Which description best captures the essential focus when selecting a dose range in development planning?

• A. To identify a dose range and schedule with safe and effective results and an acceptable risk-benefit.
• B. To analyze only pharmacokinetic data.
• C. To minimize the number of participants.
• D. To determine marketing potential.

Answer: (A)

When planning dose range, the focus is on finding doses and a dosing schedule that can show meaningful efficacy while keeping safety within an acceptable risk-benefit balance. This means looking at how different doses impact both therapeutic effect and potential adverse effects, guided by safety margins, pharmacokinetic/pharmacodynamic data, and early human signals, so the chosen range supports informed decisions for future development and regulatory review.

Relying only on pharmacokinetic data misses whether those exposures translate into real clinical benefit and acceptable safety. While designing efficiently is important, the primary aim isn’t simply reducing study size, and marketing potential doesn’t drive dose selection in trials.