Community Dent Oral Epidemiol 2000; 28: 119–25 Copyright C Munksgaard 2000

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ISSN 0301-5661

Manal A. Awad1,
Determinants of patients’ Stanley H. Shapiro2,3,
James P. Lund1,4,5 and
Jocelyne S. Feine1,2,3
treatment preferences in a clinical 1
2
McGill University Faculty of Dentistry,
McGill University Faculty of Medicine, Joint
Departments of Epidemiology and

trial Biostatistics, and Occupational Health,

3
Jewish General Hospital, Lady Davis
Institute for Medical Research, 4McGill
University Faculty of Medicine, Department
of Physiology, 5Université de Montréal, Centre
de recherche en science neurologiques,
Monatreal, Quebec, Canada

Awad MA, Shapiro SH, Lund JP, Feine JS: Determinants of patients’ treatment
preferences in a clinical trial. Community Dent Oral Epidemiol 2000; 28: 119–25.
C Munksgaard, 2000

Abstract – Several researchers have suggested that patients’ preferences for a

particular form of treatment should be taken into account in clinical trials. Prefer-
ences may influence the outcome of treatment, especially in trials when patients
cannot be blinded to the type of treatment received and the outcome is based on
patients’ evaluations of therapy. Participants in this study were 136 edentulous
patients who took part in a randomised controlled clinical trial comparing two
types of treatments for edentulism: conventional dentures and implant-support-
ed prostheses. Prior to receiving treatment, subjects were required to complete a
questionnaire regarding their satisfaction with their present prostheses. In addi-
tion, they were asked to indicate which treatment they would prefer if given a
choice. The objective of this study was to determine whether there are important
differences among study participants between patients who have a treatment pref-
erence and those who do not. The effects of satisfaction with pre-treatment pros-
theses, age, gender and level of education on preferences were examined. Level of
satisfaction with the original dentures and level of education were significant
predictors of preference. Compared to subjects who rated their satisfaction with
their current condition as ‘low’, the odds ratios associated with having a prefer-
ence for implant treatment were 0.31 (95% CI: 0.09 to 0.96) for subjects who rated
their prostheses in the ‘medium’ range and 0.11 (95% CI: 0.03 to 0.41) for those
Key words: clinical trials; oral prostheses;
who rated in the ‘high’ range. In addition, subjects with high levels of education study designs; treatment preferences
were significantly less likely to have a preference for either conventional or im-
plant treatments (ORΩ0.18, 95% CI: 0.02 to 0.77 and ORΩ0.20, 95% CI: 0.05 to 0.76, Jocelyne S. Feine, McGill University,
3640 University Street, Montreal,
respectively) compared to those with low education. Neither age nor gender was Quebec H3A 2B2, Canada
a significant predictor of preference. We suggest that study designs which incorpo- Tel: π514 398 7203
rate patients’ preferences must take into account possible differences between Fax: π514 398 8900
preference groups that might confound the relationship between preference and e-mail: feine/med.mcgill.ca
the outcome of interest. Accepted for publication 27 August 1999

The importance of considering patients’ preferenc-
es for a particular form of treatment has been a
topic of debate (1–7). It is clear that this is an im-
portant issue in daily clinical practice, and it is now
emerging as a major concern in clinical trial design.
Patients are becoming more active participants in
decisions about their health care (1), and it seems
to be generally accepted that patients should be
given the opportunity to select their preferred
treatment whenever possible from options that
have already been shown to be appropriate and ef-
ficacious (2). However, when the efficacy of treat-

119
Awad et al.
ment is assessed in clinical trials, preference is not
taken into account. This has led some authors to
question the generalisability estimates of the effec-
tiveness of therapy from trials (3–12). Patient pref-
erence can also cause other problems in clinical
trials (5, 6). For example, people with strong prefer-
ences for one treatment may decide not to partici-
pate in a randomised trial. If they do decide to en-
rol in a non-blinded trial, the motivation of those
who are not given their treatment of choice may be
low, leading to drop-outs or non-compliance. To-
gether, the effects of these phenomena would re-
duce the general applicability of the results. Allow-
ance for preference may be particularly important
in non-blinded or poorly blinded randomised clin-
ical trials (RCTs) that use patient-based outcomes,
because it is probable that patients’ emotional re-
sponses to treatment assignment would influence
the estimates of effectiveness. For instance, pa-
tients’ ‘‘disappointment’’ at having been assigned
a non-preferred treatment may be reflected in their
ratings of satisfaction.
McPherson et al. (11) developed a theoretical
model to evaluate the effect of preference in RCTs.
In this model, the treatment effect is not simply the
measured ‘‘biologic’’ difference between treat-
ments; the difference is modified depending on
whether subjects receive their preferred treatment
and by the proportion of subjects who prefer each
treatment. According to this model, the effect of the
‘‘biologically’’ superior treatment can be overesti-
mated if a higher proportion of patients prefer that
treatment. On the other hand, underestimation of
effectiveness would occur if more patients ex-
pressed a preference for the inferior treatment.
Consequently, Brewin & Bradley (12) suggested
that studies should be designed to achieve a ‘‘good
fit’’ between patients’ preferences and the type of
treatment received.
Clearly the RCT is the most appropriate design
when the efficacy of a particular form of therapy is
being first established, or when the probabilities of
different outcomes associated with each treatment
modality are unknown (13). However, once efficacy
has been estimated and the objective is to ascertain
which form of therapy would yield better results in
the field, then including preference into the study
design creates conditions that more closely resem-
ble clinical practice. As Till et al. (14) pointed out,
preference trials should be viewed as supplements
to RCTs rather than alternatives.
Cooper et al. (15) recently examined the relation-
ship between preferences and satisfaction with
120
treatment among 273 women suffering from heavy
menstrual bleeding. One hundred and thirty-five
subjects were randomly assigned to a partially ran-
domised patient preference trial (PRPP) in which
patients who prefer a particular treatment are giv-
en their choice, while those who have no prefer-
ence are randomly assigned to either treatment
(12). Another 138 subjects were assigned to a con-
ventional randomised controlled trial (CRCT). Sub-
jects randomised to the PRPP were asked about
their preferences and were given the treatment of
their choice, while those who expressed no prefer-
ence were randomly allocated to receive either
treatment. Five subjects (3%) refused to participate
in the PRPP arm, while in the CRCT group, 30% of
the subjects refused participation because of strong
preference, suggesting that subjects who agreed to
participate in the CRCT were women who did not
have strong preferences. This hypothesis is sup-
ported by the authors, who report that no statis-
tically significant difference was observed between
subjects who remained in the CRCT and those who
were randomly allocated to treatments in the PRPP
group. However, women who selected the medical
intervention were significantly more likely to find
the treatment acceptable than women who were
randomly allocated to it. This difference was not
observed among the women who chose the surgi-
cal procedure and those who were randomised to
this treatment. The authors suggested that women
who selected the surgical intervention had high ex-
pectations for this treatment but suffered more,
and their disappointment led to these results. In-
formation regarding the role of preference on out-
come would not have been possible if patient pref-
erence were not part of this trial design.
Henshaw et al. (16) measured the preferences of
women for two methods of abortion (mifepri-
stone/gemeprost or vacuum aspiration). The re-
sults showed that both interventions were highly
acceptable to women who were assigned to the
procedure that they preferred. On the other hand,
the surgical procedure was more acceptable than
the medical intervention among subjects who were
randomly allocated to treatment. The authors con-
cluded that women who prefer a specific method
of terminating a pregnancy should be given their
choice, while women who are not sure which pro-
cedure they prefer might be offered the surgical op-
tion.
However, conclusions from studies such as these
that consider patients’ preferences in the evalua-
tion of treatment outcome could be misleading if

there are differences in other potential explanatory
variables between patients with preference for
treatments and those without preferences (11). This
led to our attempt to determine whether there are
factors that distinguish between patients who ex-
press a preference and those who have no prefer-
ence for one of two types of treatment for eden-
tulism.
Material and methods
Participants in this study were patients who were
originally recruited to evaluate the efficacy of two
types of treatments for the mandible: a standard
oral prosthesis and a prosthesis partially supported
by two endosseous implants. The efficacy of this
type of implant-supported prosthesis had not been
previously evaluated in a randomised controlled
clinical trial.
One hundred and thirty-six male and female
edentulous subjects seeking replacement of their
standard oral prostheses responded to an invita-
tion to participate in this clinical trial. For patients
to be eligible to participate in this study, they had
to fulfil the following criteria: 1) must be wearing
their conventional dentures on a regular basis; 2)
be between the ages of 35 and 65 years; 3) have
been edentulous for at least 10 years. Subjects who
met these criteria attended an information session
in which they were told that the study was a ran-
domised controlled clinical trial to compare the ef-
ficacy of two types of treatments for the mandible.
They were given a description of the procedures
involved with each treatment modality, and in-
formed that there was no evidence to suggest that
an implant prosthesis using only two implants is
better than a well-made standard prosthesis. They
were asked to complete a questionnaire on socio-
demographic factors (age, sex, and level of educa-
tion) and to indicate which treatment they would
prefer to receive if given a choice. In addition, pa-
tients were asked to rate their level of general satis-
faction, comfort, ability to chew, stability, ability to
speak and aesthetics of their present oral prosthesis
on a 100-mm Visual Analogue Scale (VAS), with
higher values indicating greater satisfaction.
Analysis
This analysis focused on estimating the effect of
gender, level of education, age and general satisfac-
tion with the original conventional dentures on
subjects’ preference for treatment. Previously, we
had shown that subjects’ ratings of level of com-
Patients’ treatment preferences and study designs
fort, ease of chewing, stability, ability to speak and
aesthetics of their oral prostheses explained 89% of
the variation in ratings of general satisfaction (17).
Therefore, ratings of general satisfaction were used
in this analysis as an indicator of their current oral
condition.
It was conceivable that patients who were rela-
tively satisfied with their present condition might
express a preference for the same form of treat-
ment. In contrast, those who were very dissatisfied
might prefer the alternative treatment, while those
in the middle of the scale might be neutral. There-
fore, it was decided to divide the variable ‘overall
satisfaction’ into three levels: low, medium and
high (0–25 mm, 26–75 mm, 76–100 mm, respec-
tively). It was assumed that the effect of age on
preference would more likely depend on whether
the patients are considered relatively young or old
with respect to a chronic condition like edentulism.
A 40-year-old edentulous subject would be consid-
ered relatively young to be missing all of his or her
teeth. Therefore, the mean age (approximately 50
years) was taken as a cut-off point above which it is
more common to find people who are edentulous.
Level of education was initially defined by four
categories: less than 7 years of education, 8–12
years of education, 13–15 years (technical or college
degree) and 16 years or more (university). How-
ever, due to sample size considerations, level of
education was defined as ‘high’ when subjects
completed university or college, and ‘low’ when
they did not (12 or fewer years of education).
Chi-square tests were used to evaluate whether
the univariate distribution of age, gender, level of
education and satisfaction with current prostheses
differed according to preference status. Additional
multivariate polytomous regression was carried
out to evaluate the multivariate association be-
tween type of preference and subjects’ characteris-
tics. This method of analysis allows for the assess-
ment of nominal scaled outcome variables with
more than two categories. Analyses were imple-
mented using the statistical package SAS (18).
Results
Table 1 shows the distribution of subjects according
to gender, age, level of education and ratings of
level of satisfaction with the present oral prosthe-
ses. The majority of the subjects (79%) expressed a
treatment preference, 19% for the conventional and
60% for the implant treatments. Most subjects who
preferred the implant treatment were patients 50
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Awad et al.

Table 1. Associations between patients’ characteristics and preferences

Preferences N (%)
Neutral Conventional Implant
Variables 28 (21) 26 (19) 82 (60) P-value

Gender
Females 11 (39) 17 (65) 43 (53) 0.15
Males 17 (61) 9 (35) 39 (47)
Age
∞50 years 15 (54) 11 (42) 24 (29) 0.05
Ø50 years 13 (46) 15 (58) 58 (71)
Education level
High* 25 (89) 14 (54) 46 (56) 0.004
Low† 3 (11) 12 (46) 36 (44)
Level of satisfaction‡
Low (0–25 mm) 6 (22) 12 (46) 50 (61) 0.002
Medium (26–75 mm) 11 (39) 7 (27) 23 (28)
High (76–100 mm) 11 (39) 7 (27) 9 (11)

* University or college completed.

†
University or college degree incomplete.
‡
Rating based on 100-mm Visual Analogue Scale (VAS).

Table 2. Multivariate adjusted odds ratios (ORs)* according to preference and 95% confidence intervals (CI) for patients’
characteristics

Variables OR† CI P-value OR‡ CI P-value

Gender
Males§
Females 2.86 0.89, 2.22 0.08 1.86 0.69, 4.90 0.23
Age
∞50 years§
Ø50 years 1.43 0.44, 4.66 0.54 2.33 0.88, 6.23 0.09
Education level
Low§
High 0.18 0.04, 0.77 0.02 0.20 0.05, 0.76 0.02
Level of satisfaction
Low (0–25 mm)§
Medium (26–75 mm) 0.41 0.10, 1.67 0.22 0.31 0.09, 0.96 0.05
High (76–100 mm) 0.36 0.08, 1.54 0.17 0.11 0.03, 0.41 0.001

* ORs estimated from polytomous logistic regression including all the above variables, using neutrality as reference outcome
category.
†
Estimated ORs for preference for conventional treatment.
‡
Estimated ORs for preference implant treatment.
§
Reference category.

years and older (71%); while 46% of patients who
were neutral and 58% of subjects who preferred
conventional prostheses were in this age category
(PΩ0.05). The majority of patients who were neu-
tral had a college or university degree (89%).
Slightly more than half of those who preferred the
newer treatment or the conventional treatment had
a college or university degree (56% and 54% re-
spectively, PΩ0.004). The majority of subjects in the
neutral group rated their overall satisfaction with
their present prostheses in the ‘medium’ (39%) and
‘high’ category (39%), while significantly more sub-
jects who preferred implant treatment (61%) rated
their present prostheses in the ‘low’ category (PΩ
0.002).
Table 2 displays the findings using polytomous
logistic regression analysis. This model allowed for
the simultaneous estimation of the odds ratios

122

(ORs) for preference for implant treatment or stan-
dard treatment, using neutrality regarding treat-
ments as the reference outcome category. Although
the ORs for the association between preferences
and gender were not significantly different from 1,
there was a tendency for females to prefer conven-
tional treatment (ORΩ2.86, 95% CI: 0.89 to 2.22, PΩ
0.08). Likewise, compared to younger subjects,
older patients were more likely to prefer the im-
plant treatment. However, the OR was not statis-
tically significant (ORΩ2.33, 95% CI: 0.88 to 6.23,
PΩ0.09). As shown in Table 2, compared to ‘low’
ratings of satisfaction, the odds ratios associated
with preference for implant treatment were signi-
ficant for ratings of satisfaction in the ‘medium’
range (ORΩ0.41, 95% CI: 0.09 to 0.96), as well as in
the ‘high’ range (ORΩ0.11, 95% CI: 0.03 to 0.41).
The ORs associated with preference for standard
treatment did not significantly differ from 1. In ad-
dition, compared to subjects with ‘low’ education
(university or college degree incomplete), subjects
with ‘high’ education (university or college degree
completed) were significantly (P∞0.05) less likely
to have a preference for either conventional treat-
ment (ORΩ0.18, 95% CI: 0.04 to 0.77) or implant
treatment (ORΩ0.20, 95% CI: 0.05 to 0.76).
Discussion
It has been advocated strongly that therapeutic
choices should be based not only on predetermined
clinical criteria, but also on patient preferences (13,
14, 19), especially since more and more studies are
demonstrating that patients differ in their views of
possible outcomes (8, 20–22).
Feine et al. (7) have previously discussed some
of the problems that may arise when patients’ pref-
erences are not taken into consideration. Using ex-
amples from different studies where patients’ eval-
uations of treatment differed from that of the clini-
cians, they pointed out that, when subjects are
given a choice, anxiety and disappointment may be
reduced. This could be reflected in treatment out-
come. The authors (7) also mentioned preliminary
results from the present study that suggested that
there were differences between patients who have
a preference for a particular treatment for edentul-
ism and those who did not. In this paper we have
presented the full analysis on the determinants of
treatment preferences among edentulous patients.
These data show that, in addition to the previously
(7) reported differences between those with and
Patients’ treatment preferences and study designs
without preferences, it is equally important to con-
sider the form of the preferred therapy (Table 2).
The belief that preferences for treatment need to
be considered when estimating the potential effec-
tiveness of unblinded therapies prompted Brew-
in & Bradley (12) to propose a preference design
for clinical trials. In this form of preference con-
trolled trial (PCT), those patients who express no
strong treatment preference are randomised, while
those with preference are offered their choice. It
was hypothesised that comparing the two arms in
the randomised group would provide similar re-
sults to those obtained from an RCT design, while
comparing patients who selected a particular treat-
ment modality to those randomly allocated to the
same treatment would indicate the effect of moti-
vational factors on outcome of treatment (5). This
proposed design is based on the assumption that
preference for treatment is independent of other
factors. However, our results suggest that there are
factors that may confound the association between
preference and outcome that cannot be properly
controlled in the Brewin & Bradley design.
Moreover, in many cases, confounding factors
are not known to the investigators prior to the trial
and therefore cannot be controlled in the analysis
of the data.
We found a significant association between level
of education and preference. The suggestion by
Hack et al. (23) that well-educated patients might
tend to be more knowledgeable about disease
seems to be a plausible explanation for this finding.
Subjects who are well educated might better
understand the reasons for conducting compara-
tive research and be more likely to accept our state-
ment that there was no strong evidence to suggest
that the new treatment was superior to the stan-
dard. Also, well-educated individuals might be less
susceptible to marketing claims for new treatments
and therefore tend to be neutral.
A relationship between patients’ perception of
their level of satisfaction and preference was dem-
onstrated in the study by Cooper et al. (15), who
found that women who preferred a surgical proce-
dure for their heavy menstrual bleeding had sig-
nificantly higher baseline bleeding and pain scores
than patients who had no preference or who pre-
ferred the medical intervention. In the present
study, higher ratings of satisfaction were associated
with less likelihood of having a treatment prefer-
ence. This suggests that patients who perceive their
previous treatment positively are willing to try ei-
ther treatment, while those who are dissatisfied
123
Awad et al.
wish to have the implants. However, satisfaction
with current prostheses did not distinguish be-
tween patients who prefer conventional treatment
and those who were neutral. This finding is not
surprising, as patients who opted for the standard
treatment were as satisfied with this therapy as
those who had no treatment preference. At the con-
clusion of the trial, a sample of 50 subjects partici-
pated in focus groups to gather qualitative infor-
mation that was used to complement and interpret
quantitative data gathered during the trial. These
data provided some explanation for why subjects,
who were relatively satisfied with their original
prostheses and preferred the conventional treat-
ment, wished to participate in the trial. They indi-
cated that they had confidence in studies done in
university settings and that they knew that at some
point they would need a new prosthesis to replace
the one they currently wore.
The association between age and preference was
observed to be significant only in the univariate
analysis. After adjustment for other factors, age
was not as strongly correlated with preference (PΩ
0.09), possibly because older patients were also less
satisfied with their prostheses than younger ones.
Likewise, there was a non-significant tendency for
females to have a preference, especially for the con-
ventional treatment (Table 2). However, with a
larger sample size it may be possible that these
variables have significant effects on preference in a
multivariate model.
Few researchers have disputed the impact of pa-
tients’ characteristics on the choice of a particular
treatment modality, which in turn could affect rat-
ings of treatment outcome (4). The present study
addressed this issue by reporting on preferences
for treatment and characteristics of edentulous sub-
jects participating in a clinical trial. In light of our
results, we suggest that the design advocated by
Brewin & Bradley (12) can lead to unreliable esti-
mates of the effect of preference on outcome meas-
ures, because differences in uncontrolled variables
between neutral subjects assigned to the RCT arm
and the subjects with preference in the PCT will
modify the effects of preference in ways that are
impossible to estimate.
One possible solution is the design proposed by
Wennberg et al. (24), who suggested the random
allocation of subjects to either a PCT or a classic
RCT. If the study is sufficiently large, this initial
randomisation should control for differences be-
tween the neutral and the preference groups and
allow a better estimate of the magnitude of the ef-
124
fect of preference on the measured outcome. If one
wishes to exercise tighter control, then stratification
could be done for factors such as age and gender.
In summary, our data suggest that there are dif-
ferences between patients who express preferences
for one form of therapy and those who have no
strong preference. In our population, level of edu-
cation and level of perceived satisfaction with their
original treatment were the factors that distin-
guished the two groups.
Acknowledgements
This study was funded by Canadian Medical Research Coun-
cil Grant .UI-11195 and Health and Welfare Canada NHRDP
Grant .6605–4409–402. We wish to thank Nobel Biocare Can-
ada Inc.A for its generous support.
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