Case Studies: How Patient Input Is Changing R&D

US FDA's patient-focused drug development meeting on sickle cell disease inspired Global Blood Therapeutics to develop a new patient-reported outcomes instrument, while an externally led meeting on amyloidosis confirmed for Alnylam that its pivotal trial was studying clinically meaningful secondary endpoints.

The philosophy underpinning the patient-focused drug development (PFDD) movement – that it's important to find out what symptoms are most bothersome to patients, and what they would like to see in new treatments – is increasingly being embraced by drug developers as they plan their clinical programs for new compounds.

Below are two examples of how the growing attention to the importance of the patient voice, coupled with the US FDA's own PFDD initiatives, have impacted recent drug development programs.

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