Study Design 101

Helpful formulas Finding specific study types Glossary References

MetaAnalysis Systematic Review Practice Guideline Randomized Controlled Trial Cohort Study Case Control Study Case Report

Cohort Study
Definition
A study design where one or more samples (called cohorts) are followed prospectively and subsequent status evaluations with respect to a disease or outcome are conducted to determine which initial participants exposure characteristics (risk factors) are associated with it. As the study is conducted, outcome from participants in each cohort is measured and relationships with specific characteristics determined

Advantages
Subjects in cohorts can be matched, which limits the influence of confounding variables Standardization of criteria/outcome is possible Easier and cheaper than a randomized controlled trial (RCT)

Disadvantages
Cohorts can be difficult to identify due to confounding variables No randomization, which means that imbalances in patient characteristics could exist Blinding/masking is difficult Outcome of interest could take time to occur

Design pitfalls to look out for

The cohorts need to be chosen from separate, but similar, populations. How many differences are there between the control cohort and the experiment cohort? Will those differences cloud the study outcomes?

Fictitious Example
A cohort study was designed to assess the impact of sun exposure on skin damage in beach volleyball players. During a weekend tournament, players from one team wore waterproof, SPF 35 sunscreen, while players from the other team did not wear any sunscreen. At the end of the volleyball tournament players' skin from both teams was analyzed for texture, sun damage, and burns. Comparisons of skin damage were then made based on the use of sunscreen. The analysis showed a significant difference between the cohorts in terms of the skin damage.

Real-life Example
Ramchand, R., Ialongo, N. S., & Chilcoat, H. D. (2007). The effect of working for pay on adolescent tobacco use. American Journal of Public Health, 97(11), 2056-2062. This study uses data collected from high school students from Baltimore, Maryland, and studies the differences in initiation of tobacco use between a cohort of adolescents that started working for pay and a cohort of adolescents that did not work. The results suggest that adolescents who work for pay have a higher risk of initiating tobacco use. Lindenauer, P. K., Rothberg, M. B., Pekow, P. S., Kenwood, C., Benjamin, E. M., & Auerbach, A. D. (2007). Outcomes of care by hospitalists, general internists, and family physicians. New England Journal of Medicine, 357(25), 2589-2600. To study effects of hospitalists, general internists, and family physicians on patient care, patients that were hospitalized with certain conditions under the care of hospitalists, general internists, and family physicians were separated into three cohorts. The results showed that patients cared for by hospitalists had shorter hospital stays and lower costs than those cared for by general internists or family physicians. Nichol, K. L., Nordin, J. D., Nelson, D. B., Mullooly, J. P., & Hak, E. (2007). Effectiveness of influenza vaccine in the community-dwelling elderly. New England Journal of Medicine, 357(14), 1373-1381. To determine the long-term effectiveness of influenza vaccines in elderly people, cohorts of vaccinated elderly and unvaccinated community-dwelling elderly were studied. The results suggest that the elderly who are vaccinated have a reduced risk of hospitalization for pneumonia or influenza.

Related Formulas
Relative Risk Odds Ratio

Related Terms
Cohort Confounding Variables Population Bias/Volunteer Bias Prospective Study

Now test yourself!

1. In a cohort study, an exposure is assessed and then participants are followed prospectively to observe whether they develop the outcome.

a) True b) False 2. Cohort Studies generally look at which of the following?

a) Determining the sensitivity and specificity of diagnostic methods b) Identifying patient characteristics or risk factors associated with a disease or outcome c) Variations among the clinical manifestations of patients with a disease d) The impact of blinding or masking a study population

Study Design 101

Helpful formulas Finding specific study types Glossary References

MetaAnalysis Systematic Review Practice Guideline Randomized Controlled Trial Cohort Study Case Control Study Case Report

Randomized Controlled Trial

Definition
A study design that randomly assigns participants into an experimental group or a control group. As the study is conducted, the only expected difference between the control and experimental groups in a randomized controlled trial (RCT) is the outcome variable being studied.

Advantages
Good randomization will wash out any population bias Easier to blind/mask than observational studies Results can be analyzed with well known statistical tools Populations of participating individuals are clearly identified

Disadvantages

Expensive in terms of time and money Volunteer biases: the population that participates may not be representative of the whole Does not reveal causation Loss to follow-up attributed to treatment

Design pitfalls to look out for

An RCT should be a study of one population only. Was the randomization actually random, or are there really two populations being studied? The variables being studied should be the only variables between the experimental group and the control group. Are there any confounding variables between the groups?

Fictitious Example
To determine how a new type of short wave UVA-blocking sunscreen affects the general health of skin in comparison to a regular long wave UVA-blocking sunscreen, 40 trial participants were randomly separated into equal groups of 20: an experimental group and a control group. All participants' skin health was then initially evaluated. The experimental group wore the short wave UVA-blocking sunscreen daily, and the control group wore the long wave UVA-blocking sunscreen daily. After one year, the general health of the skin was measured in both groups and statistically analyzed. In the control group, wearing long wave UVA-blocking sunscreen daily led to improvements in general skin health for 60% of the participants. In the experimental group, wearing short wave UVA-blocking sunscreen daily led to improvements in general skin health for 75% of the participants.

Real-life Examples
Ensrud, K. E., Stock, J. L., Barrett-Connor, E., Grady, D., Mosca, L., Khaw, K., et al. (2008). Effects of raloxifene on fracture risk in postmenopausal women: The raloxifene use for the heart trial. Journal of Bone and Mineral Research, 23(1), 112-120. This research studied the effect of raloxifene on fracture risk in postmenopausal women, and found that the women who took raloxifene over the same five year period of time as the women who did not reduced their risk of clinical vertebrate fracture. Krishna, S., Balas, E. A., Francisco, B. D., & Knig, P. (2006). Effective and sustainable multimedia education for children with asthma: A randomized controlled trial. Children's Health Care, 35(1), 75-90. This article reports on the effect of multimedia education for children with asthma. A control group of pediatric patients with asthma was given standard asthma educational resources, while the experimental group of pediatric patients with asthma was given standard resources plus multimedia resources. The study found a reduction in daily symptoms, in emergency room visits, in school days missed, and in days of limited activity in the group given multimedia education resources. Mller, O., Traor, C., Kouyat, B., Y, Y., Frey, C., Coulibaly, B., et al. (2006). Effects of insecticidetreated bednets during early infancy in an African area of intense malaria transmission: A randomized controlled trial. Bulletin of the World Health Organization, 84(2), 120-126.

The study done in this example was to determine whether the early use of bed nets had an effect on the morbidity rate of infants in rural Burkina Faso. An experimental group of infants used bed nets at birth, and a control group of infants used bed nets at six months of age. Results showed no significant difference in morbidity rates, but use of bed nets from birth was related to lower incidence of both malaria and moderate to severe anemia.

Related Formulas
Relative Risk Odds Ratio

Related Terms
Blinding/Masking Causation Confounding Variables Correlation Double Blinding/Masking Null Hypothesis Population/Cohort Population Bias/Volunteer Bias Randomization Research (alternative) Hypothesis Sensitivity Specificity Type 1 error Type 2 error

Now test yourself!

1. Having a volunteer bias in the population group is a good thing because it means the study participants are eager and make the study even stronger.

a) True b) False 2. Why is randomization important to assignment in an RCT?

a) It enables blinding/masking b) So causation may be extrapolated from results c) It balances out individual characteristics between groups. d) a and c e) b and c

Study Design 101

Helpful formulas Finding specific study types Glossary References

MetaAnalysis Systematic Review Practice Guideline Randomized Controlled Trial Cohort Study Case Control Study Case Report

Randomized Controlled Trial

Definition
A study design that randomly assigns participants into an experimental group or a control group. As the study is conducted, the only expected difference between the control and experimental groups in a randomized controlled trial (RCT) is the outcome variable being studied.

Advantages
Good randomization will wash out any population bias Easier to blind/mask than observational studies Results can be analyzed with well known statistical tools Populations of participating individuals are clearly identified

Disadvantages
Expensive in terms of time and money Volunteer biases: the population that participates may not be representative of the whole Does not reveal causation Loss to follow-up attributed to treatment

Design pitfalls to look out for

An RCT should be a study of one population only. Was the randomization actually random, or are there really two populations being studied?

The variables being studied should be the only variables between the experimental group and the control group. Are there any confounding variables between the groups?

Fictitious Example
To determine how a new type of short wave UVA-blocking sunscreen affects the general health of skin in comparison to a regular long wave UVA-blocking sunscreen, 40 trial participants were randomly separated into equal groups of 20: an experimental group and a control group. All participants' skin health was then initially evaluated. The experimental group wore the short wave UVA-blocking sunscreen daily, and the control group wore the long wave UVA-blocking sunscreen daily. After one year, the general health of the skin was measured in both groups and statistically analyzed. In the control group, wearing long wave UVA-blocking sunscreen daily led to improvements in general skin health for 60% of the participants. In the experimental group, wearing short wave UVA-blocking sunscreen daily led to improvements in general skin health for 75% of the participants.

Real-life Examples
Ensrud, K. E., Stock, J. L., Barrett-Connor, E., Grady, D., Mosca, L., Khaw, K., et al. (2008). Effects of raloxifene on fracture risk in postmenopausal women: The raloxifene use for the heart trial. Journal of Bone and Mineral Research, 23(1), 112-120. This research studied the effect of raloxifene on fracture risk in postmenopausal women, and found that the women who took raloxifene over the same five year period of time as the women who did not reduced their risk of clinical vertebrate fracture. Krishna, S., Balas, E. A., Francisco, B. D., & Knig, P. (2006). Effective and sustainable multimedia education for children with asthma: A randomized controlled trial. Children's Health Care, 35(1), 75-90. This article reports on the effect of multimedia education for children with asthma. A control group of pediatric patients with asthma was given standard asthma educational resources, while the experimental group of pediatric patients with asthma was given standard resources plus multimedia resources. The study found a reduction in daily symptoms, in emergency room visits, in school days missed, and in days of limited activity in the group given multimedia education resources. Mller, O., Traor, C., Kouyat, B., Y, Y., Frey, C., Coulibaly, B., et al. (2006). Effects of insecticidetreated bednets during early infancy in an African area of intense malaria transmission: A randomized controlled trial. Bulletin of the World Health Organization, 84(2), 120-126. The study done in this example was to determine whether the early use of bed nets had an effect on the morbidity rate of infants in rural Burkina Faso. An experimental group of infants used bed nets at birth, and a control group of infants used bed nets at six months of age. Results showed no significant difference in morbidity rates, but use of bed nets from birth was related to lower incidence of both malaria and moderate to severe anemia.

Related Formulas
Relative Risk Odds Ratio

Related Terms

Blinding/Masking Causation Confounding Variables Correlation Double Blinding/Masking Null Hypothesis Population/Cohort Population Bias/Volunteer Bias Randomization Research (alternative) Hypothesis Sensitivity Specificity Type 1 error Type 2 error

Now test yourself!

1. Having a volunteer bias in the population group is a good thing because it means the study participants are eager and make the study even stronger.

a) True b) False 2. Why is randomization important to assignment in an RCT?

a) It enables blinding/masking b) So causation may be extrapolated from results c) It balances out individual characteristics between groups. d) a and c e) b and c

Study Design 101

Helpful formulas Finding specific study types Glossary References

MetaAnalysis Systematic Review

Practice Guideline Randomized Controlled Trial Cohort Study Case Control Study Case Report

Randomized Controlled Trial

Definition
A study design that randomly assigns participants into an experimental group or a control group. As the study is conducted, the only expected difference between the control and experimental groups in a randomized controlled trial (RCT) is the outcome variable being studied.

Advantages
Good randomization will wash out any population bias Easier to blind/mask than observational studies Results can be analyzed with well known statistical tools Populations of participating individuals are clearly identified

Disadvantages
Expensive in terms of time and money Volunteer biases: the population that participates may not be representative of the whole Does not reveal causation Loss to follow-up attributed to treatment

Design pitfalls to look out for

An RCT should be a study of one population only. Was the randomization actually random, or are there really two populations being studied? The variables being studied should be the only variables between the experimental group and the control group. Are there any confounding variables between the groups?

Fictitious Example
To determine how a new type of short wave UVA-blocking sunscreen affects the general health of skin in comparison to a regular long wave UVA-blocking sunscreen, 40 trial participants were randomly separated into equal groups of 20: an experimental group and a control group. All participants' skin health was then initially evaluated. The experimental group wore the short wave UVA-blocking sunscreen daily, and the control group wore the long wave UVA-blocking sunscreen daily.

After one year, the general health of the skin was measured in both groups and statistically analyzed. In the control group, wearing long wave UVA-blocking sunscreen daily led to improvements in general skin health for 60% of the participants. In the experimental group, wearing short wave UVA-blocking sunscreen daily led to improvements in general skin health for 75% of the participants.

Real-life Examples
Ensrud, K. E., Stock, J. L., Barrett-Connor, E., Grady, D., Mosca, L., Khaw, K., et al. (2008). Effects of raloxifene on fracture risk in postmenopausal women: The raloxifene use for the heart trial. Journal of Bone and Mineral Research, 23(1), 112-120. This research studied the effect of raloxifene on fracture risk in postmenopausal women, and found that the women who took raloxifene over the same five year period of time as the women who did not reduced their risk of clinical vertebrate fracture. Krishna, S., Balas, E. A., Francisco, B. D., & Knig, P. (2006). Effective and sustainable multimedia education for children with asthma: A randomized controlled trial. Children's Health Care, 35(1), 75-90. This article reports on the effect of multimedia education for children with asthma. A control group of pediatric patients with asthma was given standard asthma educational resources, while the experimental group of pediatric patients with asthma was given standard resources plus multimedia resources. The study found a reduction in daily symptoms, in emergency room visits, in school days missed, and in days of limited activity in the group given multimedia education resources. Mller, O., Traor, C., Kouyat, B., Y, Y., Frey, C., Coulibaly, B., et al. (2006). Effects of insecticidetreated bednets during early infancy in an African area of intense malaria transmission: A randomized controlled trial. Bulletin of the World Health Organization, 84(2), 120-126. The study done in this example was to determine whether the early use of bed nets had an effect on the morbidity rate of infants in rural Burkina Faso. An experimental group of infants used bed nets at birth, and a control group of infants used bed nets at six months of age. Results showed no significant difference in morbidity rates, but use of bed nets from birth was related to lower incidence of both malaria and moderate to severe anemia.

Related Formulas
Relative Risk Odds Ratio

Related Terms
Blinding/Masking Causation Confounding Variables Correlation Double Blinding/Masking Null Hypothesis Population/Cohort Population Bias/Volunteer Bias Randomization Research (alternative) Hypothesis Sensitivity Specificity Type 1 error

Type 2 error

Now test yourself!

1. Having a volunteer bias in the population group is a good thing because it means the study participants are eager and make the study even stronger.

a) True b) False 2. Why is randomization important to assignment in an RCT?

a) It enables blinding/masking b) So causation may be extrapolated from results c) It balances out individual characteristics between groups. d) a and c e) b and c

Study Design 101

Helpful formulas Finding specific study types Glossary References

MetaAnalysis Systematic Review Practice Guideline Randomized Controlled Trial Cohort Study Case Control Study Case Report

Randomized Controlled Trial

Definition

A study design that randomly assigns participants into an experimental group or a control group. As the study is conducted, the only expected difference between the control and experimental groups in a randomized controlled trial (RCT) is the outcome variable being studied.

Advantages
Good randomization will wash out any population bias Easier to blind/mask than observational studies Results can be analyzed with well known statistical tools Populations of participating individuals are clearly identified

Disadvantages
Expensive in terms of time and money Volunteer biases: the population that participates may not be representative of the whole Does not reveal causation Loss to follow-up attributed to treatment

Design pitfalls to look out for

An RCT should be a study of one population only. Was the randomization actually random, or are there really two populations being studied? The variables being studied should be the only variables between the experimental group and the control group. Are there any confounding variables between the groups?

Fictitious Example
To determine how a new type of short wave UVA-blocking sunscreen affects the general health of skin in comparison to a regular long wave UVA-blocking sunscreen, 40 trial participants were randomly separated into equal groups of 20: an experimental group and a control group. All participants' skin health was then initially evaluated. The experimental group wore the short wave UVA-blocking sunscreen daily, and the control group wore the long wave UVA-blocking sunscreen daily. After one year, the general health of the skin was measured in both groups and statistically analyzed. In the control group, wearing long wave UVA-blocking sunscreen daily led to improvements in general skin health for 60% of the participants. In the experimental group, wearing short wave UVA-blocking sunscreen daily led to improvements in general skin health for 75% of the participants.

Real-life Examples
Ensrud, K. E., Stock, J. L., Barrett-Connor, E., Grady, D., Mosca, L., Khaw, K., et al. (2008). Effects of raloxifene on fracture risk in postmenopausal women: The raloxifene use for the heart trial. Journal of Bone and Mineral Research, 23(1), 112-120.

This research studied the effect of raloxifene on fracture risk in postmenopausal women, and found that the women who took raloxifene over the same five year period of time as the women who did not reduced their risk of clinical vertebrate fracture. Krishna, S., Balas, E. A., Francisco, B. D., & Knig, P. (2006). Effective and sustainable multimedia education for children with asthma: A randomized controlled trial. Children's Health Care, 35(1), 75-90. This article reports on the effect of multimedia education for children with asthma. A control group of pediatric patients with asthma was given standard asthma educational resources, while the experimental group of pediatric patients with asthma was given standard resources plus multimedia resources. The study found a reduction in daily symptoms, in emergency room visits, in school days missed, and in days of limited activity in the group given multimedia education resources. Mller, O., Traor, C., Kouyat, B., Y, Y., Frey, C., Coulibaly, B., et al. (2006). Effects of insecticidetreated bednets during early infancy in an African area of intense malaria transmission: A randomized controlled trial. Bulletin of the World Health Organization, 84(2), 120-126. The study done in this example was to determine whether the early use of bed nets had an effect on the morbidity rate of infants in rural Burkina Faso. An experimental group of infants used bed nets at birth, and a control group of infants used bed nets at six months of age. Results showed no significant difference in morbidity rates, but use of bed nets from birth was related to lower incidence of both malaria and moderate to severe anemia.

Related Formulas
Relative Risk Odds Ratio

Related Terms
Blinding/Masking Causation Confounding Variables Correlation Double Blinding/Masking Null Hypothesis Population/Cohort Population Bias/Volunteer Bias Randomization Research (alternative) Hypothesis Sensitivity Specificity Type 1 error Type 2 error

Now test yourself!

1. Having a volunteer bias in the population group is a good thing because it means the study participants are eager and make the study even stronger.

a) True b) False

2. Why is randomization important to assignment in an RCT?

a) It enables blinding/masking b) So causation may be extrapolated from results c) It balances out individual characteristics between groups. d) a and c e) b and c

Study Design 101

Helpful formulas Finding specific study types Glossary References

MetaAnalysis Systematic Review Practice Guideline Randomized Controlled Trial Cohort Study Case Control Study Case Report

Randomized Controlled Trial

Definition
A study design that randomly assigns participants into an experimental group or a control group. As the study is conducted, the only expected difference between the control and experimental groups in a randomized controlled trial (RCT) is the outcome variable being studied.

Advantages
Good randomization will wash out any population bias Easier to blind/mask than observational studies Results can be analyzed with well known statistical tools Populations of participating individuals are clearly identified

Disadvantages
Expensive in terms of time and money Volunteer biases: the population that participates may not be representative of the whole Does not reveal causation Loss to follow-up attributed to treatment

Design pitfalls to look out for

An RCT should be a study of one population only. Was the randomization actually random, or are there really two populations being studied? The variables being studied should be the only variables between the experimental group and the control group. Are there any confounding variables between the groups?

Fictitious Example
To determine how a new type of short wave UVA-blocking sunscreen affects the general health of skin in comparison to a regular long wave UVA-blocking sunscreen, 40 trial participants were randomly separated into equal groups of 20: an experimental group and a control group. All participants' skin health was then initially evaluated. The experimental group wore the short wave UVA-blocking sunscreen daily, and the control group wore the long wave UVA-blocking sunscreen daily. After one year, the general health of the skin was measured in both groups and statistically analyzed. In the control group, wearing long wave UVA-blocking sunscreen daily led to improvements in general skin health for 60% of the participants. In the experimental group, wearing short wave UVA-blocking sunscreen daily led to improvements in general skin health for 75% of the participants.

Real-life Examples
Ensrud, K. E., Stock, J. L., Barrett-Connor, E., Grady, D., Mosca, L., Khaw, K., et al. (2008). Effects of raloxifene on fracture risk in postmenopausal women: The raloxifene use for the heart trial. Journal of Bone and Mineral Research, 23(1), 112-120. This research studied the effect of raloxifene on fracture risk in postmenopausal women, and found that the women who took raloxifene over the same five year period of time as the women who did not reduced their risk of clinical vertebrate fracture. Krishna, S., Balas, E. A., Francisco, B. D., & Knig, P. (2006). Effective and sustainable multimedia education for children with asthma: A randomized controlled trial. Children's Health Care, 35(1), 75-90. This article reports on the effect of multimedia education for children with asthma. A control group of pediatric patients with asthma was given standard asthma educational resources, while the experimental group of pediatric patients with asthma was given standard resources plus multimedia resources. The study found a reduction in daily symptoms, in emergency room visits, in school days missed, and in days of limited activity in the group given multimedia education resources.

Mller, O., Traor, C., Kouyat, B., Y, Y., Frey, C., Coulibaly, B., et al. (2006). Effects of insecticidetreated bednets during early infancy in an African area of intense malaria transmission: A randomized controlled trial. Bulletin of the World Health Organization, 84(2), 120-126. The study done in this example was to determine whether the early use of bed nets had an effect on the morbidity rate of infants in rural Burkina Faso. An experimental group of infants used bed nets at birth, and a control group of infants used bed nets at six months of age. Results showed no significant difference in morbidity rates, but use of bed nets from birth was related to lower incidence of both malaria and moderate to severe anemia.

Related Formulas
Relative Risk Odds Ratio

Related Terms
Blinding/Masking Causation Confounding Variables Correlation Double Blinding/Masking Null Hypothesis Population/Cohort Population Bias/Volunteer Bias Randomization Research (alternative) Hypothesis Sensitivity Specificity Type 1 error Type 2 error

Now test yourself!

1. Having a volunteer bias in the population group is a good thing because it means the study participants are eager and make the study even stronger.

a) True b) False 2. Why is randomization important to assignment in an RCT?

a) It enables blinding/masking b) So causation may be extrapolated from results c) It balances out individual characteristics between groups. d) a and c e) b and c