Understanding the experience, treatment preferences and goals of people living with chronic lymphocytic leukemia (CLL) in Australia.

BACKGROUND: Listening to patient voices is critical, in terms of how people experience their condition as well as their treatment preferences. This research explored the patient journey, therapy attributes and goals among treatment experienced adults with chronic lymphocytic leukemia (CLL). We sought to understand patient experiences, needs and expectations to identify areas for improvement of treatment and care delivery. METHODS: Two online surveys were developed for completion by CLL patients. In Stage 1, participants completed a best-worst scaling (BWS) task to evaluate eleven previously validated healthcare journey moments that matter (MTM). Responses were used to generate the patient experience index (PEI) score. In Stage 2, participants completed a survey that included both a discrete choice experiment (DCE) to assess drivers of treatment preferences by evaluating the relative attribute importance (RAI) of seven features and a BWS exercise which explored long-term treatment goals. RESULTS: Twenty-five patients completed Stage 1 and thirty patients Stage 2. Treatment experience was balanced between oral and intravenous medication. The most important/least satisfied MTM were treatment effectiveness, access to support and other treatments as well as monitoring progress. The median PEI score was 66.2 (out of 100). DCE results demonstrated that patients most value treatments for CLL that are associated with prolonged progression free survival (PFS; RAI: 24.6%), followed by treatments that have a lower risk of severe side effects and lower out-of-pocket costs (RAI: 19.5%, 17.4%, respectively). The remainder of the weight in decision making (38.5%) was split between the remaining attributes, namely 'mild to moderate side effects' (13.4%), 'long-term risks' (12.2%), type of treatment (i.e., oral, IV or a combination of oral and IV; 8.7%) and treatment duration (i.e., ongoing versus fixed; 4.2%). Patients preferred oral to intravenous therapy. The most valued long-term treatment goal was to be physically healthy, followed by living a long life, spending time with family/friends, and avoiding hospitalization. CONCLUSION: Treatment experienced patients with CLL are focused on receiving effective, safe therapies and value long PFS. Consideration and discussion of other attributes, such as once daily dosing, oral only medication, out-of-pocket costs and access to support services may affect patient treatment choices and ultimately enhance their healthcare experience and outcomes.

HTA community perspectives on the use of patient preference information: lessons learned from a survey with members of HTA bodies.

This research sought to assess whether and how patient preference (PP) data are currently used within health technology assessment (HTA) bodies and affiliated organizations involved in technology/drug appraisals and assessments. An exploratory survey was developed by the PP Project Subcommittee of the HTA International Patient and Citizen Involvement Interest Group to gain insight into the use, impact, and role of PP data in HTA, as well as the perceived barriers to its incorporation. Forty members of HTA bodies and affiliated organizations from twelve countries completed the online survey. PP data were reported to be formally considered as part of the HTA evidence review process by 82.5 percent of the respondents, while 39.4 percent reported that most of the appraisals and assessments within their organization in the past year had submitted PP data. The leading reason for why PP data were not submitted in most assessments was time/resource constraints followed by lack of clarity on PP data impact. Participants reported that PP data had a moderate level of influence on the deliberative process and outcome of the decision, but a higher level of influence on the decision's quality. Most (81.8 percent) felt patient advocacy groups should be primarily responsible for generating and submitting this type of evidence. Insights from the survey confirm the use of PP data in HTA but reveal barriers to its broader and more meaningful integration. Encouragingly, participants believe obstacles can be overcome, paving the way for a second phase of research involving in-depth collaborative workshops with HTA representatives.

A new subjective well-being index using anchored best-worst scaling.

Subjective well-being (SWB) describes an individual's life evaluation. Direct elicitation methods for SWB via rating scales do not force individuals to trade-off among life domains, whilst best-worst scaling (BWS) approaches only provide relative measures. This paper instead offers a dual-response BWS task, where respondents nominate areas of most and least importance and satisfaction with respect to 11 SWB domains, whilst also eliciting anchoring points to obtain an absolute measure of domain satisfaction. Combining domain satisfaction and importance produces a robust measure of individual SWB, but statistically unique relative to other life satisfaction measures utilizing single- and multi-item ratings, including global satisfaction and those aggregated over SWB domains, as well as eudemonia. Surveying 2500 Australians reveals anchored-BWS improves discrimination amongst domains in terms of importance and satisfaction, illustrating its value as a diagnostic tool for SWB measurement to focus services, policy, and initiatives in areas to most impact wellbeing. This includes highlighting a major discrepancy between health satisfaction and importance, whilst also reporting that SWB is significantly lower for Indigenous, unemployed, middle-aged, males and lower income groups.

Patient preferences for heart valve disease intervention.

BACKGROUND: This study aimed to determine how patients trade-off the benefits and risks of two different types of procedures used to treat heart valve disease (HVD). It also aimed to determine patients' preferences for HVD treatments (predicted uptake) and the relative importance of each treatment attribute. METHODS: A discrete choice experiment (DCE) was conducted in Australia and Japan with patients who required a heart valve procedure. Patients were stratified into three categories: no prior procedure experience, minimally invasive procedure experience and invasive procedure experience. DCE attributes included risk of mortality; risk of stroke; needing dialysis; needing a new pacemaker; valve durability; independence 1 month after surgery; and out-of-pocket expenses. Participants chose between two hypothetical labelled approaches to therapy ('invasive procedure' and 'minimally invasive procedure'), with a separate opt-out included. A mixed multinomial logit model was used to analyse preferences. RESULTS: The DCE was completed by 143 Australian and 206 Japanese patients. Both populations demonstrated an overall preference for the minimally invasive procedure over the invasive procedure. All attributes tested significantly predicted choice and were important to patient decision-making. However, patients' choices were most influenced by the durability of the valve and the likelihood of independence postprocedure, irrespective of their prior procedure experience. Differences in preference were observed between Australian and Japanese patients; valve durability was the most important attribute among Australian patients, while Japanese patients emphasised regaining independence postsurgery. Risk of mortality was less important relative to other key attributes in Japan; however, it remained significant to the model. CONCLUSIONS: HVD patients prefer a minimally invasive procedure over an invasive procedure, irrespective of prior treatment experience. Key attributes contributing to treatment preferences are valve durability and faster recovery. These results can be used to help inform healthcare decision-makers about what features of heart valve procedures patients value most. PATIENT AND PUBLIC CONTRIBUTION: People with lived experience of HVD were included in multiple stages of the design phase of this research. First, patients and doctors were consulted by taking part in qualitative interviews. The qualitative interviews helped inform which treatment attributes to include in the DCE based on what was important to those with lived experience and those who help make treatment decisions on behalf of patients. Qualitative interview participants also assisted with the framing of questions in the online survey to ensure the terminology was patient-friendly and relevant to those with lived experience. Following qualitative interviews, the DCE attribute list was agreed on in expert consultation with a steering committee, which included patient representatives and treating physicians (interventional cardiologists, cardiothoracic surgeons). The survey was also pilot tested with a small sample of patients and minor adjustments were made to the wording to ensure it was appropriate and meaningful to those with lived experience of HVD.

Alignment of preferences in the treatment of multiple myeloma - a discrete choice experiment of patient, carer, physician, and nurse preferences.

BACKGROUND: Multiple Myeloma (MM) is a cancer characterised by the proliferation of malignant plasma cells in the bone marrow. This study examined the treatment preferences of people living with MM compared to the treatment preferences of other groups involved in treatment decision making, including carers, as well as physicians and nurses who treat people living with MM in Australia. METHODS: Data were collected using discrete choice experiments (DCEs) through an online survey. The DCEs presented participants with a traditional treatment generic choice experiment (e.g., treatment A vs treatment B), focusing on the clinical benefits of treatments and the associated risks. The attributes and levels of the attributes were selected based on previous research, literature review, qualitative research and expert opinion. The DCE data were modelled using a Latent Class Model (LCM). RESULTS: The model revealed significant heterogeneity in preferences for treatment attributes. In particular, overall survival, remission period and annual out of pocket cost were the attributes with the most variation. In comparison to people living with MM, carers were less cost-sensitive and more concerned with quality of life (remission period). Physicians and nurses were generally more concerned with overall survival and more cost sensitive than people living with MM. CONCLUSIONS: This study demonstrated that not all people living with MM valued the same treatment attributes equally. Further, not all groups involved in MM treatment decision making had preference alignment on all treatment attributes. This has important implications for healthcare policy decisions and shared decision making. Results from this study could be used to guide decisions around the value of new MM medicines or the medical plan surrounding the needs of those living with MM, as well as those caring for them.

Treatment preferences in people with haemophilia A or caregivers of people with haemophilia A: A discrete choice experiment.

INTRODUCTION: The standard of care for people with haemophilia A (PwHA) is regular prophylactic therapy with Factor VIII. However, very little is known about the treatment preferences of PwHA with and without inhibitors, or their caregivers, when presented with the choice between intravenous and subcutaneous prophylactic treatments with different modes of action, side effect risk and frequency of administration. METHODS: We conducted a discrete choice experiment to assess the preferences of PwHA or their caregivers. The survey was conducted in Australia, New Zealand, Canada, France, Italy and the United Kingdom. RESULTS: There were 56 respondents. The majority of PwHA had hereditary HA (78%) rather than spontaneous HA (22%). Most PwHA were diagnosed when they were under 12 months old (65%). The model identified two segments based on treatment preferences. There is heterogeneity between PwHA in their treatment preferences. People with and without inhibitors prefer to receive treatment via a subcutaneous injection compared to an intravenous infusion. However, for PwHA, efficacy is key; they value a reduction in the annual bleed rate. For those without inhibitors, this also includes a reduction in the risk of developing inhibitors. The side effect risk, administration risk and storage requirements were prioritised differently in each segment. CONCLUSIONS: Results from this study may inform decisions about the value of existing and new treatments for PwHA.

Valuing injection frequency and other attributes of type 2 diabetes treatments in Australia: a discrete choice experiment.

BACKGROUND: Multiple pharmacotherapy options are available to control blood glucose in Type 2 Diabetes Mellitus (T2DM). Patients and prescribers may have different preferences for T2DM treatment attributes, such as mode and frequency of administration, based on their experiences and beliefs which may impact adherence. As adherence is a pivotal issue in diabetes therapy, it is important to understand what patients value and how they trade-off the risks and benefits of new treatments. This study aims to investigate the key drivers of choice for T2DM treatments, with a focus on injection frequency, and explore patients' associated willingness-to-pay. METHODS: A discrete choice experiment (DCE) was used to present patients with a series of trade-offs between different treatment options, injectable and oral medicines that were made up of 10 differing levels of attributes (frequency and mode of administration, weight change, needle type, storage, nausea, injection site reactions, hypoglycaemic events, instructions with food and cost). A sample of 171 Australian consenting adult T2DM patients, of which 58 were receiving twice-daily injections of exenatide and 113 were on oral glucose-lowering treatments, completed the national online survey. An error components model was used to estimate the relative priority and key drivers of choice patients place on different attributes and to estimate their willingness to pay for new treatments. RESULTS: Injection frequency, weight change, and nausea were shown to be important attributes for patients receiving injections. Within this cohort, a once-weekly injection generated an additional benefit over a twice-daily injection, equivalent to a weighted total willingness to pay of AUD$22.35 per month. CONCLUSIONS: Based on the patient preferences, the importance of frequency of administration and other non-health benefits can be valued. Understanding patient preferences has an important role in health technology assessment, as the identification of the value as well as the importance weighting for each treatment attribute may assist with funding decisions beyond clinical trial outcomes.

Patient and Healthcare Professional Preferences for Prescription Weight Loss Medications in Australia: Two Discrete Choice Experiments.

Purpose: Overweight and obesity are common in Australia and among the leading risk factors for ill health. Maintained weight loss of >5-10% can prevent and reduce the risk of obesity-related comorbidities. Prescription weight loss medications plus lifestyle interventions can result in additional weight loss compared with lifestyle interventions alone, but these medications are under-prescribed in Australia. Our aim was to develop a greater understanding of the treatment preferences of people with overweight or obesity and the healthcare practitioners (HCPs) who treat them. Participants and Methods: An online survey of Australian adults with overweight or obesity and treating HCPs was conducted in 2020. A discrete choice experiment (DCE) approach was used to determine what is most important to people when evaluating oral and injectable prescription weight loss medications. Participants were asked to choose between three hypothetical treatment alternatives: "Oral pill"; "Subcutaneous injection pen (replaceable needle)"; "Disposable subcutaneous injection pen (hidden needle)"; and an opt-out option ("None of these"). Results: The online survey and DCE were completed by 193 patients and 104 HCPs. For both patients and HCPs, all treatment alternatives (oral, replaceable injection and disposable injection) were preferred over the opt-out. Gastrointestinal side effects, followed by success rate, percentage body weight lost, and cost were the most important attributes to patients. For HCPs, percentage body weight loss was the most important treatment attribute, followed by success rate, gastrointestinal side effects and cost. While most patients reported relatively low needle fear, physicians reported relatively high perceived patient needle fear. Conclusion: Clinician-patient discussions about treatments for weight loss should cover the option of prescription weight loss medications, including injectable medications, which patients may be less apprehensive about than physicians believe. Treatments with a high success rate and low or manageable risk of gastrointestinal side effects may be preferred over alternatives.

Tradeoffs and decision-making in moderate to severe psoriasis for oral versus injectable treatments: data from patients and dermatologists in Australia.

BACKGROUND: Oral systemic and injectable biologic treatments are available in Australia to treat moderate to severe psoriasis. OBJECTIVE: To examine how patients and dermatologists in Australia choose between oral and injectable treatments for psoriasis. METHODS: In this discrete choice experiment (DCE), adults with moderate to severe psoriasis and dermatologists were asked to choose between 2 treatments labeled by mode of administration ('oral' or 'subcutaneous injection'), each with randomly assigned levels for 9 treatment attributes. Needle fear was rated by patients. RESULTS: Completed surveys from 178 patients and 43 dermatologists were included in the analysis. Symptom reduction, safety, and mode of administration were attributes found to have a significant impact on treatment choice; dosing frequency was a significant attribute for the injectable option. When treatment attributes were held equal, patients and dermatologists preferred oral versus injectable treatments for moderate disease. Patients with higher levels of needle fear were more likely to favor an oral treatment versus patients with lower levels of needle fear. LIMITATIONS: Participation bias may limit the generalizability of these findings. CONCLUSION: Participants preferred oral over injectable treatment for moderate psoriasis. These findings corroborate the need for efficacious oral therapies to treat the disease.

Treatment preferences among Japanese patients and physicians for epidermal growth factor receptor-mutant non-small cell lung cancer.

INTRODUCTION: Evidence is limited on preferences of Japanese patients and physicians in treatment for epidermal growth factor receptor (EGFR)-mutant non-small cell lung cancer (NSCLC). Several oral or intravenous novel agents for EGFR exon 20 insertions are under development. The aim of our study was to investigate which attributes of novel treatments influenced selection of oral or intravenous agents among treated patients and treating physicians in Japan. METHODS: The study was designed by board-certified oncologists, patient representatives, and analytics specialists. Eligible participants completed an online survey with a discrete choice experiment presenting two treatment profiles described by attributes: mode of administration (oral or intravenous); frequency of administration; overall response rate (ORR); average progression-free survival (PFS); chance of experiencing severe side effects (SEs); mild-moderate gastrointestinal SEs; mild-moderate skin-related SEs; and patient out-of-pocket costs. RESULTS: Fifty-four patients (all self-reported EGFR-mutant) and 74 physicians participated from December 2021 to August 2022. All attributes being equal, there was greater preference for oral administration. However, there was greater preference for intravenous over oral, when ORR and PFS improved by 10% and 1 month, and severe SEs reduced by 10%. Physicians exhibited greater preference for PFS compared to patients (p < 0.01). Ranked order of attribute importance was as follows: (1) PFS; (2) ORR; (3) severe SEs, expressed by patients and physicians alike. CONCLUSIONS: Our study revealed Japanese physician and patient preferences in treatment options for EGFR-mutant NSCLC. Compared to the strong preference for a more efficacious drug, the preference of oral versus intravenous revealed a smaller impact.

Understanding Treatment Preferences Among People Living with HIV in Australia: A Discrete Choice Experiment.

Purpose: To better understand what is most important to people living with human immunodeficiency virus (PLWH) when choosing their treatment. We assessed how PLWH trade off the potential risks and benefits of oral and long acting injectable (LAI) treatments. Participants and Methods: Firstly, in-depth interviews were conducted with 11 PLWH to develop a holistic understanding of experiences and determine attributes that contribute to treatment decision-making. Secondly, a discrete choice experiment (DCE) was used to understand the treatment preferences for PLWH with n = 99 PLWH aged 18 years or over with a diagnosis of human immunodeficiency virus (HIV) and who were currently using anti-retroviral therapy (ART). Study participants were presented with 12 scenarios and asked to select their preferred treatment among two hypothetical injectable treatment alternatives, "injection 1" and "injection 2" and their current oral ART treatment. The DCE data were modelled using a latent class model (LCM). Results: The model revealed significant heterogeneity in preferences for treatment attributes among study participants. Two segments/classes of PLWH were identified. The first segment expressed a strong preference for their current oral treatment; the second segment showed strong preference for the injection treatment and for it to be administered in a GP clinic. Overall, out-of-pocket cost was the most important attribute for participants. One-third of PLWH were willing to switch to an LAI. Conclusion: Not all PLWH valued the same treatment attributes equally. Overall, out-of-pocket costs for treatments were considered by respondents as the most determining factor in making treatment choices. Results have important implications for healthcare policy and will serve to better inform patients and stakeholders involved in the treatment decision-making process about the treatment preferences of PLWH. Clinicians are encouraged to consider shared decision-making to establish the treatment course that best aligns with PLWH's treatment goals.

Patient Satisfaction and Sensory Attributes of Nasal Spray Treatments of Olopatadine Hydrochloride/Mometasone Furoate Monohydrate and Azelastine Hydrochloride/Fluticasone Propionate for Allergic Rhinitis in Australia - An Observational Real-World Clinical Study.

Purpose: Combination intranasal corticosteroid and antihistamine sprays are a first-line treatment option for allergic rhinitis (AR), of which Azelastine Hydrochloride and Fluticasone Propionate nasal spray (AZE/FLU; Dymista®), and Olopatadine Hydrochloride and Mometasone Furoate Monohydrate nasal spray (OLO/MOM; Ryaltris®) are currently registered in Australia. As it is not known how patients value treatment attributes of current combination nasal sprays, this observational, real-world clinical study aimed to understand patients' satisfaction with, and importance of, treatment attributes of OLO/MOM and AZE/FLU using an Anchored Best-Worst Scaling (ABWS) exercise. Participants and Methods: Four hundred and twenty-six adults in Australia with moderate to severe AR using either OLO/MOM or AZE/FLU completed an online survey incorporating an ABWS with 11 domains: 7 sensory (immediate taste of medication, aftertaste of medication, smell of medication, irritation to your nose, urge to sneeze, dripping out your nose/down your throat, dryness of your nose/throat) and 4 treatment-related (convenience, fast acting, duration of effect, and AR symptom control). The ABWS involved rescaling individual BWS scores using anchored ratings (0-10) for most and least satisfied/important domains to create a total satisfaction index (TSI) (0-100) to be compared across groups. Statistical comparisons were completed using ANOVA (TSI) and MANOVA (individual domains). Results: Participants using OLO/MOM (M = 68.26, SE = 1.39) had significantly higher TSI than participants using AZE/FLU (M=62.78, SE = 0.70) (p < 0.001), significantly higher satisfaction on 7 of 11 domains and regarded 8 of 11 domains as significantly more important compared to participants using AZE/FLU (all p < 0.05). Preferred domains were predominantly sensory attributes. Conclusion: Current findings showed that participants using OLO/MOM were more satisfied with their overall treatment compared to participants using AZE/FLU, particularly with sensory attributes, thus highlighting the suitability of OLO/MOM for people with AR who value sensory attributes. Prescribers of AR treatments are encouraged to discuss treatment attributes with patients to facilitate shared decision-making.

Patient and Clinician Preferences for Genetic and Genomic Testing in Non-Small Cell Lung Cancer: A Discrete Choice Experiment.

Precision (personalised) medicine for non-small cell lung cancer (NSCLC) adopts a molecularly guided approach. Standard-of-care testing in Australia is via sequential single-gene testing which is inefficient and leads to tissue exhaustion. The purpose of this study was to understand preferences around genetic and genomic testing in locally advanced or metastatic NSCLC. A discrete choice experiment (DCE) was conducted in patients with NSCLC (n = 45) and physicians (n = 44). Attributes for the DCE were developed based on qualitative interviews, literature reviews and expert opinion. DCE data were modelled using a mixed multinomial logit model (MMNL). The results showed that the most important attribute for patients and clinicians was the likelihood of an actionable test, followed by the cost. Patients significantly preferred tests with a possibility for reporting on germline findings over those without (ß = 0.4626) and those that required no further procedures over tests that required re-biopsy (ß = 0.5523). Physician preferences were similar (ß = 0.2758 and ß = 0.857, respectively). Overall, there was a strong preference for genomic tests that have attribute profiles reflective of comprehensive genomic profiling (CGP) and whole exome sequencing (WES)/whole genome sequencing (WGS), irrespective of high costs. Participants preferred tests that provided actionable outcomes, were affordable, timely, and negated the need for additional biopsy.

Understanding the Treatment Preferences of People Living with Schizophrenia in Australia; A Patient Value Mapping Study.

Purpose: To examine the treatment and long-term outcome preferences for people living with schizophrenia. Patients and Methods: Sixty-six Australian adults, living with schizophrenia completed a novel online survey with six sections: Demographic characteristics; Disease history; Quality-of-life; Patient support programmes; Discrete Choice Experiment, and Best-Worst Scaling exercise. Results: Participants indicated that they preferred to be involved in treatment decision with their doctor. A minority of participants reported having been previously involved in a patient support programme (28.8%) and only one in six participants had a National Disability Insurance Scheme (NDIS) package (16.7%) with over a third of participants indicating that they were ineligible (37.9%). Participants' average quality-of-life score was 60%. Conclusion: Recent hospitalisation influenced the relative importance of treatment attributes, with effectiveness on hearing voices being the most important treatment attribute. The most important long-term goals were having a stable place to live, being independent, and physical health. People with schizophrenia care about their long-term functional recovery outcomes, rating symptom control and independence as their highest priority. They want to be part of the treatment conversation with their doctors. Therefore, psychiatrists are encouraged to use shared decision-making to establish the treatment course that best aligns with individuals' long-term goals.

Patient advocacy group involvement in health technology assessments: an observational study.

BACKGROUND: In some jurisdictions, patients and patient groups may be invited to provide input when Health Technology Assessment (HTA) is used to inform decisions about which medicines should be subsidised or funded. This input can help frame the evidence from a patient perspective, address uncertainties in the evidence and interpret it for the local setting. However, there is currently no evidence linking patient involvement with positive reimbursement decisions. AIM: We aimed to understand the expectations of patient involvement in the reimbursement process, especially among cancer patient advocacy groups (PAGs) in New Zealand (Aotearoa), South Korea and Taiwan. METHODS: We developed an online survey to help understand the role that cancer PAGs play in reimbursement processes and identify knowledge gaps about the processes that might impact the efforts of PAGs. The survey elicited the views of staff and patients affiliated with PAGs (n = 43) on current practices and how the assessment and reimbursement of new cancer drugs might be improved. RESULTS: There was variability in knowledge of the HTA assessment processes and in experience of being involved in them. Those with HTA experience were more likely to have confidence in the process. Those who had not been involved tended to have little awareness of, or frustration with, decision-making processes. Most identified cost, finances and economic assessments as key considerations in current processes. Some respondents had clear ideas about how their knowledge and involvement could improve processes to determine the value of new medicines. However, for many, a lack of information about the basis for decision making and opportunities to be involved was a barrier to identifying process improvement. CONCLUSIONS: HTA is implemented primarily in countries seeking to have fair and equitable processes for funding medicines. PAGs often recognise the financial challenges of funding new medicines and share the desire for procedural fairness. The connection PAGs make between patient involvement and improved access to new medicines may be based on the belief they can add information to the evidence base, help solve problems, ensure fairness through transparency and/or influence the culture towards increased access to medicines they value.

WHAT ARE HEALTH TECHNOLOGY ASSESSMENTS?: Health technologies are medicines, devices and services used to prevent, diagnose or treat medical conditions. A Health Technology Assessment (HTA) considers the value of a health technology to help governments make fair decisions about which treatments to fund or subsidise. Patients and Patient Advocacy Groups (PAGs) may be invited to provide input into this process. WHAT DID OUR STUDY LOOK AT?: We surveyed staff and patients from cancer PAGs in New Zealand (Aotearoa), South Korea and Taiwan to understand their experiences and expectations of involving patients in HTA. A total of 43 people completed our survey. WHAT WERE THE RESULTS?: Overall, 20 of the 43 surveyed people (47%) were aware of the assessment process used to decide which medicines were funded in their country. (27 of these 50 people were PAG staff members.) Patients from South Korea and New Zealand were rarely included in the assessment process. Patient involvement was more commonly reported in Taiwan. The impact of input from patients on HTA was rated low by patients in both South Korea and New Zealand and average in Taiwan. A general lack of transparency, feedback on the process, and support from assessment agencies was also reported. Many of the patients surveyed wanted to see more patient involvement in the assessment processes. WHAT IS THE MAIN TAKE-HOME MESSAGE FROM THIS STUDY?: With improved guidance and feedback from assessment agencies, PAGs and patients believe their involvement can improve decision making on treatment funding and increase fairness through transparency.

Understanding Treatment Preferences of Australian Patients Living with Treatment-Resistant Depression.

BACKGROUND: There is evidence of improved adherence and treatment outcomes when patients' treatment preferences are considered, and shared decision making is utilized. PURPOSE: We aimed to better understand treatment preferences among Australians with treatment-resistant depression (TRD), focusing on the specific treatment attributes that people value (such as effectiveness, risk of side effects and cost) and their relative importance. The risk-benefit trade-offs that characterize treatment choices were also examined. PATIENTS AND METHODS: An online survey of 75 patients with experience of TRD was conducted, consisting of two discrete choice experiment (DCE) components - a medication DCE and a treatment plan DCE. Participants were able to prioritize and trade off different features of medications and treatment plans. Additional questions aimed to better define this population group, which in Australia is poorly understood. RESULTS: In both DCEs, two distinct latent classes were identified. In the medication DCE, the classes were distinguished by willingness to consider new treatment alternatives. Participants in class 1 were reluctant to give up current treatment, while those in the slightly larger class 2 preferred new treatment options. In both classes, treatment effectiveness and cost were the greatest contributors to preference. Similar behavior was seen in the treatment plan DCE, with the larger class more likely to choose a new plan over their current treatment arrangement. Participants preferred medications that were low-cost, taken orally, had a high percentage improvement in mood symptoms, high rate of remission and low risk of weight gain. A similar result was found in preferences for treatment plans such that plans with the greatest effectiveness and lowest cost were most favorable. CONCLUSION: Patient preferences should routinely be considered and discussed to guide informed decisions regarding the value of new and existing medications for TRD and how they sit in the context of treatment plans.

Myeloma Patient Value Mapping: A Discrete Choice Experiment on Myeloma Treatment Preferences in the UK.

BACKGROUND: Myeloma is an incurable life-threatening hematological cancer. Recent treatment developments have seen improvements in survival; however, while patients are living longer, they are living with symptoms and treatment side effects. OBJECTIVE: To evaluate myeloma patients' preferences for treatment using a discrete choice experiment (DCE). This study set out to define the relative importance of key treatment attributes, characterize the risk-benefit trade-offs in patients' decision-making, and to analyze the predictive power of basic demographic factors. METHODS: Four hundred seventy-five myeloma patients in the UK were invited to participate by Myeloma UK. Data were collected using DCEs through an online survey. The DCEs presented patients with 10 choice scenarios, each with 2 treatment options described by 7 attributes, and a "no treatment" option. The DCE data were modelled using a latent class model (LCM). The effects of demographic characteristics were also examined. RESULTS: Not surprisingly, average survival was most important to all patients but there were significant contrasts between the class preferences. The LCM revealed two classes of patients. Patients in Class 1 placed greater importance on average survival and mild-to-moderate side effects, whereas patients in Class 2 focused on the mode of administration and the average out-of-pocket costs. Patients living with others and those diagnosed in the last 5 years were more likely to be in Class 1. CONCLUSION: Different treatment features were not valued equally among all myeloma patients. This has important implications for healthcare policy decisions and could be used to guide decisions around the value of new myeloma medicines.

Patient and physician preferences for atopic dermatitis injection treatments in Japan.

Background: Recently, biologic agents administered as a subcutaneous injection have been introduced as treatment options for atopic dermatitis (AD). Biologic treatments differ considerably from traditional topical and systemic anti-inflammatory treatments, and it is unclear how this may impact patient and physician preferences for treatments.Objective: To examine the treatment preferences for new injection treatments of patients and physicians.Methods: Discrete choice experiment methodology was used to quantify preferences for treatments via an online survey. Participants were presented with a series of choice scenarios; treatment options were described using the following attributes: add-on or replacement treatment, efficacy of improving rashes and itching, time until response, place of administration, injection site reaction, risk of mild-to-moderate and severe side effects, frequency of administration and cost.Results: 76.67% of physicians and 46.24% of patients opted-in to the new treatment in the scenarios presented. Of those who opted-in to treatment, physicians were more likely to value the efficacy of treating rashes and were more concerned about cost than patients. Patients preferred add-on treatments and were against self-administering the treatment at home.Conclusion: Overall patients and physicians differ in their preferences for AD treatments. These findings have implications for shared decision making and clinical practice.

What do Australian patients with inflammatory arthritis value in treatment? A discrete choice experiment.

BACKGROUND AND OBJECTIVES: The purpose of this study was to develop an understanding of treatment preferences in patients with inflammatory arthritis (IA) [rheumatoid arthritis (RA), ankylosing spondylitis (AS) and psoriatic arthritis (PsA)] focussing on treatment attributes that patients' value, their relative importance, and the risk-benefit trade-offs that characterise patients' choices around treatment. METHODS: A discrete choice experiment (DCE) approach was used. Attributes of interest were clinical efficacy; slowing of disease progression; risk of mild-moderate side effects; risk of severe side effects; frequency of administration; real-world product evidence; management of related conditions; and availability of a patient support programme. Using data from the DCE component, a restricted latent class model (LCM) was estimated to determine discrete 'classes' of treatment preferences. RESULTS: In this analysis, 206 participants were included (AS n = 59; PsA n = 62; RA n = 85). Two classes were identified. For 'class 1' (59.9%), the most important attributes (across all treatment modalities) were preventing disease progression, clinical efficacy and risk of mild-to-moderate side effects. For 'class 2' (40.1%), clinical and non-clinical attributes were important, and attribute importance depended on treatment modality. Patient demographic and treatment characteristics did not predict class membership. CONCLUSION: For most patients with IA, clinical efficacy, stopping disease progression and risks of mild-to-moderate side effects are important treatment attributes. Patients with prior biologic DMARD experience had greater preference for injection treatments. For a subset of patients, patient support programmes and the frequency of administration were important. Clinicians should be mindful of preferences when prescribing treatment to patients with IA.Key Points• Most patients consider clinical efficacy, stopping disease progression and the risk of mild-to-moderate side effects as important treatment attributes• Patients with prior biologic DMARD experience have greater preference for injection treatments.• For a subset of patients, patient support programmes, and the frequency of administration were important.• Clinicians should be mindful of preferences when prescribing treatment to patients with IA.