Inclusion of patient-reported outcome measures in registered clinical trials: Evidence from ClinicalTrials.gov (2007-2013)

Background: Patient reported outcomes (PROs) have gained a prominent place in clinical research. Previous estimates suggest that PRO measures are used in 14% of clinical trials. Online registries, such as ClinicalTrials.gov, may be useful in evaluating extent of PRO use.

Purpose: To estimate the proportion of clinical trials using at least one PRO measure and to examine associations between trial characteristics and use of PRO measures.

Methods: A copy of the ClinicalTrials.gov database was made, containing all data from November 2007 to December 2013. Content was searched for use of PRO measures. Multivariable logistic regression was used to investigate possible associations between trial-level characteristics and use of PRO measures.

Results: Of 96,736 registered trials, 26,337 (27%) were identified as using one or more PRO measures. Among oncology trials, 29% (3947/13,584) were identified as using a PRO measure, compared to 27% (22,390/83,152) of non-oncology trials. A greater proportion of trials using PRO measures were more likely to be sponsored by university/research organizations (29%) or the US government (33%), compared to industry (22%); Phase III (35%); non-randomized (32%); and evaluating devices (30%), procedures (31%) or behaviors (50%), compared to drugs (24%). Fewer were FDA-regulated (23%).

Conclusions: Evidence suggests that between 2007 and 2013, there has been an increase in the number of trials that use a PRO measure, particularly in oncology trials. With initiatives such as the Patient-Focused Drug Development and FDA PRO Guidance, the trend in the use of PRO measures in clinical research will likely increase further.