Normal Distribution
nothing is called 95% until it has been counted
Concept

Estimand — where it appears

The quantity a procedure is an estimate of, stated before the estimate is computed. Under a wrong model it is not the truth's own parameter but the projection the fit converges to, and an interval judged against the wrong one reports a misspecification as a coverage failure.

Named by 16 essays across 8 fields — each of them below, with the objects they name alongside it.

One wrong model, four designs, four slopes. The slope a straight line converges to when the truth is a quadratic, under four covariate distributions, by two routes: the population projection in closed form, and the mean of 2500 fitted slopes at 200 rows apiece. The even spread over [0, 2] gives 1.6000 and the same spread moved to [1, 3] gives 2.6000, while widening it to [0, 4] gives 2.6000 — the same number as the shifted one, because a symmetric design's target is the truth's tangent slope at the design's own mean and does not read the spread at all. An exponential spread with the SAME mean as the first gives 2.6000. So two studies of one world, each fitting the same wrong model, honestly report slopes 1.0000 apart, and neither is making an error.

What a wrong model estimates

A straight line fitted to a curved truth converges on the tangent at its own design's mean. Two honest studies of one world, fitting the same wrong model, report 2.600000 and 1.600000, and neither is in error.

sandwich · Misspecification Three mechanisms leave the slope alone; one does not. The bias of the complete-case slope under each of four missingness rules, counted over 4000 studies of 200 rows at 35.0% missing, with the closed form printed beside each count. Missingness that depends on nothing, on the regressor, or on the second covariate leaves the slope exactly where it was — the closed forms are zero to machine precision and the counts are -0.0005, -0.0005 and -0.0011 against standard errors of about 0.0018. Missingness that depends on the outcome moves it by -0.1635, which is 27.3% of the slope being estimated. The same share of rows is lost in every case.

Three mechanisms and one dataset

Four rules for which outcomes go missing, each calibrated to lose the same 35% of the rows and each leaning on what it reads with the same coefficient. Three leave the fitted slope exactly where it was, and the one that reads the outcome moves it by 0.163531.

missing · Missingness Unrepresentative in every respect but the one that matters. Three properties of the complete cases as the chance of being observed leans harder on the regressor, in closed form, at 35.0% of outcomes missing throughout. The mean of the regressor among the rows kept climbs from 0.0000 to 0.5528 against a population mean of zero, and the mean of the outcome from 0.0000 to 0.3980 above its own. The bias in the fitted slope is exactly zero at every one of the ten settings, because selection acting on the regressor alone leaves the conditional law of the outcome given the regressor untouched and least squares conditions on exactly that. The sample is wrong about almost everything and right about the one quantity being estimated.

Dropping the incomplete rows

Push the missingness until the rows that survive have a covariate mean of 0.543905 against a population zero and a variance of 0.5041 against one, and the fitted slope is still exactly right. Where the rule reads the outcome instead, the same sweep takes coverage to 2.42% at eight hundred rows.

missing · Missingness The estimator has no upper bound on what it costs. What a thinning overlap does to a stabilised inverse-probability estimate of an average effect of 1.0000, over 600 samples of 600 at each of six settings. The spread rises from 0.1965 to 0.8122 and the root mean square error from 0.1964 to 0.9219, so at the thin end the error is very nearly the whole of the quantity being estimated. The lower line is the share of the arm's weighted total the single largest observation owns, averaged over the same draws: 0.69% to 9.78%, and in the worst single draw of the sweep 82.75%. Coverage of the 95% interval goes from 93.7% to 55.5%.

The region with no comparison

A trimmed interval covers the average effect over everybody 90.8% of the time at six hundred rows and 41.0% at nine thousand six hundred, while covering the average effect over the units it kept 94.3% and 96.0% throughout. An interval that gets worse as the sample grows is an interval about something else.

weights · Weighting Four numbers, and only one of them is the question. Four quantities in a population where the effect is not the same for everybody: 40.0% compliers, 25.0% always-takers and 35.0% never-takers, with the compliers carrying an effect 1.0000 larger than everybody else's. The population average effect is 0.5000. The compliers' average effect is 1.1000. A valid instrument converges on 1.1000 — the second of those, not the first, and the two differ by 0.6000. Comparing the treated with the untreated as they stand gives 1.4182, wrong by 0.9182 in the same direction, because always-takers start 1.5000 above never-takers before any treatment happens. The instrument removes the selection and changes the question at the same time, and only one of those is reported.

Whose effect it is

With a perfectly valid instrument and no violation of anything, the estimate converges on 1.1000 where the population average effect is 0.5000. The gap is exactly θ(1 − p_c), the always-takers and never-takers cancel out of both halves of the ratio, and five per cent defiers move the answer to 1.2667.

instrument · Exclusion Two worlds, one Kaplan–Meier curve, two truths. World A gives each subject a frailty with mean one and variance 1, and multiplies both its event hazard (0.35) and its dropout hazard (0.5) by it, so the subjects likeliest to leave are the ones likeliest to fail. World B has independent event and dropout times whose hazards are world A's crude hazards. Kaplan–Meier over 1000 studies of 400 gives the same curve from both — 0.7750 and 0.7763 at t = 1; 0.6627 and 0.6641 at t = 2; 0.5924 and 0.5932 at t = 3; 0.5047 and 0.5042 at t = 5 — and that curve is world B's truth, 0.5052 at t = 5. World A's truth is 0.3636 there. The dashed lines are the two bounds that assume nothing, from every dropout failing on leaving (0.1905 at t = 5) to none ever failing (0.6667).

A dropout the data cannot see

Two worlds leave the same record to the last detail a study can write down — the same times, the same share ending in the event, the same share leaving first — and a log-rank test between them rejects at its own 5% level at every sample size from a hundred to sixteen hundred. Kaplan–Meier converges on 0.5052 at t = 5 from both. The truth is 0.5052 in one and 0.3636 in the other, and what is left to argue about is where between two bounds to stand.

survival · Censoring The damage does not stay in the term that was left out. Where each coefficient lands when the model that fills the missing outcomes and the model that analyses them disagree, over 1500 studies of 200 rows at 35.0% missing and 20 imputations. An imputer that omits a covariate the analysis fits attenuates that covariate's coefficient by exactly the missing fraction — -0.1405 counted against a closed -0.1400 — and pushes the coefficient it did impute on the other way by exactly the product of the omitted coefficient, the covariates' correlation and the missing fraction: 0.0402 counted against 0.0420. Both closed forms come out of the same two-by-two solve. Matching models leave both alone, and so does an imputer that knows more than the analysis.

An imputation model the analysis does not contain

A model that fills the gaps without a covariate the analysis fits attenuates that covariate's coefficient by exactly the missing share, 0.4 to 0.26, and moves the one it did carry by exactly γρf, 0.6 to 0.642. The reverse case is supposed to inflate the interval, and at four strengths of the extra knowledge it does not.

missing · Missingness The risk of one cause, estimated two ways. Two causes of an ending event with constant hazards 0.2 (the one of interest) and 0.3 (the competitor), random dropout at 0.1 and follow-up to 6. The lower line is the cumulative incidence, (0.2/0.5)(1 − e^(−0.5t)), the chance of actually having had this event by t; the dots on it are the Aalen–Johansen estimate over 2000 studies of 300, 0.3670 at t = 5 against 0.3672. The upper line is 1 − e^(−0.2t), and the dots on it are one minus Kaplan–Meier with the competing event treated as censoring: 0.6318 at t = 5 against 0.6321. The second is larger by a factor of 1.722 at t = 5, and it is not an error of estimation. It estimates, correctly, the risk in a population where the competing cause does not exist.

One minus Kaplan–Meier is not a risk

With two ways for observation to end, one minus Kaplan–Meier for one cause reads 0.6318 at t = 5 where the chance of actually having had that event is 0.3670. Added across the two causes, the complements reach 1.4088 — more than the whole cohort. Nothing is estimated badly: the complement estimates, correctly, the risk in a world where the other cause does not exist.

survival · Censoring An estimate reported as a function of an assumption. What the slope really is, against a shift in the outcomes nobody saw — line from the closed form, dots counted over 2000 studies of 200 rows at 35.0% missing. Every point on this line produces exactly the same observed data, and the complete-case estimate is the flat line at 0.5996 regardless. The truth moves at -0.2845 per unit of shift, which is a function of the missingness model and the missing fraction and of nothing that can be estimated: across the swept range the true slope runs from 0.8845 to 0.3155, a span of 0.5691 against a value of 0.60 in the world where the shift is zero. Reporting the line is the honest form of the answer.

The mechanism the data cannot see

Two worlds produce identical covariates, identical patterns of what is recorded and identical recorded outcomes, to the last bit. Their true slopes are 0.6 and 0.315452, and the truth moves at 0.284548 per unit of an assumption nothing in the data can inform.

missing · Missingness One treatment, a different hazard ratio at every follow-up. The hazard ratio a Cox model converges to, found as the root of its expected score by numerical integration, as the trial runs longer; dropout at 0.1 throughout. The proportional treatment reads 0.5 at every τ. The waning treatment reads 0.5000 at τ = 1, 0.6362 at τ = 3 and 0.7890 at τ = 8 — the same two arms, the same effect in the same first year, and a number that drifts towards one as later, effect-free events are added to the average. The dots are the mean of 400 Cox fits with 400 subjects an arm: 0.5014 at τ = 1, 0.7020 at τ = 2, 0.7635 at τ = 3, 0.8034 at τ = 5, 0.8208 at τ = 8. The crossing treatment reads 0.3429 at τ = 1, exactly 1 at τ = 3 by construction, and 1.0611 at τ = 8: beneficial, null or harmful according to when the trial stopped.

The hazard ratio the follow-up chose

A treatment that halves the hazard for one year and then does nothing has a Cox hazard ratio of 0.5000 if the trial stops at one year, 0.7617 at three and 0.8194 at eight. Nothing about the treatment differs between those numbers. When hazards are not proportional the hazard ratio is an average, and the length of follow-up and the dropout rate choose its weights.

survival · Censoring Weights that balance a sample by construction. What three sets of weights leave of the standardised difference between the arms on each covariate, as a root mean square over 1200 samples of 600 units. The true propensity leaves 0.1317 and 0.1186 — a sampling error, since it is right about the population and knows nothing of the draw. A likelihood fit leaves 0.0770 and 0.0657, having absorbed part of the draw's imbalance as a side effect of fitting the treatment. Weights fitted so that each arm's weighted means are the sample's leave 1.4e-14 and 1.2e-14, which is the arithmetic's floor rather than a small number: the largest gap between a weighted arm mean and the sample mean in any draw is 9.8e-14.

A weight fitted to balance

Weights fitted so that each arm's weighted covariate means equal the sample's leave a difference of 1.4×10⁻¹⁴ between the arms and give the estimate a third of the variance of weights fitted by likelihood — 0.011883, within a relative 5.8% of the bound no estimator can beat. In the world where the assignment carries a square nobody named, the same exact balance leaves the square further apart than no weighting at all, and where the outcome carries it too the estimate is wrong by 0.6973 with an interval that covers 1.5%.

weights · Weighting Six cells, and 5% is the right answer in all of them. How often a regression between two independently generated series is called significant at the 5% level, for two worlds and three treatments, at 200 observations. Every pair is independent by construction, so 5% is correct everywhere and every other reading is a failure. Untreated: 82.9% and 100.0%. With a fitted line removed: 74.2% and 33.5%. Differenced: 5.0% and 5.2%. The treatment that controls the rate in both worlds is the one that discards the level and the trend, which is the quantity a study of trending series was about.

The repair that keeps the question

A regression between two independent trending series is significant 82.9% of the time on random walks and 100.0% on trend-stationary ones. Subtracting a fitted line leaves 74.2% and 33.5%; differencing leaves 5.0% and 5.2% and throws away the trend the study was about.

timeseries · Spurious Three contrasts on one dataset, three different splits. The variance-minimising allocation for each of three ways of reporting the same two-arm comparison, against the first arm's proportion, with the second at 0.1. A risk difference wants the arm with the larger p(1 − p) to get more units; a log odds ratio wants it to get fewer, and the two curves are exact reflections of each other in the half line. A log risk ratio wants something else again. At a first-arm proportion of 0.6 they ask for 62.0%, 21.4% and 38.0% of the units. A trial reporting more than one of them cannot be optimal for either.

Two contrasts, one split

A risk difference wants 62.0% of the units in the first arm, a log risk ratio wants 21.4% and a log odds ratio wants 38.0% — on one dataset, with one pair of proportions. The difference's rule and the odds ratio's are exact reflections of each other, so no split can be near-optimal for both.

allocation · Allocation The difference in restricted mean survival at every horizon, in three worlds. Treatment minus control, in closed form, with dropout irrelevant to the truth. The proportional treatment's difference grows to 0.4766 at τ = 3 and the waning treatment's to 0.2675. The crossing treatment's rises to 0.1776 at τ = 2, near where the two survival curves cross, and falls back to 0.1366 at τ = 3. The ticks along the bottom are the eleven horizons, from 0.5 to 3 in quarters, at which a trial below reads its differences.

A horizon chosen after looking

A difference in restricted mean survival read at whichever of eleven horizons looks most convincing rejects 11.24% of trials in which the treatment does nothing, against 4.70% at a horizon fixed in advance. The correlation of the differences across horizons is closed, and the Gaussian process it defines prices the choice at a critical value of 2.317 — which brings the counted size back to 4.99% and keeps 96.92% of the power that a horizon nobody could have known to fix would have had.

survival · Censoring One weighting told the means and one told the second moments, in five worlds. The bias of the fit to balance over 600 samples of 600 units in each world, fitted to the covariates' means and fitted to their means, squares and product. Told the means it is off by -0.0004, -0.0020, 0.0103, 0.6973, 0.2698 in the worlds with no square, a square in the assignment, a square in the outcome, a square in both and a cube in both; told the second moments, by -0.0009, -0.0000, 0.0009, -0.0045, 0.3099. Its interval covers 94.0%, 94.7%, 95.0%, 1.5%, 51.0% and 93.7%, 89.8%, 94.0%, 91.0%, 48.3%.

The moments a balance is told

Weights fitted to balance the covariates' means were wrong by 0.6973 in the world where both the assignment and the outcome carry a square. Told the squares and the product as well, the same construction is off by −0.0045 there and its interval covers 91.0%. The failure moves up a moment rather than away: with a cube in both, the second-moment balance is off by 0.3099 and leaves the cube twice as far apart as no weighting. And where overlap is thin, 37.0% of samples have no such weights at all.

weights · Weighting Each interval covers one question and not the other. Coverage of each interval for the overall mean, scored against both estimands, over 20,000 two-site studies of 10 observations apiece. The fixed-effect interval covers the mean of the two sites in hand 96.37% of the time and the population mean 54.77%. The random-effects interval covers the population mean 94.96% — exactly its level, from one degree of freedom — and over-covers the two sites in hand at 98.25%. Both are correct; they are answers to different questions printed in the same place.

What a two-unit study should report

The fixed-effect interval covers the mean of the two sites in hand 96.37% of the time and the population mean 54.77%. The random-effects interval covers the population mean 94.96% — exactly its level, from one degree of freedom — and is 11.6 times wider.

multilevel · Levels

Named alongside it

The objects these essays reach for when they reach for this one.

Closed formConfidence intervalModel misspecificationCensoringKaplan–MeierMonte CarloNon-identifiabilityComplete-caseConditional distributionEffective sample sizeInverse-probability weightingLeast squares

All concepts