Innovative health technologies, like telemedicine, offer advantageous telediagnostic apps that can improve the health care of populations in remote regions. However, evidence on how these developments can enhance universal coverage for electrocardiographic (ECG) diagnosis to support a cardiovascular disease prevention program is limited. The utility of telemedicine for attaining universal coverage for ECG diagnosis according to the national cardiovascular disease prevention program in Paraguay was investigated.

This cross-sectional survey included adults (aged 19 to 80 years) and children (aged 1 to 18 years) with a medical prescription. The study was carried out by the Telemedicine Unit to evaluate the utility of a telemedicine net for a countrywide detection and prevention program for cardiovascular disease. The results obtained by the tele-ECG net, which was implemented in sixty public hospitals countrywide, were analyzed and used to verify adherence to the cardiovascular prevention program.

Between 2014 and 2019, 331,418 remote ECG diagnoses were performed. Of these, eighty-two percent (n = 270,539) were in adults and eighteen percent (n = 60,879) were in children. Among the adult diagnoses, the majority (52%) were pathological and included sinus bradycardia (13%), right bundle branch block (6%), left ventricular hypertrophy (5%), and ventricle repolarization disorder (5%). Among the children, only twenty percent of diagnoses pathological and included sinus bradycardia (11%) and sinus tachycardia (4%). The mean rate of adherence to the prevention program was 38.2 per 1,000 diagnoses performed.

The results showed that telemedicine can significantly enhance coverage for universal ECG diagnosis to support cardiovascular disease prevention and health programs. However, before carrying out the systematic implementation of such a program contextualization using the regional epidemiological profile must be performed.

India has introduced health technology assessment (HTA) as a tool for improving the allocation of health resources. The core mandate of HTA in India (HTAIn) is to undertake critical appraisal of available technologies, identify cost-effective interventions, and help the government pursue evidence-informed decisions regarding public health expenditures. We conducted a systematic review to assess economic evaluation studies published in the last four years from India.

Economic evaluations published from September 2015 to September 2019 were identified by searching various databases, including PubMed, Scopus, Embase, The Cochrane Library, and CINAHL according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guideline. Cost-effectiveness studies and HTAs reported or conducted in India were included. Two independent reviewers performed the final selection of studies by assessing the full-text articles and conducted the data extraction. Differences of opinions were resolved through discussion and mutual consensus.

After screening 2,837 articles, seventy met the inclusion criteria and were selected. The articles predominantly used secondary data (70%) to evaluate the cost effectiveness of an innovation. Among the technologies assessed, fifty-seven percent were curative in nature and most commonly addressed infectious diseases (27%), closely followed by non-communicable diseases, and maternal and child health. Principally, the cost effectiveness of a technology was expressed in terms of disability-adjusted or quality-adjusted life-years. Only two studies reported negative findings.

HTA can play a pivotal role in equipping policy makers and public health payers to make appropriate decisions for healthcare budget allocations when mapped with the true disease burden of the population. It is important to highlight negative results and to create a national repository of HTA studies to facilitate faster adoption of best practices in India.

Web-based and mobile health interventions, also called eHealth, have significant potential to deliver cost effective, quality health care. The present review maps common eHealth technology solutions for primary healthcare by evaluating their safety, efficacy, and effectiveness, and the challenges associated with their implementation in low-middle income countries (LMIC) in the last ten years.

A search of various electronic database was conducted, including PubMed, Scopus, and PsycINFO, to identify articles published between 2009 and 2019 that focused on the implementation of eHealth in the primary healthcare setting across LMICs. A total of 450 articles were screened and thirty-nine relevant articles were selected for review.

The thirty-nine included studies were classified into the following four categories: (i) assessment of intervention effects (n = 26); (ii) cost-benefit analysis (n = 4); (iii) systematic review (n = 5); and (iv) conceptual exploration of eHealth interventions (n = 4). The eHealth studies covered three domains: (i) non-communicable diseases; (ii) reproductive, maternal, newborn, and child health; and (iii) other health issues. The included eHealth technologies comprised mobile health (n = 27), telemedicine (n = 10), and information and communication technology (n = 2).

The majority of studies assessed eHealth technologies based on the following eight dimensions: safety, clinical effectiveness, technical aspects, acceptability, cost, ethical aspects, adaptability to local needs, and scalability. However, evidence on safety, cost effectiveness, and scalability were limited. The main implementation challenges identified were technology development and maintenance costs, the need for trained human resources, and acceptability among users. The methodologies and assessment frameworks of the studies were heterogeneous in nature, highlighting the need for a robust, standardized, and comprehensive framework for assessing eHealth technologies.

In 2017, the National Institute for Health and Care Excellence (NICE) and the National Health Service England established a program to identify Digitally Enabled Therapies (DET) that increase access to Improving Access to Psychological Therapies (IAPT) services. The aim was to determine whether DETs could improve service efficiency, and whether outcomes are at least as good as those achieved by NICE-recommended non-digital therapies.

An IAPT assessment briefing (IAB) was developed for each eligible DET. IABs included an assessment of content, technical standards, clinical effectiveness, and cost and resource impact. IABs were reviewed by the NICE IAPT expert panel to decide whether a DET is suitable for evaluation in IAPT services, needs further development, or is not suitable. Suitable DETs were evaluated for up to two years.

Of 154 DETs reviewed by the program, fourteen had IAB assessments. The high dropout rate was due to ineligible products or developer withdrawal. Of the fourteen IABs, five were recommended for evaluation, one was recommended for development, and eight were not recommended.

DETs can provide an alternative for patients who may not be able to access treatment. When establishing programs to review DETs, centers must consider the quality of the products submitted and, where necessary, make pragmatic decisions about assessment criteria.

Disruptive telemedicine tools can help overcome the lack of specialized care and electroencephalographic (EEG) support for diagnosing and treating nervous system disorders such as epilepsy in remote communities. However, evidence on how such cloud-based platforms could enhance data-driven health care is limited. The utility of telemedicine-based apps to achieve EEG screening of communities in rural areas of Paraguay was investigated.

This descriptive study was carried out by the Telemedicine Unit of the Ministry of Public Health in collaboration with the Department of Biomedical Engineering and Imaging of the Health Science Research Institute in Paraguay and the Basque Country University in Spain to evaluate the utility of telediagnostic apps for EEG screening. For this purpose, the results obtained by tele-EEG apps implemented in nineteen public community hospitals were analyzed to determine the utility of the apps as epidemiological surveillance tools.

Among the 10,791 remote EEG studies performed, the most common reasons for the test included epileptic seizure (44%), headache (22%), seizure disorder (8%), follow up (6%), attention deficits in children (5%), cognitive impairment (4%), cranioencephalic trauma (3%), brain death (1%), history of seizure (0.9%), abnormal movements (0.7%), and behavioral disorders (0.5%).

The results showed that telemedicine apps can significantly enhance nationwide EEG screening by freeing up professional time and increasing productivity, improving access and equity, and reducing costs. However, before their systematic implementation a contextualization of the apps using the regional epidemiological profile must be performed.

Involving patients is a core principle which governs the work of the National Institute for Health and Care Excellence (NICE). To improve how patient evidence is identified and considered in health technology assessments (HTAs), NICE worked with patient organizations to review existing HTA methods and co-designed proposals for change.

A working group, including six patient organizations, oversaw the project, identifying and co-designing options for improvement. We held a stakeholder event with twenty-two patient organizations to identify themes for improving how we find and use patient evidence. We then ran an online quantitative and qualitative survey for targeted consultation with patient organizations to capture broader views.

The fifty-two people who responded to the consultation made the following suggestions:

1. (i) Provide information about uncertainties that patient evidence might help to address;

2. (ii) Explore the role of real-world evidence in patient involvement;

3. (iii) Provide training and support to patient organizations;

4. (iv) Create inclusive committee cultures; and

5. (v) Include additional touchpoints during HTAs to incorporate patient evidence.

This work identified improvements in seeking and incorporating patient evidence into HTA processes. Precise guidance for patient organizations will help them to submit evidence that will make the most impact. This is particularly important when assessing disruptive technologies where there are likely to be greater uncertainties and cost pressures. The results of this work will be developed into formal options for NICE to consider when updating its methods guides.

In 2018, the National Centre for Pharmacoeconomics (NCPE) was commissioned to conduct a health technology assessment (HTA) of one of the first commercially available chimeric antigen receptor (CAR) T-cell therapies, tisagenlecleucel. CAR T-cells are a major advance in personalized cancer treatment, demonstrating promising outcomes in relapsed/refractory pediatric acute lymphoblastic leukemia (pALL). However, the results are based on short-term follow up, limiting their value in predicting long-term survival and leading to uncertainty about the most appropriate survival modeling method to employ. This study aimed to address these limitations by means of expert elicitation.

An expert elicitation method, the histogram technique, was employed. A predefined discrete numerical scale was presented in Microsoft Excel® and the expert was asked to place twenty crosses on a frequency chart. These crosses represented the expert's beliefs about the distribution of particular quantities. Each cross represented five percent of the probabilistic distribution. Individual distributions were then aggregated across experts using linear pooling.

A total of seventeen experts were invited to take part; eight agreed to participate and five completed the exercise. Three experts did not consider tisagenlecleucel to be a “curative” therapy because patients had a higher risk of death, compared with the age- and sex-matched general population. The aggregated distributions indicated the five-year overall survival rate to be thirty-three percent (95% CI 8.65–56.88) in patients who do not receive a subsequent stem cell transplant and twenty percent (95% CI 2.38 -52.04) in those who do.

The results of this study will be used to calibrate CD19 CAR T-cell therapy survival estimates presented in HTA submissions to the NCPE to ensure more robust assessments. They will also be used to inform the construction of a de novo cost-utility model for examining the cost effectiveness of CD19 CAR T-cell therapies for relapsed/refractory pALL in the Irish healthcare setting.

The National Health Insurance Law enacted in 1995 stipulates a minimum list of health services (benefits package) that the four health plans in Israel have to provide to their members. The recommendations on which new technologies or new indications for existing ones should be added every year to the benefits package, subject to a predetermined budget, are made by a public committee that evaluates and prioritizes candidate technologies according to their clinical merit, economic (mainly budget impact), social, ethical and other aspects. We assessed the legitimacy of this coverage decision process over the past 20 years.

The legitimacy of the process was assessed by adherence to the conditions outlined in the accountability for reasonableness (A4R) framework. A4R defines four conditions for legitimate and fair healthcare coverage decision processes: relevance, publicity, appeals/reversibility, and enforcement. We reviewed the changes made in the coverage decision process over the past 20 years and examined whether these changes have changed its legitimacy.

Our analysis suggests that despite several changes made over the years in the process for updating the benefits package, for example, increase in transparency, introducing a structured appeal process, it only partially fulfills the four A4R conditions. In order to accomplish these goals more fully, several widely used considerations such as cost-effectiveness analysis and incorporating views from patients should be included. Additionally, this decision-making process should become even more transparent than it currently is.

The annual process of updating the benefits package in Israel where hundreds of technologies are “competing” with each other for coverage under a pre-defined budget is unique and not without merit. This process has been operating in the same pattern with only minor changes made since 1999. The main barriers for fulfilling all A4R conditions may relate in part to the large number of technologies assessed each year within a short time frame. Several changes in the process including the assessment of societal values, involvement of diverse stakeholders including patient advocate groups should be made to improve its legitimacy.

One anastomosis gastric bypass (OAGB) has become a widespread technique over the last few years in France, without any prior assessment and despite existing controversies among bariatric surgeons. An older bypass technique for treating obesity, the Roux-en-Y gastric bypass (RYGB), is available and reimbursed, having been assessed and approved for use in 2005. In 2019, the French Haute Autorité de Santé (HAS) assessed OAGB for the treatment of severe and massive obesity. This assessment, the first in the world, was undertaken for OAGBs carried out with a 200- or 150-centimeter biliopancreatic-limb (BP-limb) length.

A systematic review (SR) of the literature and consultation of a working group consisting of both healthcare professionals (clinician and surgeons) and patients were carried out. The primary aim of our assessment was to determine whether the OAGB technique can replace RYGB. The efficacy and safety profile of OAGB was compared with RYGB in adult patients with massive, severe obesity. Complications and postoperative follow up specific to OAGB were identified.

The three selected randomized controlled trials (RCTs) could not confirm the superiority or the non-inferiority of OAGB, compared with RYGB, on the selected efficacy endpoints of weight loss, resolution of comorbidities, and quality of life. Adverse events reported for OAGB included severe nutritional complications and bile reflux that could potentially lead to lower esophageal cancer. In one RCT, the frequency of serious adverse events in the OAGB group was almost two times higher than in the RYGB group.

HAS considered that OAGB carried out with a longer (200 centimeter) BP-limb is not a validated technique for the surgical treatment of massive, severe obesity. Thus, it cannot be considered an alternative to RYGB. There were insufficient data available on OAGB performed with a 150-centimeter BP-limb. Thus, HAS recommended undertaking a multicenter RCT to assess the efficacy and safety of OAGB. Patients who have already undergone OAGB should receive the same follow up as patients who have received RYGB, including close monitoring for nutritional complications and lower esophageal cancer and an endoscopic examination five years after surgery.

Type 2 diabetes results mainly from weight gain in adult life and affects one in twelve people worldwide. In the Diabetes REmission Clinical Trial (DiRECT), the primary care-led Counterweight-Plus weight management program achieved remission of type 2 diabetes (for up to six years) for forty-six percent of patients after one year and thirty-six percent after two years. The objective of this study was to estimate the implementation costs of the program, as well as its two-year within-trial cost effectiveness and lifetime cost effectiveness.

Within-trial cost effectiveness included the Counterweight-Plus costs (including training, practitioner appointments, and low-energy diet), medications, and all routine healthcare contacts, combined with achieved remission rates. Lifetime cost per quality-adjusted life-year (QALY) was estimated according to projected durations of remissions, assuming continued relapse rates as seen in year two of DiRECT and the consequent life expectancy, quality of life and healthcare costs.

The two-year intervention cost was EUR 1,580 per participant, with over eighty percent of the costs incurred in year one. Compared with the control group, medication savings were EUR 259 (95% confidence interval [CI]: 166–352) for anti-diabetes drugs and EUR 29 (95% CI: 12–47) for anti-hypertensive medications. The intervention was modeled with a lifetime horizon to achieve a mean 0.06 (95% CI: 0.04–0.09) gain in QALYs for the DiRECT population and a mean total lifetime cost saving per participant of EUR 1,497 (95% CI: 755–2,331), with the intervention becoming cost-saving within six years.

The intensive weight loss and maintenance program reduced the cost of anti-diabetes drugs through improved metabolic control, achieved diabetes remission in over one-third of participants, and reduced total healthcare contacts and costs over two years. A substantial lifetime healthcare cost saving is anticipated from periods of diabetes remission and delaying complications. Healthcare resources could be shifted cost effectively to establish diabetes remission services, using the existing DiRECT intervention, even if remissions are only maintained for limited durations. However, more research investment is needed to further improve weight-loss maintenance and extend remissions.

According to international experience in the field of hospital-based health technology assessment (HB-HTA), most of the implemented new health technologies must undergo a clinical and economic assessment (CEA) of their viability by creating a mini-health technology assessment report. However, HB-HTA should not be limited only to the initial CEA; further monitoring of the effectiveness of implemented new health technologies is necessary.

We developed a special reporting form for creating a CEA of implemented new health technologies and integrated it into the hospital information system. Indicators of clinical effectiveness are determined individually for each implemented technology. The main indicators of economic effectiveness are financial results (or net profit) and profitability—high-cost and high-tech health technologies have priority for monitoring.

In order to ensure a more detailed and complete CEA of implemented health technologies, the following measures were proposed: (i) before implementing the technology, determine the key clinical effectiveness criteria for further monitoring for each implemented health technology; (ii) if possible, determine comparative technologies (alternatives or analogs) for conducting comparative CEA of the implemented health technologies; and (iii) carry out a prospective CEA of the implemented health technologies with a view to publishing the results.

The organization of a continuous monitoring process that analyzes the effectiveness and usage of new health technologies in hospital practice will allow assessment of the following: the clinical effectiveness and safety of the implemented technologies in comparison with world data; the economic effectiveness of the technology, including an accurate calculation of the payback period for investments; and the “real” data on the effectiveness of implemented health technologies in comparison with the initial request for implementation.

Advanced Therapy Medicinal Products (ATMPs) are innovative biologics (gene, cells and tissue-based products) with the potential to treat diseases with significant unmet clinical need. ATMPs pose distinct regulatory, health technology assessment (HTA) and patient access challenges, hence early identification and prioritization of ATMPs is now recognized as a key concern in England. The National Institute for Health Research Innovation Observatory (NIHRIO) uses a robust methodology to identify and monitor health technologies, including ATMPs that meet the remit of key HTA stakeholders in England. This analysis provides a global overview of the current ATMPs pipeline to administer useful insights for policymakers, funders and innovators.

NIHRIO's database tracks pharmaceuticals from phase I/II onwards, but this analysis focuses on late-stage development. The database (N > 12,000 records) was filtered to identify potential ATMPs using a predefined criteria based on the European Medicine's Agency's classification. Each record is categorized by stage: ‘Active’, (with an estimated three years to European licence); ‘Monitoring’ (in development with no licence date); and ‘Finished’, (output produced/discontinued and no longer tracked). Subsequently, records in ‘Active’ and ‘Monitoring’ were examined further.

Analysis identified 636 ATMPs: five percent ‘Active’, 40 percent ‘Monitoring’ and 55 percent ‘Finished’. ATMPs in the Active/Monitoring stages included: gene therapies (52%), somatic cells (43%) and tissue-engineered products (5%). Of these, 40 percent were oncological with the majority targeting hematological cancers (lymphomas). Prevalent non-oncology areas included musculoskeletal (10%) and ophthalmology (8%). Over one-third of trials were phase IIs, with almost half of all trials were based in the US.

The overarching findings here indicate increasing development of the ATMP pipeline towards indications with significant unmet clinical need. In oncology, the high prevalence of hematological ATMPs is largely due to recent chimeric antigen receptor T cells (CAR-T) innovation. In non-oncology areas, ATMP development is increasing due to advances in regenerative medicine. With a significant number of ATMPs projected to be licenced within three years, and many more in active late-stage trials, HTA bodies and health systems are challenged to prepare for the entry of these innovative therapies.

Current clinical practice is based on guidelines and local protocols that are informed by clinical evidence. This means that clinical variability is reduced, but can lead to inefficient clinical decision-making and can increase medical errors, decreasing patient's safety. The aim of the EXCON project is to investigate the innovative concept of Intelligent Clinical History (ICH), and to develop functional prototypes of high added-value in healthcare services.

The innovative EXCON project will take advantage of recent advances in technologies for coding, structuring and semantizing medical information. Thanks to this new structuring, the EXCON platform will be developed. The final users will be health professionals and other decision-makers. Doctors, nurses, epidemiologists and information specialists will be involved in the development and subsequent validation of the platform.

The EXCON platform identifies profiles of patients with a high probability of ischemic heart disease. In the sample analyzed (n = 4,700), 17 percent of patients were admitted to a cardiology unit with suspected coronary heart disease. Of the patients admitted, 53.7 percent did not have ischemic heart disease at discharge. If we apply the algorithm developed by the EXCON project, 24.8 percent of patients would not have been admitted and did not have ischemic heart disease.

In coming decades, patient management will be impacted by the application of new advanced data analytics tools. This will allow for safer and more efficient clinical management, decrease variability in clinical practice, and improve equity. That is why the development and assessment of these technologies is necessary.

Clinical atherosclerotic cardiovascular disease (ASCVD) patients are judged to be very-high-risk if they had a history of multiple major ASCVD events, or one major ASCVD event with multiple high-risk conditions. Very-high-risk ASCVD patients are under high risk of adverse clinical events and need more attention in the management of secondary prevention. This real-world study aimed at estimating the prevalence of very-high-risk ASCVD and investigating the occurrence of adverse clinical events and associated risk factors among patients with very-high-risk ASCVD in China.

Data were obtained from the Urban Employee Basic Medical Insurance database in Tianjin, China. Very-high-risk ASCVD patients were identified from 2014 to 2015 through the history of ASCVD events and evidence of high-risk conditions, and followed for 24 months. Adverse clinical events were measured by major adverse cardiovascular events (MACE), a composite endpoint of stroke, myocardial infarction (MI) and death. A Cox regression model was used to identify risk factors of MACE, adjusting for potential confounders.

The percentage of clinical ASCVD patients identified as very-high-risk was 35.2 (N = 41,181), while 34,740 patients with continuous enrollment were included (mean age: 67.1 years; 42.5% female). The percentage of patients who had MACE in the 24-month follow-up period was 27.7, with stroke (22.3%) as the most prevalent event followed by death (6.9%) and MI (1.3%). Male gender, older age, and having MI or ischemic stroke (versus unstable angina) as the index major ASCVD event were risk predictors of MACE.

More than one-third of patients with clinical ASCVD are under very-high-risk in China, and among them 27.7 percent experience MACE during a 24-month follow-up period. Male patients, older patients, and patients who had MI or ischemic stroke are under higher risk of experiencing MACE. Future studies are warranted for comparing the differences in characteristics, pattern of drug use, occurrence of adverse clinical events and medical burden between very-high-risk ASCVD patients and ASCVD patients not at very-high-risk.

There are large differences between the prenatal screening strategies for Down Syndrome (DS) in different provinces in China. In Henan province there is a serological triple screening in the second trimester (STS) strategy, while in Shandong province contingent non-invasive prenatal testing (NIPT) screening strategy (NIPT delivered to older pregnant women) is used, and there is a universal NIPT screening strategy in Anhui province. Moreover, many factors varied widely in different regions, such as the proportion of older pregnant woman and the ability of people to pay. This study aimed to determine the cost-effectiveness of current strategy in Shandong compared with strategies in other provinces.

A decision tree model was developed according to the screening strategies in different provinces. Four screening strategies were involved, universal STS strategy, contingent STS strategy, contingent NIPT strategy, and universal NIPT strategy. Cost-effectiveness analysis was conducted from a societal perspective in a simulated cohort of 100,000 pregnant women. The data of costs and epidemiologic parameters were collected from field surveys in Shangdong and a literature review.

The universal STS strategy, contingent STS strategy, contingent NIPT strategy, and universal NIPT strategy could prevent 17.0, 40.0, 46.2, and 53.6 DS births, respectively. There was no strategy dominated by others. The universal NIPT strategy and contingent NIPT strategy would decrease invasive procedures for prenatal diagnosis, resulting in fewer procedure-related miscarriages. The sensitivity analysis showed that the effectiveness of the screening strategy is significantly influenced by the resident's acceptance of NIPT.

From the perspective of maximizing the effect, the universal NIPT strategy is the optimal strategy. But taking into account the resident's and government's ability to pay, contingent NIPT Strategy may be appropriate for the current situation in Shandong. To ensure a better cost-effective advantage in the universal NIPT strategy, the government should provide health education to increase the acceptance of NIPT while controlling the price of NIPT.

Chronic obstructive pulmonary disease (COPD) is a leading cause of morbidity and mortality in China. However, early identification of patients with COPD in the community is challenging. This study used a real-world survey of the Chinese urban adult population to estimate the prevalence of COPD diagnosis or COPD-risk, examine the health outcomes and healthcare resource use of these groups, and investigate the sociodemographic factors associated with these statuses.

Respondents to the 2017 National Health and Wellness Survey in China (n = 19,994) were classified into: COPD (diagnosed), COPD-risk (undiagnosed), and control (undiagnosed, not at-risk) using their self-reported diagnosis and Lung Function Questionnaire (LFQ) score. These groups were compared by healthcare resource use and health outcomes (EuroQol [EQ-5D] and Work Productivity and Activity Impairment questionnaires). Factors associated with being in these groups were investigated using pairwise comparisons (t-tests and chi-square tests) and multivariable logistic regression.

In total, 3,320 respondents (16.6%) had a suspected risk of COPD but did not report receiving a diagnosis. This was projected to 105.3 million people (16.9% of urban adults). Relative to the controls, COPD-risk and COPD-diagnosed respondents had higher healthcare resource use, lower productivity, and lower health-related quality of life (HRQoL) (p < 0.05). Age, smoking, alcohol consumption, weight, exercise, comorbidities, gender, education, employment, and air pollution were associated with increased odds of COPD-risk relative to the controls (p < 0.05).

A substantial group of individuals, undiagnosed, but with a risk of COPD, have impaired HRQoL, lower productivity, and elevated healthcare resource use. A range of sociodemographic factors are predictive of COPD risk, which may support targeted screening. Case-detection tools such as the LFQ may offer a convenient approach for identifying individuals for further definitive testing and appropriate treatment in China.

Synthesis of continuous and time-to-event outcomes is often complicated by the use of multiple outcome scales and heterogeneous reporting of outcomes across trials. Simple methods of evidence synthesis for clinical effectiveness can fail to account for these issues and result in a reduction of the evidence base, which can be further reduced at the cost-effectiveness stage as common outcome measures, such as standardized mean differences, cannot easily be incorporated into the economic decision model. Recent methodological advances for synthesizing continuous and time-to-event outcomes aim to include a greater proportion of the available evidence base within a single coherent analysis.

To assess the statistical methods commonly used in health technology assessment (HTA) and establish whether recent advances in synthesis methods have been adopted in practice, we conducted a review of HTA reports and guidelines published in the United Kingdom (UK) between 1 April 2018 and 31 March 2019 reporting a quantitative meta-analysis (MA), network meta-analysis (NMA) or indirect treatment comparison (ITC) of at least one continuous or time-to-event outcome.

Forty-seven articles were considered eligible for this review. Fifty-one percent of eligible articles reported at least one continuous outcome and 55 percent at least one time-to-event outcome. Twenty-nine articles reported NMA or ITC and twenty-seven reported MA of a continuous or time-to-event outcome. Forty articles included a decision model, of which twenty-seven incorporated evidence from a synthesis of a continuous or time-to-event outcome with eleven informed by a single trial (despite synthesis being conducted).

Uptake of methods to include a greater proportion of the available evidence base within a single coherent analysis in UK HTA reports has been slow. Evaluating health technologies using an evidence-based approach often results in better outcomes for patients. Therefore, HTA analysts and decision modelers must be aware of the expanding literature for synthesis of continuous and time-to-event outcomes and appreciate the limitations of simpler approaches.

After the introduction of the seven-valent pneumococcal conjugate vaccine (PCV7) in the United States (US) in the year 2000, the incidence of invasive pneumococcal disease (IPD) caused by the seven vaccine serotypes declined by 80 percent in vaccinated children and 30 percent in unvaccinated adults. A transmission dynamic equation model developed in 2009 captured the direct and indirect effects of vaccination in the early years after vaccination. Subsequently, the vaccine program switched to the 13-valent PCV and adult PCV13 vaccination. This work explores the accuracy of the mathematical model to predict long-term IPD due to changes in US immunization practices.

The model simulates the acquisition of asymptomatic carriage of pneumococci and the development of IPD among individuals aged <2, 2–4, 5–17, 18–49, 50–64, and ≥65 years. Pneumococcal serotypes were stratified into three categories: PCV7-type (4, 6B, 9V, 14, 18C, 19F, and 23F), PCV6-type (1, 3, 5, 6A, 7F, and 19A), and non-PCV-type (all others). Model parameters were calibrated using US IPD surveillance data from 1998–2006. Model results were compared to observed epidemiology.

The model was previously shown to predict observed IPD well through 2007. After adjusting model parameters for PCV13 efficacy and adult vaccine coverage, modeled IPD closely replicated observed IPD. Observed baseline pre-vaccine incidence for children <2 years of age was 192 cases/100,000 and 13.5 cases in 2016, versus 18.5 cases estimated by the model. Similarly, observed versus modeled cases in the ≥65-year-old age group were 24 and 23.6 cases.

This epidemiologic model accurately simulates the observed US IPD surveillance data 17 years after initial introduction of PCV, highlighting the direct and indirect benefits of vaccination. Well-constructed mathematical models can accurately replicate real-world scenarios. Key input parameters of these models can then be modified to predict the impact of alternate scenarios, providing insights to inform public health policy-making.

Secondary hyperparathyroidism (SHPT) is a consequence of non-dialysis chronic kidney disease (ND-CKD), causing disturbances in metabolic parameters including increased phosphate and parathyroid hormone (PTH) and reduced calcium serum level, which can cause bone disease, extra-skeletal calcification and increased cardiac disease risk through vascular and visceral calcification. According to Kidney Disease Improving Global Outcomes (KDIGO) guidelines in 2017, treatment with paricalcitol is no longer recommended in early stage CKD due to increased risk of hypercalcemia. Prolonged release calcifediol (PRC) has been developed as a novel treatment for SHPT in ND-CKD. The objective of this study was to compare the efficacy and safety of PRC versus paricalcitol by assessing biomarkers such as PTH, calcium and phosphate.

To identify relevant randomized control trials (RCTs) to be included in the network meta-analysis (NMA), a systematic literature search was performed in PubMed. All analyses were performed with a frequentist random-effects NMA. Comparisons were made between the overall treatment effects of the two drugs (including all studies, with fixed and titrated dosage regimens), and between low fixed doses (PRC: 30 μg/day, paricalcitol: 1 μg/day) and high fixed doses (PRC: 60 μg/day, paricalcitol: 2 μg/day).

Nine RCTs, comprising a total of 1,426 patients, were included in the analyses. No statistically significant differences in PTH reduction were found. Paricalcitol showed significantly larger increases in calcium when overall effects and high doses were analyzed. No differences in effects on phosphate were observed. Although effect sizes and statistical significance levels vary somewhat across analyses, the general pattern of similar PTH reductions and larger increases in calcium from paricalcitol are observed in all analyses.

The non-inferiority shown by PRC in lowering PTH and the tendency to increase calcium serum levels observed with paricalcitol treatment indicates that PRC might be used as an equally effective but potentially safer alternative to paricalcitol in treating patients with SHPT.