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 In cancer clinical trials, it is best to measure patient-reported outcomes uniformly, according to a new draft guidance from the FDA.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 In cancer clinical trials, it is best to measure patient-reported outcomes (PROs) uniformly, according to a new draft guidance from the FDA.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.
Conversations With Perry and Friends
April 14th 2025Perry Cohen, Pharm.D., a longtime member of the Managed Healthcare Executive editorial advisory board, is host of the Conversations with Perry and Friends podcast. His guest this episode is John Baackes, the former CEO of L.A. Care Health Plan.
Listen
Breaking Down Health Plans, HSAs, AI With Paul Fronstin of EBRI
November 19th 2024Featured in this latest episode of Tuning In to the C-Suite podcast is Paul Fronstin, director of health benefits research at EBRI, who shed light on the evolving landscape of health benefits with editors of Managed Healthcare Executive.
Listen
Medicaid Expansion Linked To Timelier Lung Cancer Surgery and Access to High-Volume Hospitals
April 17th 2025New research shows that Medicaid expansion was linked to a 2.1% increase in timely lung cancer surgeries and a 2.8% rise in procedures at high-volume hospitals, highlighting how policy changes can impact cancer care access.
Read More