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Repeated Significance Testing · Tutorial 26 of 699

Interpreting Monitoring Under the Alternative

Learn to interpret the probability and timing of rejection under a specified real effect, while keeping power separate from the probability of a false positive.

Beginner 10 min read

What You'll Learn

  • Define power for a repeated-monitoring rule under a specified alternative.
  • Distinguish power from Type I error and from the probability of an early stop.
  • Calculate two-look rejection probabilities in a clearly stated independent-test illustration.
  • Break overall detection probability into probabilities of first rejection at each look.
  • Explain when adding a look can raise power and why recalibrating boundaries can change that conclusion.
  • Recognize why calculations based on independent looks may not describe cumulative trial data.

Monitoring When an Effect Is Real

Repeated monitoring is often discussed in terms of false positives: when \(H_0\) is true, how often will a monitoring rule reject it at least once? But a monitoring plan also has consequences when an effect is real. It can affect the chance that the study detects the effect, and the look at which the first rejection occurs. These are questions about power and detection timing, not Type I error.

The interpretation depends on the whole decision rule: the alternatives being considered, the planned looks, and the rejection boundary at each look. A nominal significance level for one test does not by itself describe either the probability of any rejection across repeated looks or the chance of detecting a specified effect.

Definition: For a specified parameter value under \(H_a\), the power of a monitoring rule is the probability that the rule rejects \(H_0\) at least once. The Type I error probability is the probability that the rule rejects \(H_0\) at least once when \(H_0\) is true. Power is therefore evaluated under an alternative; Type I error is evaluated under the null.

Power is not one universal number for a study. It depends on which alternative value is used. A monitoring plan may have different power for a small effect than for a large one. The probability of an early stop is also distinct: it is the probability of rejecting at a particular look, either unconditionally or conditional on not having rejected earlier. These probabilities describe different features of the monitoring rule.

A Two-Look Illustration

To make the arithmetic transparent, first consider a deliberately simplified model. At each of two planned looks, a one-sided standardized test statistic \(Z_j\) is compared with the same boundary \(c=1.645\). Under the null, each statistic has a standard normal distribution. Under a particular alternative, each has a normal distribution with variance 1 and mean \(\delta\). For this illustration only, assume \(Z_1\) and \(Z_2\) are independent. Each look rejects if its statistic exceeds 1.645, and the study stops at the first rejection.

These assumptions are useful for illustrating repeated opportunities, but independence is not generally appropriate for tests based on accumulating data: successive statistics often share observations and are dependent. The calculations below are not a substitute for modeling that dependence in an actual monitoring plan.

Worked Example: Power and Type I Error With Two Independent Looks

Suppose the hypothetical standardized statistics are independent across two looks, and at each look the study rejects for \(Z_j>1.645\). Under \(H_0\), each statistic follows \(N(0,1)\). Under a particular alternative, each follows \(N(1,1)\), so the standardized effect is \(\delta=1\). Find the per-look rejection probability and the probability of any rejection under each condition.

Under the null. The probability of rejection at one look is approximately \(P(N(0,1)>1.645)=0.0500\). By independence, the probability of no rejection at either look is \((1-0.0500)^2=0.9025\). Thus the probability of at least one rejection, the Type I error probability for this repeated rule, is

$$ 1-(1-0.0500)^2=1-0.9025=0.0975. $$

Under the specified alternative. If \(Z_j\sim N(1,1)\), subtracting the mean gives \(P(Z_j>1.645)=P(N(0,1)>0.645)\approx0.2595\). Therefore the probability of no rejection at either independent look is approximately \((1-0.2595)^2\), and the power is

$$ 1-[1-\operatorname{norm.sf}(0.645)]^2 \approx 1-(1-0.2594636)^2 \approx 0.4516058 \approx 0.4516. $$

Here \(\operatorname{norm.sf}(x)\) denotes the standard normal upper-tail probability. The repeated rule has about 45.16% power at this alternative in this illustration, compared with about 25.95% at one look. Its Type I error probability is about 9.75%, not 5%. The greater power and the greater Type I error arise from the same repeated-rejection rule, but they are probabilities under different parameter conditions.

Why the Rejection Probability Changes

The general event structure does not require independent looks. Let \(R_j\) be the event that the statistic at look \(j\) crosses its boundary, and let \(K\) be the number of planned looks. The monitoring rule rejects if the union of those events occurs. Under a parameter value \(\theta\), its rejection probability is

$$ P_{\theta}\left(\bigcup_{j=1}^{K}R_j\right). $$

When \(\theta\) is an alternative value, this is the power at \(\theta\). When \(\theta\) is a null value, it is the Type I error probability for the rule. The formula involves the joint behavior of all the looks: knowing each look's separate rejection probability is not enough unless their dependence is also known.

A useful derivation partitions the event of any rejection according to the first look at which rejection occurs. Let \(A_j\) be the event that the first rejection is at look \(j\). These events cannot occur together, and their union is the event of at least one rejection. Consequently,

$$ P_{\theta}(\text{any rejection}) =\sum_{j=1}^{K}P_{\theta}(A_j). $$

This decomposition explains how an overall detection probability can be reported by timing. In general, the probability at look \(j\) is not simply the probability of crossing its boundary at that look: a rejection at look \(j\) counts as the first rejection only if earlier looks did not reject.

Worked Example: Probability and Timing of Detection

Continue the hypothetical two-look model above under \(\delta=1\). At either look, the rejection probability is \(p=\operatorname{norm.sf}(0.645)\approx0.2594636\), and the probability of no rejection is \(q=1-p\approx0.7405364\). Calculate the probability that the first rejection is at each look and the fraction of all detected studies that reject at the first look.

The probability of a first-look rejection is \(p\approx0.2595\). For the first rejection to occur at the second look, the study must first avoid rejection and then reject at look 2. Independence gives

$$ P(\text{first rejection at look 2}) =q p \approx(0.7405364)(0.2594636) \approx0.1921422 \approx0.1921. $$

Adding the two disjoint first-rejection probabilities gives \(0.2594636+0.1921422\approx0.4516058\approx0.4516\), consistent with the power calculation. Among studies that reject at either look, the fraction that reject at the first is approximately

$$ \frac{0.2594636}{0.4516058}\approx0.5745. $$

Thus, in this hypothetical model, about 57.45% of detections occur at the first look. This is a conditional description among detected studies; it does not mean that 57.45% of all studies stop early. The unconditional probability of first-look rejection is about 25.95%.

Power Depends on the Alternative

A result described as “the power” is incomplete unless the alternative is specified. In the simple normal illustration, increasing \(\delta\) shifts the distribution of each test statistic farther above the fixed boundary. That raises the per-look rejection probability and, for the unchanged rule, the probability of at least one rejection. Smaller effects are harder to detect and generally have lower power.

Worked Example: A Smaller Effect Has Lower Power

Suppose the same hypothetical independent two-look rule uses \(Z_j>1.645\), but the alternative has \(\delta=0.5\), rather than \(\delta=1\). Each statistic follows \(N(0.5,1)\). Calculate the approximate per-look rejection probability and two-look power, and compare them with the previous alternative.

At one look,

$$ P(Z_j>1.645) =P(N(0,1)>1.645-0.5) =P(N(0,1)>1.145) \approx0.1261. $$

Using independence, the probability of at least one rejection is approximately

$$ 1-(1-0.1261)^2 \approx1-(0.8739)^2 \approx0.2363. $$

The power is about 23.63% at \(\delta=0.5\), lower than the approximately 45.16% at \(\delta=1\). The boundary and number of looks did not change; the assumed effect did. Under \(H_0\), the Type I error probability also remains approximately 9.75% in this model, because it is calculated under the null rather than under either alternative.

When an Added Look Raises Power

There is a precise but limited statement about adding monitoring opportunities. If a new look is added while all the old looks and their boundaries are left unchanged, the original rejection events remain part of the new rule. The event of any rejection under the expanded rule therefore contains the event of any rejection under the original rule. For any fixed parameter value, including a specified alternative, its rejection probability cannot decrease. It may stay the same if the new look never adds a rejection, or increase if some paths reject only at the added look.

This set-inclusion argument does not say that power must rise when the boundaries are recalculated. A design may alter its thresholds to control the overall Type I error probability. The resulting event is not necessarily a superset of the old rejection event, so power can change in either direction. The exact consequences depend on the full rule and the joint distribution across looks; a per-look significance level alone does not determine them.

This distinction helps compare common approaches. A final-look-only test has no earlier stopping opportunity, so its power and timing are defined by that one test. Repeating an unchanged per-look cutoff can raise both the chance of detecting an effect and the chance of a false positive. A prespecified monitoring plan with boundaries chosen for a stated error objective evaluates power under the alternative while keeping the null error probability as a separate design property. These approaches answer related but different questions; none can be compared solely by counting looks.

Diagnostics and Common Misinterpretations

When interpreting or checking a power calculation for repeated monitoring, verify the following before drawing conclusions:

  • Check the parameter condition. State whether a probability is calculated under \(H_0\) or under a specified alternative. Calling a null rejection probability “power” is incorrect.
  • Check the event being counted. “Reject at look 2” may mean crossing at look 2 regardless of the past, or first rejecting at look 2 after no earlier rejection. Those are not the same event.
  • Check dependence. Multiplying probabilities of no rejection across looks assumes independence. Shared observations or accumulated data can make tests dependent, so that product may be wrong.
  • Check the full rule. If boundaries change when looks are added, the new rule may not preserve the old rejection events. The simple claim that adding a look cannot reduce power then does not follow.
  • Check the alternative and units. Report the effect size or parameter value at which power is evaluated, and describe effects in the outcome's units when applicable. Power at one effect size does not automatically describe another.

A frequent mistake is to say, “More looks increase power, so the probability of a false positive is unchanged.” The first claim is only guaranteed if the old boundaries and rejection opportunities remain unchanged; the second is generally false for that same unadjusted expansion. In the two-look illustration, per-look null rejection is 5%, but the chance of at least one null rejection is 9.75%. Conversely, a plan that modifies thresholds to manage null error cannot be evaluated for power merely by counting the looks.

Key takeaway: Power is the probability of at least one rejection under a specified alternative; Type I error is that probability under the null. Repeated looks affect rejection opportunities and detection timing, while the size of those effects depends on the monitoring boundaries and the joint behavior of the tests.

Check Your Understanding

Answer each question, then compare your reasoning with the solutions below.

  1. In a two-look plan, the probability of at least one rejection under \(H_0\) is 0.08. Is this the plan's power? What is it describing?
  2. In the independent illustration, suppose the per-look rejection probability under an alternative is 0.20. Find the two-look probability of any rejection.
  3. Explain why the probability of first rejection at look 2 is not generally the same as the probability of crossing the look-2 boundary.
  4. If a new look is added and all existing boundaries are unchanged, can power at a fixed alternative decrease? Explain why.
  5. Why might adding a look while recalibrating the boundaries fail to increase power?

Solutions. (1) It is not power: it is the Type I error probability, because it is calculated under \(H_0\). Power requires a specified alternative. (2) Under independence, the probability of no rejection at either look is \((1-0.20)^2=0.64\), so the probability of any rejection is \(1-0.64=0.36\). (3) A first rejection at look 2 requires no rejection at look 1 as well as a crossing at look 2; a look-2 crossing can occur on a path already stopped after look 1. (4) No. With unchanged old boundaries, the expanded any-rejection event contains the original event, so its probability at a fixed alternative cannot decrease. (5) Recalibration can change old boundaries, so the new rejection event need not contain the old one. Its power depends on the resulting rule and the joint distribution of the tests.

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