Coronavirus disease 2019 (COVID-19) is a contagious disease caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Symptoms of COVID-19 are variable, but often include fever, cough, headache, fatigue, breathing difficulties, and loss of smell and taste. Symptoms may begin one to fourteen days after exposure to the virus. COVID-19 transmits when people breathe in air contaminated by droplets and small airborne particles containing the virus. The present analysis aims to define the cost-effectiveness profile of the anti-COVID vaccination campaign in the Italian healthcare setting.

The analysis was based on the collection and analysis of data regarding the number of hospitalizations (ordinary regime and intensive care) and infections recorded by the Italian Ministry of Health in vaccinated and unvaccinated patient cohorts. The acquisition costs of the available vaccine alternatives were considered as well as the cost of the personnel involved in the vaccination campaign. The reduction in hospitalizations was considered as a measure of effectiveness. We have compared the current scenario of campaign vaccination versus a scenario in which the total of the eligible population would be vaccinated. Results are reported in terms of Incremental Cost Effectiveness Ratio (ICER). Deterministic and probabilistic sensitivity analyses were carried out in order to test the robustness of the results.

The vaccination campaign allowed for savings amounting to EUR 9,398,012.10 (EUR 60,499,053.25 vs EUR 69,897,065.35) and 6,647 hospitalizations avoided (715 and 5,932 in the intensive care and ordinary regimen, respectively), thus resulting a dominant strategy as compared with the alternative (no vaccination). As the cost-effectiveness profile of the campaign improves, we should consider the period (May-July 2021), during which the daily threshold of 500,000 doses administered on a national basis was consistently exceeded.

The analysis underlined how the vaccination campaign represents a cost-saving alternative in the Italian healthcare setting.

Cost-effectiveness analyses of empirical participant data are frequently complicated by irregularly distributed and correlated observations, which are not well approximated by normal distributions. Things get even more difficult when observations are clustered within higher level units (for example, hospitals) or the participant (that is, multiple measurements at different timepoints). Therefore, we developed a flexible Bayesian approach to jointly model costs and effects of two competing interventions with a multilevel structure.

Our new model is presented in mathematical form and discussed in detail. We model costs and Quality-Adjusted Life-Years effects through Gamma and Beta distributions, and account for the dependency between costs and effects by adding the effects as a predictor for the costs. We further include hurdle models to account for costs of for the presence of zero costs and perfect health scores.

The full model is implemented in the probabilistic programming language Stan. To compare the performance of our Bayesian model to a frequentist approach (linear mixed model combined with bootstrapping), we simulate 1000 datasets consisting of 400 participants and 20 clusters. Performance of both models is assessed in terms of variance, bias and coverage probability with respect to the costs and effects defined in the simulation.

We ran a preliminary simulation with high intraclass correlation, strong negative correlation for patient-level costs and effects, and positive correlation of cluster effects on both outcomes. The analysis shows that the Bayesian model exhibits a slightly larger bias for estimated costs, but smaller errors and higher coverage probability compared to the frequentist alternative. We will explore different scenarios where we vary the parameters of the simulations and assess whether the results are robust to change.

It is very important that economic evaluations in health care produce precise and reliable results. Our Bayesian approach is able to handle multiple statistical complexities at once and performs better than a comparable frequentist model. Whether this conclusion holds for different simulation scenarios will be explored in further stages of this study.

All studies should report methods and findings in full, following credible and justifiable reporting guidelines. According to the guiding principles of the Ensuring Value in Research (EViR) Funders’ Forum (www.evir.org), this applies irrespective of the nature of the findings or whether the study was completed as planned.

One way for a public funding agency to address evidence quality and transparency is to adaptively implement EQUATOR reporting guidelines (www.equator-network.org) in its funding procedure to ensure research quality ‘from proposal to publication’. The Netherlands Organisation for Health Research and Development, ZonMw, has created the ZonMw Reporting Checklist (ZRC), which was derived from EQUATOR reporting guidelines in order to systematically plan, monitor, and evaluate projects. The next step is experimenting with implementing the ZRC in ZonMw’s grant management system and procedures. Customization is possible based on the ‘comply or explain’ approach (80/20 rule).

We selected 15 EQUATOR reporting guidelines that covered basic research and health technology assessment through to implementation projects, supplemented with the reporting guideline for implementation studies (StaRI checklist). We conducted comparative content analyses (including rearrangement) to provide a greatest common denominator consisting of both standard and modular reporting elements. We completed the ZRC by adding other current requirements for responsible research practices with respect to diversity and gender, data management, open access, systematic reviews, recruitment and inclusion, registration, and impact.

The ZRC results in structured and validated in-house data on the objectives, design, conduct, and results of ZonMw projects. This is an important source for good research governance, impact assessment, and research on research.

Implementation of the ZRC by a funding agency optimizes the quality, transparency, relevance, and impact of evidence, which legitimately and effectively improves health care for all.

Aging populations and specialized medicine are leading to increasing healthcare costs which are expected to rise in the next decades. The Netherlands Organization for Health Research and Development (ZonMw) funds trials that address the efficiency of healthcare interventions in order to evaluate new and existing interventions. These studies have led to considerable cost savings and increased health outcomes. However, efficiency studies often face setbacks during the start-up and inclusion which limit the available research capacity and postpone the availability of novel findings. Here, we investigate the scope of these problems and identify common causes.

Records from efficiency research trials funded by ZonMw from 2014-2020 were combined with information provided by project leaders through a survey. The combined dataset was explored through statistical analysis. Next, a subset of 30 selected projects was evaluated qualitatively to gain a better understanding of the possible underlying reasons for the experienced problems.

The response rate among project leaders was 73 percent (146/201). Data indicate that 61 percent of projects started as planned and 35 percent included the first patient as scheduled. The complexity of setting up a multicenter study and legal procedures like local ethical approval were associated with delays in starting inclusions. In addition, 56 percent of studies had to extend the inclusion period by more than 6 months. Possible reasons that were identified include the limited numbers of patients available, and treatment preferences of the doctor, the patient, or the participating center.

Our results indicate that the majority of trials face setbacks and the main reasons include time to procure legal and ethical approval, limited patient numbers, as well as unforeseen treatment preferences. More streamlined procedures regarding approvals could speed up trial initiation, and better knowledge of eligible patients and treatment preferences could lead to more realistic planning. The results and conclusions from this study can be applied by ZonMw and other relevant stakeholders to resolve the identified problems in order to accelerate healthcare efficiency research.

Updating of health technology assessments (HTAs) is generally more efficient than starting again when new evidence emerges, but there is no clear guidance on how to do this. Health Technology Wales (HTW) has developed a re-assessment process to ensure that HTAs remain current and relevant to best serve the population and health and care providers in Wales.

HTW developed a standard operating procedure (SOP) to create a consistent approach to HTA re-assessment. HTW keep a record of stakeholders who contribute to a HTA and then send them a questionnaire to ascertain whether the research question is still relevant and if there have been any developments to the evidence since publication of the HTA. The input from these stakeholders is collated and taken to HTW’s Assessment Group to decide whether or not the HTA needs updating. If the Assessment Group decides that re-assessment of a HTA is warranted, HTW perform an updated literature search to inform the re-assessment.

The HTA re-assessment SOP developed by HTW was approved by the organization’s Assessment Group. At the time of writing this abstract, HTW sent questionnaires to stakeholders of three HTAs which had HTW guidance published three years ago, and were therefore due routine consideration for re-assessment as detailed in our SOP. HTW also received a request from a clinician for a more recent HTA to be considered for re-assessment as they believed the evidence-base had changed since original publication. These questionnaires have been collated and will be taken to an upcoming Assessment Group to decide whether HTW should proceed with the re-assessments.

HTW has developed a consistent process for HTA re-assessment, which ensures that HTAs done by HTW remain current and relevant to best serve the population and health and care providers in Wales. By utilizing expertise from HTA stakeholders and HTW Assessment Group members, an informed decision can be made as to whether a HTA warrants re-assessment after three years following publication or sooner if requested.

For the analysis of clinical effects, multiple imputation (MI) of missing data was shown to be unnecessary when using longitudinal linear mixed-models (LLM). It remains unclear whether this also applies to cost estimates from trial-based economic evaluations, that are generally right-skewed. Therefore, this study aimed to assess whether MI is required prior to LLM when analyzing longitudinal cost-effectiveness data.

Two-thousand complete datasets were simulated containing five time points. Incomplete datasets were generated with 10 percent, 25 percent, and 50 percent missing data in costs and effects, assuming a Missing At Random (MAR) mechanism. Statistical performance of six different methodological strategies was compared in terms of empirical bias (EB), root-mean-squared error (RMSE), and coverage rate (CR). Six strategies were compared: (i) LLM (LLM), (ii) MI prior to LLM (MI-LLM), (iii) mean imputation prior to LLM (M-LLM), (iv) complete-case analysis prior to seemingly unrelated regression (CCA-SUR), (v) MI prior to SUR (MI-SUR), and (vi) mean imputation prior to SUR (M-SUR). To evaluate the impact on the probability of cost-effectiveness at different willingness-to-pay [WTPs] thresholds, cost-effectiveness analyses were performed by applying the six strategies to two empirical datasets with 9% and 50% of missing data, respectively.

For costs and effects, LLM, MI-LLM, and MI-SUR performed better than M-LLM, CCA-SUR, and M-SUR, as indicated by smaller EBs and RMSEs, as well as CRs closer to the nominal levels of 0.95. However, even though LLM, MI-LLM, and MI-SUR performed equally well for effects, MI-LLM and MI-SUR were found to perform better than LLM for costs at 10 percent and 25 percent missing data. At 50 percent missing data, all strategies resulted in relatively high EBs and RMSEs for costs. In both empirical datasets, LLM, MI-LLM, and MI-SUR all resulted in similar probabilities of cost-effectiveness at different WTPs.

When opting for using LLM for analyzing trial-based economic evaluation data, researchers are advised to multiply impute missing values first. Otherwise, MI-SUR may also be used.

Patients’ EQ-5D health states are preferably valued using country-specific value sets. If value sets are not available, crosswalks may be used to estimate utility values. However, up until now the impact of using crosswalks instead of value sets on cost-utility outcomes remains unclear.

Trial-based cost-utility data were simulated for four conditions (depression, low back pain, osteoarthritis, and cancer), three levels of disease severity (mild, moderate, and severe), and three treatment effect sizes (small, medium, and large), resulting in 36 scenarios. For all scenarios, utility values were estimated using four scoring methods (EQ-5D-3L value set, EQ-5D-5L value set, 3L-to-5L crosswalk, and 5L-to-3L crosswalk) for three countries (the Netherlands, the United States, and Japan). Mean utility values, quality-adjusted life years (QALYs), incremental QALYs, and cost-utility outcomes (incremental cost-effectiveness ratios [ICER], probabilities of cost-effectiveness at willingness-to-pay [WTP] thresholds) were compared between value sets and crosswalks.

Differences between value sets and crosswalks ranged from -0.33 to 0.13 for mean utility values, from -0.18 to 0.13 for QALYs, and from -0.01 to 0.08 for incremental QALYs. Because of the small differences in incremental QALYs, ICERs between scoring methods were considerably different. For small effect sizes, at a WTP of EUR 20,000/QALY gained, the largest difference in the probability of cost-effectiveness was found for moderate cancer between the 5L value set and 3L-to-5L crosswalk (difference 0.63) using Japanese valuations. For medium effect sizes, the largest difference was found for mild cancer between the 3L value set and the 5L-to-3L crosswalk (difference 0.06) using Japanese valuations. For large effect sizes, the largest difference was found for mild osteoarthritis between the 3L value set and 5L-to-3L crosswalk (difference 0.08) using Japanese valuations.

Our findings indicate that reimbursement decisions may change depending on the use of crosswalks. Crosswalks are justifiable in absence of country-specific value sets but should not be considered a sustainable alternative for value sets.

This study reviewed all publicly available Japanese cost-effectiveness appraisals delivered by the Center for Outcomes Research and Economic Evaluation for Health (C2H) from the national institute of public health (NIPH) and analyzed the relationship between the outcomes of the health-economic assessments and the final price adjustment decisions made by the Ministry of Health, Labour and Welfare (MHLW).

Data were extracted from all official health-economic assessments published by C2H website for the analysis. The extractions were structured based on the following items: indication, assessment methodology, appropriate comparators, health-economic outcomes, and key uncertainties identified by C2H. The analysis was performed on 29th November 2021. A threshold of 5 million JPY per Quality Adjusted Life Year gained (JPY/QALY) was used for the incremental cost-effectiveness ratio (ICER) analysis cut-off.

Up to the time of this analysis, six health-economic assessments had been conducted for five products: three assessments performed cost-effectiveness analysis, one performed cost-minimization analysis and two performed cost-effectiveness and cost-minimization analysis for different comparators and different patient subgroups respectively. Among the five assessments’ reported ICER values, four of them are under the 5 million JPY/QALY threshold, ranging from 328,585 JPY/QALY to 483,056 JPY/QALY. However, price adjustments were still implemented on three out of the four products which were deemed to be cost-effective, ranging from -0.5 percent to -4.3 percent (mean: -3.0%). For the only product deemed to be not cost-effective, a price adjustment of -4.3 percent was implemented.

A price discount could be implemented regardless of whether the ICER value falls under the 5 million JPY/QALY threshold. However, a lower magnitude of price discount is likely to be applied by MHLW for more cost effective treatments. The outcome of this analysis may be limited by the small sample size and continuous monitoring of further HTA publications in Japan is needed.

The Brazilian HTA process includes calls for public consultations, in which society can give its opinion on reports emitted by the National Committee for Health Technology Incorporation (CONITEC). Open and closed queries for public consultation are performed by official formularies and are available online at CONITEC webpage. There are two categories of queries: clinical protocols and guidelines, and incorporation/exclusion demands. Incorporation/exclusion queries are subdivided in two additional categories: opinion and experience, or technical. In this study we analyze health professionals’ technical contributions and their opinion (pro or con) on the inclusion or exclusion of health technologies.

On November 26th, 2021, formularies concerning concluded public consultations on health technology incorporation/exclusion reports were extracted from CONITEC website for the period, January 1, 2021 to November 26, 2021. Entries on the technical contributions formularies included a close-ended question about the opinion of participants on health technology incorporation/exclusion reports (“favorable”/“against”/“neither”).

A total of 63 health technology incorporation/exclusion queries were carried out during the study period, of which only 4 were exclusions. A total of 7783 contributions were registered. “Patients”, “Family or caregivers”, “Interest on the theme”, and “Health professionals”, accounted for 96.4% (10.9, 15.2, 17.1 and 53% respectively). Health professionals’ participation alone accounted for 4130 entries. Concerning the category “health professionals”, the total number of favorable opinions on the presented documents was 2740 (66.3%), 1306 (31.6%) disagreed, and 84 (2%) had no opinion.

Health professionals can be considered one of the main stakeholders considering HTA for technology incorporation in public health systems. Brazilian HTA reports are submitted to public consultation through queries, which are available open access at the Brazilian National Committee for Health Technology Incorporation website.

The European Network for Health Technology Assessment (EUnetHTA) was a voluntary cross-border initiative on HTA harmonization established by European Commission in 2005. Between 2016-2021, EUnetHTA completed 56 Joint Assessments (28 drugs; 28 other technologies) including 14 reviews of COVID treatments.

We conducted a review of the 14 EUnetHTA joint assessment reports of drugs in non-COVID 19 indications. We cross-referenced recommendations with national guidance in 30 member countries (including UK) and conducted an analysis of time to national assessment, choice of comparator, direct reference to EUnetHTA assessment, and time to reimbursement decision.

Six products in oncology, 2 in endocrine and metabolic diseases, 2 in infectious and parasitic diseases, and cardiovascular, digestive system, eye disorders and central nervous system (one each) were identified. On average, EUnetHTA published its recommendation 52 days after market authorization for oncology products and 33 days for non- oncology products. EUnetHTA recommendations considered on average 4 comparators (range 1-8) as part of the assessment. All of the 6 oncology products have been assessed by national HTA bodies, however uptake was low with an average of 5 reports referencing the EUnetHTA report. Similarly for the non-oncology products assessed only 3 of 30 HTA bodies cite the EUnetHTA report. Citing HTA bodies were: AETSA (Spain), HAS (France), INFARMED (Portugal), NoMA (Norway), and TLV (Sweden). There was no clear reduction in the time to reimbursement for these products in these markets.

According to EUnetHTA, there has been an increased use and dissemination of joint assessment reports since 2016. Our analysis shows that the level of implementation across countries is heterogeneous despite publication of the EUnetHTA reports shortly after market authorization. The future the EU HTA will depend on the timeliness, rigor and transparency of joint clinical assessment reports and improved uptake of these reports at a national level.

Health-related quality of life (HRQoL) data are necessary for conducting cost-utility analyses (CUAs) as part of health technology assessments (HTAs), but the lack of robust HRQoL data may delay or even prevent patient access to medicines when National Health Insurance is introduced in South Africa. This study aimed to make recommendations for evidence generation activities to support national HTA in the public health sector, with a focus on creating South African HRQoL data suitable for CUAs.

A systematic review of HRQoL research in South Africa formed the basis for three analyses. The first analysis quantified and evaluated the suitability of HRQoL studies for CUAs. The second analysis determined the performance indicators of the research output and identified collaborative networks through bibliometric analyses. The third analysis critiqued the translation methodology of the HRQoL instruments retrieved in the systematic review.

Based on the published literature, existing HRQoL data are unlikely to support CUAs because they were derived from observational or cross-sectional studies that lacked the methodological details necessary to determine their scientific merit according to HTA requirements. Overall, there was a lack of research continuity in this field, with numerous isolated research networks. Despite the strong contribution of South African based researchers and organizations in this area, their performance was below that of international counterparts. Since only a few HRQoL instruments suitable for CUAs would be valid in the South African context, HRQoL research output in South Africa could be optimized by using more rigorous study designs and by the expansion of researcher networks to include those working in HTA and related fields. The three-level EQ-5D is the tool best suited for use in South Africa, so its utilization should be encouraged and supported by establishing a South African value set.

Future data generation activities should incorporate the recommendations from this study because existing South African HRQoL data are likely to be inadequate for conducting CUAs in national HTA.

Damage to the central nervous system (CNS) in adulthood, may lead to cognitive impairments. In Germany, occupational therapy is most often prescribed for neurological diagnoses, including stroke and traumatic brain injury (351 and 343 cases per 100,000, respectively in 2018). For cognitive impairments, the primarily prescribed remedies are sensorimotor-perceptive, motor-functional and neuropsychologically oriented treatment or training of cognitive performance. Here we report the results of a health technology assessment (HTA) report on the clinical efficacy of occupational therapy for patients with cognitive impairments.

To assess clinical efficacy, a systematic overview was conducted based on published systematic reviews and HTA reports from the last ten years summarizing randomized controlled trials (RCTs) retrieved from four bibliographic databases. The target population included adult patients with cognitive impairments caused by diseases of the CNS, excluding moderate to severe dementia. The intervention studied is occupational therapy compared to no occupational therapy. Outcomes were cognitive abilities, independence, self-determination, health-related quality of life (QoL), and participation in activities of daily living (ADL).

Five systematic reviews comprising 1,316 patients were included. There is evidence for a small statistically significant positive effect on “general cognitive function” (10 RCTs, n=470) and on ADL (4 RCTs, n= 405). A non-quantified positive effect was reported on behavior control (1 RCT, n=96), and conflicting evidence on QoL (2 RCTs, n=214). No effect was found for individual components of cognition (5 RCTs, n=202), self-efficacy (1 RCT, n=98) and social participation (2 RCTs, n=194). The level of the evidence was low for all endpoints due to the high risk of bias and small sample sizes.

Based on this systematic overview, it cannot be demonstrated but also not ruled out that occupational therapy for cognitive impairment is an effective therapy for adults with cognitive impairments. The evidence is very uncertain due to small effects and high risk of bias, low statistical power, and heterogeneity of interventions and study populations.

The performance of diagnostic health technologies is usually assessed by comparing them with standard care using the kappa statistic. These comparisons are made based on comprehensive clinical information (e.g., anamnesis and complementary tests). However, not all digital applications (DAs) execute over complete information, which leads to work under non-uniform distribution of values. Using kappa statistic in this situation has serious methodological limitations. Kappa assumes that the marginal values are uniformly distributed and highly weights the discordant values when calculating concordance, which underestimates the real effectiveness of DAs (i.e., observed concordance). We aimed to present the application of the B statistic to WtsWrng, a symptom triage DA for individuals.

WtsWrng was used by 382 patients at the emergency department of a hospital. Diagnoses provided by WtsWrng, given 19 symptoms, were compared with those logged in the hospital’s electronic clinical records at discharge. Observed concordance was calculated using contingency tables. The concordance using the kappa and B statistics were compared for the 12 most frequent diagnoses at hospital discharge. Sensitivity and specificity were also calculated.

Real observed concordance fluctuated from 0.4 to 0.98 for the 12 most frequent diagnoses, eight of which had a concordance greater than 0.8. The results ranged from -0.005 to 0.37 when using the kappa statistic and from 0.36 to 0.99 when using the B statistic. The sensitivity and specificity of WtsWrng were greater than 0.8 for three and eight of the 12 diagnoses, respectively.

The results show that the B statistic is closer to the real observed concordance when kappa statistic assumptions are not fulfilled by a DA. Therefore, the B statistic is better suited for assessing the effectiveness of this type of technology. Analysis of WtsWrng using the B statistic showed that its diagnoses were close to those provided by clinicians, which were arrived at using complete clinical information. Moreover, the high specificity of the WtsWrng DA suggests that it is a good tool for determining the appropriate use of healthcare resources.

Health technology assessment (HTA) has become a critical support to health policy decision-making. The HTA evaluation process requires transparency, formalized processes, clear timelines, and standardization according to international best practice. Tunisia is establishing an HTA-based decision-making system through the National Authority for Accreditation and Assessment in Healthcare (INEAS) to ensure impartiality and fairness in decision-making, which is important for an emerging democracy. INEAS opted for a participatory approach in developing the national health economic guidelines to better engage healthcare sector stakeholders in the HTA process. We aimed to present the main phases of the process used to develop the Tunisian health economic guidelines, the methodological choices for pharmacoeconomic evaluations, and the methodological choices for budget impact analyses.

The different phases of developing the guidelines were listed and reported.

The guidelines were developed under a technical cooperation program of the World Health Organization and involved collaboration between the Institut national d’excellence en santé et en services sociaux (INESSS in Quebec, Canada) and INEAS. The first version of the guidelines was drafted following a review of international HTA guidelines and best practice reference books, taking into account the Tunisian healthcare system context. This first draft was discussed in a workshop with the main health system stakeholders and then peer reviewed by international experts. Based on the feedback from experts, a second version was prepared and published on the INEAS website for public consultation. The Union of Innovative Pharmaceutical Research Companies (SEPHIRE), the National Health Insurance Fund (CNAM), and healthcare professionals provided the majority of feedback. The comments provided by SEPHIRE were discussed during a second workshop. The guidelines were revised and updated based on the comments provided and the final version was published in November 2021.

INEAS adopted a participatory approach for developing its economic guidelines, which enhanced engagement of the major health system stakeholders in the HTA implementation process in Tunisia.

Hansen’s disease, or leprosy, is a chronic bacterial infection that affects the nerves, skin, eyes, and nose lining. In 2019, there were 202,256 new cases reported globally, and nearly 28,000 new cases are diagnosed each year in Brazil. The best way to prevent the spread of Hansen’s disease is early diagnosis and treatment of infected individuals. Most diagnoses are done clinically, but only the microscopic analysis of slit-skin smears is funded in Brazil. Serologic and polymerase chain reaction (PCR) tests have also been developed to aid in the diagnosis. The goal of this study was to identify the most cost-effective strategy for increasing the diagnosis of Hansen’s disease in Brazil.

We examined the impact of the following four strategies using a decision tree model: (i) slit-skin smear only; (ii) PCR test only; (iii) serologic testing followed by slit-skin smear for positive samples; and (iv) serologic testing followed by slit-skin smear for positive samples and PCR test for negative serologic tests and negative slit-skin smears. The accuracy of the tests was determined using a systematic review and meta-analysis and validated by experts. The costs were calculated from the Brazilian health system perspective. Univariate and probabilistic analyses were also conducted.

Serologic testing or PCR followed by slit-skin smear was dominated in the economic model (more false-negative samples and more costly). The addition of serologic testing and PCR to the diagnostic sequence made the strategy more expensive than slit-skin smears alone, but it significantly reduced the percentage of false negative results (from 7.3 to 2.9%) at an estimated cost of USD 533.61 per incremental diagnosis. Disease prevalence was the most important variable in the sensitivity analysis.

This is the first cost-effectiveness model undertaken for Hansen’s disease. The results indicate that incorporating serology and PCR testing into the Brazilian health system could be an appealing option for reducing the spread of Hansen’s disease in Brazil.

The unemployment rates among people being treated for substance use disorder (SUD) are high, with Norwegian estimates ranging from 81 to 89 percent. A promising method for improving vocational outcome is Individual Placement and Support (IPS), where employment support is integrated into the treatment regimen. However, the expense and economic gain are covered by different societal sectors, which may be a disincentive for implementing this method. Thus, the aim of this study was to model the potential socioeconomic value of a new SUD treatment service.

For the simulation study, we made qualified assumptions about costs and socioeconomic gain based on data from scientific and administrative publications, expert opinion, and a randomized controlled trial of treatments for individuals with SUD that was set in a specialized Norwegian healthcare setting. We made assumptions about the proportion of patients likely to obtain employment after participating in the following three interventions: (i) treatment as usual; (ii) a self-help guide and additional workshop; and (iii) IPS.

Based on early socioeconomic simulation modeling for the three interventions, IPS was found to be cost effective over a period of one to two years.

In this study we used early economic modeling to demonstrate the potential value of IPS for increasing employment rates among patients with SUD. Since it is important to secure evaluative support for an innovation at the earliest possible stage, early economic modeling may assist the innovator in implementing a health service that meets predefined user needs while also reducing associated risks. Although there is much uncertainty in such early stages due to a lack of valid data sources, early economic modeling may provide health authorities with much needed decision support when planning for future health services.

Fatigue in Parkinson’s Disease (PD) remains poorly understood. The comprovation derives from and is proven by psychometric scales. The objective is to prepare a Technical Scientific Report on the performance of scales in PD.

Based on the Methodological Guideline for the Preparation of Technical Scientific Report, Brazil 2014, we conducted a search on MEDLINE / PubMed, Virtual Health Library (BVS) and Cochrane, and then a review and critical evaluation of the studies and the quality of the evidence.

Nine studies were analyzed: three systematic reviews, one case-control and five cross-sectional studies. The following were evaluated: Fatigue Severity Scale (FSS), Fatigue Assessment of Chronic Illness Therapy (FACIT-F) and Parkinson’s Fatigue Scale (PFS-16). In Brazil, FSS and PFS-16 were validated. The studies have methodological weaknesses and moderate, low and very low evidence. However, FSS, FACIT-F and PFS-16 show potential for assessing fatigue in PD but further studies are needed.

Consensus on fatigue are recommended for Brazilian studies comparing FSS and FACIT-F with PFS-16 and longitudinal monitoring of patients.

Neurogenic bowel dysfunction occurs in people with central nervous system disease or injury and causes loss of bowel control and severe constipation. These problems involve a lot of anxiety and discomfort and can reduce the quality of life of those who suffer from them, therefore the management of symptoms is very important. Peristeen Plus is a transanal irrigation (TAI) system for bowel dysfunction management, used to empty the rectum and distal sigmoid colon, to prevent uncontrolled bowel movements or to relieve and prevent constipation.

A literature review was conducted. A total of 14 records were included to evaluate the benefits in terms of efficacy and safety associated with the adoption of the medical device. To assess the economic impact, two different budget impact models have been implemented. The first aimed at evaluating an incremental diffusion in a short-term time horizon (3 years) of the home distribution of the device compared to the direct and indirect distribution methods in the Italian context. The second model aimed at assessing the impact of the diffusion of the device in the clinical practice.

Overall, most studies demonstrate improved endpoints related to the severity of fecal incontinence, constipation and intestinal disorders in patients using the device. The economic assessments conducted estimate that the increase in the Italian care setting is associated with a saving of resources in each year under analysis. The diffusion of home distribution of the device would potentially be able to offer a lower absorption of resources compared to other distribution methods. In addition to this, there is an incremental saving correlated to the degree of diffusion of Peristeen Plus.

TAI is considered a safe and more effective method than conventional treatments for reducing fecal incontinence, constipation and improving quality of life. The results of our study confirm the benefits of TAI as a second-line treatment in case of failure of conventional medical therapy in the management of the neurogenic gut.

Parkinson’s disease (PD) is the second most common neurodegenerative disease in the world. High-intensity focused ultrasound (HIFU) is a new non-invasive therapeutic option for treating the motor symptoms of PD. HIFU is an imaging-guided procedure for therapeutic brain ablation that has been used for patients with essential tremor and neuropathic pain. It is indicated for patients older than 22 years of age who have PD that is refractory to drug treatment and are ineligible for surgery. The objective of this study was to conduct an early assessment of HIFU subthalamotomy for the treatment of motor symptoms in patients with PD.

HIFU was identified by the early awareness and alert system, SINTESIS- nuevas tecnologías, of the Agencia de Evaluación de Tecnologías Sanitarias at the Instituto de Salud Carlos III. Relevant literature published to October 2021 was identified by searching PubMed, EMBASE, ClinicalTrials.gov, and the Cochrane Library.

One prospective study and one randomized controlled trial (RCT) were found that assessed the efficacy and safety of HIFU subthalamotomy for treating the motor symptoms of PD. The Movement Disorder Society-Sponsored Revision of the Unified Parkinson’s Disease Rating Scale Part III (MDS-UPDRS III) was used to measure changes in symptoms (>30% change from baseline was considered clinically relevant). Both studies reported a reduction of symptoms in the intervention group. The MDS-UPDRS III score changed from 16.6 to 7.5 six months after treatment in the prospective study and from 19.9 to 9.9 four months after treatment in the RCT (a decrease of 11.6 points was observed after 12 months). The main adverse events reported were dyskinesia, speech and gait disturbances, and weakness, all of which resolved without treatment.

The results regarding the efficacy and safety of HIFU for treating the motor symptoms of PD are promising. HIFU is a non-invasive procedure that eliminates the risks associated with surgery. Although rapid diffusion of this technology is expected, further studies and economic evaluation are needed.

Migraine is a multifactorial pathology that affects 12 percent of the world’s population. Relivion® MG (Neurolief Inc., USA) is a non-invasive self-administered device for external combined occipital and trigeminal nerve stimulation that has been recently approved for marketing in Europe and the USA. The objective of this study was to conduct an early assessment of the efficacy and safety of Relivion for the treatment of migraine episodes.

Relivion was identified by the early awareness and alert system, SINTESIS- nuevas tecnologías, of the Agencia de Evaluación de Tecnologías Sanitarias at the Instituto de Salud Carlos III. An early assessment of the technology was conducted by reviewing relevant clinical studies published to 29 September 2021. The literature was identified by searching PubMed, Embase, the International Clinical Trials Registry Platform, ClinicalTrials.gov, and the Cochrane Library.

Two randomized, sham-controlled double-blind trials were found. They assessed side effects and pain relief, response rate, and pain freedom two hours after treatment. One study included 55 patients and the other (the RIME study) included 131 patients. The rate of pain relief two hours after treatment ranged from 60 to 76 percent in the treatment group and from 31 to 37 percent in the control group (p<0.01). The response rate, defined as at least 50 percent pain reduction two hours after treatment, was significantly higher in the treatment group (67 to 70% versus 32 to 42%). The percentage of patients free of pain two hours after treatment ranged from 42 to 46 percent in the treatment group, compared with 11 to 12 percent in the control group (p<0.0001). No notable adverse events were recorded.

Preliminary results indicated that Relivion effectively and safely relieves or eliminates acute migraine pain. However, more comparative studies are needed. The use of Relivion could improve the control of symptoms and improve quality of life in patients with migraine.