Eurordis - Rare Disease Europe

When testing the pharmacological efficacy of a new treatment, the criteria of eligibility may exclude a large part of the patient population from the trial?

In an intention to treat perspective, all patients may be included without considering any criteria of eligibility.

The comparison between treatments may result in greater difference for the secondary endpoints than for the main endpoint.

A statistical difference shown between two treatments may be of poor interest clinically.

According to the medical situation, subjective endpoints may be more relevant than objective endpoints.

Randomised trials are mainly used in paediatric research because children, as minors, are not able to choose their treatment.

Some criteria of exclusion are aimed at protecting individuals.

In a controlled trial, the investigational product is compared to another.

A randomised trial is a gold standard of clinical research because it avoids the risk of bias in the assignment of the treatment.

In both parallel-group and crossover design, the participants are evaluated after a fixed period of treatment.

Testing the efficacy of a new treatment may be conducted in a limited group of participants, without a control group.

“Controlled trial” means “a trail supervised by an independent monitoring committee”.