An environmental scan conducted by the Canadian Agency for Drugs and Technologies (CADTH-March-2019) revealed that several health technology assessment (HTA) organisations are currently developing standard health technology reassessment (HTR) processes. Here we present methods used to conduct an HTR of a prioritization programme for non-immediate life-threatening urgent surgeries implemented in 2017 at a tertiary referral hospital in (Quebec-Canada). This HTR initiative was conducted by a regional HTA unit to optimize the programme efficiency and resources utilization as well as to motivate change in the clinical community of other hospitals within its healthcare network. Patient and healthcare personnel satisfaction levels towards the programme were also considered.

In this case study, HTR methods and outputs were elaborated using elements presented in the CADTH environmental scan and relevant publications identified through PubMed and in the grey literature. Documents in English and French, published between January 2002 and March 2019 were considered. Key stakeholders were consulted to identify barriers of the programme implementation to other hospitals in regards to aspects related either to the local medical practice or organizational factors.

The prioritization process was conducted using the same tool applied for HTA appraisal with the additional criterion that the HTR could facilitate the programme implementation. The research processes used in this HTR included: i) systematic review of the literature, ii) hospital database search (efficacy and resource utilization), iii) perceptions of healthcare teams and patients. HTR outputs consist of specific recommendations on implementation barriers and methods to monitor the impacts of the programme.

In this evolving field, sharing lessons from HTR methods provides information to develop standard adaptable processes to different contexts. Hence, this work applies HTR to a healthcare programme while most of the literature focuses on the HTR processes on drug and interventional medicine disinvestment. These elements represented HTR methods used from prioritization appraisal, research processes for evaluation and outputs used to plan the implementation and finally monitoring from a regional HTA unit. It also showcases that HTR being conducted as a structured evidence-based assessment adds value to a healthcare programme and could also facilitate its implementation.

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.

Prevalence of dyslipidemia in Chinese adults is increasing rapidly. Dyslipidemia is one of the most important risk factors for acute myocardial infarction (AMI), which represents a serious disease burden to the country. However, there is no published research on the costs of Chinese patients diagnosed with AMI combining dyslipidemia. This study aimed to report key findings of the disease burden in China, including direct medical costs and direct non-medical costs.

Six hospitals from different geographic areas were selected in China for data collection. Patients who were hospitalized due to AMI combining dyslipidemia from January 1 2016 to December 31 2016 in the six sites were enrolled. Direct medical costs including inpatient and outpatient costs were extracted through electronic medical records; medical costs occurred in other healthcare institutions and direct non-medical costs were collected by a face-to-face questionnaire survey. Results were analyzed with descriptive statistics.

Data of 900 patients were analyzed. There were more males (78.40%) than females. The mean age was 62.1 (SD: 11.5). The times of inpatient and outpatient per year were 0.57 and 8.67, respectively. Medium direct medical costs and medium direct non-medical costs were 31,440 RMB (Interquartile range (IQR): 21,533–48,202) (4,443 USD: 3043–6812) and 665 RMB (IQR: 351–1328) (94 USD: 50–188), respectively; while corresponding medium indirect costs per year were 659 RMB (IQR: 226–1579) (93 USD: 32–223).

This is the first study comprehensively analyzing the disease burden of patients diagnosed with AMI combining dyslipidemia in China. The results suggested that the medical cost of this population is still high. Hospitalization cost accounted for 81 percent of the total cost, which was around 1.3 times of the annual per capita disposable income over the same period. Therefore, the importance of providing effective clinical management as well as dyslipidemia prevention and control intervention should be highlighted, especially for middle-aged and elderly males with dyslipidemia.

With the aging of population, miniaturization of family size and changes of diseases spectrum, the demand for long-term care of Chinese elderly is increasing, which is challenging the existing long-term care system. China is currently carrying out pilot work for a long-term care insurance system, and Jingmen is one of the pilot cities, however more detailed research on payment is needed. Therefore, this paper draws on case-mixed-adjusted prospective payment system to provide designs for long-term care insurance in pilot cities.

Adopting a case analysis method, this paper focuses on system for payment of Skilled Nursing Facility under Part A of the Medicare program—Patient Driven Payment Model, and discusses the implementation plan of a long-term care insurance in Jingmen City from the perspectives of payment methods, payment grouping and payment standards.

Currently Jingmen adopts per-diem payment for long-term care insurance, so it is necessary to establish a payment based on population characteristics and demands. So, the patients should be classified into a group for each of the five case-mix adjusted components: physical therapy, occupational therapy, speech therapy, nursing and non-therapy ancillary. In addition, this payment model also includes a “variable per diem adjustment” to account for the changes in patient costs more accurately.

The theoretical system of a long-term care insurance payment method is developed, and a localization plan for case-mixed-adjusted prospective payment system for long-term care insurance is provided. Therefore, Jingmen long-term care insurance payment should adopt “case-mixed adjustment”, strengthening the relationship between individual clinical characteristics and payment.

Malignant tumors have become a major public health problem and their treatment cost is increasing rapidly in China, but treatment aimed at healing diseases or extending patients’ life. There is little empirical research on utilization of healthcare resources of terminally ill cancer patients. In order to explore the optimal treatment decision for patients and provide information for relevant decision makers, this study analyzed the consumption status of medical resources in patients with cancer during the whole treatment period, and the current medical resource utilization efficiency in different levels of hospital for end-stage cancer patients.

This study was based on the clinical treatment and payment data of 2,536 cases of patients with lung cancer from the medical insurance database during the period of 2007 to 2014 in Hubei province. We retrospectively analyzed patients’ medical expenditure and utilization of different medical resources during their whole treatment period as well as at the end stage.

The per capita inpatient expenditures of patients under 50 years old was 193,000 CNY (27,451 USD), while that of the patients over 70 years were 80,000–90,000 CNY (11,379–12,802 USD). Secondly, the medical expenditures spent during the last 6 months of life accounted for 66.1 percent of the total expenditures. Lastly, the medical expenditure spent in tertiary hospitals accounted for 95.3 percent of the total expenditure, and the expenditure was 14,200 ± 17,030 CNY (2,019.82 ± 2,422.36 USD) per visit.

Population aging is not the only factor causing the rise of medical expenditure. The unclear objectives of treatment and the reverse of medical resource allocation are also important factors to boost the growth of medical expenditure. It is necessary to improve the healthcare insurance payment system, strengthen the capacity of primary medical institutions, and develop the palliative care system in China.

The scientific application of clinical evidence-based guidelines can reduce the variability of clinical practice, and standardize clinical diagnosis and treatment pathways. At present, many evidence-based guidelines on Chronic Obstructive Pulmonary Disease (COPD) prevention have been issued in countries around the world, but the procedures and evaluation strategies developed by different guidelines are not the same. This study aimed to evaluate the quality of published clinical practice guidelines (CPGs) relating to COPD using the Appraisal of Guidelines for Research and Evaluation II (AGREE II) instrument.

Databases were systematically searched PubMed, EMBASE, Wan Fang, and CNKI as well as guidelines websites on COPD prevention and treatment. The search period was from inception of the database up to May 2019. The inclusion criteria for this study are as follows: (i) published and in accordance with the definition of the practice guidelines; (ii)the main target population is COPD patients with the diagnostic criteria of the 2019 edition of the global initiative for COPD (GOLD), and the content of the guideline is related to the prevention and treatment practice of COPD; (ii) the same guide is included in the latest updated version; (iv) the published language is English or Chinese. Guidelines that met these inclusion criteria were evaluated for the quality of the AGREE II guidelines. Then, a descriptive analysis was made of the consensus that exists in the guidelines.

A total of fifteen guidelines/Consensuses Statements were included in the study. Two guidelines were assessed as recommended, eleven guidelines were assessed as recommended with modifications and two guidelines were not recommended. The mean scores of the included guidelines in the six domains (scope and purpose, personnel involved in guideline development, rigor of development, clarity, applicability, independence) were 90 percent, 72 percent, 49 percent, 96 percent, 60 percent, 69 percent, respectively. Thus, the study identified a consensus that disease risk factors and recommended interventions were mentioned in the guidelines, and that they comprehensively evaluated the quality of guideline reporting to provide reference for standardizing the development of practice guidelines for COPD in China.

The overall methodological quality of COPD CPGs should be improved. The key recommended areas for improvement include standardization of guideline report writing and synthesis of the latest and best evidence, to develop CPGs for COPD to improve the quality of clinical diagnosis and treatment for COPD.

Primary immunodeficiency diseases (PIDs) are a heterogeneous group of over 200 disorders with defects in the function and/or development of the immune system. Although early screening is imperative for improving therapeutic efficiency and preventing disease-associated morbidity, its widespread use has been limited, owing to the low incidence of PIDs. It is particularly important to evaluate the cost-effectiveness of PIDs screening for newborns. The aim of this study was to provide an overview of the existing cost-effectiveness evidence on newborn screening of PIDs and to provide reference for decision-makers in China and other developing countries.

We conducted a systematic review using three electronic databases (PubMed, CNKI, and CSPD) of cost and cost-effectiveness studies of PIDs screening published during 2000–2019. Two reviewers independently searched databases and screened titles, abstracts and full texts; a third reviewer resolved disputes when necessary. The initial search returned 124 references, of which 10 full articles were included in the review. Five of the studies conducted analyses using model-based techniques.

Severe combined immunodeficiency (SCID) was the predominantly studied condition (80%). Most studies (70%) examined the T-cell receptor excision circle (TREC) assay. A healthcare system's perspective was commonly used (50%) for cost calculations, and most studies (50%) were US-based. The majority (67%) of the studies found the TREC assay an effective screening tool for SCID, but the incremental cost-effectiveness ratio (ICER) varied across screening test specificity and disease incidence.

Evidence from the published literature demonstrated that newborn screening for PIDs generally appeared to be cost-effective, and most importantly, it is lifesaving and allows children with PIDs an opportunity to live a healthier life. However, the type of PIDs included in this study were limited and most studies were done in developed countries whose health systems are different from low-/middle-income countries (LMIC). Further research is required to identify the cost-effectiveness of PIDs screening both in developed and developing countries.

China bears a considerably high burden of multidrug-resistant tuberculosis (MDR-TB). Second-line anti-TB drugs are urgently needed yet domestic MDR-TB drugs are expensive and lack policy support. Patients’ living conditions are closely related to the drug affordability. The national TB prevention programs should play a critical role. The purpose of this study is to measure the cost of treating MDR-TB patients under different treatment schemes and price sources. The results of this study are expected to inform the relevant drug protection policies and provide inputs for further cost-effectiveness analyses.

Based on the treatment plan of China's Multidrug-Resistant Pulmonary Tuberculosis Clinical Path (2012 edition) and the World Health Organization (WHO) Drug-Resistant Tuberculosis Treatment Guide (2018 edition), the treatment costs of MDR-TB were measured under different scenarios. Catastrophic health expenditure was then calculated if the treatment cost exceeds 40 percent of the household's non-subsistence income. National, rural and disposable income per capita in 2018, were used to represent Chinese patients’ affordability.

Under varied treatment schemes and market price sources in China, the total costs for MDR-TB patients range from 19,401 to 126,703 CNY [2,853 to 18,633 USD] per person. Under current prices, all treatment schemes recommended by the WHO will incur catastrophic costs for Chinese MDR-TB patients. Significant differences were found between rural and urban areas as 52.8 percent of the treatment listed in the 2012 China Guideline would lead to catastrophic cost for rural patients but not urban ones.

Our study concludes that the domestic drugs are more expensive than the international purchase price and the treatment of MDR-TB imposes substantial economic burden on patients, especially in the rural areas. The results of the study also indicate that it is urgent for the state to emphasize government responsibility and initiate centralized procurement for price negotiations to reduce the market price of MDR-TB drugs. The urban-rural gap should also be addressed in the design of future policies to ensure the drug affordability for all patients in need.

Breast cancer is the most frequent cancer among women globally, impacting 2.1 million women each year, causing the greatest number of cancer-related deaths among women. In Malaysia, the new cases of breast cancer comprised of 32.7 percent of all new cancer cases in women as reported by The International Agency for Research on Cancer (IARC). The recurrence rate was about 16.4 percent post-mastectomy. This early assessment is to evaluate the effectiveness and safety of a breast cancer vaccine.

A systematic review was conducted. Searches were done through PubMed, Medline and ClinicalTrial.gov. The articles were selected based on inclusion and exclusion criteria and appraised using Critical Appraisal Skills Programme (CASP) checklist. More than twenty cancer vaccines under development were identified.

The most advanced breast cancer vaccine is Nelipepimut-S (NPS). In a Phase 2b clinical trial, improvement disease-free survival (DFS) in the NPS group was 89.8 percent. The DFS rate in the NPS group was 92.6 percent for triple negative patients. Median DFS in the NPS group [hazard ratio (HR): 0.26 (95% confidence interval, Cl: 0.08–0.81)] showed a significant difference. A projection study showed the cost-effectiveness will be 90 percent success if the cost less than USD1,000 per patient. No safety issues were reported.

Early assessment showed potential benefit in patient with triple-negative breast cancer (TNBC). However, further research is required to ensure its efficacy, safety, and cost-effectiveness.

The Health Technology Assessment (HTA) Department of the “State Expert Center of the Ministry of Health of Ukraine” was established in 2019 for conducting assessments and development of recommendations for informing decisions of the Ukrainian Ministry of Health regarding financing of the health technologies. Since 2015, procurement of medicines by the state budget in Ukraine was carried out through international specialized organizations. The central procurement program covers forty-one programs for different disease areas. The list of medicines and medical devices purchased were based of procurement agreements with specialized organizations approved by Cabinet of Ministers Decree of Ukraine № 255 of 13 March 2019.

Descriptive analysis of methodological processes around rapid review methods in HTA used for the procedure of central procurements of medicines. In the process of conducting rapid assessment we use a modern tool based on the HTA Core Model® for Rapid Relative Effectiveness Assessments version (V3.0). HTA Guidance in Ukraine was developed in 2017.

The list of government programs, for which rapid review methods in HTAhave been conducted, included following diseases: adult and pediatric oncology, juvenile rheumatoid arthritis, hemophilia, and orphan diseases. Reports include an overview of comparative efficacy, effectiveness and safety, as well as analysis of reports from HTA agencies and thresholds, budget impact for informing decisions for development of the central procurement programs in 2019. The HTA Department conducted more than forty reviews in less than 6 months.

Use of rapid review methods in HTA in Ukraine for informing decisions for the central procurement programs of medicines is the perspective for rational resource allocation and spending. This increases and improves patients’ access to effective, safe and cost-effective medicines.

The Health Technology Assessment (HTA) Department of the “State Expert Center of the Ministry of Health (MoH) of Ukraine” was established in 2019 for conducting assessments and development of recommendations for informing decisions for the Ministry of Health of Ukraine (MoH) regarding the financing of health technologies based on HTA. The study aimed to conduct cost analysis and compare the treatment costs of two available dosage forms of tocilizumab for subcutaneous and intravenous administration for systemic juvenile idiopathic arthritis in Ukraine. Currently there is a central procurement program financed by the state budget with pharmaceuticals provided to patients with juvenile idiopathic arthritis approved by the Cabinet of Ministers Decree of Ukraine №255 dated 13.03.2019.

The cost analysis was carried out for tocilizumab over a 1-year horizon per one patient. The analysis included drug manufacturers' prices from the registry of the MoH dated 19.09.2019. The annual number of tocilizumab vials for intravenous infusion for each weight category of patients is approved by the order MoH №334 from 14 February 2019. The direct medical costs were included in the analysis omitting cost of administration.

The direct medical costs of treatment with intravenous tocilizumab per one patient for one budgetary year ranged from between USD7,563.83 and USD30,255.30 depending on patient's weight that was in the range of 10–80 kg. The direct medical costs of treatment per one patient for one year with subcutaneous tocilizumab was USD7,782.40 for patients < 30 kg and USD15,564.80 for patients ≥ 30 kg.

The introduction of subcutaneous tocilizumab can potentially lead to cost savings on average USD 4,041.97 (34.2%) for patients < 30 kg and USD 5,245.82 (25.2%) for patients ≥ 30 kg per one patient for 1-year treatment compared to the intravenous route. Intravenous tocilizumab has an economic advantage over the subcutaneous route solely for the pediatric population of certain weight categories. The cost of intravenous tocilizumab was USD 218.58 lower (2.9%) for patients ≤ 13 kg and USD 2,320.25 lower (17.5%) for patients with weight 31–35 kg, compared to the cost of subcutaneous tocilizumab.