Uncertainty About the Benefits of New Cancer Drugs and Patients' Treatment Preferences: a Randomized Controlled Trial

Many new cancer drugs are approved based on uncertain benefits. Without knowledge of this information, patients may overestimate the benefits of new cancer drugs, resulting in decisions that are not reflective of their individual treatment preferences. A previous study found that learning about uncertainties with a new cancer drug's benefits (such as the drug's small magnitude of effect or limited trial population) reduced the proportion of people who were willing to take the drug. It is unclear whether these findings are generalizable to patients with cancer, who may have different treatment preferences, and may weigh uncertainty about the benefits of new cancer drugs differently from the general public.

In this study, US adults with a current or former cancer diagnosis will be recruited to evaluate the effect of learning about uncertainties with the benefits of a new cancer drug on their hypothetical decisions to take the drug and their perceptions of the drug's effectiveness. Patients will be randomized to 1 of 5 interventions: (1) no uncertainty information (control); or plain-language statements about uncertainties with (2) an unvalidated surrogate endpoint (unknown effect of the drug on survival or quality of life); (3) limited study population (the drug was not studied in patients of similar race or ethnicity); (4) small magnitude of effect (it is unknown whether patients would notice an improvement with the drug), or (5) a combination of all three uncertainties.