Acta Dermato-Venereologica 99-2CompleteContent | Page 19
INVESTIGATIVE REPORT
Measuring Patient Needs and Benefits in Dermatology using the
Patient Benefit Index 2.0: A Validation Study
Janine TOPP 1 , Matthias AUGUSTIN 1 , Kathrin VON USSLAR 1 , Ramona GOSAU 1 , Kristian REICH 2 , Michael REUSCH 3 and
Christine BLOME 1
1
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), 2 Dermatologikum Berlin and SCIderm Research Institute, and 3 Dermatological
Practice Tibarg, Hamburg, Germany
This study investigated the validity and feasibility of
the Patient Benefit Index 2.0 (PBI 2.0), a short in-
strument to assess patient-relevant treatment bene-
fit. In a cross-sectional study, patients with skin di-
seases completed the PBI 2.0 alongside instruments
on quality of life and disease-specific PBI long versions
to assess convergent validity. Feasibility questions
appraise comprehensibility, completeness, length, and
readability. Data from a longitudinal study were used
to explore responsiveness and test-retest reliability.
Most patients rated the PBI 2.0 easy to understand,
complete, legible, and not too long. The amount of
missing values was overall low. In all groups, except
for vitiligo, correlation analyses indicated good con-
vergent validity of PBI 2.0. Responsiveness of the PBI
2.0 could not be clearly confirmed. Retest-reliability
achieved satisfactory results. Thus, the PBI 2.0 may
be a suitable instrument for its use in different skin di-
seases. Its broad applicability allows for comparisons
across diagnosis groups.
Key words: patient-relevant benefit; patient-reported outco-
mes; validation; skin diseases.
Accepted Oct 11, 2018; E-published Oct 11, 2018
Acta Derm Venereol 2019; 99: 211–217.
211
Corr: Janine Topp, German Center for Health Services Research
in Dermatology, Institute for Health Services Research in Der-
matology and Nursing, University Medical Center Hamburg-
Eppendorf, Martinistr. 52, DE-20246 Hamburg, Germany.
E-mail: [email protected]
P
atients and healthcare professionals assess patients’
health state differently, and assessment results often
do not coincide (1–3). Therefore, in order to evaluate
treatment benefit comprehensively, the evaluation of
the patients’ perspective should complement objective
measures and outcomes. This demand for patient-relevant
benefit assessment is increasingly recognized in clinical
encounters as well as in research.
Very commonly, patient-relevant benefit is determined
using questionnaires on health-related quality of life
(HRQoL), asking patients to rate their impairments due to
the disease before and after treatment. A change in HRQoL
is interpreted as patient-relevant treatment benefit. This
prospective approach, however, is susceptible to recalibra-
tion response shift (4), a bias due to changes in the patients’
interpretation of the questionnaire and its response scale
SIGNIFICANCE
Considering the patients’ perspective in clinical decision-
making is a major goal in healthcare. To assess the patient
perspective, valid and reliable instruments on individual
needs and treatment benefits are needed. In the dermato-
logical setting, the Patient Benefit Index (PBI) can be used
to evaluate patient-relevant treatment benefit. This study
analyses and confirms the reliability, feasibility and validity
of a short version of the PBI, the PBI 2.0, which is applicable
for patients with different skin diseases. The brevity of the
instrument may foster its implementation in clinical practice.
(5, 6). This bias is present only in repeated measurements,
assessing changes over time. It is not present in retrospec-
tive benefit assessment, taking place after treatment only.
While potentially being susceptible to recall bias (6), the
retrospective assessment has the advantage of measuring
benefit close to the actual perception of the patient. This di-
rect rating of benefit by the patient is crucial when assessing
treatment satisfaction and estimating treatment adherence
(7). Such retrospective approach is taken by the questionn-
aire Patient Benefit Index (PBI). Furthermore, the PBI has
the advantage that, in addition to a benefit assessment, it
assesses the importance the individual patient assigns to
different treatment benefits and therefore enables the cal-
culation of an importance-weighted global benefit score.
The PBI is based on earlier approaches to individualized
benefit measurement. One of these approaches is the goal
attainment scaling (8), which comprises the formulation of
individualized treatment goals with each patient. Another
approach is the goal-oriented outcome measurement (9),
which includes the determination of individual patient-
relevant treatment goals in the field of rehabilitation.
The PBI consists of 2 parts: in the first part, patients rate
the perceived importance of different treatment goals listed
in the Patient Needs Questionnaire (PNQ). In the second
part, the Patient Benefit Questionnaire (PBQ), patients
rate the achievement of these treatment goals. Usually, the
PNQ is completed before starting a new treatment, and the
PBQ at a time when an effect of the treatment is expected
and treatment benefit will be assessed (10).
Thus far, a range of different PBI instruments have been
developed and validated for various skin diseases. A short
version of the PBI, the PBI 2.0, has been developed based
This is an open access article under the CC BY-NC license. www.medicaljournals.se/acta
Journal Compilation © 2019 Acta Dermato-Venereologica.
doi: 10.2340/00015555-3063
Acta Derm Venereol 2019; 99: 211–217