Differences between healthcare datasets in structure, content, and coding systems are widely recognized as significant barriers to generating robust evidence for regulatory and medical decision making. As a result, there is a growing interest in using common data models embedded within large data networks. By standardizing the structure, contents, and semantics of disparate healthcare databases, common data models like the Observational and Medical Outcomes Partnerships common data model (OMOP-CDM) enable multidatabase studies to be undertaken at speed and in a transparent way. To date, little attention has been given to their potential role in health technology assessment (HTA).

We identify the uses of observational data in generating evidence in HTA, some common analytical challenges faced in their estimation, and the infrastructural, technical, and data reusability constraints that limit its wider use. We discuss where and how the OMOP-CDM could overcome these barriers in relation to different types of evidence requirements.

The OMOP-CDM increases the interoperability of otherwise disparate datasets, allowing reliable evidence to be generated from multidatabase studies at speed and transparently. The current analytical tools are best suited for clinical characterization and population-level effect estimation. Further developments to these tools are required to support analyses common in HTA like parametric survival modeling. Differences in costing methods as well as the structure of healthcare delivery between countries may limit the feasibility and value of standardization.

The OMOP-CDM has the potential to support reliable and timely evidence generation in HTA. The analytical tools should be further developed to support common HTA use cases.

Ankylosing spondylitis (AS) is a common disease that causes pain and affects productivity. Tumor necrosis factor-α (TNF-α) like adalimumab can bring better clinical efficacy and improve quality of life. Adalimumab is likely to be covered by health insurance. It is necessary to assess the impact of adalimumab for patients with AS on the medical insurance budget in China. Our research aims to give support evidence for policy-making.

From the perspective of medical insurance payers, a budget impact model was established to evaluate the impact of adalimumab for the treatment of adults with severe active AS that has responded inadequately to conventional therapy. The time horizon was 5 years (2020–2024). The cost of measurement included drug and treatment costs for adverse events. Scenario analysis was conducted to evaluate the results under different drug price reimbursement ratios and treatment ratios.

Based on the current price of adalimumab (CNY 3,160 [USD 446]/unit), under the reimbursement ratio of 70 percent, adalimumab will increase medical insurance expenditure by CNY 162 [USD 22] million, CNY 152 [USD 21] million, CNY 114 [USD 16] million, CNY 100 [USD 14] million and CNY 88.11 [USD 12] million in the next 1–5 years, respectively. The increased medical insurance expenditure accounts for 0.091, 0.085, 0.064, 0.056, and 0.049 percent of the annual medical insurance expenditure in the next 1–5 years, respectively, which is assumed to be equivalent to the expenditure in 2018 of CNY 1782.2 [USD 251] billion.

The budget impact of adalimumab for AS on medical insurance expenditure is limited, and including adalimumab in the medical insurance catalogue can reduce the burden on individuals, enrich treatment options, and satisfy clinical needs better.

Spinal muscular atrophy (SMA) is a rare, life-threatening, and seriously debilitating neuromuscular disorder, which has a heavy burden on patients, caregivers and the health system. Technological advances have improved clinical effect, but have also increased the financial burden. There is limited information in the literature on the resource utilization and economic burden of SMA. Our research aims to summarize the current literature on resource use, cost and economic evaluations of treatments for SMA, to inform further research and policy decision making.

Databases, including PubMed, Embase, Cochrane Library and CRD Database, were searched from inception. Two reviewers undertook title and abstract screening followed by full-text screening, and any disagreement was resolved in consensus. Data extraction was conducted using a customized form. Included studies were summarized using narrative synthesis structured around general and economic characteristics. Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines were adhered to where applicable.

We reviewed 552 abstracts and included twenty-six from 2015 to 2019. Four-fifths were published in the United States and Europe. Five full economic evaluations and one budget impact analysis compared nusinersen with AVXS-101 or best supportive care, and the remaining evaluated the economic burden of SMA. The most common outcomes were healthcare resource utilization and direct medical costs, only a few studies evaluated direct non-medical costs or indirect cost.

SMA patients have significant medical expenditures and high utilization of healthcare services, including nusinersen-treated patients. The results highlight the substantial burden of treatment for SMA, not only for patients but also for their caregivers. SMA represents a significant hidden cost that society should be made aware of, and that should be considered in the design, implementation and evaluation of support programs for people who suffer from this disease and their families, as well as in the economic evaluation of new treatments.

Idiopathic pulmonary fibrosis (IPF) is a fatal lung disease. Due to insufficient awareness of the disease, and the lack of specificity of clinical and physiological signs, the diagnosis of IPF is often delayed. In the Lazio Region (Italy) four reference centers manage patients with IPF. The objective of this analysis is to support the definition of a new regional care and therapeutic pathway (Percorso Diagnostico Terapeutico Assistenziale [PDTA]) for IPF to anticipate the moment of diagnosis by reference centers. The delayed referral to specialized centers has clinical consequences both in terms of survival and access to treatments.

A survey collected aggregated evidence on factors associated with referral patterns to specialized centers for IPF. Its content was defined on the basis of a literature search, the experience of involved clinicians, and hospital data sources. The survey considered patients diagnosed with IPF by the network of reference centers from 2014 to 2018. Aggregated data on the pre-diagnosis pathway and evidence on organizational features of each reference center were collected.

Patients with a confirmed diagnosis of IPF increased from 2014 (n = 81) to 2018 (n = 344). A similar trend emerged considering only older patients. Incidence rates reached 11.33 cases per 100,000 residents in 2018. The majority of patients had a diagnosis in time to access to available treatments (87% in 2018). The number of specialists in multidisciplinary teams didn't change in a significant way. GPs, pneumologists, and IPF centers emerged as the pillar of the de-facto PDTA.

A new regional care and therapeutic pathway has been proposed to improve treatment of IPF. The first goal is to improve interaction among GPs, pneumologists, and IPF centers along the natural course of the disease. Criteria for referral to IPF centers has been defined (i.e. high-resolution computed tomography) as well as for adoption of a hub-and-spoke approach based on telemedicine.

According to the World Health Organization, there were approximately 0.5 million new cases of rifampicin-resistant tuberculosis in 2018, of which 78 percent were multidrug-resistant tuberculosis (MDR-TB), and China has one of the largest shares of the global burden (14%). In recent years, the Chinese government has made progress in TB control and prevention, but for MDR-TB, treatment options are still limited and expensive, and novel drugs are not always available. This research aims to evaluate the cost-effectiveness of adding bedaquiline to a background regimen (BR) of drugs for MDR-TB treatment in China, and to provide evidence for government to improve public health policies.

A cohort-based Markov model was developed to evaluate the incremental cost-effectiveness ratio (ICER) of bedaquiline plus BR (BBR) versus BR alone in MDR-TB treatment, over a 10-year time horizon. Data were sourced from a phase II clinical trial, real-world data in China, published literature, and expert opinion. Outcomes were evaluated in quality-adjusted life years (QALYs) and life-years gained (LYG). The discount rate was 3.5%. Probabilistic and deterministic sensitivity analyses were conducted.

The discounted costs per person for BBR was CNY 135,706 [USD 19,172], compared with CNY 92,465 [USD 13,063] for BR. The discounted utility per person for BBR was also higher than that for BR (3.943 QALYs versus 3.193 QALYs). The ICER of BBR was CNY 58,096 [USD 8,208]/QALY, which was lower than the willingness-to-pay threshold of CNY 212,676 [USD 30,046] (three-times the gross domestic product per capita). Therefore, BBR was considered to be cost-effective. The sensitivity analysis confirmed the robustness of the results. BBR remained cost-effective in the sensitivity analysis, with a 77.2 percent probability of being cost-effective versus BR.

In China, bedaquiline is not included in the National Reimbursement Medicine List, which results in a heavy financial burden for MDR-TB patients. From this study, BBR was cost-effective by significantly reducing time to sputum culture conversion and increasing QALYs and LYGs, which offset the higher drug costs.

To strengthen the care capacity of primary facilities, China has vigorously promoted the construction of a hierarchical medical system and a family doctor care system. In July 2017, a family doctor care plan was launched in an urban district of Chengdu, Sichuan Province, and two family doctor contracting service models were adopted, one provided a basic-service package and the other a paid-service package. In order to evaluate the effect of different models on diabetic patients, this study conducted a real world study based on the district healthcare database.

Diabetic patients who contracted family doctor services January 2018 to January 2019 as reported in the database were enrolled in the paid- or basic-service group. Propensity score matching (PSM) was conducted to balance the distribution of covariances between the groups. The results of the first and last examination of glycosylated hemoglobin, low-density lipoprotein cholesterol (LDL-C), systolic and diastolic blood pressure in the groups were compared by independent sample t-test and chi-square test.

Included were 4,871 patients in basic-service and 394 patients in paid-service. In both groups the total control rates of blood pressure, glycosylated hemoglobin and LDL-C at the last physical examination were 43.67, 79.28 and 51.11 percent, respectively, a significant increase from pre-test. The combined control rates of HbAlc, LDL-C and blood pressure in the basic- and paid-service group were 20.76% and 22.37%, respectively. After PSM, there was no significant difference between the groups.

Up to now, there is no significant difference between basic-service and paid-service family doctor contracting service models in improving the comprehensive control rate of diabetic patients. The possible reasons may be that the quality and content of paid-service is not as good as expected, the period of implementation is not long enough and the sample size of paid-service patients is limited.

The Brazilian health system has not been able to enforce the constitutional Right to Health for the entire population, leaving litigation as the last alternative for the fulfillment of the right. In order to harmonize and underpin the decision making of federal judges, an Academic Detailing (AD) program with concepts of evidence-based medicine and health policies for federal judges will be conducted nationwide in Brazil. AD is a strategy to provide information, combining an interactive outreach approach with the best evidence. This study reports the method used to define key messages to be used during visits.

Government, federal judges and academy representatives were invited to a workshop on health litigation in Brazil. They were divided into six groups to discuss five hypothetical scenarios. In each scenario, groups listed two possible key messages to disseminate during AD, addressing the legal, scientific, economic or ethical dimensions. After the definition, a vote was taken, according to the importance that each participant attributed to them (1 to 10), and then a score was generated.

Of the thirty-one participants, five were from the judiciary, three prosecutors, one health insurance representative, nine managers and thirteen from the academy. From the case study presented, fifty-five key messages were suggested. After removing duplicates, twenty-five were selected to assign importance values by each participant. The key messages were sorted from highest to lowest score, the most important being “Explain what evidence-based medicine means and what scientific evidence means”.

Knowledge of theoretical concepts of evidence-based medicine, health policies and the technology incorporation process seem to be of fundamental importance to federal judges. This information will serve as a basis for decision making regarding litigious proceedings involving the constitutional Right to Health in Brazil. From the selected key messages, bulletins to the academic detailing program to federal judges shall be produced.

Considering the World Health Organization (WHO) Astana Declaration, in order to provide universal healthcare coverage, Kazakhstan through ongoing healthcare reform committed to the promotion of a people-centered Primary Healthcare (PHC) system. Since the implemented top-down policies showed low buy-in from community members and put more constraints on PHC facilities and teams, the Kazakh National Medical University, the Medeo district mayor's office and the WHO European Centre for PHC supported the initiative of a local non-governmental organization “Community health committee” and Outpatient clinic of Almaty State hospital #5, for creation of an integrated plan to develop people-centered PHC through better coverage and engagement of patients with non-communicable disease and enhancing the health literacy of the population above 65 years.

We used a community-based participatory approach. The process consisted of: forming a steering committee with at least one member from each stakeholder group; two interactive workshops where the community worked jointly with PHC professionals in defining priority health needs and proposing actions to address selected priorities; and, after, joint development by all stakeholders of an action plan for empowerment of the community, and for assessment and review of the scope of practice of PHC teams.

The interactive workshops identified priority health needs such as low health literacy, low responsibility for health, low engagement of the elderly in prevention and self-management of non-communicable diseases. The main findings of semi-structured interviews were that there are no planning approaches (neither shared planning by a PHC team, or individually by PHC professionals) for addressing defined priority needs of the community, families and/or patients, and that the role of a PHC team in community empowerment is very limited.

Using results of the review on PHC teams’ scope of practice, we will develop, discuss and agree with the national and local stakeholders’ proposal of a conceptual model of PHC service delivery. Further, we will implement and evaluate the results of implementation.

In hospitals with standardized processes of health technology assessment (HTA), clinical studies are usually required to support incorporation requests. Factors such as conflicts of interest and confirmation bias affect the quality of the evidence. The objective of this study was to assess the quality of studies submitted to the university hospital to support the incorporation of non-drugs technologies.

All submissions received from 2017 to 2019 were reviewed. Data about methodological quality of studies sent by requesters and their utilization of final recommendation were collected, as well as about studies retrieved during additional search performed by HTA. The Jadad and Assessing the Methodological Quality of Systematic Reviews (AMSTAR) scales were used for assessing the quality of randomized clinical trials (RCTs) and systematic reviews (SRs), respectively.

Twenty-one requests for incorporation of equipment were analyzed. The average number of studies attached was 4.5. In eight requests (53.3%), both SRs and RCTs were attached. In seven (46.7%) only low quality studies were included were included; additional search identified RCTs and SRs in four of these cases. According to the Jadad and AMSTAR scales, 60 percent of the submitted RCTs showed a high risk of bias, while 57 percent of the SRs showed moderate quality, respectively.

The best evidence is not always submitted during the incorporation request process. Requirements for studies of moderate-to-high quality to accept the incorporation demand could be helpful to avoid inefficiencies in hospital-based HTA.

Tricuspid regurgitation (TR) is defined as incompetence of the tricuspid valve (TV), which produces the movement of blood flow from the right ventricle (RV) to the right atrium during systole. Pathological TR is functional in nearly 80–90 percent of cases, secondary to volume and/or pressure overload in the RV. Surgical intervention of TR is associated with mortality rates of 10 percent. Transcatheter therapy interventions (TTI) can be an alternative for severe TR. The aim of this study is to assess effectiveness and safety of TTI.

A systematic review was carried out. The scientific literature search was performed in major medical databases. Studies analyzing the efficacy and safety of the devices were included. Outcomes related with mortality rates, TR volume reduction, echocardiographic findings and adverse events were analyzed. The methodological quality of the studies was analyzed with the Canadian Institute of Health Economics Quality Appraisal Checklist.

Nine studies comprising 557 patients were included (two first-in-human studies, one retrospective, five single arm prospective studies and one international registry). The studies were small with short follow up. The outcome of procedural success ranged from 80 to 100 percent. Mortality rates at 30 days were lower than 5 percent. Improvements in reduction of TR, European System for Cardiac Operative Risk Evaluation (EuroSCORE), heart failure symptoms or quality of life scores were observed in all studies.

TTI for moderate-severe TR show significant reduction of annulus dimension, improvements in heart failure symptoms and quality of life, which are maintained in mid-term follow up. TTI present lower rates of major serious adverse events. No differences were observed between different TTI devices in terms of procedural success, mortality or safety. Randomized studies comparing TTI with optimal medical therapy are needed to confirm the preliminary clinical impact in patients with severe TR, and define aspects such as patient selection, risk factors associated with procedural success or mortality rates.

A robotically assisted surgical system, the da Vinci surgical system (DVSS), is a sophisticated surgical platform equipped with immersive 3D visualization and dexterous articulating endoscopic instruments. Surgeons can intuitively control the surgical system to perform delicate surgical tasks. Robotic surgery has gained popularity globally ever since its birth and was approved to market by the China Food and Drug Administration in 2006. This study aims to map the current use of DVSSs in mainland China and the trends from 2009 to 2019.

A full-sample survey of all hospitals equipped with DVSSs was conducted in mainland China, collecting data on hospitals and surgical departments using DVSSs, operation volume, and time of installation. Disease classification was standardized to obtain DVSS use in each department. EXCEL software was used for logging and cleaning the data. The analysis focused on descriptive analysis to map trends of DVSS use in China and present geographical and department distribution.

The DVSSs installed have grown from seven in 2009 to 135 in 2019. By the end of 2019, twenty-eight provinces in China have been equipped with the DVSSs, among which eighty-seven in the eastern regions, twenty-seven in the central regions, and twenty-one in the western regions. The annual volume of operations grew from 339 in 2009 to 38,991 in 2019, at an annual rate of 60.7 percent. The average workload conducted by a single robot is much higher than that of their counterparts in other countries. The largest share of the volume is in department of urology (48%), followed by general surgery (25%) and thoracic surgery (13%).

The use of DVSSs in China has been growing rapidly and extensively, with certain differences between geographical regions and surgical departments. We need to further explore the factors affecting its use and operation efficiency and to evaluate the effectiveness as well as cost-effectiveness in real-world clinical practice to inform public policies on application of DVSS, for example, license and insurance.

The treatment of relapsed/refractory multiple myeloma (RRMM), a common hematological malignancy, remains a great challenge in China, partially due to the limited accessibility to novel agents and inadequate public health insurance coverage. Ixazomib, a novel oral proteasome inhibitor (PI), was approved by the China Food and Drug Administration (CFDA) for RRMM in 2018. While bortezomib, a traditional PI, is the recommended agent in the clinical guideline for MM. Here, we compared their costs and effectiveness.

RRMM patients who has received an ixazomib-based regimen (at least 2 cycles) were analyzed. Using a propensity score matching method, we generated a control group of RRMM patients who received the bortezomib-based regimen. The criteria included the number of treatment lines, age, and the revised international staging system stage (R-ISS) which representing the disease stage for myeloma, and paired at a ratio of 1:2 (allowing one control to match multiples). The difference in hospitalization stay, grade 3/4 adverse events rates, overall response rate (ORR), mortality during treatment, and treatment costs was then compared.

Nineteen patients received ixazomib and twenty-seven that received bortezomib were included. The ixazomib-group demonstrated a shorter hospital stay (9 days versus 27 days, p < 0.001), lower grade 3–4 adverse events rates (42.1% versus 55.6%, p < 0.001), higher ORR (63.2% versus 48.1%, p = 0.228), and lower mortality rate during treatment (0% versus 7.4%, p = 0.169) than that of bortezomib-group. The ixazomib group had lower total costs (127,620CNY versus 156,424CNY [18,033USD versus 22,103USD], p > 0.05), lower drug costs (98,376CNY versus 103,307CNY [13,901USD versus 14,598USD], p > 0.05), and the lower costs of supportive treatment (5,507CNY versus 14,701 CNY [778USD versus 2,077USD], p < 0.001). Only in terms of self-funded costs, the bortezomib-based regimen was significantly lower (37,127CNY versus 11,521CNY [5,246USD versus 1,628USD], p < 0.001).

Compared with the bortezomib-based regimen, the ixazomib-based regimen has better therapeutic effects on MM patients while saving costs. Hence, it may be preferable for use in the treatment of RRMM in China.

Anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV) is a rare, serious and often life-threatening disease. The use of available treatments options (immunosuppressants and glucocorticoids (GCs)) improves the prognosis of AAV greatly; however, GC use is associated with significant toxicity related morbidities and the management of AAV is costly. However, information of the costs associated with AAV in the United Kingdom is limited. This study aimed to quantify the burden of AAV using a large England and Wales source of real-world data, the Clinical Practice Research Datalink (CPRD) Hospital Episode Statistics (HES) linked database, to identify healthcare resource utilization and generate estimates of costs.

Incident patients (n = 220) were included if ≥ eighteen years, with diagnosis read codes G754.00/G75A.00; ICD codes M31.3/M31.7 from January 1997 to December 2017. Costs were taken from Unit Costs of Social and Health Care, National Health Service reference costs and electronic drug tariff. Distinction was made between type of consultations, outpatient visits and inpatient admission based on Healthcare Resource Grouping. Costs were summarised as mean per member per year (PMPY) in 2016 prices and presented before and after diagnosis.

In the year preceding AAV diagnosis, mean costs PMPY were GBP12,012 [USD15,400], (GBP5,339 [USD6,845] inpatient, GBP766 [USD982] outpatient, GBP314 [USD403] GP, GBP5,594 [USD7,172] GP prescribing). In the year of AAV diagnosis (Y0) costs PMPY were GBP28,252 [USD36,220], GBP15,436 [USD19,790] inpatient, GBP1,863 [USD2,388] outpatient, GBP2,407 [USD3,086] GBP8,545 [USD10,956] GP prescribing). Costs in the years post-diagnosis remained higher than pre-diagnosis with a low of GBP22,839 [USD29,281] in Y4. The prescribing costs (GC, methotrexate and azathioprine) were the largest contributor in Y0-Y4 (GBP15,047 [USD19,291] Y1; GBP12,325 [USD15,801] Y4).

Diagnosis of AAV is associated with increased healthcare costs, including higher inpatients costs in the year of diagnosis and subsequently higher prescribing costs in the community. Given the incidence (17.2 cases per million) and considering only costs in the year of diagnosis, an additional GBP15.6 million [USD24.6 million] of healthcare resource utilization occurs every year from new diagnoses of AAV. However, this will likely be underestimated due to the lack of secondary care prescribing data in CPRD-HES and prescribing of immunosuppressant treatments in this setting.

Different value frameworks (VFs) have been proposed in order to translate available evidence on risk-benefit profiles of new treatments into Pricing & Reimbursement (P&R) decisions. However limited evidence is available on the impact of their implementation. It's relevant to distinguish among VFs proposed by scientific societies and providers, which usually are applicable to all treatments, and VFs elaborated by regulatory agencies and health technology assessment (HTA), which focused on specific therapeutic areas. Such heterogeneity in VFs has significant implications in terms of value dimension considered and criteria adopted to define or support a price decision.

A literature research was conducted to identify already proposed or adopted VF for onco-hematology treatments. Both scientific and grey literature were investigated. Then, an ad hoc data collection was conducted for multiple myeloma; breast, prostate and urothelial cancer; and Non Small Cell Lung Cancer (NSCLC) therapies. Pharmaceutical products authorized by European Medicines Agency from January 2014 till December 2019 were identified. Primary sources of data were European Public Assessment Reports and P&R decision taken by the Italian Medicines Agency (AIFA) till September 2019.

The analysis allowed to define a taxonomy to distinguish categories of VF relevant to onco-hematological treatments. We identified the “real-world” VF that emerged given past P&R decisions taken at the Italian level. Data was collected both for clinical and economical outcomes/indicators, as well as decisions taken on innovativeness of therapies. Relevant differences emerge between the real world value framework and the one that should be applied given the normative framework of the Italian Health System.

The value framework that emerged from the analysis addressed issues of specific aspects of onco-hematological treatments which emerged during an ad hoc analysis conducted on treatment authorized in the last 5 years. The perspective adopted to elaborate the VF was the one of an HTA agency responsible for P&R decisions at a national level. Furthermore, comparing a real-world value framework with the one based on the general criteria defined by the national legislation, our analysis allowed identification of the most critical point of the current national P&R process in terms ofsustainability of current and future therapies as advance therapies and agnostic-tumor therapies.

Worldwide, more than 50 million people suffer from epilepsy, and there are 16–51 new cases per 100,000 population each year. Up to 30 percent of patients with epilepsy are pharmacoresistant, who are candidates for surgical treatment. Invasive electroencephalography (iEEG) is a mandatory method in the arsenal of epileptic centers, and is gradually becoming the gold standard for invasive determination of boundaries between the affected and functional zones of the cortex and subcortical brain. Treatment costs correlate with the severity of the disease, with patients having uncontrolled seizures incurring eight times the costs compared to those with controlled epilepsy.

To assess the clinical and cost-effectiveness of the iEEG in the pre-surgical diagnosis of pharmacoresistant epilepsy, a systematic search of literature by keywords in the MEDLINE database was conducted. The search resulted in sixty-six articles. The analysis included twenty studies that met the search criteria.

Most studies including meta-analysis show very low rates of complications of iEEG. Literature data demonstrate cost-effectiveness of the method in patients with pharmacoresistant epilepsy in comparison with continued antiepileptic drug therapy. As an integrated method, rather than a simple method, it takes maximum account of clinical, neurophysiological and anatomical-functional data to achieve accurate localization of the epileptogenic zone. Currently, iEEG is a clinically effective method to improve the safety and specificity of resective surgery.

With the use of iEEG, mortality and disability of patients with pharmacoresistant epilepsy will be significantly reduced. It has also been proven that epilepsy surgery leads to significant financial savings in the treatment of pharmacoresistant epilepsy. The results of the clinical and economic evaluation (mini-HTA report) have been submitted to the Ministry of Healthcare for decision-making on including iEEG in government reimbursement system.

The interaction of health technology assessment (HTA) and health regulatory agencies has been widespread, especially for decision-making in health system coverage. The objective of this paper is to report the HTA-regulatory interaction in Brazil.

This is a case study on the interaction between HTA and regulation in Brazil. Technical documents and Brazilian legislation on health regulation and HTA were analyzed. The study was conducted in July 2019.

HTA-Regulatory Interaction in Brazil is still incipient. There is no responsible agency for interaction between agencies, as there is in Europe and Canada, for example. In the last 4 years, cooperation has started between the Brazilian Health Surveillance Agency (Anvisa) and the Oswaldo Cruz Foundation (Fiocruz) for post-registration monitoring of medicines. During this partnership, 170 post-marketing drug opinions were prepared, assisting the regulatory agency in decision-making.

Brazil legislation guarantees essential medicines at low cost or free. The interaction between HTA and regulation has the potential to reduce the time taken to incorporate technology to the patient, in addition to ensuring greater safety for users of the Unified Health System. In this sense, it was observed that the interaction between health regulation and science and technology institutions has innovative potential in this approach.

Based on the needs assessment of the medical and non-medical departments, the Investment Committee of the Hospital de Clínicas de Porto Alegre (HCPA), a teaching hospital in Brazil, recommends on which technologies the limited financial resources should be invested. Technology inclusion requests are evaluated by the hospital's technology assessment unit. For technology replacement, we have found models to assess the criticality of medical equipment, but they were insufficient to support the decision, which involves all departments of our hospital. This study aimed to develop an automated tool to support decision making regarding investments in equipment replacement in the hospital.

A working group was set up with professionals from healthcare administration, clinical engineering and research departments. From the hospital's inventory database, we developed the tool using Google SheetsR. We have defined three departments for pilot testing of the tool: hemodynamics, laundry, and basic research. These departments represent the areas of healthcare, support services, and teaching and research in the hospital.

The criticality of medical equipment is assessed based on the criteria of function, physical risk, impact, remaining equipment life cycle, intensity of use and number of corrective maintenance actions performed. For the equipment in the administrative, support and research areas, the function and physical risk criteria were replaced by the safety and by the risks to the quality of service criteria. The evaluation is carried out by a multidisciplinary team. The tool categorizes the equipment into low, medium and high criticality.

The tool prioritized the equipment based on objective criteria evaluated by the departments’ multidisciplinary team comprising experts who use the equipment in their activities, the department administrator and clinical engineers, and provided transparency regarding the decision-making of the hospital's Investment Committee. In 2019, the limited financial resources were invested only in the replacement of highly critical equipment. We believe the tool can be reproduced in hospitals in low and middle-income countries.

The Hospital de Clínicas de Porto Alegre (HCPA), a public teaching hospital, has a Hospital-based Health Technology Assessment (HB-HTA) unit to support the decision-making process on technology incorporation, rationalization or disinvestment. In 2017, the plastic adhesive drape was standardized at HCPA for use in cardiovascular, digestive, orthopedic, and neurological surgery for the purpose of preventing surgical site infection (SSI). This study evaluated whether the plastic adhesive drape technology is more effective than the no adhesive drapes in the surgical procedures in which it is used in the HCPA, so as to support the medical board's decision regarding the rationalization of use.

The primary outcome was the surgical site infection rate (SSI). Searches were performed in PubMed, Cochrane and national and international health agencies: World Health Organization (WHO), National Institute for Health and Care Excellence (NICE), Centers for Disease Control and Prevention (CDC), Society for Healthcare Epidemiology of America (SHEA), Brazilian National Commission for the Incorporation of Technologies (CONITEC) and Brazilian National Health Surveillance Agency (ANVISA) databases. The search strategy combined terms related to the technology and types of surgery in which it is used in the HCPA. The quality of the included studies was assessed. Additionally, data on technology utilization and costs in the hospital were analyzed.

Technology assessment followed AdHopHTA project recommendations. Data from the hospital showed that the technology has been used in fifteen surgical specialties, different from the proposed incorporation, with a progressive increase in consumption from 2017 to 2018. The literature review included a systematic review with seven clinical trials, which concluded that the plastic adhesive drape lacks benefits, with potential for increased risk of SSI. The evidence was of moderate quality.

The expenses associated with the use of the technology were considered unjustified as it is not reimbursed by the Brazilian Ministry of Health and its disinvestment was recommended. The Medical Board approved the disinvestment of the technology based on the evidence found by the HB-HTA unit, and the medical staff complied with the decision.

The Agency for Health Quality and Assessment of Catalonia (AQuAS) is developing an evaluation tool for mobile health (mHealth) solutions to be used by health technology assessment (HTA) agencies and evaluation experts. In order to have a practical and comprehensive tool taking into account the particularities and challenges of mobile interventions, we considered the views and opinions of key stakeholders. The objective was to present the final selection of general aspects (dimensions) to be assessed in the evaluation, as well as the specific items (criteria) to be included in each of these topics, as a result of different co-design approaches with health professionals, developers, hospital managers, HTA agencies and patients.

A list of criteria used for health apps evaluation were drawn from a literature review. The initial list included eighty-nine criteria items grouped in nine domains. Those criteria and domains were discussed during four focus groups (FG). The importance of the criteria that were not considered as mandatory were later rated through a Delphi online sub-study, in a scale from one to six points, taking as consensus value when median value (median 6, Interquartile range, 0–1) was reached.

FG reduced domains and criteria from nine to seven and from eighty-nine to thirty-three, respectively. Most mandatory criteria were related with security, user experience, and clinical effectiveness. Fifty-seven individuals (53.7% of 106 invited to participate) were registered in the online platform (50.1% women, 68.4% 35–64years old and 42.1% from HTA agencies). From fifty non-mandatory criteria under consensus, ten criteria reached consensus (most from solution's content and health problem covered domains) concluding with a 43/7 criteria/domain tool.

Insights from main stakeholders on the content of the tool for mHealth assessment were considered through the FG and Delphi technique. The dimensions of security and privacy, clinical effectiveness, solutions’ content, technological aspects, users’ experience and costs were considered mandatory. The dimension related to the impact on the organization was appraised as a secondary domain for evaluation. A workshop with AQuAS research team and HTA external researchers will help to define: the assessment methods (type of instrument, dichotomous responses and/or Likert scales) for the evaluation and the format and dimension's weights of the final design of the tool.

In recent years, the GRADE system has been adopted internationally to make judgments about evidence and/or recommendations. Recently, the Spanish Network of HTA (RedETS) has promoted among its members the use of the GRADE system, especially the “Evidence to Decision” tool (EtD) where a multidisciplinary panel is involved. The objective is to describe the methodological approach with the first AQuAS experience using this methodology in HTA, focused on inclusion/exclusion of these technologies in the Spanish National Health Service (NHS) portfolio.

The standard EtD tool was used for clinical and financial decisions. Four multidisciplinary panels were constituted by relevant professionals in clinical practice and, where possible, patients. The four panels discussed on the following four interventions: in two cases, AQuAS prepared preliminary recommendations and participants provided feedback, while in the other two cases, experts received the evidence review and were asked to formulate recommendations. These recommendations were voted on and, in the case of disagreement, adapted and then voted on for a second time. Finally, any discrepancies were noted in the report. Evaluated interventions were: maxillofacial 3D-reconstruction, cataract surgery equipment, non-invasive surgery in obesity and pharmacological treatment in secondary fracture prevention.

Especially when more than one evaluative question was addressed in the HTA report the EtD and the consensus results required discussion. Consensus was fast but not immediate. Meeting length depended on the number of HTA questions and the amount of original disagreement in the recommendations. The nuances on how to write recommendations also affected the panel duration. All panels were successful in formulating the final recommendations.

Standardizing methodologies increased the homogeneity across HTA reports. The GRADE system is a feasible and useful tool because it favors transparency and rigor in drawing up recommendations on the inclusion/exclusion of technologies in the NHS portfolio. The EtD framework complements GRADE tables, which display the relevant evidence in a way that can be used by multidisciplinary groups to reach a consensual recommendation.

Although all participants received a short training video, more experience in the use of these methodologies might shorten the duration of the process and facilitate reaching consensus. Some considerations on how to overcome the difficulties and complexity of this methodological approach are discussed.