Table 1 Included studies from literature review of methodological development in methods for specifying a target difference
Study | Journal | Method | Methodological development | |
|---|---|---|---|---|
Hedayat | 2015 | Biometrics | Anchor | Variation in threshold-based approach to estimating the MID |
Rouquette | 2014 | Journal of Clinical Epidemiology | Anchor | Use of item response model approach to calculate M(C)ID estimate from anchor assessment |
Zhang | 2015 | Journal of Clinical Epidemiology | Anchor | Assessment of expressing MID as absolute and relative difference |
Hollingworth | 2013 | Clinical Trials | HE (cost utility) | Assessment of cost-utility-based sample size approaches |
Chen MH | 2013 | Clinical Trials | HE (VOI) | VOI for multistage adaptive trials from industry perspective |
Andronis | 2016 | Medical Decision making | HE (VOI) | Expected value of sample information variant estimator |
Breeze | 2015 | Health economics | HE (VOI) | ENBS from pharmaceutical perspective using value-based pricing |
Hall | 2012 | Medical Decision making | HE (VOI) | Expected net present value of sample information approach |
Jalal | 2015 | Medical Decision making | HE (VOI) | Meta modelling approach to calculating the expected value of sample information |
Madan | 2014 | Medical Decision making | HE (VOI) | Efficient approach to calculating the expected value of partial perfect information (applicable to calculating the expected value of sample information) |
Maroufy | 2014 | Journal of Biopharmaceutical Statistics | HE (VOI) | Method for calculating expected net gain of sampling |
Mckenna | 2011 | Medical Decision making | HE (VOI) | Exploration of the role of value of sample information analysis |
Menzies | 2016 | Medical Decision making | HE (VOI) | Expected value of sampling information estimator |
Sadatsafavi | 2013 | Health economics | HE (VOI) | Expected value of sample information variant estimator |
Streuten* | 2013 | Pharmacoeconomics | HE (VOI) | Comprehensive review of VOI methodological developments |
Strong | 2014 | Medical Decision making | HE (VOI) | Meta modelling approach to calculating the expected value of sample information |
Welton | 2014 | Medical Decision making | HE (VOI) | Application of expected value of sampling information to a cluster trial |
Welton | 2015 | Medical Decision making | HE (VOI) | ENBS accounting for heterogeneity in treatment effects |
Willan | 2011 | Pharmacoeconomics | HE (VOI) | Framework for exploring the perspective of societal decision-maker and industry |
Willan | 2012 | Health economics | HE (VOI) | Accounting for between-study variation in value of information approach |
Kirkby | 2011 | BMC Medical Research Methodology | Opinion seeking | Survey approach to estimate MCID in trial setting |
Ross | 2012 | BMC Medical Research Methodology | Opinion seeking | Survey approach to estimate MCID with three treatment options |
Chen H | 2013 | Clinical Trials | Pilot/Preliminary study | Comparison of approaches to using SD from preliminary study (e.g. pilot or phase 2 study) |
Fay | 2013 | Clinical Trials | Pilot/Preliminary study | Variation in approach to using SD from preliminary study (e.g. pilot or phase 2 study) |
Kirby | 2015 | Pharmaceutical statistics | Pilot/Preliminary study | Variation in approaches to discounting evidence from preliminary study |
Sim | 2012 | Journal of Clinical Epidemiology | Pilot/Preliminary study | Inflation factor for pilot study SD estimate for use in trial sample size calculation |
Whitehead | 2016 | Statistics Method in Medical Research | Pilot/Preliminary study | Assessment of size of pilot study needed to inform main trial |
Valentine | 2016 | JCE | SES | Alternative effect size metric proposed |