Making Informed Decisions:the Value of Testing Strategies in Healthcare

In this thesis, I cover many aspects related to the economics of testing strategies in healthcare, from the organization of screening tests to the use of diagnostics to combat antimicrobial resistance. It is important to consider the testing infrastructure: where should testing take place, close to the patient or in specialized laboratories; who should perform the test and how are these health professionals organized; and what value do the test results have for public health and how are these data shared? These are some of the issues to be considered when deciding when to implement or reimburse tests. From a cost-effectiveness perspective, the underlying clinical data should be sufficient to compare the testing strategy to other health technologies, by using generalizable health outcomes, such as quality-adjusted life years, and by using sufficiently long time hori¬zons. In a cost-effectiveness analysis, the costs for society are related to the clinical benefits for patients, but for microbiological tests, the clinical value is broader than that, especially if tests can identify specific pathogens. The collected data can be used to make public health decisions, for example by updating treatment guidelines for infectious disease and by responding to antimicrobial resistance and potentially pandemic pathogens

Economic evaluation of orphan drug Lutetium-Octreotate vs. Octreotide long-acting release for patients with an advanced midgut neuroendocrine tumour in the Netherlands

OBJECTIVES: Multiple studies showed positive effects of Lutetium-Octreotate (LO) treatment in neuroendocrine tumours. LO has been used in the Netherlands since the 1980s and recently received the orphan status shortly after the acquisition by Novartis. Since then, the official list price has increased sixfold. From a value-based pricing perspective, we analysed the impact of the increase in price on the incremental cost-effectiveness ratio (ICER) of LO treatment compared to optimal best supportive care, a high dose of Octreotide long-acting release (O-LAR), using the clinical data of the NETTER-1 trial. METHODS: A Markov model was developed to evaluate the costs per quality-adjusted life-year (QALY) for LO treatment compared to O-LAR from the healthcare perspective. A scenario analysis was conducted to compare the cost-effectiveness with the initial and increased price level of the LO-treatment. RESULTS: At the increased price level, the cost-effectiveness analysis rendered a deterministic ICER of €53,500 per QALY, while at the initial pricing, the ICER was €19,000 per QALY. The probabilistic sensitivity analysis (PSA) showed that LO had a high probability of being cost-effective at both the increased and initial price level, considering a cost-effectiveness threshold of €80,000. CONCLUSIONS: Even at the increased price level, LO treatment can still be considered cost-effective using the applicable Dutch willingness-to-pay threshold of 80,000 euro per QALY. Considering the public scrutiny in relation to this price increase, these outcomes raise the question whether traditional cost-effectiveness methods are sufficient in fully capturing the societal acceptance of prices of new medicines. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s10198-021-01303-2

Health economic evaluation of current vaccination strategies and new vaccines against tuberculosis:a systematic review

Introduction: Bacillus Calmette-Guerin (BCG) is the only licensed vaccine for tuberculosis, but its effectiveness is limited and varies by age. New candidate vaccines are currently being investigated. In response to the declining incidence of TB, practices relating to BCG vaccination have changed in various countries in recent years. A valid cost-effectiveness study is therefore needed in order to assist decision-makers in the implementation of cost-effective strategies for BCG vaccination. Areas covered: Studies involving economic evaluations of BCG vaccination were reviewed in order to present current findings concerning a range of BCG vaccination strategies in a variety of regions, target populations, and vaccine types. The Quality of Health Economic Studies (QHES) instrument was used to assess the quality of the studies included in the analysis. Expert opinion: Most of the studies showed a favorable economic profile of BCG vaccination. Selective strategies seem the most cost-effective option for low-incidence areas. Varying results on revaccination strategies did not lead to any conclusive finding on the cost-effectiveness of the strategies. A novel vaccine - either a BCG replacement or booster vaccine that provides better protection, especially in adults - has the potential to enhance the cost-effectiveness of vaccinating against tuberculosis

The impact of patent expiry on drug prices:insights from the Dutch market

Background: Currently literature on the impact of patent expiry on drug prices is lacking. Objective: To determine the impact of patent expiration and generic entry on drug prices in the Netherlands. Methods: Prescription and price data from 1999 up to and including December 2016 were collected from two national databases. The overall price ratio of drugs prices up to 48 months after patent expiration was compared to the price in the month before expiry. Sub-analyses were performed to provide insights in generic uptake, length of market exclusivity and price development for originators and generics separately. Results: In total 250 drugs faced patent expiration during the study period. Forty-eight months after patent expiration the median price ratio decreased to 0.59 (IQR = 0.23-0.86) compared to the month prior patent expiry. Major differences in price developments were observed depending on the level of revenue prior to patent expiration and the time of patent expiration with ratios ranging from 0.08 (IQR = 0.07-0.16) to 0.81 (IQR = 0.62-0.97). Prior to patent expiry, the price decreased by 2.3% annually while having market exclusivity for 11.3 years on average. Conclusion: This study showed that the median drug price after patent expiration decreased by 41% after 4 years. The results of this study can be used to provide more reliable estimates on drug prices over its lifecycle and can be implemented in economic evaluations to inform the cost-effectiveness and long-term budget impact of new drugs

School health in Europe:a review of workforce expenditure across five countries

Background: Most European countries have implemented a form of school health services (SHS) to provide young children and adolescents with various types of healthcare. No estimations on SHS expenditure for European countries have been published until now. We estimated SHS workforce expenditure in Europe, expected to serve as the main driver of school healthcare costs. Methods: Using two networks of experts on healthcare provision for children we contacted various country representatives to provide data on the number of professionals working in SHS and salaries. These data were used, together with publicly available data, to estimate annual SHS workforce expenditure on the national level. Results: We received sufficient data for five European countries, and estimated the SHS workforce expenditure. Nurses were the most widely reported professionals working in this field, followed by doctors and psychologists. Our SHS expenditure estimations ranged from 43,000 for Estonia to 195,300 in Norway (per 1000 pupils). For Norway, Estonia, Finland and Iceland, school nurses were the main drivers of SHS expenditure, mainly due to their large numbers, while in Austria, school doctors played the largest role in SHS expenditure. Conclusions: We estimated the spending on SHS workforce for five European countries, which comprises relatively minor parts of total healthcare spending (0.16 to 0.69%). Many questions regarding SHS spending in Europe remain, due to a general lack of data on national levels

Costs of two vancomycin-resistant enterococci outbreaks in an academic hospital

Objective: In early 2017, the University Medical Center Groningen, the Netherlands, had an outbreak of 2 strains of vancomycin-resistant enterococci (VRE) that spread to various wards. In the summer of 2018, the hospital was again hit by a VRE outbreak, which was detected and controlled early. However, during both outbreaks, fewer patients were admitted to the hospital and various costs were incurred. We quantified the costs of the 2017 and 2018 VRE outbreaks. Design: Using data from various sources in the hospital and interviews, we identified and quantified the costs of the 2 outbreaks, resulting from tests, closed beds (opportunity costs), cleaning, additional personnel, and patient isolation. Setting: The University Medical Center Groningen, an academic hospital in the Netherlands. Results: The total costs associated with the 2017 outbreak were estimated to be €335,278 (US $356,826); the total costs associated with the 2018 outbreak were estimated at €149,025 (US$158,602). Conclusions: The main drivers of the costs were the opportunity costs due to the reduction in admitted patients, testing costs, and cleaning costs. Although the second outbreak was considerably shorter, the costs per day were similar to those of the first outbreak. Major investments are associated with the VRE control measures, and an outbreak of VRE can lead to considerable costs for a hospital. Aggressively screening and isolating patients who may be involved in an outbreak of VRE may reduce the overall costs and improve the continuity of care within the hospital.</p

Economic Analyses of Respiratory Tract Infection Diagnostics:A Systematic Review

BACKGROUND: Diagnostic testing for respiratory tract infections is a tool to manage the current COVID-19 pandemic, as well as the rising incidence of antimicrobial resistance. At the same time, new European regulations for market entry of in vitro diagnostics, in the form of the in vitro diagnostic regulation, may lead to more clinical evidence supporting health-economic analyses. OBJECTIVE: The objective of this systematic review was to review the methods used in economic evaluations of applied diagnostic techniques, for all patients seeking care for infectious diseases of the respiratory tract (such as pneumonia, pulmonary tuberculosis, influenza, sinusitis, pharyngitis, sore throats and general respiratory tract infections). METHODS: Following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, articles from three large databases of scientific literature were included (Scopus, Web of Science and PubMed) for the period January 2000 to May 2020. RESULTS: A total of 70 economic analyses are included, most of which use decision tree modelling for diagnostic testing for respiratory tract infections in the community-care setting. Many studies do not incorporate a generally comparable clinical outcome in their cost-effectiveness analysis: fewer than half the studies (33/70) used generalisable outcomes such as quality-adjusted life-years. Other papers consider outcomes related to the accuracy of the test or outcomes related to the prescribed treatment. The time horizons of the studies generally are limited. CONCLUSIONS: The methods to economically assess diagnostic tests for respiratory tract infections vary and would benefit from clear recommendations from policy makers on the assessed time horizon and outcomes used. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s40273-021-01054-1

Health-economic modelling of infectious disease diagnostics: current approaches and future opportunities

Objectives: Antimicrobial resistance (AMR) is a public health threat; infections with resistant organisms are estimated to cause over 650.000 infections and over 30.000 deaths in Europe. AMR is associated with antibiotic consumption: appropriate prescribing of antibiotics is key in combating AMR. To fight this threat, it has been suggested that point-of-care diagnostics to inform antibiotics prescribing are an important tool in reducing antibiotics prescriptions. With the objective of knowing the state of the art, we reviewed diagnostic cost-effectiveness analyses (CEAs) for infectious disease, focussing on model types, effects on prescriptions and AMR.  Methods: We searched the literature comprehensively through the PUBMED, Web of Science and EMBASE databases, as well as grey literature for the period 2000-2018. We included economic evaluations for diagnostic strategies for infectious disease in all geographic areas. Data extraction was based on the Consolidated Health Economic Evaluation Reporting Standards statement, with an additional focus on the modelling type and inclusion of AMR.Results: In general, CEAs of diagnostics for infectious disease fall in two categories: trial-based cost-effectiveness, focusing on short-term outcomes, and long-term models, focusing on outcomes extrapolated beyond the trial. Outcomes measured are for example prescriptions saved or hospitalizations saved. Cost-utility analyses seem to be difficult, as QALY differences between various diagnostic strategies are minor. Antimicrobial resistance is considered indirectly: as an ICER with prescriptions saved as an outcome; by calculating the threshold cost of resistance that would change the conclusion of cost-effectiveness; or as a point estimate; often, it is not considered at all.Conclusions: CEAs of diagnostic strategies for infectious disease are relatively scarce and it can be challenging to prove the cost-effectiveness of diagnostic strategies, especially in the context of relatively inexpensive treatments, such as antibiotics. However, there is an important opportunity of including the societal costs of AMR in future CEAs.<br/