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

Blinding — where it appears

Keeping the arm a unit was assigned to hidden from the people treating it, assessing it, or deciding who gets enrolled next. It has a second, sharper meaning for a sequential rule: a stopping rule that reads only within-block contrasts is blind to the mean it will report, which is what makes the interval afterwards exact.

Named by 20 essays across 9 fields — each of them below, with the objects they name alongside it.

One experiment, with the blocks getting smaller as the target comes into range. A single run at a requirement of 0.25, with the block sizes 5, 5, 11, 25, 11, 8, 3, 2 and a total of 70 observations in 8 blocks. The rule stops when the observations in hand reach z²σ̂²/d², with σ̂² pooled from the within-block contrasts — an estimate that moves as the run goes on, so the target moves too. Early blocks are large because the target is far away and cannot be overshot; late ones are small because a block is the granularity of the answer. The interval afterwards is built from the 8 block means and from nothing the rule looked at, and it has 7 degrees of freedom against the rule's 62.

A block size that changes

The blinded rule's exactness never needed the blocks to be the same size. Letting the size be chosen from the contrasts as the run goes on leaves the coverage exactly where it was — and runs straight into an identity that says what a schedule can and cannot buy.

pace · Stopping The construction survives a difference of two weighted means. Coverage of δ̂ ± t√(S_D²/H) on b − 1 degrees of freedom, over 900 runs at a requirement of 0.3, where δ̂ is the block differences weighted by h_b = (1/m_A + 1/m_B)⁻¹ and H is their total. The theorem the one-mean field rests on goes through with h_b in place of the block size, and the reason is that the weights a weighted least squares decomposition needs are the inverse variances — which is exactly what h_b is. The stopping rule reads only within-arm within-block contrasts, so it is a function of nothing the interval reports, whatever it does with the block sizes. Each bar is within 2.9% of the level it claims.

A width promised for a difference

The exact fixed-width interval was built for one mean. Two arms make the target 42.7 units of effective size and each unit costs four observations, so the same promise about a difference costs 169.4 rather than 42.7 — and the theorem survives untouched with the harmonic size in place of the block size.

contrast · Width The stopping rule costs more than the weighting does. Coverage over 2000 runs of a trial whose variance ratio drifts by a factor of twenty, at three ways of deciding when to stop. Twelve blocks fixed in advance is the top line and reproduces what a trial of fixed length delivers. Stopping when the reported interval is short enough is the bottom line, and it costs between 3.0% and 5.5% of coverage — including for the rule that is told every block's true ratio, which is what says the shortfall belongs to the stopping and not to the weights. Stopping on a width predicted from the within-arm sums of squares is the middle line, and it is back at the fixed-length values. The standard error on each point is 0.49%.

A width the trial has to stop for

The weighting that covers at 94.9% on twelve blocks covers at 91.5% when the trial stops as soon as its interval is short enough — and so does the rule that is told every block's true variance ratio. The shortfall is the stopping, not the weights.

stop · Stopping What the interim sees, at an effect of 1. The same 60 observations, estimated two ways. Keeping the arms separate gives 0.995, which is σ. Pooling them without separating the arms — the price of staying blind to the comparison — gives 1.114, against the identity √(1 + Δ²/4σ²) = 1.118. The sample size is proportional to the variance, so a blinded design at this effect asks for 25% more units than it needs, and it does so systematically rather than by chance.

Choosing n after looking

Re-estimating the sample size from an interim is the one adaptation with a defence, and the defence is exactly what it costs: an analyst kept blind to the arms measures a spread that contains the effect, so the design overshoots by 1 + Δ²/4σ². Re-estimating the effect instead breaks the error rate.

adaptive · Stopping How wrong the ratio is allowed to be. λ enters only through the weights, so misstating it leaves the estimate unbiased and moves two things — the interval's calibration and its efficiency — both of which are closed forms of the design. Coverage stays at its level over a factor of two in either direction (94.93% at half the truth, 94.27% at twice it) and starts to go at a factor of five. An estimate on hundreds of within-arm degrees of freedom is never wrong by anything like that, which is what makes the feasible rule usable rather than merely definable.

Blinded, and still exact

The one number the exact interval needs is a ratio of within-arm spreads, which is a contrast and contains no mean — so a rule forbidden to look at the effect may compute it, on more degrees of freedom than the interval itself has.

corner · Width Two promises, and no rule here keeps both. A fixed-width procedure promises two things: that the interval covers at its nominal rate, and that it is no wider than the width asked for. Over 1500 runs of the modelled weighting, a rule that stops when the interval it will report is short enough keeps the width — only 2.0% of runs come out wider than 0.34 — and covers at 91.13% against a nominal 95%. A rule that stops on a width predicted from the within-arm sums of squares covers at 94.80% and comes out wider than promised on 42.3% of runs. The two promises are in conflict because keeping the second one exactly requires conditioning on the very quantity that has to be independent of the stopping time for the first.

Stopping on the arms

The width a trial will report is predictable from quantities the interval is not about. A rule that stops on the prediction covers at 94.5% where one that stops on the interval covers at 91.5, and it costs two blocks and half of the width promise.

stop · Width What a rule gives away by being predictable. Minimisation run at every probability from a coin to fully deterministic, over 500 cohorts of 120 at each. The upper line is the share of assignments an investigator who knows the rule and the enrolled patients can name in advance: 49.7% at p = 0.5, which is a coin and cannot be beaten, and 87.6% at p = 1 — short of everything only where the two arms tie and the rule falls back on a coin. The lower line is what that is worth: an investigator who enrols a patient 0.5 of a standard deviation better than average whenever they predict their favoured arm produces a treatment effect of 0.75 where the truth is zero. Nothing about the randomisation was broken; the bias entered through who was enrolled, which is the one thing an allocation rule cannot control. The dashed line is the closed form 2δ(2g − 1).

The rule that can be guessed

A balancing rule improves as it becomes more deterministic, and a deterministic rule can be worked out in advance from information the person enrolling the patient already has. At full determinism 87.6% of assignments are guessable, and an investigator who acts on the guess produces a treatment effect of three quarters of a standard deviation where the truth is zero.

covadapt · Assignment Three sets of weights, five designs, and no estimator that is exact everywhere. Coverage of the same interval under three weightings. h_b is the inverse variance when the arms share a variance or the allocation is constant; equal weights are right when every block has the same two counts; the estimated precision weights are right in the limit and exact nowhere, because the decomposition needs the weights to be the constants they are only estimating. In the corner — two variances, changing sizes, changing allocation — the two exact estimators are the ones that miss, at 98.45% and 95.65%, and the one with no theorem behind it is at 95.05%. That is the whole statement: there is an exact estimator under either condition, and none under both.

Which weights are the inverse variances

There is an exact estimator when the two arms share a variance and another when every block has the same two counts, and between them they cover every trial anybody designs on purpose. In the corner where neither holds, both cover 98.45% instead of 95%, and the only estimator at its level is the one with no theorem behind it.

contrast · Nuisance The weights may not read the block they weight. A weighted least squares decomposition needs weights that are constants, or at least independent of the differences they multiply. One λ̂ pooled across the trial is estimated on hundreds of degrees of freedom and is effectively a constant; a λ̂ estimated inside each block is estimated on that block's own two or three, and is correlated with the difference it weights. Coverage falls from 94.68% to 82.76% — and the interval gets wider while doing it, 0.5163 against 0.3024, which is the signature of weights that are noise.

The condition that cannot be dropped

The weights may not read the block they weight. Estimate the variance ratio inside each block rather than across the trial and the coverage falls to 83% — on an interval that is at the same time seventy per cent wider.

corner · Allocation Two arms leave one degree of freedom per block unaccounted for. Each point is one run. The one-mean field's identity is (b − 1) + (N − b) = N − 1, and every schedule moves along that line rather than off it. Two arms give the rule N − 2b and the interval b − 1, which come to N − b − 1 — short of the N − 2 two arms leave by exactly one per block, since a block's arm counts absorb one degree of freedom each and only one of the two directions carries the difference. The hollow points add what the block sums are worth, b − 1 more, and land on the total. The missing degrees of freedom are not lost; they are in a place the interval has to be shown it may read.

The degrees of freedom in the sums

One arm partitions N − 1 exactly. Two arms give the rule N − 2b and the interval b − 1, which is short by one per block — and the missing ones are in the block sums, which are correlated with the differences at −0.79 and are usable anyway.

contrast · Blocking Exact coverage, at every block size. Coverage of the interval each rule reports, at a nominal 95%, over 2,500 runs each with a standard error of 0.44 points. The blinded rule stops on the within-block contrasts and reports an interval built from the block means, and those two are independent whatever the rule does — so the interval is an ordinary t interval on b − 1 degrees of freedom and its coverage is exact. It is exact at every block size drawn. The interval a practitioner writes at the purely sequential rule's stopping time covers 91.72%, and Stein's two-stage rule is exact for the same reason as the blinded rule and spends 2.10 times the observations to be so. The bars are truncated at 86% so the differences can be seen.

The rule that cannot see the mean

A sequential rule stops when its own estimate of the spread is small, which is more often on the samples whose spread came out low — so the interval afterwards is short. There is a way to keep updating the estimate and stop being able to see the mean at all.

blind · Stopping The overshoot is the last block size and nothing else. A run stops at a multiple of its own block sizes and cannot land between them, so it ends past its own target by about half a block. Fixed sizes overshoot by 1.5, 2.2, 3.1, 4.7, 8.5 observations as the size goes 2, 3, 5, 8, 16. Every schedule here ends in blocks of two and every one of them lands where blocks of two land — 1.62, 1.32, 1.37 against 1.48 — while having spent most of the run inside blocks four and eight times larger. That is the one thing on this page a schedule genuinely takes from both ends.

What a schedule actually buys

Big blocks early and small blocks late is the right instinct and it does not take both ends of the trade, because there are not two ends to take. What it does take is the overshoot — about four per cent of the observations — and a steadier stopping point.

pace · Nuisance What a fixed-width interval covers, by the number of blocks the trial ran before it stopped. Two thousand runs of each rule, the modelled weighting, a promise of 0.34. Reading its report: 4–8 blocks, 22.3% of runs, 78.2%; 9–12 blocks, 16.6% of runs, 90.4%; 13–16 blocks, 18.4% of runs, 96.2%; 17–20 blocks, 17.4% of runs, 96.0%; 21–28 blocks, 17.9% of runs, 96.4%; 29–36 blocks, 7.4% of runs, 99.3% — 91.45% overall. Reading the arms: 4–8 blocks, 0.0%, none; 9–12 blocks, 0.9%, 94.4%; 13–16 blocks, 30.4%, 95.6%; 17–20 blocks, 50.0%, 93.9%; 21–28 blocks, 18.0%, 94.4%; 29–36 blocks, 0.7%, 92.3% — 94.50% overall.

The trials that stopped early

A fixed-width trial that stops when its own interval is short enough covers 91.45% — an average of 78.2% among the 22.3% of runs that stop within eight blocks and 96% to 99% among those that run longer. Widening every interval by 17.1% brings the average to 95% and leaves the early stops at 85.6%, while 92.8% of runs now report an interval wider than the width they promised. Even doubling every interval leaves the early stops short.

stop · Width A wrong weight costs width; a random weight costs level. Five weightings on a trial whose variance ratio drifts by a factor of 20.1 between the first block and the last, over 4000 runs. The rule that knows every λ_b covers at 95.1% and sets the width. One ratio for the whole trial is wrong for every block and costs nothing in level — 94.8% — while being 20% wider; equal weights are calibrated by an identity and 22% wider. The ratio estimated inside each block is the only rule aimed at the quantity that actually varies, and it is the only one that misses the level, at 92.0%: a weight computed from a handful of degrees of freedom is mostly noise, and noise in a weight is not a wrong weight. Modelling the drift across blocks recovers the oracle's width at 94.8%.

A ratio that changes between blocks

A wrong weight costs width and a random weight costs level. The rule aimed at the quantity that actually varies is the only one that misses its own coverage, and the rule that models it across blocks recovers the whole of what knowing it is worth.

blocks · Nuisance What a schedule is allowed to read, and what happens when it reads more. The construction allows the block sizes to be anything at all as long as they are functions of the within-block contrasts, which are independent of every block mean. A schedule that shrinks the block whenever the between-block spread is running above what the contrasts say is a direct attempt to hold down the quantity the interval will be built from, and it succeeds: the estimate lands at 0.8373σ² against the honest 0.9831, and the coverage goes with it. Reading the running mean instead pushes the other way and over-covers — which is not a repair, it is the same violation with the sign reversed, and the level is no longer a property of the procedure at all.

A schedule that reads the mean

The block sizes may be anything at all provided they are functions of the contrasts. Two natural schedules break that, in opposite directions — and the most natural mistake of the three is not a schedule at all but a stopping rule, at 86.87% coverage and fewer observations.

pace · Stopping What a guesser gets, and what a guesser gets for nothing. 600 trials of 150 patients under a fully deterministic rule, with an investigator who knows the rule, the factors and every assignment so far. The guess rate falls with the number of arms — 87.8% at 2, 86.2% at 3, 81.0% at 4 — which reads like a trial getting safer and is not: what a guesser can trade on is the excess over the 50%, 33%, 25% they would get by naming an arm at random, and that goes the other way, from 1.76× chance at two arms to 3.24× at 4. The gap a guesser manufactures between the best and worst arm under a true null is 0.759, 0.733, 0.711 standard deviations — nearly unchanged.

Guessing one arm in three

A balancing rule is guessable because it is balancing. With three arms the next assignment is worked out less often than with two — and by more, relative to what a guesser gets for nothing, and the damage they can do is almost unchanged.

multiarm · Assignment Free until the sums stop seeing what the differences see. Coverage with and without the block sums pooled into the interval's variance estimate. With one effect and one level they are free. With an effect that varies between blocks they are still free, because a block's sum picks that variation up exactly as its difference does. With a level that varies they make the interval 37% wider and conservative. And where the effect falls as the level rises — a ceiling, and not an exotic thing to suppose — the sums carry none of the between-block variation while the differences carry all of it, the pooled estimate is short, and the interval that uses it covers 88.75% on a width 20% narrower than the honest one.

What a two-arm rule may not pool

A spread computed "within the block" without the arm label carries a share of the effect, so the trial runs 173 observations at a null and 282 at an effect of 1.5. The stopping rule is reading the thing it exists to measure, and the phrase that produced it is one word long.

contrast · Allocation What the exactness costs, and the dial it is bought with. The median half-width of the interval each rule reports, at a requirement of 0.4 and a first look after 5 observations. The flat line is the interval a practitioner writes at the purely sequential rule's stopping time, which covers 91.72% rather than 95%. The curve is the blinded rule, which covers its nominal level at every block size: it reads b − 1 degrees of freedom where the other reads n − 1, and pays for the exactness in width. The best block size is 3, at 0.4712. Larger blocks give the stopping rule a better estimate and the interval a worse one, and the two costs go opposite ways, which is what puts the minimum in the middle.

What the blindfold costs

The exactly-covering rule pays for it in the width of the interval, and the block size is a dial between two costs that run in opposite directions. And on an interval whose width was fixed in advance, the same repair buys nothing at all.

blind · Nuisance The fixed-width trial's coverage when the outcomes are not normal, for both stopping rules. normal: stopping on the arms 94.05% after 18.1 blocks, on the report 89.95%; log-normal, skewness 0.95: stopping on the arms 94.70% after 18.5 blocks, on the report 90.80%; log-normal, skewness 2.26: stopping on the arms 94.15% after 19.3 blocks, on the report 90.25%; log-normal, skewness 4.75: stopping on the arms 94.45% after 18.7 blocks, on the report 90.50%; t, five degrees of freedom: stopping on the arms 94.35% after 18.3 blocks, on the report 90.30%; skewness 4.75, arm A only: stopping on the arms 93.80% after 26.0 blocks, on the report 89.90%; skewness 4.75, arm B only: stopping on the arms 93.60% after 14.2 blocks, on the report 89.95%; equal variances, normal: stopping on the arms 94.75% after 11.4 blocks, on the report 90.90%; equal variances, skewness 4.75: stopping on the arms 94.05% after 11.1 blocks, on the report 92.00%.

A width rule on skewed outcomes

The blinded fixed-width rule rests on a within-arm spread being independent of the arm means, which only normal samples guarantee. On outcomes with a skewness of 4.75 the independence fails and the overall coverage barely notices — 93.60% to 94.70% across every shape counted, against 94.05% on normal outcomes. What skew moves is the runs that stop by twelve blocks, which cover about 90% with the skew in one arm, and the trial's length: a variance ratio corrected on normal theory lengthens it from 18.1 blocks to 26.0 with the skew in the first arm and shortens it to 14.2 with the skew in the second.

stop · Width Where the bias lands. The drift in the log variance ratio, fitted across 12 blocks over 4000 trials. E[log λ̂_b] is log λ_b plus ψ(k_B/2) − log(k_B/2) − ψ(k_A/2) + log(k_A/2), which depends on nothing but the degrees of freedom — so the tempting sentence is that it goes into the intercept and leaves the slope alone. It does not, because the blocks alternate between allocations and the alternation is correlated with the covariate being fitted: the lopsided blocks carry 0.5383 of bias and the even ones carry none. Uncorrected the slope reads 1.5597 against a truth of 1.5, which is 8.0 standard errors. Subtracting the two digammas block by block leaves 1.4976.

The bias that lands in the slope

The bias in a log variance estimate depends on nothing but its degrees of freedom, so it goes into the intercept — unless the degrees of freedom alternate with the design, which is exactly what a block-randomised trial makes them do.

blocks · Width

Named alongside it

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

CoverageFixed-width intervalDegrees of freedomStopping ruleNuisance parameterInterval widthBlockingEstimated varianceIndependenceSample sizeVariance ratioWeighted least squares

All concepts