Research Design

Effect Size, ATE, ITT, and TOT

A reading guide for lecture p-04 — what counts as an effect, and the difference between being offered a treatment and actually taking it.

Section one

Key concepts

The three ways of defining who counts as treated, and what an effect actually is.

Key concepts

An "effect" is a magnitude *and* an interval, not a p-value.

The deck’s own definition: the observed change plus its confidence interval — the size of the impact together with how accurately it was measured, so that you can say with confidence whether it is positive.

In regression terms, b1 = MEAN(treat) − MEAN(control), the null is b1 = 0, and significance is the question of whether the interval around b1 contains zero.

The average treatment effect is the default because it is the easiest thing to measure.

The ATE is the difference in average outcomes between treatment and comparison groups. It is the most compact way to report program effectiveness — and it silently averages over everyone, including people who never engaged with the program at all.

Intention to treat counts everyone who was *offered* the treatment.

ITT measures the effect of being assigned to the program, whether or not the person used it. In the bed-net example: group T is everyone given bed nets.

Treatment on the treated counts only those who actually took it.

TOT measures the effect among people who were given bed nets and used them.

The deck’s framing question is exactly this: Is group T those who are given bed nets, or those who use them? Both are legitimate. They answer different questions.

The contraception app makes the distinction concrete.

From the news story the deck reproduces:

  • Used perfectly, 5 in 1,000 women per year become pregnant — 99.5% effective
  • Under typical use, 7 in 100 become pregnant — about 93% effective

The deck asks which number is the TOT and which is the ITT. Perfect use is treatment on the treated. Typical use is intention to treat.

Then the harder question: is real effectiveness 99.5% or 93%? For a policy-maker deciding whether to fund distribution, the ITT number is the honest one, because real people forget. For a clinician advising a highly motivated patient, the TOT number is more informative.

ITT is usually the conservative estimate, and usually the policy-relevant one.

Because ITT includes non-compliers, it dilutes the measured effect toward zero. That makes it a lower bound on what the program does — and an accurate picture of what a funder can expect when they roll the program out to people who behave normally.

Selection and attrition are the two ways the comparison breaks.

Reviewed here from earlier lectures, with the fixes stated plainly:

  • Selection into the study — participants differ from non-participants. The fix: randomization or matching, to force an apples-to-apples comparison.
  • Selection out of the study (attrition) — leavers differ from stayers. The fix: compare the characteristics of those who stay against those who leave.

Section two

Key relationships

The mental map. Why this deck reviews the whole course before adding one idea.

This deck is a survey, and ITT/TOT is the new idea in it

Roughly two-thirds of these slides revisit material developed in p-01, p-02, and p-03: the suicide-rate example, the caffeine hypothesis-testing frame, the Bonferroni correction, the microfinance selection table, the attrition sequence, the three counterfactuals, and the staggered-start case study. If those are fresh, skim to slide 42.

The genuinely new content is slides 42–46: different interpretations of program effects — ATE, ITT, and TOT — plus the sampling-distribution refresher at the end.

ITT versus TOT is a choice about the denominator

Every effectiveness number is a fraction, and this pair of terms is a dispute about what goes underneath. ITT divides by everyone offered the program; TOT divides by everyone who complied. Neither is a correction of the other. Reporting only one, without saying which, is the actual error.

The distinction has teeth precisely because compliance is not random

If people who use the bed nets differ systematically from people who were given them and did not — more health-literate, more able to plan, less exhausted — then the TOT estimate carries all the selection problems from lecture p-02 back into the analysis. TOT is an estimate on a self-selected subgroup. That is why ITT preserves the randomization and TOT generally does not.

The two considerations for choosing an estimator, restated

The deck compresses lecture p-03 into two questions to ask of any design:

  1. Does C1 = T1? Usually not, for comparison groups. Randomization is what buys it, which is why post-test-only estimators are legitimate in experiments.
  2. Is C2 − C1 an accurate reflection of the trend? Sometimes a comparison group captures it adequately even when it is not equivalent.

Answer the first “yes” and post-test-only works. Answer the second “yes” and diff-in-diff works. Answer both “no” and you do not yet have a study.

Why the sampling distribution shows up at the end

The closing slides return to the sampling distribution at n = 10 and n = 50, and to the anatomy of a confidence interval — 2.5% of sample statistics above, 2.5% below, the true slope somewhere inside. This is not filler. It is the justification for the deck’s opening definition: an effect is a point estimate plus an interval, and the interval comes from the sampling distribution. Sample size is the lever that tightens it.

How to read the slides

Treat this as the integrative deck for the first half of the course. Read slides 42–46 closely and carefully; use the rest as a self-check. If any of the review slides is not immediately obvious, that is a signal to go back to the lecture where it was introduced rather than to keep going here.

Section three

What should be clear in my mind?

The questions to ask before you accept a reported effectiveness number.

Questions to ask of any reported effectiveness number

Key takeaways