RCTs

Phase I trial: Conducted in healthy subjects when expected toxicity is minor.

Phase II: small sample size. First time patients are exposed to drug

Phase III: aims to give a definitive assessment of intervention against a control. Evaluate toxicity and efficacy.

Double blind trial-neither patient nor researcher knows if in placebo or treatment

Single blind trial- sometimes necessary depending on drug. Patient or assessing physician does not know which treatment is being taken

Randomisation is why phase III trials are seen as gold standard

Equipoise- we don’t know if the new treatment is better than standard treatment

outcomes= endpoints

Primary endpoint- trial designed to have a single primary question

Secondary endpoint- there may be secondary questions of interest eg symptom of relief

Hazard ratio of 1 means no different

Clinical endpoints-Reflect survival or symptomatic status of patient

Surrogate endpoints- Associated with clinical endpoints but do not directly reflect survival or clinical status of the subject

Everyone included even if didn’t cohere to it

Classical superiority trials- is this treatment better than placebo or standard tirals

New treatments may not be more efficacious but fewer side effects treatment regime that people adhere more to.