Alonso, J

Brugulat, P

Cunillera, O

Ferrer, M

Herdman, M

Medina, A

Rajmil, L

Tresserras, R

Vilagut, G

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dependent covariate dose and medication type. Efﬁcacy was
assumed identical for each subsequent injection. RESULTS: The
systematic review revealed that different regimens were applied
across pre-FENOC studies. Furthermore the Bayesian analysis
demonstrated that medication type, alone or combined with
dosage, had a signiﬁcant inﬂuence on modelled efﬁcacy. Using a
standard treatment regimen of one 90 mg/kg rFVIIa injection
every 3 hours, the model estimated that at 12, 24, and 36 hours
the probability of bleed resolution was 76%, 94%, and 99%,
respectively. In contrast, using a standard regimen of one
75 IU/kg APCC injection every 12 hours, the probability of bleed
resolution at the corresponding times was 40%, 60%, and 79%,
respectively. CONCLUSION: The present Bayesian meta-
regression suggests that, despite the outcome of the FENOC
study, on-demand treatment with rFVIIa may be associated with
a faster time to joint bleed resolution than APCC using the
standard treatment regimens speciﬁed. Future research should
include time-independent confounding factors (e.g., efﬁcacy
rating scale method, efﬁcacy rater, treatment setting, bleed
severity).
PHM18
BEST-WORST CASE SCALING IN DISCRETE CHOICE
EXPERIMENTS:AN APPLICATION IN A RARE DISEASE
POPULATION
Lee WC1, Joshi AV2, Pashos CL3
1Abt Associates Inc, Bethesda, MD, USA, 2Novo Nordisk Inc,
Princeton, NJ, USA, 3Abt Associates Inc, Lexington, MA, USA
OBJECTIVES: Although discrete choice experiments are
increasingly being used in health care applications to elicit pref-
erences, their use has been limited in diseases with low preva-
lence because it is challenging to design a statistically and
clinically meaningful study with a small sample of respondents.
This study used best-worst case scaling to enhance the design of
a discrete choice experiment eliciting physician preferences
related to the care of patients being treated for hemophilia with
inhibitors. METHODS: Thirty hematologists provided data on
factors having an impact on their treatment decisions by com-
pleting a survey instrument via face-to-face interviews at a sci-
entiﬁc meeting. To increase the amount of useful information
obtained from each respondent, best-worst scaling was used.
Speciﬁcally, each choice task was structured so that respondents
provided input on and ranked three scenarios from most to
least preferred. This innovative method had not previously been
used in discrete choice modeling. RESULTS: With the increase
in data due to the applied best-worse scaling method, an aggre-
gate multinomial logit model established stable parameter esti-
mates while obtaining a ‘consensus’ view of hematologists’
preferences. In substantive terms, the time required to stop
bleeding was the most important factor affecting treatment
decisions [relative importance (RI) = 16.3%]. Physicians also
preferred treatments that resulted in quick pain relief
[RI = 12.9%]. CONCLUSION: This example indicates that
best-worst case scaling can effectively be used in discrete choice
experiments involving aggregate multinomial logit modeling.
This method can enhance and increase the use of discrete
choice experiments to elicit preferences from relatively small
numbers of physicians or patients with rare diseases, or when
few respondents are available.
PHM19
USINGTHE EQ-5DTO MONITOR HEALTH-RELATED
QUALITY OF LIFE OVERTIME INTHE CATALAN HEALTH
INTERVIEW SURVEY
Herdman M1, Ferrer M2, Rajmil L3, Brugulat P4,Vilagut G2,
Medina A4, Cunillera O2,Tresserras R4,Alonso J2
1CIBER en Epidemiologia y Salud Pública (CIBERESP), Barcelona,
Cataluña, Spain, 2IMIM-Hospital del Mar, Barcelona, Cataluña, Spain,
3Agency for Quality, Research, and Assessment (AQuRA) in Health,
Barcelona, Cataluña, Spain, 4Departament de Salut, Barcelona,
Cataluña, Spain
OBJECTIVES: Health Related Quality of Life (HRQL) mea-
sures are infrequently used to track changes in population
health. This study aimed to monitor the evolution of HRQL
between 1994 and 2006 in Catalonia (Spain) using the EQ-5D.
METHODS: EQ-5D measures HRQL in 5 dimensions and pro-
vides a preference-based index from 1 (best health status) to
negative values, with 0 equal to death. The index used in the
present study was constructed from time trade-off (TTO) values
from a sample of the Catalan general population. The Catalan
Health Interview Survey (CHIS) is a cross-sectional study of a
representative sample of the non-institutionalised general popu-
lation. The EQ-5D was administered to respondents aged 15
and over in the 1994 (n = 12,567), 2002 (n = 7,157), and 2006
(n = 15,926) editions. Analyses were performed using weights
to restore general population representativeness. Mean
(95%CI) EQ-5D index scores and prevalence of problems on
EQ-5D dimensions were calculated for groups deﬁned by
gender and age. RESULTS: Mean EQ-5D index scores
decreased over the 3 editions in men and women and all age
groups. Reductions in HRQL between 1994 and 2006
exceeded the minimal clinically important difference on the EQ
index (0.07) in women aged 65–74 (mean [95%CI] index
scores of 0.76 [0.73, 0.79] and 0.66 [0.63, 0.70] in 1994 and
2006, respectively), and in women and men aged 75 years
(0.67 [0.63, 0.70] and 0.49 [0.46, 0.52] in women; 0.79
[0.76,0.83] and 0.70 [0.67,0.73] in men, respectively, for the
two editions). Rates of respondents reporting problems
increased in all dimensions, particularly in women and in self-
care, usual activities, and anxiety/depression. CONCLUSION:
Using the EQ-5D, HRQL appeared to worsen from 1994 to
2006 in this population, particularly in older age groups.
Further research should determine the extent to which results
are due to increased health problems and/or changes in report-
ing patterns and expectations.
PHM20
VALUE OFTRANSFUSION-FREE LIVING IN MDS: RESULTS OF
HEALTH UTILITY INTERVIEWS WITH MDS PATIENTS
Goss TF1, Szende A2, Schaefer C1, Knight R3, Heptinstall K4,
Lübbert M5, Deschler B5, Fenaux P6, Mufti GJ7, Killick S8, List A9
1Covance Market Access, Gaithersburg, MD, USA, 2Covance Market
Access, Leeds, UK, 3Celgene Corporation, Summit, NJ, USA, 4MDS
Foundation, Crosswicks, NJ, USA, 5University of Freiburg, Freiburg,
Germany, 6Hôpital Avicenne, University of Paris XIII, Bobigny, France,
7King’s College Hospital, London, UK, 8Royal Bournemouth Hospital
Foundation Trust, Bournemouth, UK, 9University of South Florida,
Tampa, FL, USA
OBJECTIVES: To elicit how MDS patients value transfusion
independence (TI), reduced transfusions (RT) and transfusion-
dependence (TD). METHODS: Forty-seven MDS patients were
interviewed, US (n = 8), France (n = 9), Germany (n = 9) and the
UK (n = 21), to elicit the utility value of TI, RT and TD. Health
states (HS), based on literature, focus groups and validated by a
Abstracts A285


PHM19 USING THE EQ-5DTO MONITOR HEALTH-RELATED QUALITY OF LIFE OVERTIME IN THE CATALAN HEALTH INTERVIEW SURVEY 

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PHM19 USING THE EQ-5DTO MONITOR HEALTH-RELATED QUALITY OF LIFE OVERTIME IN THE CATALAN HEALTH INTERVIEW SURVEY

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