8 research outputs found
Can the General Public Be a Proxy for an “At-Risk” Group in a Patient Preference Study? A Disease Prevention Example in Rheumatoid Arthritis
Background: When selecting samples for patient preference studies, it may be difficult or impractical to recruit participants who are eligible for a particular treatment decision. However, a general public sample may not be an appropriate proxy. Objective: This study compares preferences for rheumatoid arthritis (RA) preventive treatments between members of the general public and first-degree relatives (FDRs) of confirmed RA patients to assess whether a sample of the general public can be used as a proxy for FDRs. Methods: Participants were asked to imagine they were experiencing arthralgia and had screening tests indicating a 60% chance of developing RA within 2 yrs. Using a discrete choice experiment, participants were offered a series of choices between no treatment and 2 unlabeled hypothetical treatments to reduce the risk of RA. To assess data quality, time to complete survey sections and comprehension questions were assessed. A random parameter logit model was used to obtain attribute-level estimates, which were used to calculate relative importance, maximum acceptable risk (MAR), and market shares of hypothetical preventive treatments. Results: The FDR sample (n = 298) spent more time completing the survey and performed better on comprehension questions compared with the general public sample (n = 982). The relative importance ranking was similar between the general public and FDR participant samples; however, other relative preference measures involving weights including MARs and market share differed between groups, with FDRs having numerically higher MARs. Conclusion: In the context of RA prevention, the general public (average risk) may be a reasonable proxy for a more at-risk sample (FDRs) for overall relative importance ranking but not weights. The rationale for a proxy sample should be clearly justified. Participants from the general public were compared to first-degree relatives on their preferences for rheumatoid arthritis (RA) preventive treatments using a discrete choice experiment. Preferences were similar between groups in terms of the most important and least important attributes of preventive treatments, with effectiveness being the most important attribute. However, relative weights differed. Attention to the survey and predicted market shares of hypothetical RA preventive treatments differed between the general public and first-degree relatives. The general public may be a reasonable proxy for an at-risk group for patient preferences ranks but not weights in the disease prevention context; however, care should be taken in sample selection for patient preference studies when choosing nonpatients
Design, Conduct and Use of Patient Preference Studies in the Medical Product Life Cycle
Objectives: To investigate stakeholder perspectives on how patient preference studies
(PPS) should be designed and conducted to allow for inclusion of patient preferences in
decision-making along the medical product life cycle (MPLC), and how patient preferences
can be used in such decision-making.
Methods: Two literature reviews and semi-structured interviews (n = 143) with healthcare
stakeholders in Europe and the US were conducted; results of these informed the design
of focus group guides. Eight focus groups were conducted with European patients,
industry representatives and regulators, and with US regulators and European/Canadian
health technology assessment (HTA) representatives. Focus groups were analyzed
thematically using NVivo.
Results: Stakeholder perspectives on how PPS should be designed and conducted
were as follows: 1) study design should be informed by the research questions and patient
population; 2) preferred treatment attributes and levels, as well as trade-offs among
attributes and levels should be investigated; 3) the patient sample and method should
match the MPLC phase; 4) different stakeholders should collaborate; and 5) results from
PPS should be shared with relevant stakeholders. The value of patient preferences in
decision-making was found to increase with the level of patient preference sensitivity of
decisions on medical products. Stakeholders mentioned that patient preferences are hardly
used in current decision-making. Potential applications for patient preferences across
industry, regulatory and HTA processes were identified. Four applications seemed most
promising for systematic integration of patient preferences: 1) benefit-risk assessment
by industry and regulators at the marketing-authorization phase; 2) assessment of major contribution to patient care by European regulators; 3) cost-effectiveness analysis; and 4)
multi criteria decision analysis in HTA.
Conclusions: The value of patient preferences for decision-making depends on the level
of collaboration across stakeholders; the match between the research question, MPLC
phase, sample, and preference method used in PPS; and the sen
Factors and situations affecting the value of patient preference studies: semi-structured interviews in Europe and the US.
Objectives: Patient preference information (PPI) is gaining recognition among the
pharmaceutical industry, regulatory authorities, and health technology assessment (HTA)
bodies/payers for use in assessments and decision-making along the medical product
lifecycle (MPLC). This study aimed to identify factors and situations tha
Exploring preferences of at-risk individuals for preventive treatments for rheumatoid arthritis
Objective: Some immunomodulatory drugs have been shown to delay the onset of, or lower the risk of developing, rheumatoid arthritis (RA), if given to individuals at risk. Several trials are ongoing in this area; however, little evidence is currently available about the views of those at risk of RA regarding preventive treatment. Method: Three focus groups and three interviews explored factors that are relevant to first degree relatives (FDRs) of RA patients and members of the general public when considering taking preventive treatment for RA. The semi-structured qualitative interview prompts explored participant responses to hypothetical attributes of preventive RA medicines. Transcripts of focus group/interview proceedings were inductively coded and analysed using a framework approach. Results: Twenty-one individuals (five FDRs, 16 members of the general public) took part in the study. Ten broad themes were identified describing factors that participants felt would influence their decisions about whether to take preventive treatment if they were at increased risk of RA. These related either directly to features of the specific treatment or to other factors, including personal characteristics, attitude towards taking medication, and an individual’s actual risk of developing RA. Conclusion: This research highlights the importance of non-treatment factors in the decision-making process around preventive treatments, and will inform recruitment to clinical trials as well as information to support shared decision making by those considering preventive treatment. Studies of treatment preferences in individuals with a confirmed high risk of RA would further inform clinical trial design
Exploring preferences of at-risk individuals for preventive treatments for rheumatoid arthritis
Some immunomodulatory drugs have been shown to delay the onset of, or lower the risk of developing, rheumatoid arthritis (RA), if given to individuals at risk. Several trials are ongoing in this area; however, little evidence is currently available about the views of those at risk of RA regarding preventive treatment. Three focus groups and three interviews explored factors that are relevant to first degree relatives (FDRs) of RA patients and members of the general public when considering taking preventive treatment for RA. The semi-structured qualitative interview prompts explored participant responses to hypothetical attributes of preventive RA medicines. Transcripts of focus group/interview proceedings were inductively coded and analysed using a framework approach. Twenty-one individuals (five FDRs, 16 members of the general public) took part in the study. Ten broad themes were identified describing factors that participants felt would influence their decisions about whether to take preventive treatment if they were at increased risk of RA. These related either directly to features of the specific treatment or to other factors, including personal characteristics, attitude towards taking medication, and an individual’s actual risk of developing RA. This research highlights the importance of non-treatment factors in the decision-making process around preventive treatments, and will inform recruitment to clinical trials as well as information to support shared decision making by those considering preventive treatment. Studies of treatment preferences in individuals with a confirmed high risk of RA would further inform clinical trial design
Exploring preferences of at-risk individuals for preventive treatments for rheumatoid arthritis
Some immunomodulatory drugs have been shown to delay the onset of, or lower the risk of developing, rheumatoid arthritis (RA), if given to individuals at risk. Several trials are ongoing in this area; however, little evidence is currently available about the views of those at risk of RA regarding preventive treatment. Three focus groups and three interviews explored factors that are relevant to first degree relatives (FDRs) of RA patients and members of the general public when considering taking preventive treatment for RA. The semi-structured qualitative interview prompts explored participant responses to hypothetical attributes of preventive RA medicines. Transcripts of focus group/interview proceedings were inductively coded and analysed using a framework approach. Twenty-one individuals (five FDRs, 16 members of the general public) took part in the study. Ten broad themes were identified describing factors that participants felt would influence their decisions about whether to take preventive treatment if they were at increased risk of RA. These related either directly to features of the specific treatment or to other factors, including personal characteristics, attitude towards taking medication, and an individual’s actual risk of developing RA. This research highlights the importance of non-treatment factors in the decision-making process around preventive treatments, and will inform recruitment to clinical trials as well as information to support shared decision making by those considering preventive treatment. Studies of treatment preferences in individuals with a confirmed high risk of RA would further inform clinical trial design
Exploring preferences of at-risk individuals for preventive treatments for rheumatoid arthritis
Some immunomodulatory drugs have been shown to delay the onset of, or lower the risk of developing, rheumatoid arthritis (RA), if given to individuals at risk. Several trials are ongoing in this area; however, little evidence is currently available about the views of those at risk of RA regarding preventive treatment. Three focus groups and three interviews explored factors that are relevant to first degree relatives (FDRs) of RA patients and members of the general public when considering taking preventive treatment for RA. The semi-structured qualitative interview prompts explored participant responses to hypothetical attributes of preventive RA medicines. Transcripts of focus group/interview proceedings were inductively coded and analysed using a framework approach. Twenty-one individuals (five FDRs, 16 members of the general public) took part in the study. Ten broad themes were identified describing factors that participants felt would influence their decisions about whether to take preventive treatment if they were at increased risk of RA. These related either directly to features of the specific treatment or to other factors, including personal characteristics, attitude towards taking medication, and an individual’s actual risk of developing RA. This research highlights the importance of non-treatment factors in the decision-making process around preventive treatments, and will inform recruitment to clinical trials as well as information to support shared decision making by those considering preventive treatment. Studies of treatment preferences in individuals with a confirmed high risk of RA would further inform clinical trial design.</p
sj-docx-1-mdm-10.1177_0272989X231218265 – Supplemental material for Can the General Public Be a Proxy for an “At-Risk” Group in a Patient Preference Study? A Disease Prevention Example in Rheumatoid Arthritis
Supplemental material, sj-docx-1-mdm-10.1177_0272989X231218265 for Can the General Public Be a Proxy for an “At-Risk” Group in a Patient Preference Study? A Disease Prevention Example in Rheumatoid Arthritis by R. L. DiSantostefano, G. Simons, M. Englbrecht, Jennifer H. Humphreys, Ian N. Bruce, K. Schölin Bywall, C. Radawski, K. Raza, M. Falahee and J. Veldwijk in Medical Decision Making</p