Arch Dermatol Res (2009) 301:561–571
DOI 10.1007/s00403-009-0928-8
ORIGINAL PAPER
The patient beneWt index: a novel approach in patient-deWned
outcomes measurement for skin diseases
Matthias Augustin · Marc A. Radtke · Ina Zschocke · Christine Blome ·
Julia Behechtnejad · Ines Schäfer · Michael Reusch · Volker Mielke ·
Stephan JeV Rustenbach
Received: 25 September 2008 / Revised: 19 January 2009 / Accepted: 22 January 2009 / Published online: 27 March 2009
© Springer-Verlag 2009
Abstract Evaluation of therapeutic beneWts from the
patient’s perspective is important in medical decision-mak-
ing and reimbursement. This study aimed at developing and
validating an instrument on patient-deWned needs and bene-
Wts in dermatology. The questionnaire was developed
according to international guidelines. The beneWt assess-
ment consists of two steps: before treatment, every patient
deWnes his treatment needs according to a standardized list.
After treatment, the patient rates the degree of beneWts
achieved. A “patient beneWt index” (PBI) is calculated by
averaging the preference-weighed results of all items. The
PBI questionnaire was validated in a sample of 500 patients
with ten skin diseases and in a treatment study on 906
patients with acne. The patients deWned a broad spectrum of
needs and treatment beneWts, indicating disease-speciWc
patterns. The PBI showed good feasibility, reliability
(Cronbach’s alpha >0.91) and construct validity, high
responsiveness, and discrimination between subgroups.
The PBI permits valid evaluation of patient-relevant bene-
Wts in dermatological treatment.
M. Augustin (&) · M. A. Radtke · C. Blome · J. Behechtnejad ·
I. Schäfer · S. J. Rustenbach
Department of Dermatology,
German Center for Health Services Research in Dermatology
(CVderm), University Clinics of Hamburg, Martinistr. 52,
20246 Hamburg, Germany
e-mail: m.augustin@uke.uni-hamburg.de
I. Zschocke
SCIderm GmbH Hamburg, Institute for Clinical Studies,
Stephansplatz 5, 20354 Hamburg, Germany
M. Reusch · V. Mielke
Dermatology Practice, Tibarg 7-9, Hamburg, Germany
Keywords Skin diseases · Goal attainment scaling ·
Outcomes measurement · Patient preferences ·
Patient reported outcomes · Quality of life
Introduction
The scientiWc assessment of therapeutic procedures is based
on the formulation and testing of eYcacy criteria. These
criteria should be recorded using objective methods under
standardized measuring conditions. The selection of valid
endpoints in controlled studies has become one of the pre-
requisites for evidence-based medicine. In many countries,
criteria have been introduced in recent years for the assess-
ment of medical procedures with which not only their
eYcacy but also their clinical beneWt can be determined, in
particular the beneWt from the patient’s point of view [1].
These treatment outcomes from the patient’s perspective
are summarized as patient-reported outcomes (PRO). In
most health systems, allocation decisions consider such
patient-relevant criteria. Clinical research in the future must
thus also include patient-deWned beneWt parameters as out-
comes variables.
However, thus far neither a generally accepted deWnition
of “therapeutic beneWt” has been published, nor has a clear
method for recording “patient-deWned beneWt” been devel-
oped. Studies to date only show that beneWt from the
patient’s perspective cannot be assessed by the physician,
but that the patient himself must be questioned directly,
since there is divergence between physicians’ and patients’
estimates [21]. Measurement of health-related quality of
life (HRQOL) has proven useful in recent years as a possi-
ble procedure for recording patient-deWned beneWt [4, 5,
12]. In particular, disease-speciWc quality of life question-
naires have gained considerable importance, such as the
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dermatology life quality index (DLQI) [10] and the Skin- numerous studies on health economy and on quality of
dex [6] in dermatology. Their advantage lies in maintaining life.
the patient perspective, their disadvantage in the fact that In the initial phase, n = 100 patients with skin diseases
the importance of individual therapeutic goals in total of various types were asked in an open text form about
beneWt is not taken into account by individual weighting. It their burden of disease and treatment needs. A broad spec-
is known from previous studies that patients with skin trum of skin diseases of diVerent geneses was purposely
diseases may have individual preferences with respect to included:
their treatment goals. These preferences vary in diVerent
a. psoriasis vulgaris (n = 20)
patient subgroups, and they show particular diVerences
b. atopic eczema (n = 20)
between patients and physicians [21]. Thus far, there is no
c. ulcus cruris (n = 10)
usable instrument for such an individualized beneWt assess-
d. viral and bacterial skin diseases (n = 10)
ment which is not only patient-relevant, but also patient-
e. malignant skin tumors (n = 10)
deWned.
f. bacterial skin diseases (n = 10)
The present study was conducted to develop a methodo-
g. hair diseases (n = 10)
logically sound procedure for the required beneWt assess-
h. allergic diseases (contact eczema, type I allergies,
ment in dermatology that is usable under conditions of
n = 10)
practice and enables determination of the individual
patient-relevant beneWt. The item pool was reduced to a standardized item list by a
panel of experts in dermatology, psychology and health
economics (n = 6 persons) with participation of n = 4
Materials and methods patients with chronic skin diseases. Protocols were written
of the expert meetings, including all item discussions.
The study was performed in the following steps: Each item was examined for its representativeness and
its suitability for use in a questionnaire. The Wnal version
a. Creation of an item pool of beneWt parameters based on
consisted of 23 items on patient-relevant therapy needs and
open patient and physician surveys; development of a
beneWts.
questionnaire for patient-deWned beneWts based on the
In the Wrst part of the questionnaire (patient needs ques-
item pool by experts and patients (Patient BeneWt
tionnaire, PNQ), a Wve-step Likert scale (0 = “not important
Index, PBI).
at all” to 4 = “very important”) records how relevant these
b. Validation of the questionnaire in a cross-sectional
items are as therapy goals for the individual patient. In the
study of n = 500 patients of ten dermatological diagno-
second part of the questionnaire (patient beneWt question-
ses.
naire, PBQ), the identical items are covered with the ques-
c. Testing and validation of the questionnaire in a longitudi-
tion of the extent to which the current therapy has
nal clinical study of n = 906 patients with acne vulgaris.
contributed to attaining the therapy goals (scaled from
In accordance with the AWMF Guidelines 2004 of the 0 = “treatment did not help at all” to 4 = “treatment helped
German Dermatological Society [1], the following valida- a lot”). As an alternative, there was the option to tick “does
tion criteria were evaluated: not apply to me”. The construction of the PBI is shown
diagrammatically in Fig. 1.
• internal consistency, retest-reliability
• construct validity: convergent and discriminant validity
• discriminatory power (between diagnosis groups)
• feasibility
• responsiveness

Item pool generation and development of the questionnaire

Generation of the pilot questions and Wnally of the ques-

tionnaire was done according to the international standards
of developing test instruments and the methodical guide-
lines of the AWMF (Association of the ScientiWc Medical
Societies in Germany) and the DDG (German Dermato-
logic Society) for recording quality of life in skin patients Fig. 1 Methodological approach of evaluating patient preferences,
[1, 14]. These methods have been tested and validated in patient beneWt and patient beneWt index

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The PBI total score is derived from the ratings on both
questionnaires. SpeciWcally, the PBI is the arithmetic mean
of all rated beneWts (PBQ items) weighted by the relative
importance of each corresponding need item (PNQ) for
each patient (Fig. 2).
This procedure is an extension of previous work on
goal attainment scaling (GAS) [7, 9, 13, 18, 19] and on
goal-oriented measurement of outcomes (zielorientierte
Ergebnismessung, ZOE) [11, 17] taking pertinent criti-
cism into account [8, 22]. It is an economical solution
between individualized and generic recording, whereby
two diVerent but related constructs (goal importance and
goal attainment) are questioned at two diVerent time
points.
Validation in a cross-sectional study
The pilot version of the questionnaire was initially vali-
dated in a cohort of n = 500 patients with a broad spectrum
of dermatoses, in which n = 50 patients each had one of the
following skin diseases: acne vulgaris, atopic eczema, auto-
immune diseases of the skin, hand and foot eczema, hair
diseases, herpes zoster, hyperhidrosis, psoriasis, ulcus cru-
ris, urticaria.
Item characteristics were analyzed descriptively, by
computing means and frequencies/percentages. Reliabil-
ity of the PNQ was assessed by analysis of internal
consistency and multivariate analysis of variance was per-
formed to detect the discriminability of diagnoses by the
PNQ-items.
In addition, the PBI was calculated as a global measure
of treatment beneWt. As criteria of convergent validity, the
patients were asked how successful they would rate their
treatment with respect to physical and emotional well-
being, their performance capacity on the job and in every-
day living, their social contacts, their leisure activities, and
their quality of life. Response was scaled on Wve levels
from 1 = “not at all” to 5 = “very successful”. The correla-
tion (Pearson method) of each of these criteria with the PBI
was calculated. SigniWcance levels of p · 0.05 were
deWned.
Fig. 2 Patient beneWt index with k preference items (PNQ range 0–4)
and beneWt items (PBQ range 0–4)
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Validation in a longitudinal study
The PBI was tested in another study on n = 906 patients
with acne vulgaris in pre-post comparison. Outpatients
under therapy with a topical combination preparation of
benzoylperoxide and clindamycin (“DUAC gel”) were
enrolled in this nation-wide multicenter observational study
[3]. The PNQ was recorded at T1 (therapy onset), the PBQ
at both T2 (ca. 4 weeks after therapy onset) and at T3 (after
8–12 weeks) under practice conditions.
Therapy needs and beneWts are presented descriptively
and at the single-item level. In addition, the beneWt index
PBI was calculated.
The feasibility of the questionnaire under practice condi-
tions was determined from the percentage of missing val-
ues. The sensitivity to change of the PBI in the course of
therapy was tested by a t-test for dependent samples
between T2 and T3. Additionally, retest-reliability was
computed in this interval.
The following criteria of convergent validity were
assessed:
• Improvement in acne Wndings from T1 to T3. This was
determined by taking the mean of 5 single Wndings, rated
by the physician on a scale from 1 = “not at all” to
5 = “very much”.
• Improvement in acne severity, rated by the patient, from
T1 to T3 (response scale 1 = “low” to 5 = “very severe”).
• Improvement in quality of life from T1 to T3, recorded
based on the validated questionnaire “ADI” (acne dis-
ability index) [15].
• Rating of the eYcacy of the medication by the patient at T3
(response scale 1 = “not eVective” to 5 = “very eVective”).
• Recommendation of the medication by the patient at T3
(response scale 1 = “not at all” to 5 = “in any case”).
The Pearson correlation of the PBI (computed using PNQ
T1 and PBQ T3) with each of these criteria and the corre-
sponding signiWcance levels were calculated.
Results
Item pool generation and development of the instrument
A total of 213 potentially relevant items were collected in
an open survey of n = 100 patients and n = 23 dermatolo-
gists. This item pool was reduced by a panel of experts and
patients to a list of 23 non-redundant and non-overlapping
items. Both questionnaires (PNQ and PBQ) include these
23 items in identical format, but diVerent instructions are
given. Whereas in the PNQ, the individual relevance of
treatment shall be rated, the beneWt achieved by the most
recent treatment is assessed in the PBQ.
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Cross-sectional validation of the instrument
Patient needs questionnaire
The tabulation of PNQ means by diagnoses (Table 1)
shows disease-speciWc, clinically recognizable patterns of
patient needs. The PNQ item means signiWcantly discrimi-
nated between the diseases in a multivariate analysis of var-
iance (p · 0.001, results not shown). Disease-speciWc
patterns are especially observed for diagnoses associated
with greater emotional stress (such as pain, itching, loss of
sleep, and impaired productivity) or involve physical and
emotional stigmatization as well as aesthetic distress. The
high need for improvement of physical well-being in the
form of reduced pain is conspicuous for chronic hand and
foot eczema, herpes zoster, and in some cases of atopic der-
matitis (PNQ item means > 3.0).
Impressively high for all diagnoses is the mean need for a
“clear diagnosis and therapy” and “conWdence in the therapy”
(PNQ item means > 3.5). In contrast, the need for treatment
beneWt regarding “partnership” and “sex life” were rated
lower by all patients. The items “fewer out-of-pocket treat-
ment expenses” and “fewer side eVects” for most of the diag-
noses (PNQ item means around 2.5) were equally distributed.
To “have no fear that the disease will become worse” is most
marked in hair diseases and eczemas, while the “healing of all
skin defects” was an important need in nearly all diagnoses
(PNQ item means largely between 3.4 and 3.8).
The percentages of patients who rated their needs as high
or very high (PNQ item ratings of 3 and 4) by diagnosis are
presented in Table 2. In sum, the table shows that (a) patients
deWned a broad spectrum of needs regardless of diagnosis,
(b) there are disease-speciWc need-patterns, and (c) the needs
to “Wnd a clear diagnosis and therapy” and to “have conW-
dence in the therapy” are particularly high for all diagnoses.
The reliability of the PNQ in terms of internal consis-
tency was considerably high (Cronbach’s alpha = 0.91, the
part-whole-corrected item-total correlations ranged from
0.32 to 0.68).
Patient beneWt questionnaire
Only few beneWts were rated to be achieved at the time the
questionnaire was given, i.e. only for one item more than 50%
of all patients ticked moderate to high beneWts (Table 3). Tak-
ing into account that a considerable proportion of patients had
just begun their treatment, this result was to be expected.
Patient beneWt index
The PBI was found to be skewed to the left and not nor-
mally distributed; again, this has to be attributed to the
cross-sectional nature of the study. The major Xoor eVect
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Arch Dermatol Res (2009) 301:561–571
(a PBI of 0 was achieved over proportionally frequent,
Fig. 3) might be ascribed to the proportion of patients in an
early stage of treatment.
Convergent validity of the PBI was established in
respect to patients’ rating of therapy success and was con-
siderably high (r > 0.5) for most of the criteria (Table 4) but
not suYciently high to interpret the PBI as a redundant
measure of treatment success (i.e. the PBI captures some
unique variance indicating some discriminant validity, too).
Longitudinal testing of the instrument in clinical practice
Of the 906 patients enrolled in the longitudinal study of
DUAC in acne vulgaris, 824 patients completed the PBI at
T1, 782 at T2, and 732 at T3. The low percentages of missing
values (Table 5) indicate high feasibility of the instrument: In
the PNQ, the amount of missing values was between 0.2 and
1.7% (mean 0.7%), and in the PBQ well under 2% at both
time points (T2: mean 0.8%; T3: mean 0.6%).
Patient needs questionnaire
Table 5 shows that all need items (except for “ability to
sleep better”) were rated at least moderately important
(item means > 2.0), whereas “healing of all skin defects”
was rated most important; at the same time, this item was
the need aVecting most patients (“does not apply to
me” = 2.3%). Internal consistency was even higher in this
sample (Cronbach’s alpha = 0.97, part-whole-corrected
item-total correlations between 0.50 and 0.86).
Patient beneWts questionnaire
The vast majority of beneWt items were rated as well or
very-well achieved by 40 to 60% of the patients (Table 5);
the beneWt “to have conWdence in therapy” was rated as
best achieved (67%), and “to be able to sleep better” as
least achieved (39%).
The proportion of patients rating the beneWts as well or
very-well achieved clearly increased from T2 to T3 (T2:
49.5%, T3: 62.3%) on all items.
Patient beneWt index
The improvement is reXected especially by the distribution
of the diVerence in PBI at T2 and T3 (Fig. 4) and the
respective PBI means of 3.43 (SD = 1.01) and 3.76 (SD =
1.04), which diVered signiWcantly in a t-test for dependent
samples (ttwo-tailed = ¡10.65; df = 722; p · 0.001). This
shows that the PBI is sensitive to change. Taking into
account that treatment was continued from T2 to T3, the
obtained retest-reliability (r = 0.68) of the PBI within this
period is highly acceptable.
 Table 1 Cross-sectional study: means of the patient need items (PNQ), scaled from 1 = “not at all” to 5 = “strongly” (n = 500, each diagnosis n = 50)
Item All Acne Atopic Autoimmune Hand/foot Alopecia Herpes Hyperhidrosis Psoriasis Leg Urticaria
diagnoses dermatitis eczema zoster ulcer

Be free of pain 2.56 2.50 2.70 3.02 3.28 1.22 3.68 0.74 2.86 3.26 2.32
Be free of itching 2.46 1.86 3.68 2.28 3.54 1.58 2.42 0.66 3.22 1.88 3.50
Arch Dermatol Res (2009) 301:561–571

No longer have burning sensations on ones skin 2.33 1.92 3.18 2.66 3.02 1.16 3.04 0.62 2.64 2.12 2.98
Be healed of all skin defects 3.39 3.80 3.42 3.68 3.84 2.88 3.54 1.86 3.76 3.58 3.58
Be able to sleep better 2.34 1.74 2.98 2.54 2.62 1.80 3.08 0.92 2.70 2.38 2.60
Feel less depressed 2.60 2.68 2.44 2.88 2.92 2.74 2.48 2.34 2.68 2.32 2.56
Experience a greater enjoyment of life 2.97 2.98 2.96 3.22 3.08 3.04 2.70 3.00 3.12 2.74 2.82
Have no fear that the disease will become worse 3.22 3.34 3.00 3.38 3.48 3.68 3.24 2.38 3.00 3.42 3.30
Be able to lead a normal everyday life 3.03 2.68 3.12 3.18 3.26 2.28 3.20 2.98 3.16 3.56 2.84
Be more productive in everyday life 2.59 2.10 2.74 2.98 2.88 1.82 2.84 2.24 2.48 3.10 2.76
Be less of a burden to relatives and friends 2.30 1.96 2.44 2.62 2.86 1.96 2.04 1.40 2.62 2.68 2.46
Be able to engage in normal leisure activities 2.57 2.20 2.70 2.70 2.90 1.82 2.48 2.70 2.98 2.52 2.70
Be able to lead a normal working life 2.05 2.04 2.56 2.40 2.32 1.62 1.90 2.24 2.28 0.86 2.26
Be able to have more contact with other people 2.14 2.30 2.08 2.56 2.44 1.96 1.74 2.04 2.40 1.82 2.02
Be comfortable showing oneself more in public 2.53 3.10 2.68 2.74 2.56 2.82 1.88 2.94 2.86 1.60 2.16
Be less burdened in ones partnership 2.24 2.64 2.48 2.52 2.60 1.56 1.70 2.08 2.64 1.72 2.44
Be able to have a normal sex life 1.84 2.32 1.96 2.46 1.84 1.22 1.26 1.38 2.40 1.28 2.30
Be less dependent on doctor and clinic visits 3.06 2.72 2.98 3.44 3.56 2.94 3.08 2.34 3.06 3.32 3.20
Need less time for daily treatment 2.67 2.70 2.50 2.88 3.08 2.56 2.46 1.84 3.10 2.94 2.60
Have fewer out-of-pocket treatment expenses 2.43 2.36 2.48 2.78 2.58 2.48 1.80 1.40 2.84 2.82 2.72
Have fewer side eVects 2.50 2.56 2.64 2.92 2.36 2.62 2.26 1.56 3.00 2.58 2.52
Find a clear diagnosis and therapy 3.63 3.40 3.48 3.70 3.78 3.86 3.80 3.06 3.50 3.84 3.90
Have conWdence in the therapy 3.66 3.52 3.46 3.78 3.78 3.90 3.82 3.22 3.68 3.74 3.68
Bold results indicate the two highest-ranked results for each item

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Table 2 Cross-sectional study: percentage of patients indicating moderate or high need on a scale from 0 = “not at all” to 4 = “strongly” (n = 500, each diagnosis n = 50)
Item All diagnoses Acne Atopic Autoimmune Hand/foot Alopecia Herpes Hyperhidrosis Psoriasis Leg Urticaria
dermatitis eczema zoster ulcer

Be free of pain 64.0 62.0 72.0 76.0 82.0 28.0 92.0 18.0 68.0 80.0 62.0
Be free of itching 61.0 42.0 98.0 42.0 86.0 36.0 60.0 14.0 84.0 44.0 88.0
No longer have burning sensations on ones skin 59.0 48.0 84.0 72.0 78.0 28.0 24.0 14.0 60.0 54.0 76.0
Be healed of all skin defects 85.8 96.0 84.0 94.0 98.0 70.0 90.0 48.0 98.0 92.0 88.0
Be able to sleep better 57.4 38.0 80.0 66.0 62.0 42.0 78.0 16.0 34.0 60.0 66.0
Feel less depressed 61.6 60.0 54.0 68.0 70.0 70.0 58.0 60.0 58.0 58.0 60.0
Experience a greater enjoyment of life 72.2 74.0 68.0 78.0 74.0 74.0 64.0 72.0 78.0 70.0 70.0
Have no fear that the disease will become worse 78.4 80.0 72.0 86.0 84.0 90.0 80.0 58.0 70.0 84.0 80.0
Be able to lead a normal everyday life 76.0 62.0 78.0 84.0 80.0 58.0 82.0 76.0 80.0 90.0 70.0
Be more productive in everyday life 63.8 48.0 68.0 78.0 70.0 44.0 74.0 54.0 56.0 78.0 68.0
Be less of a burden to relatives and friends 55.4 46.0 56.0 68.0 68.0 50.0 48.0 28.0 64.0 68.0 58.0
Be able to engage in normal leisure activities 63.6 52.0 64.0 68.0 74.0 46.0 60.0 68.0 78.0 62.0 64.0
Be able to lead a normal working life 50.4 52.0 62.0 60.0 56.0 42.0 48.0 56.0 48.0 22.0 54.0
Be able to have more contact with other people 49.6 52.0 46.0 66.0 60.0 44.0 38.0 50.0 52.0 44.0 48.0
Be comfortable showing oneself more in public 61.8 80.0 64.0 72.0 64.0 72.0 48.0 76.0 64.0 32.0 46.0
Be less burdened in ones partnership 55.4 60.0 62.0 60.0 64.0 40.0 44.0 54.0 68.0 42.0 60.0
Be able to have a normal sex life 44.4 52.0 46.0 64.0 46.0 32.0 30.0 32.0 60.0 28.0 54.0
Be less dependent on doctor and clinic visits 75.6 62.0 76.0 86.0 92.0 70.0 78.0 60.0 70.0 84.0 78.0
Need less time for daily treatment 63.4 62.0 54.0 66.0 20.0 56.0 64.0 46.0 76.0 68.0 62.0
Have fewer out-of-pocket treatment expenses 55.8 54.0 56.0 70.0 58.0 56.0 40.0 30.0 64.0 68.0 62.0
Have fewer side eVects 60.8 62.0 68.0 76.0 58.0 62.0 52.0 36.0 72.0 62.0 60.0
Find a clear diagnosis and therapy 90.4 82.0 86.0 94.0 94.0 96.0 96.0 76.0 86.0 96.0 98.0
Have conWdence in the therapy 92.6 88.0 82.0 98.0 96.0 98.0 98.0 80.0 98.0 94.0 94.0
Bold results indicate the two highest-ranked results for each item
Arch Dermatol Res (2009) 301:561–571
 Table 3 Cross-sectional study: means of patient beneWt (PBQ), percentage of patients indicating moderate or high beneWt on a scale from 0 = “not at all” to 4 = “strongly” (n = 500, each
diagnosis n = 50)
Item All Acne Atopic Autoimmune Hand/foot Alopecia Herpes Hyperhidrosis Psoriasis Leg ulcer Urticaria
diagnoses dermatitis eczema zoster

Be free of pain 42.7 15.8 47.4 56.8 28.6 38.9 77.1 10.0 32.6 48.9 33.3
Arch Dermatol Res (2009) 301:561–571

Be free of itching 36.3 13.3 36.7 60.0 30.6 24.0 75.8 18.2 31.1 34.5 27.7
No longer have burning sensations on ones skin 38.5 13.8 43.2 63.4 23.3 22.2 67.5 9.1 35.7 38.7 31.7
Be healed of all skin defects 26.2 12.0 22.0 51.0 22.0 7.9 41.7 16.7 20.0 36.2 25.0
Be able to sleep better 35.1 13.8 37.2 44.7 30.8 32.1 60.5 15.8 26.7 41.2 31.6
Feel less depressed 30.2 20.5 33.3 43.2 24.4 30.0 35.1 23.7 26.1 45.7 22.5
Experience a greater enjoyment of life 32.6 22.7 29.8 51.1 25.6 27.9 42.1 26.7 28.3 44.7 29.3
Have no fear that the disease will become worse 31.7 14.9 30.0 52.1 22.4 14.0 46.5 25.7 25.0 62.5 24.4
Be able to lead a normal everyday life 32.9 17.5 36.2 50.0 32.6 29.0 31.0 26.2 27.1 47.7 28.6
Be more productive in everyday life 30.7 15.6 29.8 43.9 31.0 30.8 27.0 25.7 29.5 46.3 23.3
Be less of a burden to relatives and friends 31.1 9.4 25.6 46.2 29.3 17.2 39.3 26.9 31.7 52.9 28.9
Be able to engage in normal leisure activities 31.2 18.8 29.5 40.5 30.0 23.1 33.3 33.3 26.7 37.8 35.0
Be able to lead a normal working life 29.6 20.0 24.3 44.1 24.1 22.7 27.3 33.3 30.8 26.7 36.4
Be able to have more contact with other people 28.6 18.4 21.1 43.6 25.0 13.3 37.0 32.3 29.5 37.0 30.3
Be comfortable showing oneself more in public 28.9 17.8 26.7 39.0 28.9 18.4 42.3 33.3 30.4 25.0 32.4
Be less burdened in ones partnership 33.1 15.8 30.6 34.3 29.4 18.2 59.1 32.3 39.5 42.3 37.1
Be able to have a normal sex life 30.0 11.4 24.2 37.5 25.0 6.7 55.6 30.0 37.1 38.9 36.4
Be less dependent on doctor and clinic visits 24.8 11.6 22.9 34.7 20.8 9.3 35.0 22.9 27.1 41.3 21.3
Need less time for daily treatment 22.6 6.8 17.4 38.3 20.0 6.8 33.3 17.9 25.5 40.4 17.5
Have fewer out-of-pocket treatment expenses 23.3 10.5 10.0 40.5 21.6 7.3 44.0 16.7 21.3 47.4 19.0
Have fewer side eVects 30.2 28.2 22.0 42.9 26.7 23.5 46.4 21.4 22.7 47.2 22.2
Find a clear diagnosis and therapy 48.7 37.0 36.0 68.0 32.7 30.0 87.8 42.9 46.9 68.0 36.0
Have conWdence in the therapy 51.2 33.3 36.0 72.0 46.9 32.0 83.7 42.2 48.0 80.0 36.7
The percentages refer to the total number of patients in each case who did not tick “doesn’t apply to me” for the item

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study in clinical routine. In addition, the PBI was sensitive

Fig. 3 Distribution of the PBI (n = 500, cross-sectional study)
In this sample, the convergent validity of the PBI (Table
4) regarding patients' rating of the eYcacy of the medica-
tion and their recommendation of the medication was
acceptable high (r > 0.5) and acceptable for ratings of clini-
cal improvement and improvement in quality of life
(r between 0.34 and 0.44).
Discussion
The instrument was found to be suYciently feasible, reli-
able and valid in a cross-sectional study of patients with a
broad spectrum of diagnoses and in a longitudinal treatment
to change. Due to the concept of the instrument, reliability
assessment is complicated, since two diVerent constructs
are measured by the PNQ and PBQ and patients individu-
ally select and weight their treatment needs. As the separate
summation of the items from both questionnaires is neces-
sary for computing the PBI, internal consistency was cho-
sen as the primary method for the estimation of reliability.
The retest-reliability of the PBI obtained in the longitudinal
study has to be interpreted as a lower bound of reliability,
since (a) 8 to 12 weeks passed before the retest was given,
and (b) treatment was continued in this interval.
The correlations to related constructs (convergent valid-
ity) were in the medium range for a variety of constructs
like quality of life, treatment success and, to less extent,
clinical eYcacy as rated by patients and practitioners.
Apparently, the PBI also captures some unique variance not
accounted for by the chosen criteria.
Patient-deWned outcomes parameters have only rarely
been studied as main target parameters in clinical studies
thus far. As secondary criteria, primarily pain and quality of
life are found among the possible outcomes parameters
from the patient’s perspective. Instruments to assess these
parameters (e.g. dermatology life quality index DLQI,
Skindex, Freiburg quality of life questionnaire FLQA,
Short-Form-36 Health Survey SF-36), were developed with
sound methodology and are internationally accepted and
implemented in clinical studies.
As part of the introduction of requirements for beneWt
evaluation for cost reimbursement of drugs, the patient-
deWned beneWt assessment has become a deWnite health-
political and economic standard. This applies at least
for the beneWt assessment by the Institute for Quality
and Economy in Health (Institut für Qualität und

Table 4 Convergent validity: correlation of the PBI with criteria applied

Study Criterion of convergent validity Correlation SigniWcance n
(r) (two-tailed)

Cross-sectional Rating of treatment success with respect to …

Physical well-being 0.76 ·0.001 499
Emotional well-being 0.69 ·0.001 499
Performance capacity in job and everyday living 0.54 ·0.001 499
Social contacts 0.53 ·0.001 499
Leisure activities 0.53 ·0.001 499
Quality of life 0.71 ·0.001 499
Longitudinal Improvement of mean acne Wnding (physician’s rating) 0.34 ·0.001 728
Improvement of acne severity (patient’s rating) 0.34 ·0.001 715
Improvement in quality of life 0.44 ·0.001 711
Patient’s rating of the eYcacy of medication 0.57 ·0.001 728
Patient’s recommendation of the medication to others 0.53 ·0.001 725
Improvement scores were computed by removing the inXuence of baseline values (T1) from the respective outcomes (T3) by linear regression

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 Table 5 Longitudinal study: means and frequencies of the PNQ (patient needs questionnaire) and PBQ (patient beneWt questionnaire) items (T1: n = 824; T2: n = 782, T3: n = 732)
Item T1: PNQ T2: PBQ T3: PBQ

Mean SD Important/very Does not Missing Helped/ Does not Missing Helped/ Does not Missing
important (%) apply (%) helped apply (%) helped apply (%)
to me (%) a lota (%) to me (%) a lot (%) to me (%)

Be free of pain 2.27 1.42 40.3 37.0 0.4 53.3 48.0 0.8 64.2 48.2 0.3
Arch Dermatol Res (2009) 301:561–571

Be free of itching 2.36 1.41 51.6 36.2 0.5 53.9 41.7 0.3 63.5 43.9 0.3
No longer have burning sensations on ones skin 2.50 1.35 53.1 33.7 1.7 49.5 39.3 1.7 62.8 39.8 1.0
Be healed of all skin defects 3.61 0.77 90.7 2.3 0.7 45.6 4.3 1.7 68.1 5.7 1.1
Be able to sleep better 1.88 1.46 37.0 55.3 0.5 39.3 60.0 0.6 50.0 59.8 1.0
Feel less depressed 2.56 1.35 57.2 21.5 0.5 49.3 28.1 0.8 63.0 29.5 0.3
Experience a greater enjoyment of life 2.80 1.28 65.0 20.1 1.1 52.0 26.0 0.9 67.0 24.6 0.5
Have no fear that the disease will become worse 2.84 1.32 65.9 15.7 0.6 55.7 21.2 0.9 66.4 19.0 0.4
Be able to lead a normal everyday life 2.74 1.30 63.0 27.9 1.0 52.7 33.5 0.3 64.9 31.6 0.8
Be more productive in everyday life 2.26 1.36 46.2 42.2 0.7 43.1 45.4 0.6 52.9 43.2 0.8
Be less of a burden to relatives and friends 2.07 1.40 41.9 39.2 0.6 40.8 42.1 1.2 54.0 39.5 0.5
Be able to engage in normal leisure activities 2.55 1.37 57.4 34.5 0.2 49.8 37.1 0.6 66.4 35.0 0.3
Be able to lead a normal working life 2.32 1.46 49.1 51.3 0.6 46.1 53.7 0.6 58.9 53.8 0.4
Be able to have more contact with other people 2.63 1.33 58.6 26.7 0.7 49.9 26.2 1.0 64.1 27.3 0.3
Be comfortable showing oneself more in public 2.97 1.21 69.1 11.5 0.8 54.2 10.1 0.8 70.2 11.5 1.0
Be less burdened in ones partnership 2.67 1.35 62.3 42.4 0.5 53.0 46.2 0.9 65.4 46.3 0.4
Be able to have a normal sex life 2.43 1.47 53.7 48.1 1.1 46.6 53.3 0.6 57.7 51.9 0.3
Be less dependent on doctor and clinic visits 2.46 1.30 52.8 15.7 0.5 40.6 21.0 0.9 52.6 21.0 1.1
Need less time for daily treatment 2.62 1.27 57.3 7.3 0.7 44.2 8.3 0.9 57.7 8.6 0.5
Have fewer out-of-pocket treatment expenses 2.50 2.14 52.6 21.5 0.8 43.9 30.1 0.8 53.1 28.7 0.5
Have fewer side eVects 2.49 1.39 53.7 22.7 0.7 50.1 27.0 1.0 64.1 28.4 0.4
Find a clear diagnosis and therapy 2.99 1.21 70.8 11.2 0.6 58.8 13.7 0.8 70.1 14.5 0.5
Have conWdence in the therapy 3.22 1.04 85.3 7.4 0.2 66.7 7.8 0.5 75.8 8.1 0.4
a
Means, standard deviations (SD) and percentages of ticked “important/very important” or “helped/helped a lot“ refer to the total number of patients who did not tick “doesn’t apply to me”. All
other percentages refer to the total number of all patients in the sample

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569
570
Fig. 4 Changes in the PBI from T2 to T3 (n = 721, longitudinal study,
bracket indicates patients with improvement in PBI)
Wirtschaftlichkeit im Gesundheitswesen, IQWiG) and for
the Joint Federal Committee (Gemeinsamer Bundesauss-
schuss, GBA) as the central authorities in Germany. It can
be expected that such beneWt assessment will gain in inter-
national importance [20].
The PBI was developed, because no reliable, valid and
accepted method to assess patient-relevant beneWt—at least
for dermatological treatments— was found in the literature.
The logic is to present a structured list of predeWned items
of empirically well-known patient-relevant needs in derma-
tology, from which each individual patient selects and
weights his personal treatment needs, and to request after
(or in the course of) treatment the extent to which these
individual needs had been achieved. This method is new
and adds a lot to the usual assessment of patient-reported
outcome or quality of life.
The only approaches similar to the presented method are
“Goal Attainment Scaling, GAS” and “ZielorientierteErgeb-
nismessung, ZOE”, which indeed served as a basis for the
PBI development and have been implemented in numerous
published studies in the area of rehabilitation medicine and
psychiatry. Since these methods are controversially dis-
cussed and shortcomings are well known to date, the PBI
was developed to overcome these relevant criticisms [12].
A major advantage, for example, is the separation of the
construct measured into “needs” and beneWts” which mini-
mizes eVects relevant to internal validity (e.g. repeated test-
ing and regression towards the mean).
Moreover, the prestructured item lists of empirically
established relevant goals in dermatology provide a better
standardization than the open questioning in GAS, and the
individual weighting of relevant needs on 5-point Likert-
scales provides a more realistic and diVerentiated approach
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Arch Dermatol Res (2009) 301:561–571
to patient-relevant beneWt assessment than the simple “rele-
vant/not relevant” decisions requested in ZOE.
The large number of patient-relevant need items
included enables to identify need patterns within and
between diagnoses, corresponds to the claims to implement
a wide diversity of outcome parameters in clinical studies,
and meets the requirements of modern beneWt assessment
[16]. Moreover, it was demonstrated empirically that a vari-
ety of individually relevant treatment needs and beneWts do
exist. This diversity has to be accounted for in studies of
eYcacy and eVectiveness and in the decision processes on
product authorisation and reimbursement.
In conclusion, the PBI provides the Wrst valid and feasi-
ble self-assessment procedure for weighted beneWt evalua-
tion by the patient in dermatology. The PBI is suitable for a
patient-deWned beneWt assessment under practice and study
conditions.
The next steps in further developing the PBI are to
expand disease-speciWc modules, and to develop a cut-oV
of clinically relevant beneWt.
ConXict of interest statement The authors declare that they have no
conXict of interest. The clinical trial was supported by a grant from
Stiefel GmbH, OVenbach/Germany
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