A team of researchers from the University of Maryland School of Pharmacy say they were unable to find information about the development or validation of many of the patient reported outcomes (PROs) listed in the US Food and Drug Administration's (FDA) pilot Clinical Outcome Assessment (COA) Compendium.
In an article published
in Value in Health
earlier this month, the researchers also say they were only able to verify patient engagement in the development of one-third of the PROs listed in the COA Compendium that were reviewed for the article.
Development of the COA Compendium kicked off in 2016
as part of FDA's efforts to foster patient-focused drug development and is meant to serve as a resource for drugmakers and COA developers to facilitate the use of COAs in clinical trials.
According to FDA, there are four types of COA measures: PROs, clinician-reported outcomes (ClinRO), observer-reported outcomes (ObsRO) and performance outcomes (PerfO).
The COA Compendium
itself lists more than 100 COAs sourced from the Center for Drug Evaluation and Research's Drug Development Tool (DDT) Clinical Outcome Assessment Qualification Program and labeling from new molecular entities (NMEs) approved from 2003-2014.
However, FDA notes that the COA Compendium is not a comprehensive listing, nor does a COA's listing in the compendium indicate that it is endorsed by FDA or fit to be "the sole (or primary) determinant of a clinical benefit in a clinical trial."
Despite FDA's stated goal for the COA Compendium to foster patient-focused drug development, the authors say they were only able to verify patient engagement in the development of 9 of the 26 PROs they reviewed.
Of those, six were found to have engaged patients during concept elicitation and through cognitive interviewing. Of the remaining three, two only engaged patients during concept elicitation and one only engaged patients through cognitive interviews.
"There are concerns that COA Compendium-listed measures do not measure a concept of interest that reflects out-comes that matter to patients," the authors write.
For 12 of the PROs, the authors said they "were unable to identify information on development or validation" and for the remaining five, the authors were unable to find evidence of patient engagement, despite identifying some information about their development.
The authors attribute this to the fact that many of the COAs included in the compendium are proprietary and are not shared to protect commercial interests.
"For the FDA Pilot COA Compendium to fulfill its purpose of fostering [patient-focused drug development], it needs fine-tuning to reflect today's standards, improving transparency and facilitating clear identification of included measures so that the level of patient engagement, among other factors, can be properly assessed," the authors write.
The authors also say they are concerned that FDA's 2009 guidance Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims
is not being followed, or that PRO developers are not providing enough information to show whether the guidance was followed in published material for the PROs.
"Proper development of PRO measures is critical for all downstream [patient-focused drug development] activities. Without meaningful patient engagement in the development of PRO measures, the reliability and true value-add in patient-centered benefit-risk assessment, patient-centered value assessment, and health technology assessment become uncertain," the authors write.
Patient-Reported Outcome Measures in the Food and Drug Administration Pilot Compendium: Meeting Today's Standards for Patient Engagement in Development?