Standardize PROs, Says FDA Draft Guidance

MHE Publication, MHE July 2021, Volume 31, Issue 7

In carpentry, it is best to measure twice and cut once. In cancer clinical trials, it is best to measure patient-reported outcomes uniformly, according to a new draft guidance from the FDA.

In carpentry, it is best to measure twice and cut once. In cancer clinical trials, it is best to measure patient-reported outcomes (PROs) uniformly, according to a new draft guidance from the FDA.

“Heterogeneity of PRO measurement has lessened the “regulatory utility” of PRO data, says the guidance, which was issued in June. The document recommended sticking to five core PROs: disease-related symptoms, symptomatic adverse events, overall side effect summary measures, physical function and role function. Disease-related symptoms include pain, anorexia and fatigue. Neuropathy is an example of a symptomatic adverse event.

The overall adverse event measure can help investigators assess the tolerability of a medication and adjusts for some patients emphasizing certain adverse events more than others, the guidance says. Measuring “role function” is assessing the effect a treatment has on a patient’s ability to work and carry out daily activities.

None of this is uncharted territory, and the guidance points to existing scales and instruments for measuring the core PROs. The guidance also has some suggestions for how often patients should be asked to supply PROs and mentions taking into account the “response burden to patients.” The guidance says baseline assessments should be done to establish a reference point for change and that assessments ought to be done more often during the first few treatment cycles.