Diabetes mellitus (DM) is one of the most frequent metabolic complications associated with pregnancy, affecting both the prognosis of the pregnant woman and the newborn. Pregestational DM type 1 (T1DM) and type 2 (T2DM) and gestational DM (GDM) are associated with an increased risk of pregnancy complications such as miscarriage, fetal malformations, macrosomia, preeclampsia, and neonatal hypoglycemia, among others. The aim of this review was to evaluate the efficacy and safety of using the Dexcom G6 device (Dexcom, Co., USA) to continuously self-monitor blood glucose levels during pregnancy. This report was requested by the Spanish Ministry of Health.

We systematically searched for articles published to July 2021 in the MEDLINE, Embase, and Web of Science databases. We included experimental and observational primary studies addressing the safety, efficacy, and cost effectiveness of the Dexcom G6 device for gestational and pregestational diabetes.

Two non-comparative prospective studies were identified. One study of 25 pregnant women with T1DM, which evaluated glycemic control and complications during pregnancy and postpartum, reported stable hemoglobin A1c levels during gestation in women using the Dexcom G6 device. The percentage of time spent in the therapeutic glucose range (63 to 140 mg/dL) was 59 percent; 38 percent was in the hyperglycemic range and 3 percent was in the hypoglycemic range. Although some patients reported mild erythematous and edematous reactions to the sensor, no moderate or severe reactions or infections occurred at the sensor insertion site. The other study in pregnant women with T1DM (n=20), T2DM (n=3), or GDM (n=9) showed adequate accuracy of the Dexcom G6 device, compared with the reference method, especially when the sensor is placed on the arm.

Randomized controlled trials are required to assess the effectiveness and safety of the Dexcom G6 device in maintaining adequate glucose control during pregnancy in women with DM. Studies are also needed to compare the Dexcom G6 device with conventional capillary blood glucose self-monitoring or other monitoring methods. No cost-effectiveness studies have been conducted for the Dexcom G6 device in this patient population.

Health technology assessment (HTA) reports are complex technical documents that address multiple aspects of the incorporation of a technology into the healthcare system applying complicated methodologies coming from different disciplines. The purpose of HTA is to support decision makers, who should have an adequate level of training to fully understand these assessments. However, most HTA education programs and courses are intended for HTA doers and there is a lack of practical guidance training aimed at preparing health managers or policy makers in HTA. The objective is to describe an HTA training program developed for decision makers of the three levels (health care administration, hospital management and clinical practice).

The education program has been designed through a collaboration between the Public Health Department of the Faculty of Medicine of the University of Santiago de Compostela (USC) and the Galician Health Technology Assessment Agency that belongs to the Spanish HTA Bodies Network. The duration of the course is 200 hours and the methodology will be distance learning, through the virtual classroom of the USC. The teaching collaborators come from the academic field and the HTA area.

The course will cover the legal, clinical and organizational framework in which the HTA is developed in Spain and in Europe; and will approach the methodology used in HTA. The course is structured in six modules: (i) Research, development and regulation of health technologies; (ii) Role of HTA as a decision support tool; (iii) HTA Methodology; (iv) Health information systems (including use of real world data); (v) Incorporation of HTA into society (stakeholders); (vi) Future challenges (personalized medicine and e-health).

A specific training about HTA from a practical approach not theoretical could be of interest for different stakeholders involved in the decision-making process across the health systems. This type of educational program will allow decision makers to have a good understanding of the wide range of information they handle.

Most European countries use Health Technology Assessment (HTA) as input for decisions on reimbursement of (new) medicines. In 2018, following the approval of European Social Funding, the Directorate for Pharmaceutical Affairs (DPA) within the Malta Ministry for Health led a specific Work Package aimed at enhancing knowledge, skills and HTA processes to inform reimbursement decisions.

Since the start of the project, the Institute for Medical Technology Assessment (iMTA) and the National Health Care Institute of the Netherlands (ZIN) collaborate in providing guidance and training to DPA on relative effectiveness assessments (REAs) and pharmacoeconomic assessments (PEAs) of pharmaceuticals. Several activities were organized: site visits, face-to face interviews with stakeholders, a qualitative assessment of the core process of the Maltese system, health economics training and tutorials, meetings introducing the European Network for Health Technology Assessment (EUnetHTA) and Dutch HTA processes, development of a new framework for assessment, and a shadow assessment.

Our assessment identified important methodological challenges and crucial processes interdependencies to optimize within the Maltese system. Based on the learnings, DPA created a template based on the EUnetHTA REA assessment format. IMTA created a template to perform PEAs. Currently, a shadow assessment is ongoing in which DPA performs the REA and PEA of a pharmaceutical using the new templates. ZIN and iMTA will provide feedback to DPA on several aspects, including, but not limited to, the PICO, assessment of therapeutic efficacy and safety, identifying uncertainties, input costs and effects, and formulating a clear discussion of the assessment. If necessary, the templates will be adapted during the process. The project will be evaluated and finalized in 2022.

With the guidance and training provided by iMTA and ZIN, DPA structured and aligned their REA and PEA to enhance their assessment process and to improve the presentation of their HTA report to the two appraisal committees. This project emphasizes the importance of international collaboration to enhance HTA processes within the

As part of the cyclic Appropriate Care programme of the National Health Care Institute in the Netherlands, a systematic analysis of hearing health care is taking place. Parties in hearing health care are actively involved throughout the entire process. This abstract focuses on lessons learned from the cooperation as a HTA body with a diverse group of stakeholders.

We carried out an in-depth analysis for the patient journey of both children and adults with ear complaints or hearing impairment. Different kinds of information were included in the analyses, including claims data, quantitative and qualitative research, analyses of (international) guidelines and patient information. A range of strategies were used to co-operate and interact with patient organizations, hearing health care professionals, institutes/hospitals and insurance companies.

Close collaboration between the project team and patient organizations turned out to be effective to comprehend patient’ perspectives. Data analyses were often found to be challenging in hearing health care, as the reimbursement data lacked sufficient information. In several cases, building bridges between parties, but also in relation to our HTA body was needed. Conclusions from the analyses were being shared and discussed with a panel of involved stakeholders, leading to support, but not always consensus on potential room for improvement. An internal review process turned out to be helpful in sharing experiences on effective multi-stakeholder management.

We believe that the process did influence the way stakeholders think about the appropriate use of the different available treatment options. Building bridges, and combining different perspectives from patient organizations, health-care professionals and insurance companies is necessary in a cyclic approach. The cyclic appropriate Care programme proved to be a constructive approach for collaboration with stakeholders.

At various stages of the COVID-19 pandemic, face coverings have been recommended and encouraged as one of the interventions to reduce transmission of the SARS-CoV-2 virus. However, in the earlier stages of the pandemic, decisions on face coverings relied primarily on evidence based on other viral respiratory infections. More direct evidence on the use of face coverings with COVID-19 developed in tandem with the pandemic.

Health Technology Wales undertook an ultra-rapid review to inform national guidelines, the work assessed the evidence on the effectiveness of face coverings to reduce SARS-CoV-2 transmission. We also reviewed evidence on the efficacy of different types of face coverings.

We conducted a systematic literature search for evidence to address (i) the effectiveness of face coverings to reduce the spread of COVID-19 in the community, and (ii) the efficacy of different types of face coverings designed for use in community settings. We identified a rapid review in 2021 by Public Health England that closely aligned with our review questions. This provided the main source for identifying relevant studies, supplemented by a search for publications following their search date.

We identified two evidence reviews (including the Public Health England review) that examined the effectiveness of face coverings on reducing transmission of SARS-CoV-2; reporting on 31 and 39 studies, respectively. Two further primary studies were published after the two evidence review searches were included. Overall, the evidence suggested that face coverings may provide benefits in preventing SARS-CoV-2 transmission, although the higher-quality studies suggested that these benefits may be modest. Medical masks appeared to have higher efficacy than fabric masks, although the evidence was mixed.

At the time of this review, evidence on the effectiveness of face coverings remains limited and conclusions rely on low-quality sources of evidence with high risk of bias, although higher-quality evidence points to some benefit. Face coverings may play a role in preventing transmission of SARS-CoV-2, particularly as part of a bundle of other preventative measures.

The Scottish Medicines Consortium (SMC) conducts early health technology assessment of new medicines in Scotland. While budget impact is not a factor in decisions on reimbursement, budget impact information is provided initially through horizon scanning reports for high impact medicines (estimated net budget impact >GBP 500,000 [EUR 585,710] per annum) to aid financial planning and implementation of advice at the local level, and later through budget impact templates from the submitting company issued alongside SMC advice. This research aimed to understand how the information is used and to evaluate the benefits of a lifecycle approach to budget impact analysis.

Health Board users of the budget impact templates were surveyed to explore the degree of utilization and identify areas for improvement, including the need to cross-validate the horizon scanning estimates with those of the submitting company. Responses were analyzed quantitatively and qualitatively, with comments coded in Nvivo (QSR International) and themes established through thematic analysis.

The initial responses received (n=17) provided representation from 57 percent of Health Boards (i.e., payers) covering 79 percent of the population. Preliminary results showed that while the budget impact templates were valued, 69 percent of respondents found them ‘somewhat useful’, suggesting scope for improvement. Almost half (48%) of the respondents used the templates for high impact medicines, with only 30 percent using them for all medicines. The majority (76%) of those surveyed thought there would be value in linking budget impact information throughout the SMC process. An emerging theme was that some users found the templates complex and inflexible, and that a simpler, more adaptable tool to aid the planning process would be welcomed. Priorities identified for improving template included adapting them to the local population and adjusting medicine prices to reflect confidential discounts.

This research suggests that budget impact information is valued by Health Boards and that there is strong support for linking budget impact estimates and engaging stakeholders throughout a medicine’s lifecycle. Simplifying the templates, increasing their adaptability, and providing guidance and training in their use will be key steps in improving this important part of SMC process.

The use of telemedicine services has increased worldwide during recent years because of national strategies for digitalization of health care and the coronavirus disease 2019 (COVID-19) pandemic. However, healthcare professionals often express uncertainty regarding the effectiveness of telemedicine interventions. The TELEMED database (https://telemedicine.cimt.dk/) was introduced by the Centre for Innovative Medical Technology (CIMT) at Odense University Hospital to ensure that hospital managers, healthcare professionals, and other stakeholders have access to scientific studies of telemedicine interventions.

The database constitutes a structured literature search in PubMed for randomized and non-randomized controlled trials on the use of telemedicine for treating somatic diseases in the hospital setting. The search was conducted by staff members in the health technology assessment unit at CIMT. Identified studies were sorted by first screening titles and abstracts and then by reading full-text versions. The data extracted from the studies included setting, intervention, patient group, type of telemedicine, clinical effect, patient perception, and implementation challenges. The value of each study was also assessed with respect to effectiveness.

A total of 510 articles were selected for data extraction and assessment. The database provides results from 22 different specialties and can be searched using the criteria of medical specialty, country, technology, clinical effect, patient experience, and economic effect. The database serves as an information platform for clinical departments who wish to implement telemedicine services. It has great potential for supporting digital transformation during COVID-19 by providing accessible evidence-based information on patient groups and relevant technologies and their effects. More than 95 percent of the studies in the database that compared telemedicine with a control group showed either statistically significant improvements in clinical outcomes with telemedicine or no statistically significant difference between the two groups.

The TELEMED database provides an easily accessible overview of existing evidence-based telemedicine services. The database is freely available and is expected to be continuously improved and broadened over time.

Telemedicine has been introduced in health services, but uncertainties about the real value of this strategy in the management of neurological diseases remain.

A systematic review was undertaken of available scientific literature on the safety, effectiveness, and cost effectiveness of telemedicine combined with in-person visits, compared with usual care, for the treatment and follow-up assessment of patients with neurological diseases. The overall effect size for each neurological disease was estimated using meta-analysis. An economic analysis was performed from the societal and Spanish healthcare system perspectives.

Two economic studies were included for cost effectiveness and 25 randomized controlled trails (n=8,976 patients) were included for the effectiveness and safety assessment (11 on cerebrovascular diseases, four on Parkinson’s disease, three on multiple sclerosis, two on epilepsy, and one each on brain damage, dementia, spina bifida, migraine, and cerebral palsy). The types of telemedicine evaluated included: virtual visits (11 studies); telerehabilitation (seven studies); telephone calls (three studies); smartphone apps (two studies); and online software for computers (two studies). Subgroup analysis by type of telemedicine indicated no discernible effect for telemedicine combined with in-person visits on most of the outcomes analyzed for the various neurological diseases. Given the heterogeneity of diseases, types of telemedicine, and the results observed, a cost-minimization analysis was conducted. Combining telemedicine with in-person visits would cost EUR 2.55 per patient from the perspective of the healthcare system, but it would result in cost savings (EUR 27.34 per patient) from the societal perspective.

The safety and effectiveness of combining in-person visits with telemedicine was similar to that of usual care, but it could be a cost-saving strategy in Spain from a societal perspective.

FreeStyle Libre System (FSL) is a minimally invasive technology, which provides frequent information about interstitial glucose levels, which allows adjustment of insulin dose and a reduction in the number of fingersticks. This study aims to evaluate the effectiveness and safety of FSL in childhood and adolescence.

Prospective case series in 27 Spanish hospitals. Patients aged 4-17 years with type 1 diabetes mellitus (T1DM) were included. Follow-up was done at 3, 6 and 12 months after starting to use the FSL. Outcome measures were HbA1c levels, acute complications of DM (severe hypoglycemia, ketoacidosis), DM knowledge, health-related quality of life, satisfaction and adverse effects. Biochemical glycemic outcomes (e.g., glycemic variability, time in therapeutic range) were available from 3 to 12 months. Mixed regression models with repeated time measurements were implemented.

The mean age of patients was 12.6 years, with 56.4 percent had HbA1c values above 7.5 percent at baseline. This subgroup significantly improved their HbA1c levels at 3, 6 and 12 months (-0.46%, -0.44% and -0.35%, respectively). Patients with controlled HbA1c levels significantly worsened at 12 months (0.29%). There was a significant reduction in severe hypoglycemic episodes, but only in the multiple imputation analysis. In patients controlled at baseline, there were significant reductions between 3 and 12 months in the percentage of time under 55mg/dl (-0.64%), above 250mg/dl (-1.8%) and glycemic variability (-2.6%). In uncontrolled patients, there was a significant reduction in time above 250mg/dl (-5.8%) between 3 and 12 months follow-up. There was no significant improvement in knowledge about disease, although general self-perceived health worsened and general satisfaction improved. Mild adverse events such as skin reactions (14%) and discomfort or pain (11.3%) with no significant reductions in follow-up were recorded.

The use of FSL in childhood and adolescence with T1DM produces a significant reduction in HbA1c levels in patients with uncontrolled HbA1c levels along with a reduction in severe hypoglycemic episodes (in the multiple imputation analysis). FSL-related adverse effects are considered mild.

Major depressive disorder (MDD) severely limits a person’s psychosocial functioning and reduces quality of life. According to world statistics, about 3.8 percent of the population, or about 280 million people, suffer from depression. Approximately one-third of patients with MDD have treatment-resistant depression (TRD). Meanwhile, Vagus Nerve Stimulation (VNS) therapy was approved by the US Food and Drug Administration and received CE marking in Europe for the treatment of chronic or recurrent depression in the early 2000s. The aim of this analysis is to determine the impact of VNS use in the treatment of TRD.

A comprehensive literature search was performed in MEDLINE/PubMed and Google Scholar databases in order to estimate the clinical effectiveness of neurostimulator implantation for treatment of TRD. The main assessment methods were the Hamilton Rating Scale for Depression, the Montgomery-Asberg Depression Rating Scale and the Beck Depression Inventory.

In total, 6 systematic reviews with meta-analyses on the effectiveness of VNS in TRD were studied. The identified meta-analyses did not report any statistically significant differences in treatment outcomes favoring VNS compared to placebo and treatment as usual (TAU). However, the results of two studies demonstrate its positive clinical effect in the form of additional treatment to the TAU with longer follow-up period. An improvement in the clinical response is observed on average after 12 months as a decrease of about 50 percent in the initial estimates of depression.

Despite the lack of clinical evidence of the benefits of treating depression, VNS therapy should be used as a standard adjunct treatment to antidepressants or other treatments for people with TRD. Many studies tend to suggest that the efficacy and safety of VNC in depression is still unclear, and additional further research is still needed to establish clinically significant effects.

The transition from the budget model of the healthcare system to compulsory social health insurance has created a competitive environment among hospitals in Kazakhstan. Managers are interested in introducing the most effective new technologies. Implementation of the health technology assessment (HTA) process in Kazakhstan began in 2010 but few managers have created a structure for HTA development in their hospitals. Our aim was to identify issues in the implementation of new health technologies in hospitals.

Structured interviews were held with hospital managers and physicians in June 2020, and September 2021. In the first stage, the needs of hospitals in the implementation of new technologies were considered. In the second stage, the impact of COVID-19 on the introduction of new technologies in the hospital was addressed. Interviews were held on-line by mobile phone or zoom and lasted 25-30 minutes.

The first interviews involved 8 managers and 14 physicians from 5 hospitals. The needs of HTA for physicians was noted by respondents of both groups. Only a few physicians had been trained in HTA. Hospital staff lacked time and experience in preparing applications for new technologies by a national assessment unit and could not meet deadlines. Managers were interested in use of HTA for hospitals’ technologies in short-term timeframes within existing policies. However, physicians believed that long-term performance of technologies over 5 years or more should also be considered in hospital management. Physicians were aware of the importance of ethical considerations in the HTA of new health technologies. Managers did not consider ethical issues.

At the second stage of the project, 5 managers and 8 physicians were interviewed. COVID-19 had shown the importance and necessity of developing the scientific potential of doctors, and of introducing HTA and training medical personnel in its use.

Positive outcomes from the interviews were the interest of respondents in increasing their knowledge of the HTA process and acceptance of its importance at the hospital level.

The success of the health technology assessment (HTA) implementation depends on the level of communication efficiency between all stakeholders. Taking this into consideration, the leading HTA experts of HTA Department of State Expert Center of the Ministry of Health of Ukraine launched the new educational initiative which includes training programs for pharmaceutical companies.

Сomprehensive review of the first developed HTA training programs based on the HTA Guideline (Number 593) “The state health technology assessment for medicines” approved on 29 March 2021 was conducted.

Leading HTA experts of the HTA Department developed a training program, which was conducted for industry representatives according to the provisions of the HTA Guideline. In 2021 over twelve training sessions and two webinars were conducted. These learning events reflect harmonized international recommendations and approaches to HTA training programs, The Professional Society for Health Economics and Outcomes Research, ISPOR short courses in particular, European Network for Health Technology Assessment (EUnetHTA), The National Institute for Health and Care Excellence (NICE), Institute for Clinical and Economic Review (ICER), Agency for Health Technology Assessment and Tariff System (AOTMiT) guidelines. Two-hour webinars were aimed at giving theoretical and practical bases for building a Markov model with the help of Excel and TreeAge Software. Training sessions titled “HTA as a tool for assessing the value of health technology” differ by duration (7-hour, 4-hour, 3-hour) and cover core topics adapted to the needs of the audience. The main program components include HTA Roadmap in Ukraine, clinical section, economic section and practical case studies of building a Markov model. All training options included questionnaires at the beginning and i end to assess the quality of each program. Questionnaires are the tool that gives presenters the possibility to trace progress and transform the training material accordingly.

Development of extended capacity building programs in HTA for users and doers is highly prospective for further steps in HTA institutionalization in Ukraine.

To provide a complete picture of the economic impact of the coronavirus disease 2019 (COVID-19) emergency for the Italian National Health System (NHS), an estimate was made of the costs to the NHS of vaccination hesitation. The concept of economic impact was investigated with reference to the volume of hospitalizations and days of intensive care required for patients with COVID-19 related to missed vaccinations, considering a vaccine efficacy of less than 100 percent.

Data from the Istituto Superiore di Sanità were analyzed with respect to the number of people vaccinated in the general population, and the number of people with severe acute respiratory syndrome coronavirus 2 infection who were hospitalized or died in a one-month period stratified by vaccination status. The costs for unvaccinated patients admitted to a general hospital ward (Medical Area) or the intensive care unit were calculated.

Based on the number of preventable hospitalizations among unvaccinated people, the economic impact of missed vaccinations on the NHS in the 30-day period from 13 August 2021 to 12 September 2021 was estimated. Among the unvaccinated hospitalized patients, 5,932 would have avoided hospitalization in the Medical Area and 715 would have avoided admission to the intensive care unit. Thus, each unvaccinated hospitalized patient had an average per capita cost of EUR 17,408. The total costs amounted to EUR 69,894,715, comprising EUR 51,166,079 for hospitalizations in the Medical Area and EUR 18,728,636 for hospitalizations in intensive care.

By evaluating the weekly incidence of hospitalizations per 100,000 people stratified by vaccination status (unvaccinated, partially vaccinated, and fully vaccinated), it is possible to see that we are facing two distinct pandemics running together.

The role of associations dedicated to patient advocacy has assumed strategic importance within the most advanced health systems, including the Italian NHS. In this period of strong national and international emergency, the associations of citizens and patients have also changed their actions and have implemented others to alleviate the discomforts of sick people in Italy, collaborating with institutions and health services.

Data were collected using a semi-structured survey, with both yes / no and open questions, developed and administered by the Patient Advocacy LAB (ALTEMS- Catholic University of Sacred Heart) to 150 patient advocacy associations. The organizational changes and the initiatives adopted by patient associations during the COVID-19 emergency was investigated.

The majority of the initiatives adopted by patient advocacy associations during the COVID-19 pandemic have been introduced during the first wave (March- June 2020), and that most of them have been maintained to (December 2020). These initiatives included improvements and updating of the communication tools aimed at reaching the higher number of patients. Thanks to these new approaches, the empathy and the assistance to patients have been increased. In addition, a number of training initiatives have been developed online and they have been followed by a large number of patients and caregivers. At an operative level, during the pandemic, many patient advocacy associations have provided their support to distribute masks, gloves, and therapies to patients. Unfortunately the fundraising activities suffered due to the lockdown, and the main problems were related to social distancing and lack of public campaigns. The institutional relationships have increased during the COVID-19 pandemic and they mainly regarded the participation in legislative interventions. Among the initiatives adopted, our sampled associations affirmed that they would like to maintain the social support provided to patients experienced during the pandemic.

Finally we calculated a resiliency score and we discovered that the majority of the sampled associations developed an intermediate level of resiliency and that it is positively correlated with their tenure.

Our results provide a fresh view about the role of patient advocacy associations during the pandemic indicate their important role within the NHS.

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.