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Download: Case studies on Rare Disease and Pediatrics

In Clinical Development by Freya SmaleLeave a Comment

This guest blog post was provided by Premier Research.

We’ve done a lot of trials. In fact, we’ve done 100 pediatric and more than 90 rare disease trials in the past five years across multiple therapeutic areas. But what made them work? Why were they successful? More important, what best practices did we learn for the future?

We reviewed our most challenging studies to better understand how we addressed the rigors of pediatrics and rare diseases trials, and the strategies and tactics that proved successful.

Here are two that you might find interesting.

Recruitment: Challenge Accepted

Premier Research_Peds Dermatology_Blogpost

When a customer came to us to field a PIV pediatric study of a dermatology product previously approved for adult prescription, we knew going in that it would be the perfect recruitment nightmare.

We were looking for children aged two to 12 for a dermatology study that involved long visits and extensive blood draws. Of course, most parents didn’t want to have anything to do with it. Behind from the get-go, it was clear we needed to figure it out – quickly.

Read how we changed our approach to recruitment here.


Premier Research_Rare Diseases_BlogpostThe Elusive Protocol

When the regulatory requirements are different from country to country, finding the right protocols are a challenge. We needed to do a chart review of patients with an extremely rare metabolic bone disease, with a live birth incidence of about one in 100,000. And then settle on the protocol.

Find out how we found the right page – and kept everyone on it – here.

These are just two of our recent case studies – click here to see the rest and to discover how our experienced study teams can guide you through the clinical trial process.

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