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.

Budget impact of oral nirmatrelvir/ritonavir in adults at high risk for progression to severe COVID-19 in the United States.

BACKGROUND: Nirmatrelvir/ritonavir (NMV/r) is indicated for the treatment of mild-to-moderate COVID-19 in adults who are at high risk for progression to severe COVID-19. NMV/r has also been authorized for emergency use by the US Food and Drug Administration for the treatment of mild-to-moderate COVID-19 in pediatric patients (aged 226512 years and weighing at least 40 kg) who are at high risk for progression to severe COVID-19. Understanding the budget impact of introducing NMV/r for the treatment of adults with COVID-19 is of key interest to US payers. OBJECTIVE: To estimate the annual budget impact of introducing NMV/r in a US commercial health plan setting in the current Omicron COVID-19 era. METHODS: A budget impact model was developed to assess the impact of NMV/r on health care costs in a hypothetical 1-million-member commercial health insurance plan over a 1-year period in the US population; clinical and cost inputs were derived from published literature with a focus on studies in the recent COVID-19 era that included vaccinated population and predominance of the Omicron variant. In the base-case analysis, it was assumed the only effect of NMV/r was a reduction in incidence (not severity) of hospitalization or death; its potential effect on post-COVID conditions was assessed in a scenario analysis. Outcomes included the number of hospitalizations, total cost, per patient per year (PPPY) costs, and per member per month (PMPM) costs. Sensitivity and scenario analyses were conducted to assess uncertainty around key model inputs. RESULTS: An estimated 29,999 adults were eligible and sought treatment with oral antiviral for COVID-19 over 1 year. The availability of NMV/r was estimated to reduce the number of hospitalizations by 647 with a total budget impact of $2,733,745, $91 PPPY, and $0.23 PMPM. NMV/r was cost saving when including post-COVID conditions with a -$1,510,780 total budget impact, a PPPY cost of -$50, and a PMPM cost of -$0.13. Sensitivity analyses indicated results were most sensitive to the risk of hospitalization under supportive care, risk of hospitalization with NMV/r treatment and cost of NMV/r. CONCLUSIONS: Treatment with NMV/r in the current COVID-19 era is estimated to result in substantial cost offsets because of reductions in hospitalization and modest budget impact to potential overall cost savings.

Cost-efficiency of specialist inpatient rehabilitation for adults with multiple sclerosis: A multicentre prospective cohort analysis of the UK Rehabilitation Outcomes Collaborative national clinical dataset for rehabilitation centres in England.

BACKGROUND: Rehabilitation is effective for multiple sclerosis, but is it value for money? OBJECTIVES: To evaluate functional outcomes, care needs and cost-efficiency of specialist inpatient rehabilitation for adults with multiple sclerosis (MS). METHODS: A multicentre cohort study of prospectively collected clinical data from the UK Rehabilitation Outcomes Collaborative national clinical database. Data included all adults with MS (n = 1007) admitted for specialist inpatient (Level 1 or 2) rehabilitation in England, 2010-2018. OUTCOME MEASURES: Dependency/care needs: Northwick Park Dependency Scale/Care Needs Assessment, Functional independence: UK Functional Assessment Measure (UK FIM+FAM). Cost-efficiency. Patients were analysed in three dependency groups (High/Medium/Low). RESULTS: All groups showed significant reduction in dependency between admission and discharge on all measures (paired t-tests: p < 0.001). Mean reduction in care costs/week was greatest in the most dependent patients: High: £519 (95% CI: 447-597), Medium: £148 (76-217), Low: £36 (12-83). Despite longer stays, time taken to offset the cost of rehabilitation was shortest in the most dependent patients: High: 12.9 (12.0-14.1) months; Medium: 29.3 (21.3-51.8); Low: 76.8 (0-36.1). Item-level changes corresponded with clinical experience. CONCLUSIONS: Specialist rehabilitation provided good value for money in patients with MS, yielding improved outcomes and substantial savings in ongoing care costs, especially in high-dependency patients.

Estimated Life-Time Savings in the Cost of Ongoing Care Following Specialist Rehabilitation for Severe Traumatic Brain Injury in the United Kingdom.

OBJECTIVES: To evaluate cost-efficiency of rehabilitation following severe traumatic brain injury (TBI) and estimate the life-time savings in costs of care. SETTING/PARTICIPANTS: TBI patients (n = 3578/6043) admitted to all 75 specialist rehabilitation services in England 2010-2018. DESIGN: A multicenter cohort analysis of prospectively collated clinical data from the UK Rehabilitation Outcomes Collaborative national clinical database. MAIN MEASURES: Primary outcomes: (a) reduction in dependency (UK Functional Assessment Measure), (b) cost-efficiency, measured in time taken to offset rehabilitation costs by savings in costs of ongoing care estimated by the Northwick Park Dependency Scale/Care Needs Assessment (NPDS/NPCNA), and (c) estimated life-time savings. RESULTS: The mean age was 49 years (74% males). Including patients who remained in persistent vegetative state on discharge, the mean episode cost of rehabilitation was £42 894 (95% CI: £41 512, £44 235), which was offset within 18.2 months by NPCNA-estimated savings in ongoing care costs. The mean period life expectancy adjusted for TBI severity was 21.6 years, giving mean net life-time savings in care costs of £679 776/patient (95% CI: £635 972, £722 786). CONCLUSIONS: Specialist rehabilitation proved highly cost-efficient for severely disabled patients with TBI, despite their reduced life-span, potentially generating over £4 billion savings in the cost of ongoing care for this 8-year national cohort.

Development and validation of a casemix classification to predict costs of specialist palliative care provision across inpatient hospice, hospital and community settings in the UK: a study protocol.

INTRODUCTION: Provision of palliative care is inequitable with wide variations across conditions and settings in the UK. Lack of a standard way to classify by case complexity is one of the principle obstacles to addressing this. We aim to develop and validate a casemix classification to support the prediction of costs of specialist palliative care provision. METHODS AND ANALYSIS: Phase I: A cohort study to determine the variables and potential classes to be included in a casemix classification. Data are collected from clinicians in palliative care services across inpatient hospice, hospital and community settings on: patient demographics, potential complexity/casemix criteria and patient-level resource use. Cost predictors are derived using multivariate regression and then incorporated into a classification using classification and regression trees. Internal validation will be conducted by bootstrapping to quantify any optimism in the predictive performance (calibration and discrimination) of the developed classification. Phase II: A mixed-methods cohort study across settings for external validation of the classification developed in phase I. Patient and family caregiver data will be collected longitudinally on demographics, potential complexity/casemix criteria and patient-level resource use. This will be triangulated with data collected from clinicians on potential complexity/casemix criteria and patient-level resource use, and with qualitative interviews with patients and caregivers about care provision across difference settings. The classification will be refined on the basis of its performance in the validation data set. ETHICS AND DISSEMINATION: The study has been approved by the National Health Service Health Research Authority Research Ethics Committee. The results are expected to be disseminated in 2018 through papers for publication in major palliative care journals; policy briefs for clinicians, commissioning leads and policy makers; and lay summaries for patients and public. TRIAL REGISTRATION NUMBER: ISRCTN90752212.

Variations in the cost of formal and informal health care for patients with advanced chronic disease and refractory breathlessness: A cross-sectional secondary analysis.

BACKGROUND: Refractory breathlessness in advanced chronic disease leads to high levels of disability, anxiety and social isolation. These result in high health-resource use, although this is not quantified. AIMS: To measure the cost of care for patients with advanced disease and refractory breathlessness and to identify factors associated with high costs. DESIGN: A cross-sectional secondary analysis of data from a randomised controlled trial. SETTING/PARTICIPANTS: Patients with advanced chronic disease and refractory breathlessness recruited from three National Health Service hospitals and via general practitioners in South London. RESULTS: Of 105 patients recruited, the mean cost of formal care was £3253 (standard deviation £3652) for 3 months. The largest contributions to formal-care cost were hospital admissions (>60%), and palliative care contributed <1%. When informal care was included, the total cost increased by >250% to £11,507 (standard deviation £9911). Increased patient disability resulting from breathlessness was associated with high cost (£629 per unit increase in disability score; p = 0.006). Increased breathlessness on exertion and the presence of an informal carer were also significantly associated with high cost. Patients with chronic obstructive pulmonary disease tended to have higher healthcare costs than other patients. CONCLUSION: Informal carers contribute significantly to the care of patients with advanced disease and refractory breathlessness. Disability resulting from breathlessness is an important clinical cost driver. It is important for policy makers to support and acknowledge the contributions of informal carers. Further research is required to assess the clinical- and cost-effectiveness of palliative care interventions in reducing disability resulting from breathlessness in this patient group.

Measures to assess commonly experienced symptoms for people with dementia in long-term care settings: a systematic review.

BACKGROUND: High symptom burden is common in long-term care residents with dementia and results in distress and behavioral challenges if undetected. Physicians may have limited time to regularly examine all residents, particularly those unable to self-report, and may rely on reports from caregivers who are frequently in a good position to detect symptoms quickly. We aimed to identify proxy-completed assessment measures of symptoms experienced by people with dementia, and critically appraise the psychometric properties and applicability for use in long-term care settings by caregivers. METHODS: We searched Medline, EMBASE, PsycINFO, CINAHL and ASSIA from inception to 23 June 2015, supplemented by citation and reference searches. The search strategy used a combination of terms: dementia OR long-term care AND assessment AND symptoms (e.g. pain). Studies were included if they evaluated psychometric properties of proxy-completed symptom assessment measures for people with dementia in any setting or those of mixed cognitive abilities residing in long-term care settings. Measures were included if they did not require clinical training, and used proxy-observed behaviors to support assessment in verbally compromised people with dementia. Data were extracted on study setting and sample, measurement properties and psychometric properties. Measures were independently evaluated by two investigators using quality criteria for measurement properties, and evaluated for clinical applicability in long-term settings. RESULTS: Of the 19,942 studies identified, 40 studies evaluating 32 measures assessing pain (n = 12), oral health (n = 2), multiple neuropsychiatric symptoms (n = 2), depression (n = 8), anxiety (n = 2), psychological wellbeing (n = 4), and discomfort (n = 2) were included. The majority of studies (31/40) were conducted in long-term care settings although none of the neuropsychiatric or anxiety measures were validated in this setting. The pain assessments, PAINAD and PACSLAC had the strongest psychometric evidence. The oral health, discomfort, and three psychological wellbeing measures were validated in this setting but require further psychometric evaluation. Depression measures were poor at detecting depression in this population. All measures require further investigation into agreement, responsiveness and interpretability. CONCLUSIONS: Measures for pain are best developed for this population and setting. All other measures require further validation. A multi-symptom measure to support comprehensive assessment and monitoring in this population is required.

Public health and palliative care in 2015.

Palliative care is a public health concern, because the problems faced by patients and their families represent a substantial burden of illness and cost to the society that is likely to increase markedly in the future as the world's population continues to age. There are also inequities in access to palliative care and continued unmet need. There is evidence to support palliative care services, but not yet enough information on the cost-effectiveness of many specific treatments/interventions.

Patient self-assessment of hospital pain, mood and health-related quality of life in adults with sickle cell disease.

INTRODUCTION: Acute pain is a hallmark of sickle cell disease (SCD) for which frequent hospital admissions may be required, affecting the quality of life of patients. OBJECTIVES: To characterise the relationship between adult patient self-reported sickle cell pain, mood and quality of life during and after hospital admissions. DESIGN: Longitudinal study across three time-points. SETTING: Secondary care, single specialist sickle cell centre. PARTICIPANTS: 510 adult patients with SCD admitted to hospital daycare or inpatient units. OUTCOME MEASURES: Self-assessments of pain, mood and health-related quality of life with health utility (measured on the EQ-5D) on admission, before discharge and at 1-week postdischarge. RESULTS: Mood, general health and quality of life showed significant steady improvements with reduction of pain in patients with SCD on admission to hospital, before discharge and at 1-week follow-up (p<0.01). Health utility scores derived from the EQ-5D showed a negative association with pain in regression analysis over the three time-points. CONCLUSION: Examining health-related quality of life and health utility in relation to pain during hospital admissions is valuable in terms of targeting appropriate psychological interventions within the context of a multidisciplinary approach to managing sickle cell pain. This has implications for healthcare costs.