Patient Preference and Adherence (Jun 2023)
Development of a Specific Variant of Patient Benefit Index (PBI) Assessing Patient Needs, Goals and Benefits in Rosacea Treatment
Abstract
Matthias Augustin, Rachel Sommer, Christine Blome, Natalia Kirsten, Anna Langenbruch German Center for Health Services Research in Dermatology (CVderm), Institute for Health Services Research in Dermatology and Nursing (IVDP), University Medical Center Hamburg-Eppendorf (UKE), Hamburg, GermanyCorrespondence: Matthias Augustin, German Center for Health Services Research in Dermatology (CVderm), Institute for Health Services Research in Dermatology and Nursing (IVDP), University Medical Center Hamburg-Eppendorf (UKE), Martinistraße 52, Hamburg, 20246, Germany, Tel +49 40 7410 55428, Fax +49 40 7410 55348, Email [email protected]: Evaluation of patient-reported outcomes including health-related quality of life (HRQoL) and perceived benefits from treatment has become a fundamental component of medical decision-making. Standardized evaluation of treatment benefits in rosacea based on patient preferences is still lacking.Objective: Development and validation of an instrument for recording patient-defined benefits in rosacea therapy based on the Patient Benefit Index (PBI) methodology.Patients and Methods: In an open survey of n = 50 patients, potential benefits of therapy from the patient’s perspective were examined. The generated item pool was combined with pre-existing PBI items for other skin conditions and reviewed by an expert panel of dermatologists, psychologists and patients. Items were condensed to n = 25 and converted into a Likert-scaled questionnaire. The validity and feasibility of the resulting Patient Benefit Index for rosacea (PBI-RO) were tested on individuals with rosacea recruited from a German rosacea patient organization.Results: N = 446 patients with rosacea completed the PBI-RO. The internal consistencies measured by Cronbach’s alpha were high (Patient Needs Questionnaire [PNQ] 0.94). Mean PBI-RO was 1.9 ± 1.2 (scale from 0 = no benefit to 4 = maximum benefit), 23.5% of the patients experienced a PBI-RO < 1 (no clinically relevant benefit). The PBI-RO correlated with HRQoL, health state, current extent of rosacea lesions and treatment satisfaction. The highest correlation was found between PBI-RO and satisfaction with previous treatment (r = − 0.59, p < 0.001); correlation with the extent of rosacea lesions was low (r = 0.16, p < 0.001).Conclusion: The PBI-RO shows satisfying internal consistency and construct validity. It offers the option of a patient-weighted evaluation of the therapeutic benefit of rosacea therapy and may add to more stringent goal orientation in therapy.Keywords: patient needs, treatment goals, disease burden, health-related quality of life, Patient Benefit Index, rosacea
