Clinical Trials in Rare Diseases 2023

We are pleased to announce the launch of a brand new event: Clinical Trials in Rare Diseases will take place on 13th-14th September 2023 in Princeton!

13 - 14

September

2023
  • Princeton, New Jersey
  • Complimentary
  • Why attend?
  • Agenda
  • Speakers
  • Sponsors
  • Media Centre
  • Contact Us
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Why attend?

A FANTASTIC NETWORK OF YOUR PEERS AND COLLEAGUES

With over 40 solution providers the virtual meeting 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

  • 7 Dec 2022
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EUROPE – TIMES IN EUROPEAN CENTRAL TIME

8:55 AM

Chair’s opening remarks

9 AM

Enrolling and engaging patients in a rare disease clinical study

  • Best practice for thoughtful and engaged recruitment for a rare disease study
  • Developing a more individualized approach to recruitment and retention, and the importance of this when running a study on smaller patient populations
  • Working closely with patient advocacy groups as part of your recruitment strategy

Speakers

9 AM

OPENING KEYNOTE: A patient perspective on participating in a rare disease clinical trial

  • An overview of participating in a clinical trial as a patient: what can sponsor companies do to improve the patient experience?
  • Understanding the financial burden that clinical trials have on patients
  • The importance of working closely with patients and advocacy groups throughout your trial, and best ways to approach this

Speakers

9:30 AM

Reserved for Premier Research

9:30 AM

Reserved for Premier Research

10 AM

Running a successful rare disease clinical trial: key considerations and best practice

  • Overview: understanding challenges specific to rare disease clinical trials
  • Finding the right vendors: ensuring your partners are equipped to handle a rare disease clinical study
  • Keeping costs for rare diseases and orphan drugs low while innovating trials

Speakers

10 AM

Targeted Personalized Cancer Therapy: The Holy Grail of Cancer Treatment

  • An overview of Andarix’s technology to target lung and pancreatic cancer
  • Emphasising how to incorporate flexibility into rare disease trials from the beginning
  • Developing personalized cancer treatments: challenges and opportunities
  • Shipping products from lab to patient: what is required?
  • Results from Phase 1 studies and plans for Phase 2
  • Identifying national and international partners to make trials a success

10:30 AM

Reserved for THREAD

10:30 AM

Available for sponsor

11 AM

CASE STUDY: Challenges and learnings from running a clinical trial in brain protection

  • Developing therapeutics for rare diseases: the case of the ADNP syndrome and davunetide
  • Exploring the tools, technologies and data management techniques used to streamline the process
  • Pitfalls encountered and how they were overcome
  • Advice on vendor negotiation and management – how to ensure you are working with a partner who can cater to your specific trial needs

Speakers

11 AM

The movement towards decentralized clinical trials in 2023

We welcome GlobalData to present their research into the state of the Biopharmaceutical Industry in 2023. Including:

  • A review of how big pharma have reacted to the trend: who is going virtual with their trials?
  • Supply chain concerns due to COVID-19
  • Predictions for the future

Don’t miss this talk; it is crucial for the biopharmaceutical industry to recognize the factors shaping the clinical trial landscape in order to maximize their future success.

Speakers

11:30 AM

Available for sponsor

11:30 AM

Available for sponsor

12 PM

END OF CONFERENCE

12 PM

BREAK – VISIT EXHIBITION HALL

Sessions resume at 9am US Eastern Time/3pm EU time

US – TIMES IN US EASTERN TIME

Speakers

Select a speaker to learn more

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Michele Rhee
Vice President, Patient Affairs and Advocacy, X4 Pharma

Session Details:

OPENING KEYNOTE: A patient perspective on participating in a rare disease clinical trial

2023-09-13, 9:00 AM

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Stephen Partridge
VP of Clinical Development, Galecto inc

Session Details:

Running a successful rare disease clinical trial: key considerations and best practice

2023-09-13, 10:00 AM

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Illana Gozes
Professor of Clinical Biochemistry/Chief Science Officer, Tel Aviv University/ ATED Therapeutics Ltd.

Professor of Clinical Biochemistry the Lily and Avraham Gildor Chair for the Investigation of Growth Factors, Head of the Elton Laboratory for Molecular Neuroendocrinology, Department of Human Molecular Genetics and Biochemistry, Sackler Faculty of Medicine, Sagol School of Neuroscience and Adams Super Center for Brain Studies, Tel Aviv University. Former Director, the Adams Super Center for Brain Studies & the Edersheim Levie-Gitter Institute for Functional Brain Imaging. Serves as Editor-in-Chief of the Journal of Molecular Neuroscience. The inventor of CP201 and CP102. Holds a B.Sc. from Tel Aviv University and a Ph.D. from the Weizmann Institute of Science.

Session Details:

CASE STUDY: Challenges and learnings from running a clinical trial in brain protection

2023-09-13, 11:00 AM

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Halley Losekamp
Director, Patient Enrollment, AGTC

Session Details:

Enrolling and engaging patients in a rare disease clinical study

2023-09-13, 9:00 AM

View In Agenda
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Scotty Chung-Siu
Senior Analyst, GlobalData Healthcare

Session Details:

The movement towards decentralized clinical trials in 2023

2023-09-13, 11:00 AM

View In Agenda
Next speaker

Sponsors

Select a sponsor to learn more

Session Sponsor

Exhibitor

Media Centre

MEDIA PARTNERS

Become A Media Partner

A FANTASTIC NETWORK OF YOUR PEERS AND COLLEAGUES

 

With over 40 solution providers the virtual meeting 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

Jaz Sidhu

Senior Sponsorship Manager

+44 (0) 20 7947 2755

SPEAKING OPPORTUNITIES

Louisa Manning

Programme Director

louisa.manning@arena-international.com

MARKETING ENQUIRIES

Moona Popal

Marketing Manager

SPONSORSHIP ENQUIRIES

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

Jaz Sidhu

Senior Sponsorship Manager


+44 (0) 20 7947 2755

SPEAKER ENQUIRIES

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

Louisa Manning

Programme Director


+44 (0) 207 661 0906