Clinical trials
Mnemonic
Does the drug SWIM?
"Is it Safe?” Assesses safety, toxicity, pharmacokinetics, and pharmacodynamics
“Does it Work?” Assesses treatment efficacy, optimal dosing, and adverse effects.
“Is it as good or better?” Compares the new treatment to the current standard of care (any Improvement?).
“Can it stay?” Detects rare or long-term adverse effects (eg, black box warnings). Can result in treatment being withdrawn from Market.
- Phase 0 trial
- Purpose: Exploratory phase with no therapeutic or diagnostic intent, aimed at gaining insight into pharmacokinetics and pharmacodynamics.
- Only < 1% of the therapeutic dose is used.
- Safety and toxicity are not assessed.
- Study population: Small sample consisting of either healthy individuals or a population with a disease of interest (∼ 10–15).
- Study design: Open-label.
- Phase I trial
- Purpose: Evaluation of pharmacodynamic and pharmacokinetic properties of the drug, safety, and toxicity.
- Evaluation of the maximum tolerated dose.
- Study population: Small number of healthy individuals or patients with a specific disease (∼ 15–30).
- Study design: Open-label.
- Phase II trial
- Purpose: Evaluation of efficacy, optimal dose range, and side effects (especially common and short-term side effects).
- Study population: Moderate number of patients with a specific disease (∼ 10–100).
- Study design: Randomized, Controlled, Anonymized.
- Phase III trial
- Purpose: Final confirmation of safety and evaluation of efficacy against placebo or the current standard of care.
- Study population: Randomized control trial with a large number of patients with a specific disease (∼ 100–1000).
- Study design: Randomized Controlled Trial.
- Phase IV trial
- Purpose: Postmarketing surveillance, comparing real-life efficacy to that described in research studies. Safety studies following approval (especially evaluation of rare and long-term side effects).
- Study population: Large number of patients with a specific disease after drug approval.
- Study design: Open-label.