Economic evaluations of non-communicable diseases conducted in Sub-Saharan Africa: a critical review of data sources.

BACKGROUND: Policymakers in sub-Saharan Africa (SSA) face challenging decisions regarding the allocation of health resources. Economic evaluations can help decision makers to determine which health interventions should be funded and or included in their benefits package. A major problem is whether the evaluations incorporated data from sources that are reliable and relevant to the country of interest. We aimed to review the quality of the data sources used in all published economic evaluations for cardiovascular disease and diabetes in SSA. METHODS: We systematically searched selected databases for all published economic evaluations for CVD and diabetes in SSA. We modified a hierarchy of data sources and used a reference case to measure the adherence to reporting and methodological characteristics, and descriptively analysed author statements. RESULTS: From 7,297 articles retrieved from the search, we selected 35 for study inclusion. Most were modelled evaluations and almost all focused on pharmacological interventions. The studies adhered to the reporting standards but were less adherent to the methodological standards. The quality of data sources varied. The quality level of evidence in the data domains of resource use and costs were generally considered of high quality, with studies often sourcing information from reliable databases within the same jurisdiction. The authors of most studies referred to data sources in the discussion section of the publications highlighting the challenges of obtaining good quality and locally relevant data. CONCLUSIONS: The data sources in some domains are considered high quality but there remains a need to make substantial improvements in the methodological adherence and overall quality of data sources to provide evidence that is sufficiently robust to support decision making in SSA within the context of UHC and health benefits plans. Many SSA governments will need to strengthen and build their capacity to conduct economic evaluations of interventions and health technology assessment for improved priority setting. This capacity building includes enhancing local infrastructures for routine data production and management. If many of the policy makers are using economic evaluations to guide resource allocation, it is imperative that the evidence used is of the feasibly highest quality.

Dynamics of Patient-Based Benefit-Risk Assessment of Medicines in Chronic Diseases: A Systematic Review.

Background: A critical gap exits in understanding the dynamics of patient-based benefit-risk assessment (BRA) of medicines in chronic diseases during the disease journey. Purpose: To systematically review and synthesize current evidence on the changes of patients' preferences about the benefits and risks of medicines during their disease journey including the influence of disease duration and severity, and previous treatment experience. Methods: A systematic review of studies identified in PubMed and Embase, from inception to November 2020, was conducted in accordance with the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) statement. Articles were eligible if they analyzed adult patient-based BRA of medicines with a chronic disease, based on at least one of the pre-specified dimensions: disease severity, disease duration, or previous treatment experience. Results: A total of 26,228 articles were identified and 105 were eligible for inclusion. Of these, 85 detected a variation in patient-based BRA of medicines with at least one of the pre-specified criteria. Patients with higher disease severity and more treatment experience have increased risk tolerance. It remains inconclusive whether disease duration directly affects the relative importance of a patient's preference. Conclusion: Factors important for patients' BRA of their medicines during a chronic disease journey vary more with their clinical situation and previous treatment experience than with time since diagnosis. Due to the importance of these factors on patients' perspectives and potential impact on their decision-making and eventually their clinical outcomes, there is a need for more studies to assess the dynamics of patients' BRA in every disease.

Patient-based benefit-risk assessment of medicines: development, refinement, and validation of a content search strategy to retrieve relevant studies.

Introduction: Poor indexing and inconsistent use of terms and keywords may prevent efficient retrieval of studies on the patient-based benefit-risk assessment (BRA) of medicines. We aimed to develop and validate an objectively derived content search strategy containing generic search terms that can be adapted for any search for evidence on patient-based BRA of medicines for any therapeutic area. Methods: We used a robust multistep process to develop and validate the content search strategy: (1) we developed a bank of search terms derived from screening studies on patient-based BRA of medicines in various therapeutic areas, (2) we refined the proposed content search strategy through an iterative process of testing sensitivity and precision of search terms, and (3) we validated the final search strategy in PubMed by firstly using multiple sclerosis as a case condition and secondly computing its relative performance versus a published systematic review on patient-based BRA of medicines in rheumatoid arthritis. Results: We conceptualized a final search strategy to retrieve studies on patient-based BRA containing generic search terms grouped into two domains, namely the patient and the BRA of medicines (sensitivity 84%, specificity 99.4%, precision 20.7%). The relative performance of the content search strategy was 85.7% compared with a search from a published systematic review of patient preferences in the treatment of rheumatoid arthritis. We also developed a more extended filter, with a relative performance of 93.3% when compared with a search from a published systematic review of patient preferences in lung cancer.

Community-based integrated care versus hospital outpatient care for managing patients with complex type 2 diabetes: costing analysis.

Objective This study compared the cost of an integrated primary-secondary care general practitioner (GP)-based Beacon model with usual care at hospital outpatient departments (OPDs) for patients with complex type 2 diabetes. Methods A costing analysis was completed alongside a non-inferiority randomised control trial. Costs were calculated using information from accounting data and interviews with clinic managers. Two OPDs and three GP-based Beacon practices participated. In the Beacon practices, GPs with a special interest in advanced diabetes care worked with an endocrinologist and diabetes nurse educator to care for referred patients. The main outcome was incremental cost saving per patient course of treatment from a health system perspective. Uncertainty was characterised with probabilistic sensitivity analysis using Monte Carlo simulation. Results The Beacon model is cost saving: the incremental cost saving per patient was A$365 (95% confidence interval -A$901, A$55) and was cost saving in 93.7% of simulations. The key contributors to the variance in the cost saving per patient course of treatment were the mean number of patients seen per site and the number of additional presentations per course of treatment associated with the Beacon model. Conclusions Beacon clinics were less costly per patient course of treatment than usual care in hospital OPDs for equivalent clinical outcomes. Local contractual arrangements and potential variation in the operational cost structure are of significant consideration in determining the cost-efficiency of Beacon models. What is known about this topic? Despite the growing importance of achieving care quality within constrained budgets, there are few costing studies comparing clinically-equivalent hospital and community-based care models. What does this paper add? Costing analyses comparing hospital-based to GP-based health services require considerable effort and are complex. We show that GP-based Beacon clinics for patients with complex chronic disease can be less costly per patient course of treatment than usual care offered in hospital OPDs. What are the implications for practitioners? In addition to improving access and convenience for patients, transferring care from hospital to the community can reduce health system costs.

Disease modifying therapies for relapsing-remitting multiple sclerosis: Use and costs in Australia (1996-2019).

BACKGROUND: New disease modifying therapies (DMT) to control relapsing-remitting multiple sclerosis (RRMS) have been introduced to the market in the past few years and are now widely used in Australia. OBJECTIVE: To analyse the dispensed use of government subsidised RRMS DMTs in Australia from 1996 to 2019. METHODS: We obtained data of dispensed use of DMTs from the Australian Government's Pharmaceutical Benefits Scheme (PBS) administered by Medicare Australia. We measured use as defined daily dose (DDD) per 100,000 population per day. We obtained jurisdictional population data from the Australian Bureau of Statistics. RESULTS: Total DMT use increased by an average of 18% annually, from 2.4 (in 1996) to 69.9 DDD/100,000/day in 2019. Interferon ß1B was the most commonly used medicine between 1996 and 2000, Interferon ß1A between 2001 and 2014, and fingolimod subsequently. Among Australian states, Tasmania (the southernmost state) had the highest dispensed DMT use of 94.6 DDD/100,000/day in 2019. Concession beneficiaries under the Government's PBS had both lower use and cost per patient than general beneficiaries did. Fingolimod and ocrelizumab accounted for 55% of total expenditure on MS drug therapy in 2019. CONCLUSION: The use of oral DMTs might increasingly replace parenteral treatments in the near future. Given the current substantial government expenditure on oral DMTs, it will be imperative to examine the real world effectiveness of DMTs.

Duration of opioid use and association with socioeconomic status, daily dose and formulation: a two-decade population study in Queensland, Australia.

Background There is an association between the duration of prescription opioids use and an increased risk of serious harm, often unintentional. Objective (1) Describe the trends in duration of prescription opioids dispensing and, (2) determine the risk of long-term use (≥4 months) based on patients' socioeconomic status, daily dose in oral daily morphine milligram equivalent, and opioid formulation. Setting Residents of Queensland (2,827,727), Australia from the age 18 years and who were dispensed pharmaceutical opioids from 1 January 1997 to 31 December 2018. Method Retrospective, longitudinal population-based analysis using data obtained from the Monitoring of Drugs of Dependence system of the Monitored Medicines Unit of Queensland Health. Main outcome measure Contribution of socioeconomic status, and daily dose and opioid formulation (modified-release or immediate-release) to the risk of long-term opioid use. Results There was little difference between the number of patients dispensed opioids for ≥4 months and ≤3 months between 1997 and 2011. Thereafter, the number for those using opioids long-term increased. The highest risk of having opioids dispensed for ≥4 months were for patients in the lowest level of socioeconomic status (adjusted odds ratio 1.36; 95% CI, 1.34, 1.38), compared to people in the highest socioeconomic status areas, followed by the low-socioeconomic status areas, mid-socioeconomic status areas, and high-socioeconomic status areas respectively. The risk of being dispensed prescription opioids for ≥4 months significantly increased as the dose increased: adjusted odds ratio 1.73; 95% CI, 1.71, 1.75, adjusted odds ratio 1.89; 95% CI, 1.87, 1.92, and adjusted odds ratio 3.63; 95% CI, 3.58, 3.69 for the ≥20 to <50 oral daily morphine milligram equivalent, ≥50 to <100 oral daily morphine milligram equivalent and ≥100 oral daily morphine milligram equivalent dose categories, respectively. Conclusion Higher doses and living in a low socioeconomic status areas were associated with increased risk of long-term dispensing of opioid prescriptions.

Pharmaceutical opioids utilisation by dose, formulation, and socioeconomic status in Queensland, Australia: a population study over 22 years.

Background Prescription opioids are a central aspect of pain management and as the prevalence of pain is increasing so is the rate of use of prescription opioids. Increased opioid prescriptions increases the risk of deaths and morbidity. Objective To (a) describe the 22-year trend of prescription opioid dispensing in Queensland, (b) examine the effect of opioid dose, formulation and socioeconomic status on the number of prescriptions dispensed. Design/setting Retrospective analysis of data from the Monitoring of Drugs of Dependence system of the Monitored Medicines Unit of Queensland Health, Australia. Participants Queensland residents (3.3 million) from 18 years old dispensed 18.8 million opioid prescriptions from January 1997 to December 2018. Results Opioid prescriptions dispensed annually increased to over two million in 2018 from about 150,000 prescriptions in 1997. The number of prescriptions for modified-release formulations dispensed annually was three times higher compared to the immediate-release formulations. Oxycodone accounted for over 60% of prescriptions for pharmaceutical opioids since 2013. There was an increase in the number of prescriptions dispensed as socioeconomic status decreased and modified-release opioid formulations positively affects the pattern of dispensing. The highest increase in number of prescriptions dispensed (for all opioids) was observed among the high socioeconomic status (IRR = 1.25, 95% CI 1.25, 1.26). The disparities in the annual number of prescriptions across dose categories are wider in the modified-release than the immediate-release formulations. Conclusion The dispensing of opioids increased significantly in Queensland. There was a positive relationship between the increased dispensing of opioids and locations of lower socioeconomic status.

What do we need to know? Data sources to support evidence-based decisions using health technology assessment in Ghana.

BACKGROUND: Evidence-based decision-making for prioritising health is assisted by health technology assessment (HTA) to integrate data on effectiveness, costs and equity to support transparent decisions. Ghana is moving towards universal health coverage, facilitated mainly by the National Health Insurance Scheme (NHIS) established in 2003. The Government of Ghana is committed to institutionalising HTA for priority-setting. We aimed to identify and describe the sources of accessible data to support HTA in Ghana. METHODS: We identified and described data sources encompassing six main domains using an existing framework. The domains were epidemiology, clinical efficacy, costs, health service use and consumption, quality of life, and equity. We used existing knowledge, views of stakeholders, and searches of the literature and internet. RESULTS: The data sources for each of the six domains vary in extent and quality. Ghana has several large data sources to support HTA (e.g. Demographic Health Surveys) that have rigorous quality assurance processes. Few accessible data sources were available for costs and resource utilisation. The NHIS is a potentially rich source of data on resource use and costs but there are some limits on access. There are some data on equity but data on quality of life are limited. CONCLUSIONS: A small number of quality data sources are available in Ghana but there are some gaps with respect to HTA based on greater use of local and contextualised information. Although more data are becoming available for monitoring, challenges remain in terms of their usefulness for HTA, and some information may not be available in disaggregated form to enable specific analyses. We support recent initiatives for the routine collection of comprehensive and reliable data that is easily accessible for HTA users. A commitment to HTA will require concerted efforts to leverage existing data sources, for example, from the NHIS, and develop and maintain new data (e.g. local health utility estimates). It will be critical that an overarching strategic and mandatory approach to the collection and use of health information is developed for Ghana in parallel to, and informed by, the development of HTA approaches to support resource allocation decisions. The key to HTA is to use the best available data while being open about its limitations and the impact on uncertainty.

Health technology assessment capacity at national level in sub-Saharan Africa: an initial survey of stakeholders.

Background: Health technology assessment (HTA) is an effective tool for supporting priority setting (PS) in health. Stakeholder groups need to understand HTA appropriate to their role and to interpret and critique the evidence produced. We aimed to rapidly assess current health system priorities and policy areas of demand for HTA in Sub-Saharan Africa, and identify key gaps in data and skills to inform targeted capacity building. Methods: We revised an existing survey, delivered it to 357 participants, then analysed responses and explored key themes. Results: There were 51 respondents (14%, 30 full completions) across 14 countries. HTA was considered an important and valuable PS tool with a key role in the design of health benefits packages, clinical guideline development, and service improvement. Medicines were identified as a technology type that would especially benefit from the application of HTA. Using HTA to address safety issues (e.g. low-quality medicines) and value for money concerns was particularly highlighted. The perceived availability and accessibility of suitable local data to support HTA varied widely but was mostly considered inadequate and limited. Respondents also noted a need for training support in research methodology and data gathering. Conclusions: While important in raising awareness of HTA as a tool for PS, this study had a low response rate, and that respondents were self-selected. A more refined survey will be developed to support engagement strategies and capacity building.

Consumption of medicines used for gastric acid-related disorders in Australia and South Korea: a cross-country comparison.

PURPOSE: The study's aim was to compare the use of proton pump inhibitors (PPIs), histamine 2-receptor antagonists (H2RAs) and mucoprotective medicines (MPs) used for gastric acid-related disorders (GARD) in Australia and South Korea (Korea) from 2004 to 2017. METHODS: Prescription data for PPIs, H2RAs and MPs for Australian outpatients were extracted from the Australian Statistics on Medicines annual reports, with dose-specific and expenditure data obtained from Medicare. Similar data were obtained from Korean National Health Insurance Service claims data. We analysed the volume and expenditure of medicines use annually using the defined daily dose per 1,000 population per day. We calculated which medicines accounted for 90% of use and estimated the proportions of use for low- and high-dose PPIs. RESULTS: While total utilisation for GARD medicines increased over time in both countries, patterns of use differed. Overall, use was somewhat higher in Australia but increased more rapidly in Korea. PPIs were used more extensively in Australia, while more MPs and H2RAs were used in Korea. Expenditure and use of low-dose PPIs is escalating in Korea. CONCLUSION: There were substantial differences in the use of GARD medicines in Australia and Korea over 14 years. Both countries face similar challenges to promote rational medicines use and contain medical care costs. The discrepant prescribing patterns can be attributed to differences in healthcare systems, pharmaceutical policies and demographics. This study provides a baseline to influence more rational use of these medicines. It provides insight into medicines policies for other countries that face similar challenges.

Cost-effectiveness Analysis of Routine Screening Using Massively Parallel Sequencing for Maturity-Onset Diabetes of the Young in a Pediatric Diabetes Cohort: Reduced Health System Costs and Improved Patient Quality of Life.

OBJECTIVE: Maturity-onset diabetes of the young (MODY) is an autosomal dominant form of diabetes, with multiple causative genes. Some MODY subtypes can be treated with sulfonylureas instead of insulin, improving glycemic control, complication rates, quality of life (QoL), and costs. Using massively parallel sequencing (MPS), we recently determined the prevalence of pathogenic/likely pathogenic MODY variants in an Australian pediatric diabetes cohort. Here, these data are used to estimate cost-effectiveness of using MPS for MODY in all pediatric diabetes cases compared with standard practice (sequencing limited to individuals with specific clinical features). RESEARCH DESIGN AND METHODS: A Markov decision model was developed to estimate incremental costs and quality-adjusted life-years (QALYs) of MPS screening, modeled over 30 years. We used our observed prevalence of 2.14% compared with 0.7% for standard practice, based on published data. The probabilities and utility weightings of long-term diabetes complications were based on HbA1c and estimated from published data. A series of one-way sensitivity analyses were performed using the net monetary benefit framework. RESULTS: Routine MPS screening for MODY was more effective and less costly than standard care screening, with 26 QALYs gained and 1,016,000 AUD (782,000 USD) saved per 1,000 patients. Cost of screening was fully offset within 10 years. Routine MPS screening remained dominant until MODY prevalence fell to <1.1%. CONCLUSIONS: Routine MPS screening for MODY in the pediatric population with diabetes could reduce health system costs and improve patient QoL. Our results make a compelling argument for routine genetic screening in all children with presumed type 1 diabetes mellitus.

Using pharmacy management systems for research: survival outcomes for lenalidomide in multiple myeloma in the clinical setting.

Background Health records can be used to measure medicine use and health outcomes. The public subsidy of lenalidomide in Australia was based on two phase III trials showing improved survival. Objective To use hospital pharmacy information management systems to determine survival outcomes for lenalidomide as a second line treatment in relapsed or refractory multiple myeloma (RRMM) patients. Setting Five public hospitals in Queensland, Australia. Method We extracted data on medicine use and survival for RRMM patients planned to start lenalidomide from pharmacy management and pathology databases. Descriptive statistical analyses (Kaplan-Meier curves) were used to calculate overall survival. Main outcome measure Overall survival. Results There were 136 patients who received at least one lenalidomide dose and 2234 cycles were ordered. The median age was 69 years and 54% were male. Two lenalidomide containing protocols were considered: 90% of patients had lenalidomide plus dexamethasone; 18% had lenalidomide plus dexamethasone with cyclophosphamide. The median starting lenalidomide dose was 20 mg (range 4.3-25 mg) on days 1-21 of a 28-day cycle. Median time on treatment 9.4 months (range 0.5-71.7 months). Median overall survival was 45.4 months (range 12.0-70.5 months). Conclusion The median survival in our study compared favourably to clinical trials. Patients and clinicians can be reassured that outcomes in this clinical setting are as good as those observed in trials.

Impact of a general practitioner-led integrated model of care on the cost of potentially preventable diabetes-related hospitalisations.

AIM: To estimate potential savings for Australia's health care system through the implementation of an innovative Beacon model of care for patients with complex diabetes. METHODS: A prospective controlled trial was conducted comparing a multidisciplinary, community-based, integrated primary-secondary care diabetes service with usual care at a hospital diabetes outpatient clinic. We extracted patient hospitalisation data from the Queensland Hospital Admitted Patient Data Collection and used Australian Refined Diagnosis Related Groups to assign costs to potentially preventable hospitalisations for diabetes. RESULTS: 327 patients with complex diabetes referred by their general practitioner for specialist outpatient care were included in the analysis. The integrated model of care had potential for national cost savings of $132.5 million per year. CONCLUSIONS: The differences in hospitalisations attributable to better integrated primary/secondary care can yield large cost savings. Models such as the Beacon are highly relevant to current national health care reform initiatives to improve the continuity and efficiency of care for those with complex chronic disease in primary care.

Patterns in use and costs of conventional and biologic disease-modifying anti-rheumatic drugs in Australia.

OBJECTIVES: The aim of this study was to characterise the use and costs of subsidising conventional disease-modifying anti-rheumatic drugs (DMARDs) and biologic DMARDs in Australia from 2004-2014 through pharmaceutical benefits schemes. METHODS: Dispensing and expenditure data on conventional and biologic DMARDs were extracted from Medicare Australia and temporal trends were analysed. Medicine use was standardised in terms of the defined daily dose (DDD) per 1,000 population per day (DDD/1,000 population/day). RESULTS: Conventional and biologic DMARD use increased 74% over the study period (4.86 to 8.46 DDD/1,000 population/day; average annual increase 6.7%). Conventional DMARDs accounted for the vast majority of total use and increased 55% (4.81 to 7.43 DDD/1,000 population/day), while biologic DMARD use increased 1,784% (0.055 to 1.030 DDD/1,000 population/day). The most frequently used conventional DMARD was methotrexate (56% total conventional DMARD use) and use increased 95%. Hydroxychloroquine and leflunomide use increased marginally while sulfasalazine use declined 4.2%. Etanercept was the most commonly used biologic DMARD in 2004 and adalimumab in 2014. Conventional DMARD expenditure decreased 4.2% to AUD$33.3 million but biologic DMARD expenditure increased 2,089% to AUD$585.4 million. CONCLUSIONS: The use of conventional and biologic DMARDs increased substantially over a decade in Australia. Patterns of use of conventional DMARDs have changed, and costs have decreased. In contrast a significant escalation in both the use and cost of biologic DMARDs has occurred. Further research is required to address cost-effectiveness, regulation and quality use of these medicines in clinical practice.