Despite increased healthcare systems costs, limited opportunities for health economics training are available to healthcare professionals. From 2016-2018, with a grant from the Brazilian Ministry of Health, the Federal University of Goias with 7 other universities, implemented the distance learning Postgraduate Certificate in Health Economics for Health Care Professionals (PCHE) aimed at enhancing technical capacity of professionals working in the Brazilian Public Healthcare System (SUS).

This is a descriptive and qualitative assessment of the PCHE implemented in Brazil 88 healthcare professionals working in SUS and involved in decision making in all levels of managament were enrolled in a health economics training, through long-distance learning strategy. We present course metrics, describe its workload, content, modalities and structure of training.

PCHE was structured with 3-day workshops introducing each of the modules, during which students were also evaluated regarding the previous module content. With a total workload of 360 hours, structured in four modules: Public Health and Epidemiology; Introduction to health economics and healthcare funding; Management of healthcare resources; and Healthcare economic evaluation. The module coordinator was resposible for supervision of course materials development, workshop, distance based tutoring activities, and evaluation. Course material included theorethical content and practical tools for economic evaluation and health technology assessment in the workplace, applying problem-based learning strategies. Certificates were granted to students with 75 percent presence and approved in all modules, and final papers approved by an examination board. Each module was completed in 8 weeks (90 hours/module). Within groups of 20 students, tutors performed communication witn chats twice weekly and discussion forums by topic.. A total of 88 students were enrolled. Drop-out rate was 35.2 percent (n = 31). Additional 10 students did not pass the exams. In total, 47 students completed the training.

Health economics training through distance learning is a more efficient use of resources with good results.

Principal component analysis (PCA) is used for dimension reduction and data summary. However, principal components (PCs) cannot be easily interpreted. To interpret PCs, this study compares two methods to approximate PCs. One uses the PCA loadings to understand how input variables are projected to PCs. The other uses forward-stepwise regression to determine the proportions of PC variances explained by input variables.

Two data sets derived from the Canadian Health Measures Survey (CHMS) were used to test the concept of PC approximation: a spirometry subset with the measures from the first trial of spirometry; and, full data set that contained representative variables. Variables were centered and scaled. PCA were conducted with 282 and twenty-three variables respectively. PCs were approximated with two methods.

The first PC (PC1) could explain 12.1 percent and 50.3 percent of total variances in respective data sets. The leading variables explained 89.6 percent and 79.0 percent of the variances of PC1 in respective data sets. It required one and two variables to explain more than 80 percent of the variances of PC1, respectively. Measures related to physical development were the leading variables to approximate PC1 and lung function variables were leading to approximate PC2 in the full data set. The leading variable to approximate PC1 of the spirometry subset were forced expiratory volume (FEV) 0.5/forced vital capacity (FVC) (percent) and FEV1/FVC (percent).

Approximating PCs with input variables were highly feasible and helpful for the interpretation of PCs, especially for the first PCs. This method is also useful to identify major or unique sources of variances in data sets. The variables related to physical development are the variables related to the most variations in the full data set. The leading variable in the spirometry subset, FEV0.5/FVC (percent), is not well studied for its application in clinical use.

For the last decades, health technology assessment has been strongly promoted in order to provide evidence-based rather than eminence-based healthcare. However, when it comes to implementing interventions that are based on processes and behavior rather than products, importing evidence-based interventions is likely to fail because it ignores the strong influence of contextual factors. In a recently started research project, an alternative approach is tested.

The project aims at improving identification and support of children who have parents with a mental illness in an Austrian region (Tyrol). A theory- and stakeholder-driven approach has been designed in order to co-develop, implement and evaluate practice approaches for improving the situation for children. The former addresses the questions whether, how and why suggested practices may work and the later brings together evidence and practice to develop interventions that are feasible and take the regional context and service settings into account.

Based on evidence from various sources (literature, international and local stakeholder interviews), theories that describe the key mechanisms of action to influence outcomes for children have been developed. A regional and interdisciplinary group of practitioners and people with lived experience has been established to facilitate the development of the theory of change and practice approaches for the region and local service settings. The final practice model is then monitored in the respective organizations with implementation support from the research team.

Theory-driven and co-designed-based approaches are a feasible alternative to ‘off-the-shelf’ evidence-based practices for supporting decision makers in implementing complex interventions. However, they require a broad variety of skills within the research team as well as willingness to accept uncertainties of the final outcomes produced, which can also be of risk to funders. The ongoing project will demonstrate whether the developed practices will be implemented successfully and result in benefits for the affected children.

To provide insights into the attributes of health-related quality of life (HRQoL) research within the context of economic evaluations for a potential national health technology assessment process in South Africa, and make evidence generation recommendations.

A systematic review was conducted in January 2019 using Medline, the Web of Science (WoS) Core Collection and the South African SciELO collection via the WoS Platform, and in the Cochrane Library. No time restrictions were applied. Duplicate records were removed before first- and second-pass screening by two reviewers working independently.

The review identified 123 publications representing 104 studies since the first-published article appeared in 1996. Only eight studies were randomized controlled trials, most were cross-sectional (n = 54). The EQ-5D, SF-36, and WHOQOL-BREF were the most used HRQoL instruments (n = 35, n = 23, and n = 10, respectively). Instruments were frequently administered in multiple languages, reflecting the cultural groups in which the study was conducted, with the English version of instruments used most often. Studies were predominantly conducted within the public health sector (n = 67), in the Western Cape province (n = 46), in adults (n = 92) and people with HIV (n = 24).

South African specific HRQoL studies have been conducted in a range of settings and populations using mostly generic HRQoL instruments in multiple languages. These studies may provide generalizable, real-world data due to their observational nature. However, more comparative and longitudinal studies should be conducted as this is preferred for economic evaluations and patient, disease, and treatment characteristics should be reported in full.

Australia and Canada have parallel submission processes that allow companies to submit their dossier to the respective Health-Technology-Assessment (HTA) body before the market authorization is issued, aiming to provide timelier access to drugs. The objective of this study is to investigate the associations of parallel submissions with the rollout times and HTA recommendations of new active substances (NASs).

Public data from 208 HTA appraisals were collected from the Pharmaceutical Benefits Advisory Committee (PBAC) from Australia and the Canadian Agency for Drugs and Technology in Health (CADTH) for NASs obtaining regulatory approval between 2012 and 2020. We implemented multivariable logistic and linear regression models allowing for type of submission (parallel or sequential) and jurisdiction (Australia and Canada) to examine associations with first HTA recommendation (positive and positive with restrictions vs negative) and rollout time (regulatory submission to HTA recommendation), respectively.

A total of 121 appraisals followed a parallel submission. The therapeutic products that most used a parallel submission were antineoplastic agents (Anatomical Therapeutic Chemical Code=L;47.11%). A similar proportion of chemical and biotechnological products followed parallel submissions.

Multivariable linear regression showed that parallel submission presented 14-months decrease in rollout time when compared to sequential (p<0.001). Regarding jurisdictions, longer rollout times were seen for Canada when compared to Australia (β:4.0, p-value=0.024).

Parallel submission showed no association with HTA recommendation. Canada had higher odds of receiving a positive recommendation (Odds Ratio:4.84, 95% confidence interval:2.63-9.18) when compared with Australia (p<0.001).

Antineoplastic agents were the main products using parallel submissions. Appraisals following a parallel submission showed a considerably faster rollout time than those following the traditional sequential submission, illustrating the advantage of this approach for dossier submission. The submission type did not have an impact on the HTA recommendation, indicating that although quicker, the HTA decision was not affected. Canada has a more restrictive criteria regarding the timing of dossier submission compared to Australia, which may lead to disparities in their rollout time.

Postoperative delirium has been associated with poorer long term survival in Transcatheter aortic valve replacement (TAVR) and Surgival aortic valve replacement (SAVR) patients. However, its effect on hospitalization costs and length of stay in these populations has not been formally assessed.

Using the Medicare Provider Analysis and Review File, we retrospectively analyzed elderly (80 years of age and older) Medicare patients receiving TAVR and SAVR in the United States during the 2015 fiscal year. ICD-9-CM codes were used to identify postoperative delirium diagnoses. The incremental hospital resource consumption, measured as hospital cost and length of stay, was estimated for patients with postoperative delirium during their TAVR or SAVR index hospitalization. Multivariate regression models were used for the adjusted cost estimates controlling for patient demographics, comorbidities, and complications.

A total of 21,088 claims were available for analysis (12,114 TAVR and 8,974 SAVR). The mean age of the TAVR group was older compared to the SAVR group (87 versus 84; p < .001) and TAVR patients presented with a higher comorbidity burden (Charlson Index score 3.0 versus 2.1; p < .0001). TAVR patients experiencing postoperative delirium during the index hospitalization was 1.6 percent compared to 3.6 percent of surgical patients (p < .0001). For the overall cohort, the regression adjusted incremental cost of postoperative delirium was (USD15,592; p < .0001). Patients experiencing delirium also had significantly longer hospital length of stay (4.16 days; p < .0001). When stratified by treatment approach, the adjusted incremental cost was USD13,862 for TAVR (p < .0001) and USD16,656 for SAVR (p < .0001).

While infrequent, postoperative delirium significantly increased hospital cost and length of stay following transcatheter or surgical aortic valve replacement (AVR). Despite a significantly higher comorbidity burden, TAVR was associated with lower postoperative delirium rates compared to SAVR. Moreover, post-TAVR delirium may be associated with less resource consumption than post-SAVR delirium. Future studies should seek to determine whether general anesthesia avoidance in appropriately selected transfemoral TAVR patients can further decrease rates of delirium.

Long-chain fatty acid oxidation disorders (LC-FAOD) are a group of serious diseases in which patients are at risk of metabolic decompensation, resulting in cardiomyopathy, hypoglycemia and rhabdomyolysis and premature mortality. In addition, LC-FAOD are a burdensome disease that adversely effects quality of life (QoL) via symptoms of muscle pain, fatigue, and a difficult diet. Previous studies have reported improvements in QoL during treatment with triheptanoin as measured by short form (SF) instruments. This study sought to convert the QoL measure into a utility value for a sample of patients with LC-FAOD at baseline and 78 weeks during treatment with triheptanoin.

In an open-label Phase 2 study of triheptanoin (UX007-CL201, NCT01886378), caregivers of patients (n = 9/23 enrolled) or patients aged 18+ years (n = 6/6 enrolled) completed the SF-10 or the SF-12v2, respectively. Component summary scores at baseline and 78-week during treatment period were converted to EuroQol-Five Dimension (EQ-5D) utility (with zero representing death and 1.0 perfect health) using a published conversion algorithm (Lawrence and Fleishman 2004). Generalized linear mixed-effects models with individual-level random effects were used to estimate the utility values.

At baseline, patients’ utility was estimated to be 0.365 (standard error [SE] = 0.090) compared with 0.629 (SE = 0.072) 78-weeks during treatment, a significant improvement (p = 0.0073). In a sensitivity analysis using SF-12v2 data only (that is, only adult patients), utility estimates were 0.498 (SE = 0.084) at baseline versus 0.690 (SE = 0.068) during treatment (p = 0.0499). No patients had a major clinical event during the SF instrument recall period, indicating the benefit was driven by day-to-day improvement in QoL.

Treatment with triheptanoin resulted in a substantial improvement in daily QoL for patients with LC-FAOD. Limitations of this study include that the estimation of utilities is from a single-arm study with small sample sizes and that the assessment of utility was based on a conversion algorithm rather than direct measurement. Nevertheless, results indicate significant improvement in QoL for patients treated with triheptanoin.

The Canadian Agency for Drugs and Technologies in Health (CADTH) pan-Canadian Oncology Drug Review (pCODR) plays an important role in public reimbursement decision-making for oncology drugs in Canada. This research studies the relation of positive pCODR decisions to new cancer treatment and their subsequent inclusion in Canada's public drug plans.

We studied all oncology drugs that received an approval from Health Canada and were reviewed by the pCODR from inception till 26th Sep, 2017. The data was obtained from CADTH and Health Canada. Data such as indication, submission type and date, recommendation date, final recommendation, and subsequent provincial funding status was extracted and analyzed. Impact was evaluated by analyzing the percentage of drug submissions with assessment outcome (positive recommendation rate and conditional recommendation rate) and time taken for the final decision (recommendation gap). The percentage of drugs included in public formulary after positive recommendation by pCODR (coverage rate) and the gap in days from positive recommendation to subsequent coverage in provinces (coverage gap) was also assessed.

Among 119 drugs reviewed by pCODR, the positive recommendation rate was eight percent. Nine applications comprising seven drugs for six indications received positive recommendations, and genitourinary treatments received maximum positive recommendations. The conditional recommendation rate was 52 percent; 62 applications of 45 drugs for 46 indications received conditional recommendation. Lymphoma and myeloma treatments received maximum conditional recommendations. The average recommendation gap for positive and conditional recommendations was 180 and 172 days, respectively. The coverage rate for drugs with positive recommendation was 100 percent for all provinces except 89 percent for Newfoundland and Labrador, and 67 percent for Prince Edward Island. Among the provinces, British Columbia had a maximum of 433 days and Saskatchewan has the minimum of 165 days coverage gap.

Despite Health Canada's approval, only a fraction of oncology drugs receive positive pCODR recommendation; furthermore, provincial drug plans take time to include these in the reimbursement formularies. While health technology assessment is crucial for appropriate allocation of limited resources, efforts should also be made to reduce access barriers, particularly to positively recommended oncology drugs inclusion in provincial formularies.

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.

Public consultation is one of the phases provided by the law that rules the health technology incorporation process in Brazilian Public Health System (SUS). In the Brazilian model, anyone can participate as long as he/she identifies her/himself. During the decision-making process these suggestions are analyzed by the National Committee for Health Technology Incorporation (CONITEC) and, sometimes, they are responsible for changing a preliminary recommendation for a technology. This study aims to identify the health technologies for which CONITEC revised the initial negative recommendations due to the contributions received during the public consultation.

A descriptive study using as input data the information on coverage decisions available on the CONITEC website.

Since CONITEC's creation until October 2017, CONITEC enacted 241 public consultations. Fifteen cases of change to the preliminary negative recommendation were found and among these eight (53 percent) had the economic studies or proposed technology price reconsidered by the companies. In the other seven decisions, the Board also regarded as important the reasons for changing the initial recommendation: new evidence on efficacy and safety as well as the analysis of different outcomes previously unconsidered in the preliminary assessment.

During the public consultations, besides technical-scientific information, personal experiences and opinion reports on each health technology analyzed, CONITEC received new price offers and economic studies from the applicants. This new material has allowed, in some cases, these technologies to become competitive and to be included as alternatives to those already available, provided there is no clinical impairment. This study reinforces the importance of the public consultation and social participation in the process of health technologies incorporation in Brazil, considering its capacity to add new information to the decision-making process.