Abstract
ObjectivesSleep disturbance is a common experience in fibromyalgia (FM). The field lacks a sleep specific patient reported outcome (PRO) measure developed and validated in a FM population. The study objective is to gain an in-depth understanding of sleep in FM and to develop a PRO measure of it.MethodsResearch involved the following stages: 1) A literature review conducted to identify key concepts associated with FM patient experience of sleep and PRO measures that have been used to assess this; 2) Qualitative interviews with therapeutic area experts; 3) Focus groups with FM patients who experienced sleep disturbance; 4) Development of a conceptual framework and the Fibromyalgia Sleep Diary (FMSD); and 5) Cognitive interviews with patients to explore content validity of the FMSD.ResultsThe literature review and expert interviews supported sleep disturbance being an important aspect of the FM patient experience, and underscored the need for a new FM specific sleep PRO measure. Results from the focus groups demonstrated that FM patients experience sleep disturbances that they attribute to their FM symptoms, such as pain and stiffness, confirming the importance of understanding more about sleep changes. Aspects of sleep raised by FM patients included poor sleep quality and insufficient quantity including difficulty with falling asleep, getting comfortable, and staying asleep; restlessness; light sleep; not feeling rested upon awakening; and difficulty starting the day. Cognitive interview results showed that the 8-item FMSD, developed to reflect the concepts identified above, was relevant to FM patients with content that was interpreted as intended.ConclusionsThe FMSD was developed in line with the recommendations of the FDA PRO guidance and ISPOR PRO Task Force. The qualitative evidence generated thus far strongly supports the content validity of the FMSD as a PRO measure of sleep disturbance in FM populations. Psychometric evaluation of the FMSD to demonstrate reliability, validity and sensitivity to change is recommended as a next step.
Highlights
Introduction and backgroundFibromyalgia (FM) is characterized by chronic widespread pain and tenderness [1]
The Food and Drug Administration (FDA) issued a guidance document describing the necessary evidence for a Patient Reported Outcome (PRO) instrument to support label claims [5], which includes the evaluation of content validity as a key recommendation
These searches were supplemented by a review of FM or sleep-specific patient reported outcome (PRO) measures listed in the PRO research database PROQOLID, as well as a review of clinical trials that targeted adult FM patients, assessed sleep outcomes, and were listed in the clinical trial search engine provided by the U.S National Institutes of Health (NIH; http://clinicaltrials.gov)
Summary
Introduction and backgroundFibromyalgia (FM) is characterized by chronic widespread pain and tenderness [1]. The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) PRO Task Force published five “good research practices” that are crucial steps in establishing and documenting the evidence of content validity for new PRO instruments [6]. These are: 1) identification of the PRO measurement concept and the context of use; 2) development of the qualitative concept elicitation protocol; 3) qualitative concept elicitation data collection among the population of interest (i.e. a pre-specified patient population); 4) analysis of the qualitative data; and 5) documentation of methods used and results of the validity assessment of the content of the PRO measure (e.g., instructions, items, response options, recall period). Current standards focus on the need for concrete evidence of the direct link between the patient perspective and the concept and item coverage in a PRO measure
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