Keeping Surveys Valid,Reliable, and Useful: A Tutorial

This tutorial focuses on how to produce reliable and generalizable data from random‐digit‐dialing (RDD) landline and cell phone surveys. The article notes that RDD response rates have declined and explores the impact of this pronounced decline. The tutorial addresses order, response mode, and many other biases, sample size, cooperation and response rates, weighting, and hybrid designs‐all using examples from risk analysis to illustrate the key points. The article ends with a brief review of the advantages and disadvantages of major Internet and paper surveys tools, and how these can be molded and sometimes combined in repeated, longitudinal, and other designs to answer questions about risk preferences and perceptions.     

Back to basics: explaining sample size in outcome trials,are statisticians doing a thorough job?

Time to event outcome trials in clinical research are typically large, expensive and high‐profile affairs. Such trials are commonplace in oncology and cardiovascular therapeutic areas but are also seen in other areas such as respiratory in indications like chronic obstructive pulmonary disease. Their progress is closely monitored and results are often eagerly awaited. Once available, the top line result is often big news, at least within the therapeutic area in which it was conducted, and the data are subsequently fully scrutinized in a series of high‐profile publications. In such circumstances, the statistician has a vital role to play in the design, conduct, analysis and reporting of the trial. In particular, in drug development it is incumbent on the statistician to ensure at the outset that the sizing of the trial is fully appreciated by their medical, and other non‐statistical, drug development team colleagues and that the risk of delivering a statistically significant but clinically unpersuasive result is minimized. The statistician also has a key role in advising the team when, early in the life of an outcomes trial, a lower than anticipated event rate appears to be emerging. This paper highlights some of the important features relating to outcome trial sample sizing and makes a number of simple recommendations aimed at ensuring a better, common understanding of the interplay between sample size and power and the final result required to provide a statistically positive and clinically persuasive outcome. Copyright © 2009 John Wiley & Sons, Ltd.     

A Class of Sampling Two Units with Probability Proportional to Size

A class of sampling two units without replacement with inclusion probability proportional to size is proposed in this article. Many different well known probability proportional to size sampling designs are special cases from this class. The first and second inclusion probabilities of this class satisfy important properties and provide a non-negative variance estimator of the Horvitz and Thompson estimator for the population total. Suitable choice for the first and second inclusion probabilities from this class can be used to reduce the variance estimator of the Horvitz and Thompson estimator. Comparisons between different proportional to size sampling designs through real data and artificial examples are given. Examples show that the minimum variance of the Horvitz and Thompson estimator obtained from the proposed design is not attainable for the most cases at any of the well known designs.     

On Approximability of Boolean Formula Minimization

For a Boolean function given by a Boolean formula (or a binary circuit) S we discuss the problem of building a Boolean formula (binary circuit) of minimal size, which computes the function g equivalent to , or -equivalent to , i.e., . In this paper we prove that if P NP then this problem can not be approximated with a good approximation ratio by a polynomial time algorithm.     

Modified Wald statistics for generalized linear models

Summary: Wald statistics in generalized linear models are asymptotically ² distributed. The asymptotic chi–squared law of the corresponding quadratic form shows disadvantages with respect to the approximation of the finite–sample distribution. It is shown by means of a comprehensive simulation study that improvements can be achieved by applying simple finite–sample size approximations to the distribution of the quadratic form in generalized linear models. These approximations are based on a ² distribution with an estimated degree of freedom that generalizes an approach by Patnaik and Pearson. Simulation studies confirm that nominal level is maintained with higher accuracy compared to the Wald statistics.     

Center and Distinguisher for Strings with Unbounded Alphabet

Consider two sets and of strings of length L with characters from an unbounded alphabet , i.e., the size of is not bounded by a constant and has to be taken into consideration as a parameter for input size. A closest string s* of is a string that minimizes the maximum of Hamming¹ distance(s, s*) over all string s : s . In contrast, a farthest string t* from maximizes the minimum of Hamming distance(t*,t) over all elements t: t . A distinguisher of from is a string that is close to every string in and far away from any string in . We obtain polynomial time approximation schemes to settle the above problems.     

Estimating population size with truncated sampling

Let X₁, X₂,…,X_n be independent, indentically distributed random variables with density f(x,θ) with respect to a σ-finite measure μ. Let R be a measurable set in the sample space X. The value of X is observable if X ? (X?R) and not otherwise. The number J of observable X’s is binomial, N, Q, Q = 1?P(X ? R). On the basis of J observations, it is desired to estimate N and θ. Estimators considered are conditional and unconditional maximum likelihood and modified maximum likelihood using a prior weight function to modify the likelihood before maximizing. Asymptotic expansions are developed for the [Ncirc]’s of the form [Ncirc] = N + α√N + β + o_p(1), where α and β are random variables. All estimators have the same α, which has mean 0, variance σ² (a function of θ) and is asymptotically normal. Hence all are asymptotically equivalent by the usual limit distributional theory. The β’s differ and Eβ can be considered an “asymptotic bias”. Formulas are developed to compare the asymptotic biases of the various estimators. For a scale parameter family of absolutely continuous distributions with X = (0,∞) and R = (T,∞), special formuli are developed and a best estimator is found.     

Adapting by calibration the sample size of a phase III trial on the basis of phase II data

The problem of estimating the sample size for a phase III trial on the basis of existing phase II data is considered, where data from phase II cannot be combined with those of the new phase III trial. Focus is on the test for comparing the means of two independent samples. A launching criterion is adopted in order to evaluate the relevance of phase II results: phase III is run if the effect size estimate is higher than a threshold of clinical importance. The variability in sample size estimation is taken into consideration. Then, the frequentist conservative strategies with a fixed amount of conservativeness and Bayesian strategies are compared. A new conservative strategy is introduced and is based on the calibration of the optimal amount of conservativeness – calibrated optimal strategy (COS). To evaluate the results we compute the Overall Power (OP) of the different strategies, as well as the mean and the MSE of sample size estimators. Bayesian strategies have poor characteristics since they show a very high mean and/or MSE of sample size estimators. COS clearly performs better than the other conservative strategies. Indeed, the OP of COS is, on average, the closest to the desired level; it is also the highest. COS sample size is also the closest to the ideal phase III sample size M_I, showing averages and MSEs lower than those of the other strategies. Costs and experimental times are therefore considerably reduced and standardized. However, if the ideal sample size M_I is to be estimated the phase II sample size n should be around the ideal phase III sample size, i.e. n?2M_I/3. Copyright © 2010 John Wiley & Sons, Ltd.     

Some sampling effects of pairwise correlated observations on likelihood ratio tests for the difference between two means

We study the effects of the inclusion of pairs of correlated observations in a sample on likelihood ratio tests for the difference in two means. In particular, we assess how the inclusion of correlated data pairs (e.g., such as data inadvertently collected from sib-pairs) affects the sample size requirements necessary for the implementation of a Likelihood Ratio (LR) test for the difference between two means. Our results suggest that correlated data pairs beneficially or adversely effect sample size requirements for an LR test to a degree functionally related to the mixture parameters dictating their relative frequencies in the larger sample on which the test will be performed, the strength of the correlation between the observations, and the size of imbalances in the sample with respect to the number of observations in each group. The relevance and implications of the results for genetic and epidemiologic research are discussed.     

Reporting cumulative proportion of subjects with an adverse event based on data from multiple studies

Experience has shown us that when data are pooled from multiple studies to create an integrated summary, an analysis based on naïvely‐pooled data is vulnerable to the mischief of Simpson's Paradox. Using the proportions of patients with a target adverse event (AE) as an example, we demonstrate the Paradox's effect on both the comparison and the estimation of the proportions. While meta analytic approaches have been recommended and increasingly used for comparing safety data between treatments, reporting proportions of subjects experiencing a target AE based on data from multiple studies has received little attention. In this paper, we suggest two possible approaches to report these cumulative proportions. In addition, we urge that regulatory guidelines on reporting such proportions be established so that risks can be communicated in a scientifically defensible and balanced manner. Copyright © 2010 John Wiley & Sons, Ltd.