Challenge #1: Ensure feasibility and scientific rigor of trial design

Rare disease clinical trials: Foreseeing and overcoming the obstacles

Rare disease studies are not miniature versions of standard trials. They present unique obstacles, and Premier Research has the hands-on experience it takes to anticipate and overcome.

It may be a small study, but that doesn’t make it easy. Fifty rare disease patients require a tremendous amount of attention compared to 50 patients with a more common condition. Collecting meaningful data is also hard with a small number of patients, and when you’re working with small sample sizes, there is no room for error.

We often recommend adaptive trial design or companion diagnostics if the disease is genetic. Many sponsors include an expanded-access or compassionate-use program. It can be immensely productive to work with patient advocacy groups and medical networks. And you may have to work with only one patient at a given site — or in a given country. It’s difficult, but feasible — and we know how to make it work.

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