The evolution of advances in informatics, technology in medicine, and artificial intelligence (AI) offers opportunities to enhance health care during the coronavirus disease 2019 (COVID-19) pandemic. The challenge for biomedical engineers is to implement these developments in clinical practice to improve global health. Populations living in low-income countries do not have access to specialist care and quality diagnostic services for COVID-19. Therefore, an AI system based on a telemedicine platform for diagnosing COVID-19 could help mitigate the lack of highly trained radiologists at regional hospitals and serve as a triage system for rationalizing the use of reverse transcription polymerase chain reaction (RT-PCR) testing and other health resources in low-income countries. Thus, the utility of an AI system for diagnosing COVID-19 in Paraguay was investigated.

This is a descriptive multicenter observational feasibility study of an AI tool for the rapid detection of COVID-19 in chest computed tomography (CT) images of patients with respiratory difficulties who attended public hospitals across the country.

Between March 2020 and August 2021, 3,514 rapid diagnostic tests were carried out on patients with respiratory disorders to rule out COVID-19 in 14 hospitals nationwide. The average age of the patients was 48.6 years (52.8% were men); the most common age ranges were 27 to 59 years, followed by older than 60 years and 19 to 26 years. The most frequent findings on the CT images were severe pneumonia, bilateral pneumonia with pleural effusion, bilateral pulmonary emphysema, diffuse ground glass opacity, hemidiaphragmatic paresis, calcified granuloma in the lower right lobe, bilateral pleural effusion, sequelae of tuberculosis, bilateral emphysema, and fibrotic changes. Overall, there was 93 percent agreement and 7 percent discordance between the AI system and the RT-PCR test results. Compared with RT-PCR testing, the AI system had a sensitivity of 93 percent and a specificity of 80 percent.

Paraguay has an AI-based telemedicine screening system for the rapid detection of COVID-19 that uses chest CT images of patients with respiratory conditions.

The diagnosis and management of chronic diseases during the coronavirus disease 2019 (COVID-19) pandemic was one of the biggest challenges facing healthcare systems globally, especially in low-income countries. Since basic health care for chronic diseases can overwhelm the capacity of conventional face-to-face healthcare services, there is growing interest in using information and communication technology and telemedicine to improve access to medical services that are often not consistently available in rural communities. In this context, telemedicine tools should be directed toward maintaining basic health services for patients with chronic conditions in rural and underserved hospitals. This study evaluated a telemedicine system in remote public hospitals in Paraguay to demonstrate how telemedicine improved access to tertiary level diagnostic services for patients with chronic conditions.

This descriptive study evaluated the use of telemedicine for diagnosing patients in remote public hospitals to improve provision of basic health services to patients with chronic disease during the COVID-19 pandemic. The type and frequency of diagnostic studies performed were determined.

During the study 677,023 telediagnoses were performed in 67 hospitals. The 435,568 electrocardiograms performed in 61 hospitals indicated normal physiology (60.1%), unspecified arrhythmias (10.5%), and sinus bradycardia (8.4%). The 227,360 teletomography tests performed in 12 hospitals were undertaken on the head (52.4%) because of trauma (motorcycle accidents) and cerebrovascular diseases, chest (15.8 %), and other anatomical regions. The 14,076 electroencephalograms performed in 19 hospitals were undertaken for antecedents of seizure (53.3%), disease progression controls (14.0%), and headache (12.5%). Nineteen prenatal ultrasound scans were conducted.

Although the results are promising for using telemedicine to bridge gaps and improve equity in the provision of basic health services for patients with chronic diseases in remote locations during the COVID-19 pandemic, a widespread use assessment should be undertaken before this tool is adopted.

Cataract surgery is the most commonly performed surgical procedure in the UK (approx. 472,000 annually). The suspension of interventions due to the COVID-19 pandemic, has had a devastating impact on patients’ access to care. In the UK a complete cessation of elective cataract surgery during the crisis has been an unfortunate reality and encompassed a 14 week hiatus to services in the National Health Service. Patients on prolonged waiting lists may experience negative outcomes during the wait period, including vision loss, increased risk of falls, and ultimately, poorer health-related quality of life (HRQoL). The objective of this research was to estimate the potential societal costs associated with vision-loss related to prolonged waiting times for cataract surgery, as a consequence of COVID-19 in the UK.

In this analysis, we present estimates relating to two cohorts: a hypothetical cohort of 1,000 cataract surgeries and quarterly estimates of cataract surgeries in the UK. Quarterly estimates (n=122,969) were chosen to reflect a suspension of cataract surgeries for 14 weeks during the COVID-19 crisis. UK cataract surgery numbers were attained from EUROSTAT. Estimates for decreasing visual acuity for those waiting for surgery were attained from the literature, as were the cost estimates associated with cataract-related sight-loss, which were made up of direct, indirect and intangible costs. Five scenarios (at 20% intervals) were simulated for the cost estimates, assuming from 20 percent to 100 percent clearing of waiting lists.

For cohort 1 (1,000 patients), the societal costs associated patients remaining on waiting list for one year, ranged between GBP 237,765 (EUR 279,533) (20% of patients remain untreated) to GBP 1.18m (EUR 1.39m) (100% remain untreated). For cohort 2 (n=122,969) cost estimates are in the region of GBP 29.23m to GBP 146.18m (EUR 34.36m to EUR 171.73m). Estimates consist of direct (15.6%), indirect (28.7%) and intangible costs (55.6%).

Cataract surgery is a sight saving procedure and its impact on HRQoL is overwhelmingly positive. Prolonged waiting times for cataract patients due to COVID-19 is likely to be associated with significant societal costs.

Realistic Review (RR) is a methodology for systematic review proposed by Pawson (2002), to guide policy formulation. It is aimed at policy makers and decision makers and seeks to explain how a given intervention succeeds or fails in a certain context, allowing the identification of more suitable alternatives to solve problems. The objective of this study was to develop a theoretical model for Monitoring and Evaluation (M&E) of Health Technology Assessment (HTA) management.

The realistic review sought to identify aspects that influence HTA contributions. Studies were included if they provided a description of context and description of HTA contributions. Key purpose elements were extracted from selected studies and the contexts of HTA contributions were analyzed. The association between context and types and areas of contributions of HTA in different realities was synthesized. We analyzed thirty-one articles published in international journals in the area of health technology assessment and three articles on assessment policy in Brazil, nominated by experts.

It was possible to identify situations that generate demands for HTA; favorable and unfavorable contexts and factors for contributions; main functions of networks; main products resulting from the application of evaluations; results and impact predicted by the action of the networks.

The logical model starts with conjunctural aspects that generate demands: economic, social and cultural. In a favorable context, the definition of priorities and debates in the negotiation arenas influence research and the policy formulation process. Networks identify evidence and contribute to knowledge management, generating products, results and impacts.

The use of the theoretical model created from the realistic review is a tool that allows the M&E of HTA management.

Digital transformation has been promoted by the World Health Organization (WHO), Food and Drug Administration (FDA) and the European Commission (EC) to help improve health outcomes. To ensure sustainability, digital health interventions (DHI) require funding by payers. Evidence-informed decision and policy making requires an assessment of the impact on relevant outcomes vs current healthcare practice. Various national and international organizations are involved in creating or guiding the development of standards for the evidence required for digital technologies.

We undertook an intensive individual investigation of the websites of leading payer and health technology assessment (HTA) bodies in France, UK, Germany, Belgium, Austria, Finland, Canada, Australia, and the USA to identify new frameworks and any updated information. As the objective focused on evaluation frameworks which were used across DHIs by a particular payer to support pricing and reimbursement decisions, we excluded individual case studies where DHIs had been assessed, regulatory frameworks for approval of DHIs and frameworks which assessed feasibility or applicability of a DHI since these were not directly influencing the decision for funding.

We found six frameworks which directly address digital health interventions for the purposes of pricing and reimbursement: NICE Evidence Standards, FinCCHTA, MSAC, Germany BfArM, Belgium RIZIV and France HAS. The context for the framework and the requirements were compared on parameters including those normally found in HTA and for criteria related to digital technologies. The parameters included varied considerably across the frameworks as did the level of evidence expected to be available for the assessment. In some cases, these related to the level of risk or impact of the intended DHI.

While DHIs are increasingly used in health, HTA is struggling to adapt to assess these technologies. Due to the multidisciplinary nature of digital health (combination of health care and technology), and the speed and rate of change of innovations in this area, an approach based upon the risk assessment posed by the technology seems reasonable. In this way the level of effort can be tailored to those interventions which seek to influence care or predict outcomes rather than those which are tailored to increased awareness of the patient about their condition.

Multi-criteria decision analysis (MCDA) is a useful tool in complex decision-making situations and has been used in medical fields to evaluate treatment options and drug selection. We aimed to provide valuable insights on the use of MCDA in health care through examining the research focus of existing studies, major fields, major applications, most productive authors and countries, and most common journals in the domain using a scientometric and bibliometric analysis.

Publications related to MCDA in health care were identified by searching the Web of Science Core Collection on 14 July 2021. Three bibliometric software programs (VOSviewer, Bibliometrix, and CiteSpace) were used to conduct the analysis.

A total of 410 publications were identified from 196 academic journals (average yearly growth rate of 32% from 1999 to 2021), with 23,637 co-cited references by 871 institutions from 70 countries or regions. The USA was the most productive country (n=80), while the Universiti Pendidikan Sultan Idris (n=16), Université de Montréal (n= 13), and Syreon Research Institute (n=12) were the most productive institutions. The biggest nodes in every cluster of author networks were Aos Alaa Zaidan, Mireille Goetghebeur, and Zoltan Kalo. The top journals in terms of number of articles (n=17) and citations (n=1,673) were Value in Health and the Journal of Medical Systems, respectively. The research hotspots mainly included the analytic hierarchy process (AHP), decision-making, health technology assessment, and healthcare waste management. In the recent literature there was more emphasis on coronavirus disease 2019 (COVID-19) and fuzzy Technique for Order Preference by Similarities to Ideal Solution (TOPSIS). Big data, telemedicine, TOPSIS, and the fuzzy AHP, which are well-developed and important themes, may be the trends in future research.

This study provides a holistic picture of the MCDA-related literature published in health care. MCDA has a broad application in different topic areas and would be helpful for practitioners, researchers, and decision makers working in health care when faced with complex decisions. It can be argued that the door is still open for improving the role of MCDA in health care, both in its technologies and its application.

The orphan designation has been used by the European Medicines Agency to incentivize the development of drugs treating rare diseases with high-unmet medical needs by supporting their development process and economic returns. This study evaluated the impact of the regulatory orphan designation and other drug development-related factors on the rollout times and Health-Technology-Assessment (HTA) recommendations of new active substances (NASs).

A total of 656 HTA appraisals from 6 European countries were collected for NASs that received regulatory approval between 2012 and 2020. Multivariable logistic (positive and positive with restrictions vs. negative HTA recommendation as dependent variable) and linear regression (rollout time as dependent variable) models examined associations with regulatory orphan designation, expedited process, product type (biotechnological vs chemical), and jurisdiction (France, England, Germany, Poland, Scotland and Sweden). Rollout time was defined as months elapsed from regulatory submission to HTA recommendation (mean± standard deviation).

Multivariable logistic regression analysis identified disparities in HTA recommendations between countries. Every month increase in rollout time conferred a 3 percent reduction in the odds of a positive recommendation (p<0.001). Review and product type did not show associations with HTA recommendation. Interestingly, orphan products showed a 99% increase in the odds of obtaining a positive HTA recommendation compared to non-orphan (p-value=0.003). We found 244 appraisals (37%) assessing an orphan product, of which 202 (83%) received a positive HTA recommendation.

Multivariable linear regression analysis indicated that orphan products presented a 4.4-month rise in rollout time when compared to non-orphan products (p<0.001). The mean rollout time in months for orphan products were 25±12 in France, 30±15 in England, 21±9.1 in Germany, 37±16 in Poland, 25±12 in Scotland and 27±14 in Sweden.

Orphan designated products showed greater odds of receiving a positive HTA recommendation compared with non-orphan. A more detailed review of orphan products could result in their longer rollout time compared with non-orphan counterparts. Considerable differences were found between HTA recommendations and rollout times between jurisdictions.

The Scottish Medicines Consortium (SMC) defines end-of-life medicines as drugs used for treating conditions that usually lead to death within three years using currently available treatments. Orphan medicines are drugs used for the treatment of very rare conditions and ultra-orphan drugs are used for the treatment of extremely rare conditions. The objective of this study is to determine the influence of unmet need on SMC health technology assessment (HTA) decisions for rare disease conditions.

The reimbursement data between 2004 and 2021 from SMC for rare disease conditions were included. These data were categorized based on the presence of an unmet need, that is, drugs considered under the orphan or ultra-orphan process, or those that fulfilled SMC end-of-life criteria. A chi square test was conducted to determine an association between the presence of an unmet need and the HTA decision. HTAs without a decision were excluded.

A total of 91 HTAs were included in the analysis of which, 57.1 percent (n = 52) were recommended, and 42.9 percent (n = 39) were not recommended. Out of the recommended reviews, 32.7 percent (n = 17) addressed an unmet need and 67.3 percent (n = 35) did not. Recommended drugs had positive clinical evidence and high cost-effectiveness or the submission of a patient access scheme while negative decisions were associated with lower or uncertain cost-effectiveness. The chi square test result showed no association between the presence of an unmet need and the HTA decision (p = 0.315).

Unmet need does not influence the SMC HTA decisions for rare disease conditions. Economic elements were the driving factors in the decision-making process.

The number of anterior cruciate ligament reconstruction (ACLR) procedures is increasing. However, the likelihood of not allocating sufficient funds for the ACLR procedure in a developing country, especially in Indonesia, is high. This study aimed to analyze costs for ACLR procedures in Indonesia’s resource-limited context, determine the burden of ACLR, and propose national prices for ACLR reimbursement.

A retrospective observational study was conducted on the cost of ACLR from a payer perspective using inpatient billing records from 1 January 2019 to 31 December 2019 from four hospitals. The national burden of ACLR was calculated and national prices for reimbursement were developed.

Of the 80 ACLRs performed, 53 (66%) were isolated ACLRs and 27 (34%) were combined with meniscus treatment. The mean hospital costs incurred per ACLR procedure were USD 2,853 (IDR 40.4 million), which was mainly attributed to the orthopedic implants (USD 1,387; IDR 19.6 million). The costs of ACLR with combined meniscus treatment were estimated to be 35 percent higher than for isolated ACLR. The national burden of ACLR revealed a total budget of USD 367.4 million (IDR 5.2 trillion) per 100,000 patients for ACLR with additional meniscus treatment and USD 271.3 million (IDR 3.8 trillion) per 100,000 patients for the isolated ACLR procedure.

ACLR procedures in Indonesia are underbudgeted. Adjustment of reimbursement prices for ACLR procedures is needed to facilitate access for the Indonesian people. This study showed varying cost estimates for ACLR in Indonesia, necessitating a new reimbursement system that takes various clinical and patient factors into account.

Implementing value-based healthcare (VBHC) services requires the ability to assess and integrate evolution of patient-centered outcomes (PCO), clinical/epidemiological data and resource consumption. Aligned with Porter’s framework of value, the “Value of Healthcare Journey for Patients With Rare Diseases Project” (JAV-RARAS) was implemented to evaluate the value of the healthcare journey of patients with rare diseases (RD) in the Brazilian Public Healthcare System (SUS). The goal of this project is to identify the value (clinical outcomes and costs) associated with the management of RD in the SUS.

Patients diagnosed with eight pre-defined RD under active clinical follow-up in SUS medical centers were invited. Retrospective data on diagnosis and assistance carried out after diagnosis were collected from medical records. Prospective follow-up of one year will be collected through validated clinical questionnaires on three visits, with an average interval of 6 months. Clinical outcomes include exams and disease-specific features, quality of life, productivity loss, treatment adherence and satisfaction. Total cost of patient’s journey in each medical center will be assessed through Time-Driven Activity-Based Costing method. All data collection is being carried out through the Think Patient Value (TPValue®), a computerized management support system, based on Porter’s paradigm of value.

Thirty-five medical centers currently assisting patients with RD were enrolled, representing all macro-regions in Brazil: Midwest (n=4), North (n=5), Northeast (n=10), South (n=5) and Southeast (n=11). Recruitment started in March, and until November 2021, 28 centers had reported retrospective data of at least one RD. So far, JAV-RARAS recruited patients with osteogenesis imperfecta (number of patients=106), phenylketonuria (n=52), familial amyloidotic polyneuropathy (n=32), classical homocystinuria (n=25), Prader–Willi syndrome (n=30), acromegaly (n=33), hereditary angioedema associated with C1 esterase-inhibitor deficiency (n=42) and mucopolysaccharidosis type-II (n=17).

Future results of JAV-Raras real-world evidence study will bring PCO and costs according to Porter’s Value, bringing insights for decision-makers in SUS. This study was funded by National Council for Scientific and Technological Development –CNPq and Ministry of Health of Brazil –MoH.

The Patient Perspective is an initiative to include the testimonials of patients or their representatives at the time of the initial recommendation of health technology assessment (HTA) processes developed by the National Committee for Health Technology Incorporation in the Brazilian Public Health System (CONITEC). It allows these actors to be involved prior to the public consultation. This action is articulated with a framework of strategies to encourage patient and public involvement (PPI) in HTA in Brazilian Public Health System (SUS). This paper aims to analyze the first year of its implementation.

This is a descriptive study, based on document analysis (minutes of meetings, technical and society reports, videos of CONITEC meetings, tables for internal control, etc.) and systematic observation of the activities related to the initiative. Field notes were taken and their contents were coded, following content analysis criteria.

From December 2020 to November 2021, 1,052 subscribers were registered in 75 public calls, which resulted in the participation of 42 people. The testimonials presented information about the illness experience, its impact on quality of life, use of technologies, and difficulties of accessing and adherence to treatment. The patient perspective has been useful to give visibility to specific dimensions of experiences regarding not only the health conditions but SUS as well. Furthermore, some testimonials produced tensions and repercussions about HTA processes, such as the consideration of aspects or technologies not covered in the clinical studies and thus the HTA, as well as the need of reviewing some results of the technical report.

The patient perspective has had the support of the target audience. Additionally, it has been successful in including various types of participants and different aspects of the experience with the health condition and/or the technologies under evaluation. In general, it has been possible to observe the diversification of modes of PPI and institutional learning towards the improvement of HTA in SUS.

Public consultation is a strategy for patient and public involvement (PPI) assured by the National Committee for Health Technology Incorporation in Brazilian Public Health System (CONITEC)´s normative framework. It aims to obtain society’s views on the matters under evaluation. It is made available after CONITEC issues an initial recommendation, which will be reviewed in light of society contributions. This paper examines the cases where the final recommendations differed from the initial ones in technology assessments carried out between 2012 and 2020, taking into account the arguments used by the CONITEC Plenary to support the modification.

From a total of 361 public consultations referring to the assessment of health technologies carried out by CONITEC between 2012 and 2020, 40 final reports of health technology assessment (HTA) processes that had changes from the initial to the final recommendation were examined. Then, analytical categories were built so that justifications for the change were investigated and the interpretations produced. The empirical approach excluded public consultations regarding clinical protocols and therapeutic guidelines.

It was observed that, in 39 cases, the change occurred from an unfavorable position to a favorable position for the technology incorporation. The main reasons for changing the plenary’s decision were proposals to renegotiate the prices of technologies (that generate changes in the results of economic evaluation and budget impact analysis), the receipt of new evidence and experience, and opinion arguments sent mainly by health professionals.

The analyzed data has shown that the changes in recommendations are mostly based on the content of contributions which led to changes in the assessment of clinical evidence, economic evaluation and budget impact. Thus, it appears that public consultation has been consolidated as a relevant instrument to inform the deliberative process in HTA in Brazilian Public Health System.

Involving patients in the health technology assessment (HTA) lifecycle is a core principle at the National Institute for Health and Care Excellence (NICE) that helps build public confidence in healthcare decision-making. The last stage in this lifecycle is an appeal whereby stakeholders, including patient organizations, can appeal against the HTA committee’s decision based on two specific grounds: (i) the committee has failed to act fairly or NICE has exceeded its powers; and (ii) the recommendation is unreasonable considering the evidence submitted to NICE. Improving patient involvement in the appeals process was identified as a key area from consultative work undertaken with patient organizations in 2019.

In September 2020, surveys were sent to patient organizations for their feedback. The organizations received a survey tailored to their circumstances in relation to the following four outcomes.

1. (i) An appeal was lodged that: (a) resulted in an appeal hearing; or (b) did not result in an appeal hearing.

2. (ii) An appeal was not lodged but the organization had received: (a) negative guidance; or (b) ‘optimized’ guidance.

Sixteen responses were received across the four surveys. The key findings in priority order were as follows.

1. (i) The legalist nature of appeal hearings is off-putting.

2. (ii) The online appeals guide and template letter are not easy to use.

3. (iii) Organizations would like more information on appeal hearings and timelines.

4. (iv) Organizations would like patient-friendly materials and training on par with the support they receive for other HTA participation.

As a result of these findings, we are iteratively developing materials to support patient organizations, including:

• • a glossary;

• • a redesigned appeals web page with clearer timelines and a template letter;

• • a ‘Frequently Asked Questions’ document specifically for patient organizations; and

• • a video podcast from a lay appeals panel Chair explaining appeals in a patient-friendly way.

We are also including a section on appeals in our introductory training for all patient organizations participating in medicines HTAs.

According to the Healthy China 2030 Plan, children under 5 years is the main focus group to achieve universal health and sustainable development of China. To identify the major threats to children’s health, we analyzed and compared the burden of disease and risk factors among children under 5 years in China and other regions.

Indicators were gathered from the Global Burden of Disease 2019, which included the standardized rates and risk factors of mortality and DALYs of children under 5 years in China, Western Europe, North America and the world from 1990 to 2019. Paired t-test or Wilcoxon test were used to compare the rates based on gender. A joinpoint regression model was used to analyze the trend, and the Annual Percent of Change (APC) was calculated and statistically tested.

From 1990 to 2019, the all-cause mortality and DALYs of children under 5 years in China decreased from 1 153.81/100 000 to 160.39/100 000 and 104 426.40/100 000 to 16 479.01/100 000, respectively. The top 3 causes of both death and DALYs were neonatal preterm birth, congenital heart anomalies, lower respiratory infections. The top 3 risk factors of both death and DALYs were low birth weight, short gestation, child wasting. Unintentional injuries, behavioral and environmental risks posed greater threats to children compared with other regions. The rates of mortality and DALYs of the top 15 diseases and injuries in boys and girls showed a downward trend (p<0.05), and most of them were higher in boys than girls (p<0.05).

The burden of diseases among children under 5 years in China has decreased significantly from 1990 to 2019. Compared to other regions, it remains to strengthen the prevention and control of preterm birth, birth defects and unintentional injuries, and to adopt targeted gender-specific interventions. Promoting the parenting behavior and multiple social security may also affects children’s health status.

Children and youth in out-of-home care face many challenges regarding their development and general well-being. It is necessary to put in place best practices to support them where they are most vulnerable. Providing recommendations to health and social services for best practices regarding social and academic success interventions, this study puts forward a synthesis method that combines empirical data and the expertise of key practitioners in the context of Quebec social services.

A systematic review was first undertaken to identify the most effective interventions. Included studies (n = 31) were analyzed according to their methodological quality, collaboration processes, and type of care (foster home vs. residential care). To ensure the applicability of the recommendations made in the context of Quebec social services, contextual data, and clinical expertise were collected. Contextual data was gathered through local research reports, administrative data, and government documents. The expertise of multiple stakeholders was obtained in follow-up committees and semi-structured interviews (n = 4). In addition, to ensure their relevance and scope, recommended guidelines were debated in a deliberation committee.

The analysis between expert, contextual and empirical data led to several recommended evidence-based practices. In accordance with expert opinions, experimental and quasi-experimental studies show that various types of collaboration are beneficial for children and youth in out-of-home care. Notably, intersectoral collaborations were warranted in more complex situations (i.e., youth in residential centers), while partnership agreement was sufficient in less complex situations (i.e., children in foster homes). However, even if effective, some interventions are difficult to apply in real life and certain considerations must be taken into account (e.g. confidentiality issues, availability of resources).

Utilizing three sets of data, guidelines have been proposed to help health and social services to identify best practices and promote the academic development of out-of-home care children and youth.

Assessing the cost utility of health technologies for pediatric patients requires robust utility values for child health states, but the methods for valuing these pediatric health states are much less established than those for valuing adult health states. This is partly because the elicitation of preferences for child health states poses many normative, ethical, and practical challenges.

This presentation examines the conceptual issues in the valuation of health states in children by addressing the following questions.

• (i) Normative theories of health state values: What are we attempting to elicit?

• (ii) Sources of preferences: Whose preferences should we elicit, and from which perspective?

• (iii) Valuation methodologies: How should we elicit preferences?

• (iv) Attaching different values to child and adult health: Is a lack of consistency problematic?

To answer these questions, we used desk research (non-systematic literature reviews) and findings from a two-part workshop held in April 2021. The workshop included 25 participants with expertise in health economics, health state valuation, child health, health technology assessment (HTA) decision-making, and ethics.

We identified a lack of consensus on what is being elicited for both adults and children. Many HTA agencies recommend that the public be involved in utility generation exercises, but some criteria for defining who constitutes a member of the public exclude children. Of the many candidate sample types, perspectives, and methodologies, only a few were deemed relevant, acceptable, and feasible for use in the child health context. In addition, there were diverging views on whether it is possible to compare and integrate adult and child value sets with different properties.

Several questions remain to be answered before the public and other stakeholders can have confidence in child health state valuation protocols. We propose a research agenda, including both empirical and conceptual work, to inform future methodological development and to help HTA agencies make recommendations about how child utility values should be generated.

In 2019, the Québec provincial health technology assessment body (INESSS) recommended that lung cancer screening with low-dose computed tomography (LDCT) be accessible in Québec only within the context of an evaluation in the ‘real-world’ care setting. Based on this recommendation, the ministry of health (MSSS) decided, in 2020, to implement a screening pilot project and to conduct a formal evaluation, partnering with a clinical leader (principal investigator), participating hospitals, the provincial public health agency (INSPQ) and INESSS. The goal of this evaluation is to facilitate decision-making regarding the implementation of a province-wide screening program.

To support the implementation of the pilot project, algorithms and recommendations were developed to guide management of screening program participants. This material, based on Lung-RADS (Lung Computed Tomography Screening Reporting and Data System of the American College of Radiology), was developed by reviewing the literature and by consulting clinical experts. The evaluation plan proposes various indicators, focusing on six main topics: (i) costs, (ii) screening and investigation processes, (iii) clinical effectiveness and other effects on health, (iv) effects on smoking cessation, (v) organizational impact and (vi) implementation issues.

INESSS has developed 12 algorithms and close to 50 recommendations for lung cancer screening and investigation, a tool for assessing lung cancer risk and a benefits/risks table. For the evaluation of the pilot project, MSSS, INSPQ and INESSS developed more than 100 indicators; short-term indicators are currently being measured and others will follow in the longer term. Since starting in June 2021, the pilot project is progressing well (as of November 28, 2021): 2,365 people have been referred, 1,272 were eligible for screening, 678 have had their first LDCT and 19 were Lung-RADS 4B or 4X. Results on indicators will help the ministry decide on the feasibility of scaling up screening to the provincial level and will highlight aspects to be improved.

This project shows how health technology assessment products can elicit changes in the health system, and how multi-stakeholder collaboration can actively support practice implementation and inform decision-making.

The Brazilian National Agency for private healthcare system (ANS) regulates the private healthcare system in Brazil. ANS, since 2019, has been running the pilot value-based new payment models project. In total, 13 projects were selected by ANS. This investigation aims to identify opportunities and challenges to implement value based healthcare (VBHC) in Brazil.

We interviewed managers participating in the ANS’ Value-Based Payment Models. Data were collected through semi-structured interviews during 2021. Twelve managers were invited to participate in the interview and eight accepted the invitation. The key questions were: “what are the main factors that facilitate – or limit - the transition from the fee for service model to a value-based model in the private healthcare system? And “will the payment models be scalable?” For data analysis, Bardin’s content analysis was chosen. Data validation was performed using the debriefings technique.

The interviews identified two key facilitating factors: people (identified by 50% of respondents) and processes (identified by 50% of respondents). Reponses relating to people nominated the need for professionals with VBHC knowledge (33%), support of senior management (25%), support from the provider (25%) and care team (17%). Reponses relating to processes nominated the need for partnership (58%), health-driven management (25%) and results (17%). We also identified that limiting factors (49%) were: providers (39%), in details: non-support from the provider, (56%), fear of financial loss (22%) and provider only wanting profit (22%); information system (30%), with data management; culture (17%), current versus innovative models; and peoples (13%), knowledge. More than 90 percent found it to be scalable, particularly, in vertical health plans (38%), large operators (38%); and provide diagnostic services (13%). We found that non-scalable situations are those where fee for service is hegemonic in terms of payments.

Our study found that knowledge and culture management positively impacts the projects. Service providers can be limiting when they are exclusively focused on economic perspectives. The new payment model is considered scalable.

The Brazilian National Agency for private healthcare system (ANS) makes the regulation for private healthcare system in Brazil. ANS, since 2019, is running the pilot value-based new payment models project. In total, 13 projects were selected by ANS. This research aims to identify the key drivers for moving from fee for service (FFS) to value-based payment models in the Brazilian healthcare private system.

We interviewed managers of private healthcare plans (13 in total) participating in the Value-Based Payment Models run by ANS. Data were collected through semi-structured interviews during 2021. Twelve managers were invited to the interview and eight accepted the invitation. The key questions were: “Why are healthcare providers transitioning from the fee for service model to value-based models?” and “What are your motivations to participate in the ANS project?” For data analysis, Bardin’s content analysis was chosen. Data validation was performed using the debriefing technique.

The main reasons for transitioning from FFS to value-based models were related to weaknesses of FFS (58%), strengths of the value-based payment model (14%) and sector needs (14%). Fee-for-service weaknesses are related to financial impacts – including waste and unsustainability (55%), and lack of transparency – including lack of trust and conflict of interest (28%). Strengths of the value-based payment model were related to financial benefits (100%), in other words, greater return on investment. The key unmet needs of the sector are related to improvement of the financial status - including lower costs and less waste (71%), and improvement of care delivery quality (29%). Continuity was reported as a benefit of FFS, according to 43 percent of respondents.

Our results suggest that financial motivations are the main reason to transition from fee-for-service to value-based models.

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.