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Usually pilot experiments are conducted to gain insights for design of the clinical trial to follow.There are two goals to testing medical treatments: to learn whether they work well enough, called "efficacy" or "effectiveness"; and to learn whether they are safe enough, called "safety".
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Certain functions necessary to the trial, such as monitoring and lab work, may be managed by an outsourced partner, such as a contract research organization or a central laboratory.
Only 10 percent of all drugs started in human clinical trials become an approved drug.
The researchers send the data to the trial sponsor, who then analyzes the pooled data using statistical tests.
Examples of clinical trial goals include assessing the safety and relative effectiveness of a medication or device: While most clinical trials test one alternative to the novel intervention, some expand to three or four and may include a placebo.
For instance, Lady Mary Wortley Montagu, who campaigned for the introduction of inoculation (then called variolation) to prevent smallpox, arranged for seven prisoners who had been sentenced to death to undergo variolation in exchange for their life.