| Literature DB >> 25726010 |
Esther W de Bekker-Grob1, Bas Donkers2, Marcel F Jonker3, Elly A Stolk3.
Abstract
Discrete-choice experiments (DCEs) have become a commonly used instrument in health economics and patient-preference analysis, addressing a wide range of policy questions. An important question when setting up a DCE is the size of the sample needed to answer the research question of interest. Although theory exists as to the calculation of sample size requirements for stated choice data, it does not address the issue of minimum sample size requirements in terms of the statistical power of hypothesis tests on the estimated coefficients. The purpose of this paper is threefold: (1) to provide insight into whether and how researchers have dealt with sample size calculations for healthcare-related DCE studies; (2) to introduce and explain the required sample size for parameter estimates in DCEs; and (3) to provide a step-by-step guide for the calculation of the minimum sample size requirements for DCEs in health care.Entities:
Mesh:
Year: 2015 PMID: 25726010 PMCID: PMC4575371 DOI: 10.1007/s40271-015-0118-z
Source DB: PubMed Journal: Patient ISSN: 1178-1653 Impact factor: 3.883
Background information and sample size (method) used of published health care-related discrete-choice experiment studies in 2012 (N = 69)
| Item |
|
|---|---|
| Country of origina | |
| UK | 16 (23) |
| USA | 13 (19) |
| Canada | 10 (14) |
| Australia | 7 (10) |
| Germany | 6 (9) |
| Netherlands | 4 (6) |
| Denmark | 3 (4) |
| Other | 19 (28) |
| Number of attributesa | |
| 2–3 | 5 (7) |
| 4–5 | 24 (35) |
| 6 | 25 (36) |
| 7–9 | 17 (25) |
| >9 | 3 (4) |
| Number of choices per respondent | |
| 8 or fewer | 14 (20) |
| 9–16 choices | 47 (68) |
| More than 16 choices | 5 (7) |
| Not clearly reported | 3 (4) |
| Sample size useda | |
| <100 | 22 (32) |
| 100–300 | 28 (41) |
| 300–600 | 17 (25) |
| 600–1,000 | 10 (14) |
| >1,000 | 6 (9) |
| Sample size method useda | |
| Parametric approach | 4 (6) |
| Louviere et al. [ | 3 (4) |
| Rose and Bliemer [ | 1 (1) |
| Rule of thumb | 9 (13) |
| Johnson and Orme [ | 5 (7) |
| Pearmain et al. [ | 2 (3) |
| Lancsar and Louviere [ | 3 (4) |
| Referring to studies | 8 (12) |
| Review studies | 3 (4) |
| Applied studies | 5 (7) |
| Not (clearly) reported | 49 (71) |
aTotals do not add up to 100 % as some studies were conducted in different countries, used a different number of attributes per discrete-choice experiment, used several subgroups of respondents, and/or used multiple sample size methods
Alternatives, attributes and levels for preventive osteoporosis drug treatment, their parameter labels, initial belief about parameter values, and discrete-choice experiment design codes (based on de Bekker-Grob et al. [12])
| Parameter label | Initial belief parameter value | DCE design code | ||
|---|---|---|---|---|
| Alternative | Alternative label | |||
| Constant (i.e., alternative specific constant for drug treatment; intercept) | A | 1.23 | ||
| Alternative 1 | Drug treatment alternative I | 1 | ||
| Alternative 2 | Drug treatment alternative II | 1 | ||
| Alternative 3 | Opt-out alternative | 0 | ||
| Attribute | Attribute levels | |||
| Drug administration | Tablet once a month | |||
| Tablet once a week | B1 | –0.31 | 1 | |
| Injection every 4 months | B2 | –0.21 | 1 | |
| Injection once a month | B3 | –0.44 | 1 | |
| Effectiveness ( %) | C | 0.028 | ||
| 5 | 5 | |||
| 10 | 10 | |||
| 25 | 25 | |||
| 50 | 50 | |||
| Side effect nausea | D | –1.10 | ||
| No | 0 | |||
| Yes | 1 | |||
| Treatment duration (years) | E | –0.04 | ||
| 1 | 1 | |||
| 2 | 2 | |||
| 5 | 5 | |||
| 10 | 10 | |||
| Cost (€) | F | –0.0015 | ||
| 0 | 0 | |||
| 120 | 120 | |||
| 240 | 240 | |||
| 720 | 720 | |||
DCE design
| Choice task | Alternative | Constant | I. Route of drug administration | II. Effectiveness | III. Nausea | IV. Duration | V. Costs | ||
|---|---|---|---|---|---|---|---|---|---|
| A | B1 | B2 | B3 | C | D | E | F | ||
| 1 | 1 | 1 | 1 | 0 | 0 | 5 | 0 | 10 | 120 |
| 1 | 2 | 1 | 0 | 1 | 0 | 10 | 1 | 1 | 240 |
| 1 | 3 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| 2 | 1 | 1 | 0 | 0 | 1 | 5 | 1 | 5 | 720 |
| 2 | 2 | 1 | 0 | 0 | 0 | 10 | 0 | 10 | 0 |
| 2 | 3 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| 3 | 1 | 1 | 0 | 0 | 0 | 25 | 1 | 10 | 240 |
| 3 | 2 | 1 | 1 | 0 | 0 | 50 | 0 | 1 | 720 |
| 3 | 3 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| . | . | . | . | . | . | . | . | . | . |
| . | . | . | . | . | . | . | . | . | . |
| . | . | . | . | . | . | . | . | . | . |
| 16 | 1 | 1 | 0 | 1 | 0 | 10 | 0 | 10 | 720 |
| 16 | 2 | 1 | 0 | 0 | 1 | 25 | 1 | 1 | 0 |
| 16 | 3 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
alternative 1 = drug treatment alternative I; alternative 2 = drug treatment alternative II; alternative 3 = opt-out alternative; values 0 and 1 in column A mean ‘opt-out alternative’ and ‘drug treatment alternative’, respectively; value 1 in columns B1, B2, B3 means ‘tablet every week’, ‘infusion every 4 months’, and ‘infusion every month’, respectively; column C presents how effective (risk reduction of a hip fracture in %) a drug treatment alternative is; values 0 and 1 in column D mean ‘no nausea as a side effect’ and ‘nausea as a side effect’, respectively; column E presents the total treatment duration in years; and the values in column F present the out-of-pocket costs (€)
Minimum sample size required to obtain the desired power level 1−β for finding an effect when testing at a specific confidence level 1−α
|
| 1− | Constant | I. Route of drug administration | II. Effectiveness | III. Nausea | IV. Duration | V. Costs | ||
|---|---|---|---|---|---|---|---|---|---|
| A | B1 | B2 | B3 | C | D | E | F | ||
| 0.1 | 0.6 | 2 | 28 | 72 | 13 | 2 | 1 | 17 | 3 |
| 0.05 | 0.6 | 3 | 43 | 111 | 19 | 2 | 1 | 27 | 4 |
| 0.025 | 0.6 | 4 | 58 | 151 | 26 | 3 | 2 | 36 | 6 |
| 0.01 | 0.6 | 6 | 79 | 205 | 35 | 5 | 3 | 49 | 8 |
| 0.1 | 0.7 | 3 | 39 | 100 | 17 | 2 | 1 | 24 | 4 |
| 0.05 | 0.7 | 4 | 56 | 145 | 25 | 3 | 2 | 35 | 6 |
| 0.025 | 0.7 | 6 | 73 | 190 | 33 | 4 | 3 | 46 | 7 |
| 0.01 | 0.7 | 7 | 96 | 250 | 43 | 6 | 3 | 60 | 10 |
| 0.1 | 0.8 | 4 | 53 | 139 | 24 | 3 | 2 | 33 | 5 |
| 0.05 | 0.8 | 6 | 73 | 190 | 33 | 4 | 3 | 46 | 7 |
| 0.025 | 0.8 | 7 | 93 | 241 | 42 | 5 | 3 | 58 | 9 |
| 0.01 | 0.8 | 9 | 119 | 308 | 53 | 7 | 4 | 74 | 12 |
| 0.1 | 0.9 | 6 | 78 | 202 | 35 | 5 | 3 | 49 | 8 |
| 0.05 | 0.9 | 8 | 102 | 263 | 45 | 6 | 4 | 64 | 10 |
| 0.025 | 0.9 | 10 | 125 | 323 | 56 | 7 | 4 | 78 | 13 |
| 0.01 | 0.9 | 12 | 154 | 400 | 69 | 9 | 5 | 97 | 16 |
Parameter estimates and precision from an actual discrete-choice experiment study [12] relative to those predicted by the sample size calculations
| Attribute | MNL results actual study ( | Predicted results based on 117 subjects | |||
|---|---|---|---|---|---|
| Parameter value | SE | 95 % CI | SE | 95 % CI | |
| Constant (drug treatment) | 1.23 | 0.218 | 0.81–1.66 | 0.109 | 1.02–1.45 |
| Drug administration (base level tablet once a month): | |||||
| Tablet once a week | –0.31 | 0.070 | −0.45 to −0.17 | 0.099 | –0.50 to –0.12 |
| Injection every 4 months | –0.21 | 0.097 | −0.41 to −0.02 | 0.108 | –0.43 to –0.01 |
| Injection once a month | –0.44 | 0.100 | −0.64 to −0.25 | 0.094 | –0.63 to –0.26 |
| Effectiveness (1 % risk reduction) | 0.03 | 0.003 | 0.02–0.03 | 0.002 | 0.02–0.03 |
| Side effect nausea | –1.10 | 0.104 | −1.30 to −0.89 | 0.065 | –1.22 to –0.97 |
| Treatment duration (1 year) | –0.04 | 0.010 | −0.06 to −0.02 | 0.010 | –0.06 to –0.02 |
| Cost (€1) | –0.0015 | 0.0002 | −0.002 to −0.001 | 0.0002 | –0.002 to –0.001 |
CI confidence interval, SE standard error
aNumber of observations 5589 (117 respondents × 16 choices × 3 options per choice, minus 27 missing values), Pseudo R 2 = 0.185, log pseudolikelihood = −1668.7
| The minimum sample size needed for a discrete-choice experiment (DCE) depends on the specific hypotheses to be tested. |
| DCE practitioners should realize that a small size effect may still be meaningful, but that a limited sample size prevents detection of such small effects. |
| Policy makers should not make a decision on non-significant outcomes without considering whether the study had a reasonable power to detect the anticipated outcome. |