Value of information methods for planning and analyzing clinical studies optimize decision making and research planning.

OBJECTIVE The results of two randomized clinical trials (RCTs) demonstrate the clinical effectiveness of alternatives to casting for certain ankle and wrist fractures. We illustrate the use of value of information (VOI) methods for evaluating the evidence provided by these studies with respect to decision making. STUDY DESIGN AND SETTING Using cost-effectiveness data from these studies, the expected value of sample information (EVSI) of a future RCT can be determined. If the EVSI exceeds the cost of the future trial for any sample size, then the current evidence is considered insufficient for decision making and a future trial is considered worthwhile. If, on the other hand, there is no sample size for which the EVSI exceeds the cost, then the evidence is considered sufficient, and no future trial is required. RESULTS We found that the evidence from the ankle study was insufficient to support the adoption of the removable device and determined the optimal sample size for a future trial. Conversely, the evidence from the wrist study was sufficient to support the adoption of the removable device. CONCLUSIONS VOI methods provide a decision-analytic alternative to the standard hypothesis testing approach for assessing the evidence provided by cost-effectiveness studies and for determining sample sizes for RCTs.

[1]  K Claxton,et al.  The irrelevance of inference: a decision-making approach to the stochastic evaluation of health care technologies. , 1999, Journal of health economics.

[2]  Hamid Pezeshk,et al.  Bayesian techniques for sample size determination in clinical trials: a short review , 2003, Statistical methods in medical research.

[3]  K Claxton,et al.  An economic approach to clinical trial design and research priority-setting. , 1996, Health economics.

[4]  John Gittins,et al.  Quantitative Methods in the Planning of Pharmaceutical Research , 1996 .

[5]  Andrew R Willan,et al.  The Option Value of Delay in Health Technology Assessment , 2008, Medical decision making : an international journal of the Society for Medical Decision Making.

[6]  John Gittins,et al.  A Behavioral Bayes Method for Determining the Size of a Clinical Trial , 2000 .

[7]  Howard Raiffa,et al.  Applied Statistical Decision Theory. , 1961 .

[8]  Hamid Pezeshk,et al.  A Fully Bayesian Approach to Calculating Sample Sizes for Clinical Trials with Binary Responses , 2002 .

[9]  Andrew R Willan,et al.  Optimal clinical trial design using value of information methods with imperfect implementation. , 2009, Health economics.

[10]  H. Raiffa,et al.  Applied Statistical Decision Theory. , 1961 .

[11]  Andrew R Willan,et al.  Time and expected value of sample information wait for no patient. , 2008, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[12]  Andrew R Willan,et al.  Expected value of information and decision making in HTA. , 2007, Health economics.

[13]  B. Alman,et al.  A Randomized, Controlled Trial of a Removable Brace Versus Casting in Children With Low-Risk Ankle Fractures , 2007, Pediatrics.

[14]  D. Lindley.  The choice of sample size , 1997 .

[15]  B W Brown,et al.  The sample size for a clinical trial: A Bayesian–decision theoretic approach , 2001, Statistics in medicine.

[16]  A R Willan,et al.  Argon green vs. krypton red laser photocoagulation for extrafoveal choroidal neovascularization secondary to age-related macular degeneration: 3-year results of a multicentre randomized trial. Canadian Ophthalmology Study Group. , 1996, Canadian journal of ophthalmology. Journal canadien d'ophtalmologie.

[17]  Andrew R Willan,et al.  The value of information and optimal clinical trial design , 2005, Statistics in medicine.

[18]  Hamid. Pezeshk,et al.  How large should a clinical trial be , 2000 .

[19]  J Halpern,et al.  Designing a cost-effective clinical trial. , 1995, Statistics in medicine.

[20]  C. J. Adcock,et al.  The choice of sample size. Commentaries. Author's reply , 1997 .

[21]  J Hornberger,et al.  The cost-benefit of a randomized trial to a health care organization. , 1998, Controlled clinical trials.

[22]  Matthew Kowgier,et al.  Determining optimal sample sizes for multi-stage randomized clinical trials using value of information methods , 2008, Clinical trials.

[23]  J. Wright,et al.  Measurement properties of the activities scale for kids. , 2000, Journal of clinical epidemiology.

[24]  A O'Hagan,et al.  Bayesian Assessment of Sample Size for Clinical Trials of Cost-Effectiveness , 2001, Medical decision making : an international journal of the Society for Medical Decision Making.

[25]  M. J. R. Healy,et al.  Economic Choice of the Amount of Experimentation , 1956 .

[26]  Andrew R Willan,et al.  Planned caesarean section versus planned vaginal birth for breech presentation at term: a randomised multicentre trial , 2000, The Lancet.

[27]  K M Thompson,et al.  A dynamic programming approach to the efficient design of clinical trials. , 2001, Journal of health economics.

[28]  R. Goeree,et al.  Cast versus splint in children with minimally angulated fractures of the distal radius: a randomized controlled trial , 2010, Canadian Medical Association Journal.