Dataset Information

Social Preferences for Orphan Drugs: A Discrete Choice Experiment Among the French General Population.

ABSTRACT: Objectives: While several authors have suggested using a multi-criteria approach for orphan drug assessment and proposed lists of determinants of orphan drug value, studies on social preferences regarding these determinants remain limited. The current study aimed to identify preferences of the French general population regarding attributes characterizing the value of orphan drugs in a discrete choice experiment. Methods: The list of attributes was formed based on a literature search and was refined through expert interviews, a focus group, and a pilot study. The final list included nine attributes: disease-associated disability, disease-associated mortality, number of patients, availability of alternative treatments, treatment impact on disease disability, treatment impact on mortality, treatment safety, uncertainty around therapeutic effect, and annual treatment cost per patient. Members of the General Public were presented with 12 choice sets containing two drug profiles described according to the attributes and an option to fund neither of these treatments. The questionnaire was disseminated online. A conditional logit model with random effects was used to estimate the weight of each attribute. Results: A total of 958 persons participated in the study (48.7% male, mean age: 47.5 years). All attributes except for disease-associated disability had a statistically significant influence on the choices made by participants. The attribute with the highest weight was treatment impact on mortality (p < 0.0001), followed by uncertainty around therapeutic effect (p < 0.0001). The direction of results was generally consistent with intuition: patients preferred a drug with a larger impact on mortality, a larger impact on disability, with mild or no adverse events, with less uncertainty. Although patients appeared to prefer drugs with a lower budget impact, the relationship between patient preferences and costs was more complex. Conclusions: Preferences of the general public between orphan drugs are mostly driven by the impact on mortality and the degree of certainty regarding the available evidence.

SUBMITTER: Toumi M

PROVIDER: S-EPMC7379418 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Social Preferences for Orphan Drugs: A Discrete Choice Experiment Among the French General Population.

Toumi Mondher M Millier Aurélie A Cristeau Olivier O Thokagevistk-Desroziers Katia K Dorey Julie J Aballéa Samuel S

Frontiers in medicine 20200717

<b>Objectives:</b> While several authors have suggested using a multi-criteria approach for orphan drug assessment and proposed lists of determinants of orphan drug value, studies on social preferences regarding these determinants remain limited. The current study aimed to identify preferences of the French general population regarding attributes characterizing the value of orphan drugs in a discrete choice experiment. <b>Methods:</b> The list of attributes was formed based on a literature searc ...[more]

PMID: 32766260

Similar Datasets

Project description:BackgroundCervical cancer screening is effective in reducing mortality due to uterine cervical cancer (UCC). However, inequalities in participation in UCC screening exist, especially according to age and social status. Considering the current situation in France regarding the ongoing organized UCC screening campaign, we aimed to assess general practitioners' (GPs) and gynaecologists' preferences for actions designed to reduce screening inequalities.MethodsFrench physicians' preferences to UCC screening modalities was assessed using a discrete choice experiment. A national cross-sectional questionnaire was sent between September and October 2014 to 500 randomly selected physicians, and numerically to all targeted physicians working in the French region Midi-Pyrénées. Practitioners were offered 11 binary choices of organized screening scenarios in order to reduce inequalities in UCC screening participation. Each scenario was based on five attributes corresponding to five ways to enhance participation in UCC screening while reducing screening inequalities.ResultsAmong the 123 respondents included, practitioners voted for additional interventions targeting non-screened women overall (p < 0.05), including centralized invitations sent from a central authority and involving the mentioned attending physician, or providing attending physicians with the lists of unscreened women among their patients. However, they rejected the specific targeting of women over 50 years old (p < 0.01) or living in deprived areas (p < 0.05). Only GPs were in favour of allowing nurses to perform Pap smears, but both GPs and gynaecologists rejected self-collected oncogenic papillomavirus testing.ConclusionsFrench practitioners tended to value the traditional principle of universalism. As well as rejecting self-collected oncogenic papillomavirus testing, their reluctance to support the principle of proportionate universalism relying on additional interventions addressing differences in socioeconomic status needs further evaluation. As these two concepts have already been recommended as secondary development leads for the French national organized screening campaign currently being implemented, the adherence of practitioners and the adaptation of these concepts are necessary conditions for reducing inequalities in health care.

Project description:BACKGROUND:Reaching an acceptable participation rate in screening programs is challenging. With the objective of supporting the Belarus government to implement mammography screening as a single intervention, we analyse the main determinants of breast cancer screening participation. METHODS:We developed a discrete choice experiment using a mixed research approach, comprising a literature review, in-depth interviews with key informants (n = 23), "think aloud" pilots (n = 10) and quantitative measurement of stated preferences for a representative sample of Belarus women (n = 428, 89% response rate). The choice data were analysed using a latent class logit model with four classes selected based on statistical (consistent Akaike information criterion) and interpretational considerations. RESULTS:Women in the sample were representative of all six geographic regions, mainly urban (81%), and high-education (31%) characteristics. Preferences of women in all four classes were primarily influenced by the perceived reliability of the test (sensitivity and screening method) and costs. Travel and waiting time were important components in the decision for 34% of women. Most women in Belarus preferred mammography screening to the existing clinical breast examination (90%). However, if the national screening program is restricted in capacity, this proportion of women will drop to 55%. Women in all four classes preferred combined screening (mammography with clinical breast examination) to single mammography. While this preference was stronger if lower test sensitivity was assumed, 28% of women consistently gave more importance to combined screening than to test sensitivity. CONCLUSION:Women in Belarus were favourable to mammography screening. Population should be informed that there are no benefits of combined screening compared to single mammography. The results of this study are directly relevant to policy makers and help them targeting the screening population.

Project description:BackgroundPatient preferences for treatment outcomes are important to guide decision-making in clinical practice, but little is known about the preferences of patients with metastatic hormone-sensitive prostate cancer (mHSPC).ObjectiveTo evaluate patient preferences regarding the attributed benefits and harms of systemic treatments for mHSPC and preference heterogeneity between individuals and specific subgroups.Design setting and participantsWe conducted an online discrete choice experiment (DCE) preference survey among 77 patients with metastatic prostate cancer (mPC) and 311 men from the general population in Switzerland between November 2021 and August 2022.Outcome measurements and statistical analysisWe evaluated preferences and preference heterogeneity related to survival benefits and treatment-related adverse effects using mixed multinomial logit models and estimated the maximum survival time participants were willing to trade to avert specific adverse effects. We further assessed characteristics associated with different preference patterns via subgroup and latent class analyses.Results and limitationsPatients with mPC showed an overall stronger preference for survival benefits in comparison to men from the general population (p = 0.004), with substantial preference heterogeneity between individuals within the two samples (both p < 0.001). There was no evidence of differences in preferences for men aged 45-65 yr versus ≥65 yr, patients with mPC in different disease stages or with different adverse effect experiences, or general population participants with and without experiences with cancer. Latent class analyses suggested the presence of two groups strongly preferring either survival or the absence of adverse effects, with no specific characteristic clearly associated with belonging to either group. Potential biases due to participant selection, cognitive burden, and hypothetical choice scenarios may limit the study results.ConclusionsGiven the relevant heterogeneity in participant preferences regarding the benefits and harms of treatment for mHSPC, patient preferences should be explicitly discussed during decision-making in clinical practice and reflected in clinical practice guidelines and regulatory assessment regarding treatment for mHSPC.Patient summaryWe examined the preferences (values and perceptions) of patients and men from the general population regarding the benefits and harms of treatment for metastatic prostate cancer. There were large differences between men in how they balanced the expected survival benefits and potential adverse effects. While some men strongly valued survival, others more strongly valued the absence of adverse effects. Therefore, it is important to discuss patient preferences in clinical practice.

Project description:OBJECTIVES:It is desirable that public preferences are established and incorporated in emergency healthcare reforms. The aim of this study was to investigate preferences for local versus centralised provision of all emergency medical services (EMS) and explore what individuals think are important considerations for EMS delivery. DESIGN:A discrete choice experiment was conducted. The attributes used in the choice scenarios were: travel time to the hospital, waiting time to be seen, length of stay in the hospital, risks of dying, readmission and opportunity for outpatient care after emergency treatment at a local hospital. SETTING:North East England. PARTICIPANTS:Participants were a randomly sampled general population, aged 16 years or above recruited from Healthwatch Northumberland network database of lay members and from clinical contact with Northumbria Healthcare National Health Service Foundation Trust via Patient Experience Team. PRIMARY AND SECONDARY OUTCOME MEASURES:Analysis used logistic regression modelling techniques to determine the preference of each attribute. Marginal rates of substitution between attributes were estimated to understand the trade-offs individuals were willing to make. RESULTS:Responses were obtained from 148 people (62 completed a web and 86 a postal version). Respondents preferred shorter travel time to hospital, shorter waiting time, fewer number of days in hospital, low risk of death, low risk of readmission and outpatient follow-up care in their local hospital. However, individuals were willing to trade off increased travel time and waiting time for high-quality centralised care. Individuals were willing to travel 9 min more for a 1-day reduction in length of stay in the hospital, 38 min for a 1% reduction in risk of death and 112 min for having outpatient follow-up care at their local hospital. CONCLUSIONS:People value centralised EMS if it provides higher quality care and are willing to travel further and wait longer.

Project description:Choosing hypodontia treatment requires young people and parents to consider a large amount of information, identify what is important to them, and make trade-offs between options. This study aimed to examine young people and parents' preferences for hypodontia treatment using discrete choice experiment (DCE). This was a cross-sectional survey of young people (12-16 y) with hypodontia of any severity, at any stage of treatment, and their parents. Participants were recruited from NHS Hospitals in England and Wales. A bespoke DCE questionnaire was developed to measure preferences for 6 attributes of hypodontia treatment (waiting time, treatment time, problems during treatment, discomfort during treatment, bite, appearance). The questionnaire was completed 1) online by young people and parents, individually or together, and 2) by child-parent dyads under observation. Preferences were analyzed using regression models. In total, 204 participants (122 young people, 56 parents, 26 dyads) completed the online questionnaire and 15 child-parent dyads completed the questionnaire under observation. The most important attribute in hypodontia treatment was improvement in appearance, but significant heterogeneity was found in preferences. Four distinct groups of participants were found: group 1 (39%): severe discomfort and problems were most important; group 2 (31%): most concerned about improvement in appearance of teeth and improvement in bite; group 3 (22%): appearance 3 times more important than any other attribute; and group 4 (9%): preferences difficult to interpret. There was variation in how child-parent dyads approached decision-making, with some negotiating joint preferences, while for others, one individual dominated. Making trade-offs in DCE tasks helped some people think about treatment and identify their preferences. Appearance is an important outcome from hypodontia treatment, but preferences vary and potential risks and functional outcome are also important to some people. There is a notable level of uncertainty in decision-making, which suggests further shared decision support would be valuable.

Dataset Information

Social Preferences for Orphan Drugs: A Discrete Choice Experiment Among the French General Population.

Publications

Social Preferences for Orphan Drugs: A Discrete Choice Experiment Among the French General Population.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets