Episode: 48 - Florence Mowlem on the Challenges and Solutions of Pediatric Rare Disease Trials

How can electronic capture of clinical outcome assessments (eCOA) help with the unique challenges of pediatric rare disease trials? Florence Mowlem, chief scientific officer of uMotif, joins The Scope of Things to offer her expertise on eCOA, share advice for companies on vetting technology providers, and discuss where sponsors are tripping up when it comes to pediatric rare disease trials. Plus, host Deborah Borfitz brings you the latest news on a possible probiotic for preventing immune system disorders in babies, misunderstood mechanisms of body weight regulation, a promising primary care-based weight management program, GLP-1 drugs potentially benefitting the brain, how patient-reported outcomes can improve clinician adverse event reporting, and a discovery-to-reimbursement model for cell and gene therapies. 

Show Notes   

News Roundup 

Preventing allergies and asthma 

• Study in Nature Microbiology 
• News from the Technical University of Denmark 

Competing body weight regulation theories 

• Study in Nature Reviews Endocrinology 
• News on the Pennington Biomedical Research Center website 

PATHWEIGH weight management process 

• Pragmatic study in Nature Medicine 
• News on the University of Colorado Anschutz website 

Diabetes drugs reduce risk of dementia 

• Study in Drug Safety 
• News on McGill University website 

PRO data improved side effect grading 

• Study in The Lancet Oncology 
• Blog post on EORTC website 

Development model for cell and gene therapies 

• Research article in Molecular Therapy, Methods and Clinical Development 
• News on the Lund University website 

Guest 

Florence Mowlem, chief scientific officer at uMotif  

The Scope of Things podcast explores clinical research and its possibilities, promise, and pitfalls. Clinical Research News senior writer, Deborah Borfitz, welcomes guests who are visionaries closest to the topics, but who can still see past their piece of the puzzle. Focusing on game-changing trends and out-of-the-box operational approaches in the clinical research field, the Scope of Things podcast is your no-nonsense, insider’s look at clinical research today.