| Literature DB >> 24479410 |
Mickaël Hiligsmann, Benedict G Dellaert, Carmen D Dirksen, Trudy van der Weijden, Stefan Goemaere, Jean-Yves Reginster, Verity Watson, Annelies Boonen.
Abstract
INTRODUCTION: The patient's perspective is becoming increasingly important in clinical and policy decisions. In this study, we aimed to evaluate the preferences of patients with, or at risk of, osteoporosis for medication attributes, and to establish how patients trade between these attributes.Entities:
Mesh:
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Year: 2014 PMID: 24479410 PMCID: PMC3979104 DOI: 10.1186/ar4465
Source DB: PubMed Journal: Arthritis Res Ther ISSN: 1478-6354 Impact factor: 5.156
Attributes and levels for osteoporosis drug treatment
| Efficacy in reducing the risk of future fractures | 20% |
| | 30% |
| | 40% |
| | 50% |
| Possible side effects (affecting one in 50 patients) | Gastrointestinal disorders |
| | Flu-like symptoms |
| | Skin reactions |
| Mode of administration | Oral tablet |
| | Subcutaneous injection |
| | Intravenous injection |
| Frequency of administration | Weekly |
| | Monthly |
| | Every 3 months |
| | Every 6 months |
| | Yearly |
| Cost to you (per month) | €5 |
| | €15 |
| | €25 |
| | €40 |
| €60 |
Figure 1Example of a choice set.
Patients’ characteristics
| Age (years, mean ± standard deviation) | 67.1 ± 10.4 |
| Female gender | 83.3% |
| Educational level | |
| Primary | 8.4% |
| Some high school | 35.9% |
| High school graduate | 30.3% |
| College or university | 25.5% |
| Size of household | |
| One person | 29.9% |
| Two people | 55.1% |
| Three or more people | 15.0% |
| Monthly household income | |
| Up to €999 | 5.5% |
| €1,000 to 1,499 | 33.1% |
| €1,500 to 1,999 | 19.1% |
| €2,000 to 2,499 | 17.8% |
| €2,500 to 2,999 | 11.9% |
| €3,000+ | 12.7% |
| Diagnosis of osteoporosis | 89.8% |
| Years since osteoporosis (mean ± standard deviation) | 8.9 ± 0.3 |
| With prior fracture(s) | 52.5% |
| In the last year | 22.8% |
| Patients on osteoporotic treatment | 69.8% |
| Administration mode of current treatment | |
| Oral | 72.2% |
| Subcutaneous injection | 15.4% |
| Intravenous injection | 12.4% |
| Number of co-treatments | |
| Zero or one | 19.3% |
| Two or three | 40.6% |
| Four or more | 40.2% |
| 10-year probability of a major osteoporotic fracture (FRAX) (mean ± standard deviation) | 14.3 ± 7.5% |
| 10-year probability of a hip fracture (FRAX) (mean ± standard deviation) | 6.1 ± 5.3% |
Results from the panel mixed logit model
| Constant | 0.90*** (0.62 to 1.17) | 0.00 | – |
| Efficacy (1% risk reduction) | 0.07*** (0.05 to 0.08)a | 0.00 | 1.19*** (1.06 to 1.30) |
| Cost per month (€1) | -0.05*** (–0.04 to –0.06)a | 0.00 | 1.24*** (1.09 to 1.39) |
| Drug administration (reference level: weekly oral tablet) | |||
| Monthly oral tablet | 0.69*** (0.36 to 1.03) | 0.00 | 0.92*** (0.65 to 1.19) |
| Subcutaneous injection 3-monthly | 0.16 (–0.09 to 0.42) | 0.21 | NSb |
| Subcutaneous injection 6-monthly | 0.75*** (0.44 to 1.07) | 0.00 | NS |
| Intravenous injection 3-monthly | -0.57** (–1.12 to –0.01) | 0.05 | 2.62*** (2.04 to 3.20) |
| Intravenous injection yearly | 0.28 (–0.12 to 0.68) | 0.17 | 1.56*** (1.17 to 1.94) |
| Side-effects (reference level: gastrointestinal disorders) | |||
| Flu-like symptoms | 0.97*** (0.76 to 1.18) | 0.00 | 0.90*** (0.65 to 1.15) |
| Skin reactions | 0.63*** (0.41 to 0.85) | 0.00 | 1.04*** (0.81 to 1.26) |
Number of observations 3,822 (257 respondents × 15 choices, minus 33 missing values).
Pseudo-R2 = 0.42; log-likelihood = –2,456.03; Akaike information criterion = 1.29.
aFor the coefficients of efficacy and cost to you, exp(β) is shown. The standard deviation of the log-normal distribution is reported. bNot significant and not included in the final model. **P < 0.05. ***P < 0.01.
Willingness to pay and willingness to trade efficacy for osteoporosis medication attributes
| Efficacy (1% risk reduction) | 3.73 (3.01 to 4.44) | – |
| Cost (€1) | – | -2.27 (–1.58 to –2.96) |
| Drug administration (reference level: weekly oral tablet) | | |
| Monthly oral tablet | 16.16 (12.85 to 19.47) | -10.16 (–7.88 to –12.50) |
| Subcutaneous injection 3-monthly | 4.24 (3.72 to 4.76) | -2.93 (–2.57 to –3.30) |
| Subcutaneous injection 6-monthly | 19.53 (17.15 to 21.92) | -13.52 (–11.82 to –15.22) |
| Intravenous injection 3-monthly | -15.28 (–23.23 to –7.34) | 8.66 (14.31 to 3.01) |
| Intravenous injection yearly | 11.75 (5.64 to 17.85) | -5.83 (–1.88 to –9.77) |
| Side-effects (reference level: gastrointestinal disorders) | | |
| Flu-like symptoms | 25.21 (13.06 to 20.50) | -16.68 (–14.20 to –19.16) |
| Skin reactions | 16.78 (13.06 to 20.50) | -9.48 (–7.13 to –11.83) |
Data presented as mean (95% confidence interval). A positive willingness to pay means that patients are willing to pay a personal contribution for the attribute/level, while a negative willingness to trade efficacy means that patients are willing to give up treatment efficacy for the attribute/level. aUsing the conditional constrained distribution.
Differences between high-risk and low-risk patients’ preferences for osteoporosis drug treatment
| Number of patients | 139 | 114 | |
| Pseudo- | 0.39 | 0.42 | |
| Log-likelihood | -1,378.35 | -1,085.55 | |
| Constant | 1.50*** (1.17 to 1.83) | -0.05 (–0.52 to 0.43) | 0.01 |
| Efficacy (1% risk reduction) | 0.04*** (0.03 to 0.04) | 0.14*** (0.11 to 0.17) | 0.00 |
| SD: 1.65*** | SD: 1.01*** | ||
| Cost per month (€1) | -0.02*** (–0.02 to –0.03) | -0.08*** (–0.06 to –0.09) | 0.00 |
| SD: 1.45*** | SD: 0.67*** | ||
| Drug administration (reference level: weekly oral tablet) | | ||
| Monthly oral tablet | 0.57** (0.08 to 1.06) | 1.14*** (0.47 to 1.82) | 0.14 |
| SD: 0.94*** | SD: 1.87*** | ||
| Subcutaneous injection 3-monthly | 0.14 (–0.19 to 0.47) | 0.28 (–0.17 to 0.74) | 0.14 |
| SD: NS | SD: NS | ||
| Subcutaneous injection 6-monthly | 0.57*** (0.17 to 0.96) | 1.55*** (0.97 to 2.14) | 0.06 |
| SD: NS | SD: NS | ||
| Intravenous injection 3-monthly | -0.28 (–0.88 to 0.31) | -0.24 (–1.39 to 0.91) | 0.25 |
| SD: 1.82*** | SD: 4.84*** | ||
| Intravenous injection yearly | 0.28 (–0.13 to 0.69) | 0.75** (0.05 to 1.45) | 0.33 |
| SD: 0.81*** | SD: 2.15*** | ||
| Side effects (reference level: gastrointestinal disorders) | | ||
| Flu-like symptoms | 0.66*** (0.36 to 0.95) | 1.51*** (1.07 to 1.95) | 0.57 |
| SD: 0.91*** | SD: 1.18*** | ||
| Skin reactions | 0.45** (0.05 to 0.85) | 0.49** (0.10 to 0.87) | 0.05 |
| SD: 1.31*** | SD: 1.04*** | ||
Data presented as estimate (95% confidence interval). SD, standard deviation; NS, not significant. P value was estimated in a joint model with interaction terms. **P < 0.05. ***P < 0.01.