussing challenges, successes and real-world experience

Speakers

Bruce Bloom
Chief Collaboration Officer, Healx

9:30 AM

Reserved for event sponsor

10 AM

Case study: Tackling patient recruitment to ensure your trial is delivered on time

  • Considering ways to boost recruitment through investigator meetings and recruitment workshops
  • Communicating effectively and working together with sites to assist in recruitment
  • Sharing recruitment challenges and successes from a rare disease trial that started during Covid-19 pandemic

Speakers

Jamie Switzer
Associate Director Clinical Operations, Insmed

10:30 AM

Reserved for event sponsor

11 AM

Morning refreshments and networking

11:30 AM

Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

  • Finding innovative solutions for patient engagement
  • Collaborating with advocacy groups, patient organizations and care givers to extend patient reach
  • Utilizing decentralized trials with remote visits and monitoring to reduce patient burden where possible

Moderator: Anka Ehrhardt, Director Cell-Based Assays / Analytical Research & Development, Merck

Speakers

Anka Ehrhardt
Director Cell-Based Assays / Analytical Research & Development, Merck
Peter Covitz
Executive Director, Head Digital Innovation, Data Generation & Managed Access, Alexion
Jamie Switzer
Associate Director Clinical Operations, Insmed
Gurdyal Kalsi
Chief Medical Officer, Asklepion Pharmaceuticals

12:15 PM

Reserved for event sponsor

12:45 PM

Running decentralized rare disease trials: challenges and benefits

  • Discussing processes and procedures of a DCT model
  • Comparing outcomes of a DCT model versus non DCT model
  • Sharing benefits and lessons learned from experiences within rare disease DCTs

Speakers

Maria Dunaway-Bryant
Director Clinical Operations - Neuroscience & Oncology, Jazz Pharmaceuticals
Jennifer Oertli
Senior Clinical Trials Manager, Jazz Pharmaceuticals

1:15 PM

Lunch and networking

2:15 PM

Navigating clinical trials for rare diseases: hurdles and opportunities

  • Best practice in ensuring your rare disease trial is a success
  • Using precision medicine in clinical trials to advance therapeutics for rare diseases
  • Overcoming limited data sets and patient pools

Speakers

Gurdyal Kalsi
Chief Medical Officer, Asklepion Pharmaceuticals

2:45 PM

Engaging with advocacy groups to aid with trial design, patient enrolment and patient engagement

  • Using advocacy experience when designing trials with patient centric focus
  • Tapping into advocacy access to patient networks to spread information about trials
  • Sharing best practice in engaging advocacy groups early to drive recruitment and ensure trial success

Speakers

Eve Dryer
Board Member, NephCure

3:15 PM

Afternoon refreshments and networking

3:45 PM

Case Study: Sharing an approach to quality and clinical operations oversight in a rare disease trial at a small biotech

  • Deciding the right level of oversight: key considerations
  • Addressing communication hurdles and how to overcome them
  • Supporting sites for operational improvement and inspection readiness

Speakers

Amy DeWerth
Senior Director Regional Clinical Lead, Pharvaris
Vinay Edwin
Director Clinical Quality, Pharvaris

4:15 PM

Panel Discussion: Looking at the future of clinical trials in rare diseases: where are we now and where are we headed

  • Considering advances in trial designs and master protocols
  • Innovating through technology, digital health tools, AI and machine learning
  • Expanding collaborations and partnerships
  • Addressing ethical and diversity challenges
  • Discussing long-term vision in rare disease therapy development

Moderator: Anka Ehrhardt

Speakers

Anka Ehrhardt
Director Cell-Based Assays / Analytical Research & Development, Merck
Bruce Bloom
Chief Collaboration Officer, Healx

5 PM

Chairperson’s closing remarks

Speakers

John Seman
Chief Executive Officer, REVITALE Pharma

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

 

Roundtable 1: Sharing experiences and highlighting importance of decentralizing in rare disease trials

Leigh Dallow, Patient Advocate & Caregiver

Roundtable 2: Overcoming patient retention challenges in rare disease trials

Jenifer Waldrop, Executive Director, Rare Disease Diversity Coalition

 

Roundtable 3: Using new technologies in rare disease trials

Bruce Bloom, Chief Collaboration Officer, Healx

Speakers

Leigh Dallow
Patient Advocate & Caregiver
Jenifer Waldrop
Executive Director, Rare Disease Diversity Coalition
Bruce Bloom
Chief Collaboration Officer, Healx

10:30 AM

Morning refreshments and networking

11 AM

Participating and care giving in a rare disease trial: a family perspective

  • Understanding how living with a rare disease impacts decision on enrolling in a trial
  • Sharing experience of participating in trial from start to finish
  • Considering hurdles and barriers and what biopharma can do to relieve patient burden and improve access to studies

Speakers

Leigh Dallow
Patient Advocate & Caregiver

11:30 AM

Reserved for event sponsor

12 PM

Medically underserved populations in rare disease research

  • Discussing population inclusion and access to care and clinical trials regardless of diversity and socioeconomics, with a focus on improving patient care
  • Mental health and well-being: the emotional toll rare diseases take on patients and caregivers
  • Insights on the differences between represented and underrepresented populations on their barriers to care
  • Identifying realistic ways to improve opportunities for all patients

Speakers

Jenifer Waldrop
Executive Director, Rare Disease Diversity Coalition

12:30 PM

Lunch and networking

1:30 PM

Panel Discussion: Selecting a CRO for a rare disease trial: key considerations

  • Assessing experience in trials with small patient populations an recruitment challenges
  • Evaluating ability to adapt and innovate for unique needs of rare disease trials
  • Considering regulatory knowledge and patient access

Speakers

Bruce Bloom
Chief Collaboration Officer, Healx

2:15 PM

Fireside Chat: Contracting clinical sites for rare disease trials: accelerating timelines

  • Attracting an academic organization’s attention to prioritize your need
  • Considerations for contract negotiations and speeding up processing time scales
  • Building relationships and providing two-way support

Speakers

Kinjal Patel
Director Medical Affairs – Operations, Mitsubishi Tanabe Pharma America
Janine Sampong
Senior Manager Medical Affairs – Operations, Mitsubishi Tanabe Pharma America

2:45 PM

Development strategies for orphan-designated programs

  • Understanding regulatory pathways and incentives
  • Identifying clinical development challenges and solutions
  • Considering pricing, reimbursement and patient advocacy engagement

Speakers

Venkat Nelabhotla
Co-Founder, President & CEO, Vyome Therapeutics

3:15 PM

Afternoon break, networking and prize draw

3:45 PM

Case study: Developing a company and setting up rare disease trials: challenges and successes

  • Creating a company and ensuring the brand becomes known
  • Leveraging patient advocacy as part of enrolment strategy
  • Learnings from first trial challenges to improve in next study

Speakers

Niha Kamat
Senior Director Clinical Operations, Tenaya Therapeutics

4:15 PM

Overcoming challenges and optimizing a phase II rare disease trial using data and technology: a case study

  • Understanding successful protocol design trends and competitive activity in the rare disease space
  • Utilizing data analytics and benchmark models for assessing geographic feasibility, timelines, enrolment, and trials cost forecasting
  • Improving subject accrual and the site selection process
  • Execution phase and beyond – tracking investigators and competitive trials, mitigating potential complications

 

Senior Representative, GlobalData

4:45 PM

END OF CONFERENCE

Speakers

Select a speaker to learn more

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Peter Covitz
Executive Director, Head Digital Innovation, Data Generation & Managed Access, Alexion

Session Details:

Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

2025-09-17, 11:30 AM

View In Agenda
Next speaker
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Gurdyal Kalsi
Chief Medical Officer, Asklepion Pharmaceuticals

Session Details:

Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

2025-09-17, 11:30 AM

Session Details:

Navigating clinical trials for rare diseases: hurdles and opportunities

2025-09-17, 2:15 PM

View In Agenda
Next speaker
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Bruce Bloom
Chief Collaboration Officer, Healx

Dr. Bruce Bloom is Chief Collaboration Officer of Healx, a Cambridge UK biotech using AI and drug redevelopment to create novel therapies for rare disease patients.  He is the founder and former CEO of the global charity Cures Within Reach that brought over a dozen redeveloped drug therapies to patients through proof of concept clinical trials, and developed CureAccelerator®, the online drug redevelopment collaboration marketplace.  Dr. Bloom is an Ashoka Social Entrepreneur Fellow, the Patient Advisory Board Chair for the Institute for Translational Medicine, board member of the Drug Discovery Center and Chair of the Cancer Center Advancement Advisory Boards at the University of Illinois Chicago, member of the Vanderbilt Institute for Clinical and Translational Research External Advisory Board, Executive Board member of Mission: Cure, and is on the Science Advisory Boards of Rediscovery Life Sciences, the Dr. Ralph and Marian Falk Medical Research Trust Awards Programs, the Findacure Fundamental Disease Charity, the Rare Disease Research Hub of the Westchester Biotech Project, OneThree Biotech, and ReBootRx, a member of the IRDiRC Working Group on Rare Disease Treatment Access and the editorial board of ASSAY and Drug Development Technologies.

Session Details:

Case Study: Cutting edge technology – using AI in rare diseases

2025-09-17, 9:00 AM

Session Details:

Interactive Speaker-Hosted Roundtable Discussions

2025-09-18, 9:00 AM

Session Details:

Panel Discussion: Looking at the future of clinical trials in rare diseases: where are we now and where are we headed

2025-09-17, 4:15 PM

Session Details:

Panel Discussion: Selecting a CRO for a rare disease trial: key considerations

2025-09-18, 1:30 PM

View In Agenda
Next speaker
Back
Jamie Switzer
Associate Director Clinical Operations, Insmed

Session Details:

Case study: Tackling patient recruitment to ensure your trial is delivered on time

2025-09-17, 10:00 AM

Session Details:

Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

2025-09-17, 11:30 AM

View In Agenda
Next speaker
Back
Maria Dunaway-Bryant
Director Clinical Operations - Neuroscience & Oncology, Jazz Pharmaceuticals

Session Details:

Running decentralized rare disease trials: challenges and benefits

2025-09-17, 12:45 PM

View In Agenda
Next speaker
Back
Jennifer Oertli
Senior Clinical Trials Manager, Jazz Pharmaceuticals

Session Details:

Running decentralized rare disease trials: challenges and benefits

2025-09-17, 12:45 PM

View In Agenda
Next speaker
Back
Anka Ehrhardt
Director Cell-Based Assays / Analytical Research & Development, Merck

Dr. Ehrhardt is a biophysicist with a Ph.D. in human physiology. Throughout her career, she focused on driving biomedical research through efficient application of innovative technologies and strategies. She built and led successful teams in industry, including at Merck, where she headed a laboratory providing leading technologies for cell-based research from early discovery through manufacturing; and at BMS where Dr. Ehrhardt led and grew an international clinical assay team to robustly cover large scale registrational and translational clinical analysis generating clinical decision-driving data and mechanistic insights for immuno-oncology, cardiovascular, immunology, metabolic and rare disease studies. Before returning to Merck, Dr. Ehrhardt fulfilled one of her scientific aspirations at CHDI Foundation, designing and implementing successful clinical biomarker discovery and development strategies for Huntington’s disease. Currently, at Merck, Dr. Ehrhardt is providing strategic and technical leadership for the development and implementation of industry-leading and QC-friendly cell-based assays and models for potency determination that reflect the unique mechanisms of biologics products in clinical studies and beyond.

Session Details:

Panel Discussion: Overcoming patient recruitment challenges in rare diseases: what can we do to increase enrolment?

2025-09-17, 11:30 AM

Session Details:

Panel Discussion: Looking at the future of clinical trials in rare diseases: where are we now and where are we headed

2025-09-17, 4:15 PM

View In Agenda
Next speaker
Back
Kinjal Patel
Director Medical Affairs – Operations, Mitsubishi Tanabe Pharma America

Session Details:

Fireside Chat: Contracting clinical sites for rare disease trials: accelerating timelines

2025-09-18, 2:15 PM

View In Agenda
Next speaker
Back
Janine Sampong
Senior Manager Medical Affairs – Operations, Mitsubishi Tanabe Pharma America

Session Details:

Fireside Chat: Contracting clinical sites for rare disease trials: accelerating timelines

2025-09-18, 2:15 PM

View In Agenda
Next speaker
Back
Eve Dryer
Board Member, NephCure

Session Details:

Engaging with advocacy groups to aid with trial design, patient enrolment and patient engagement

2025-09-17, 2:45 PM

View In Agenda
Next speaker
Back
Leigh Dallow
Patient Advocate & Caregiver

Session Details:

Interactive Speaker-Hosted Roundtable Discussions

2025-09-18, 9:00 AM

Session Details:

Participating and care giving in a rare disease trial: a family perspective

2025-09-18, 11:00 AM

View In Agenda
Next speaker
Back
Amy DeWerth
Senior Director Regional Clinical Lead, Pharvaris

Session Details:

Case Study: Sharing an approach to quality and clinical operations oversight in a rare disease trial at a small biotech

2025-09-17, 3:45 PM

View In Agenda
Next speaker
Back
Vinay Edwin
Director Clinical Quality, Pharvaris

Session Details:

Case Study: Sharing an approach to quality and clinical operations oversight in a rare disease trial at a small biotech

2025-09-17, 3:45 PM

View In Agenda
Next speaker
Back
Paul Lupo
Senior Director Registry Operations, PTC Therapeutics
Next speaker
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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:

Interactive Speaker-Hosted Roundtable Discussions

2025-09-18, 9:00 AM

Session Details:

Medically underserved populations in rare disease research

2025-09-18, 12:00 PM

View In Agenda
Next speaker
Back
John Seman
Chief Executive Officer, REVITALE Pharma

John Seman is the CEO and Founder of REVITALE Pharma.  He began his career working in Big Pharma - Johnson & Johnson and Bristol Myers Squibb - in sales, marketing and business development.  The second half of his career was spent as a serial entrepreneur having Founded a half dozen companies that provided either products or services for the pharmaceutical industry.  John earned his bachelor's and master's degrees from the Massachusetts College of Pharmacy.

Session Details:

Chairperson’s opening remarks

2025-09-17, 8:50 AM

Session Details:

Chairperson’s closing remarks

2025-09-17, 5:00 PM

View In Agenda
Next speaker
Back
Niha Kamat
Senior Director Clinical Operations, Tenaya Therapeutics

Session Details:

Case study: Developing a company and setting up rare disease trials: challenges and successes

2025-09-18, 3:45 PM

View In Agenda
Next speaker
Back
Venkat Nelabhotla
Co-Founder, President & CEO, Vyome Therapeutics

Session Details:

Development strategies for orphan-designated programs

2025-09-18, 2:45 PM

View In Agenda
Next speaker

Plan Your Visit

Venue

Princeton Marriott at Forrestal 100 College Road East Princeton, New Jersey, USA, 08540

Accommodation

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Sponsors

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Session Sponsors

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Exhibitors

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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 Sales Manager

+44 (0) 20 7936 6870

SPEAKING OPPORTUNITIES

Ruth Atterbury

Clinical Trial Supply Portfolio Manager

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:

Nick McCudden

Commercial Director


+61 280 978 126

SPEAKER ENQUIRIES

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

Ruth Atterbury

Clinical Trial Supply Portfolio Manager