Immunization is one of the most effective public health interventions, saving millions of lives annually. However, complex and heterogenous market access pathways can impact timely and equitable population access to vaccines. Focusing on five Asia-Pacific (APAC) countries, this study described the current vaccine market access landscape and analyzed the success factors for National Immunization Program (NIP) inclusion.

The study was conducted in two phases (i) pragmatic literature review to map current regulatory and funding pathways and identify information gaps in China, Japan, Korea, Taiwan and Australia (ii) targeted interviews with market access and policy experts in each market to identify drivers and barriers to NIP inclusion.

Regulatory approval, driven by safety and efficacy data, followed by introduction in the private market was commonly observed in all markets. However, pathways to NIP inclusion varied within and between markets. In all markets expert panels such as National Immunization Technical Advisory Groups (NITAG) were identified to play a crucial role in providing advice to governments on NIP inclusion. Health technology assessment was increasingly used to inform decision making. However, the assessment frameworks utilized were typically designed for medicines, rather than the unique features of vaccines.

Japan, Korea and Australia provided relatively consistent coverage via national programs, despite the different processes employed to review, recommend and reimburse vaccines. In some cases, the reimbursement timeframe varied significantly for different vaccines within the same market. China and Taiwan provided coverage at the regional level, which required engagement with multiple local authorities to enable access. The key barriers to patient access were budget limitations, long reimbursement timeframes and a lack of coverage consistency. Early stakeholder engagement, local epidemiology and cost-effectiveness evidence were the main success factors for NIP inclusion.

Funding pathways for vaccines in APAC are heterogenous. Adopting the Asia-Pacific Economic Cooperation (APEC) Action Plan on Vaccination Across the Life Course could increase vaccine coverage through alignment, collaboration, and improvement of reimbursement pathways in APAC.

The increasing pace at which artificial intelligence medical devices (AI-MDs) or digital health technologies (DHTs) have been introduced and integrated in healthcare has not been matched with appropriate selection criteria for health technology assessment (HTA) to inform funding decision-making. To align with international best practice and local regulatory guidance, the Agency for Care Effectiveness (ACE) developed criteria to include AI-MDs as part of its 2022 topic prioritization process for medical technologies. This abstract describes ACE’s approach to develop the inclusion criteria.

To develop key principles for including AI-MDs in ACE’s topic prioritization process, relevant information from overseas HTA agencies, local regulatory guidelines, and ACE’s existing topic selection criteria were reviewed. A search of international HTA agency websites was conducted in September 2022 to identify relevant information on inclusion of AI-MDs in healthcare for reimbursement recommendations.

Additionally, local regulatory guidelines for AI-MDs in healthcare were also identified. The inclusion criteria were then piloted with AI-MDs identified from ACE’s horizon scanning workstream to examine their feasibility for HTA topic selection.

One overseas framework on DHTs from the National Institute for Health and Care Excellence (NICE) and two local regulatory guidelines were identified. Based on the key finding that the purpose of AI-MD use in guiding clinical management and its associated risks were important considerations, the following criteria were developed: (i) full registration with the regulatory body;(ii) device characteristics should be interventional, have direct impact on patient safety, or support accurate diagnosis or treatment which is critical to avoid death and serious health deterioration; and (iii) the AI algorithm should be fixed as opposed to adaptable as per regulatory requirements. Using this inclusion criteria, eight AI-MDs surfaced from horizon scanning were screened with the above criteria and deemed suitable for HTA topic selection.

As AI technologies are increasingly used to replace or supplement current clinical practice, continuous adaptation of HTA method is needed to ensure appropriate topic selection.

Computerized Clinical Decision Support Systems (cCDSS) are promising digital health tools whose development and use are increasing. The Agency for Health Quality and Assessment of Catalonia (AQuAS) received a request from the Spanish Ministry of Health, through the Spanish Network of Health Technology Assessment (HTA) Agencies (RedETS), to perform an HTA of cCDSS for cancer. We present the challenges that arose during the evaluation process.

We evaluated the safety, effectiveness and cost-effectiveness through a systematic literature review. We involved two clinicians and a technology expert to gain insight into the pathology and technology, respectively. To identify cCDSS used in Spain, we consulted with the Spanish regulatory agency (AEMPS) and the Spanish Federation of Healthcare Technology Companies (FENIN), plus did a survey among Spanish hospitals. To understand applicable regulations, we reviewed the European regulation (MDR) and consulted the Medical Device Coordination Group from the European Commission, AEMPS, and a regional regulatory expert.

The scientific literature revealed large heterogeneity in the definition of cCDSS (e.g., from simple online prognostic calculators to complex commercial software using machine learning), making the literature search and screening arduous. Many articles dealt with cCDSS that do not qualify as medical devices, are not in the market anymore, are currently used as newer versions or are developed in-house. Next, we faced the difficulty to attain a comprehensive overview of the tools in use in the country. In terms of legislation, we observed that similar tools might receive different classifications in different jurisdictions, and the complexity of the MDR might lead to the need for a case-by-case discussion at a National level.

We identified many challenges in the HTA of cCDSS. The first step for proper assessment is a clear definition of the device and version to be evaluated. Multiple stakeholders must be involved, and alignment between regulatory and HTA agencies is key. We expect that the European Database on Medical Devices (EUDAMED) will help in the identification of existing cCDSS and hence ease their assessment.

Experts highlight the need to reconsider the frameworks used in health technology assessment (HTA) in order to cover the particularities of digital health technologies (DHT). However, there is a large heterogeneity in the literature about terminologies and definitions. Our objective was to identify the methodological frameworks used worldwide for HTA of DHT and generate a proposal of domains to consider in the assessment through a thematic analysis, a qualitative data analysis method that consists of identifying, analysing and reporting patterns across the data.

Methodological frameworks were identified through a scoping review (ScR) in accordance with the PRISMA extension for ScR. We searched five different databases for peer-reviewed and grey literature published between 2011 and 2021. The retrieved references were screened using Rayyan by two authors in a single-blind manner. References included were analyzed thematically by three reviewers using ATLAS.ti.

The systematic search retrieved 3,061 references (2,238 unique), of which 26 were included. These, in turn, synthesized 102 frameworks designed for the assessment of DHT. The included documents contained a wide variety of items to consider in HTA of DHT and often used different wording to refer to similar concepts. Through the thematic analysis, we reduced this heterogeneity. Specifically, in the first analysis phase we established 176 provisional codes related to different assessment domains. In the second one, these codes were grouped in 86 descriptive themes that, in turn, were clustered in the third phase in 61 analytical themes. The latter were organized through a vertical hierarchy of three levels: level 1 (13 domains), level 2 (38 dimensions), and level 3 (11 sub-dimensions).

There is a need to reconsider the frameworks and assessment domains used for HTA of DHT. Our thematic analysis of 26 references led to the definition of 13 domains, 38 dimensions and 11 sub-dimensions to consider in HTA of DHT. From our perspective, thematic analysis is the most appropriate method to identify assessment items for HTA of DHT from literature.

The global artificial intelligence (AI) healthcare market is predicted to grow rapidly. Various technologies for AI-based Software as Medical Device (SaMD) have been developed, and demand for their health insurance reimbursement coverage is increasing. Reimbursement policies for new medical technologies need to be thoroughly examined, despite their role in stimulating the market. The reason is that health insurance finance can have significant impact on the entire country, including patients, providers, and industry.

Based on guidelines for applying Korea’s innovative medical technologies, especially AI-based imaging medical technology, to health insurance, we examined outcome factors such as procedures and benefits. After the guidelines’ publication in 2019, we examined their impact on the medical device market through changes in the number of clinical trials and identified cases in which health insurance was listed.

The process of registering SaMD’s health insurance occurs in accordance with the existing medical technology evaluation system, and it can take up to 460 days from application to approval. If new technologies, including SaMDs demonstrate significant improvement in diagnostic capabilities and cost-effectiveness compared to existing practices, separate health insurance claims are available. Since the scheme’s announcement in 2019, items approved for SaMD clinical trials have increased (2018: ‘n=4; 2020: n=44; 2021: n=37). However, as of November 2022, only one was listed for health insurance benefits (VUNO Med-DeepBrain®), and one case was not listed on benefits but was recognized for its innovation and entered the market on the premise of suspending the health technology assessment process and accumulating real-world data (VUNO Med-DeepCARS®).™ DeepBrain® is a deep learning-based image reading technology costing about KRW80,000 (USD60) higher than conventional brain-magnetic resonance imaging and readings.

The number of SaMDs attempting clinical trials is increasing, but there is a low number of cases of reimbursement because most technologies are often classified as existing technologies and do not receive additional compensation. Since SaMD continuously is developed by accumulating data and feedback, a flexible system that can reflect this is required.

The Corona Virus Disease 2019 (COVID-19) pandemic is the biggest public health crisis of all time. Private health care plays a major role in health globally. We conducted a study to document the engagement of a Brazilian private health care organization to influence decisions to private and public health emergencies of COVID-19.

This retrospective study evaluated the outputs of the health technology assessment (HTA) group of Unimed-BH, a private Health Maintenance Organization (HMO) with 1.5 million participants in Belo Horizonte, Minas Gerais state. The study evaluated the impacts on the overview of health local decisions in the municipality and the national supplementary health agency (ANS) during the period from March 2020 to December 2022.

During the pandemic, Unimed-BH made all its sanitary decisions based on scientific research, such as the use of masks and appropriate medications. Even though some medicines, such as regdanvimab, were authorized for emergency use by the Brazilian Health Regulatory Agency (ANVISA), Unimed-BH did not recommend their use due to uncertain evidence, and months later, ANVISA withdrew the registration.

Unimed-BH also conducted a systematic review of ivermectin for COVID-19 treatment, which showed no effect, and therefore did not recommend its use. Additionally, Unimed-BH provided weekly updates on COVID-19 data, including suspected and confirmed cases, hospitalizations, and deaths in their customer portfolio. The organization also actively supported the decisions made by ANS and municipal managers using evidence and statistics on the pandemic. The Unimed-BH HTA group produced a total of 167 reports from March 2020 to December 2022.

Belo Horizonte had the lowest in-hospital mortality rates with COVID-19 in Brazil. Unimed-BH’s HTA reports provided evidence-based assessments for decision-making, proposed partnership with policymakers, fomented information transparency, and strict follow-up on pandemic numbers, which may have contributed to the lower fatality rate in our city. These findings underscore the importance of private healthcare organizations in responding to COVID-19 emergencies, and their potential to support evidence-based decision-making and minimize the impact of the pandemic.

The fast track in drug registration by the Brazilian Health Regulatory Agency, ANVISA, began in 2017 and is intended to prioritize analysis related to drugs relevant to public health. This process is appropriate in situations where there are no therapeutic alternative available or for technologies show significant improvement in safety, efficacy, or adherence to treatment.

The Brazilian public administration has a tool for accessing information called Transparency Portal. Thus, data on the number of drugs approved by fast track between 2018 and 2021 were requested through this tool and evaluated.

The data received by the Brazilian transparency portal shows that the number of requests for fast track had an increase from one in 2018 to 32 in 2021. There is an important increase of registrations, being drugs with phase II trials and with single-arm clinical trials. With registration based on a phase II trial, these patients are in fact receiving drugs in a context similar to clinical trials, but with funding from the healthcare system. Given that phase II studies are conducted in a limited population, there has been an increase in the registration of drugs with suboptimal efficacy and safety concerns. This scenario of uncertainty leads to non-adherence to treatment and a discrepancy in real-world outcomes in comparison to the clinical trial. About 70 percent of phase II trials, show no benefit, and only 30 percent proceed to later phases. It is noteworthy that about 50 percent of the studies that move on to later phases fail to show benefits. Nowadays the number of drugs approved by the fast track has increased, many probably with phase II studies and no comparator group.

Given the uncertainties in the efficacy and safety of a drug registered via fast track, often based on phase II studies, implementing provisional registration with real-world evaluation of outcomes, and coupled with financing based on risk-sharing agreements, may be a sustainable alternative for health systems.

Hepatocellular carcinoma (HCC) is the most common form of liver cancer and the fourth leading cause of cancer-related death globally. There are unmet needs for effective systematic therapy. The findings of the RESORCE trial highlighted the improvement in overall survival with regorafenib in advanced HCC patients progressing on sorafenib treatment. This study aimed to assess the cost-effectiveness of regorafenib compared with best supportive care (BSC) for advanced HCC from the Canadian healthcare system perspective.

We developed a Markov model based on four health states: live with adverse events, live without adverse events, progression, and dead. Health outcomes were measured using life-years (LYs), and quality-adjusted life-years (QALYs), and costs were presented in Canadian dollars (CAD). Clinical inputs were derived from the RESORCE trial. A 1.5 percent discount rate was applied to costs and outcomes. One-way and probabilistic sensitivity analyses were performed to assess the uncertainty in findings due to variability in parameters. TreeAge Pro software was used for model implementation.

The use of regorafenib results in a gain of 0.38 LYs and 0.25 QALYs as compared to BSC with a high incremental cost of CAD26,954 (USD22,313). The ICER for regorafenib compared with BSC was CAD105,850/QALY (USD87,624/QALY) in the base-case analysis. Further, probabilistic sensitivity analyses revealed regorafenib not to be cost-effective at a willingness-to-pay threshold of CAD50,000/QALY.

Regorafenib was not found to be cost-effective in the treatment of advanced HCC because of the lower health benefits and higher incremental costs. Lowering the official price of regorafenib or use for only selected patients who can achieve maximum benefits would enhance its cost-effectiveness and treatment preference value.

Immersion in water is a non-pharmacological method for pain relief during childbirth. The aim was to describe the experiences, values and preferences of women regarding water immersion during childbirth identified in the evidence.

A systematic review and thematic synthesis of qualitative evidence was conducted. Databases were searched from 2009 to 2022 and screened for inclusion using pre-determined criteria. Studies that used qualitative methods for data collection and analysis to investigate the opinions of women or health professionals in hospital settings were included. Non-qualitative studies, mixed methods studies that did not separately report qualitative findings and studies in languages other than English or Spanish were excluded. The Critical Appraisal Skills Programme Qualitative Research Checklist was used to assess study quality and findings were synthesized using thematic synthesis, as described by Thomas and Harden.

The final report was reviewed by several categories of health professionals that care for mothers and infants, as well women.

Thirteen studies met inclusion criteria and were included in this review. Nine studies have been identified that reflect the experience of women in relation to immersion in water during childbirth, a study exploring the factors that determine the use of immersion during childbirth according to the point of view of both women and midwives, and three more studies on midwives’ experience with water immersion during childbirth. In the qualitative studies the following key themes emerged: Reasons identified by the women/professionals for choosing a water birth, benefits experienced in water births and barriers and facilitators of immersion in water during childbirth.

The evidence from qualitative studies indicates that women associated water birth with a sense of autonomy and control over labor, and a lower level of associated pain. Further, a water birth was considered a positive experience. From the point of view of midwives, to make water births safe, there is a need for adequate resources, as well as rigorous standardized protocols.

Certain doubts about immersion in water during birth mainly regarding the safety of the infant, warrant analysis of the data to determine whether immersion in water during childbirth is safe and effective. The aim is to describe the situation regarding the use of water immersion during childbirth in hospitals of the Spanish National Health System across Spanish Autonomous Regions and Cities.

A questionnaire was developed to assess the use of water immersion on maternity wards of National Health System hospitals. The survey was reviewed by several categories of health professionals and stakeholders. The online questionnaire was distributed via email. A database was created using the Microsoft Excel 365® computer program. Quantitative results were described through percentages and frequency distributions. In the case of free responses, a content analysis was performed, coding the responses into different categories.

Regarding the status of water birth in Spain, the availability of the option of water birth varies across hospitals of the National Health System. Forty-six hospitals in 13 autonomous regions indicated that they had birthing pools on their delivery wards. Among these hospitals, 20 percent reported having more than 10 years of experience in water births, 45 percent between five and 10 years and 35 percent less than five years. Of the 46 responses received, 78 percent of the hospitals indicated that there was a demand for information on waterbirth by pregnant women. Regarding the existence of criteria for the adequate selection of pregnant women who could opt for immersion in water during childbirth, 89 percent of the hospitals indicated that these did exist, while 11 percent indicated that they did not have agreed criteria for the selection of candidates for water birth.

The availability of the option of water birth varies in hospitals across the Spanish National System. All the hospitals that have birthing pools offer them in the first stage of labor (dilation), while 32 percent also use them in the pushing stage and 15 percent during delivery of the placenta. It would be advisable to have standardized protocols and training to ensure the possibility that all pregnant women, regardless of their place of residence, can safely opt for water immersion during childbirth with satisfactory results.

The Patient Involvement (PI) Interest Group of the Spanish Network of Health Technology Assessment Agencies (RedETS) was set up in 2017 by a group of health technology assessment (HTA) researchers interested in PI. Since its inception, training and capacity-building to support PI and patient-based evidence in HTA processes has been one of its main aims. The objective of this work was to identify the needs and priorities related to training and capacity building activities to be developed within the framework of the PI Interest Group.

The PI Interest Group met on November 14, 2022, for its Annual Meeting. The group discussed the needs, priorities and possibilities on training, and carried out a prioritization exercise. For this purpose, a self-reported and anonymous questionnaire was used, which included 16 training activities. Every item was scored with a Likert-type scale ranging from 0 to 10.

The questionnaire was answered by twenty participants. The most highly rated training activities (mean less than or equal to 8) were: qualitative evidence synthesis (8.75); PI case studies (basic (8.65) and advanced (8.56) level); quality assessment tools for qualitative evidence (8.37); and qualitative research (8.11). Other proposals scoring above 7 points were: ethical aspects related to PI, evaluation of patient participation and impact, identification and recruitment procedures, and discrete choice experiments. The group agreed to organize bi-monthly webinars and three structured training activities for the whole RedETS network on: Qualitative Evidence Synthesis, Qualitative Research and PI Case Studies.

The prioritization of training activities according to PI Interest Group members allowed planning a tailored capacity-building program adapted to the needs of RedETS.

As India makes rapid strides towards universal health coverage, focusing on medical device reimbursement is key to ensuring patient access to device-based technologies. The nascent medical device reimbursement process offers a promising opportunity for interventions driven by a diverse group of stakeholders. We conducted policy research to capture these diverse perspectives and highlight key elements to develop a structured framework for reimbursement.

This research was a two-part process, including secondary research with expert interviews followed by policy research using focus group discussions (FGDs) through an online workshop with key stakeholders. We developed a white paper proposing changes to the reimbursement pathway, based on a benchmarking study of global markets and interviews with experts in the field. As a next step, key changes proposed in the white paper were deliberated upon by three focus groups (six to eight participants). Group participants were selected by quota sampling and represented key stakeholders in the reimbursement process. A discussion guide was used to capture participants’ opinions and an addendum to the white paper was released highlighting small, actionable, and impactful changes to the reimbursement process.

FGDs with key stakeholders highlighted the need to establish a more structured, inclusive, and transparent process. Accordingly, we proposed key recommendations to the medical device reimbursement process in India. A first change is the creation of an online submission portal allowing different healthcare stakeholders to submit new technologies for consideration through a streamlined pathway. Secondly, we proposed enhancing evaluation transparency by improving availability of publicly shared information on the evaluation process, metrics, and assessment timelines. We also suggested adoption of adaptive health technology assessments to leverage existing evidence for faster, efficient decision-making.

Through this process, we created a pragmatic and concrete call for a stronger voice from care-providers and patient groups in the evaluation process. Consecutively, the proposed innovative framework introducing value-based incentives for implantable medical devices will be instrumental in enabling access to quality health care for poor patients. These strategies follow the principles of value-based care and will go a long way in achieving better health outcomes for the population. The scientific initiative has been made possible with the support of St. Jude Medical India Pvt Ltd (now Abbott).

The Micra Transcatheter Pacing System (Micra TPS) is a single-chamber transcatheter leadless pacemaker (LPM). LPMs do not require leads or a subcutaneous pocket, which represent the primary sources of device-related complications with conventional transvenous pacemakers (TVPMs). Complications such as infections and lead dislodgements cause significant patient burden, which have significant economic consequences. Running a randomized controlled trial (RCT) to estimate risk differences of infrequent events requires large sample sizes and long follow-up periods. Real-world observational data, while informative, requires an appropriate study design and statistical adjustments to control for potential biases.

The Micra Coverage with Evidence Development (CED) study was a cohort study of LPM versus TVPM based on US Medicare claims data of 16,431 patients with 2-year follow up (LPM: n=6,219; TVPM: n=10,212). Propensity score matching (PSM) was applied to account for differences in baseline characteristics. As no RCT was identified in the literature, this study was presented to the Australian payer as the primary source of clinical evidence, upon which a cost-utility analysis was conducted.

After PSM, the CED study demonstrated significantly more complications with TVPM versus LPM with adjusted rates of 6.5 percent and 4.6 percent (p<0.001). Significant differences favoring LPM (p<0.01) were observed in device breakdown (1.4% vs 2.0%), dislodgment (0.4% vs 1.2%) and infection (<0.1% vs 0.6%). Based on these findings, a claim of superior safety was accepted by Medical Services Advisory Committee (MSAC) to support reimbursement. In making this decision, MSAC considered that the large sample size and propensity weighting overcame some of the potential biases and the magnitude of the benefit supported cost-effectiveness relative to TVPM.

The lack of a sufficiently powered RCT with an extended follow-up period can mean the impact and benefits of new technologies that reduce clinically important adverse events of relative infrequency are not formally incorporated into payer decision making, particularly where RCTs are a requirement. A well-designed observational study can provide valuable, real-world evidence to support a HTA for reimbursement decisions.

The National Committee for Health Technology Incorporation of the Brazilian Public Health System’ s (Conitec) principle is to advise the Ministry of Health (MS) in the tasks related to incorporation, exclusion or modification of any health technologies into the Unified Health System (SUS). Moreover, this also involves alteration of clinical protocols or therapeutic guidelines. All of the recommendations consider the international classification of diseases (CID) as a common language that allows health professionals and managers to understand standardized information, to identify trends and benefits of recommendations in each therapeutic area.

This exploratory, descriptive and retrospective study aims to provide qualitative and quantitative data from the technologies evaluated by the Conitec in the period June 2012 to November 2022. Data were extracted in Conitec’s website.

The searches resulted in 763 recommendations in total. Among them, the most evaluated therapeutic area was Infectology with 126 technologies (16.5%). In this field the highlighted diseases and conditions were Hepatitis 42 (33.3%); HIV 23 (18.3%) and COVID-19 11 (8.7%). In Oncology, 113 recommended technologies (14.8%) were identified, in order of prominence for the diseases: Breast Cancer 21 (18.6%); Colorectal Cancer 11 (9.7%); Leukemias 17 (15.0%). In the Respiratory Diseases area, 89 technologies (11.7%) were recommended, among them: Chronic obstructive pulmonary disease (COPD) 17 (19.1%); Asthma 15 (16.9%) and COVID-19 11 (12.4%). These results clarify which diseases are most needing new technologies to be treated.

The results show what conditions and fields in health needs to be prioritized for public policies and prevention measures. This study demonstrates how important is to make accessible the public health information, improving public knowledge and social actions in SUS.

Earlier cancer diagnosis and advances in science are resulting in improved patient and societal outcomes. However, payer frameworks and methods can find it difficult to keep pace with scientific progress, evolution of endpoints, and assess the wider value of these advances.

A multidisciplinary, international group of experts working in the cancer field was brought together to reach consensus on key principles of defining and assessing of cancer treatment value. A Delphi-based approach including surveys, virtual panels, interviews and structured online discussions was used to reach consensus. This work was initiated and funded by AstraZeneca.

Twenty-four experts from across the world (including patient advocates, oncologists, health economists, regulators, members of payer and health technology assessment (HTA) bodies) reached consensus on seven key principles across two themes, oncology relevant endpoints and dimensions of value. Three of the seven principles were found to be of particular relevance to HTA bodies and payers: assessing broad economic impact of new medicines (including socio-economic and caregiver impact), where early-stage cancer treatments can enhance patients’ ability to lead productive lives and contribute to economic activity; consider other value aspects of relevance to patients and society; use of Managed Entry Agreements (MEAs) supported by ongoing evidence collection to help address decision-maker evidence needs and address clinical uncertainty.

Incentivizing access to early-stage treatments can promote cancer control, improved outcomes and generate long-term societal benefit. Furthermore, early diagnosis and treatment at earlier stages of cancer can be cost-effective, and sometimes cost-saving, as well as provide opportunities for cure. Expanding value components in therapy assessments to include, for example, insurance value, the value of choice, scientific spillovers, and wider societal perspectives, along with structured MEAs to manage clinical uncertainty and balance budgets will help realize the potential to eliminate cancer as the cause of death.

South Korea has introduced conditionality to coverage decisions for certain difficult or high-risk procedures. The transcatheter aortic valve implantation (TAVI) was included in the coverage with evidence development (CED) in 2014. This study reviewed the results of reassessment for the TAVI using real world data (RWD) and suggested its implications.

Healthcare providers authorized to use the promising technologies are required to collect the RWD for suitability evaluation, safety monitoring, and cost-effectiveness, differing from the general reassessment process. In 2021, 45 healthcare providers collected clinical information for TAVI patients. Their registries were linked with the national health insurance claims, which provided data on 19 items to assess safety and effectiveness such as overall mortality, reoperation rates, hospital readmission rates, and degree of functional improvement.

According to the Society of Thoracic Surgeons’ predicted risk of mortality (STS), 988 TAVI patients were classified into three groups; high (STS >8 percent, n=347), intermediate (STS 4-8 percent, n=272), and low (STS <4 percent, n=369); We compared main outcomes and estimated survival probabilities between subgroups. Within 30 days, the overall mortality rates were 4.9 percent (high), 2.6 percent (intermediate), and 1.4 percent (low); major bleeding rates were 7.6 percent (high), 6.2 percent (intermediate),and 1.4 percent (low); incidence of new atrial fibrillation were 6.8 percent (high), 4.2 percent (intermediate), and 3.2 percent (low). Based on the quantitative results using RWD and systematic review for the safety and effectiveness, TAVI is reported to have essential benefits for high-risk group and elderly patients (>80 years). Whereas, intermediate and low-risk groups have out-of-pocket payment rates of 50 percent and 80 percent, respectively.

The reassessment system through RWD accumulation enabled the evidence-based evaluation for the TAVI. Based on the transition to CED for essential benefits, a systematic framework such as RWD collection from treatment commencement should be introduced to broaden RWD use for benefit management of medical technologies with uncertain levels of evidence. Therefore, this ensures overall quality of care and effective coverage in health.

Cardiac Implantable Electronic Device (CIED) infection is a serious complication associated with morbidity, mortality and high healthcare costs. Internationally, the published rate of CIED infection ranges from 1.0 percent to 1.6 percent. There is a lack of data on CIED infection rates in Australia; the reported range is from less than 1 percent at 30 days to 7 percent over 5 years. Due to the variability within the limited number of studies there is a need for further analyses of CIED infection rates in Australia.

This was a retrospective cohort study using secondary linked hospital (the NSW Admitted Patient Data Collection) and mortality data for patients who underwent CIED procedures between July 2017 and June 2020 in NSW. Overall and procedure-and patient-specific incidence of infection was calculated.

A total of 23,786 CIED procedures were performed among 22,404 patients and 422 CIED infections were identified, giving an overall infection rate of 1.77 percent. When infections were limited to those following a CIED procedure in the period July 2017-June 2020 (n=309), the procedure-specific CIED infection rate was 1.30 percent, ranging from 1.01 percent for permanent pacemaker (PPM) to 2.71 percent for cardiac resynchronization therapy-defibrillator (CRT-D). The proportion of patients undergoing CIED procedures in this period who had a subsequent CIED infection was 1.29 percent, ranging from 0.97 percent for permanent pacemaker (PPM) to 3.05 percent for cardiac resynchronization therapy defibrillator (CRT-D). Procedure-based infection rate in high-risk patients (generator replacement; system upgrade; revision; or CRT-D procedure) was 1.47 percent and patient-based infection rate was 1.68 percent. Infection rate was highest within the first month following the CIED procedure that dropped significantly over time.

Rates of infection were highest among patients with cardiac resynchronization therapy (CRT) devices, and those who underwent revision or upgrade procedures. Ongoing monitoring of CIED infection rates and preventative measures are necessary, especially for high-risk patients. This study highlights the important role linked secondary data has in reducing uncertainty and removing the reliance on international estimates by providing targeted, local data for health technology assessment.

In Australia, approximately 200,000 patients have a cardiac implantable electronic device (CIED), and in an aging population that number is rising. CIED-related infections are also increasing, causing considerable morbidity and mortality, and substantial healthcare costs. Internationally, the rate of CIED infection ranges from 1.0 percent to 1.6 percent, while in Australia, the reported range is from less than 1 percent to 7.0 percent. The average hospital cost to treat an infection in the US ranges between USD48,000–USD83,000. To date, few publications have estimated the cost of CIED infections in Australia. Critical appraisal of these studies has highlighted issues in their methodology, making them unreliable sources for use in economic evaluations. The purpose of this study was to utilize Australian routinely collected health data to robustly model costs of CIED infections to reduce uncertainty for future health technology assessment (HTA).

The cost of treating a CIED infection was modeled for the public and private sector including cost of system removal and re-implantation procedures, hospital and intensive care unit (ICU) stay, and outpatient follow-up. Cost inputs were obtained from the Australian Prostheses List, Medicare Benefits Schedule, Australian Institute of Health and Welfare, and Private Hospital Data Bureau. Other inputs were obtained by surveying Australian clinicians, which were validated with published data. Phone interviews and online surveys were conducted with clinicians to elicit specific Australian practice pathways for patients with a CIED infection.

The majority of patients with a CIED have their device system removed (95-100%) and re-implanted (83%) once the infection has cleared. In the private sector, cost of infection ranged from AUD80,869 (USD54,384) for a single chamber pacemaker (PM), to AUD140,103 (USD94,248) for a dual chamber Implantable Cardioverter-defibrillator (ICD). Modeled costs of CIED infection were slightly lower in the public sector (AUD73,643-AUD88,446 (USD49,555 – USD59,516) for the same devices).

The cost of a CIED infections to the healthcare system is high and differs by device type. Utilizing local real-world data to estimate costs will improve accuracy of economic evaluations and reduce uncertainty for decision makers.

Critical congenital heart disease (CCHD) refers to a group of heart defects that cause serious, life-threatening symptoms in the neonatal period and requires timely surgical or catheter interventions. We tried to explore current status of CCHD burden and the effect of early diagnosis of CCHD to mortality using the Korean national health insurance (NHI) data.

We analyzed the national health insurance (NHI) data from 2014 to 2018. We identified CCHD patients using the diagnosis codes and intervention codes from the claim data and the prevalence, mortality and medical expenditure of CCHD were analyzed. We linked neonatal data with their mother’s medical claim data and developed retrospective cohort data set for analyzing the effect of early diagnosis to mortality and related outcomes of CCHD treatment.

The annual prevalence of neonatal CCHD in Korea was 0.144% percent. A total of 2,241 CCHD neonates, 1,546 (69.0%) underwent cardiac ultrasound within three days after birth, and mothers of 419 neonates had a record of prenatal fetal ultrasound (18.7%). In our comparison of neonates diagnosed with CCHD within three days of birth with those diagnosed with CCHD on or after day 4 of birth, the probability of early diagnosis increased for preterm infants and infants with low birth rate. Regarding mortality rate, most types of CCHD showed a significantly higher mortality rate in the early diagnosis group.

The reason for the high mortality rate despite a high early diagnosis rate pertains to the high percentage of patients with severe conditions that induce a serious heart rate within three days of birth. More than half of the neonates with CCHD were found to have not undergone a prenatal fetal ultrasound, rendering this an important policy target.

In December 2021 the European Union (EU) Health Technology Assessment (HTA) Regulation, a key pillar of the EU Pharmaceutical Strategy, was adopted by the Council and the European Parliament. The focus areas of Joint HTA Cooperation include Joint Clinical Assessments (JCA), Joint Scientific Consultations (JSC), and joint early-stage horizon scanning. The European HTA regulation will be adopted in a stepwise approach and from 2030 onwards, all products (drugs, high-risk medical devices, and in vitro diagnostics) approved in all indications will be subjected to JCA in EU.

A targeted literature research was performed for policies and the European Network for HTA (EUnetHTA) methodological guidelines describing the HTA methods including scoping process, comparators, endpoints, the applicability of evidence, and validity of clinical studies. Additionally, the anticipated opportunities and challenges were also summarized with respect to these methods.

EUnetHTA put forward a timeline for different activities over the next three years as part of the new EU HTA Regulation, including key deadlines for ongoing EUnetHTA consultations on the processes and methods. EUnetHTA will set up a new ecosystem across the EU as it aims to reduce duplication and time to access by supplementing multiple national clinical assessments with a joint central assessment. In any case, assessment of added value and pricing and reimbursement decisions will still occur at the national level.

Additionally, EU HTA may promote harmonization of processes, standards, and evidence requirements, which will increase predictability and simplify evidence requirements. However, differences in clinical practice, standard of care, and national priorities may lead to assessments that are not generalizable to all Member States.

The joint EU HTA cooperation will benefit countries which have less developed or do not have established HTA expertise or infrastructure. However, the JCA process could result in increased requirements for clinical evidence generation as relative effectiveness and relevance of outcomes to patients gain further importance for products to successfully gain access across countries.