The financial burden of accessing care for people with scleroderma in Canada: a patient-oriented, cross-sectional survey.

BACKGROUND: Patients with scleroderma require a lifetime of treatment and frequent contacts with rheumatologists and other health care professionals. Although publicly funded health care systems in Canada cover many costs, patients may still face a substantial financial burden in accessing care. The purpose of this study was to quantify out-of-pocket costs borne by people with scleroderma in Canada and compare this burden for those living in large communities and smaller communities. METHODS: We analyzed responses to a Web-based survey of people living in Canada with scleroderma. Respondents reported annual out-of-pocket medical, travel and accommodation and other nonmedical costs (2019 Canadian dollars). We used descriptive statistics to describe travel distance and out-of-pocket costs. We used a 2-part model to estimate the impact on out-of-pocket costs of living in a large urban centre (≥ 100 000 population), compared with smaller urban centres or rural areas (< 100 000 population). We generated combined mean estimates from the 2-part models using predictive margins. RESULTS: The survey included 120 people in Canada with scleroderma. The mean, annual, total out-of-pocket costs were $3357 (standard deviation $5580). Respondents living in smaller urban centres and rural areas reported higher mean total costs ($4148, 95% confidence interval [CI] $3618-$4680) and travel or accommodation costs ($1084, 95% CI $804-$1364) than those in larger urban centres (total costs $2678, 95% CI $2252-$3104; travel or accommodation costs $332, 95% CI $207-$458). INTERPRETATION: Many patients with scleroderma incur considerable out-of-pocket costs, and this burden is exacerbated for those living in smaller urban centres and rural areas. Health care systems and providers should consider ways to alleviate this burden and support equitable access to care.

Clinician preferences for neurotechnologies in pediatric drug-resistant epilepsy: A discrete choice experiment.

OBJECTIVE: Novel and minimally invasive neurotechnologies offer the potential to reduce the burden of epilepsy while avoiding the risks of conventional resective surgery. Few neurotechnologies have been tested in randomized controlled trials with pediatric populations, leaving clinicians to face decisions about whether to recommend these treatments with insufficient evidence about the relevant risks and benefits. This study specifically explores the preferences of clinicians for treating pediatric drug-resistant epilepsy (DRE) with novel neurotechnologies. METHODS: A discrete-choice experiment (DCE) was designed to elicit the preferences of clinicians with experience in treating children with DRE using novel neurotechnological interventions. The preferences for six key attributes used when making treatment decisions (chances of clinically significant improvement in seizures, major and minor risks from intervention, availability of evidence, financial burden for the family, and access to the intervention) were estimated using a conditional logit model. The estimates from this model were then used to predict the adoption of existing novel neurotechnological interventions. RESULTS: Sixty-eight clinicians completed the survey: 33 neurosurgeons, 28 neurologists, and 7 other clinicians. Most clinicians were working in the United States (74%), and the remainder (26%) in Canada. All attributes, apart from the nearest location with access to the intervention, influenced preferences significantly. The chance of clinically significant improvement in seizures was the most positive influence on clinician preferences, but low-quality evidence and a higher risk of major complications could offset these preferences. Of the existing neurotechnological interventions, vagus nerve stimulation was predicted to have the highest likelihood of adoption; deep brain stimulation had the lowest likelihood of adoption. SIGNIFICANCE: The preferences of clinicians are drive primarily by the likelihood of achieving seizure freedom for their patients, but preferences for an intervention are largely eradicated if only low quality of evidence supporting the intervention is available. Until better evidence supporting the use of potentially effective, novel neurotechnologies becomes available, clinicians are likely to prefer more established treatments.

Understanding Attributes that Influence Physician and Caregiver Decisions About Neurotechnology for Pediatric Drug-Resistant Epilepsy: A Formative Qualitative Study to Support the Development of a Discrete Choice Experiment.

OBJECTIVE: This study reports formative qualitative research used to analyze decision making regarding neurotechnological interventions for pediatric drug-resistant epilepsy from the perspective of physicians and caregivers and the derivation of attributes for a discrete choice experiment. METHODS: Purposive and convenience sampling was used to recruit physicians and caregivers. Physician focus group sessions were held at key national conferences in the USA and Canada. Caregivers were approached through clinics with established epilepsy surgery programs in the USA and Canada. Thematic analysis was used to identify critical features of decisions about treatment outcomes, procedural trade-offs, values, and concerns surrounding conventional and novel pediatric drug-resistant epilepsy interventions among physicians and caregivers. RESULTS: The results highlight the presence of central attributes that are considered by both groups in decision making, such as "chances of seizure freedom", "risk", "availability of evidence", and "cost to families", as well as attributes that reflect important differences between groups. Physicians were focused on the specifics of treatment options, while caregivers thought more holistically, considering the overall well-being of their children. DISCUSSION: The findings shaped the development of a discrete choice experiment to understand the likely uptake of different neurotechnologies. We identified differences in decision making and thus designed two discrete choice experiments to elicit preferences for pediatric drug-resistant epilepsy treatments, one aimed at clinicians and one at caregivers. The variation we observed highlights the value of seeking to understand the influences at the point of clinical decision making and incorporating this information into care.

Understanding multidisciplinary care for people with rheumatic disease in British Columbia, Canada, through patients, nurses and physicians voices: a qualitative policy evaluation.

BACKGROUND: In 2011, the province of British Columbia (BC) moved to allow patients with complex rheumatic disease to be seen by nurses along with their rheumatologist by introducing a 'Multidisciplinary Care Assessments' (MCA) billing code (G31060). OBJECTIVE: To describe multidisciplinary care introduced as part of MCAs across BC and investigate the perceived impact of this intervention, the addition of nurses to the care team, on patient care from the perspective of patients, nurses, and rheumatologists. METHODS: We conducted semi-structured interviews, informed by a qualitative evaluation approach with patients, nurses, and rheumatologists from September 2019 - August 2020. Interviews investigated 1) the experiences of all stakeholders with adopting the multidisciplinary care billing code, 2) the perceived role of the nurse in the care team, and 3) the perceived impact of multidisciplinary care on patient experience and outcomes. We purposefully sampled practices for maximum variation of geographical location (rural vs. urban), size of practice (i.e., patient caseload), and number of nurses employed. RESULTS: We interviewed 21 patients, 13 nurses, and 12 rheumatologists from across BC. Our analysis identified variation in the way rheumatologists adopted multidisciplinary care across BC. Our analysis showed some heterogeneity in the way the MCA was delivered in rheumatology practices; however, patient education was identified as the core role of nurses across practices. We identified six core themes describing the impact of this model of care, all representing improvements in the way practices functioned, from improved efficiency to access, patient experience, time management, clinician experience, and patient health outcomes. Contextual factors that influenced the presence of these themes were related to the time the nurses spent with patients and the professional roles they performed. CONCLUSION: Our results suggest nurse care can complement physician care by extending contact time for patients and promoting the efficient use of health care professionals' skills, time, and resources. These data may encourage future uptake of the billing code to help ensure the policy delivers maximum benefits to patients given the wide range of perceived benefits described by clinicians and patients.

Co-production of randomized clinical trials with patients: a case study in autologous hematopoietic stem cell transplant for patients with scleroderma.

BACKGROUND: Increasingly, it is argued that clinical trials struggle to recruit participants because they do not respond to key questions or study treatments that patients will be willing or able to use. This study explores how elicitation of patient-preferences can help designers of randomized controlled trials (RCTs) understand the impact of changing modifiable aspects of treatments or trial design on recruitment. METHODS: Focus groups and a discrete choice experiment (DCE) survey were used to elicit preferences of people with scleroderma for autologous hematopoietic stem cell transplant (AHSCT) treatment interventions. Preferences for seven attributes of treatment (effectiveness, immediate and long-term risk, care team composition and experience, cost, travel distance) were estimated using a mixed-logit model and used to predict participation in RCTs. RESULTS: Two hundred seventy-eight people with scleroderma answered the survey. All AHSCT treatment attributes significantly influenced preferences. Treatment effectiveness and risk of late complications contributed the most to participants' choices, but modifiable factors of distance to treatment center and cost also affected preferences. Predicted recruitment rates calibrated with participation in a recent trial (33%) and suggest offering a treatment closer to home, at lower patient cost, and with holistic, multidisciplinary care could increase participation to 51%. CONCLUSIONS: Through a patient engaged approach to preference elicitation for different features of AHSCT treatment options, we were able to predict what drives the decisions of people with scleroderma to participate in RCTs. Knowledge regarding concerns and the trade-offs people are willing to make can inform clinical study design, improving recruitment rates and potential uptake of the treatment of interest.

Designing Discrete Choice Experiments Using a Patient-Oriented Approach.

Patient-oriented research is a process whereby patients or caregivers are included as research partners so that research focusses on topics that are priorities and lead to findings that translate into practice. Using a case study of preferences for stem cell transplant in scleroderma, we report on a patient-oriented research approach to developing a discrete choice experiment. Our patient-oriented research application followed the four guiding principles in Canada's Strategy for Patient-Oriented Research: inclusiveness, support, mutual respect and co-build. In this case study, patient partners were involved at different levels of engagement to match individual availability, skillset and roles in the team. They advised, to different degrees, on all aspects of the study from design to analyses. Using a patient-oriented research approach led to the inclusion of attributes that would likely have been excluded (e.g. support from a multidisciplinary team), and realistic framing of patient-relevant and sometimes sensitive attributes (e.g. mortality and cost). Meeting locations and times were adjusted to accommodate all-team circumstances. Institutional constraints on the reimbursement for patient partners influenced the timing and extent of involvement. We found that adopting a patient-oriented research approach to discrete choice experiment design injected unique knowledge and expertise into the team, improved the representativeness of the sample recruited, minimised researcher biases, and ensured appropriate attribute selection and descriptions. The patient-oriented research approach highlighted some constraints of discrete choice experiment designs and, while not a solution, might ensure the methodological trade-offs remain patient relevant. Institutional challenges must be addressed to progress patient-oriented health economics research.

Patient-provider communication about medication cost in rheumatoid arthritis.

OBJECTIVE: To examine the perceived importance and frequency with which out-of-pocket medication costs are discussed between rheumatologists and patients with rheumatoid arthritis (RA) in Canada. METHODS: A cross-sectional online survey was distributed to patients with RA and rheumatologists; both were asked to rate their perceived importance of discussing medication costs, and how often these discussions occurred. Predictors of (1) patients discussing costs with their rheumatologist and (2) the perceived importance of discussing medication cost for patients were explored. RESULTS: Seventy-eight patients and 64 rheumatologists completed the survey; 68% patients and 75% of physicians rated the perceived importance of discussing medication costs as "quite" or "very important"; 22% of patients reported never talking about medication cost, but no physicians reported never discussing costs with patients. The only predictor of talking about cost among patients (at 10% level) was whether they perceived it as highly important (p = 0.058). Higher perceived importance of discussing out-of-pocket costs was associated with a more positive attitude to shared decision-making (p = 0.044). CONCLUSION: Discussions about cost do not always happen, even with diseases with potentially high medication costs like RA. Cost was more likely to be discussed by patients who perceived it as "very important," suggesting the onus might be on patients to initiate these conversations. Without any significant predictors regarding what may make physicians more likely to think it was important to discuss medication costs, there is a need to reinforce recommendations that all physicians seek to discuss costs with all of their patients when suggesting medications. Key Points • There is a need for patients and physicians to discuss costs in the treatment decision-making process. Our findings suggest this does not always happen. • Among patients, medication cost was more likely to be discussed by those who perceived it as "very important" and higher perceived importance of discussing out-of-pocket costs was associated with a more positive attitude to shared decision-making. • Our results did not reveal any significant predictors regarding what may make physicians more likely to think it was important to discuss medication costs, suggesting that there is a need to reinforce recommendations that all physicians seek to discuss medication costs with all of their patients when suggesting medications.

The Clinical Research Landscape of Pediatric Drug-Resistant Epilepsy.

OBJECTIVE: To characterize the clinical research landscape of pediatric drug-resistant epilepsy (DRE) with a focus on neurotechnology. METHOD: We searched the ClinicalTrials.gov registry using the terms "epilepsy" and "drug resistant" for studies including participants age 0-17 years. Returns were grouped by intervention (eg, neurotechnological, drug). Key trial features such as age range, trial status and outcomes were compared across interventions. RESULTS: We identified 101 registered trials with pediatric DRE patients. Thirty-two (32%) investigate neurotechnological interventions, devices, or diagnostic procedures; 13 (41%) are currently active. Among neurotechnology trials, 15 (46%) investigate vagus nerve stimulation, transcranial direct current stimulation, or deep brain stimulation; few are specific to children. Of the remaining 69 trials, 37 investigate a drug, 17 investigate a dietary therapy, and 15 investigate another intervention. Seizure frequency is the most frequent primary outcome measured in the trials identified. SIGNIFICANCE: The landscape of registered trials pertaining to pediatric DRE reflects a lag between clinical research and clinical practice, and highlights the need for timely evidence before novel neurotechnological interventions are widely adopted into clinical practice.

The use of routinely collected patient-reported outcome measures in rheumatoid arthritis.

This study systematically reviewed commonly used patient-reported outcome measures (PROMs) for rheumatoid arthritis (RA), routinely collected in clinical practice, and evaluated objectives of their use. An additional survey conducted among identified RA registries provided additional information about collection of PROMs. Medline Ovid and Embase were searched for observational studies using data of RA registries/cohorts, published between 2011 and 2016. The search combined a validated search algorithm for PROMs and RA. Study characteristics, objective, registry, country, type of PROMs collected, and time interval of collection were systematically recorded. The survey asked about PROMs collected by the registries, timing, response rates, and barriers to collection. Ninety-eight articles from 15 countries were identified making use of 37 registries and large cohorts. Thirty-three PROMs were collected routinely, with VAS, EQ-5d and HAQ being the most used tools. Health domains reported the most were functional assessment, pain and patient global assessment. Despite the wide variety of collected PROMs, foci of the articles were similar and reported results narrow. This review suggests rethinking the role of PROMs in rheumatology research to use this information as broadly as possible, including evaluation of treatments, economic analyses, and decision-making based on patients' experiences at system, provider, and physician level.

Cancer-related traumatic stress reactions in siblings of children with cancer.

The purpose of this study was to explore cancer-related posttraumatic stress (PTS) reactions in siblings of children with cancer including prevalence, common symptoms, comorbidity with anxiety and depression, and gender- and age-related patterns. A total of 125 children (63 girls) between the ages of 8 and 17 (M = 12.4; SD = 2.9 years) with a brother or sister with cancer, diagnosed 4 to 38 months prior to the study (M = 1.3 years; SD = 6.7 months), completed the Child PTSD Symptom Scale (CPSS), Revised Children's Manifest Anxiety Scale, and Child Depression Inventory-Short Form. Over half of the sample (60%) scored in the moderate to severe range for PTS and 22% fulfilled full criteria for PTSD based upon CPSS responses. Nearly 75% reported "Feeling upset when you think about or hear about the cancer," and "Trying not to think about, talk about, or have feelings about the cancer." Over 60% reported arousal symptoms. PTS symptoms reportedly interfered with functioning for 75% of the sample and co-occurred with anxiety and depressive symptoms. Gender and age-related patterns were not found. Siblings of children with cancer experience cancer-related PTS reactions and greater attention should be paid to ameliorating their cancer-related distress with empirically based treatments.