Causation and Experiments

CO-3: Describe the strengths and limitations of designed experiments and observational studies.
LO 3.2: Explain how the study design impacts the types of conclusions that can be drawn.
LO 3.3: Identify and define key features of experimental design (randomized, blind etc.).

Recall that in an experiment, it is the researchers who assign values of the explanatory variable to the participants. The key to ensuring that individuals differ only with respect to explanatory values — which is also the key to establishing causation — lies in the way this assignment is carried out. Let’s return to the smoking cessation study as a context to explore the essential ingredients of experimental design.


In our discussion of the distinction between observational studies and experiments, we described the following experiment: collect a representative sample of 1,000 individuals from the population of smokers who are just now trying to quit. We divide the sample into 4 groups of 250 and instruct each group to use a different method to quit. One year later, we contact the same 1,000 individuals and determine whose attempts succeeded while using our designated method.

This was an experiment, because the researchers themselves determined the values of the explanatory variable of interest for the individuals studied, rather than letting them choose.

We will begin by using the context of this smoking cessation example to illustrate the specialized vocabulary of experiments. First of all, the explanatory variable, or factor, in this case is the method used to quit. The different imposed values of the explanatory variable, or treatments (common abbreviation: ttt), consist of the four possible quitting methods. The groups receiving different treatments are called treatment groups. The group that tries to quit without drugs or therapy could be called the control group — those individuals on whom no specific treatment was imposed. Ideally, the subjects (human participants in an experiment) in each treatment group differ from those in the other treatment groups only with respect to the treatment (quitting method). As mentioned in our discussion of why lurking variables prevent us from establishing causation in observational studies, eliminating all other differences among treatment groups will be the key to asserting causation via an experiment. How can this be accomplished?

Randomized Controlled Experiments

Your intuition may already tell you, correctly, that random assignment to treatments is the best way to prevent treatment groups of individuals from differing from each other in ways other than the treatment assigned. Either computer software or tables can be utilized to accomplish the random assignment. The resulting design is called a randomized controlled experiment, because researchers control values of the explanatory variable with a randomization procedure. Under random assignment, the groups should not differ significantly with respect to any potential lurking variable. Then, if we see a relationship between the explanatory and response variables, we have evidence that it is a causal one.

A visual representation of the experimental study. A large circle represents the entire population. Through random selection we generate the sample, which is represented as a smaller circle. The circle representing the samples is divided up evenly into 4 pieces, each piece representing one value of the explanatory variable (method). The pieces are treatment groups randomly assigned by researchers.


  • Note that in a randomized controlled experiment, a randomization procedure may be used in two phases. First, a sample of subjects is collected. Ideally it would be a random sample so that it would be perfectly representative of the entire population.
  • Often researchers have no choice but to recruit volunteers. Using volunteers may help to offset one of the drawbacks to experimentation which will be discussed later, namely the problem of noncompliance.
  • Second, we assign individuals randomly to the treatment groups to ensure that the only difference between them will be due to the treatment and we can get evidence of causation. At this stage, randomization is vital.

Let’s discuss some other issues related to experimentation.

Inclusion of a Control Group

A common misconception is that an experiment must include a control group of individuals receiving no treatment. There may be situations where a complete lack of treatment is not an option, or where including a control group is ethically questionable, or where researchers explore the effects of a treatment without making a comparison. Here are a few examples:


If doctors want to conduct an experiment to determine whether Prograf or Cyclosporin is more effective as an immunosuppressant, they could randomly assign transplant patients to take one or the other of the drugs. It would, of course, be unethical to include a control group of patients not receiving any immunosuppressants.


Recently, experiments have been conducted in which the treatment is a highly invasive brain surgery. The only way to have a legitimate control group in this case is to randomly assign half of the subjects to undergo the entire surgery except for the actual treatment component (inserting stem cells into the brain). This, of course, is also ethically problematic (but, believe it or not, is being done).


There may even be an experiment designed with only a single treatment. For example, makers of a new hair product may ask a sample of individuals to treat their hair with that product over a period of several weeks, then assess how manageable their hair has become. Such a design is clearly flawed because of the absence of a comparison group, but it is still an experiment because use of the product has been imposed by its manufacturers, rather than chosen naturally by the individuals. A flawed experiment is nevertheless an experiment.


  • The word control is used in at least three different senses.
    • In the context of observational studies, we control for a confounding variable by separating it out.
    • Referring to an experiment as a controlled experiment stresses that the values of the experiment’s explanatory variables (factors) have been assigned by researchers, as opposed to having occurred naturally.
    • In the context of experiments, the control group consists of subjects who do not receive a treatment, but who are otherwise handled identically to those who do receive the treatment.

Blind and Double-Blind Experiments

Suppose the experiment about methods for quitting smoking were carried out with randomized assignments of subjects to the four treatments, and researchers determined that the percentage succeeding with the combination drug/therapy method was highest, and the percentage succeeding with no drugs or therapy was lowest. In other words, suppose there is clear evidence of an association between method used and success rate. Could it be concluded that the drug/therapy method causes success more than trying to quit without using drugs or therapy? Perhaps.

Although randomized controlled experiments do give us a better chance of pinning down the effects of the explanatory variable of interest, they are not completely problem-free. For example, suppose that the manufacturers of the smoking cessation drug had just launched a very high-profile advertising campaign with the goal of convincing people that their drug is extremely effective as a method of quitting.

Even with a randomized assignment to treatments, there would be an important difference among subjects in the four groups: those in the drug and combination drug/therapy groups would perceive their treatment as being a promising one, and may be more likely to succeed just because of added confidence in the success of their assigned method. Therefore, the ideal circumstance is for the subjects to be unaware of which treatment is being administered to them: in other words, subjects in an experiment should be (if possible) blind to which treatment they received.

How could researchers arrange for subjects to be blind when the treatment involved is a drug? They could administer a placebo pill to the control group, so that there are no psychological differences between those who receive the drug and those who do not. The word “placebo” is derived from a Latin word that means “to please.” It is so named because of the natural tendency of human subjects to improve just because of the “pleasing” idea of being treated, regardless of the benefits of the treatment itself. When patients improve because they are told they are receiving treatment, even though they are not actually receiving treatment, this is known as the placebo effect.

Next, how could researchers arrange for subjects to be blind when the treatment involved is a type of therapy? This is more problematic. Clearly, subjects must be aware of whether they are undergoing some type of therapy or not. There is no practical way to administer a “placebo” therapy to some subjects. Thus, the relative success of the drug/therapy treatment may be due to subjects’ enhanced confidence in the success of the method they happened to be assigned. We may feel fairly certain that the method itself causes success in quitting, but we cannot be absolutely sure.

When the response of interest is fairly straightforward, such as giving up cigarettes or not, then recording its values is a simple process in which researchers need not use their own judgment in making an assessment. There are many experiments where the response of interest is less definite, such as whether or not a cancer patient has improved, or whether or not a psychiatric patient is less depressed. In such cases, it is important for researchers who evaluate the response to be blind to which treatment the subject received, in order to prevent the experimenter effect from influencing their assessments. If neither the subjects nor the researchers know who was assigned what treatment, then the experiment is called double-blind.

The most reliable way to determine whether the explanatory variable is actually causing changes in the response variable is to carry out a randomized controlled double-blind experiment. Depending on the variables of interest, such a design may not be entirely feasible, but the closer researchers get to achieving this ideal design, the more convincing their claims of causation (or lack thereof) are.

Did I Get This?: Experiments

Pitfalls in Experimentation

Some of the inherent difficulties that may be encountered in experimentation are the Hawthorne effect, lack of realism, noncompliance, and treatments that are unethical, impossible, or impractical to impose.

We already introduced a hypothetical experiment to determine if people tend to snack more while they watch TV:

  • Recruit participants for the study.
  • While they are presumably waiting to be interviewed, half of the individuals sit in a waiting room with snacks available and a TV on. The other half sit in a waiting room with snacks available and no TV, just magazines.
  • Researchers determine whether people consume more snacks in the TV setting.

Suppose that, in fact, the subjects who sat in the waiting room with the TV consumed more snacks than those who sat in the room without the TV. Could we conclude that in their everyday lives, and in their own homes, people eat more snacks when the TV is on? Not necessarily, because people’s behavior in this very controlled setting may be quite different from their ordinary behavior.

If they suspect their snacking behavior is being observed, they may alter their behavior, either consciously or subconsciously. This phenomenon, whereby people in an experiment behave differently from how they would normally behave, is called the Hawthorne effect. Even if they don’t suspect they are being observed in the waiting room, the relationship between TV and snacking in the waiting room might not be representative of what it is in real life.

One of the greatest advantages of an experiment — that researchers take control of the explanatory variable — can also be a disadvantage in that it may result in a rather unrealistic setting. Lack of realism (also called lack of ecological validity) is a possible drawback to the use of an experiment rather than an observational study to explore a relationship. Depending on the explanatory variable of interest, it may be quite easy or it may be virtually impossible to take control of the variable’s values and still maintain a fairly natural setting.

In our hypothetical smoking cessation example, both the observational study and the experiment were carried out on a random sample of 1,000 smokers with intentions to quit. In the case of the observational study, it would be reasonably feasible to locate 1,000 such people in the population at large, identify their intended method, and contact them again a year later to establish whether they succeeded or not.

In the case of the experiment, it is not so easy to take control of the explanatory variable (cessation method) merely by telling all 1,000 subjects what method they must use. Noncompliance (failure to submit to the assigned treatment) could enter in on such a large scale as to render the results invalid.

In order to ensure that the subjects in each treatment group actually undergo the assigned treatment, researchers would need to pay for the treatment and make it easily available. The cost of doing that for a group of 1,000 people would go beyond the budget of most researchers.

Even if the drugs or therapy were paid for, it is very unlikely that most of the subjects contacted at random would be willing to use a method not of their own choosing, but dictated by the researchers. From a practical standpoint, such a study would most likely be carried out on a smaller group of volunteers, recruited via flyers or some other sort of advertisement.

The fact that they are volunteers might make them somewhat different from the larger population of smokers with intentions to quit, but it would reduce the more worrisome problem of non-compliance. Volunteers may have a better overall chance of success, but if researchers are primarily concerned with which method is most successful, then the relative success of the various methods should be roughly the same for the volunteer sample as it would be for the general population, as long as the methods are randomly assigned. Thus, the most vital stage for randomization in an experiment is during the assignment of treatments, rather than the selection of subjects.

There are other, more serious drawbacks to experimentation, as illustrated in the following hypothetical examples:


Suppose researchers want to determine if the drug Ecstasy causes memory loss. One possible design would be to take a group of volunteers and randomly assign some to take Ecstasy on a regular basis, while the others are given a placebo. Test them periodically to see if the Ecstasy group experiences more memory problems than the placebo group.

The obvious flaw in this experiment is that it is unethical (and actually also illegal) to administer a dangerous drug like Ecstasy, even if the subjects are volunteers. The only feasible design to seek answers to this particular research question would be an observational study.


Suppose researchers want to determine whether females wash their hair more frequently than males.

It is impossible to assign some subjects to be female and others male, and so an experiment is not an option here. Again, an observational study would be the only way to proceed.


Suppose researchers want to determine whether being in a lower income bracket may be responsible for obesity in women, at least to some extent, because they can’t afford more nutritious meals and don’t have the means to participate in fitness activities.

The socioeconomic status of the study subject is a variable that cannot be controlled by the researchers, so an experiment is impossible. (Even if the researchers could somehow raise the money to provide a random sample of women with substantial salaries, the effects of their eating habits during their lives before the study began would still be present, and would affect the study’s outcome.)

These examples should convince you that, depending on the variables of interest, researching their relationship via an experiment may be too unrealistic, unethical, or impractical. Observational studies are subject to flaws, but often they are the only recourse.

Let’s summarize what we’ve learned so far:

1. Observational studies:

  • The explanatory variable’s values are allowed to occur naturally.
  • Because of the possibility of lurking variables, it is difficult to establish causation.
  • If possible, control for suspected lurking variables by studying groups of similar individuals separately.
  • Some lurking variables are difficult to control for; others may not be identified.

2. Experiments

  • The explanatory variable’s values are controlled by researchers (treatment is imposed).
  • Randomized assignment to treatments automatically controls for all lurking variables.
  • Making subjects blind avoids the placebo effect.
  • Making researchers blind avoids conscious or subconscious influences on their subjective assessment of responses.
  • A randomized controlled double-blind experiment is generally optimal for establishing causation.
  • A lack of realism may prevent researchers from generalizing experimental results to real-life situations.
  • Noncompliance may undermine an experiment. A volunteer sample might solve (at least partially) this problem.
  • It is impossible, impractical, or unethical to impose some treatments.