Consolidated Health Economic Evaluation Reporting Standards 2022 (CHEERS 2022) statement: updated reporting guidance for health economic evaluations.

Health economic evaluations are comparative analyses of alternative courses of action in terms of their costs and consequences. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement, published in 2013, was created to ensure health economic evaluations are identifiable, interpretable, and useful for decision making. It was intended as guidance to help authors report accurately which health interventions were being compared and in what context, how the evaluation was undertaken, what the findings were, and other details that may aid readers and reviewers in interpretation and use of the study. The new CHEERS 2022 statement replaces previous CHEERS reporting guidance. It reflects the need for guidance that can be more easily applied to all types of health economic evaluation, new methods and developments in the field, as well as the increased role of stakeholder involvement including patients and the public. It is also broadly applicable to any form of intervention intended to improve the health of individuals or the population, whether simple or complex, and without regard to context (such as health care, public health, education, social care, etc). This summary article presents the new CHEERS 2022 28-item checklist and recommendations for each item. The CHEERS 2022 statement is primarily intended for researchers reporting economic evaluations for peer reviewed journals as well as the peer reviewers and editors assessing them for publication. However, we anticipate familiarity with reporting requirements will be useful for analysts when planning studies. It may also be useful for health technology assessment bodies seeking guidance on reporting, as there is an increasing emphasis on transparency in decision making.

Patient Stakeholder Versus Physician Preferences Regarding Amyloid PET Testing.

BACKGROUND: Patient and caregiver perspectives on amyloid positron emission tomography (PET) use are largely unexplored, particularly as compared with clinician views. METHODS: We surveyed clinicians, patients, caregivers, and dementia advocates on topics relating to an evidence-based guideline on amyloid PET use. Topic importance was rated on a 9-point scale. Patient stakeholder and clinician views were compared using the Mann-Whitney U test. RESULTS: Patient representatives (n=107) rated all survey topics as equal to or more important than clinicians (n=114) except 1 item discussing potential harms of false-positive diagnoses. Differences between patient representative and clinician populations were greatest when comparing the competing values of false-positive and false-negative diagnoses and the value of testing asymptomatic individuals. CONCLUSIONS: Patients and caregivers emphasized the importance of having a dementia diagnosis and placed more value on testing and outcomes for asymptomatic populations than clinicians. This underscores the importance of research investigating the effect of amyloid PET results on asymptomatic individuals and the need for amyloid PET ordering and disclosure standards.

Understanding systemic lupus erythematosus patients' desired outcomes and their perceptions of the risks and benefits of using corticosteroids.

Introduction The use of corticosteroids in systemic lupus erythematosus (SLE) patients requires difficult trade-offs between efficacy and risk of toxicity. This qualitative study examined SLE patients' most desired outcomes and their concerns with corticosteroid use in SLE treatment. Methods SLE patients with current/past experience with using corticosteroids were recruited from the clinics at the Johns Hopkins Lupus Center and the University of Maryland Medical Center. Five in-depth interviews ( N = 5) and four focus groups ( N = 15) were conducted during which discussions were transcribed and analyzed based on a grounded theory approach. Results We identified five major themes describing SLE patients' most desired outcomes: reduction in flares, maintenance of normal activities, minimization of treatment side effects, prevention of future organ damage, and finding a cure. Further, SLE patients reported these primary concerns with the adverse effects of corticosteroids: weight gain, organ damage (particularly bone-related damage), mood swings/irritability, sleep disturbances, and dental issues. Patients appeared to be more concerned with adverse effects that immediately affected their day-to-day lives. Conclusion Knowledge gained during this study better informs how patients view the benefits and risks of corticosteroids. This can facilitate discussions between physicians and patients as they work together to determine the appropriate use of corticosteroids.

Comparative analysis of various prognostic nodal factors, adjuvant chemotherapy and survival among stage III colon cancer patients over 65 years: an analysis using surveillance, epidemiology and end results (SEER)-Medicare data.

AIM: The prognostic effects of chemotherapy and various lymph node measures [positive nodes, total node count and the positive lymph node ratio (PLNR)] have been established. It is unknown whether the cancer-specific survival benefit of chemotherapy differs across these nodal prognostic categories. METHOD: This retrospective analysis of linked Surveillance, Epidemiology and End Results (SEER) data and Medicare data (SEER-Medicare)included patients ≥ 65 years of age with a diagnosis of stage III colon cancer between 1997 and 2002. We grouped patients according to the number of positive nodes (N1 and N2), total node count (≥ 12 and < 12 total nodes) and PLNR (below the 75th percentile and at least at the 75th percentile of the PLNR). The end point was colon cancer-specific mortality. RESULTS: Fifty-one per cent (3701) of the 7263 patients received adjuvant therapy during the time period 1997-2002. The mean (standard deviation) number of total nodes examined was 13 (9) and the number of positive nodes identified was 3 (3). Patients with N2 disease, < 12 total nodes examined and a high PLNR had a worse survival at 2, 3 and 5 years following colectomy. Utilization of chemotherapy demonstrated a colon cancer-specific survival benefit (hazard ratio at median follow up = 0.7; P < 0.001) that was consistent and statistically significant across the three nodal prognostic categories examined. CONCLUSION: The benefit of chemotherapy did not vary based on N stage, total node count or PLNR. The results favour a broad-based approach towards increasing the chemotherapy treatment rates in stage III patients of ≥ 65 years of age, rather than an approach that targets clinical subgroups.

What works when: mapping patient and stakeholder engagement methods along the ten-step continuum framework.

Aim: This study provides a recommended 'patient engagement translation table' that identifies evidence-based methods for meaningful patient engagement along a ten-step framework for continuous engagement. Materials & methods: We used a mixed methods research design to collect data on preferred engagement methods, including an environmental scan of available literature, interviews and focus groups with patient-centered outcomes research stakeholders to match methods with research steps and a modified Delphi process with subject matter experts to create the final translation table. Results: Evidence-based engagement methods included community partnerships, focus groups, interviews, meetings, sharing print materials, social media, storytelling, surveys and including patients as research team members. Conclusion: Our recommended patient engagement translation table is designed to assist investigators in determining appropriate engagement methods for meaningful interactions with stakeholders.

The association between thiazolidinediones and hospitalisation for fracture in type 2 diabetic patients: a Taiwanese population-based nested case-control study.

AIMS/HYPOTHESIS: Evidence from the USA has emerged that thiazolidinediones may have a negative effect on the skeleton and increase the risk of fracture, but the association between thiazolidinediones use and fractures has not been evaluated in an Asian population. Using the 2000-2005 Taiwan National Health Insurance claims database, this Taiwanese population-based nested case-control study explored the association between thiazolidinediones use and hospitalisation for bone fracture in type 2 diabetic patients. METHODS: In the study cohort of type 2 diabetic patients, we identified 18,003 patients with fracture and 90,015 matched controls. Multivariable conditional logistic regressions were used to estimate the association between exposure to thiazolidinediones and fractures. Duration of thiazolidinediones use was defined on the basis of cumulative days of exposure to thiazolidinediones during the year prior to the index date, i.e. <30 days, 30 to 180 days and >180 days. RESULTS: More type 2 diabetic patients with fractures than controls used thiazolidinediones (fractures 5.99% vs control 4.06%). Thiazolidinediones use was associated with hospitalisation for fracture and the association was stronger with longer term exposure to thiazolidinediones (<30 days OR 1.32 [95% CI 1.09-1.54], p = 0.005; 30-180 days 1.42 [1.24-1.62], p < 0.0001; and >180 days 1.54 [1.37-1.74], p < 0.0001). This dose-response relationship was significantly evident in women (<30 days, 1.20 [0.93-1.55], p = 0.17; 30-180 days, 1.57 [1.32-1.86], p < 0.0001; and >180 days, 1.76 [1.52-2.04], p < 0.0001), but not in men. CONCLUSIONS/INTERPRETATION: Long-term exposure of type 2 diabetic patients to thiazolidinediones was associated with higher odds of fractures among women without a significant increase in odds of fractures among men.

Pulmonary artery intimal injury associated with blunt trauma.

In recent years, intimal injuries to the aorta (minimal aortic injuries) have been diagnosed more frequently. We report the first case of pulmonary artery intimal injury in the setting of blunt trauma. We propose a number of theories regarding the pathogenesis, outcome, and treatment of pulmonary artery intimal injuries, drawing inferences from aortic intimal injuries. We conclude with a discussion on differentiating pulmonary artery intimal injury from the more common (but still rare) pulmonary artery dissection, using our case as an example.

The direct medical costs of alpha(1)-antitrypsin deficiency.

BACKGROUND: For individuals with emphysema because of severe alpha(1)-antitrypsin deficiency, specific therapy called IV augmentation therapy has been available since 1989. Such therapy consists of IV infusion of pooled human plasma alpha(1)-antiprotease. METHODS: To assess the direct medical costs of having alpha(1)-antitrypsin deficiency, the current study surveyed members of the Alpha One Foundation Registry for Individuals With alpha(1)-Antitrypsin Deficiency regarding their annual expenditures for treatment of this disease. Data regarding demographic features, alpha(1)-antitrypsin status, and health-resource utilization were collected from a self-administered questionnaire. Respondents were asked to provide total health-care expenditures, but costs by specific items of care (eg, drugs, physician visits, etc) were not available. RESULTS: Mean annual cost estimates were higher for PI*ZZ-phenotype individuals ($30,948, n = 292) than for non-PI*ZZ-phenotype individuals ($20,673, n = 53; p = 0.049). Among PI*ZZ-phenotype individuals, self-reported costs of health-care services were further analyzed for those 288 individuals whose alpha(1)-antiprotease use status was reported. For the 185 current alpha(1)-antiprotease users, the mean annual cost was $40,123 (median, $36,000). CONCLUSIONS: Annual health-care expenditures by individuals with alpha(1)-antitrypsin deficiency are very high, whether or not they are currently receiving augmentation therapy. Augmentation therapy adds substantial costs, especially for heavier individuals who are receiving weekly infusions.

Emerging standardization in pharmacoeconomics.

In this study, we reviewed pharmacoeconomic guidelines from the United Kingdom, Spain, Italy, Australia, Canada, and the United States to determine areas of emerging standardization. We examined the published literature, publication guidelines of major health journals, and published and unpublished recommendations from various task forces and conferences on related topics. The review revealed several general principles for which there was consensus across guidelines. These common features included the importance of using and reporting transparent methods so that readers can easily understand what calculations are being performed on which data elements, minimizing bias, and providing justification for the methods and assumptions used. Differences were detected across guidelines on the following topics: type of pharmacoeconomic assessment, perspective, comparators and data sources, data analysis, cost analysis, future cost analysis, outcomes assessment, modeling, time horizon, discounting, disclosure, and generalizability. Evolution of economic guidelines hinges on whether the primary goal is to increase the consistency or increase the validity of economic assessments. Some balance between these two objectives is desirable.

Indirect cost of HIV infection in England.

BACKGROUND: Few studies have estimated the indirect costs of care for HIV infection in England by stage of infection at a population level. OBJECTIVE: This study estimated annual indirect costs of the HIV epidemic in England in 1997-1998 from both a public-sector and societal perspective. METHODS: Service costs for HIV-infected individuals were indexed to 1997-1998 English prices. Average annual indirect costs included the costs of statutory, community, and informal services; disability payments; and lost economic productivity by stage of HIV infection. Disability payments were excluded from the societal perspective, whereas the degree of lost economic productivity was varied for the sensitivity analyses. Total average annual indirect costs by stage of HIV infection were calculated, as were population-based costs by stage of HIV infection and overall population costs. RESULTS: Annual indirect costs from the public-sector and societal perspectives, respectively, ranged from pound sterling 3169 (dollars 5252) to pound sterling 3931 (dollars 6515) per person-year for asymptomatic individuals, pound sterling 5302 (dollars 8787) to pound sterling 7929 (dollars 13,140) for patients with symptomatic non-AIDS, and pound sterling 9956 (dollars 16,499) to pound sterling 21,014 (dollars 34,825) for patients with AIDS. Estimated population-based indirect costs from the public-sector perspective varied between pound sterling 109 million (dollars 181 million) and pound sterling 145 million (dollars 241 million) for 1997-1998, respectively, comprising between 58% and 124% of direct treatment costs for triple drug therapy in England during 1997. From the societal perspective, estimated population-based costs varied between pound sterling 84 million (dollars 138 million) and pound sterling 119 million (dollars 198 million) in 1997-1998, comprising between 45% and 102% of direct treatment costs and cost of care, respectively, during 1997. CONCLUSIONS: Average indirect costs increase as HIV-infected individuals' illness progresses. Whether one takes a public-sector or societal perspective, indirect costs add a considerable amount to the cost of delivering health care to HIV-infected individuals. Both direct and indirect costs, when obtainable, should be used to assess the economic consequences of HIV infection and treatment interventions.

Selection of clinical, patient-reported, and economic end points in acute exacerbation of chronic bronchitis.

BACKGROUND: Acute exacerbation of chronic bronchitis (AECB) places tremendous burden on patients, providers, employers, and health care systems. OBJECTIVE: The purpose of this paper is to (1) review the clinical, patient-reported, and economic measures used to evaluate disease burden and treatment effectiveness in AECB in clinical trials and (2) propose a guide for selecting study end points in AECB that will help capture all the relevant disease outcomes. METHODS: Two literature searches of the PubMed database were conducted to identify studies of clinical trials in bronchitis and evaluate the clinical, patient-reported, and economic end points used in these studies. RESULTS: Previous studies have focused primarily on clinician-assessed outcomes, which do not capture the full impact of AECB on patients' lives. Reporting mechanisms for most end points have been inconsistent, limiting the ability to compare information or interpret differences. Previous studies have given limited attention to patient-reported outcomes and the economic implications of AECB. Patient-reported outcomes such as speed of symptom relief and work productivity are important parameters for assessing treatment effectiveness and provide practical information for treatment evaluation. CONCLUSIONS: Additional research is needed to develop, examine, and validate patient-reported outcomes and the indirect costs of AECB. Measuring the relevant clinical, economic, and patient-reported outcomes in AECB patients using standardized methods may lead to a clearer understanding of the disease burden and the role, effectiveness, and cost-effectiveness of antibiotic treatment.

Cost effectiveness of abciximab during routine medical practice.

OBJECTIVE: To estimate the cost per ischaemic event (death, nonfatal myocardial infarction, subsequent revascularisation procedure) avoided at 6 months in high risk patients undergoing coronary revascularisation treated with abciximab during routine medical care. DESIGN: Retrospective, matched cohort design. SETTING: University teaching hospital. PATIENTS: 62 abciximab-treated patients and 62 patients not treated with abciximab with high risk coronary lesions were matched according to gender, hyperlipidaemia, diabetes mellitus and stenting. MAIN OUTCOME MEASURES: Using a third-party payer's perspective, an incremental cost-effectiveness ratio (ICER) was computed as the cost per ischaemic event avoided over 6 months. Fieller's theorem was used to estimate confidence sets and confidence ellipses were generated to visually represent the variability in the data. RESULTS: At 6 months, abciximab-treated patients experienced an approximately 40% lower rate of ischaemic events (16.1 vs 27.4%; p = 0.128). The point estimate of the ICER was $US21,789 per ischaemic event avoided. Fieller's theorem resulted in a 95% confidence set consisting of 2 half-lines (-infinity to -$US115,461) and ($US391 to +infinity), reflecting the finding that the ICER denominator was not significantly different from zero at the p = 0.05 level. CONCLUSIONS: In high risk patients treated during routine care, the effectiveness of abciximab was consistent with efficacy rates from clinical trials. However, abciximab-treated patients remained approximately $US2400 more costly at 6 months.

Pharmacy benefit management, cost-effectiveness analysis and drug formulary decisions.

Pharmacy benefit management companies (PBMs) have evolved over the past decade in response to the increased demand for health care cost containment. Their activities include the implementation of drug formularies and the negotiation of rebates from manufacturers. Our analysis of this industry is based on interviews and materials provided by the top five ranked PBM companies which account for over 80% of beneficiaries covered within formulary plans. The formularies of these companies are relatively inclusive, but they are becoming more restrictive over time. At present the use of cost-effectiveness (C-E) studies in the formulary decisions of PBMs has been limited. In this regard, the surveyed PBMs emphasized that most C-E studies have not compared therapeutic substitutes in populations with characteristics that are similar to those of their clients. Pharmacy benefit management companies also have had strong incentives to focus narrowly on drug costs because they typically manage drug benefits on a "carved-out" basis. However, PBMs anticipate a growing future role in the integrated management of patient care (disease management) for certain high cost chronic diseases and conditions. All of the leading firms we surveyed have disease management programs in development. The importance of C-E studies to PBM decisions is expected to increase significantly as disease management programs are implemented. The data infrastructure inherent to the PBM industry and the increasing number of employees with advanced training in pharmacoeconomics will permit firms to perform their own internal C-E studies. They are also establishing various alliances and joint ventures with drug manufacturers, health maintenance organizations, and academic institutions to perform these analyses. The leading PBMs tend to favor active participation in the development of methodological approaches to C-E studies over government regulations such as those proposed by the FDA in 1995.

Direct health care costs for treatment of diabetes mellitus and hypertension in an IPA-group-model HMO.

The differences in direct health care costs and use between HMO enrollees with both diabetes mellitus and hypertension and enrollees with either disease alone were studied. Two years' worth of medical and pharmacy claims data from a hybrid (independent practice association and group)-model HMO were evaluated. Diagnoses were determined from medical claims data and cross-referenced with prescription information from pharmacy claims data. Aggregate costs associated with each disease, including pharmacy costs, costs of physician office visits, and laboratory costs, were compiled. Comparisons were made of all costs (any cost incurred by the health plan for the member, regardless of disease) and disease-specific costs. The frequency of comorbid conditions was identified. A total of 6195 patients (670 with diabetes and hypertension, 1756 with diabetes alone, and 3769 with hypertension alone) were assessed. Patients with both diseases incurred much higher costs per year than patients with diabetes or hypertension alone (mean costs, $13,446, $8,493, and $8,424, respectively). Hospitalization costs contributed the greatest amount to total costs, while emergency room costs contributed the least. Disease-specific costs for diabetes and hypertension represented less than one quarter of total health care costs per patient. Average disease-specific costs were highest for patients with both diseases ($2,955), followed by costs for patients with hypertension alone ($1,803) and patients with diabetes alone ($689). The percentage spent on prescriptions was much higher for disease-specific costs than for total costs. The three most common comorbid conditions were dyslipidemia, coronary artery disease, and chronic obstructive pulmonary disease, with the frequency of cerebrovascular disease and myocardial infarction more than double in patients with diabetes and hypertension compared with patients with either disease alone. The cost of care for a patient with both diabetes and hypertension, although not double that for a patient with diabetes or hypertension alone, was higher than the cost of treating either disease.

The economic burden of skeletal-related events among elderly men with metastatic prostate cancer.

BACKGROUND AND OBJECTIVE: Advanced prostate cancer patients with bone metastasis are predisposed to skeletal complications termed skeletal-related events (SREs). There is limited information available on Medicare costs associated with treating SREs. The objective of this study was to ascertain SRE-related costs among older men with metastatic prostate cancer in the US. METHODS: We analysed patients aged 66 years or older who were diagnosed with incident stage IV (M1) prostate cancer between 2000 and 2007 from the linked Surveillance, Epidemiology and End Results (SEER)-Medicare dataset. A propensity score for the incidence of an SRE was estimated using a logistic regression model including demographic and clinical baseline variables. Patients with SREs (cases) were matched to patients without SREs (controls) based on the propensity score, length of follow-up (i.e. date of prostate cancer diagnosis to last date of observation) and death. Health resource utilization cost differences between cases and controls over time were compared using generalized linear models. Healthcare costs were examined by type of SRE (pathological fracture only, pathological fracture with concurrent surgery, spinal cord compression only, spinal cord compression with concurrent surgery, and bone surgery only) and by source of care (inpatient, physician/non-institutional provider, skilled nursing facility, outpatient and hospice). All costs were adjusted to 2009 US dollars, using the medical care component of the Consumer Price Index. RESULTS: Application of the inclusion criteria resulted in 1,131 metastatic prostate cancer patients with SREs and 6,067 patients without SREs during follow-up. The average age of the sample was 79 years, and 14 % were African American. A total of 928 patients with SREs were matched to 928 patients without SREs. The average health care utilization cost of patients with SREs was US$29,696 (95 % confidence interval [CI] US$24,730-US$34,662) higher than that of the controls. The most expensive SRE group was spinal cord compression with concurrent surgery (US$82,868: 95 % CI US$67,472-US$98,264) followed by bone surgery only (US$37,496: 95 % CI US$29,684-US$45,308), pathological fracture with concurrent surgery (US$34,169: 95 % CI US$25,837-US$ 42,501), spinal cord compression only (US$25,793: 95 % CI US$20,933-US$30,653) and pathological fracture only (US$14,649: 95 % CI US$6,537-US$22,761). The largest cost difference by source of care was observed for hospitalizations (p < 0.01). CONCLUSION: Metastatic prostate cancer patients with SREs incur higher costs compared to similar patients without SREs. SRE costs among older stage IV (M1) prostate cancer patients vary by SRE type, with spinal cord compression and concurrent surgery costing at least twice as much as other SREs.

Clinic visits and prescribing patterns among Veterans Affairs Maryland Health Care System dementia patients.

OBJECTIVE: Our objective was to determine how patient demographics and outpatient referrals to specialized dementia (DEM) or mental health (MH) clinics influence receipt of anti-dementia (AD), antidepressant (ADEP), antipsychotic (APSY) and sedative-hypnotic (SEDH) medications among veterans with dementia. DESIGN: Retrospective, cross-sectional observational study. SETTING: Veterans Affairs Maryland Health Care System (VAMHCS). PARTICIPANTS: Veterans aged ≥ 60 years with Alzheimer's or related dementia diagnosis after 1999 with minimum of one-year follow-up or death were included. MEASUREMENTS: Retrospective analysis of VAMHCS electronic medical records were used to determine predictors of AD, ADEP, APSY, and SEDH prescribing using logistic regression models that examined visits to DEM or MH clinics, patient age, follow-up time, race/ethnicity and marital status. RESULTS: Among 1209 veterans with average follow-up of 3.2 (SD 1.9) years, 36% percent had MH visits, 38% had DEM visits and 19% visited both clinics. DEM visits were associated with AD and ADEP but not APSY medication receipt (OR(AD:DEM) = 1.47, 95% CI = (1.052, 2.051); OR(ADEP:DEM) = 1.66, 95% CI = (1.193, 2.302); OR(APSY:DEM) = 1.35, 95% CI = (0.941, 1.929)). MH visit was associated with ADEP and APSY medication receipt (OR(AD:MH)\ = 1.16, 95% CI = (0.821, 1.631); OR(ADEP:MH) = 2.83, 95% CI = (2.005, 4.005); OR (APSY:MH) = 4.41, 95% CI = (3.109, 6.255)). CONCLUSION: In the VAMHCS dementia population, visits to DEM or MH specialty clinics increase the odds of receiving AD, ADEP, and APSY medications.