The “Patient-Reported Outcome Measures in Clinical Research” research paper describes the importance and also some limitations of the use of patient-reported outcome measures (PROMs) in clinical research.
During the last years, patient-reported outcomes (PROs) have gained relevance in clinical research. As a matter of fact, PROs often capture information about patients’ experiences with their disease or treatment that cannot be captured any other way, being quicker, less burdensome, and less costly than clinical measures.
On the other hand, PROMs are reports of the “the status of a patient’s health condition that come directly from the patient without interpretation of their response by a clinician or anyone else” and are assessed using standardized questionnaires that collect the feelings and functioning abilities of the patients as well as the effects of the interventions being investigated in clinical research.
Despite being a valuable tool in clinical research, PROMs have some limitations, since they cannot be used for patients who cannot reliably self-report their own health experiences (e.g., someone who is too young). In those cases, measures based on caregiver or clinician reports should be considered. Furthermore, PROMs can be influenced by respondents’ expectations, leading to biased estimated of treatment effects.
Incorporating PROMs into clinical research requires careful consideration of many issues, including the mode of administration (e.g., tablet, computer), prevention of missing data, cultural or linguistic relevance of PROMs in multinational studies, and construction of well-defined end points.
According to Dr. Giorgio Reggiardo, senior biostatistician and head of CVBF’s Biostatistics Unit “when designing a clinical study, it is important to consider that PROMs may be less familiar than other types of outcomes to clinicians. Considering that the PROMs design will increase in the near future, it is likely to require greater engagement of clinicians so that collection of PROMs becomes more a part of clinical routine. Poor quality trial design refers to the lack of PRO-specific methodological rigor during the design phase and can have a direct impact on the quality of the trial results.”
Moreover “PROMs require active input from the patient or respondent, and it is necessary to consider that the response rate can be influenced by a number of factors (response bias). Furthermore, there is evidence that response rates have deteriorated over time and the PROMs questionnaires may be returned incomplete. From a methodological point of view, response bias can be detected if important characteristics of patients are associated with differential response rates”, says Dr. Reggiardo.
More information about this paper can be found here.