Clinical Trial

• research study to answer ?'s about vaccines/therapies/new ways of using known tx's
• aka medical research & research studies
• used to determine whether new drugs or tx's are safe & effective
• carefully conducted clinical trials are the fastest & safest way to find tx's that work in ppl
• trials are in 4 phases

Phase I

tests a new drug or tx in a small group

initial studies to determine the metabolism & pharmacologic actions of drugs in humans, the side effects, associated with increasing doses, & to gain early evidence of effectiveness

may include healthy participants &/or pts

Phase II

expands the study to a larger group of ppl

controlled clinical studies conducted to evaluate the effectiveness of the drug for a particular indication or indications in pts with the disease or condition under study & to determine the common short-term side effects & risks

Phase III

expands the study to an even larger group of ppl

expanded controlled & uncontrolled trials after preliminary evidence suggesting effectiveness of the drug has been obtained, and are intended to gather additional info to evaluate the overall benefit-risk relationship of the drug & provide adequate basis for physician labeling

Phase IV

takes place after the drug or tx has been licensed & marketed

post-marketing studies to delineate additional info including the drug's risks, benefits, & optimal use

Dose-Ranging Study

A clinical trial in which 2 or more doses of an agent (drug, etc) are tested against each other to determine which dose works best and is least harmful.

Double-Blind Study

A clinical trial design, neither the subject or researcher knows which subjects receiving experimental drug or placebo or another therapy.

"objective" results - expectations of experiment doesn't affect the outcome

aka double-masked study

Empirical

Based on experimental data, not on a theory

Epidemiology

brach of medical science that deals with the study of incidence & distribution & control of a disease in a population

Hypothesis

A supposition or assumption advanced as a basis for reasoning or argument, or as a guide to experimental investigation

Inclusion/Exclusion Criteria

The medical or social standards determining whether a person may or may not be allowed to enter a clinical trial

based on factors like age, gender, type/stage of disease, previous tx hx, & other medical conditions, etc.

Used to identify appropriate participants & keep them safe (not to reject ppl personally)

Off-Label Use

A drug prescribed for conditions other than those approved by the FDA

Orphan Drugs

An FDA category that refers to meds used to tx diseases and conditions that occur rarely

little financial incentive for pharmaceutical industry to develop meds for these diseases/conditions

orphan drug status gives a manufacturer specific financial incentives to develop & provide such meds

Placebo

an inactive pill, liquid, or powder that has no tx value

in clinical trials, experimental tx's are often compared with placebos to assess the tx's effectiveness

Randomization

A method based on chance by which study participants are assigned to a tx group

minimizes the differences among groups by equally distributing ppl with particular characteristics among all the trial arms

researchers do not know which tx is better

Randomized Trial

A study in which participants are randomly assigned

(i.e., by chance), to one or two or more tx arms of a clinical trial

occasionally placebos are utilized

Side Effects

Any undesired actions or effects of a drug or tx

(negative effects)

Experimental drugs must be evaluated for both immediate and long-term side effects

Single-Blind Study

A study in which one party, either the investigator or participant, is unaware of what med the participant is taking

aka single-masked study

Standards of care

Tx regimen or medical management based on state of the art participant care

Statistical Significance

The probability that an event or difference occurred by chance alone. In clinical trials, the level of statistical significance depends on the # of participants studied & the observations made, as well as the magnitude of differences observed

Study Endpoint

A primary or secondary outcome used to judge the effectiveness of a tx