Tutorials › Propensity Score Matching and Instrumental Variables › The Instrument as an Assignment That Shifts Treatment

IV Assumptions and Local Average Treatment Effects · Tutorial 440 of 699

The Instrument as an Assignment That Shifts Treatment

Learn to distinguish an instrument’s assignment from treatment receipt, quantify how an encouragement shifts uptake, and identify what that shift does—and does not—establish.

Advanced 11 min read

What You'll Learn

  • Distinguish instrument assignment, treatment receipt, and outcome
  • Calculate an assignment-group difference in treatment uptake
  • Interpret an uptake shift without mistaking it for a treatment effect
  • Explain why random assignment alone does not establish instrument validity
  • Recognize design features that can undermine an encouragement instrument
  • Compare randomized encouragement with direct treatment assignment and observational instruments

Assignment Is Not the Same as Treatment

In an instrumental-variable design, the instrument is often an assignment, offer, or encouragement that changes who receives a treatment without determining receipt for everyone. This distinction matters because people may decline an offer, fail to access the treatment, or obtain it through another route. The assigned condition and the treatment actually received are therefore different variables.

Let \(Z\) denote an assignment or encouragement, \(D\) indicate whether a person receives the treatment, and \(Y\) denote an outcome. In a common binary setup, \(Z=1\) means assigned or offered the encouragement and \(Z=0\) means not assigned or offered it; \(D=1\) means treatment was received and \(D=0\) means it was not. These labels describe separate stages: \(Z\) is the assignment, \(D\) is treatment receipt, and \(Y\) is the outcome of interest.

Definition: An instrument candidate is an assignment, offer, or other variable used to generate variation in treatment receipt \(D\), rather than a direct record of treatment itself. In a randomized encouragement design, \(Z\) records the randomized offer or encouragement, while \(D\) records actual treatment receipt. A change in uptake across assignment groups is evidence that the assignment shifts treatment receipt; it does not, by itself, establish that the candidate is a valid instrument for estimating a treatment effect.

For example, randomizing an invitation to a counseling program does not randomize counseling attendance. Some invited people may not attend, and some people not invited may attend through another route. The invitation is \(Z\); attendance is \(D\). Treating these as interchangeable would erase the very distinction that makes an encouragement design useful.

A direct randomized treatment assignment can look different. If every person assigned to treatment receives it, then assignment and receipt coincide in the data. With nonadherence, however, the assigned treatment group and the treatment-receipt groups diverge. An instrumental-variable analysis uses the assignment variation, not a claim that every person followed the assignment.

What an Uptake Shift Shows

For a binary assignment, a basic descriptive check compares the treatment-receipt rates in the two assignment groups. If \(D\) is binary, the rate in each group is the proportion with \(D=1\). The difference tells us whether the encouragement is associated with a change in treatment uptake in the observed data.

$$ \widehat{\Delta}_D = \widehat{P}(D=1\mid Z=1) - \widehat{P}(D=1\mid Z=0) $$

When \(Z\) is randomized, properly implemented, and analyzed according to assignment, this contrast estimates the effect of assignment on treatment receipt in the study population, subject to the usual design and measurement conditions. It is an effect of the offer on uptake—not the effect of the treatment on the outcome \(Y\). Even a large uptake difference does not show that the assignment affects \(Y\) only through treatment, or that the assignment is independent of all relevant potential outcomes. Those are separate requirements for instrumental-variable interpretation.

The arithmetic is straightforward. If \(n_1\) people are assigned the encouragement and \(r_1\) receive treatment, then the observed uptake rate in that group is \(r_1/n_1\). Define \(n_0\) and \(r_0\) analogously for the comparison group. Subtracting the two rates gives the observed uptake contrast. A positive value indicates higher uptake among those assigned the encouragement; a negative value indicates lower uptake. A value near zero may indicate little practical shift, but sampling uncertainty should be considered before concluding that the assignment has no effect on uptake.

$$ \widehat{\Delta}_D = \frac{r_1}{n_1} - \frac{r_0}{n_0} $$

Worked Example: An Invitation Changes Counseling Uptake

In a hypothetical trial, 100 people are randomly assigned an invitation to attend counseling and 100 are assigned no invitation. Of those invited, 60 attend; of those not invited, 30 attend through other routes. Calculate and interpret the uptake contrast.

The invited-group uptake rate is \(60/100=0.60\), or 60%. The comparison-group rate is \(30/100=0.30\), or 30%. Thus,

$$ \widehat{\Delta}_D = \frac{60}{100} - \frac{30}{100} = 0.60-0.30 = 0.30 $$

The invitation group has a 30-percentage-point higher observed counseling uptake rate. Given that assignment was randomized and implemented as planned, this is an estimate of how assignment to the invitation changed receipt in this hypothetical study. It is not an estimate that counseling improves the outcome by 30%, nor does it show that the invitation is a valid instrument for an outcome analysis.

Encouragement Designs in Practice

An encouragement can be an invitation, reminder, referral, scheduling offer, or other assignment intended to make treatment receipt more likely. It need not force treatment, and it should not be confused with a treatment itself. Its defining role is to create a difference in the opportunity or motivation to receive treatment.

Worked Example: A Reminder and a Modest Uptake Shift

Suppose a hypothetical study randomly assigns 120 patients to receive a reminder about an available prevention program and 120 to receive no reminder. Fifty-four reminder-group patients and 42 comparison-group patients attend. Find the uptake rates and their difference.

The reminder-group rate is \(54/120=0.45\), or 45%. The comparison-group rate is \(42/120=0.35\), or 35%. The difference is

$$ \widehat{\Delta}_D = \frac{54}{120} - \frac{42}{120} = 0.45-0.35 = 0.10 $$

In this hypothetical sample, assignment to a reminder is associated with a 10-percentage-point increase in attendance. This is a smaller uptake shift than in the counseling example, but whether it is estimated precisely depends on sample size and sampling variation. The rate difference describes assignment’s relationship to receipt; it does not say what effect attendance has on health outcomes.

The assignment should also be described accurately. If the reminder includes practical advice, transportation assistance, or additional clinical attention, it may do more than encourage program attendance. Such features raise a concern that assignment could affect the outcome through a route other than treatment receipt. A positive uptake contrast does not resolve that concern.

Worked Example: A Relevant Offer With a Design Concern

In a hypothetical trial, 200 people are randomized to receive either an offer of a diet program bundled with grocery vouchers or no offer. Eighty of the 100 people assigned the bundle enroll in the program, while 20 of the 100 people in the comparison group enroll through other channels.

The uptake rates are \(80/100=0.80\) and \(20/100=0.20\), so the assignment-group difference is

$$ \widehat{\Delta}_D = \frac{80}{100} - \frac{20}{100} = 0.80-0.20 = 0.60 $$

The assignment is associated with a 60-percentage-point higher enrollment rate, a substantial shift in treatment receipt. But the grocery vouchers may affect health outcomes even for people who do not enroll in the diet program. Therefore, the uptake calculation alone cannot establish that this assignment is suitable as an instrument for enrollment. The study would need to address whether the assignment has a pathway to the outcome outside the treatment being analyzed.

What Must Be Checked Before Calling It an Instrument

“Instrument” is sometimes used informally for any encouragement. For causal analysis, it is safer to distinguish an instrument candidate from an instrument whose assumptions are credible for the question at hand. An observed uptake shift is necessary for the usual binary-instrument strategy to provide useful treatment variation, but it is not sufficient for a causal treatment interpretation.

The design and analysis should establish, or justify, several points. First, the assignment must be defined clearly and recorded correctly. Second, assignment should be implemented as intended; a randomized allocation that is overridden or misrecorded may not provide the planned comparison. Third, treatment receipt should be measured consistently in both groups, including treatment received outside the study. Finally, the assignment must satisfy the instrumental-variable assumptions relevant to the chosen analysis. The standard names include independence, exclusion, and—when targeting a local average treatment effect—monotonicity. These are distinct from the question of whether uptake differs.

For a randomized encouragement, randomization supports independence of assignment from pre-assignment characteristics in expectation. It does not guarantee that assignment has no direct effect on \(Y\), and it does not guarantee that every person’s receipt moves in the same direction. A reminder might change attendance for some people and have no effect for others; another assignment might discourage some people. The assignment procedure and the real-world pathways it creates both matter.

A simple uptake contrast is also a sample estimate. Random assignment can produce different groups by chance, so a small observed difference does not prove the population uptake shift is exactly zero, and a positive difference does not prove it is large or precisely estimated. Consider uncertainty, the size of the shift, and whether the comparison was pre-specified. Do not rely on a single noisy percentage difference as a complete diagnostic.

Derivation: Why the Difference in Rates Is the Assignment Contrast

For binary treatment receipt, the sample mean of \(D\) in an assignment group equals the proportion receiving treatment, because each person’s \(D\) is either zero or one. If the group contains \(n_z\) people and \(r_z\) of them receive treatment, the sum of their \(D\) values is \(r_z\). Therefore, the group mean is \(r_z/n_z\), which is also the group’s treatment-receipt rate. Subtracting the two group means gives the rate difference above.

$$ \bar{D}_{Z=1} = \frac{1}{n_1}\sum_{i:Z_i=1}D_i = \frac{r_1}{n_1}, \qquad \bar{D}_{Z=0} = \frac{1}{n_0}\sum_{i:Z_i=0}D_i = \frac{r_0}{n_0} $$

Thus, \(\bar{D}_{Z=1}-\bar{D}_{Z=0}\) is not a special adjustment: it is exactly the difference in observed treatment-receipt proportions. Randomization gives this comparison a causal interpretation as the assignment’s effect on receipt under appropriate implementation and measurement conditions. It does not transform the comparison into the causal effect of receipt on the outcome.

Diagnostics, Alternatives, and Limitations

A useful review starts with a flow of people from assignment to receipt. Report the number assigned to each group, the number with observed treatment status, and the number receiving treatment in each group. Check missing treatment records and treatment access outside the study. If missingness differs by assignment, the observed rates may not represent the actual uptake rates. Also verify that the group labels correspond to the original assignment rather than a post-assignment status.

Common warning signs include an almost identical uptake rate across groups, substantial missing receipt information, inconsistent definitions of treatment, or an encouragement that changes other services at the same time. A weak observed shift can make an IV estimate unstable; a strong shift cannot repair an exclusion or independence problem. These are different failure modes and should not be conflated.

The main alternatives answer different questions. A direct randomized treatment assignment with nearly complete adherence can support a comparison of assigned treatment groups, while an encouragement design retains the assignment comparison when uptake is incomplete. A per-protocol or as-treated comparison instead groups people by treatment received and can lose the protection of random assignment, because uptake may be related to prognosis or motivation. An observational instrument may offer variation in treatment receipt, but its credibility depends on a defensible source of assignment variation rather than randomization.

An encouragement design is limited by the population and setting in which the offer operates. Its uptake shift may be modest, its effect on receipt may differ across groups, or the offer may create pathways to the outcome besides treatment. The uptake contrast is therefore an essential design diagnostic, not a complete analysis. Interpret it narrowly, then evaluate the remaining assumptions and the target causal question.

  • Mistake: “The invitation raised attendance by 30 percentage points, so treatment reduced the outcome by 30%.”
  • Correction: The 30-percentage-point contrast is in treatment receipt across assignment groups. It is not an outcome effect, and it is not a treatment effect.
  • Mistake: “Assignment was randomized, so it must be a valid instrument.”
  • Correction: Randomization supports independence, but instrument validity also requires other conditions, including a credible exclusion restriction; randomization alone does not establish them.
Key takeaway: In an encouragement design, \(Z\) is the assignment or offer and \(D\) is treatment receipt. Compare receipt rates to determine whether assignment shifts uptake, but do not confuse that shift with a treatment effect or treat it as proof of instrument validity.

Check Your Understanding

Answer each question, then compare with the solutions below.

  1. A hypothetical trial assigns 80 people an offer; 48 receive treatment. Among 80 people not offered treatment, 32 receive it. What are the two uptake rates and their difference?
  2. In a randomized encouragement study, what does a positive difference in treatment-receipt rates establish, and what does it not establish?
  3. Why is it incorrect to call an invitation and treatment receipt the same variable when some people decline and some comparison-group members obtain treatment?
  4. A randomized offer substantially increases program enrollment, but it also provides free transportation that could affect the outcome. What concern does this create?
  5. What additional information should be checked if treatment-receipt rates are almost identical across assignment groups?

Solutions. (1) The offer-group rate is \(48/80=0.60\); the no-offer rate is \(32/80=0.40\). The difference is \(0.60-0.40=0.20\), or 20 percentage points. (2) It establishes an observed uptake difference; with properly implemented randomization and adequate measurement, it estimates the assignment’s effect on receipt. It does not establish the effect of treatment on the outcome or all conditions for instrument validity. (3) Assignment records the offer, while receipt records whether treatment was actually taken. Declining and outside access make the variables differ. (4) Transportation may affect the outcome through a pathway other than enrollment, raising an exclusion concern. (5) Check the assignment implementation, sample size and uncertainty, missing receipt data, treatment definition, and outside access before concluding that the encouragement failed to shift uptake.

Clinical Biostats