Clinical Development & Regulatory

 

Clinical Development & Regulatory, Wednesday 24 April 2024

Melissa Penn
Clinical Development & Regulatory
11:24

Chair's Remarks

Melissa Penn, Director Patient Engagement Research & Development, Bayer
Melissa Penn
Clinical Development & Regulatory
11:24

Chair's Remarks

Melissa Penn, Director Patient Engagement Research & Development, Bayer
Clinical Development & Regulatory
11:25

The pivotal role patient advocacy organizations play in driving rare disease therapeutic development

  • Understand how patient advocacy organizations drive therapeutic development in the rare disease space
  • How to effectively engage and partner with patient advocacy organizations to develop meaningful treatments
  • Gain insight into the “how, when and where” patient advocacy can significantly impact the drug development process
Juliane Mills
Clinical Development & Regulatory
12:05

Uniting for the uncommon: Successes and lessons in pharma-patient advocacy partnerships

What the audience will learn:

  • This will be a transparent discussion on the types of collaborations and agreements that have worked (or not worked) between rare disease patient advocacy groups and drug development companies.
  • Learn what you’re doing right (or wrong!) when trying to build bridges with advocacy organizations.
  • Learn key questions to ask about advocacy organizations and drug companies before starting a partnership.
Rahul Kukreja
Clinical Development & Regulatory
12:25

Navigating cell and gene therapy studies in rare diseases

  • Site Identification and Competition
  • Overcoming Interdepartmental Challenges
  • Logistics Management
  • The Impact of Box Warnings

 

Richard Wilson
Clinical Development & Regulatory
12:45

The critical role of partnerships in rare disease drug advancement: From lab bench to bedside

Rachael Sawaya
Clinical Development & Regulatory
13:05

Mobile visits are critical for successful rare disease research

Jennifer McNary
Clinical Development & Regulatory
14:59

Chair's Remarks

Jennifer McNary, Founder, One Rare
Panel discussion
Clinical Development & Regulatory
15:00

Patient-first innovation: FDA shifting rare diseases endpoints

Jamie Sullivan, Vice President of Public Policy, EveryLife Foundation for Rare Diseases
Lavni Varyani
Clinical Development & Regulatory
15:40

The future of rare disease funding: Saudi Arabia's investment wave

Lavni Varyani, Founder, Pharma Business Partners
Panel discussion
last published: 20/Apr/24 16:35

Clinical Development & Regulatory, Thursday 25 April 2024

Carolyn Schwartz
Clinical Development & Regulatory
11:59

Chair's Remarks

Panel discussion
Clinical Development & Regulatory
12:00

Putting science first in rare disease studies

Anthy Tsatoumas, Manager, Project Management, Veristat
Mridula Shukla
Clinical Development & Regulatory
12:40

Evolving trends in gene therapy

Mridula Shukla
Clinical Development & Regulatory
12:40

Evolving trends in gene Therapy

Carolyn Schwartz
Clinical Development & Regulatory
15:14

Chair's Remarks

Adrian Kielhorn
Clinical Development & Regulatory
15:35

Measuring diversity of patient in clinical trials: Do we have the right measures to measure the right things?

David Allison
Clinical Development & Regulatory
15:55

Collaboration: the TREAT-NMD model bringing therapies to patients with a rare disease

David Allison, Chief Executive Officer, TREAT-NMD
Carolyn Schwartz
Clinical Development & Regulatory
16:15

Developing patient-reported outcomes for orphan disease: Challenges and solutions

last published: 20/Apr/24 16:35

 

Get Involved At World Orphan Drug Congress USA

 

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