When planning trials, what should the sponsor ensure regarding safety and efficacy data?

• A. Safety data from nonclinical studies only are sufficient.
• B. Efficacy data should be the only driver for human exposure.
• C. The sponsor should ensure that sufficient safety and efficacy data from nonclinical studies and/or clinical trials are available to support human exposure by the route, at the dosages, for the duration, and in the trial population to be studied.
• D. The sponsor should not rely on prior data.

Answer: (C)

When planning a trial, the key idea is to confirm there is enough safety and efficacy information to justify exposing humans to the investigational product. This means using data from nonclinical studies (such as animal and in vitro work) and any available clinical data, and ensuring those data support the planned route of administration, the doses, the duration of exposure, and the characteristics of the trial population. This evidence base underpins a responsible risk–benefit assessment, informs starting doses and dose-escalation schemes, guides who can participate, and shapes safety monitoring and stopping rules. Relying on safety data alone or efficacy data alone would leave important gaps, and disregarding prior data would ignore useful information. Therefore, the sponsor should ensure that sufficient safety and efficacy data from nonclinical studies and/or clinical trials are available to support human exposure by the route, at the dosages, for the duration, and in the trial population to be studied.