Sample size — where it appears
Named by 88 essays across 37 fields — each of them below, with the objects they name alongside it.
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.
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.
Not half and half
The same units, the same measurements, the same analysis — and a different variance, decided before anything is measured. When the two arms have different spreads the best split is σ₁ : σ₂, equal allocation costs 2(σ₁²+σ₂²)/(σ₁+σ₂)², and at three to one that is a quarter of the experiment.
The observations that repeat each other
Almost every standard error divides by √n, which claims the observations carry independent information. At a lag-one correlation of 0.8 a fifty-point series is worth about six independent observations, and its 95% interval covers 47%.
The slope that borrows
Pooling a mean makes it look as though how much a group borrows depends on how much data it has. Pool a slope instead and the illusion breaks — ten groups with ten observations each can borrow anything from 28% to 91%, decided entirely by where those ten observations were placed.
What the 95% refers to
An interval that claims 95% is making a checkable statement about a procedure, not about the interval in front of you. Build every possible sample and count, and the interval taught first turns out to cover 87.6% of the time.
What a prior is worth
A prior is not a philosophical position, it is a component with a stated size. For a proportion it is worth exactly a + b observations, which turns "how much does the prior matter" from an argument into a subtraction.
Sums of almost anything
The theorem says sums converge on one shape whatever they are sums of, which is remarkable and true. Watching it happen from a one-sided skewed source, with the rate of convergence predicted in advance, is more convincing than watching the shape appear.
Two standard deviations of what
The 95.45% inside two standard deviations is a fact about a curve whose centre and width are given. Drawn from ten observations, the same band holds 91.1% on average and less than 95% on 59.9% of samples — and the average is the reading that hides it.
The reversal a coin cannot prevent
Randomisation removes Simpson's reversal in expectation, which is not the same as removing it. A correctly randomised trial of eighty units, on a population where the treatment helps in both groups, reports it losing overall on 3.40% of trials — and stratifying the randomisation takes that to zero at every size.
Where the two tails disagree
A 95% t interval on an exponential source at 120 observations covers 94.81%, which reads as very nearly right. It misses below the mean on 4.08% of samples and above on 1.11% — one tail 63% too heavy and the other 56% too light, and the total is the statistic that hides it.
The spread a pilot supplies
A trial sized for 80% power from a pilot's standard deviation is sized from an estimate that is too small more often than not. With a pilot of ten, 55.9% of the trials it sizes have less than 80% power and 11.1% less than 50%, although the planned sample is right on average. Sizing from the pilot's 80% upper confidence limit instead leaves 19.8% short, at 1.65 times the sample; from its 90% limit, 10.0% short at 2.12 times.
More data is not monotonically better
Coverage of an interval for a proportion does not improve smoothly as the sample grows. It oscillates, and there are larger samples that cover materially worse than smaller ones — a sample of twenty covers twelve points worse than a sample of nineteen.
One number for a table of candidates
An effective sample size is a real quantity, it is exactly right about one thing, and that thing is a mean. Substituted into Akaike's criterion it changes nothing at all, because the penalty it is meant to fix has no sample size in it.
The cost of a unit
Change the constraint from units to money and the allocation rule changes with it — from σᵢ to σᵢ/√cᵢ, which can point the other way. An arm that is noisy and expensive gets fewer units than the same arm would if the money were not the thing running out.
The tail converges last
The central limit theorem is usually shown as a shape arriving. What the demonstration leaves out is the rate — and the rate is wildly different in the middle and in the tail, which is where every approximation in the subject is actually read.
![No block size is best at both things the procedure claims. Two claims and one dial. The honest interval's half-width falls as the blocks get smaller, because the interval's degrees of freedom are the number of blocks: 0.2602 at blocks of two against 0.2933 at blocks of sixteen. The fixed-width claim — that the mean is within 0.25 of the truth — gets more reliable as they get larger, because the sample size is less variable: 92.40% against 95.00%. Both are computed from the same runs, and the second is reproduced to within a tenth of a point by E[2Φ(d√N/σ) − 1], which needs the sample-size distribution and nothing else. The schedules sit at the bottom left: as narrow as the smallest fixed block and as few observations, with the rule's spread estimate on half as many degrees of freedom again.](/f/no-block-size-is-best-at-both-things-the-procedure-claims-c75edaf0.svg)
Two degrees of freedom, one total
The block size is a dial, and the two things a fixed-width procedure claims move in opposite directions along it. Divide the width by the square root of the sample size and one of them turns out to depend on the number of blocks and on nothing else.
What a credible interval covers
A credible interval makes the statement everyone wants and does not claim to have a coverage. It has one anyway, it can be summed over the sample space exactly, and on a reasonable prior it beats the interval taught first.
What a p-value does not say
The same p of 0.04 corresponds to a large effect in ten observations and a negligible one in two thousand. A p-value alone cannot be interpreted, and the number that makes it interpretable is almost never printed beside it.
The two worlds that look the same
Three causal structures were fitted to one covariance matrix and agree with it to 4.4·10⁻¹⁶. The regression returns 0.5000 under all three; the effect they hold is 0.5000, 0.8481 and 0.8481. What separates structures is a missing edge, and the signature of one is a correlation of exactly zero.
A tenth as wide, and both of them right
The interval for a mean and the interval for one future observation are both labelled 95%, and at a hundred observations one is 10.05 times the other — exactly the square root of n + 1. Read the narrow one as the wide one and it covers a new value 15.7% of the time.
The t statistic wearing different clothes
For a simple regression, t² = (n − 2)R²/(1 − R²), exactly, on every dataset — checked to sixteen significant figures over five hundred fits. So a paper reporting R² and a p-value has reported one number twice, and two studies with the same R² have points four times further from the line.
A degrees of freedom that is not a count
The pooled two-sample test's size runs from 0.55% to 18.91% across forty units split five ways against five variance ratios, with a true null in every cell. Welch's runs from 4.63% to 5.51% — bought with a degrees of freedom that is a function of the data, not an integer, and not a count of anything.
The chance a trial succeeds
A trial of sixty-four per arm has 80% power at an effect of half a standard deviation. If the effect is only believed to be about half a standard deviation, give or take a quarter, the chance the trial reaches significance is 69.2%; give or take a half, 61.4%. Reaching 80% then takes 113 per arm, or 1,268 — and when the belief is uncertain by three quarters of a standard deviation no number of patients reaches 80%, because the chance can never exceed the 74.8% probability that the effect is positive at all.
Dropping the losers
Carrying the best of eight arms forward and testing it at 1.96 rejects a true null 10.3% of the time — the hypothesis was chosen by looking at the data, so the statistic is a maximum wearing a single comparison's clothes. The value that holds the rate is 2.313, and it has to be solved for.
R² is not a measure of fit
Adding a predictor with no relationship to anything cannot reduce R², and in expectation raises it by 1/(n − 1). Twenty useless predictors on thirty points give an R² of 0.69 from pure noise.
Stopping when it is precise enough
An experiment that runs until its estimate is precise enough is the natural design and the one with a theorem against it. Its two-stage cousin keeps its promise exactly, for every unknown spread, and pays twice the observations for it.
The price of control
Every correction is paid for in power, and the exchange rate can be measured. Holm buys familywise control for 33 percentage points of power; Benjamini–Hochberg buys a weaker guarantee for 10. Neither is free and neither is a matter of taste.
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.
The winner's curse
Filter honest studies down to the ones that reached significance and the effects they report are systematically too large. At low power the inflation is a factor of two, nobody has done anything wrong, and the selection did all of it.
Twenty intervals and one expected miss
The 95% belongs to the procedure, not to the interval in front of you. Twenty intervals from twenty samples make that visible in a way no definition does, and the one that misses is not a mistake.
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.
What differencing costs
Differencing takes the false-positive rate between two unrelated walks from 76.7% to 4.9%, and takes a genuine relationship's R² from 0.91 to 0.33. Applied to a series that did not need it, it doubles the variance and installs a correlation of −0.5 that the data never had.
What normal actually looks like
A single quantile plot of forty normal points wanders enough to look suspicious. Twenty of them, all genuinely normal, show what the noise looks like — and any single panel a reader would have rejected is in there.
Where the gain is, and where the decision is
A bigger proposal is worth a factor of six at a loose tolerance and nothing at a tight one. The tolerances where it helps are the ones where a hunt costs two evaluations a draw, and the crossing barely moves.
Robust is not free
A robust standard error's promise is asymptotic and its use is not. Its 95% interval covers 88.73% at twenty rows, and under mild heteroskedasticity it is the worse of the two intervals until a hundred.
A coverage table with its own error
Twenty cells estimating the coverage of an interval that is exactly 95%, at a thousand replications each, read from 93.9% to 96.5% — and a table like that flags at least one of its correct cells on 69.9% of honest runs. Ten times the replications does not repair it: at ten thousand the same table still flags one 63.3% of the time.
The effect a stopped trial reports
An O'Brien–Fleming trial at 88.45% power holds its error rate exactly and reports an effect 9.6% too large on average. The 11.39% of trials that stop at the second look report 1.83 times the truth, the ones that cross at the last look report 0.80 times it, and pooling every trial by its size gives the truth back to the last digit.
The prevalence the test has to estimate
Every predictive value takes a prevalence as given, and the prevalence is usually estimated from the same test's positive rate. At a true prevalence of one in a thousand that rate reads 5.09% — fifty times the truth — and the correction that inverts it is unbiased, 18% more variable, and negative on 48.6% of samples of a thousand.
Ninety-three observations, and nothing assumed
The interval between the smallest and largest of a sample holds a share of the population whose distribution does not depend on the population — Beta(n − 1, 2), for anything continuous. Buying the 95/95 that normality buys at ten observations costs 93 of them, and that number is the exchange rate between an assumption and data.
Significant in one, not in the other
Two studies of exactly the same effect, each with 50% power, disagree about significance half the time — and when they do, the test of the difference between them is significant in 9.75% of cases. A p of 0.01 beside a p of 0.20 is a difference with p = 0.36. Among four subgroups sharing one effect, at least one significant and one not happens 87.5% of the time, and the test that would tell a real difference apart needs four times the sample the effect itself needed.
A league table of a hundred
A hundred groups with sizes from 4 to 400, and a top ten to publish. Ranked by their own means, small groups fill 62.1% of the top ten against their 36.3% share of the true top ten. Ranked by posterior means they fill 13.4%. The ranking built from each group's chance of being in the top ten recovers 5.47 of the true ten, the best of three and barely half; and the group ranked first could hold any rank from 1 to 31.
An outcome cut in two
Replacing a measured outcome with whether it crossed a threshold keeps 63.7% of the information when the cut is at the mean, 34.2% at the top tenth and 13.1% two standard deviations out. A trial that needs 63 patients per arm on the measured outcome needs 102 cut at the mean and 185 cut at one and a half standard deviations. The responder rates that result read as a share of patients who respond — 50.0% against 69.1% — when every patient moved by the same amount; and a cut chosen after looking turns a 5% test into a 17.7% one.
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.
Allocating on a guess
Every allocation rule in this field is a function of quantities the experiment is being run to find out. Fed a pilot's estimate of them, the rule that minimises the variance makes the experiment worse than not bothering — until the arms differ by about a factor of two, which is further than anyone would guess.
How many subjects
Sixty-four per arm for 80% power at half a standard deviation — a power figure that could only be simulated, with nothing to disagree with, until the non-central t was written. Two routes now, agreeing to within the simulation's own error.
The check before the standard error
One number decides whether every interval in an analysis is trustworthy, and the check for it flags a lag-one correlation of 0.5 nine times in ten — and one of 0.2 only one time in five, where the interval already covers 88.6% instead of 95%.
The design that has to be integers
The optimal design is a set of real weights and an experiment is a set of runs, so the theory's answer is never available. Thirteen runs reach 99.77% of it and fourteen reach 99.44% — adding a run makes the design worse per run, and the search that finds it does not always find the same one.
The shortest interval is the one that misses
Four intervals for the same data, with their widths and their coverage measured together. The narrowest is the one that fails its stated level, which is exactly why it looks the most appealing.
Twenty residual plots
Judging whether a residual plot looks wrong requires knowing what a correct one looks like, and almost nobody has seen twenty of those. Here they are, from a model that is exactly right, at the sample size that matters.
Two levels at once
A third level of grouping adds no new arithmetic and produces one number — the design effect — that decides how many independent observations a clustered study is worth. It is the same quantity the time-series field computes for autocorrelated data, arrived at from a completely different picture.
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.
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.
The same draws for both methods
Two intervals computed on the same simulated datasets give a difference in coverage whose variance can be 4.891 times smaller than on separate datasets — or, for a pair that covers different samples, 1.164 times larger. Which one a comparison gets is an exact sum over the counts each interval covers, and a standard error that ignores the sharing covers 100.00% for one pair and 93.07% for the other.
The side a bound is read from
On thirty exponential observations the upper limit of a 95% t interval is exceeded by the true mean 6.38% of the time, against the 2.5% a safety margin set from it assumes. Widen the interval until its total coverage is exactly 95% and the upper limit is still exceeded 4.69% of the time. A symmetric repair fixes the number that is reported and not the one that is used; Hall's transformation, which bends the interval, takes the same rate to 3.31%.
All of the next ten
A warranty, a batch release or a monitoring rule promises something about every one of the next ten observations, not about one. From a sample of ten, the band that holds all ten with 95% probability reaches 3.716 sample standard deviations either side of the mean — already wider than the 3.382 of a tolerance interval for 95% of the population — and it keeps widening: 4.942 for a hundred, 6.008 for a thousand, with no ceiling. A 95% prediction interval, read as the answer, holds all ten 67.9% of the time: more than 0.95 to the tenth power, because the ten succeed and fail together.
The verdicts watch the threshold, not the gap
Two studies whose effects really differ are read through their verdicts as often as through their difference. Halves of an 80%-power study whose effects differ by the whole effect split 72.87% of the time against 49.99% when they do not — a split is never more than 1.86 times as likely under any difference up to twice the effect. And a halving of the effect in two large studies leaves both significant 97.93% of the time, while the test of the difference finds it 80.74%.
A tail the sample never saw
Given the source, the saddlepoint reads the tail of a sum of thirty exponential draws to within a fifth of a per cent six standard deviations out. Given only a sample of thirty from that source, the same tilt reads it at a median of 0.061 of the truth, and nine samples in ten read it low. The error has moved from the approximation to the sample: thirty draws put the skewness at a median of 1.30 against a true 2, and hold nothing past their largest value.
A baseline cut in two
Adjusting a trial's result for a baseline measurement that predicts the outcome shrinks the sample it needs to 1 − ρ² of the unadjusted one. Adjusting for whether that measurement was above its median shrinks it only to 1 − (2/π)ρ² — the cut keeps 63.7% of what the covariate could remove, exactly the fraction a cut outcome keeps. In patients the loss grows with the covariate's strength: a cut costs 12% more patients at a correlation of 0.5, 35% at 0.7 and 2.55 times as many at 0.9, where a simple change from baseline would have done better than the cut adjustment.
An upper limit is the finding
A survey of a thousand people with a 90%-sensitive, 95%-specific test, estimating a prevalence of one in a thousand, prints a corrected 95% interval that covers the truth 95.17% of the time — and reaches below zero in 97.95% of surveys and lies entirely below zero in 3.35%. Coverage is not the problem. The problem is what the interval can say, and the honest summary of a typical survey is one number: the prevalence is below 1.64%. Bringing that limit down to twice the truth takes 184,521 people at this specificity and 5,083 with a test that never gives a false positive.
What a longer sample settles
At a hundred and twenty rows, letting each candidate choose its own whitening changes which of fifteen candidates wins on about an eighth of draws, whatever the list, the table or the law. At four hundred and eighty rows it changes the winner on 1.0% of draws for a window and 2.3% for an order, and at nine hundred and sixty on a quarter of one per cent. The sample settles it through one factor only: how often the candidates disagree about the tuning value hardly moves, and the share of their disagreements that change the winner collapses.
The charge at four times the sample
A band of lags is charged a full unit apiece and is worth about three eighths of one at a hundred and twenty rows. The field's last essay said the error in that convention was harmless only because the error surface is flat, and that a steeper surface would give it a cost. A longer sample was the obvious place for one. It is not there: from 120 rows to 480 the error's spread across band widths falls by three quarters, and the derived charge's advantage over the convention falls from 5.5 standard errors to 1.7. What the sample does move is the charge itself, from 0.37 of a unit a lag towards the half a unit the window's weights predict.
A boundary for giving up
Adding "stop if z is below zero" to an O'Brien–Fleming trial costs 5.20 points of power at the effect it was designed for and halves the observations a trial with no effect uses. Stopping when conditional power at the observed trend falls under 10% costs 13.23 points and stops 21.28% of trials with a real effect. Making that rule binding lowers the benefit boundary from 2.040 to 1.901, and a binding rule that is then ignored rejects a true null 3.523% of the time instead of 2.5%.
The fewest groups that can borrow
At three groups the estimator that shrinks towards its own data's mean returns the group means untouched, on every dataset, because its constant is J − 3. At two it expands instead of shrinking. And the number of groups at which partial pooling starts to be worth doing is five, or two, or never — it depends on how far apart the groups are.
The arm whose variance is its answer
With a binary outcome the allocation rule is a function of the proportions the trial exists to estimate. It costs at most 4.36% of variance to ignore it anywhere between a tenth and nine tenths, because √(p(1−p)) stays within a factor of two of its peak across 98% of the unit interval.
A band allowed a few misses
From ten observations, a band holding every one of the next hundred with 95% probability reaches 4.942 sample standard deviations either side of the mean. Allowing one of the hundred outside brings it to 4.289; allowing five, to 3.378 — the 95%-content tolerance factor, 3.382, to within half a hundredth. The tolerance interval turns out to be the limit of a promise about a share of failures. And the misses arrive together: when this band misses once, it misses again 45.4% of the time, where independent misses at the same rate would do so 12.5%.
How many functions a trial can hold
A rule that reads more functions of one covariate protects more shapes, and every function it reads costs the shapes it already held: the imbalance it leaves in a linear outcome falls like one over the trial size, with a constant that runs from about 3 for one function to about 16 for six. At fifty units that cost eats the protection, and the worst case over four shapes sits between 37% and 42% of a coin's from two functions to six. At four hundred it falls from 42% to 6%. By six functions the worst shape, at every size, is one the rule is reading.
The share a sample removes
A rule balancing eight main effects of two independent covariates removes exactly none of their interaction — in the population. A trial is a sample, and in a sample of a hundred units exact balance on the same eight functions removes 24% of the interaction's variance on average, between 10% and 42% from one trial to the next. At twenty-five units it removes 62%. The zero is the centre of nothing: it is the limit the in-sample share falls towards, by about two fifths with every doubling of the trial, carried there by an interaction whose spread grows with the very functions it is regressed on. At a correlation of a half the closed form says 64%; a trial of a hundred gets 69% on average and anything from 53% to 83%.
A share at a hundred units
On a skewed covariate the closed form credits a rule that balances the means with removing 26% of the covariates' product and 52% of a square, shares that rest on symmetry having failed. A trial does not collect them. At a hundred units the rule removes 12% of the product and 37% of the square on the clearly skewed covariate, and at four hundred units barely more, because the shares live in the long tail and the tail is a handful of units. On the most skewed covariate the rule removes a fifth of the median split at a hundred units and nothing of it at four hundred: balancing the mean of such a covariate is balancing its largest few values.
A cap that decides nothing
Every band-width list this field priced stopped at thirty lags, and long memory at a large sample is the one dependence whose best band wants to be wider. At 960 rows it does: the draw's own best width lies past the cap on 48.7% of draws. Lift the cap to eighty and the measured charge follows, from 28 lags to 46 — and the error moves by two ten-thousandths, in the wrong direction and inside its standard error, because the surface behind the cap is flat. The field's last conditional fails where it had its best chance.
The table decides when the sample settles it
Letting each candidate regression choose its own whitening changes which candidate wins on about an eighth of draws at 120 rows, and a longer sample settles it — for fifteen nested-and-crossed subsets, at 0.25% by 960 rows. A nested sequence of four candidates gets there a sample size sooner, from half the rate. Six candidates that all hold two predictors never get there: at 960 rows their tuning list still changes the winner on 2.3% of draws, nine times the full table's, because a disagreement among candidates of one size goes on deciding the winner as often at 960 rows as at 240.
The correction for not knowing the spread
The t distribution exists because the standard deviation is estimated rather than known. At eight observations, using the normal instead makes every interval 12% too short — and the coverage that follows can be measured rather than argued about.
Where the borrowing goes
Pooling cuts the total squared error across eight groups by 56%. Two of the eight take 61% of that reduction, the four best-measured groups share 11% between them, and the largest group gets 1.5% of what the smallest does. The headline is a fact about the groups nobody was asking about.
A level with two units
A variance estimated from two units is a scaled chi-square on one degree of freedom. Its interquartile range spans a factor of thirteen, its ten-to-ninety range a factor of a hundred and seventy-one, and it comes out exactly zero on 26.7% of studies — so the design effect it decides runs from 1.00 to 7.01 against a truth of 4.69.
The level a limit should be set at
When a failure costs twenty times a standard deviation of margin, a t limit on fifteen exponential observations set at the conventional 97.5% has an expected loss of 2.143. Set at 99.95%, the same formula's loss is 1.263 — within 1% of the best any fixed multiple of s/√n can do, and ahead of both Hall's transformation and the fitted gamma family at their own best levels. Hall's is the worst at every level on every source. Choosing the level does more than choosing the construction, and the correction that reads the sample is the one no level rescues.
A visit before or a visit after
A trial that can afford one more measurement per patient can take it before treatment, to sharpen the baseline adjustment, or after it, to average the outcome. With every pair of visits correlated 0.5, the second visit after treatment removes a quarter of the variance and the second visit before it removes a twelfth, and it is never the other way round: the baseline's contribution is ρ²(1 − ρ)/(1 + ρ), which peaks at ρ⁵ = 0.090 when ρ is the golden ratio's reciprocal. Below a correlation of one half, a single extra follow-up beats any number of baselines. When the correlation fades with the time between visits, averaging an earlier baseline into the adjustment makes the trial less precise, not more.
The threshold a validation study can see
When diseased scores are twice as spread as healthy ones, the harm-minimising threshold jumps near a prevalence of 26% from an interior cut to flagging everyone, because the true operating curve keeps climbing in a tail where few diseased people score. A validation study of a hundred diseased and three hundred healthy cannot see that tail: its own curve reaches 100% sensitivity at its lowest diseased score and goes flat. Just past the jump, only 7.7% of such studies recommend flagging everyone, and their recommended thresholds cause 15.4% more harm than the best on average. Fitting a normal curve to each group first sees the tail: 47.4% of studies then recommend flagging everyone and the excess harm falls to 2.5%. More healthy people in the study change nothing; more diseased people do.
A range allowed a few misses
Without assuming a shape, the band a sample can offer is its own smallest and largest values, and the number of future draws falling outside it is beta-binomial for every continuous population. A promise that at most one of the next hundred lands outside, with 95% probability, then needs 637 observations; none of the hundred, 3,850; at most five, 107. The normal-theory band keeps the one-in-a-hundred promise from ten. On normal data, at the sizes the range needs, it is only 6% wider than the normal band at the same size — the price of dropping the assumption is paid in observations, not in width — and its misses cluster far less than a band from ten: a second follows a first 19.7% of the time, against 45.4%.
A floor that reads the first stage
A three-stage stopping rule fails at a first stage of five because a low first reading asks for nothing more, and the only floors that repair it were set near the answer, by somebody who already knew roughly what the spread was. Inflating the first reading by Stein's own factor needs no such knowledge, and it does cut the experiments left stuck from a fifth to a fifteenth. But those still stuck cover less than before, the rule needs a different dial at every requirement, and a floor of 26 observations covers 96.5% at the spend where the rule covers 92.1%, even met by a spread it was not set for.
A rule that sets its own κ
A stopping rule may read the spread inside its blocks and may not read the spread between them, and the dial between the two costs nothing for a long stretch and then a lot. The share an efficiency argument recommends is each estimate's share of the degrees of freedom, and on blocks of two at a half-width of 0.15 that loses nothing and steadies the experiment by 30%. At 0.35 it loses 1.18 points. Deferring the forbidden reading until twenty blocks costs nothing measurable at any requirement, and it keeps most of the steadiness where steadiness can be had.
When the prior is confident and wrong
A prior worth thirty-five observations, centred in the wrong place, produces a 95% interval that covers nothing at all — and reports a width 5% narrower than an honest one. It takes seventeen thousand observations to repair, not thirty-five, and the worst study to run is the one whose sample size equals the prior's weight, exactly.
A range that grows with the lot
A band set from the range of everything seen so far flags a unit only when it sets a new record, and for any continuous population those flags are independent, with the k-th unit flagged with probability 2/(n + k). That makes a few-misses promise far cheaper than the fixed range's — at most five of the next hundred from 36 starting observations rather than 107 — and exactly as expensive when no miss is allowed, because a lot with no flag never moves the band. The saving is bought by believing every flag. After a two-standard-deviation step in the mean, the fixed range flags 25.03 of the eighty units that follow and the learning range 3.62; by the last twenty units it sees a unit outside in 25.5% of lots, against 18.4% with no step at all.
The correlation a pilot can see
Two models of repeated visits agree that adjacent visits correlate 0.7 and disagree about how many follow-ups a trial should buy: three under equal correlation, one when the correlation fades. Planning on the wrong one costs 28.0% or 9.9%. Two follow-ups cost at most 3.2% more than the best under either, and at most 11.1% under any curve with that adjacent correlation. A pilot of twenty patients at four visits chooses worse on average than that rule when the correlation fades; it takes about forty to draw level with it, and at an effect of half a standard deviation those forty patients' extra visits cost fifty times what the better schedule saves.
A second stage sized by the first
A drop-the-losers trial can size its second stage by what the first stage says about the arms' shape — more patients when the chosen arm's lead looks like luck, fewer when it looks real. The rule does what it was built to do: at sixteen arms with a genuine winner it spends 51.0 patients a group when the interim chose the wrong arm and 24.2 when it chose the right one. And it buys nothing. Against a fixed second stage of the same average size, every estimate of the chosen arm errs by the same to within a few thousandths; the published combination's bias rises from 0.014 to 0.022, and the second stage alone, still unbiased, errs by 0.319 against 0.281.
A failure charged by its size
Charge an upper limit's failure by how far the true mean exceeds it rather than by a fixed amount, and the level a limit should be set at falls further than any other change in this comparison moves it: on fifteen exponential observations, a penalty of twenty per standard deviation of shortfall asks for a t limit at 97% where a penalty of twenty per failure asked for 99.95%. The failures are small whichever limit produces them — about an eighth of a standard deviation — so a charge by size is a small charge per failure. The ranking hardly moves: the t limit stays within about 1% of the fitted family, whose failures are no smaller than its own, and Hall's transformation stays last by 11%.
A level set before the source is known
Every best level for an upper limit on skewed data was found knowing the source, and the fifteen observations that would tell the sources apart are the ones being used to set the limit. Chosen instead to protect the worst of four skewed sources, the t limit's level is 97.25% when a failure is charged by the size of its shortfall, and it costs at most 0.8% more than each source's own best — the sources disagree about the best level and agree that missing it a little is cheap. The fitted gamma family, the construction meant to adapt to the source, is the one that needs it: its best levels run from 90% to 96.5% and its minimax level costs 3.4%. Charged a fixed amount per failure, the t limit's minimax level is 99.97% and costs 6.5%; the conventional 97.5% costs up to 119.9%.
Patients spent before the choice
A trial that picks the best of eight arms and then confirms it splits its patients between choosing and estimating, and the familiar shape — sixty an arm before the choice, thirty after — spends ninety per cent of a six-hundred-patient budget on the choice. Measured against the effect a reader is told about, the best arm's, that split is nearly right when one arm is modestly ahead and twice as wrong as it needs to be when the arms are alike. A split of thirty-two an arm before the choice is never more than 21.3% worse than the best split for whichever world holds; sixty an arm can be 98.8% worse. The two targets an estimate could serve — the chosen arm's effect and the best arm's — pull the split in opposite directions, and only one of them is what gets published.
A schedule the trial chooses for itself
A separate pilot cannot pay for itself in choosing a trial's follow-up schedule, because its patients are recruited, measured and discarded. A trial can instead measure its own first patients on the schedule that needs no pilot — two follow-ups — fit the correlation curve to them, and give everyone after them the schedule that curve makes cheapest. Those first patients are counted in the analysis and cost no extra visit. Forty of them cut the trial's worst-case excess cost across every curve with adjacent correlation 0.7 from 11.1% to 5.9% in a trial of two hundred an arm, and eighty cut it to 3.6%; twenty buy almost nothing. And because the curve is read from within-arm deviations, the choice cannot see the effect: the pilot's effect estimate is the same whichever schedule it chose.
Named alongside it
The objects these essays reach for when they reach for this one.
CoverageStatistical powerMonte CarloDegrees of freedomStopping ruleConfidence intervalExperimental designStandard deviationClosed formEstimated varianceFixed-width intervalSkewness