Tutorials › Marginal Structural Models › Worked Example With Two Binary Decisions

Dynamic Treatment Regimes · Tutorial 434 of 501

Worked Example With Two Binary Decisions

Follow a numerical example from terminal Q-model predictions to the pseudo-outcome, then derive and verify the first- and second-stage treatment rules.

Advanced 11 min read

What You'll Learn

  • Specify the histories and conditional means used in a two-stage binary-treatment Q-learning calculation.
  • Calculate a terminal fitted Q-value for each treatment choice at every observed history.
  • Construct pseudo-outcomes by maximizing terminal predictions over the second treatment.
  • Average pseudo-outcomes over the intermediate covariate to compare first-stage choices.
  • Distinguish a second-stage rule on the full history from its behavior along the derived regime.
  • Check fitted rules for ties, inadequate support, and model misspecification.

From Terminal Predictions to Two Decisions

In a two-stage treatment problem, the best second treatment can depend on what happened between decisions. The best first treatment must account for that later opportunity: its value is the expected outcome after choosing the best available second treatment at each subsequent history. This tutorial carries out that calculation for binary treatments and covariates, beginning with fitted terminal Q-values and ending with both treatment rules.

Let \(X\) be a baseline covariate, \(A_1\in\{0,1\}\) the first treatment, \(L\) an intermediate covariate measured before the second decision, \(A_2\in\{0,1\}\) the second treatment, and \(Y\) the final outcome, with larger values preferred. The stage-two history is \(H_2=(X,A_1,L)\). The data must contain support for the treatment choices and histories to which the fitted rules will be applied.

Definition: The terminal Q-function is \(Q_2(x,a_1,l,a_2)=E[Y\mid X=x,A_1=a_1,L=l,A_2=a_2]\). Its optimized value at a stage-two history is \(V_2(x,a_1,l)=\max_{a_2\in\{0,1\}}Q_2(x,a_1,l,a_2)\). The stage-one Q-function is \(Q_1(x,a_1)=E[V_2(x,a_1,L)\mid X=x,A_1=a_1]\). The plug-in rules choose an action maximizing the corresponding fitted Q-function.

These conditional means have a causal interpretation under consistency, sequential exchangeability at both treatment decisions, and positivity for treatment options in the histories being evaluated. In particular, consistency links observed outcomes to the potential outcomes under received treatments; sequential exchangeability rules out unmeasured confounding of each treatment decision given its measured history; and positivity requires that the relevant actions have positive probability at those histories. For estimation by Q-learning, the conditional-mean models must also be correctly specified, or sufficiently flexible to estimate the relevant means. With binary covariates, saturated cell means provide a transparent illustration.

Worked Two-Stage Calculation

Suppose \(X\) and \(L\) are binary. A saturated terminal model is fitted by allowing a separate intercept and treatment contrast for every \((X,A_1,L)\) history. Equivalently, it gives a separate predicted mean for each value of \(A_2\) in each history. Assume the fitted predictions are as follows.

\(X\)\(A_1\)\(L\)\(\hat Q_2(\cdot,A_2=0)\)\(\hat Q_2(\cdot,A_2=1)\)\(\hat V_2\)Maximizing \(A_2\)
000910101
0011412140
010119110
0111316161
1001311130
1011718181
1101210120
1111413140

Worked Example: Constructing the Pseudo-Outcome

For each observed \((X,A_1,L)\), construct the pseudo-outcome by taking the larger of the two terminal predictions. For example, at \(X=0,A_1=0,L=1\), the predictions are 14 and 12, so the pseudo-outcome is \(\max(14,12)=14\), and the maximizing action is \(A_2=0\). At \(X=0,A_1=1,L=1\), it is \(\max(13,16)=16\), with \(A_2=1\).

The complete pseudo-outcome values, in the table’s row order, are \(10,14,11,16,13,18,12,14\). These are fitted optimized values, not observed outcomes and not a new treatment assignment. The second-stage decision has been optimized separately at each history before these values are carried back to stage one.

To fit the stage-one Q-function, average the pseudo-outcomes over the distribution of \(L\) conditional on each \((X,A_1)\). Suppose that distribution is estimated, or known for this illustration, to satisfy \(P(L=1\mid X=0,A_1=0)=0.25\), \(P(L=1\mid X=0,A_1=1)=0.75\), and \(P(L=1\mid X=1,A_1=a_1)=0.5\) for either \(a_1\).

Worked Example: Fitting the Stage-One Q-Values

At \(X=0,A_1=0\), the pseudo-outcome is 10 when \(L=0\) and 14 when \(L=1\). Therefore, \( \hat Q_1(0,0)=0.75(10)+0.25(14)=7.5+3.5=11. \)

At \(X=0,A_1=1\), the corresponding calculation is \( \hat Q_1(0,1)=0.25(11)+0.75(16)=2.75+12=14.75. \) At \(X=1,A_1=0\), it is \( \hat Q_1(1,0)=0.5(13)+0.5(18)=6.5+9=15.5. \) At \(X=1,A_1=1\), it is \( \hat Q_1(1,1)=0.5(12)+0.5(14)=6+7=13. \)

Thus the stage-one fitted values are:

$$ \begin{array}{c|cc|c} X & \hat Q_1(X,0) & \hat Q_1(X,1) & \hat d_1(X)\\ \hline 0 & 11 & 14.75 & 1\\ 1 & 15.5 & 13 & 0 \end{array} $$

The derived first-stage rule is \(\hat d_1(0)=1\) and \(\hat d_1(1)=0\): choose treatment 1 when \(X=0\), and treatment 0 when \(X=1\). Each choice follows from comparing both fitted values at that baseline history.

Why the Backward Calculation Works

Theorem: Suppose the treatment and covariate histories take values in finite sets, the conditional means in the Q-functions are well defined, and the sequential identification assumptions stated above hold. Then a rule choosing an action that maximizes \(Q_2(x,a_1,l,a_2)\) at every stage-two history, and a rule choosing an action that maximizes \(Q_1(x,a_1)\) at every baseline history, maximize the expected outcome among the available two-stage rules.

Proof. Fix a baseline value \(x\) and any first-stage choice \(a_1\). For each possible \(l\), every second-stage action has conditional expected outcome \(Q_2(x,a_1,l,a_2)\). Hence no second-stage rule can have conditional expected outcome greater than \(V_2(x,a_1,l)=\max_{a_2}Q_2(x,a_1,l,a_2)\) at that history. Averaging over \(L\) gives an upper bound of \(E[V_2(x,a_1,L)\mid X=x,A_1=a_1]=Q_1(x,a_1)\) for the expected outcome after choosing \(a_1\). A rule selecting a maximizing second-stage action at every \(l\) attains this bound, since the history sets are finite and a maximum exists. Finally, among the two first-stage choices, selecting one that maximizes \(Q_1(x,a_1)\) attains the greatest of these upper bounds. This establishes optimality at every \(x\), and averaging over \(X\) establishes optimality in the population. The identification assumptions ensure that these conditional means correspond to the expected potential outcomes under the specified treatment choices. \(\square\)

In a fitted analysis, the same steps produce a plug-in rule: replace the population Q-functions by their estimates and maximize those estimates. The theorem explains the target of this calculation; it does not by itself guarantee that a fitted rule is accurate in a finite sample.

The Second-Stage Rule Uses the Full History

Worked Example: Checking the Second-Stage Decisions

At \(X=0,A_1=0,L=0\), the terminal predictions are 9 for \(A_2=0\) and 10 for \(A_2=1\), so the maximizing choice is \(A_2=1\). At \(X=0,A_1=0,L=1\), they are 14 and 12, so the maximizing choice is \(A_2=0\). These two rows alone show that \(L\) does not determine the full second-stage rule: for \(L=1\), the optimal action can be 0.

In fact, the full fitted rule depends on \((X,A_1,L)\), as recorded in the terminal table. Along the derived regime, however, the relevant histories are narrower. When \(X=0\), the first-stage rule sets \(A_1=1\); at those histories the second-stage choice is 0 for \(L=0\) and 1 for \(L=1\). When \(X=1\), the first-stage rule sets \(A_1=0\); there too the choice is 0 for \(L=0\) and 1 for \(L=1\). Thus along this particular derived regime the second choice equals \(L\), even though that statement is not true for the full table.

This distinction is important when describing a regime. A rule defined for every possible history is not the same as its behavior on histories that occur after the rule’s own first-stage choice. Reporting only the behavior along the regime can be useful, but it must not be mistaken for a rule that ignores \(X\) and \(A_1\).

Verification, Alternatives, and Failure Modes

One verification is to compute the value of the derived rule from the stage-one fitted values. If \(P(X=0)=0.4\) and \(P(X=1)=0.6\), its fitted value is \(0.4(14.75)+0.6(15.5)=5.9+9.3=15.2\). For comparison, a rule that always selects \(A_1=1\), while still using the maximizing second-stage action for each history, has fitted value \(0.4(14.75)+0.6(13)=5.9+7.8=13.7\). The difference is \(15.2-13.7=1.5\) fitted outcome units. This is a model-based comparison, not evidence of a causal improvement unless the identification and model assumptions are credible.

For binary histories, direct enumeration of complete regimes is a useful check: calculate the value of each candidate combination of first- and second-stage decisions and verify that none exceeds the backward maximum. Enumeration becomes cumbersome as the number of histories grows; backward Q-learning exploits the sequential structure. Other approaches, such as inverse-probability-weighted comparisons, target regime values differently and can provide a useful contrast, but do not remove the need for adequate treatment support and careful causal assumptions.

  • Check the maximization row by row. For every history, compare both terminal predictions. A verbal rule based only on \(L\) can contradict rows where \(X\) or \(A_1\) changes the treatment contrast.
  • Check the distribution used for averaging. The stage-one calculation weights each pseudo-outcome by \(P(L\mid X,A_1)\), not by an overall marginal distribution of \(L\). Using the wrong weights changes the Q-values.
  • Check support and model form. Sparse histories can make cell means unstable or undefined. A terminal model that omits important treatment interactions can also conceal history-dependent contrasts. Inspect cell counts, fitted contrasts, and sensitivity to plausible model specifications.
  • Do not treat estimated ties as decisive evidence. If two fitted predictions are equal, either action maximizes the fitted value at that history. If they are merely close, sampling variability can easily reverse their ordering; the earlier material on ties and nonregular inference explains why such decisions warrant caution.

The example uses finite binary variables and fitted means to make every step visible. In practice, continuous covariates require modeling conditional means over a larger history space, and estimated treatment rules can be sensitive to extrapolation, model misspecification, and overfitting. Correct backward arithmetic cannot repair a lack of support or a poorly estimated terminal model.

Key takeaway: First maximize the terminal Q-function at each complete second-stage history. Then average those optimized predictions over the intermediate covariate conditional on baseline history and first treatment, and maximize the resulting stage-one Q-function. Keep the full-history second-stage rule distinct from its restricted behavior along the chosen regime.

Check Your Understanding

Answer each question, then compare with the solutions below.

  1. At a history with terminal predictions 7 for \(A_2=0\) and 9 for \(A_2=1\), what are the pseudo-outcome and maximizing action?
  2. If a pseudo-outcome is 8 at \(L=0\), 12 at \(L=1\), and \(P(L=1\mid X=x,A_1=a_1)=0.25\), what is the stage-one Q-value for that \((x,a_1)\)?
  3. Why is it not generally valid to describe the second-stage rule as depending on \(L\) alone?
  4. At a stage-two history, the fitted predictions for both actions are equal. What can be concluded about the fitted maximizing actions?

Solutions. (1) The pseudo-outcome is \(\max(7,9)=9\), and the maximizing action is \(A_2=1\). (2) The conditional mean is \(0.75(8)+0.25(12)=6+3=9\). (3) The terminal contrast can vary with the complete history \((X,A_1,L)\), so two histories with the same \(L\) may favor different actions. (4) Both actions maximize the fitted terminal Q-value at that history; this fitted tie does not establish that the population actions are truly equivalent.

Clinical Biostats