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Instrumental Variable Extensions · Tutorial 556 of 972

Randomized Encouragement Designs and Treatment Uptake

Distinguish randomized encouragement from treatment uptake and map the pathways needed to assess an encouragement design.

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What You'll Learn

  • Define encouragement assignment, treatment receipt, and noncompliance in a randomized encouragement design.
  • Distinguish randomized assignment from self-selected uptake.
  • Map the intended pathway from encouragement through treatment to outcome.
  • Identify direct and indirect pathways that can undermine an encouragement's use as an instrument.
  • Explain why randomization alone does not establish instrument validity.

Why Encourage Rather Than Assign Treatment?

In some studies, researchers cannot or do not want to compel people to receive a treatment. They may instead randomize an offer, reminder, referral, or other encouragement intended to increase uptake. A reminder to attend a counseling appointment, for example, can be randomly assigned even though attendance remains each participant’s choice. The resulting design has two distinct events: being assigned an encouragement and actually receiving the treatment.

That distinction is central to causal inference. Randomization governs the encouragement assignment; it does not automatically randomize who takes up treatment. Comparing people who received treatment with those who did not can therefore reintroduce selection: their decisions to take up treatment may reflect health, motivation, resources, or other factors that also relate to the outcome. An encouragement design uses random assignment as a possible source of variation in treatment uptake, but the design must also be assessed for other requirements.

Definition: A randomized encouragement design is a study in which an encouragement is randomly assigned to participants to influence whether they receive a treatment, while treatment receipt remains at least partly a choice or otherwise is not fully dictated by assignment. Let \(Z\) denote encouragement assignment, \(D\) denote treatment receipt, and \(Y\) denote the outcome. For a binary encouragement, \(Z=1\) means assigned the encouragement and \(Z=0\) means not assigned it. For a binary treatment, \(D=1\) means received the treatment and \(D=0\) means did not.

The encouragement may be an invitation, reminder, subsidy, access offer, referral, or other intervention intended to change treatment uptake. It is not itself the treatment merely because it is designed to promote treatment. Whether a particular activity counts as the treatment or as an encouragement depends on the study’s precise definitions and causal question. Those definitions should be fixed before looking at outcomes.

Assignment, Receipt, and Noncompliance

Assignment describes what randomization allocated. Receipt describes what a participant actually experienced. In a fully enforced treatment assignment, these may coincide. In an encouragement design, they commonly differ: some people assigned an offer do not take up treatment, while some not assigned the offer obtain treatment through another route. This mismatch is called noncompliance with the intended treatment status or encouragement pathway.

A participant assigned an encouragement is not necessarily treated. Likewise, a participant not assigned an encouragement is not necessarily untreated. For that reason, the study should record \(Z\) and \(D\) separately rather than replacing one with the other. The distinction also applies when \(D\) is not a simple yes-or-no event: treatment receipt may require an operational definition, such as attending at least one session or completing a specified course.

The assignment may be randomized even when receipt is not. A participant who accepts a treatment may differ from one who declines it in ways that matter for \(Y\). Thus a comparison by \(D\) is not automatically protected by the randomization that was used to allocate \(Z\). Randomization supports a comparison by assigned encouragement; a causal interpretation involving treatment receipt requires additional reasoning about how assignment affects outcomes.

Worked Example: A Randomized Appointment Reminder

Suppose a hypothetical clinic randomly assigns 200 patients to receive either a reminder about a lifestyle-counseling appointment or no reminder. In each group, 100 patients are assigned. The study defines treatment receipt as attending at least one counseling session. Among those assigned a reminder, 60 attend; among those assigned no reminder, 25 attend.

The encouragement assignment is \(Z\): reminder (\(Z=1\)) or no reminder (\(Z=0\)). Treatment receipt is \(D\): attendance (\(D=1\)) or no attendance (\(D=0\)). These are not interchangeable. The observed attendance proportion among those assigned a reminder is

$$ \frac{60}{100}=0.60. $$

The observed attendance proportion among those assigned no reminder is

$$ \frac{25}{100}=0.25. $$

There are 40 nonattenders among the 100 reminder-assigned patients, since \(100-60=40\). There are also 25 attenders among the 100 patients assigned no reminder. The example therefore shows why “assigned reminder” cannot be used as a synonym for “attended counseling.” These counts describe treatment uptake by randomized assignment; they do not, by themselves, establish that attending counseling causes any later outcome.

The Causal Pathways an Encouragement Design Requires

A simple causal map helps distinguish the design’s intended mechanism from competing explanations. The desired pathway is from randomized encouragement \(Z\) to treatment receipt \(D\), and then from \(D\) to outcome \(Y\). Other variables can create additional paths. Let \(X\) represent measured characteristics recorded before assignment, and let \(U\) represent other causes that may not be measured.

$$ Z \longrightarrow D \longrightarrow Y \qquad\text{and}\qquad U \longrightarrow D,\;\; U \longrightarrow Y $$

The \(U\)-paths illustrate why treatment receipt can be confounded with the outcome. For example, motivation might affect both attendance and later health. Random assignment of \(Z\) is intended to break systematic links between assignment and pre-existing causes of the outcome. But it does not erase the \(U\)-paths between receipt and outcome, and it does not guarantee that every effect of \(Z\) on \(Y\) travels through \(D\).

Key design condition: To use encouragement assignment as an instrument for treatment receipt, the intended causal structure is that \(Z\) changes \(D\), and that any effect of \(Z\) on \(Y\) operates through \(D\). In potential-outcome notation, the latter exclusion condition is \(Y(z,d)=Y(d)\): holding treatment receipt \(d\) fixed, changing encouragement assignment \(z\) does not change the outcome. Randomization supports independence of assignment from pre-assignment causes; it does not, on its own, establish this exclusion condition or that assignment changes uptake.

The exclusion condition is substantive, not a consequence of randomization. A reminder might change attendance and thereby affect the outcome through counseling. But it might also increase patients’ attention to their health, prompt medication adherence, or create additional contact with clinicians. If any of these routes affect \(Y\) without operating through the defined treatment \(D\), the encouragement has a direct or alternative pathway to the outcome. Calling an intervention an “encouragement” does not make those pathways disappear.

The study’s definitions matter here. If \(D\) is defined narrowly as attending one counseling session, but the reminder also causes people to complete a separate clinical service, that separate service is not automatically part of \(D\). The researcher must ask whether it is a consequence of the defined treatment or a distinct route from \(Z\) to \(Y\). Clear definitions and a realistic account of how participants experience the encouragement are essential to that assessment.

Worked Example: An Access Offer and a Separate Pathway

Consider a hypothetical randomized offer of transportation to a physical-therapy program. Let \(Z=1\) mean the transportation offer was assigned, \(D=1\) mean the participant attended physical therapy, and \(Y\) be mobility measured in points on a 0–100 scale. Among 80 people assigned the offer, 48 attend; among 80 assigned no offer, 20 attend. The observed attendance proportions are \(48/80=0.60\) and \(20/80=0.25\).

The intended route is \(Z\rightarrow D\rightarrow Y\): transportation makes attendance more feasible, and therapy may affect mobility. Now suppose the transportation program also includes a driver who helps participants obtain an assistive device, and that device can improve mobility even for someone who does not attend therapy. This creates a second route, \(Z\rightarrow\text{device access}\rightarrow Y\), that does not pass through the defined \(D\).

The arithmetic confirms that attendance differs descriptively across assignment groups: 0.60 versus 0.25. It does not settle whether the offer is a valid instrument for attendance. The device-access route would violate the exclusion condition if it changes mobility independently of therapy attendance. To assess the design, the investigators would need to establish whether that route exists and whether the treatment definition appropriately captures it; the uptake counts alone cannot answer that question.

What Randomization Does—and Does Not—Guarantee

Randomization means assignment is generated by a chance mechanism rather than chosen according to participants’ characteristics. In expectation over that mechanism, assignment is independent of pre-assignment characteristics and potential outcomes. This is a statement about the assignment process, not a promise that every realized sample will have identical groups or that all other instrumental-variable conditions hold.

A short justification makes the scope clear. Suppose \(Z\) is assigned with probability \(q\), independently of a fixed pre-assignment quantity \(W\), where \(W\) might be a potential outcome or a baseline characteristic. Let \(I(Z=1)\) equal 1 when \(Z=1\) and 0 otherwise. Independence gives

$$ E[I(Z=1)W] =E[I(Z=1)]E[W] =qE[W]. $$

Since \(P(Z=1)=q\), it follows that

$$ E[W\mid Z=1] =\frac{E[I(Z=1)W]}{P(Z=1)} =\frac{qE[W]}{q} =E[W], $$

provided \(q>0\). The same reasoning applies to \(Z=0\) when that assignment has positive probability. Thus the two assignment groups have the same expected value of \(W\) under the randomization mechanism. In a particular finite sample, their observed averages can still differ by chance.

This argument concerns variables determined before assignment and potential outcomes under the assignment mechanism. It does not show that \(D\) is randomized: treatment receipt occurs after assignment and may depend on participant choices and circumstances. Nor does it show that \(Z\) affects \(Y\) only through \(D\), or that \(Z\) changes \(D\). These are separate design questions.

Worked Example: Distinguishing Balance from Validity

In a hypothetical study, researchers randomize 120 adults to receive an offer of a home blood-pressure monitor or to receive no offer. They record baseline age, whether a participant has a smartphone, monitor use over the next month, and systolic blood pressure at follow-up. Suppose the groups each contain 60 participants, and 42 offer-assigned participants use the monitor, compared with 18 participants assigned no offer.

Here \(Z\) records the offer, \(D\) records monitor use, and \(Y\) is follow-up systolic blood pressure measured in mmHg. The observed use proportions are \(42/60=0.70\) and \(18/60=0.30\). The numbers show that assignment and use differ, and that some participants use a monitor without being assigned the offer.

Randomization supports comparing the offer-assigned and no-offer groups as groups assigned by chance; it does not make the monitor users and nonusers comparable. Further, suppose the offer includes a nurse call that encourages medication review. If that call changes systolic blood pressure independently of monitor use, then the offer has a route to \(Y\) beyond the defined \(D\). Random assignment cannot rule out that route. The design assessment must therefore distinguish baseline comparability by \(Z\) from the separate question of whether the encouragement affects \(Y\) only through monitor use.

Practical Design Questions and Common Pitfalls

Before interpreting an encouragement study, specify what is assigned, what counts as treatment receipt, and what outcome is measured. Also describe the timing: assignment should precede the uptake it is meant to influence, and outcome measurement should follow the relevant treatment period. These details help prevent ambiguous classifications, such as counting a referral as treatment in one part of the analysis and as encouragement in another.

A useful pathway audit asks whether assignment is genuinely randomized; whether the encouragement could plausibly change receipt; and whether it could affect the outcome through attention, cointerventions, access, or any other route that bypasses the defined treatment. The study should also state how receipt and outcome are measured and consider whether participants’ assignment could influence one another’s receipt or outcomes. Those checks do not establish validity mechanically; they make the assumptions and design choices visible.

1
Name the randomized variable.
State exactly what \(Z\) records, including the offer or encouragement and its comparison condition.
2
Define treatment receipt.
State what event or exposure makes \(D=1\), and how participants who do not receive it are classified.
3
Draw the intended pathway.
Describe how the encouragement is expected to change treatment receipt and how receipt could affect \(Y\).
4
Look for other routes.
Ask whether the encouragement can change \(Y\) through attention, services, behavior, or access apart from the defined treatment.
5
Keep assignment and uptake separate.
Report who was assigned each condition and who actually received treatment as distinct facts.

Common mistake: “The offer was randomized, so treatment receipt was randomized and any difference between treated and untreated participants is causal.” The first clause concerns \(Z\), not \(D\). Participants may select into receipt, and their reasons for doing so may also predict the outcome. Randomization of \(Z\) does not by itself remove that selection or rule out a direct path from \(Z\) to \(Y\). The correction is to preserve the distinction between assignment and receipt, then assess the assumptions needed for the intended instrumental-variable interpretation.

Key takeaway: A randomized encouragement design randomizes an offer or prompt, not necessarily the treatment itself. Its central causal map is \(Z\rightarrow D\rightarrow Y\), but credible instrumental-variable use also requires careful attention to alternative pathways and to the difference between random assignment and self-selected uptake.

Check Your Understanding

Use the definitions and pathway logic above to answer these questions.

  1. A randomized text message invites patients to a screening visit. What should \(Z\) represent, and what separate event might define \(D\)?
  2. Why does random assignment of an encouragement not make people who accept treatment comparable to those who decline it?
  3. A transportation offer changes treatment attendance and also provides a health consultation. What pathway should be investigated if the outcome is affected by the consultation?
  4. In a study with 50 participants assigned an offer, 35 receive treatment. What is the observed receipt proportion in that assigned group, and why is it not the same thing as the assignment proportion?
  5. State one conclusion supported by randomization and one important instrument-validity question that randomization alone cannot settle.
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