Assessment of Patient Reported Outcomes in Chronic Versus Acute Disease: A Comparison of Study Design and Analytic Approaches

OBJECTIVES: The assessment of patient reported outcomes (PRO) in new drug development has been systematically aligned to study visits using recall periods such as daily diary, weekly, or monthly reports. In chronic disease, this approach has little implication in the evaluation of change as the patient is often in a continuous health state so the derived endpoint can be scored without consideration of an event. However, in acute disease, which is generally event driven, systematic collection of PROs may pose a challenge as the event will likely contribute to immediate changes in the patient’s health state. In acute conditions, it is important to consider both the general state of patient functioning as well as the impact of unexpected events such as disease exacerbations or flares, adverse events, treatment side effects, and potential use of rescue medication to get a better understanding of a patient’s experience during the course of a clinical trial.

METHODS: This research presents common PRO assessment schedules as well as considerations for a variety of chronic and acute diseases to provide guidance for collecting and analyzing patient outcomes in chronic and acute conditions.

RESULTS: Guidance will include the presentation of schedule of assessments and analytic methodologies for assessing the temporal relationship between acute events and change in patient outcomes, with examples from depression, dysphagia, and pain conditions.

CONCLUSIONS: In conclusion, this research offers PRO measurement study design and analytic methodologies to address shortcomings of current approaches by providing potential solutions to improve the ability to quantity treatment efficacy in chronic and acute disease.