The Patient Voice: Exploring Treatment Preferences in Participants with Mild Cognitive Concerns to Inform Regulatory Decision Making.

Autor: Mansfield C; Department of Health Preference Assessment, RTI Health Solutions, 3040 East Cornwallis Road, Research Triangle Park, NC, 27709, USA. carolm@rti.org., Bullok K; Eli Lilly and Company, Lilly Corporate Center, Indianapolis, IN, USA., Fuhs JV; Eli Lilly and Company, Lilly Corporate Center, Indianapolis, IN, USA., Tockhorn-Heidenreich A; Eli Lilly and Company, Lilly Corporate Center, Indianapolis, IN, USA., Andrews JS; Eli Lilly and Company, Lilly Corporate Center, Indianapolis, IN, USA.; Takeda Pharmaceuticals, Cambridge, MA, USA., DiBenedetti D; Department of Patient-Centered Outcomes Assessment, RTI Health Solutions, Research Triangle Park, NC, USA., Matthews BR; Eli Lilly and Company, Lilly Corporate Center, Indianapolis, IN, USA., Darling JC; Eli Lilly and Company, Lilly Corporate Center, Indianapolis, IN, USA.; Seagen Inc, Bothell, WA, USA., Sutphin J; Department of Health Preference Assessment, RTI Health Solutions, 3040 East Cornwallis Road, Research Triangle Park, NC, 27709, USA.; Duke Clinical Research Institute, Duke University, Durham, NC, USA., Hauber B; Department of Health Preference Assessment, RTI Health Solutions, 3040 East Cornwallis Road, Research Triangle Park, NC, 27709, USA.; Pfizer, Inc., and the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington School of Pharmacy, Seattle, WA, USA.
Jazyk: angličtina
Zdroj: The patient [Patient] 2022 Sep; Vol. 15 (5), pp. 551-564. Date of Electronic Publication: 2022 Apr 18.
DOI: 10.1007/s40271-022-00576-w
Abstrakt: Objective: We aimed to assess the feasibility of developing a discrete-choice experiment survey to elicit preferences for a treatment to delay cognitive decline among people with a clinical syndrome consistent with early Alzheimer's disease, including the development of self-reported screening criteria to recruit the sample.
Methods: Using input from qualitative interviews, we developed a discrete-choice experiment survey containing a multifaceted beneficial treatment attribute related to slowing cognitive decline for respondents with self-reported cognitive concerns. In two rounds of in-person pretest interviews, we tested and revised the survey text and discrete-choice experiment questions, including examples, language, and levels associated with the Alzheimer's Disease Assessment Scale-Cognitive Subscale, along with a set of de novo self-reported questions for identifying respondents who had neither too mild nor too advanced cognitive decline. Self-reported memory and thinking problems were compared with symptoms from studies of patients with early Alzheimer's disease (e.g., mild cognitive impairment, mild Alzheimer's disease) to determine whether those studies' recruited patients were similar to our anticipated target population. Round 1 pretest interviews resulted in significant simplifications in the survey instrument, revisions to the inclusion and exclusion criteria, and revisions to the screening process. In round 2 of the pretest interviews, the ability of participants to provide consistent responses to the self-reported screening questions was further assessed. In addition, to evaluate participants' ability to understand and independently complete the discrete-choice experiment survey, two interviewers independently evaluated each participant's ability to make trade-offs in the discrete-choice experiment questions and to understand the content of the survey.
Results: Round 1 (15 pretest interviews) identified challenges with the survey instrument related to the complexity of the choice questions. The screening process did not screen out seven respondents with more advanced cognitive decline, as determined qualitatively by the interviewers and by these participants' inability to complete the survey. The survey instrument and screening criteria were revised, and an initial online screener was added to the screening process before round 2 pretests. In round 2 pretests, 12 participants reported cognitive problems similar to the target population for the survey but were judged able to understand and independently complete the discrete-choice experiment survey.
Conclusions: We developed self-reported screening criteria that identified a sample of individuals with memory and thinking concerns who were similar to individuals with clinical symptoms of early Alzheimer's disease and who were able to independently complete a simplified discrete-choice experiment survey. Quantitative patient preference studies provide important information on patients' willingness to trade off treatment benefits/risks. Adapting the technique for patients with cognitive decline requires careful testing and adjustments to survey instruments. This work suggests it is the severity of cognitive impairment, rather than its presence, that determines the ability to complete a simplified discrete-choice experiment survey.
(© 2022. Eli Lilly.)
Databáze: MEDLINE
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