# Quantitative patient preference studies set the benefit-risk bar before phase 3

Source: https://onco.cc/ideas/idea-moon-preference-studies-before-phase3/  
OnCo record `idea-moon-preference-studies-before-phase3` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Before a big trial starts, ask hundreds of patients how much extra survival they would trade for a given side-effect, so the trial is designed to test something patients would actually want.

## Summary

Discrete choice experiments and best-worst scaling quantify how patients trade benefits against harms and burdens. Regulators (FDA CDRH, EMA) have accepted such data in medical devices and some drugs. The proposal is that for each new indication, a preference study defines the minimum clinically important benefit and acceptable toxicity profile, which is then written into the phase 3 design and the regulatory review.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- Hypothesis: Preference-informed designs produce approvals with higher patient-relevant value and fewer post-approval label restrictions, and shift phase 3 endpoints towards those with the highest patient weights.
- Rationale: Minimum clinically important differences are currently set by convention and statistics; patients have measurable and heterogeneous preferences that should anchor them.
- Proposed test: Run preference studies in three tumour types with upcoming phase 3 programmes; compare the preference-derived benefit thresholds with those in current protocols and with eventual approval decisions.
- Maturity: early-clinical
- Actor: regulator

## Sources

- Bottleneck evidence (Patients lack understanding, navigation and agency): Basch et al., Overall survival results of a trial assessing patient-reported outcomes for symptom monitoring during routine cancer treatment (JAMA 2017): https://doi.org/10.1001/jama.2017.7156

## Connected records

- bottlenecks: [Patients lack understanding, navigation and agency](https://onco.cc/bottlenecks/b-patient-voice/), [Trial design, endpoints and cost](https://onco.cc/bottlenecks/b-trial-design/)

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