BioTalk Unzipped

Rare disease clinical trials present a problem unlike almost anything else in drug development: how do you run a rigorous trial when the patients you need may be exceptionally difficult to find?

Scott Schliebner joins BioTalk Unzipped to explore rare disease development, clinical research, artificial intelligence, and the operational realities of building organizations across international boundaries.

The episode begins with a discussion of Rare Givers, an organization supporting caregivers in the rare disease community.

Scott then explains what led him into clinical research before the conversation turns to the Epitomee weight-loss capsule and the changing landscape of obesity treatment.

From there, Scott shares lessons from building North American infrastructure for a European MedTech-focused CRO, including the cultural differences that can influence how global teams work together.

The discussion then moves into AI and machine learning. Rather than treating AI as a buzzword, Scott, Gregory, and Chad examine practical applications across clinical trials, patient recruitment, data analysis, quality, and regulatory compliance.

Finally, Scott breaks down why rare disease trials are so challenging and why successful development often requires collaboration among patients, caregivers, investigators, sponsors, and other stakeholders.

Chapters

00:53 - Introduction and charity donation to Rare Givers

02:30 - The mission of Rare Givers

04:08 - What inspired Scott to enter clinical research

06:12 - New News: The Epitomee weight-loss capsule

11:25 - Epitomee, targeted weight-loss approaches, and competition with Wegovy

14:05 - Building North American infrastructure for a European MedTech-focused CRO

16:35 - Cultural differences when doing business across countries

23:53 - AI, machine learning, and generative AI in clinical trials, drug development, FDA compliance, and quality

30:13 - Understanding rare disease trials and overcoming their unique challenges

33:12 - Is it a zebra or a horse?

Key takeaways

• Rare disease trials often require unusually sophisticated patient-identification and recruitment strategies.
• Caregivers are an essential part of the rare disease ecosystem.
• AI may improve patient recruitment, data analysis, and other areas of clinical research, but implementation must be thoughtful.
• Building international clinical-development organizations requires understanding cultural as well as operational differences.

Rare Givers

Learn more or donate:

https://www.raregivers.global/

Connect with Scott Schliebner

https://www.linkedin.com/in/scott-schliebner-1087789/

Connect with the hosts

Dr. Chad Briscoe

https://www.linkedin.com/in/chadbriscoe/

Gregory Austin

https://www.linkedin.com/in/gregoryaustin1/

New News

Epitomee announces filing of a novel weight-loss capsule for FDA clearance:

https://www.prnewswire.com/news-releases/epitomee-announces-the-filing-of-a-novel-weight-loss-capsule-for-fda-clearance-302081225.html

NIH publication, Safety, Tolerability and Efficacy of a Novel Self-Use Biodegradable Device for Management of Obesity:

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6700511/

Image credit

Rare Disease Day:

https://www.rarediseaseday.org/event/6th-annual-rare-disease-day-virtual-race/

#clinicaltrials #raredisease #AI #drugdevelopment #biotechnology