EXPERIMENTAL STUDY DESIGNS

Dr. Adora M. Abat

Definition a.k.a. Intervention studies the researcher administers or withholds a factor to see how the dependent variable is affected by the independent variable Description subjects are followed up for the outcome of interest the investigator assigns the subjects to the exposure or treatment groups (experimental or intervention group vs. control or reference group) allocation or treatment done randomly studies how the dependent variable affects the independent variable while controlling the affects of confounders Uses

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the risk of death from a particular disease, may diminish signs and symptoms, or prevent recurrence of the disease example: randomized trial among breast cancer patients that compared radical mastectomy with more limited resection

Types of experimental study according to allocation of treatment 1. Clinical Trials a. Allocation to individuals b. Ex. Effectiveness of a Drug A vs. Drug B in the treatment of hypertension; randomized trial among breast cancer patients that compared radical mastectomy with more limited resection; field trial of the poliomyelitis vaccine among healthy children Community Trials a. Allocation to group of individuals or communities b. Less expensive than large scale clinical trials c. Ex. Water fluoridation experiment

most powerful design in being able to elucidate possible cause and effect relationship done to resolve issues about the beneficial effect of a treatment that are generated from the observational study provides data on the harmful effects of the treatment as well

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Advantages the investigator is the one who assigns the subjects to the exposure or treatment groups the allocation of treatment is done randomly Disadvantages 1. Ethical issues a. treatment should not be withheld to the subject b. harmful experiments should not be done Feasibility a. Others want to be on the treatment's side than control, sometimes not feasible More costly than observational studies

Reference or Control Population The population to which one would like to generalize the results of the experimental study Ex. Males and females above 40 years for an experimental study on the effects of aspirin on cardiovascular mortality Experimental or Intervention Population The population that is actually studied SCHEMA

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(+) Outcome Expt'l group Eligible subjects Control group (+) Outcome

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(-) Outcome

Types of experimental studies according to type of exposure 1. Prophylactic Trials a. Involve the evaluation of whether a treatment or procedure reduces the risk of developing disease b. Conducted among healthy individuals who are considered at risk of dose c. example: field trial of the poliomyelitis vaccine among healthy children 2. Therapeutic Trials a. Conducted among patients with particular disease b. Done to see whether a certain treatment or procedure may reduce

(-) Outcome Direction inquiry of

Considerations in the choice of an experimental population 1. should be sufficiently large 2. should experience a sufficient number of endpoints or outcomes within the study group

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obtaining complete and accurate follow up information from the participants is feasible

intervention is not effective to bring about change Solve for sample size n= 2 (Z + Z)2 S2 d2 Where: n = sample size Z = z deviate corresponding to the error Z = z deviate corresponding to the error S2 = variance d = difference to be detected

Allocation of Treatment 1. Randomization a. Procedure for assigning treatments to individuals in such a way that all possible assignments of treatments to individuals are equally likely b. advantages: i. eliminates selection bias ii. with sufficiently large sample size, the different groups tend to be more comparable in terms of known and unknown factors iii. gives validity to the statistical treatment of data 2. Blocking a. Individuals with the same characteristics that affect the outcome are first grouped into blocks before treatment is allocated to the individuals in a block b. In community trials, randomization may not be necessary Maintaining and Assessment of Compliance Strategies: 1. selection of populations who are both interested and reliable 2. frequent contact with participants 3. use of incentives 4. use of calendar packs of study population Measurement of compliance levels: 1. Self reports 2. Pill counts on the returned (unused) pills 3. Laboratory determinations Ascertainment of Outcome Strategies: 1. Blinding a. Single blind observer b. Double blind subject and observer c. Triple blind subject, observer and data analyst 2. Use of more objective definition of outcome 3. Use of same procedure for diagnosis Interpretation of Results In community trials, if there are changes in the risk factor levels and no changes in the disease rates, these may be explained by: 1. No association between the risk factor and the disease 2. Changes in the risk factor levels were not large enough to effect changes in disease rate 3. Risk factor changes should have been identified earlier in life to produce changes 4. The risk factor changes should be sustained for longer periods to be effective In community trials, absence of many changes in the levels of the risk factors may mean either the intervention package is not effective or the efforts in the implementation of the

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