Something Is Better Than Nothing: The Value of Active Intervention in Stated Preferences for Treatments to Delay Onset of Alzheimer's Disease Symptoms

DiSantostefano, Rachael L.; Yang, Jui‐Chen; Reed, Shelby D.; Streffer, Johannes; Levitan, Bennett

doi:10.1016/j.jval.2019.03.022

Cited by 15 publications

(22 citation statements)

References 22 publications

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“…In line with former studies, trial participants experienced neuropsychological testing and the lumbar puncture as high burden assessments. [9][10][11][12][13][14][15][16][17][18][19][20][21][22]28 Most trials include of a broad array of psychological tests at its endpoint and may result in frustration and distress for the participant. 23 For trial participants, the confrontation with their cognitive struggles can be overwhelming and may result in unwillingness to participate.…”

Section: Discussionmentioning

confidence: 99%

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Experiences of and recommendations on clinical trial design in Alzheimer’s disease from the participant’s point of view

Ottenhoff

Vijverberg

Visser

et al. 2022

Preprint

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Introduction. In the context of the development of pharmaceutical interventions, expectations and experiences of participants are essential. Their insights may be particularly helpful to address the challenges of recruiting and retaining participants for Alzheimer’s disease (AD) clinical trials. We examined clinical trial participants’ experiences to optimize trial design in Alzheimer’s disease (AD). Method. In this mixed-methods study, we included adults who participated in sponsor-initiated AD trials at Brain Research Center, a clinical trial organization in the Netherlands. Participants (N = 71, age 69 ± 6.5, 54%F, 19 cognitively normal (CN), 19 Mild Cognitive Impairment (MCI) and 33 AD dementia) first completed an online survey. Next, a subsample (N = 12; 8 = CN, 4 = MCI) participated in focus groups to gain in-depth insight into their views on optimizing trial design. Audio recordings from focus group interviews were transcribed verbatim and analysed by thematic content analysis by two independent researchers. Results. To benefit future generations was most frequently (34%) reported as a motive for enrolment. The lumbar puncture (40%) and memory assessments (15%) were most often experienced as being of high burden. Structurally receiving individualized test results and smaller chance to get placebo, increased willingness to participate. Two main themes emerged from the focus groups: “trials design”, such as follow-up visit(s) after participating, and “trial center”, including the relevance of a professional and empathic staff. Conclusion. Relevant factors include expectation management and careful planning of high burden assessments, provision of individual feedback and prioritizing professionalism and empathy throughout conduct of the trial. Our findings provide insight into participants’ priorities to increase willingness to participate and can be used to optimize trial success.

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Section: Discussionmentioning

confidence: 99%

“…Finally, this study did not use a hypothetical clinical trial, as is customary in focus group research, but rather took the participants real life experience in actual clinical trials as starting point. [6][7][8][9][10][11][12][13][14]…”

mentioning

confidence: 99%

Experiences of and recommendations on clinical trial design in Alzheimer’s disease from the participant’s point of view

Ottenhoff

Vijverberg

Visser

et al. 2022

Preprint

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“…If a disease-modifying treatment becomes available, the preferences of the disease community may change in accordance with the drug's efficacy and side effect profile. Previous research has also demonstrated that this current pro-treatment mindset can confound results with some participants illogically selecting treatments with no benefit and high side effect risk 43 . Additional investigations should be performed to further understand the HD community's views on preventive medications over time, taking into consideration the community's pro-treatment bias https://doi.org/10.1017/cts.2022.372 and other treatment attributes that are outside the scope of this study including the method of treatment administration, the durability of the treatment, and even higher side effect risk.…”

Section: Discussionmentioning

confidence: 99%

Preventive drugs for Huntington’s disease: A choice-based conjoint survey of patient preferences

Parrish

Hanson‐Kahn

Srinivasan

et al. 2022

J. Clin. Trans. Sci.

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“…Studies on preferences for preventive and risk-reducing treatments have been conducted, for example, in individuals at risk of rheumatoid arthritis, 26,27 women with the BRCA1/2 gene, 28 and individuals at risk of Alzheimer's disease. 29 The results of the DCE survey should be interpreted in the context of limitations related to the survey instrument and sample. The survey presents hypothetical scenarios to respondents.…”

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Parent Preferences for Delaying Insulin Dependence in Children at Risk of Stage III Type 1 Diabetes

DiSantostefano

Sutphin

Hedrick

et al. 2020

Diabetes Technology & Therapeutics

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Background: Autoantibody screening in type 1 diabetes (T1D) may reduce the chances of potentially lifethreatening diabetic ketoacidosis (DKA) at diagnosis by allowing individuals at risk of progression to more actively monitor for and/or manage progression to insulin dependence. We investigated parents' preferences for treatments to delay the onset of insulin dependence in children who are at high risk of developing Stage III T1D. Methods: A web-based survey (n = 1501) was administered to a stratified sample of parents (children <18 years) in the United States from an online panel. Parents were told to hypothetically assume that their youngest child would become insulin dependent within 6 months or 2 years and were offered a series of choices between no treatment and two hypothetical treatments that would delay insulin dependence. Random-parameters logit analysis and maximum acceptable risks were used to evaluate the relative importance of treatment benefits and risks. Results: Most parents chose at least one active treatment (2% always chose monitoring only). For parents of children without T1D (n = 901), delaying insulin dependence and reducing the risk of long-term health complications and serious infection were the most important treatment attributes. In addition, parents of children with T1D (n = 600) also valued reducing the risk of hospitalizations due to DKA. Conclusions: When told to assume their child would develop Stage III T1D, most parents considered active treatments to delay progression. For medicines under development to delay insulin dependence in T1D, the preferences expressed in this survey provide guidance on acceptable benefit-risk trade-offs.

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Something Is Better Than Nothing: The Value of Active Intervention in Stated Preferences for Treatments to Delay Onset of Alzheimer's Disease Symptoms

Cited by 15 publications

References 22 publications

Experiences of and recommendations on clinical trial design in Alzheimer’s disease from the participant’s point of view

Experiences of and recommendations on clinical trial design in Alzheimer’s disease from the participant’s point of view

Preventive drugs for Huntington’s disease: A choice-based conjoint survey of patient preferences

Parent Preferences for Delaying Insulin Dependence in Children at Risk of Stage III Type 1 Diabetes

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