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1.
BMC Cancer ; 23(1): 66, 2023 Jan 19.
Artigo em Inglês | MEDLINE | ID: mdl-36658529

RESUMO

BACKGROUND: Historically, high hepatocellular carcinoma (HCC)-related mortality has been, in part, due to lack of effective therapies; however, several systemic therapies have been recently approved for HCC treatment, including regorafenib and ramucirumab. These two treatments utilize different routes of administration (four daily tablets and biweekly intravenous infusions, respectively) and have different risks of adverse events (AEs). However, we lack data on patient preferences in balancing the route of administration and risk of AEs in patients with HCC. We aimed to determine patient preferences and trade-offs for second-line treatment in patients with HCC.  METHODS: Patients with advanced or metastatic HCC were recruited through their physicians for this study. Patient preferences were assessed by using a modified threshold technique (TT) design in which respondents were asked two direct-elicitation questions before (assuming same safety and efficacy and only varying mode of administration) and after (incorporating the safety profiles of ramucirumab and regorafenib) the TT series on seven risks of clinically relevant AEs. RESULTS: In total, of the 157 patients recruited by their physicians, 150 were eligible and consented to participate. In the first elicitation question (assuming risk and efficacy were equivalent), 61.3% of patients preferred daily tablets. However, 76.7% of patients preferred the biweekly infusion when the safety profiles of the two available second-line therapies were included. The TT analysis confirmed that preferences for oral administration were not strong enough to balance out the risk of AEs that differentiate the two therapies. DISCUSSION: We found that when patients were asked to choose between a daily, oral medication and a biweekly IV medication for HCC, they were more likely to choose a daily, oral medication if efficacy and safety profiles were the same. However, when risks of AEs representing the safety profiles of two currently available second-line treatments were introduced in a second direct-elicitation question, respondents often selected an IV administration with a safety profile similar to ramucirumab, rather than oral tablets with a safety profile similar to regorafenib. Our findings indicate that the risk profile of a second-line treatment for HCC may be more important than the mode of administration to patients.


Assuntos
Carcinoma Hepatocelular , Neoplasias Hepáticas , Humanos , Carcinoma Hepatocelular/patologia , Neoplasias Hepáticas/patologia , Preferência do Paciente , Comprimidos/uso terapêutico
2.
Epilepsia ; 63(2): 451-462, 2022 02.
Artigo em Inglês | MEDLINE | ID: mdl-34921391

RESUMO

OBJECTIVE: This study was undertaken to elicit patients' preferences for attributes characterizing antiseizure medication (ASM) monotherapy options before treatment consultation, and to explore the trade-offs patients consider between treatment efficacy and risks of side effects. Further objectives were to explore how treatment consultation may affect patient preferences, to elicit physicians' preferences in selecting treatment, and to compare patient and physician preferences for treatment. METHODS: This prospective, observational study (EP0076; VOTE) included adults with focal seizures requiring a change in their ASM monotherapy. Patients completed a discrete choice experiment (DCE) survey before and after treatment consultation. Physicians completed a similar survey after the consultation. The DCE comprised 12 choices between two hypothetical treatments defined by seven attributes. The conditional relative importance of each attribute was calculated. RESULTS: Three hundred ten patients (mean [SD] age = 46.8 [18.3] years, 52.3% female) were enrolled from eight European countries, of whom 305 completed the survey before consultation and 273 completed the survey before and after consultation. Overall, this preference study in patients who intended to receive a new ASM monotherapy suggests that patient preferences were ordered as expected, with better outcomes being preferred to worse outcomes; patients preferred a higher chance of seizure freedom, lower risk of developing clinical depression, and fewer severe adverse events; avoiding moderate-to-severe "trouble thinking clearly" was more important than avoiding any other side effect. There were qualitative differences in what patients and physicians considered to be the most important aspects of treatment for patients; compared with patients, physicians had a qualitatively stronger preference for greater chance of seizure freedom and avoiding personality changes. Patients' preference weights were qualitatively similar before and after treatment consultation. SIGNIFICANCE: For patients, seizure freedom and avoiding trouble thinking clearly were the most important treatment attributes. Physicians and patients may differ in the emphasis they place on specific attributes.


Assuntos
Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos , Médicos , Adulto , Comportamento de Escolha , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Preferência do Paciente , Estudos Prospectivos , Convulsões , Inquéritos e Questionários
3.
Value Health ; 25(5): 685-694, 2022 05.
Artigo em Inglês | MEDLINE | ID: mdl-35500943

RESUMO

OBJECTIVES: Discrete choice experiments (DCEs) are increasingly used to elicit preferences for health and healthcare. Although many applications assume preferences are homogenous, there is a growing portfolio of methods to understand both explained (because of observed factors) and unexplained (latent) heterogeneity. Nevertheless, the selection of analytical methods can be challenging and little guidance is available. This study aimed to determine the state of practice in accounting for preference heterogeneity in the analysis of health-related DCEs, including the views and experiences of health preference researchers and an overview of the tools that are commonly used to elicit preferences. METHODS: An online survey was developed and distributed among health preference researchers and nonhealth method experts, and a systematic review of the DCE literature in health was undertaken to explore the analytical methods used and summarize trends. RESULTS: Most respondents (n = 59 of 70, 84%) agreed that accounting for preference heterogeneity provides a richer understanding of the data. Nevertheless, there was disagreement on how to account for heterogeneity; most (n = 60, 85%) stated that more guidance was needed. Notably, the majority (n = 41, 58%) raised concern about the increasing complexity of analytical methods. Of the 342 studies included in the review, half (n = 175, 51%) used a mixed logit with continuous distributions for the parameters, and a third (n = 110, 32%) used a latent class model. CONCLUSIONS: Although there is agreement about the importance of accounting for preference heterogeneity, there are noticeable disagreements and concerns about best practices, resulting in a clear need for further analytical guidance.


Assuntos
Comportamento de Escolha , Opinião Pública , Atenção à Saúde , Humanos , Análise de Classes Latentes , Projetos de Pesquisa
4.
Future Oncol ; 17(34): 4697-4709, 2021 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-34581197

RESUMO

Aims: We evaluated physicians' willingness to trade-off benefits, risks and time to infusion for CAR T-cell therapy for relapsed or refractory diffuse large B-cell lymphoma. Materials & methods: In a discrete-choice experiment survey, 150 US oncologists/hematologists chose between two hypothetical CAR T-cell treatments defined by six attributes. Results: Decreasing time to infusion from 113 to 16 days yielded the greatest change in preference weight (1.91). Physicians were willing to accept a >20% increase in risk of severe cytokine release syndrome and 15% increase in risk of severe neurological events in exchange for an increase in the probability of overall survival at 24 months from 40 to 55%. Conclusion: Physicians value reducing time to infusion and will accept incremental increases in serious adverse event risks to gain survival improvements.


Lay abstract CAR-T therapy is a treatment option for patients with diffuse large B-cell lymphoma that has not responded to at least two other kinds of treatments. CAR-T therapies are manufactured from a patient's white blood cells, modified to attack lymphoma cells. A CAR-T therapy takes time to manufacture after these cells are collected. CAR-T therapies can result in the reduction or disappearance of lymphoma tumors and can increase the chances of survival, but also cause serious side effects for a few patients. One of these is cytokine release syndrome (CRS), in which high levels of inflammation throughout the body may cause fever, heart problems or difficulty breathing. Another is the development of temporary but serious neurological problems such as confusion, seizures and memory problems. To understand how important physicians consider certain features of CAR-T therapies to be when deciding whether to recommend them, we asked physicians to choose between two treatment options resembling CAR-T therapies in a series of questions, with the CAR-T features varying in each question. Their answers indicated whether disappearance of tumors, a patient's chances of survival after 1 and 2 years of treatment, manufacturing time, or the risk of CRS or neurological problems was the most important factor. Physicians most wanted to reduce manufacturing time from 113 to 16 days, but also were willing to accept a >20% increase in risk of severe CRS and a 15% increase in risk of severe neurological events to increase a patient's chance of survival from 40 to 55% at 2 years.


Assuntos
Tomada de Decisão Clínica , Síndrome da Liberação de Citocina/epidemiologia , Imunoterapia Adotiva/métodos , Linfoma Difuso de Grandes Células B/terapia , Recidiva Local de Neoplasia/terapia , Protocolos de Quimioterapia Combinada Antineoplásica/farmacologia , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Ciclofosfamida/farmacologia , Ciclofosfamida/uso terapêutico , Síndrome da Liberação de Citocina/diagnóstico , Síndrome da Liberação de Citocina/imunologia , Doxorrubicina/farmacologia , Doxorrubicina/uso terapêutico , Resistencia a Medicamentos Antineoplásicos , Feminino , Humanos , Imunoterapia Adotiva/efeitos adversos , Imunoterapia Adotiva/estatística & dados numéricos , Linfoma Difuso de Grandes Células B/imunologia , Linfoma Difuso de Grandes Células B/mortalidade , Linfoma Difuso de Grandes Células B/patologia , Masculino , Pessoa de Meia-Idade , Recidiva Local de Neoplasia/imunologia , Recidiva Local de Neoplasia/mortalidade , Médicos/estatística & dados numéricos , Prednisona/farmacologia , Prednisona/uso terapêutico , Receptores de Antígenos Quiméricos/imunologia , Rituximab/farmacologia , Rituximab/uso terapêutico , Índice de Gravidade de Doença , Inquéritos e Questionários/estatística & dados numéricos , Fatores de Tempo , Tempo para o Tratamento/estatística & dados numéricos , Vincristina/farmacologia , Vincristina/uso terapêutico
5.
Value Health ; 23(10): 1373-1383, 2020 10.
Artigo em Inglês | MEDLINE | ID: mdl-33032782

RESUMO

OBJECTIVES: Approximately 800 000 people die globally from colorectal cancer (CRC) every year. Prevention programs promote early detection, but for people with precancerous lesions, tailoring surveillance to include lifestyle-change programs could enhance prevention potential and improve outcomes. METHODS: Those with intermediate or high-risk polyps removed during CRC screening colonoscopy within the Northern Ireland CRC Screening Programme were invited to complete 8 discrete choice questions about tailored surveillance, analyzed using random-parameters logit and a latent class modeling approach. RESULTS: A total of 231 participants (77% male) self-reported comorbid hypertension (53%), high cholesterol (48%), and mean body mass index of 28.7 (overweight). Although 39% of participants were unaware of their CRC risk status, 30.9% indicated they were already making changes to reduce their risk. Although all respondents were significantly risk- and cost-averse, the latent class analysis identified 3 segments (classes): 1. Class 1 (26.8%) significantly favored phone or email support for a lifestyle change, a 17-month testing interval, and noninvasive testing. 2. Class 2 (48.4%) preferred the status quo. 3. Class 3 (24.7%) significantly favored further risk reduction and invasive testing. CONCLUSIONS: This is the first documented preference study focusing on postpolypectomy surveillance offering lifestyle interventions. Although current care is strongly preferred, risk and cost aversion are important for participants. Latent class analysis shows that some respondents are willing to change diet and lifestyle behaviors, reflecting a teachable moment, with opportunities to personalize and optimize surveillance. Significant discordance between perceived and known risk of recurrence and limited recall of risk information provided within current practice suggest necessary improvements to surveillance programs.


Assuntos
Comportamento de Escolha , Neoplasias Colorretais/diagnóstico , Detecção Precoce de Câncer/psicologia , Preferência do Paciente , Pólipos do Colo/cirurgia , Colonoscopia/psicologia , Detecção Precoce de Câncer/métodos , Feminino , Seguimentos , Humanos , Masculino , Pessoa de Meia-Idade , Irlanda do Norte , Preferência do Paciente/psicologia , Preferência do Paciente/estatística & dados numéricos , Comportamento de Redução do Risco
6.
AIDS Behav ; 24(5): 1443-1451, 2020 May.
Artigo em Inglês | MEDLINE | ID: mdl-31696371

RESUMO

As new female-initiated HIV prevention products enter development, it is crucial to incorporate women's preferences to ensure products will be desired, accepted, and used. A discrete-choice experiment was designed to assess the relative importance of six attributes to stated choice of a vaginally delivered HIV prevention product. Sexually active women in South Africa and Zimbabwe aged 18-30 were recruited from two samples: product-experienced women from a randomized trial of four vaginal placebo forms and product-naïve community members. In a tablet-administered survey, 395 women chose between two hypothetical products over eight choice sets. Efficacy was the most important, but there were identifiable preferences among other attributes. Women preferred a product that also prevented pregnancy and caused some wetness (p < 0.001). They disliked a daily-use product (p = 0.002) and insertion by finger (p = 0.002). Although efficacy drove preference, wetness, pregnancy prevention, and dosing regimen were influential to stated choice of a product, and women were willing to trade some level of efficacy to have other more desired attributes.


Assuntos
Infecções por HIV , Adolescente , Adulto , Comportamento de Escolha , Feminino , Infecções por HIV/prevenção & controle , Humanos , Preferência do Paciente , Gravidez , África do Sul , Inquéritos e Questionários , Vagina , Adulto Jovem , Zimbábue
7.
Health Econ ; 29(10): 1307-1315, 2020 10.
Artigo em Inglês | MEDLINE | ID: mdl-32627284

RESUMO

Health researchers design discrete choice experiments (DCEs) to elicit preferences over attributes that define treatments. A DCE can accommodate a limited number of attributes selected by researchers based on numerous factors (e.g., respondent comprehension, cognitive burden, and sample size). For situations where researchers want information about the possible impact of an attribute excluded from the DCE, we propose a method to use a question after the DCE. This follow-up question includes the attributes in the DCE with fixed levels and an additional attribute originally excluded from the DCE. The DCE data can be used to predict the probability that respondents would select one treatment profile over another without the additional attribute. Comparing the prediction to the percentage of the sample who selected each profile when it includes the additional attribute provides information on the potential impact of the additional attribute. We provide an example using data from a DCE on treatments for chronic lymphocytic leukemia. Cost was excluded from the DCE, but the survey included a follow-up question with two fixed treatment profiles, similar to two treatments currently on the market, and a cost for each. Preferences were sensitive to modest changes in cost, highlighting the importance of gathering this information.


Assuntos
Comportamento de Escolha , Preferência do Paciente , Seguimentos , Humanos , Projetos de Pesquisa , Inquéritos e Questionários
8.
Value Health ; 20(1): 126-131, 2017 01.
Artigo em Inglês | MEDLINE | ID: mdl-28212953

RESUMO

BACKGROUND: This study applies attribute nonattendance to medical decision making. We aimed to demonstrate how this type of analysis can be used in medical decision making to assess whether psychiatrists were influenced in their treatment recommendations by information on the genotype of a patient, despite knowing the patient's response to treatment as measured by the Positive and Negative Syndrome Scale. A patient's genetic information may be used to predict their response to therapy; such information, however, becomes redundant, and should not influence decisions, once a clinician knows the patient's actual response to treatment. METHODS: Sixty-seven psychiatrists were presented with patients' pre- or post-treatment scores on the Positive and Negative Syndrome Scale for two hypothetical treatments for schizophrenia. Psychiatrists were also informed whether the patient possessed a genotype linked to hyper-responsiveness to one of the treatments, and were asked to recommend one of these two treatments. Attribute nonattendance assessed whether the information on genotype influenced psychiatrists' treatment recommendations. RESULTS: Years of experience predicted whether psychiatrists were influenced by the genetic information. Psychiatrists with 1 year or less of experience had a 46% probability of considering genetic information, whereas psychiatrists with at least 15 years of experience had a lower probability (7%). CONCLUSIONS: Psychiatrists and other clinicians should be cautious about allowing a patient's genetic information to carry unnecessary weight in their clinical decision making.


Assuntos
Antipsicóticos/uso terapêutico , Genótipo , Padrões de Prática Médica/estatística & dados numéricos , Psiquiatria/estatística & dados numéricos , Esquizofrenia/tratamento farmacológico , Adulto , Atitude do Pessoal de Saúde , Biomarcadores , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Farmacogenética
9.
Prev Med ; 72: 64-9, 2015 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-25575799

RESUMO

OBJECTIVE: To investigate the characteristics of those doing no moderate-vigorous physical activity (MVPA) (0 days/week), some MVPA (1-4 days/week) and sufficient MVPA (≥ 5 days/week) to meet the guidelines in order to effectively develop and target PA interventions to address inequalities in participation. METHOD: A population survey (2010/2011) of 4653 UK adults provided data on PA and socio-demographic characteristics. An ordered logit model investigated the covariates of 1) participating in no PA, 2) participating in some PA, and 3) meeting the PA guidelines. Model predictions were derived for stereotypical subgroups to highlight important policy and practice implications. RESULTS: Mean age of participants was 45 years old (95% CI 44.51, 45.58) and 42% were male. Probability forecasting showed that males older than 55 years of age (probability=0.20; 95% CI 0.11, 0.28), and both males (probability=0.31; 95% CI 0.17, 0.45) and females (probability=0.38; 95% CI 0.27, 0.50) who report poor health are significantly more likely to do no PA. CONCLUSIONS: Understanding the characteristics of those doing no MVPA and some MVPA could help develop population-level interventions targeting those most in need. Findings suggest that interventions are needed to target older adults, particularly males, and those who report poor health.


Assuntos
Exercício Físico , Conhecimentos, Atitudes e Prática em Saúde , Política de Saúde , Nível de Saúde , Adolescente , Adulto , Fatores Etários , Idoso , Exercício Físico/psicologia , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Irlanda do Norte , Comportamento Sedentário , Adulto Jovem
10.
Patient ; 2024 Apr 17.
Artigo em Inglês | MEDLINE | ID: mdl-38632181

RESUMO

The interest in quantifying stated preferences for health and healthcare continues to grow, as does the technology available to support and improve health preference studies. Technological advancements in the last two decades have implications and opportunities for preference researchers designing, administering, analysing, interpreting and applying the results of stated preference surveys. In this paper, we summarise selected technologies and how these can benefit a preference study. We discuss empirical evaluations of the technology in preference research, with examples from health where possible. The technologies reviewed include serious games, virtual reality, eye tracking, innovative formats and decision aids with values clarification components. We conclude with a critical reflection on the benefits and limitations of implementing (often costly) technology alongside stated preference studies.

11.
J Cyst Fibros ; 23(1): 73-79, 2024 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-38042750

RESUMO

BACKGROUND: Patient-centred trial design optimises recruitment and retention, reduces trial failure rates and increases the diversity of trial cohorts. This allows safe and effective treatments to reach clinic more quickly. To achieve this, patients' views must be incorporated into trial design. METHODS: A discrete choice experiment was used to quantify preferences of pwCF for trials features; medicine type, trial location, stipend, washout, drug access on trial completion and trial design. Respondents were presented pairs of hypothetical trial scenarios with different level combinations assigned through experimental design. Respondents were asked to pick their preferred option or decline both. The cross-sectional data were explored using a Random Parameters Logit model. RESULTS: We received 207 eligible responses between Oct2020-Jan2021. The strongest influence on the decision to participate was trial location; pwCF favour participation at their usual clinical centre. Greater travel distances made respondents less willing to participate. Post-trial drug access ranked second. pwCF would rather participate in modulator trials than trials of other drugs. In general, pwCF did not favour a washout period, but were more prepared to washout non-modulators than modulators. Stipend provision was not ranked highly, but higher stipends increased intention to participate. Trial design (placebo vs open-label) had minimal influence on the decision to participate. There are complex interactions between placebos and washouts. CONCLUSIONS: We used quantitative methods to systematically elicit preferences of pwCF for clinical trials' features. We explore the relevance of our findings to trial design and delivery in the current CF trials landscape.


Assuntos
Fibrose Cística , Humanos , Fibrose Cística/tratamento farmacológico , Estudos Transversais , Projetos de Pesquisa
12.
Curr Med Res Opin ; 40(5): 893-904, 2024 05.
Artigo em Inglês | MEDLINE | ID: mdl-38501272

RESUMO

OBJECTIVE: To understand the preferences of healthcare providers (HCPs) in Switzerland for pediatric hexavalent vaccine attributes. METHODS: A discrete-choice experiment included a series of choices between 2 hypothetical pediatric hexavalent vaccines with varying attributes: device type (including preparation time and risk of dosage errors), proportion of infants seroprotected against Haemophilus influenzae type b (Hib) at 11-12 months (pre-booster), packaging size, years on the market, and the thermostability at room temperature. Odds ratios (ORs) and conditional relative attribute importance (CRAI) were calculated using random-parameters logit. RESULTS: HCPs (150 pediatricians and 40 nursing staff) in Switzerland were unlikely to choose a vaccine conferring 50% (OR 0.00; 95% CI 0.00-0.00) or 70% (OR 0.01; 95% CI 0.00-0.01) of infants with Hib seroprotection at 11-12 months (pre-booster) compared with a vaccine conferring 90% seroprotection. The odds of choosing a vaccine available on the market for more than 3 years were nearly 5 times the odds of choosing a vaccine available on the market for less than 1 year (OR 4.76; 95% CI 1.87-7.65). The odds of choosing a vaccine in a prefilled syringe were nearly 3 times the odds of choosing a reconstituted vaccine (OR 2.77; 95% CI 1.39-4.15), and the odds of choosing a vaccine with a smaller package size were nearly 2 times the odds of choosing a vaccine with larger package size (OR 1.89; 95% CI 1.23-2.55). HCPs were equally likely to choose vaccines that can stay at room temperature for 6 versus 3 days (OR 1.07; 95% CI 0.73-1.42). According to CRAI, the most important attribute was Hib seroprotection, followed by years on the market, device type, and packaging size. CONCLUSION: Hib seroprotection at 11-12 months was the most important hexavalent vaccine attribute to HCPs in this study.


Assuntos
Vacinas Anti-Haemophilus , Humanos , Suíça , Masculino , Vacinas Anti-Haemophilus/administração & dosagem , Lactente , Feminino , Pessoal de Saúde/psicologia , Pessoal de Saúde/estatística & dados numéricos , Vacinas Combinadas/administração & dosagem , Adulto , Comportamento de Escolha , Haemophilus influenzae tipo b/imunologia
13.
Orphanet J Rare Dis ; 19(1): 173, 2024 Apr 22.
Artigo em Inglês | MEDLINE | ID: mdl-38649872

RESUMO

BACKGROUND: Genetic testing can offer early diagnosis and subsequent treatment of rare neuromuscular diseases. Options for these tests could be improved by understanding the preferences of patients for the features of different genetic tests, especially features that increase information available to patients. METHODS: We developed an online discrete-choice experiment using key attributes of currently available tests for Pompe disease with six test attributes: number of rare muscle diseases tested for with corresponding probability of diagnosis, treatment availability, time from testing to results, inclusion of secondary findings, necessity of a muscle biopsy, and average time until final diagnosis if the first test is negative. Respondents were presented a choice between two tests with different costs, with respondents randomly assigned to one of two costs. Data were analyzed using random-parameters logit. RESULTS: A total of 600 online respondents, aged 18 to 50 years, were recruited from the U.S. general population and included in the final analysis. Tests that targeted more diseases, required less time from testing to results, included information about unrelated health risks, and were linked to shorter time to the final diagnosis were preferred and associated with diseases with available treatment. Men placed relatively more importance than women on tests for diseases with available treatments. Most of the respondents would be more willing to get a genetic test that might return unrelated health information, with women exhibiting a statistically significant preference. While respondents were sensitive to cost, 30% of the sample assigned to the highest cost was willing to pay $500 for a test that could offer a diagnosis almost 2 years earlier. CONCLUSION: The results highlight the value people place on the information genetic tests can provide about their health, including faster diagnosis of rare, unexplained muscle weakness, but also the value of tests for multiple diseases, diseases without treatments, and incidental findings. An earlier time to diagnosis can provide faster access to treatment and an end to the diagnostic journey, which patients highly prefer.


Assuntos
Testes Genéticos , Doenças Raras , Humanos , Testes Genéticos/métodos , Adulto , Masculino , Feminino , Pessoa de Meia-Idade , Doenças Raras/diagnóstico , Doenças Raras/genética , Adulto Jovem , Adolescente , Doenças Musculares/diagnóstico , Doenças Musculares/genética , Doença de Depósito de Glicogênio Tipo II/diagnóstico , Doença de Depósito de Glicogênio Tipo II/genética , Preferência do Paciente
14.
Front Dement ; 3: 1421556, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-39081616

RESUMO

Introduction: The development of high-quality stated preference (SP) surveys requires a rigorous design process involving engagement with representatives from the target population. However, while transparency in the reporting of the development of SP surveys is encouraged, few studies report on this process and the outcomes. Recommended stages of instrument development includes both steps for stakeholder/end-user engagement and pretesting. Pretesting typically involves interviews, often across multiple waves, with improvements made at each wave; pretesting is therefore resource intensive. The aims of this paper are to report on the outcomes of collaboration with a Lewy body dementia research advisory group during the design phase of a SP survey. We also evaluate an alternative approach to instrument development, necessitated by a resource constrained context. Method: The approach involved conducting the stages of end-user engagement and pretesting together during a public involvement event. A hybrid approach involving a focus group with breakout interviews was employed. Feedback from contributors informed the evolution of the survey instrument. Results: Changes to the survey instrument were organized into four categories: attribute modifications; choice task presentation and understanding; information presentation, clarity and content; and best-best scaling presentation. The hybrid approach facilitated group brainstorming while still allowing the researcher to assess the feasibility of choice tasks in an interview setting. However, greater individual exploration and the opportunity to trial iterative improvements across waves was not feasible with this approach. Discussion: Involvement of the research advisory group resulted in a more person-centered survey design. In a context constrained by time and budget, and with consideration of the capacity and vulnerability of the target population, the approach taken was a feasible and pragmatic mechanism for improving the design of a SP survey.

15.
J Dermatolog Treat ; 35(1): 2304020, 2024 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-38221777

RESUMO

Purpose: Topical treatments for mild-to-moderate (MM) atopic dermatitis (AD) include emollients, corticosteroids, calcineurin inhibitors, a Janus kinase inhibitor, and a phosphodiesterase 4 inhibitor, which differ in multiple ways. This study aimed to quantify the conditional relative importance (CRI) of attributes of topical treatments for MM AD among adult and adolescent patients and caregivers of children with MM AD.Materials and methods: A discrete-choice experiment (DCE) survey was administered to US adults and adolescents with MM AD and caregivers of children with MM AD. Each choice task comprised 2 hypothetical topical treatments characterized by efficacy, adverse events, vehicle, and application frequency. Data were analyzed using a random-parameters logit model to calculate the CRI of each attribute.Results and conclusions: 300 adults, 331 adolescents, and 330 caregivers completed the DCE. Avoiding changes in skin color (CRI 29.0) and time until itch improves (26.6) were most important to adults, followed by time until clear/almost clear skin (17.8). Application frequency (3.0) did not have a statistically significant impact on adults' choices. Adolescents were less concerned about changes in skin color than adults or caregivers; caregivers were less concerned about time until clear/almost clear skin than patients. Physicians should consider age-relevant aspects of preferences in treatment discussions with patients and caregivers.


Assuntos
Dermatite Atópica , Criança , Adulto , Humanos , Adolescente , Dermatite Atópica/tratamento farmacológico , Cuidadores , Administração Tópica , Inibidores de Calcineurina/uso terapêutico , Emolientes/uso terapêutico
16.
J Dermatolog Treat ; 34(1): 2247105, 2023 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-37587862

RESUMO

BACKGROUND: Non-immunosuppressed patients with a history of multiple non-melanoma skin cancers (NMSCs) taking oral nicotinamide supplementation experienced a 23% decrease in annual NMSC risk in a randomized clinical trial. Patient preferences for risks and costs associated with nicotinamide are unknown. OBJECTIVES: To understand how patients prioritize NMSC reduction, infection risk, and cost. METHODS: A sample of adults with history of ≥2 NMSC within the past five years undergoing Mohs procedure completed a discrete-choice experiment comprising two hypothetical treatments-characterized by varying reductions in NMSC incidence, increased severe infection risk, and cost-and no treatment. The data were analyzed with random-parameters logit models. RESULTS: A total of 203 subjects (mean age 71.5 years, 65.5% males) participated. For a 23% annual reduction in NMSC incidence, a 26% [95% CI: 8%-45%] annual increase in severe infection risk and $8 [95% CI: $2-14] monthly cost was acceptable. Outcomes across analyzed subgroups (before vs. during COVID pandemic, site of interview, less vs. more prior NMSCs) were similar. CONCLUSIONS: Patients were unwilling to accept high severe infection risks to obtain the reduction in NMSC incidence observed in a nicotinamide trial, suggesting that routinely recommending nicotinamide may run counter to some patients' preferences.


Assuntos
COVID-19 , Neoplasias Cutâneas , Adulto , Masculino , Humanos , Idoso , Feminino , Modelos Logísticos , Niacinamida/efeitos adversos , Pandemias , Neoplasias Cutâneas/epidemiologia , Neoplasias Cutâneas/prevenção & controle
17.
JRSM Open ; 14(6): 20542704231166621, 2023 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-37325779

RESUMO

Patient-centred trial design and delivery; improves recruitment and retention; increases participant satisfaction; encourages participation by a more representative cohort; and allows researchers to better meet participants' needs. Research in this area mostly focusses on narrow facets of trial participation. We aimed to systematically identify the breadth of patient-centred factors influencing participation and engagement in trials, and collate them into a framework. Through this we hoped to assist researchers to identify factors that could improve patient-centred trial design and delivery. Robust qualitative and mixed methods systematic reviews are becoming increasingly common in health research. The protocol for this review was prospectively registered on PROSPERO, CRD42020184886. We used the SPIDER (Sample, Phenomenon of Interest, Design, Evaluation, Research Type) framework as a standardised systematic search strategy tool. 3 databases were searched as well as references checking, and thematic synthesis was conducted. Screening agreement was performed and code and theme checking were conducted by 2 independent researchers. Data were drawn from 285 peer-reviewed articles. 300 discrete factors were identified, and sorted into 13 themes and subthemes. The full catalogue of factors is included in the Supplementary Material. A summary framework is included in the body of the article. This paper focusses on outlining common ground that themes share, highlighting critical features, and exploring interesting points from the data. Through this, we hope researchers from multiple specialities may be better able to meet patients' needs, protect patients' psychosocial wellbeing, and optimise trial recruitment and retention, with direct positive impact on research time and cost efficiency.

18.
Pediatr Rheumatol Online J ; 21(1): 129, 2023 Oct 21.
Artigo em Inglês | MEDLINE | ID: mdl-37865801

RESUMO

BACKGROUND: This study aimed to elicit and quantify preferences for treatments for juvenile idiopathic arthritis (JIA). METHODS: We conducted a discrete-choice experiment among adolescents with JIA in the United States (US) (n = 197) and United Kingdom (UK) (n = 100) and caregivers of children with JIA in the US (n = 207) and UK (n = 200). In a series of questions, respondents chose between experimentally designed profiles for hypothetical JIA treatments that varied in efficacy (symptom control; time until next flare-up), side effects (stomachache, nausea, and vomiting; headaches), mode and frequency of administration, and the need for combination therapy. Using a random-parameters logit model, we estimated preference weights for these attributes, from which we derived their conditional relative importance. RESULTS: On average, respondents preferred greater symptom control; greater time until the next flare-up; less stomachache, nausea, and vomiting; and fewer headaches. However, adolescents and caregivers in the US were generally indifferent across varying modes and frequencies of administration. UK adolescents and caregivers preferred tablets, syrup, or injections to intravenous infusions. US and UK adolescents were indifferent between treatment with monotherapy or combination therapy; caregivers in the UK preferred treatment with combination therapy to monotherapy. Subgroup analysis showed preference heterogeneity across characteristics including gender, treatment experience, and symptom experience in both adolescents and caregivers. CONCLUSIONS: Improved symptom control, prolonged time to next flare-up, and avoidance of adverse events such as headache, stomachache, nausea, and vomiting are desirable characteristics of treatment regimens for adolescents with JIA and their caregivers.


Assuntos
Artrite Juvenil , Criança , Humanos , Adolescente , Estados Unidos , Artrite Juvenil/tratamento farmacológico , Cuidadores , Cefaleia , Náusea , Vômito
19.
Pharmacoeconomics ; 40(7): 653-661, 2022 07.
Artigo em Inglês | MEDLINE | ID: mdl-35696072

RESUMO

This technical note discusses how dummy and effects coding of categorical respondent characteristics in a class membership probability function should be interpreted by researchers employing a latent class analysis to explore preference heterogeneity in a discrete-choice experiment. Previous work highlighted issues arising from such coding when interpreting an alternative specific constant that represents an opt-out alternative or current situation in a discrete-choice experiment and did not fully address how this coding impacts the interpretation of parameters resulting from the membership probability function in a latent class analysis. Although latent class membership probability could be predicted separately for each respondent or subgroup of respondents, conclusions are often drawn directly from the model estimation using the full sample, which requires correctly interpreting the estimated parameters. In these cases, the misinterpretation that may arise if the problem is ignored could impact the policy conclusions and recommendations drawn based on the discrete-choice experiment results. This note provides an example comparing dummy and effects coding used to model respondent characteristics in the membership probability function in a discrete-choice experiment aimed to explore preferences for the treatment of chronic pain in the USA.


Assuntos
Comportamento de Escolha , Preferência do Paciente , Humanos , Análise de Classes Latentes , Funções Verossimilhança , Inquéritos e Questionários
20.
Expert Rev Vaccines ; 21(10): 1495-1504, 2022 10.
Artigo em Inglês | MEDLINE | ID: mdl-36154795

RESUMO

BACKGROUND: This study aimed to quantify preferences and risk tolerance for a tick-borne encephalitis (TBE) vaccination. RESEARCH DESIGN AND METHODS: A stated-preference survey instrument was administered to international travelers living in the United States to elicit preferences for a no-cost TBE vaccine when planning an international trip, conditional upon four different qualitative levels of endemic TBE risk. RESULTS: The likelihood of choosing the vaccine increased with a destination's level of endemic risk. Most respondents (94%) would choose to receive the vaccine at the highest risk level presented in the survey (i.e. when multiple TBE cases among humans are reported year after year); 6% of the sample would choose not to receive the vaccine at any risk level. Respondents who engage in outdoor activities were twice as likely as the average respondent to choose vaccination rather than opting out of vaccination, and were one-third more likely than the average respondent to choose to receive the vaccine at the lowest risk level. CONCLUSIONS: Respondents were highly interested in a TBE vaccine, assuming no cost, and most were willing to be vaccinated at all qualitative TBE risk levels. Respondents who participated in outdoor activities were more likely than the average respondent to choose the vaccine.


Assuntos
Encefalite Transmitida por Carrapatos , Vacinas Virais , Encefalite Transmitida por Carrapatos/prevenção & controle , Humanos , Tolerância Imunológica , Inquéritos e Questionários , Vacinação
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