TY - JOUR
T1 - Experiences of and recommendations on clinical trial design in Alzheimer’s disease from the participant’s point of view
T2 - a mixed-methods study in two clinical trial centers in the Netherlands
AU - Ottenhoff, Lois
AU - Vijverberg, Everard G. B.
AU - Visser, Leonie N. C.
AU - Verijp, Merike
AU - Prins, Niels D.
AU - Van der Flier, Wiesje M.
AU - Sikkes, Sietske A. M.
N1 - Funding Information: Research programs of Wiesje van der Flier have been funded by ZonMW, NWO, EU-FP7, EU-JPND, Alzheimer Nederland, CardioVascular Onderzoek Nederland, Health~Holland, Topsector Life Sciences & Health, stichting Dioraphte, Gieskes-Strijbis fonds, stichting Equilibrio, Biogen MA Inc, Boehringer Ingelheim, Life-MI, AVID, Roche BV, Fujifilm, and Combinostics. WF holds the Pasman chair. WF is recipient of ABOARD, which is a public-private partnership receiving funding from ZonMW (#73305095007) and Health~Holland, Topsector Life Sciences & Health (PPP-allowance; #LSHM20106). WF has performed contract research for Biogen MA Inc and Boehringer Ingelheim. WF has been an invited speaker at Boehringer Ingelheim, Biogen MA Inc, Danone, Eisai, and WebMD Neurology (Medscape). WF is consultant to Oxford Health Policy Forum CIC, Roche, and Biogen MA Inc. WF participated in an advisory board of Biogen MA Inc. All funding is paid to her institution. WF was associate editor of Alzheimer, Research & Therapy in 2020/2021. WF is associate editor at Brain. Funding Information: This work was co-funded by the PPP Allowance made available by Health-Holland, Top Sector Life Sciences & Health to stimulate public-private partnerships and Brain Research Center (grant no. LSHM19051). Funding Information: L. Ottenhoff is part-time employee of Amsterdam UMC and part time of Brain Research Center, The Netherlands. Her work is funded by Health~Holland, Topsector Life Sciences & Health (PPP-allowance; LSHM19051) Funding Information: Research of Alzheimer Center Amsterdam is part of the neurodegeneration research program of Amsterdam Neuroscience. Alzheimer Center Amsterdam is supported by Stichting Alzheimer Nederland and Stichting VUmc fonds. The chair of Wiesje van der Flier is supported by the Pasman stichting. LO is appointed on OTAPA, a collaboration project which is co-funded by the PPP Allowance made available by Health-Holland, Top Sector Life Sciences & Health to stimulate public-private partnerships and Brain Research Center (grant no. LSHM19051). Funding Information: S.A.M. Sikkes received funding from Health~Holland Topsector Life Sciences & Health (LSHM19051 & LSHM20084). She provided consultancy services for Biogen, Boehringer, Toyama, and Lundbeck. SAMS is the developer of the Amsterdam IADL Questionnaire and received license fees from Green Valley, VtV Therapeutics, Alzheon, Vivoryon, Roche, Janssen, Axon Neuroscience, Genentech, and Medavante. All funds were paid to her institution. Publisher Copyright: © 2023, The Author(s).
PY - 2023/12/1
Y1 - 2023/12/1
N2 - 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. Diagnostic group differences were investigated using chi-square tests or one-way ANOVAs. Next, a subsample (N = 12; 8 = CN, 4 = MCI) participated in focus groups to gain in-depth insight into their opinions on optimizing trial design from a participants’ point of view. Audio recordings from focus group interviews were transcribed verbatim and analyzed by thematic content analysis by two independent researchers. Results: Most reported motives for enrolment included “to benefit future generations” (89%), followed by “for science” (66%) and “better monitoring” (42%). Frequent suggestions for increasing willingness to participate included a smaller chance to receive placebo (n = 38, 54%), shorter travel times (n = 27, 38%), and sharing individual results of different assessments (n = 57, 80%), as well as receiving trial results (n = 52, 73). Highest visual analogue burden scores (0–100) were found for the lumbar puncture (M = 47.2, SD = 38.2) and cognitive assessments (M = 27.2, SD = 25.7). Results did not differ between diagnostic groups, nor between patient and caregiver participants (all p-values>.05). Two additional themes emerged from the focus groups: “trial 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.
AB - 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. Diagnostic group differences were investigated using chi-square tests or one-way ANOVAs. Next, a subsample (N = 12; 8 = CN, 4 = MCI) participated in focus groups to gain in-depth insight into their opinions on optimizing trial design from a participants’ point of view. Audio recordings from focus group interviews were transcribed verbatim and analyzed by thematic content analysis by two independent researchers. Results: Most reported motives for enrolment included “to benefit future generations” (89%), followed by “for science” (66%) and “better monitoring” (42%). Frequent suggestions for increasing willingness to participate included a smaller chance to receive placebo (n = 38, 54%), shorter travel times (n = 27, 38%), and sharing individual results of different assessments (n = 57, 80%), as well as receiving trial results (n = 52, 73). Highest visual analogue burden scores (0–100) were found for the lumbar puncture (M = 47.2, SD = 38.2) and cognitive assessments (M = 27.2, SD = 25.7). Results did not differ between diagnostic groups, nor between patient and caregiver participants (all p-values>.05). Two additional themes emerged from the focus groups: “trial 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.
KW - Alzheimer’s disease
KW - Alzheimer’s disease clinical trials
KW - Engagement in research trials
KW - Mild cognitive impairment
KW - Patient preferences
KW - Patients
KW - Qualitative research
KW - Quantitative research
UR - http://www.scopus.com/inward/record.url?scp=85151795449&partnerID=8YFLogxK
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85151795449&origin=inward
UR - https://www.ncbi.nlm.nih.gov/pubmed/37016435
U2 - https://doi.org/10.1186/s13195-023-01190-0
DO - https://doi.org/10.1186/s13195-023-01190-0
M3 - Article
C2 - 37016435
SN - 1758-9193
VL - 15
SP - 72
JO - Alzheimer's Research and Therapy
JF - Alzheimer's Research and Therapy
IS - 1
M1 - 72
ER -