Manuel B. Garcia

Manuel B. Garcia serves as the Senior Director for Educational Technology and Digital Learning at FEU Institute of Technology, Manila, Philippines. Read More

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Should You Broaden the Outcomes When Direct Research Evidence Is Scarce?

Broader outcomes can add useful information when direct evidence is scarce, but an easier-to-find outcome is not necessarily an adequate substitute for the outcome you actually care about. The key is whether the broader outcome measures the same underlying construct or provides defensible indirect evidence.

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Broadening Outcomes When Evidence Is Scarce Guide 825 of 899
01 · The Question

What if studies exist, but they do not report the outcome you need?

You may encounter an evidence base that looks substantial until you examine the outcomes. Perhaps many studies evaluate the intervention, exposure, or phenomenon of interest, but only two report your primary outcome. Others measure related outcomes, intermediate outcomes, different instruments, or outcomes assessed at different time points.

Should those outcomes be brought into the evidence base?

Sometimes yes. Different measures may genuinely represent the same outcome domain, and related outcomes may provide useful supporting evidence. But broadening outcomes can also quietly change the question. An outcome that is easier to measure, more frequently reported, or statistically convenient is not automatically a substitute for the outcome that matters to your research question.

02 · The Short Answer

Broaden outcomes only when their relationship to the target outcome is defensible

In Brief

You may broaden the outcomes when direct evidence is scarce if the additional outcomes represent the same meaningful construct or can legitimately provide indirect evidence about the outcome of interest.

Do not treat related, intermediate, surrogate, differently timed, or differently defined outcomes as interchangeable without justification. The farther an outcome moves from what you actually want to know, the more indirect the resulting evidence becomes.

03 · What You Need to Know

First determine what kind of outcome difference you are dealing with

“Different outcome” can describe several quite different situations. Two studies may measure the same underlying construct with different instruments. They may assess the same outcome at different time points. One may measure an intermediate outcome while another measures the final outcome that matters to participants. Or they may simply measure different things.

These distinctions determine whether broadening is sensible.

Different measures can still represent the same outcome

Suppose your outcome is depressive symptoms. One study uses one validated depression scale while another uses a different validated scale. The numerical scores are not directly interchangeable, but both may represent the same underlying outcome domain.

Similarly, studies of academic achievement might use different validated achievement measures. Patient functioning may be captured with different instruments. Quality of life can be operationalized through different established scales.

This is not necessarily the same as broadening from one outcome to another. Cochrane guidance distinguishes broad outcome domains from the more detailed specification and grouping of outcome measures for synthesis. Decisions about which outcomes matter and how related measures will be grouped should ideally be planned in advance.

The important question is whether the measures are credible operationalizations of the same construct and whether differences in measurement affect interpretation.

A related outcome may still answer a different question

Now consider a more substantial change. Your target outcome is whether students can independently perform a clinical procedure, but most studies measure only their knowledge of the procedure. Knowledge and performance are related, but they are not the same outcome.

Or perhaps the target is reduction in disease, while available studies report a biological marker. The marker may be associated with disease risk, but evidence that an intervention changes the marker does not automatically establish that it changes the clinical outcome.

Alternative measure A different method or instrument used to assess substantially the same outcome construct.
Alternative outcome A different endpoint that may be related to the target outcome but does not measure the same thing.

Confusing these situations can make a review appear to have more direct evidence than it actually does.

Surrogate outcomes require a stronger inferential bridge

A surrogate outcome is used as a substitute for an outcome of direct interest. Examples can include biomarkers, physiological measurements, intermediate events, or other endpoints expected to predict an outcome that matters more directly.

Surrogates can be scientifically useful. They may occur earlier, require smaller studies, or make research feasible when the final outcome is uncommon or takes years to observe. But usefulness as a research endpoint does not by itself validate a surrogate as a substitute for the outcome of interest.

GRADE identifies surrogate outcomes as an important source of outcome indirectness when evidence for the surrogate is used to inform conclusions about another outcome. The strength of the relationship between the surrogate and the target outcome therefore matters.

Watch Out

An intervention can improve an intermediate or surrogate outcome without producing the expected improvement in the final outcome. Do not translate an effect on a surrogate directly into an effect on the target outcome unless that inferential relationship is adequately supported.

Timing can make the same named outcome indirect

Outcome directness also depends on when it is measured. A study reporting anxiety one week after an intervention and another reporting anxiety twelve months later may use the same scale and outcome label, yet answer meaningfully different questions.

If your decision concerns sustained benefit, immediate post-intervention improvement is not equivalent to long-term improvement. Conversely, if the question concerns an immediate procedural outcome, a distant follow-up may capture additional influences that were not central to the original question.

Recent GRADE guidance explicitly identifies duration of follow-up as one way in which study outcomes can mismatch the outcome specified in the target question.

Outcome definitions can hide important differences

Even identically named outcomes may be defined differently across studies. “Treatment success,” “recovery,” “engagement,” “adherence,” “academic performance,” or “completion” can represent quite different thresholds and measurement rules.

Before combining them, inspect how each study operationalized the outcome. A shared label is not sufficient evidence that the measurements represent the same construct.

Do not use outcome reporting to decide which studies exist

There is another important issue that is easy to miss. In many intervention systematic reviews, studies are not supposed to be excluded simply because they fail to report a particular outcome of interest.

Cochrane guidance states that outcome reporting should rarely determine study eligibility. Excluding otherwise eligible studies because a desired result is unavailable can create bias, particularly when outcomes are selectively reported according to the results.

This means “only three studies report my primary outcome” is not necessarily the same as “only three studies are eligible for my review.” The review may contain many eligible studies while the evidence available for a particular outcome remains sparse.

Important outcomes should not be replaced merely because inconvenient outcomes are sparse

Outcome selection should reflect the research question and what matters for interpretation, not merely what investigators most commonly measured. Cochrane recommends considering outcomes that are meaningful and including both beneficial and adverse outcomes where relevant.

If the outcome most important to your question is rarely reported, replacing it with a frequently reported but less meaningful outcome can conceal a genuine evidence gap.

This is one reason sparse evidence should first be treated as an informational finding rather than a numerical problem to solve. The broader question of what to do when direct evidence remains scarce should be resolved before expanding the evidence base simply to obtain more analyzable data.

Broadening can occur without pretending all outcomes are equivalent

You may decide that several related outcomes are worth including while keeping their roles distinct. A review could identify a patient-important outcome as primary, examine a validated surrogate as supporting evidence, and report other related outcomes separately.

Similarly, different instruments assessing the same domain may sometimes be synthesized using statistical methods appropriate for differently scaled measurements, while genuinely different outcome domains remain separate.

The critical methodological move is classification. Decide which measures represent the same construct, which are distinct but related, and which require an inferential bridge to inform the target outcome.

If that bridge becomes too long or depends on assumptions that cannot be defended, you have reached the broader problem of when additional evidence becomes too indirect.

04 · A Practical Example

What if your target is clinical performance but most studies measure knowledge?

Hypothetical Example

An educational intervention with many knowledge studies but little performance evidence

Suppose you are reviewing whether a simulation-based educational intervention improves students' clinical performance. Only three eligible studies assess performance in a clinical or realistically simulated task. Another fifteen studies assess knowledge using written tests.

Define the target outcome Your question concerns whether learners can perform, not merely whether they know factual or procedural information.
Classify the additional outcome Knowledge is plausibly related to performance, but a knowledge test does not directly measure whether a learner can execute the clinical behavior successfully.
Identify the inferential assumption Using knowledge outcomes to support a performance conclusion requires assuming that improvements in tested knowledge translate into improved clinical performance.
Preserve the distinction You may include knowledge as a separate secondary outcome if it falls within the review's scope, but you do not combine knowledge scores with performance measures or describe the fifteen knowledge studies as fifteen studies demonstrating improved clinical performance.
Interpret the sparse direct evidence Your conclusion about clinical performance remains primarily dependent on the three studies that actually measured performance. The knowledge evidence can add context without erasing the direct evidence gap.

This distinction can feel frustrating because the broader outcome produces a much larger literature. Methodologically, however, fifteen studies answering a neighboring question do not automatically outweigh three studies answering the question you actually asked.

05 · What Researchers Often Get Wrong

Common mistakes when expanding outcomes

Misconception

“If two outcomes are correlated, they are interchangeable”

Correlation does not establish interchangeability. Two outcomes can move together while still representing different constructs, and an intervention's effect on one may not translate predictably into an effect on the other.

Misconception

“Different measurement scales mean different outcomes”

Not necessarily. Different validated instruments can sometimes measure the same underlying outcome domain. Determine what each instrument actually measures before deciding whether the outcomes are conceptually different.

Misconception

“A surrogate is good enough when the real outcome is unavailable”

A surrogate may provide useful indirect evidence, but its adequacy depends on how reliably it informs the target outcome in the relevant context. Scarcity of direct evidence does not validate the surrogate.

Misconception

“The same outcome measured at different times is automatically comparable”

Timing can alter the question substantially. Immediate response, short-term improvement, and sustained long-term benefit may have different meanings even when assessed with the same instrument.

Misconception

“I should exclude studies that do not report my primary outcome”

For many intervention reviews, this can introduce bias. Otherwise eligible studies may have measured but selectively failed to report an outcome. Cochrane therefore advises that outcome reporting should rarely determine study eligibility.

Misconception

“More outcome data means more certainty about my target outcome”

Only evidence that actually informs the target outcome contributes directly to that conclusion. Adding large amounts of evidence about increasingly distant outcomes may increase information while simultaneously increasing indirectness.

06 · What This Means for You

Classify broader outcomes before deciding what they can tell you

When direct outcome evidence is scarce, determine the relationship between the available outcome and the target outcome before deciding how it belongs in the review.

A simple decision framework

If different instruments measure substantially the same outcome construct
They may be eligible for the same outcome domain, using synthesis methods appropriate to the measurement differences.
If the outcome is related but conceptually distinct
Consider including it as a separate outcome rather than treating it as equivalent to the target outcome.
If the available outcome is a surrogate or intermediate endpoint
Assess the evidence supporting its relationship with the target outcome and explicitly account for indirectness.
If follow-up timing differs materially from the time horizon of interest
Keep the time points distinct or explain why evidence from one time point can inform another.
If the broader outcome requires several unsupported assumptions to answer the target question
Do not use it as a substitute merely because direct evidence is sparse.

Broadening outcomes is only one possible response to scarcity. In another review, the more relevant question may be whether to broaden the population or incorporate evidence from related conditions, interventions, or contexts. These decisions should be considered independently because each changes a different part of the evidentiary relationship.

07 · A Quick Checklist

Before broadening the outcomes

Before adding broader outcomes, check:
Define the outcome that directly answers the research question before examining which outcomes are most commonly reported.
Determine whether the proposed broader outcome is another measure of the same construct or a genuinely different outcome.
Check how each study defines and operationalizes outcomes rather than relying only on outcome labels.
Examine whether differences in follow-up time change the substantive meaning of the outcome.
If using a surrogate or intermediate outcome, evaluate the basis for assuming that it predicts or represents the target outcome.
Keep conceptually distinct outcomes separate during synthesis rather than combining them merely to increase the amount of data.
Do not automatically exclude otherwise eligible studies solely because the outcome of interest is unreported.
Report outcome indirectness explicitly when broader outcomes are used to inform conclusions about the target outcome.
08 · Frequently Asked Questions

Questions about broadening outcomes in sparse evidence

Can different measurement instruments be combined?

Sometimes. If the instruments measure substantially the same construct, their results may be synthesized using appropriate methods. Before combining them, confirm conceptual comparability and consider differences in scale direction, measurement properties, and interpretation.

Can I use a surrogate outcome when the final outcome is unavailable?

A surrogate can provide indirect evidence, but its usefulness depends on the strength and relevance of its relationship with the target outcome. Clearly identify the surrogate and avoid presenting its effect as though the final outcome itself had been measured.

Are short-term and long-term outcomes the same outcome?

They may measure the same construct but answer different temporal questions. If duration or persistence of an effect matters, short-term evidence should not automatically be treated as direct evidence of long-term benefit.

Can knowledge scores be used as evidence of skills?

Knowledge may contribute to skill development, but a knowledge assessment and a performance assessment measure different constructs. Evidence of improved knowledge should not automatically be interpreted as evidence of improved performance.

Should I add secondary outcomes if my primary outcome has little evidence?

Potentially, if those outcomes are meaningful to the research question and their inclusion is methodologically justified. They should retain their own interpretive role rather than being promoted to substitutes for the primary outcome simply because more data are available.

What if a study measured my outcome but did not report the results?

Do not assume that the study provides no relevant evidence. Selective non-reporting can itself create bias. Depending on the review, you may examine protocols, registrations, supplementary materials, other reports, or contact study authors when appropriate.

Does using an indirect outcome always mean the evidence is unusable?

No. Indirect evidence can be informative. The issue is how much confidence you can place in the inference from the measured outcome to the outcome of interest. The degree of indirectness should influence interpretation rather than being treated as an automatic include-or-exclude rule.

09 · The Bottom Line

More outcomes do not necessarily mean more evidence for your outcome

The Bottom Line

When direct outcome evidence is scarce, broaden the outcomes only when you can explain how the additional measures or endpoints relate to the outcome your research question actually asks about.

Different instruments may measure the same construct, while related, surrogate, intermediate, or differently timed outcomes may provide increasingly indirect evidence. Preserve those distinctions so that a larger body of available data does not become a stronger claim than the evidence can support.

10 · Sources and Further Reading

Sources and further reading

11 · Cite this Guide

How to Cite This Guide

This guide is intended to be read, shared, and used in research, teaching, and academic work. If you draw on its ideas, explanations, or other content, please acknowledge the source by citing the guide. Doing so gives appropriate credit and helps your readers locate the original resource.

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