Preferences for speed of access versus certainty of the survival benefit of new cancer drugs: a discrete choice experiment
Menée aux Etats-Unis à partir d'une enquête réalisée auprès d'un échantillon de 870 personnes âgées représentatives de la population nationale et ayant un ami proche ou un membre de la famille atteint ou ayant été atteint d'un cancer (âge : 55 ans ou plus ; 53 % d'hommes), cette étude examine leur volonté d'accéder rapidement à un anticancéreux (médicament autorisé par la FDA via la voie d'approbation accélérée) malgré l'incertitude concernant le bénéfice de ce médicament en termes de survie
Résumé en anglais
Background : The extent to which patients with cancer are willing to accept uncertainty about the clinical benefit of new cancer drugs in exchange for faster access is not known. This study aims to examine preferences for access versus certainty, and to understand factors that influence these preferences.
Methods : A US nationally representative sample of older adults were recruited via Cint, an online platform for survey research, to take part in an online discrete choice experiment. To be eligible, respondents had to self-report some experience with cancer—ie, they themselves, a close friend or a family member, previously or currently diagnosed with cancer. In the experiment, respondents chose between two cancer drugs, considering five attributes: functional status, life expectancy, certainty of the survival benefit of a new drug, effect of the drug on a surrogate endpoint, and delay in US Food and Drug Administration (FDA) approval time. The first primary outcome was the relative importance of certainty of survival benefit and wait time to respondents. The second primary outcome was willingness to wait for greater certainty of survival benefit, including subgroup analysis by cancer experience, age, education status, race or ethnicity and income. Secondary outcomes were changes in sensitivity to certainty and wait time, depending on the drug's effect on a surrogate endpoint, respondents' functional status, and life expectancy. The study plan was registered with ClinicalTrials.gov, NCT05936632.
Findings : Between July 7 and July 20, 2023, 998 eligible respondents completed the survey. 870 respondents (461 [53%] male, 406 [47%] female, and three [<1%] other) were included in the final analysis. Respondents showed strong preferences for high certainty of survival benefit (coefficient 2·61, 95% CI 2·23 to 2·99), and strong preferences against a 1-year delay in FDA approval time (coefficient –1·04, 95% CI –1·31 to –0·77). Given very low certainty a drug would provide survival benefit (no evidence linking a surrogate endpoint to overall survival), respondents were willing to wait up to 21·68 months (95% CI 17·61 to 25·74) for high certainty (strong evidence) of survival benefit. A drug's effect on a surrogate endpoint had no significant impact on drug choices (coefficient 0·02, 95% CI –0·21 to 0·25). Older respondents (aged
