Women's preferences for HIV prevention service delivery in pharmacies during pregnancy in Western Kenya: a discrete choice experiment.

INTRODUCTION: Pharmacy-delivered HIV prevention services might create more options for pregnant women to use HIV prevention tools earlier and more consistently during pregnancy. We quantified preferences for attributes of potential HIV prevention services among women of childbearing age in Western Kenya. METHODS: From June to November 2023, we administered a face-to-face discrete choice experiment survey to women aged 15-44 in Kenya's Homa Bay, Kisumu and Siaya counties. The survey evaluated preferences for HIV prevention services, described by seven attributes: service location, travel time, type of HIV test, sexually transmitted infection (STI) testing, partner HIV testing, pre-exposure prophylaxis (PrEP) and service fee. Participants answered a series of 12-choice questions. Each question asked them to select one of two service options or no services-an opt-out option. We used hierarchical Bayesian modelling levels to estimate each attribute level's coefficient and understand how attributes influenced service choice. RESULTS: Overall, 599 participants completed the survey, among whom the median age was 23 years (IQR: 18-27); 33% were married, 20% had a job and worked regularly, and 52% had been pregnant before. Participants, on average, strongly preferred having any HIV prevention service option over none (opt-out preference weight: -5.84 [95% CI: -5.97, -5.72]). The most important attributes were the availability of PrEP (relative importance 27.04% [95% CI: 25.98%, 28.11%]), followed by STI testing (relative importance 20.26% [95% CI: 19.52%, 21.01%]) and partner HIV testing (relative importance: 16.35% [95% CI: 15.79%, 16.90%]). While, on average, participants preferred obtaining services at the clinic more than pharmacies, women prioritized the availability of PrEP, STI testing and partner HIV testing more than the location or cost. CONCLUSIONS: These findings suggest the importance of providing comprehensive HIV prevention services and ensuring PrEP, STI testing and partner HIV testing are available. If pharmacies can offer these services, women are likely to access those services at pharmacies even if they prefer clinics.

A latent class analysis of factors influencing preferences for infant respiratory syncytial virus (RSV) preventives among pregnant people in the United States.

A maternal vaccine and long-acting monoclonal antibody (mAb) were recently approved to protect infants against respiratory syncytial virus (RSV). We identified subgroups of pregnant people with different preferences for RSV preventives and respondent characteristics associated with subgroup membership. An online survey, including a discrete choice experiment (DCE), was conducted among US pregnant people. RSV preventive attributes included effectiveness, duration of protection during RSV season, injection recipient/timing, preventive type (vaccine or mAb), and type of visit required to receive injection. In DCE choice tasks, pregnant people selected between two hypothetical preventive profiles with varying attribute-levels and a no-preventive option. Logistic regression, including latent class analysis (LCA), was used to analyze the data. Of 992 pregnant people (mean age: 30.0 years), 60.3% were expecting their second/later birth. LCA identified three preference subgroups: 'Effectiveness' (preventive choice mostly driven by increases in effectiveness; 51.4% class membership probability), 'Season' (preventive choice mostly driven by improvement in duration of protection during the RSV season; 39.2% class membership probability), and 'No Preventive' (frequently chose no-preventive option; 9.4% class membership probability). 'Effectiveness' and 'Season' preferred maternal vaccine over mAb; mAb was preferred by 'No Preventive.' Perceiving RSV as serious for infants, higher health literacy, and lower household income were associated with 'Effectiveness.' Perceiving RSV as serious for pregnant people was associated with 'Season.' Perceiving RSV to not be serious for pregnant people and not being employed were associated with 'No Preventive.' Subgroups of pregnant people vary in preferences for RSV preventives. Most pregnant people preferred a maternal vaccine, although some may be more willing to accept alternative preventive options.

Patient Acceptability and Preferences for Solid Oral Dosage Form Drug Product Attributes: A Scoping Review.

Background: There is no consistent framework for patient-centric drug product design, despite the common understanding that drug product acceptability and preferences influence adherence and, therefore, drug product effectiveness. The aim of this review was to assess current understanding of patient acceptability and preferences for solid oral dosage form (SODF) drug product attributes, and the potential impact of these attributes on patient behaviors and outcomes. Patients and Methods: A scoping review was conducted. Embase, Ovid MEDLINE®, and PubMed® were searched for full-text articles published between January 2013 and May 2023. Following screening and assessment against predefined inclusion criteria, data were analyzed thematically. Results: Nineteen studies were included. Four overarching domains of drug product attributes were identified and summarized in a framework: appearance, swallowability, palatability, and handling. Each domain was informed by specific drug product attributes: texture, form, size, shape, color, marking, taste, mouthfeel, and smell. The most frequently studied domains were swallowability and appearance, while the most studied attributes were size, shape, and texture. Smell, marking, and mouthfeel were the least studied attributes. Texture intersected all domains, while form, shape, and size intersected appearance, swallowability, and handling. Swallowability and size appeared to be the key domain and attribute, respectively, to consider when designing drug products. Few studies explored the impact of drug product attributes on behaviors and outcomes. Conclusion: While existing studies of drug product attributes have focused on appearance and swallowability, this review highlighted the importance of two less well-understood domains-palatability and handling-in understanding patients' acceptability and preferences for SODF drug products. The framework provides a tool to facilitate patient-centric design of drug products, organizing and categorizing physical drug product attributes into four overarching domains (appearance, swallowability, palatability, and handling), encouraging researchers to comprehensively assess the impact of drug product attributes on patient acceptability, preferences, and outcomes.

Medicines come in a variety of types and forms. These include tablets and capsules. Factors, such as the size and shape of tablets, can affect how people take medicines. However, patients are rarely involved in designing the medicines that they take. In this study, researchers summarized 19 studies published between 2013 and 2023. They wanted to understand how different factors, like size and shape, affect patients' preferences, ability, and willingness to take medicines. Researchers focused on the "physical" aspects of medicines and found 4 common themes: 1) what they look like (appearance), 2) how easy they are to swallow (swallowability), 3) how they taste and feel in the mouth (palatability), and 4) how easy they are to handle (handling). Eight factors were also found: color, markings, shape, size, smell, taste, texture, and how a medicine feels in the mouth (mouthfeel). Most studies focused on what medicines look like and how easy they are to swallow. The factors that researchers mostly looked at were the size, shape, and texture of medicines. The design of medicines can impact patients of different ages, though there may be specific needs for certain groups of patients, including children, older adults, and people with certain diseases. Patient input should become a part of future medicines design to ensure their acceptability.

Healthcare Providers' and Pregnant People's Preferences for a Preventive to Protect Infants from Serious Illness Due to Respiratory Syncytial Virus.

We assessed the impact of respiratory syncytial virus (RSV) preventive characteristics on the intentions of pregnant people and healthcare providers (HCPs) to protect infants with a maternal vaccine or monoclonal antibodies (mAbs). Pregnant people and HCPs who treated pregnant people and/or infants were recruited via convenience sample from a general research panel to complete a cross-sectional, web-based survey, including a discrete choice experiment (DCE) wherein respondents chose between hypothetical RSV preventive profiles varying on five attributes (effectiveness, preventive type [maternal vaccine vs. mAb], injection recipient/timing, type of medical visit required to receive the injection, and duration of protection during RSV season) and a no-preventive option. A best-worst scaling (BWS) exercise was included to explore the impact of additional attributes on preventive preferences. Data were collected between October and November 2022. Attribute-level preference weights and relative importance (RI) were estimated. Overall, 992 pregnant people and 310 HCPs participated. A preventive (vs. none) was chosen 89.2% (pregnant people) and 96.0% (HCPs) of the time (DCE). Effectiveness was most important to preventive choice for pregnant people (RI = 48.0%) and HCPs (RI = 41.7%); all else equal, pregnant people (RI = 5.5%) and HCPs (RI = 7.2%) preferred the maternal vaccine over mAbs, although preventive type had limited influence on choice. Longer protection, protection starting at birth or the beginning of RSV season, and use for both pre-term and full-term babies were ranked highest in importance (BWS). Pregnant people and HCPs strongly preferred a preventive to protect infants against RSV (vs. none), underscoring the need to incorporate RSV preventives into routine care.

Priorities for contraceptive method and service delivery attributes among adolescent girls and young women in Kenya: a qualitative study.

Introduction: Despite increasing global commitment to meeting the family planning needs of adolescent girls and young women (AGYW), there is limited research on how they prioritize contraceptive method and service delivery characteristics. In this qualitative study, we examine the specific elements that drive the contraceptive choices of Kenyan AGYW, and apply our findings to the development of attributes and levels for a discrete choice experiment (DCE). Methods: Our four-stage approach included data collection, data reduction, removing inappropriate attributes, and optimizing wording. Between June-October 2021, we conducted in-depth interviews with 30 sexually-active 15-24 year-old AGYW in Kisumu county, Kenya who were non-pregnant and desired to delay pregnancy. Interviews focused on priorities for contraceptive attributes, how AGYW make trade-offs between among these attributes, and the influences of preferences on contraceptive choice. Translated transcripts were qualitatively coded and analyzed with a constant comparative approach to identify key concepts. We developed and iteratively revised a list of attributes and levels, and pre-tested draft DCE choice tasks using cognitive interviews with an additional 15 AGYW to optimize comprehension and relevance. Results: In-depth interview participants' median age was 18, 70% were current students, and 93% had a primary sexual partner. AGYW named a variety of priorities and preferences related to choosing and accessing contraceptive methods, which we distilled into six key themes: side effects; effectiveness; user control; privacy; source of services; and cost. Bleeding pattern was top of mind for participants; amenorrhea was generally considered an intolerable side effect. Many participants felt more strongly about privacy than effectiveness, though some prioritized duration of use and minimizing chance of pregnancy above other contraceptive characteristics. Most AGYW preferred a clinic setting for access, as they desired contraceptive counseling from a provider, but pharmacies were considered preferable for reasons of privacy. We selected, refined, and pre-tested 7 DCE attributes, each with 2-4 levels. Conclusions: Identifying AGYW preferences for contraceptive method and service delivery characteristics is essential to developing innovative strategies to meet their unique SRH needs. DCE methods may provide valuable quantitative perspectives to guide and tailor contraceptive counseling and service delivery interventions for AGYW who want to use contraception.

Key informant views on potential acceptability and feasibility of long-acting antiretroviral treatment for HIV in Kenya.

BACKGROUND: In 2020, 14% of diagnosed persons living with HIV (PLWH) in Kenya were not taking antiretroviral therapy (ART), and 19% of those on ART had unsuppressed viral loads. Long-acting antiretroviral therapy (LA-ART) may increase viral suppression by promoting ART uptake and adherence. We conducted key informant (KI) interviews with HIV experts in Kenya to identify product and delivery attributes related to the acceptability and feasibility of providing LA-ART to PLWH in Kenya. METHODS: Interviews were conducted via Zoom on potential LA-ART options including intra-muscular (IM) injections, subcutaneous (SC) injections, implants, and LA oral pills. KI were asked to discuss the products they were most and least excited about, as well as barriers and facilitators to LA-ART roll-out. In addition, they were asked about potential delivery locations for LA-ART products such as homes, pharmacies, and clinics. Interviews were recorded and transcribed, and data were analyzed using a combination of inductive and deductive coding. RESULTS: Twelve KI (5 women, 7 men) participated between December 2021 and February 2022. Overall, participants reported that LA-ART would be acceptable and preferable to PLWH because of fatigue with daily oral pills. They viewed IM injections and LA oral pills as the most exciting options to ease pill burden and improve adherence. KI felt that populations who could benefit most were adolescents in boarding schools and stigmatized populations such as sex workers. SC injections and implants were less favored, as they would require new training initiatives for patients or healthcare workers on administration. In addition, SC injections would require refrigeration and needle disposal after use. Some KI thought patients, especially men, might worry that IM injections and implants would impact fertility, given their role in family planning. Pharmacies were perceived by most KI as suboptimal delivery locations; however, given ongoing work in Kenya to include pharmacies in antiretroviral delivery, they recommended asking patients their views. CONCLUSION: There is interest and support for LA-ART in Kenya, especially IM injections and LA oral pills. Identifying patient preferences for modes and delivery locations and addressing misconceptions about specific products as they become available will be important before wide-scale implementation.

Patient preferences in genetic newborn screening for rare diseases: study protocol.

INTRODUCTION: Rare diseases (RDs) collectively impact over 30 million people in Europe. Most individual conditions have a low prevalence which has resulted in a lack of research and expertise in this field, especially regarding genetic newborn screening (gNBS). There is increasing recognition of the importance of incorporating patients' needs and general public perspectives into the shared decision-making process regarding gNBS. This study is part of the Innovative Medicine Initiative project Screen4Care which aims at shortening the diagnostic journey for RDs by accelerating diagnosis for patients living with RDs through gNBS and the use of digital technologies, such as artificial intelligence and machine learning. Our objective will be to assess expecting parent's perspectives, attitudes and preferences regarding gNBS for RDs in Italy and Germany. METHODS AND ANALYSIS: A mixed method approach will assess perspectives, attitudes and preferences of (1) expecting parents seeking genetic consultation and (2) 'healthy' expecting parents from the general population in two countries (Germany and Italy). Focus groups and interviews using the nominal group technique and ranking exercises will be performed (qualitative phase). The results will inform the treatment of attributes to be assessed via a survey and a discrete choice experiment (DCE). The total recruitment sample will be 2084 participants (approximatively 1000 participants in each country for the online survey). A combination of thematic qualitative and logit-based quantitative approaches will be used to analyse the results of the study. ETHICS AND DISSEMINATION: This study has been approved by the Erlangen University Ethics Committee (22-246_1-B), the Freiburg University Ethics Committee (23-1005 S1-AV) and clinical centres in Italy (University of FerraraCE: 357/2023/Oss/AOUFe and Hospedale Bambino Gesu: No.2997 of 2 November 2023, Prot. No. _902) and approved for data storage and handling at the Uppsala University (2022-05806-01). The dissemination of the results will be ensured via scientific journal publication (open access).

Understanding Clinician Preferences for Treatment Attributes in Oncology: A Discrete Choice Experiment of Oncologists' and Urologists' Preferences for First-Line Treatment of Locally Advanced/Unresectable Metastatic Urothelial Carcinoma in Five European Countries.

INTRODUCTION: Prior discrete choice experiments (DCE) in oncology found that, on average, clinicians rank survival as the most important treatment attribute. We investigate heterogeneity in clinician preferences within the context of first-line treatment for advanced urothelial carcinoma in Spain, France, Italy, Germany, and the UK. METHODS: The online DCE included 12 treatment choice tasks, each comparing two hypothetical therapy profiles defined by treatment attributes: grade 3/4 treatment-related adverse events (TRAEs), induction and maintenance administration schedules, progression-free survival, and overall survival (OS). We used a random parameters logit model to estimate attribute relative importance (RI) (0-100%) and generate preference shares for four treatment profiles. Results were stratified by country. Preference heterogeneity was evaluated by latent class analysis. RESULTS: In August and September 2022, 498 clinicians (343 oncologists and 155 urologists) completed the DCE. OS had the strongest influence on clinicians' preferences [RI = 62%; range, 51.6% (Germany) to 63.7% (Spain)] followed by frequency of grade 3/4 TRAEs (RI = 27%). Among treatment profiles, the chemotherapy plus immune checkpoint inhibitor maintenance therapy profile had the largest preference share [51%; range, 38% (Italy) to 56% (UK)]. Four latent classes of clinicians were identified (N = 469), with different treatment profile preferences: survival class (30.1%), trade-off class (22.4%), no strong preference class (40.9%), and aggressive treatment class (6.6%). OS was not the most important attribute for 30.0% of clinicians. CONCLUSION: While average sample results were consistent with those of prior DCEs, this study found heterogeneity in clinician preferences within and across countries, highlighting the diversity in clinician decision making in oncology.

Using Patient Preferences in Health Technology Assessment: Evaluating Quality-Adjusted Survival Equivalents (QASE) for the Quantification of Non-health Benefits.

Interest in using patient preference (PP) data alongside traditional economic models in health technology assessment (HTA) is growing, including using PP data to quantify non-health benefits. However, this is limited by a lack of standardised methods. In this article, we describe a method for using discrete choice experiment (DCE) data to estimate the value of non-health benefits in terms of quality-adjusted survival equivalence (QASE), which is consistent with the concept of value prevalent among HTA agencies. We describe how PP data can be used to estimate QASE, assess the ability to test the face-validity of QASE estimates of changes in mode of administration calculated from five published DCE oncology studies and review the methodological and normative considerations associated with using QASE to support HTA. We conclude that QASE may have some methodological advantages over alternative methods, but this requires DCEs to estimate second-order effects between length and quality of life. In addition, empirical work has yet to be undertaken to substantiate this advantage and demonstrate the validity of QASE. Further work is also required to align QASE with normative objectives of HTA agencies. Estimating QASE would also have implications for the conduct of DCEs, including standardising and defining more clear attribute definitions.

From Qualitative Research to Quantitative Preference Elicitation: An Example in Invasive Meningococcal Disease.

BACKGROUND: Qualitative research is fundamental for designing discrete choice experiments (DCEs) but is often underreported in the preference literature. We developed a DCE to elicit preferences for vaccination against invasive meningococcal disease (IMD) among adolescents and young people (AYP) and parents and legal guardians (PLG) in the United States. This article reports the targeted literature review and qualitative interviews that informed the DCE design and demonstrates how to apply the recent reporting guidelines for qualitative developmental work in preference studies. METHODS: This study included two parts: a targeted literature review and qualitative interviews. The Medline and Embase databases were searched for quantitative and qualitative studies on IMD and immunization. The results of the targeted literature review informed a qualitative interview guide. Sixty-minute, online, semi-structured interviews with AYP and PLG were used to identify themes related to willingness to be vaccinated against IMD. Participants were recruited through a third-party recruiter's database and commercial online panels. Interviews included vignettes about IMD and vaccinations and three thresholding exercises examining the effect of incidence rate, disability rate, and fatality rate on vaccination preferences. Participant responses related to the themes were counted. RESULTS: The targeted literature review identified 31 concepts that were synthesized into six topics for the qualitative interviews. Twenty AYP aged 16-23 years and 20 PLG of adolescents aged 11-17 years were interviewed. Four themes related to willingness to be vaccinated emerged: attitudes towards vaccination, knowledge and information, perception of IMD, and vaccine attributes. Most participants were concerned about IMD (AYP 60%; PLG 85%) and had positive views of vaccination (AYP 80%; PLG 60%). Ninety percent of AYP and 75% of PLG always chose vaccination over no vaccination, independent of IMD incidence rate, disability rate, or fatality rate. CONCLUSION: Willingness to be vaccinated against IMD was affected by vaccine attributes but largely insensitive to IMD incidence and severity. This article provides an example of how to apply the recent reporting guidelines for qualitative developmental work in preference studies, with 21 out of 22 items in the guidelines being considered.

Adult, adolescent, and caregiver preferences for attributes of topical treatments for mild-to-moderate atopic dermatitis: a discrete-choice experiment.

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.

Current Practices and Challenges When Submitting Patient Experience Data for Regulatory Decisions by the US Food and Drug Administration: An Industry Survey.

OBJECTIVES: To understand industry practices and challenges when submitting patient experience data (PED) for regulatory decisions by the US Food and Drug Administration (FDA). METHODS: A two-part online survey related to collection, submission, and use of PED by FDA in regulatory decision-making (part 1) and a best-worst exercise for prioritizing potential PED initiatives (part 2) was completed by industry and contract research organization (CRO) members with ≥ 2 years of recent experience with patient-reported outcome (PRO), natural history study (NHS), or patient preference (PP) data; and direct experience with FDA filings including PED. RESULTS: A total of 50 eligible respondents (84% industry) completed part 1 of the survey, among which 46 completed part 2. Respondents mostly had PRO (86%) and PP (50%) experience. All indicated that FDA meetings should have a standing agenda item to discuss PED. Most (78%) reported meetings should occur before pivotal trials. A common challenge was justifying inclusion without knowing if and how data will be used. Most agreed that FDA and industry should co-develop the PED table in the FDA clinical review (74%), and the table should report reason(s) for not using PED (96%) in regulatory decision-making. Most important efforts to advance PED use in decision-making were a dedicated meeting pathway and expanded FDA guidance (51% each). CONCLUSIONS: FDA has policy targets expanding PED use, but challenges remain regarding pathways for PED submission and transparency in regulatory decision-making. Alignment on the use of existing meeting opportunities to discuss PED, co-development of the PED table, and expanded guidance are encouraged.

Treatment preferences of adults and adolescents with alopecia areata: A discrete choice experiment.

PRODUCTS with janus kinase (JAK) inhibition have been shown to promote hair regrowth in patients with alopecia areata (AA). To guide drug-approval and treatment decisions, it is important to understand patients' willingness to accept the potential risks of JAK inhibition in exchange for potential benefits. We quantified the treatment preferences of adult (≥18 years) and adolescent patients (12-17 years) with AA in the US and Europe to determine the trade-offs they are willing to make between benefits and risks. Preferences for oral AA treatment attributes were elicited using a discrete choice experiment consisting of 12 tasks in which patients chose between two hypothetical treatment alternatives and no treatment. Benefits included the probability of 80%-100% scalp hair regrowth (Severity of Alopecia Tool score ≤ 20) and achieving moderate-to-normal eyebrow and eyelash hair. Treatment-related risks included 3-year probabilities of serious infection, cancer, and blood clots. Preference estimates were used to calculate the maximum level of each risk that patients were willing to accept for increases in treatment benefits. The most important attribute to both adults (n = 201) and adolescents (n = 120) was a 50% probability of achieving hair regrowth on most or all the scalp; however, adolescents placed greater relative importance on this attribute than did adults. Adults were averse to the risks of serious infection, cancer, and blood clots, whereas adolescents were averse to the risk of cancer. For a 20% increase in the probability of 80%-100% scalp hair regrowth, adults were willing to accept a mean (95% confidence interval) 3-year risk of serious infection, cancer, and blood clots of 7.4% (5.5-9.3), 2.5% (1.9-3.1), and 9.3% (6.4-12.2). Adolescents were willing to accept a 3-year risk of cancer of 3.3% (2.4-4.2). Patients with AA in the US and Europe are willing to accept substantial risks to obtain an effective treatment.

Priority Outcomes in Sickle Cell Disease Treatment: Co-Creation and Implementation of a Preference Exercise With Patients and Caregivers to Inform Drug Development.

Involving patients as co-leaders and co-creators in research is key to reflecting the patient's voice in decision-making. However, co-creation of patient-centered data to inform decisions is rare, especially in early drug development where patient input is critical to prioritizing patient-relevant outcomes and endpoints for use in clinical trials. Despite the industry's growing commitment to patient centricity, most patients are excluded from sharing their expertise in research; more inclusive methods of engaging patients as research partners are needed. We describe a collaboration between a pharmaceutical company and a patient organization in co-leading and co-creating a program to understand priorities of patients and caregivers for treatment features and outcomes in sickle cell disease to inform endpoint selection in clinical development. The results of this program will be used as a basis for continued interaction between patients and the sponsor and to inform ongoing clinical development and evidence-generation activities. This case study demonstrates an approach to meaningful collaborations between patient organizations and pharmaceutical companies aimed at including the patient's voice early in the medical product lifecycle.

Maximum Acceptable Risk Estimation Based on a Discrete Choice Experiment and a Probabilistic Threshold Technique.

OBJECTIVE: We aimed to empirically compare maximum acceptable risk results estimated using both a discrete choice experiment (DCE) and a probabilistic threshold technique (PTT). METHODS: Members of the UK general public (n = 982) completed an online survey including a DCE and a PTT (in random order) measuring their preferences for preventative treatment for rheumatoid arthritis. For the DCE, a Bayesian D-efficient design consisting of four blocks of 15 choice tasks was constructed including six attributes with varying levels. The PTT used identical risk and benefit attributes. For the DCE, a panel mixed-logit model was conducted, both mean and individual estimates were used to calculate maximum acceptable risk. For the PTT, interval regression was used to calculate maximum acceptable risk. Perceived complexity of the choice tasks and preference heterogeneity were investigated for both methods. RESULTS: Maximum acceptable risk confidence intervals of both methods overlapped for serious infection and serious side effects but not for mild side effects (maximum acceptable risk was 32.7 percent-points lower in the PTT). Although, both DCE and PTT tasks overall were considered easy or very easy to understand and answer, significantly more respondents rated the DCE choice tasks as easier to understand compared with those who rated the PTT as easier (7-percentage point difference; p < 0.05). CONCLUSIONS: Maximum acceptable risk estimate confidence intervals based on a DCE and a PTT overlapped for two out of the three included risk attributes. More respondents rated the DCE as easier to understand. This may suggest that the DCE is better suited in studies estimating maximum acceptable risk for multiple risk attributes of differing severity, while the PTT may be better suited when measuring heterogeneity in maximum acceptable risk estimates or when investigating one or more serious adverse events.

Eliciting preferences for cancer screening tests: Comparison of a discrete choice experiment and the threshold technique.

OBJECTIVE: To compare results of three preference elicitation methods for a cancer screening test. METHODS: Participants (undergraduate students) completed a discrete choice experiment (DCE) and a threshold technique (TT) task. Accuracy (false positives, false negatives), benefits (lives saved), and cost for a cancer screening test were used as attributes in the DCE and branching logic for the TT. Participants were also asked a direct elicitation question regarding a hypothetical screening test for breast (women) or prostate (men) cancer without mortality benefit. Correlations assessed the relationship between DCE and TT thresholds. Thresholds were standardized and ranked for both methods to compare. A logistic regression used the thresholds to predict results of the direct elicitation. RESULTS: DCE and TT estimates were not meaningfully correlated (max ρ = 0.17). Participant rankings of attributes matched only 20% of the time (58/292). Neither method predicted preference for being screened (ps > 0.21). CONCLUSIONS: The DCE and TT yielded different preference estimates (and rank orderings) for the same participant. Neither method predicted patients' desires for a screening test. PRACTICE IMPLICATIONS: Clinicians, patients, policy makers, and researchers should be aware that patient preference results may be sensitive to the method of eliciting preferences.

U.S. patient preferences for long-acting HIV treatment: a discrete choice experiment.

INTRODUCTION: Recent advances in long-acting antiretroviral therapy (LA-ART) could provide new options for HIV treatment and reduce adherence barriers, if regimens are acceptable to patients. We elicited preferences for key attributes of potential LA-ART regimens among people with HIV (PWH) in the United States, focusing on four treatment modes (oral tablets, subcutaneous injections, intramuscular injections, and implants), product characteristics and location of administration. METHODS: A discrete choice experiment was conducted among PWH aged ≥18 years recruited from HIV clinics in Washington State and Atlanta, Georgia from March 2021 to June 2022. Participants responded to 17 choice scenarios, each with three options: two systematically generated hypothetical LA-ART regimens and a constant opt-out (their current daily oral treatment). LA-ART regimen descriptions included treatment mode, pain, dosing frequency, location, pre-treatment time with undetectable viral load, pre-treatment negative reaction testing and "late-dose leeway" (i.e. flexibility or forgiveness in timing the next dose). We used conditional logistic regression, with an interaction between treatment mode and pain, to estimate preference weights for all attribute levels. RESULTS: Seven hundred participants (350 at each site) enrolled, with median age 51 years (range 18-73); 70% identified as cisgender male, 24% as cisgender female and 6% as non-binary or transgender. LA oral tablets were the only mode preferred over current daily oral treatment, with annual implants and injections the next most preferred LA-ART option. Longer time between doses was preferred, and administration at home was preferred to clinics, which were preferred to pharmacies. Attributes with less impact on preferences included oral lead-in treatment to achieve viral suppression or test for negative reactions and late-dose leeway around the prescribed dosing interval. Participants in Atlanta were more likely to prefer their current daily oral ART than participants from Seattle. CONCLUSIONS: PWH in the United States may soon have several options for LA-ART. Our results suggest that LA oral tablets will be preferred by many patients over their current daily oral treatment, while implants and injections with longer duration may be acceptable to some. Future research should investigate sources of preference heterogeneity and actual uptake of and adherence to LA-ART products, when available.

Preference for daily oral pills over long-acting antiretroviral therapy options among people with HIV.

OBJECTIVE: To examine the characteristics of people with HIV (PWH) who prefer remaining on daily oral antiretroviral therapy (ART), rather than switching to long-acting ART (LA-ART). DESIGN: Building upon a discrete choice experiment (DCE), we examined characteristics of individuals who always selected their current daily oral tablet regimen over either of two hypothetical LA-ART options presented in a series of 17 choice tasks. METHODS: We used LASSO to select sociodemographic, HIV-related, and other health-related predictors of preferring current therapy over LA-ART, and logistic regression to measure the associations with those characteristics. RESULTS: Among 700 PWH in Washington State and Atlanta, Georgia, 11% of participants ( n  = 74) chose their current daily treatment over LA-ART in all DCE choice tasks. We found that people with lower educational attainment, good adherence, more aversion to injections, and who participated from Atlanta to be more likely to prefer their current daily regimen over LA-ART. CONCLUSIONS: Gaps in ART uptake and adherence remain, and emerging LA-ART treatments show promise to address these challenges and help a larger portion of PWH to achieve viral suppression, but preferences for these new treatments are understudied. Our results show that certain drawbacks of LA-ART may help to maintain demand for daily oral tablets, especially for PWH with certain characteristics. Some of these characteristics (lower educational attainment and Atlanta participation) were also associated with a lack of viral suppression. Future research should focus on overcoming barriers that impact preferences for LA-ART among those patients who could benefit most from this innovation.

Use of Bayesian decision analysis to maximize value in patient-centered randomized clinical trials in Parkinson's disease.

A fixed one-sided significance level of 5% is commonly used to interpret the statistical significance of randomized clinical trial (RCT) outcomes. While it is necessary to reduce the false positive rate, the threshold used could be chosen quantitatively and transparently to specifically reflect patient preferences regarding benefit-risk tradeoffs as well as other considerations. How can patient preferences be explicitly incorporated into RCTs in Parkinson's disease (PD), and what is the impact on statistical thresholds for device approval? In this analysis, we apply Bayesian decision analysis (BDA) to PD patient preference scores elicited from survey data. BDA allows us to choose a sample size (n) and significance level (α) that maximizes the overall expected value to patients of a balanced two-arm fixed-sample RCT, where the expected value is computed under both null and alternative hypotheses. For PD patients who had previously received deep brain stimulation (DBS) treatment, the BDA-optimal significance levels fell between 4.0% and 10.0%, similar to or greater than the traditional value of 5%. Conversely, for patients who had never received DBS, the optimal significance level ranged from 0.2% to 4.4%. In both of these populations, the optimal significance level increased with the severity of the patients' cognitive and motor function symptoms. By explicitly incorporating patient preferences into clinical trial designs and the regulatory decision-making process, BDA provides a quantitative and transparent approach to combine clinical and statistical significance. For PD patients who have never received DBS treatment, a 5% significance threshold may not be conservative enough to reflect their risk-aversion level. However, this study shows that patients who previously received DBS treatment present a higher tolerance to accept therapeutic risks in exchange for improved efficacy which is reflected in a higher statistical threshold.