J. Stokes

Development of a Questionnaire to Assess the Impact of Chronic Low Back Pain for Use in Regulated Clinical Trials

BackgroundChronic low back pain (CLBP) is the most common chronic pain condition and is associated with clinical, economic, social, and public health impacts. The effect of CLBP on patients’ health-related quality of life (HRQoL) is significant. The symptoms and impacts most often associated with CLBP include pain and disability; patients most affected are often crippled by the condition. CLBP also affects patients’ mental, physical, and psychosocial well-being. A variety of self-report measures have been developed for the assessment of CLBP, such as the Roland Morris Disability Questionnaire (RMDQ); however, existing measures may not meet current regulatory expectation for the development, documentation, and use of patient-reported outcomes (PRO) questionnaires (U.S. Department of Health and Human Services, Food and Drug Administration, 2009).ObjectivesThis report describes the qualitative development of the Chronic Low Back Pain Impact Questionnaire (CLBP-IQ), created for use in clinical trials.MethodsA total of 22 CLBP patients recruited by clinicians participated in concept elicitation interviews to identify target measurement concepts. An instrument development team generated the instructions, items, and response options guided by patient input. Cognitive debriefing interviews were conducted with 21 patients recruited by the same clinicians who recruited for concept elicitation interviews. During cognitive interviews, a draft instrument composed of 28 items was presented to individuals with CLBP to evaluate its readability and comprehensiveness. All research activities were conducted in the US.ResultsDuring concept elicitation interviews, participants reported a variety of physical, emotional, and social impacts associated with CLBP. Participants also reported CLBP impacts on sleep, energy, daily activities, work, household activities, leisure activities, cognition, self-care, and sex life. Impacts deemed simple, important, and relevant to CLBP patients became targets of measurement for the CLBP-IQ. During cognitive debriefing, seventeen items were interpreted as intended by at least 90xa0% of participants, and no items were interpreted incorrectly by more than five patients (24xa0%). Additionally, seventeen items were experienced by at least 90xa0% of participants, and no single item was experienced by less than 67xa0% of participants (nxa0=xa014).ConclusionsThe CLBP-IQ was developed in accordance with current US Food and Drug Administration guidance on instrument development. Results from both concept elicitation and cognitive debriefing interviews support the content validity of the CLBP-IQ in patients with CLBP. Future development should proceed with psychometric evaluation.

Informing a priori Sample Size Estimation in Qualitative Concept Elicitation Interview Studies for Clinical Outcome Assessment (COA) Instrument Development

OBJECTIVEnEvidence-based recommendations for the a priori estimation of sample size are needed for qualitative concept elicitation (CE) interview studies in clinical outcome assessment (COA) instrument development. Saturation is described as the point at which no new data is expected to emerge from the conduct of additional qualitative interviews.nnnSTUDY DESIGNnA retrospective evaluation of 26 CE interview studies conducted with patients between 2006 and 2013 was completed to assess the point at which saturation of concept was achieved in each study.nnnMETHODSnFor each of the 26 interview studies, saturation of symptom concepts was assessed by dividing the sample into quartiles and then comparing the number of responses elicited from the first 25% of participants to the next 25% of participants, from the first 50% of participants to the next 25% of participants, and then from the first 75% of participants to the last 25% of participants. The number of interviews required to achieve saturation was documented for each study and then summarized across studies.nnnRESULTSnFindings indicate that 84% of symptom concepts emerged by the 10th interview, 92% emerged by the 15th interview, 97% emerged by the 20th interview, and 99% by the 25th interview.nnnCONCLUSIONSnResults provide practical guidance for estimating the number of interviews that may be needed to achieve saturation in a qualitative CE interview study for COA instrument development; address an important gap in qualitative research for the development of COAs in the context of medical product development; and offer useful information for study design and implementation.

Development and psychometric evaluation of the self-assessment of psoriasis symptoms (SAPS) – clinical trial and the SAPS – real world patient-reported outcomes

Abstract Objective: The Self-Assessment of Psoriasis Symptoms – Clinical Trials (SAPS-CT) and SAPS – Real World (SAPS-RW) were simultaneously created to assess the experience of plaque psoriasis in two unique contexts. Methods: Qualitative and quantitative research was conducted in four phases namely concept elicitation, questionnaire construction, content evaluation and psychometric evaluation. Results: Following concept elicitation, 18 concepts were selected to inform questionnaire construction of the SAPS-CT and SAPS-RW. To accommodate each context of use, the SAPS-CT asks respondents to rate the target symptom ‘at its worst’ in the 24u2009h prior to assessment, while the SAPS-RW asks respondents to rate the target symptom “on average” in the 7 days prior to assessment. Cognitive debriefing confirmed that patients could comprehend and provide meaningful responses to both versions and, after minor modifications, resulted in 11-item questionnaires administered in an observational study (Nu2009=u2009200). Results from the observational study informed further item reduction (SAPS-RW to six items and SAPS-CT to nine items) and demonstrated that scores from each were reliable (Cronbach’s αu2009>u20090.90, test–retest intraclass correlation coefficient >0.70), construct valid and able to differentiate among clinically distinct groups. Conclusion: The SAPS-CT and SAPS-RW are content-valid PRO questionnaires capable of producing psychometrically sound scores when administered chronic to plaque psoriasis patients.

Psychometric evaluation of the Rheumatoid Arthritis Symptom Questionnaire (RASQ) in an observational study

Abstract Objective: To describe the psychometric performance of the scores produced by the Rheumatoid Arthritis Symptom Questionnaire (RASQ), a new patient-reported outcome (PRO) questionnaire developed to assess the signs and symptoms of rheumatoid arthritis (RA). Methods: Adult subjects with clinically confirmed RA completed a set of questionnaires (including the RASQ) at an initial study visit (Day 1), and then completed the RASQ and the Patient Global Impression of Change (PGI-C) on their own on Day 8. Demographic and health data were summarized using descriptive statistics, and psychometric analyses were conducted, including: acceptability, item and scale distribution, reliability (internal consistency and test–re-test reliability), and construct-related validity (convergent validity and known-groups methods). Results: In total, 200 subjects (femalesu2009=u200961.5%; whiteu2009=u200972.0%; and age [mean]u2009=u200960.7 years) with RA were recruited across the US and included in the analysis. There were no missing data recorded for the RASQ, and scores were well distributed for both timepoints. The RASQ Total Symptom Score surpassed the threshold (αu2009≥u20090.70) for internal consistency at Day 1 (αu2009=u20090.967) and test–re-test score reliability (intra-class correlation coefficient [ICC]u2009>u20090.70) (ICCu2009=u20090.960). Convergent validity analyses demonstrated that the RASQ items and Total Symptom Score had high correlations (convergent validity) with other PRO questionnaires. Known-groups methods demonstrated that the RASQ (Total Symptom Score and all single items) can differentiate between clinically distinct groups. Conclusions: The RASQ is capable of producing psychometrically sound scores when administered to adults with RA.