Entwicklung von Instrumenten zur Erfassung eines Patientennutzens bei hellem Hautkrebs, Rheumatoider Arthritis und Multipler Sklerose sowie die Validierung des Instrumentes für die Multiple Sklerose

Background: In the context of treatment benefit evaluation as well as driven by health service research requirements patient-centeredness gets increasingly important. The objective was to develop assessment instruments of patient-relevant benefits in the disorders rheumatoid arthritis (RA), multiple sclerosis (MS) and non-melanoma skin cancer (NMSC) based on the established Patient Benefit Index (PBI) methodology and the validation of the instrument for MS. Methods: Based on a multi-step development approach comprising an open item collection and multidisciplinary expert panel with clinicians, methodologists and patients, the pilot-versions were investigated for applicability and comprehensibility in cognitive pretests. Subsequently the PBI-MS was evaluated regarding its convergent validity, reliability and feasibility in a non-controlled prospective validation study with n=100 patients. Results: Three standardized and disease-specific PBI versions were developed (PBI-NMSC-S S (sirurgical): 9 items; PBI-RA: 20 items and PBI-MS: 27 items). They capture patient-relevant benefits of treatment and collect physical, mental and treatment-specific aspects, but also treatment-goals regarding leisure, social and working life. In a longitudinal cohort study the convergent validity of the PBI-MS was shown. The highest correlation was found cross-sectional with patients global impression of change (PaGIC) on treatment benefit with most content match to the construct (r=0.60, p 0.001). Comparably high correlations were found with generic EQ-5D-3L and EQ-VAS (r=0.55, p 0.001; r=0.61, p 0.001), while the PBI-MS correlation to the disease-specific HRQoL instruments HALEMS and MS-QOL were unexpectedly found to be lower (r=-0.36, p 0.05, r=-0.49, p 0.001). The correlation with physicians global impression of change (PhGIC) was on moderate effect size (r=0,44, p 0,001). Reliability was high (Cronbachs A A /-=0,90) and multidimensionality of the PBI-MS was given. A low level of missing values and the results of the feasibility survey indicated comprehensiveness, acceptance and applicability. Conclusion: The PBI-MS is a valid instrument to capture patient-relevant benefits for relapse treatment suitable especially for use in clinical routine, but potentially also contributing in clinical or quality of care studies and patient-centeredness health care research. Due to their relevance and potential the PBI-NMSC-S and PBI-RA should also be tested concerning psychometric criteria in follow-up studies.