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Effect Modification and Subgroup Effects · Tutorial 851 of 972

Conditional Average Treatment Effects and Subgroup Estimands

Learn to distinguish a treatment effect at a covariate profile from an average effect within a prespecified subgroup, and calculate both from potential outcomes.

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What You'll Learn

  • Define individual treatment effects and conditional average treatment effects for binary treatment.
  • Distinguish an effect at a covariate profile from an average effect over a subgroup.
  • Calculate conditional and subgroup effects from hypothetical potential-outcome contrasts.
  • Derive subgroup averages as weighted averages of conditional effects.
  • Explain how subgroup sizes determine a population average treatment effect.
  • Identify assumptions needed to learn these estimands from observed data.

Why Define the Target Before Comparing Effects?

A treatment may have different effects for people with different baseline characteristics. A useful analysis therefore needs to specify not just which treatment contrast is of interest, but for whom the contrast is being averaged. The effect for people with a particular covariate profile and the average effect for everyone in a prespecified subgroup are related, but they are not the same estimand.

This tutorial uses a binary treatment and a fixed outcome scale. A potential outcome is the outcome an individual would have under a specified treatment condition. The contrasts below are causal estimands: they are defined in terms of potential outcomes, whether or not those outcomes can all be observed in a study.

Definition: Let \(A\in\{0,1\}\) denote treatment status. For individual \(i\), \(Y_i(1)\) is the potential outcome under treatment and \(Y_i(0)\) is the potential outcome under control. The individual treatment effect is \(\tau_i=Y_i(1)-Y_i(0)\). Its units are the units of the outcome.

The individual effect is a contrast between two potential outcomes for the same person. In ordinary parallel-group studies, only one of those outcomes is observed for each person. Thus, individual effects are useful for defining population targets, but they are generally not directly calculable person by person from observed data.

Conditional Average Treatment Effects

Let \(X\) denote baseline characteristics that may describe an individual’s covariate profile. For a profile \(x\), the conditional average treatment effect (CATE) is the mean individual effect among people with that profile. If \(X\) is continuous, the profile \(x\) may represent a value or specified set of values; in practice, the definition also requires a meaningful target population with those characteristics.

Definition: The conditional average treatment effect at covariate profile \(x\) is $$ \tau(x)=E\!\left[Y(1)-Y(0)\mid X=x\right]. $$ Equivalently, by linearity of expectation, $$ \tau(x)=E[Y(1)\mid X=x]-E[Y(0)\mid X=x]. $$

The CATE is an average, not a claim that every person with profile \(x\) has the same individual effect. For example, \(\tau(x)=5\) points means that the average contrast between treatment and control potential outcomes among the target individuals with profile \(x\) is 5 points. Individual effects within that profile could differ.

Worked Example: A CATE at One Covariate Profile

Suppose a hypothetical treatment is intended to improve a recovery score measured in points. Consider four hypothetical individuals who all have the same specified baseline profile \(X=x\). Their potential outcomes are stipulated as follows.

Individual\(Y_i(0)\), control score\(Y_i(1)\), treatment score\(Y_i(1)-Y_i(0)\), points
170766
273818
367692
482886

The CATE for this finite set of individuals is the mean of their potential-outcome contrasts: $$ \tau(x)=\frac{1}{4}\sum_{i=1}^{4}\{Y_i(1)-Y_i(0)\} =\frac{6+8+2+6}{4} =\frac{22}{4} =5.5\text{ points}. $$

The same result follows by subtracting the two mean potential outcomes. The treatment mean is \((76+81+69+88)/4=78.5\) points, and the control mean is \((70+73+67+82)/4=73\) points. Their difference is \(78.5-73=5.5\) points. This schedule supplies both potential outcomes for calculation; it is hypothetical and does not describe what could ordinarily be observed for each person in a trial.

The result is an average for people with profile \(x\), not a prediction that treatment raises every person’s score by 5.5 points. The individual contrasts in this example range from 2 to 8 points.

Average Treatment Effects Within Prespecified Subgroups

A subgroup is defined by a baseline rule, such as belonging to an age category or having a particular diagnosis before treatment. Let \(S=g\) mean that an individual belongs to subgroup \(g\). The average treatment effect within that subgroup averages individual potential-outcome contrasts over the members of that subgroup.

Definition: The subgroup average treatment effect for subgroup \(g\) is $$ \tau_g=E\!\left[Y(1)-Y(0)\mid S=g\right]. $$ It is the average causal contrast for the target individuals who meet the subgroup definition, expressed in the outcome’s units.

A prespecified subgroup rule is set using baseline information, rather than chosen after examining which groups appear to have the largest observed effects. Prespecification helps make the target clear and limits outcome-driven selection; it does not, by itself, guarantee that the effect is identified or that an estimate is precise.

If \(X\) distinguishes profiles within subgroup \(g\), the subgroup effect averages the corresponding conditional effects using the profile distribution in that subgroup. For discrete profiles, the relationship follows from conditioning on \(X\): $$ \tau_g =\sum_x E[Y(1)-Y(0)\mid X=x,S=g]P(X=x\mid S=g). $$ When subgroup membership is determined by \(X\), this can be written using \(\tau(x)\) for the profiles included in \(g\). With continuous covariates, the corresponding expression is an expectation, or integral, over the distribution of profiles in the subgroup. The weights are the proportions of the subgroup at each profile, not automatically equal weights across profile categories.

Worked Example: Averaging Conditional Effects Within a Subgroup

In a hypothetical study setting, a prespecified subgroup consists of five people with a baseline characteristic of interest. Within this subgroup, three people are in a younger age band and two are in an older age band. The outcome is days symptom-free. The stipulated potential outcomes are:

Age band within subgroupIndividual\(Y_i(0)\), days\(Y_i(1)\), daysContrast, days
Younger1102010
Younger210188
Younger310166
Older410144
Older59112

First calculate the mean contrast in each age band. For the younger band, the mean contrast is \((10+8+6)/3=8\) days. For the older band, it is \((4+2)/2=3\) days. The subgroup contains three-fifths younger and two-fifths older people, so its average effect is $$ \tau_g=\frac{3}{5}(8)+\frac{2}{5}(3) =4.8+1.2 =6\text{ days}. $$

A direct calculation over all five subgroup members gives the same answer: $$ \tau_g=\frac{10+8+6+4+2}{5} =\frac{30}{5} =6\text{ days}. $$ The subgroup effect is not the unweighted mean of the two age-band effects, which would be \((8+3)/2=5.5\) days. That calculation assigns half the weight to each band even though the younger band contains three of the five people in this target subgroup.

Subgroup Weights Determine the Population Target

When subgroups partition a population, the population average treatment effect (ATE) is a weighted average of the subgroup effects. The weights are the proportions of the target population in each subgroup. Changing the target population can therefore change the overall average, even if each subgroup-specific effect remains the same.

Result: If mutually exclusive subgroups \(g\) cover the target population, then $$ E[Y(1)-Y(0)]=\sum_g P(S=g)\,E[Y(1)-Y(0)\mid S=g] =\sum_g P(S=g)\tau_g. $$ The weights sum to one because the subgroups partition the target population.

This result is a direct application of the law of total expectation. Let \(D=Y(1)-Y(0)\). For a partition into subgroups, $$ E[D]=\sum_g E[D\,\mathbf{1}(S=g)] =\sum_g P(S=g)E[D\mid S=g], $$ where \(\mathbf{1}(S=g)\) equals 1 for members of subgroup \(g\) and 0 otherwise. Thus, each subgroup’s mean contrast contributes in proportion to the subgroup’s share of the target population.

Worked Example: Population ATE From Two Subgroup Effects

Suppose a hypothetical target population is divided by a baseline risk category set in advance. Eighty percent of the population is in the higher-risk subgroup and 20% is in the lower-risk subgroup. The stipulated mean potential outcomes, in recovery-score points, are:

Prespecified subgroupPopulation proportionMean \(Y(0)\), pointsMean \(Y(1)\), pointsSubgroup effect, points
Higher risk0.8058624
Lower risk0.20637512

The subgroup effects are \(62-58=4\) points and \(75-63=12\) points. The ATE in the stated target population is $$ \tau=0.80(4)+0.20(12)=3.2+2.4=5.6\text{ points}. $$

As a check, the population mean treatment potential outcome is \(0.80(62)+0.20(75)=64.6\) points. The population mean control potential outcome is \(0.80(58)+0.20(63)=59.0\) points. Their difference is \(64.6-59.0=5.6\) points, matching the weighted subgroup calculation.

Giving the two subgroups equal weight would instead produce \((4+12)/2=8\) points. That is a valid average for a different target that gives each subgroup half the weight; it is not the ATE for this population, in which the subgroups have proportions 0.80 and 0.20.

Estimands and What Observed Data Can Establish

The definitions above specify targets in terms of potential outcomes. Estimating those targets from observed data requires additional design or identification assumptions. In the usual no-interference setup, consistency means that the observed outcome is \(Y=Y(A)\): a person assigned treatment has observed outcome \(Y(1)\), and a person assigned control has observed outcome \(Y(0)\). No interference means one person’s potential outcome is not changed by other individuals’ treatment assignments. If treatment conditions are not well defined or outcomes depend on others’ assignments, the potential-outcome definition must be adapted to the setting.

For an observational comparison, a common sufficient set of conditions for identifying conditional effects given baseline covariates \(X\) is conditional exchangeability, consistency, and positivity. Conditional exchangeability states that, given \(X\), treatment assignment is independent of the pair of potential outcomes. Positivity requires a positive probability of receiving either treatment at each covariate profile in the target population. Under these conditions, the conditional effect can be represented as the difference in conditional observed-outcome means: $$ \tau(x)=E[Y\mid A=1,X=x]-E[Y\mid A=0,X=x]. $$ Random assignment can support exchangeability by design, but it does not make every subgroup estimate precise, and it does not change the definition of the estimand. The subgroup target still averages over the subgroup population of interest.

These assumptions clarify a crucial distinction: defining an estimand is not the same as identifying it from observed data, and identifying it is not the same as estimating it accurately in a finite sample. The examples used complete hypothetical potential-outcome schedules so that the target calculations could be seen directly; real studies generally require design-based or model-based methods to learn about those targets.

Common Mistakes

  • Treating a CATE as an individual effect. A CATE is the mean contrast among people with a profile, not a guarantee that each person with that profile experiences that contrast.
  • Replacing a subgroup average with an effect at one profile. A subgroup can contain several profiles. Its effect averages their contrasts according to how common each profile is in that subgroup.
  • Using equal weights without defining an equal-weight target. Population subgroup weights come from the target population’s subgroup proportions. Equal weighting defines a different target when subgroup sizes differ.
  • Choosing subgroups after inspecting apparent effects. This can make the reported target depend on outcome results. Prespecifying the subgroup rule makes the intended population clearer, although it does not eliminate all inferential concerns.
  • Assuming both potential outcomes are observed for each person. The contrast \(Y_i(1)-Y_i(0)\) defines an individual effect, but ordinary studies observe only the outcome under the treatment actually received.
Key takeaway: A CATE averages potential-outcome contrasts among individuals with a specified covariate profile. A subgroup estimand averages those contrasts over a prespecified subgroup, using that subgroup’s distribution of profiles. The target population and its weights are part of the estimand, not incidental details of the calculation.

Check Your Understanding

Try these questions before moving on.

  1. In one sentence, distinguish an individual treatment effect from a conditional average treatment effect.
  2. A subgroup contains 25% of people with a conditional effect of 2 outcome units and 75% with a conditional effect of 6 units. Calculate the subgroup average effect and show the weights used.
  3. Why can an unweighted average of two subgroup effects differ from the population ATE?
  4. If the mean potential outcomes in a subgroup are 48 under control and 53 under treatment, what is the subgroup average treatment effect, and in what units should it be reported?
  5. Why does defining a CATE not, by itself, establish that observed data identify it?
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