This project aimed to identify methodological issues reported by Evidence Review Groups (ERGs) in the systematic reviews (SR) supporting single technology appraisal (STA) submissions to the National Institute for Health and Care Excellence (NICE). STA submissions contain SRs related to the clinical and cost-effectiveness of the proposed intervention and NICE require the methods for these reviews to be clearly detailed in the submission. The intention of this project was to identify methodological aspects of submissions where companies may need additional guidance or support to provide the evidence required for efficient and effective decision-making and in turn facilitate timely access to clinical innovation.

From 2019, 61 STAs were identified from the NICE website, of which 46 were included. We extracted information about the data requests or clarification questions raised by the ERG in relation to the methodological section of both the clinical and cost effectiveness SRs reported in the STA. We then categorized these data and grouped by theme to determine the most common methodological issues faced by companies. We did not assess whether comments made by the ERG were accurate or justified.

For both clinical and cost-effectiveness SRs, the most frequent clarification questions arose from the search methods section, specifically seeking information about missing intervention or comparator terms, the use of search filters and search platforms. Clarification questions were also commonly asked about the appropriateness of interventions and comparators. There were very few clarification questions asked about screening, data extraction or risk of bias assessment.

Companies looking to submit an STA should align their submission methodology to established best practice guidance for systematic reviewing to ensure their methods are fit for purpose and avoid unnecessary delays to the STA process. Consistency with the PRISMA reporting standards would help ensure that the ERG is provided with the information needed to assess the appropriateness of the STA methodology and is likely to reduce the need for clarification questions.

The rising antimicrobial resistance (AMR) and the difficulty in developing new antibiotics are causing a global public health problem. This analysis aims to better understand the clinical and economic value of new antibiotic treatment strategies, in order to inform clinical and antibiotic formulary decisions.

We applied a published and validated dynamic disease transmission and cost-effectiveness model of AMR with a 10-year time horizon and discount rate of five percent to evaluate the clinical and economic outcomes of introducing a new antibiotic, namely, Ceftazidime/Avibactam (CAZ-AVI) for treating AMR infections in Zhejiang Province, China. Together with piperacillin-tazobactam (pip/taz) and meropenem, we explored the impact of six treatment strategies across three common infections (complicated intra-abdominal infection (cIAI), hospital-acquired/ventilator-associated pneumonia (HAP/VAP) and infections with limited treatment options (LTO)), and pathogens (Escherichia coli, Klebsiella spp., and Pseudomonas aeruginosa). These treatment strategies included (i) current treatment strategy (pip/taz and meropenem, no CAZ-AVI), (ii) CAZ-AVI at the third line, (iii) CAZ-AVI at the second line, (iv) CAZ-AVI at the first line, (v) first line diversity (i.e., equal pip/taz and CAZ-AVI at the first line; meropenem at the last line) and (vi) all-lines diversity (pip/taz, meropenem and CAZ-AVI used randomly and only once). The data with a total of 10,905 patients were collected from a tier-3 hospital from 2018 to 2021.

Under the current treatment strategy, the hospital length of stay (LOS) and costs over ten years were estimated to be 1,588,763 days and CNY3,898,198,802 (USD559,781,348), respectively, associated with 142,999 quality-adjusted life-years (QALYs) lost, resulting in the resistance of pip/taz and meropenem being 42.0 percent and 49.9 percent respectively. In contrast, the other five treatment strategies all have shown improved outcomes, among which the “all-lines diversity” carried the greatest benefit, saving CNY1,646.04 (USD236.37) for each additional QALY gained, with the net monetary benefit being CNY24,727,102,215 (USD3,550,811,878).

Introducing CAZ-AVI had positive impact on clinical and economic outcomes for treating AMR, and diversifying early the antibiotics might yield the best benefits.

The heart rate characteristics (HRC) analysis is a technique for predicting and monitoring neonatal infectious diseases in infants with a risk for infectious disease, such as sepsis, using the HRC index based on the electrocardiogram (ECG) data transmitted from a HeRO® device connected to an ECG monitor.

The assessments were performed via a systematic review. Searching five databases (KoreaMed, Ovid-MEDLINE, Ovid-EMBASE, and Cochrane) yielded 284 related studies. Two reviewers independently assessed the quality of these studies, using the Scottish Intercollegiate Guidelines Network checklist and the assessment results were described based on the results of the quality appraisal and level of evidence.

The safety and effectiveness of the technique were assessed based on eight non-Korean studies (one randomized controlled trial (RCT), five cohort studies, one case-control study, one case series). The four studies that reported area under the curve (AUC) values, the timing of the analysis and AUC values (0.67– 0.75) were inconsistent among the studies. The elevation of the HRC index prior to the diagnosis of an infectious disease was not observed for meningitis or for multiple infectious diseases. In addition, although the group with additional monitoring using the technique had a significantly lower mortality rate, whether the HRC index had a direct impact on the infection prevention activities and the possibility of bias arising from the researchers’ precautionary treatment based on the clinical symptoms and other test results as well as the actual time of disease onset are unknown.

Therefore, the committee presented the following recommendations based on the available evidence and expert opinion. HRC analysis is a safe technique for predicting and monitoring neonatal infectious diseases in infants with a risk for infectious disease, such as sepsis, using the HRC index based on the ECG data transmitted from a HeRO® device connected to an ECG monitor; however, further research is needed to determine its effectiveness (Level of evidence C, Technology category II-a).

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 reviewed evidence for incorporating a mandatory neonatal CCHD screening test as a national public health project for all neonates born in Korea by analyzing the validity and cost-effectiveness of neonatal CCHD screening using pulse oximetry in Korea.

We performed a rapid literature review to establish models for the diagnostic accuracy and economic evaluation of pulse oximetry. Also, we analyzed the prevalence, mortality, and medical expenditure for different types of CCHD using the national health insurance (NHI) data. We analyzed the cost-effectiveness of pulse oximetry by comparing the group of neonates who received a combination of a physical examination and pulse oximetry, and group of neonates who only received a physical examination. For the cost-effectiveness analysis for the CCHD screening test in this study, we used a duration of one year, diagnostic accuracy as the clinical endpoint, and Life Year Gain (LYG) as the effectiveness indicator.

We used recent systematic review he pooled sensitivity can be enhanced from 76.5 percent (pulse oximetry alone) to 92 percent (combined with physical examination). We used a total of 2,334 neonates with CCHD data for the economic model. Our analysis revealed that adding pulse oximetry to the routine neonatal physical examination leads to 2.34 of LYG and a cost difference of USD1,080,602, showing a ICER of KRW610,063,240 (USD461,857)/LYG.

Considering the benefit of LYG and cost of reducing the complications and after effects of newborns with CCHD who survived early diagnosis, it is considered to be worthwhile in Korea for a mandatory screening test.

Hereditary Transthyretin amyloidosis (hATTR) is a rare autosomal dominant, multisystemic, progressive, and potentially fatal genetic disease. Currently, the only drug made available in the Brazilian National Health System to treat hATTR is tafamidis meglumine, indicated for symptomatic adult patients in early stage (stage 1) and not undergoing liver transplantation for amyloidosis associated with hATTR.

A systematic review was conducted in the databases MEDLINE via Pubmed, Embase, The Cochrane Library, and LILACS addressing the question “Is patisiran treatment effective and safe for patients diagnosed with ATTRh amyloidosis with stage 2 polyneuropathy or who have an inadequate response to tafamidis?”

The 13 studies included in the review demonstrate the efficacy of patisiran in reducing the neuropathic progression of the disease, as evidenced by decreased mNIS+7 scale scores following 18-month use of the drug. Improvements in the quality of life of patients taking patisiran have been reported, as measured by reduced scores on the Norfolk-QoL-DN scale. Patisiran has also been shown to be effective in reducing NT-proBNP, a marker related to cardiac stress. Improvements in the nutritional status of patients taking patisiran were demonstrated by increasing modified body mass index (BMI). Good tolerability of patisiran was observed by patients using it. Most adverse events were classified as mild or moderate. The studies indicated that the occurrence of deaths is similar between the patisiran and placebo groups. Most deaths were related to cardiac events and were not associated with the use of patisiran.

The use of patisiran in patients with hATTR demonstrated efficacy in reducing the neuropathic progression of the disease, evidenced by decreased mNIS+7 scale scores, improvements in quality of life as measured by reduced Norfolk-QoL-D scale scores, and reduced NT-proBNP. The drug patisiran was well tolerated, with most adverse events rated as mild and moderate.

Despite intense efforts in development of new treatments over the last two decades, symptomatic treatments remain the only option for the vast majority patients diagnosed with dementia due to Alzheimer’s disease (AD). There remains a significant unmet need for disease modifying therapies (DMTs) to slow or stop AD progression. DMTs in development are targeting early stages of AD (pre-clinical, mild cognitive impairment and mild dementia stages), thereby creating an entirely new treatment paradigm for patients, clinicians, and payers. A key challenge will be in identifying the appropriate patient for treatment in a very heterogenous population. We have performed a literature review to better understand and define the AD population, with a view to enabling more targeted treatment in future.

Embase, MEDLINE and the Cochrane Library were searched to identify publications between 2010-2021 on observational studies reporting evidence on prevalence and subgroup identification, including clinical feasibility of identification. The search was restricted to English language.

We identified 45 studies, mostly from Europe, USA and Asia. Populations were primarily grouped based on generic demographic factors (e.g., age, sex, gender), AD staging, comorbidities or biomarkers. Prevalence data was available for six subpopulations: pre-dementia stage, mild dementia, age, Apolipoprotein E (APOE) genotype, comorbid obesity and hypertension. Across these, data on prevalence were heterogenous depending on study design and country of origin, and ranging between 66 million to 102 million for people with mild AD dementia, or as another example, ranging between 46 million to 92 million for APOE genotype carriers worldwide.

The heterogeneity and the uncertainty in prevalence of the AD population represent big challenges to clinicians and payers. Future discussions on target patient identification for new treatments should be aligned and integrated with current clinical practice e.g. leveraging validated biomarkers as diagnostic tools. Additional research on an integrated approach to identify patients who would benefit the most from DMTs will be needed.

Medical technologies are evolving rapidly, with many new and expensive technologies entering the market constantly, challenging the sustainability of the public healthcare system. Early health technology assessment (HTA) to inform subsidy decision for innovative medical technologies, before they diffuse into the public healthcare system, may drive appropriate early adoption or curtail inappropriate use. This abstract describes the Agency for Care Effectiveness (ACE)’s experience in conducting early HTAs and key challenges faced.

During ACE’s 2021 topic prioritization exercise, ACE took a proactive approach by considering medical technologies identified from horizon scanning (HS) for subsidy evaluation. Two topics were shortlisted from HS. Standard HTA evaluation framework and local clinician consultation were used to define the evaluation scope and clinical pathways. Literature search and appraisal were conducted for safety, effectiveness, and economic evidence. Budget impact estimations and organizational feasibility assessment were additional domains considered for subsidy decision-making by the Ministry of Health Medical Technology Advisory Committee (MTAC).

MTAC did not recommend subsidy for the two technologies due to weak evidence base, largely due to a lack of comparative evidence, small samples, short-term follow-ups, or heterogeneity of population. Additional considerations included potentially high budget impact or organizational feasibility issues such as substantial capital and maintenance cost and infrastructure reconfiguration required. During the evaluation, key challenges of assessing such technologies in their early diffusion within the healthcare system were: (i) differing clinical opinions on whether the technology meets an unmet need; (ii) uncertain place in the clinical management algorithm for the relevant indications; (iii) sparse and weak evidence; (iv) uncertain financial implications to the healthcare system due to a lack of available local costs.

Early HTA on medical technologies identified from HS can be a useful tool to guide subsidy decisions; however, several challenges exist. Careful selection of technologies and timing of evaluation are critical. Seeking stakeholder inputs earlier would ensure shortlisting appropriate technologies with greater clinical need for HTA.

Defining drug innovation can be challenging and there is no consensus on what a truly “innovative” medicine is. The Italian Medicine Agency (AIFA) has established an approach to assess innovativeness based on therapeutic need, added therapeutic value, and quality of evidence. However, judgment can be subjective and may not be adequate for assessment at the time of marketing authorization, when only preliminary evidence – often from non-comparative or non-randomized trials – are available. We developed a transparent methodology for early assessment of innovativeness at the time of marketing authorization, based on AIFA guidelines.

Since the perspective was the marketing authorization date, only data available at agency’s Medical Review or pivotal trial publications were considered. AIFA criteria were revisited, using oncology medicines approved in the last 10 years as a base case. Impact of preliminary evidence and inadequate study design was considered.

Each assessment should refer to the first approved specific indication and predefined clinically relevant outcomes. When more than one study was presented, best methodological quality, larger sample and/or longer follow-up was selected. Four domains were established: Therapeutical need: existence and clinical benefits of alternative therapies; Clinical benefit added when compared to those alternatives; Suitability of study design considering adequate comparator group, relevant outcome assessed and randomization; Risk of bias. For each domain, clear and specific criteria were defined in consensus by a group of experts in health technology assessment (HTA) and were applied to all cancer drugs evaluated.

Efficacy evidence available for marketing authorization are often based on preliminary data, arising from single randomized clinical trials or even non-comparative studies, which difficult early assessments of innovativeness. For this reason, transparent and reproducible methodologies can be useful not only to HTA bodies, but also for other key stakeholders in the pharmaceutical market, such as investors, researchers, doctors, and governments.

In the UK over 12,400 yearly cases of head and neck cancers are reported (2021). Pharyngolaryngeal biopsies (OLB) may improve the speed of diagnosis and treatment of head and neck cancers under local anesthetic. The Scottish Health Technologies Group (SHTG) published advice on this technology in 2018. Since this, additional evidence has been published to warrant a health technology assessment (HTA) for Wales. The aim of this review was to provide update on the clinical and cost-effectiveness of OLB when compared to undergoing biopsy in an operating theatre (OTB) under general anesthetic to inform decision making in Wales.

A rapid review was undertaken of relevant databases since 2018 of the clinical evidence, health economics and patient perspectives relevant to Wales. Health Technology Wales (HTW) developed a de-novo cost-utility analysis comparing OLB to OTB over a lifetime horizon. Inputs were sourced from the SHTG budget impact analysis, updated with values more relevant to a Welsh setting.

From consultation to biopsy procedure, the mean number of days was 1.3 for OLB compared to 17.4 days under OTB (p < 0.05). The mean time from consultation to start of treatment was 27 days for OLB compared to 41.5 days for OTB (p < 0.05). The economic analysis found a resulting ICER of GBP21,011 (EUR23,824.23) in a population with 2,183 at risk patients. As OLB was associated with lower costs (GBP816 per person) (EUR925.26) and fewer quality adjusted life years than OTB (-0.04), this ICER corresponds to OLB being considered a cost-effective diagnostic strategy.

HTW guidance was able to recommend use of OLB within the diagnostic pathway for head and neck cancers within Wales. For people with a positive test, OLB is sufficient to confirm a diagnosis but should not be used to rule out a diagnosis due to the potential in reducing the time to diagnosis and treatment in a cost-saving way.

Extracorporeal shock wave therapy (ESWT) has been used since the 1990s to treat various musculoskeletal disorders, but there is considerable controversy regarding the effectiveness of ESWT. Our aim was to conduct a systematic review of randomized controlled trials (RCT) to investigate the effectiveness of ESWT for plantar fasciitis.

A comprehensive search was conducted via electronic databases including MEDLINE, Embase, the Cochrane Controlled trials register, and 5 Korean databases from inception date to April 2022. Two review authors independently assessed studies for inclusion and risk of bias, and extracted study data. Major outcomes were pain relief, function, and quality of life.

We identified a total of 48 RCTs comparing ESWT with corticosteroid injection (n=14), conventional therapies (n=19), and sham control (n=21). Most studies included participants with chronic heel pain diagnosed as plantar fasciitis. All trials were susceptible to bias. In terms of pain results, ESWT showed no significant difference when compared with the steroid injection group and the conventional therapy group, and significant pain relief was confirmed only compared to the sham control group (Mean Difference -1.71; 95% confidence interval [CI] -2.44,-0.98; I2=70%;). Functional outcomes were significantly improved in the ESWT group compared to the steroid injection group (standardized mean difference 0.45; 95% CI 0.27,0.63; I2=0%) and the sham control group (SMD 0.84, 95% CI 0.23,1.45; I2=91%), but no significant difference was found when compared to the conventional therapy group.

Based upon the currently available low certainty evidence because of wide clinical diversity and varying treatment protocols of included trials, ESWT is associated with improved function and may be associated with pain reduction in plantar fasciitis. Further evidence is needed from well-designed studies with a standard dose and treatment protocol.

Health technology assessment (HTA) agencies are increasingly embracing patient experience data (PED) to support reimbursement decisions. This study aimed to describe the European Network for HTA (EUnetHTA) and HTA agencies expectations regarding PED to support reimbursement in France, Germany, Italy, Spain and the UK.

Published HTA guidance documents were reviewed to identify recommendations related to clinical outcomes assessment (COA) (including disease-specificity, validation, analyses, endpoints and interpretation) and other forms of PED (e.g., patient preference information) in HTA decision-making. Insights from guidance documents were supplemented with a review of literature and published HTA cases and interviews with key opinion leaders (KOLs) focused on current and future states.

The German and French guidance documents include PED recommendations focused on relevant COA and health-related quality of life data, without detailing preferred COA measures. However, key differences were noted between these two countries in the methodological approaches regarding responder definitions, acceptable missing data threshold and multiplicity analyses. These differences were reinforced by the case studies and the KOLs. UK’s sources also focused on COA, in general proposing specific use of the EQ-5D to derive utility values for modelling, but included limited details on other PED-related elements. The Italian and Spanish guidance documents do not detail COA or other PED expectations, but the Italian KOL described that COA is considered if submitted. The currently developed EUnetHTA21 guidelines include PED-related information that bear the signature of certain individual HTA bodies. Globally, there is limited interest in PED beyond COA across the agencies.

The level of expectations with regards to PED varies across EUnetHTA and several European HTA agencies. Interest in PED derived from non-COA sources is limited across the countries. Knowing each agency’s expectations with regards to PED is key when submitting HTA evidence dossiers and should be considered early in clinical trial design to integrate market access perspectives and optimize drug development. Global harmonization would help advancing PED measurement standards.

Health technology assessment (HTA) agencies are increasingly embracing patient experience data (PED) to support reimbursement decisions. This study aimed to review HTA agencies’ expectations with regards to utilizing PED to support drug reimbursement in Australia, Japan, and China.

Published HTA guidance documents were reviewed in 2021 to identify any PED-specific information. If available, recommendations related to the type of PED (e.g., generic vs. disease-specific clinical outcomes assessment (COA)); COA validation, analyses, endpoints and interpretation; and the interest in PED beyond COA in HTA decision-making (e.g., patient preference information) were reviewed. Literature review and semi-structured interviews with key opinion leaders (KOLs) were conducted to further explore these themes and future trends with regards to PED.

Australia’s Pharmaceutical Benefits Advisory Committee guidance document includes a dedicated section on patient-reported outcomes (PRO), providing details on preferred PRO instruments; validation expectations; and recommended methods to explore score interpretation, assess and report PRO results and handle missing data. While PED derived from non-PRO sources are not discussed in the guidance, the KOL noted that they should not be ruled out. Japan’s Center for Outcomes Research and Economic Evaluation for Health guideline includes a section dedicated to PROs without details related to instrument validation, analyses and interpretation, however, is focused on the use of PRO to inform health economic assessments. In China, the HTA center of China National Health Commission drafted two disease-specific technical guidance documents recommending the inclusion of PROs in efficacy assessments and use of instruments relevant in the Chinese population; these points were echoed by the KOL interviewed.

There are recommendations on PED use included in country-specific guidance documents, however their level of detail varies greatly. Knowing each agency’s expectations with regards to PED is key when submitting HTA evidence dossiers and should be considered early in clinical trial design to integrate market access perspectives and optimize drug development.

Health technology assessment (HTA) bodies support healthcare decision-making by producing different kind of products. The high speed of the healthcare innovations and the scenarios such as the COVID-19 pandemic challenge HTA organizations to adapt their services to better respond to these demands. The Spanish Network of HTA Agencies (RedETS) is redefining the services and the products in its portfolio. The first step has been conducting a review in order to identify the most relevant HTA products.

A scoping review with two sections was conducted: (i) analysis of results from a bibliographic search performed in the main biomedical databases; and (ii) analysis of results from a manual review of the official websites of seven international HTA agencies: CADTH (Canada), INESSS (Canada), SBU (Sweden), NICE (United Kingdom), IQWIG (Germany), HAS (France), IECS (Argentina) and IETS (Colombia). The EUnetHTA website was also reviewed.

The search identified 1,311 references; 21 studies were considered relevant. The main topic found was about rapid responses services. The standard timeline for these should be less than six months, with even some produced in days. Transparency about methodology and involvement of decision-makers were considered key points to be included. Website analysis revealed similar HTA reports production but variation in the domains and elements considered. The timeframe for conducting a full HTA report can be up to 24 months, with a median of 12 months. Agencies also offer some kinds of rapid response services. Scientific consultation and horizon scanning systems for emerging technologies are other services performed by some agencies.

The review reveals that agencies have different products to address different needs throughout the life cycle of technologies: from scientific advice to full HTA. In addition, HTA agencies have incorporated rapid responses into their services. According to literature, these products could support short-term decision-making.

Hyperparathyroidism is a medical condition characterized by an excess of parathyroid hormone (PTH) in the blood. This hormone is produced by the parathyroid glands, located behind the thyroid gland. PTH plays a crucial role in regulating calcium levels in the body, which is necessary for muscle activity, nerve transmission, and maintaining bone density. In this study, the authors aimed to determine the parameters necessary for defining the delivery process of interventions for managing hyperparathyroidism.

To achieve this, a group of Key Opinion Leaders (KOLs) was surveyed using a questionnaire to investigate specific drivers such as the duration of interventions, drug therapies employed, and materials required. The authors also estimated the indirect costs associated with patients and caregivers. The economic analysis considered the perspectives of both the Italian National Health Service (SSN) and the community. The analysis was conducted using Activity Based Costing (ABC) methods to determine the full cost sustained for a parathyroidectomy surgery and pharmacological therapies per patient treated, as well as the average resources absorbed by a patient managed through pure surveillance.

The results showed that the average annual cost of parathyroidectomy and pharmacological therapies per patient was EUR5,193.20 and EUR1,726.96, respectively. Productivity losses due to the interventions amounted to EUR858.21 and EUR66.80 for the patient and caregiver, respectively. The pure surveillance strategy incurred an average yearly cost of EUR197.42.

The study’s major limitation was the lack of evidence available concerning the therapies under analysis, particularly in the Italian context. Nonetheless, the survey of clinicians provided useful insights into the expenditures associated with implementing interventions for managing hyperparathyroidism. In conclusion, managing hyperparathyroidism involves considerable costs, and healthcare providers must consider the perspectives of both the SSN and the community when estimating the economic impact of interventions. The authors’ analysis provides insights into the cost of different interventions, which could help healthcare providers make informed decisions when managing hyperparathyroidism.

Lack of awareness has been recognized as one of the most important barriers in the implementation of health technology assessment (HTA) across numerous countries. Medical students have been identified as a vital target group to instill an awareness, basic knowledge, and positive attitude towards HTA from the beginning of their medical career. Presently, there was no study found conducted on HTA awareness in medical students in Malaysia. This study aims to determine the level of awareness of HTA, and its predictors among clinical year medical students in public universities in Klang Valley, Malaysia.

A cross-sectional study using the stratified random sampling method was conducted among clinical year medical students in four public universities in Klang Valley, Malaysia. The data on level of awareness of HTA and its associated factors were collected using a self-administered online questionnaire. Data were analyzed using IBM SPSS version 27 to determine the level of awareness of HTA, its associated factors and predictors.

The total number of study participants was 323 and the response rate was 90 percent. The analysis showed that 69 percent of respondents had low level of awareness of HTA while 31 percent had high level of awareness of HTA. Factors associated with the level of awareness of HTA was attitude towards HTA (p < 0.001). The predictors of level of awareness of HTA were attitude towards HTA (Adjusted Odds Ratio (AOR) = 7.417, 95% confidence interval [CI]: 3.491, 15.758), peer interaction on HTA (AOR = 0.320, 95% CI: 0.115, 0.888), and previous training on HTA (AOR = 4.849, 95% CI: 1.096, 21.444).

The majority of clinical year medical students in public university have low awareness of HTA which can be predicted by the level of attitude, peer interaction and training of HTA. Therefore, it is important to increase awareness among these future medical practitioners to encourage evidence-based decision-making. Early exposure and introduction of HTA in the current university curriculum in collaboration with a local HTA agency can be recommended to increase awareness as well as to improve students’ attitude towards HTA.

Alzheimer’s disease (AD), the most common cause of dementia, is becoming increasingly prevalent worldwide. Understanding the current burden of AD is important in health economic evaluations of new therapies. We aimed to estimate the burden of illness, and healthcare costs of people living with AD using a large, comprehensive real-world database in England.

A retrospective cohort study was undertaken in the Discover-NOW dataset, a real-world database containing the linked primary and secondary care electronic health records of ˜3 million people living in North West London, England. Patients diagnosed with AD were followed from the later of 1 January 2010 or AD diagnosis date, to the earlier of 31 December 2021 or end of follow up (maximum 10 years). Baseline prevalence of 33 comorbidities, incidence of 7 outcomes (survival, cardiovascular, care home admission, hepatic and renal outcomes), healthcare resource utilisation and total direct healthcare costs (using National Health Service tariffs and unit cost approaches) were calculated.

Of 18,116 patients diagnosed with AD, at baseline the mean age was 81 years, 62 percent were female, 65 percent were White, 16.5 percent Asian and 8.9 percent Black. At baseline, hypertension prevalence was 60.2 percent, chronic kidney disease 35.5 percent and Type 2 diabetes 22.4 percent. The highest incidence rates across these outcomes were 13.4 (95% confidence interval [CI]:12.2,14.7) per 1,000 person years for stroke, 7.5 (95% CI: 6.6, 8.5) for myocardial infarction, and 83.6 (95% CI: 80.1, 87.0) for care home admission. Median survival was 4.9 years from diagnosis. Their annual total direct healthcare cost was GBP4,547 per patient, of which 58 percent were from hospital admissions. The majority (75%) of healthcare contacts were from primary care. AD patients had an average length of stay of 11.5 days per inpatient admission, and spent on average one week per year as inpatients.

AD is associated with high direct healthcare costs, with patients’ annual costs ˜1.7 times that of the UK population. The majority of these costs are associated with inpatient hospital admissions.

The Corona Virus Disease 2019 (COVID-19) pandemic has impacted the functioning of health systems, imposing the need for adaptations. Elective surgeries also needed to adapt, and research has shown higher mortality in newly infected surgical patients after or during procedures. Thus, was recommended the suspension of elective surgeries during the pandemic. Early studies evaluating the effect of COVID-19 pandemic on bariatric surgery have reported a substantial reduction in procedures performed.

This retrospective study evaluated the impact of the suspension of bariatric surgeries for a Brazilian Health Maintenance Organization: UNIMED-BH, based on the analysis of data from before and during the pandemic of COVID-19.

There were 2,641 bariatric procedures conducted in 2019 with a 14.1 percent reduction in volume to 2,314 procedures in 2020. In 2021, there were 2,813 bariatric procedures and 1,700 procedures were observed from January to August 2022. Therefore, it appears that in 2022 the demand for bariatric procedures will be similar to the year 2019, which was before the COVID-19 pandemic.

From the analysis of the data, a decrease in bariatric surgical volume was evidenced during the year 2020 when compared to 2019. Post-pandemic, monitoring is necessary to assess whether the system was able to meet the demand for bariatric surgical procedures.

The REAL World Data (RWD) In Asia for Health Technology Assessment (HTA) guidance was developed by a regional working group to facilitate the increasing acceptance of real-world evidence (RWE) in Asia. We compared the consistency of REALISE against guidance from Japan and China.

Country-specific guidance for RWE/RWD use in pharmaceutical development were identified in May 2022 through governmental websites, with validation searches via Google. Sections from local guidance were mapped onto REALISE and categorized as “agree”, “mixed”, “disagree” or “missing” based on coverage and consistency.

Five Japanese and three Chinese documents were mapped. Most sections in Chinese guidance (77%) and 36 percent of sections in Japanese guidance were tagged “agree” or “mixed”, with general alignment on definitions and good practice considerations (study design, accountability); however, 63 percent of Japanese sections were tagged “missing” from REALISE. As local documents took the regulatory perspective, they lacked REALISE’s discussion of translating RWD to RWE for HTA/economic evaluations specifically. Local guidance focused on practicalities of RWD collection in local contexts, including descriptions of specific actions (e.g., evaluating RWD sources, ensuring data security) rather than overarching principles described in REALISE; specifically, Japanese guidance described how to access and analyze databases/registries, reflecting Japan’s landscape of robust sources of national healthcare data, but lacked discussion of other RWE study types, data sources and specialized analytical methods. While Chinese guidance had a broader view of RWD types (more similar to REALISE), they also contained discussions on pharmacovigilance and omics data, communication with regulatory bodies, and incorporation of RWE into the approval pathway for traditional Chinese medicines.

Despite differing purposes (with no RWE guidance from local HTA bodies), local and regional guidance align on general principles/good practice in generating/using RWE, providing common ground for increasing usage of RWE in HTA in Asia.

Medical device health technology assessment (HTA) in Australia is largely coordinated by the Medical Services Advisory Committee (MSAC). Its remit to improve the public’s health by deciding where to allocate public healthcare funding, can be enhanced by considering real world evidence (RWE). Existing data sources have limitations that can be addressed through RWE, including coverage of Australian patient populations who may not meet trial eligibility criteria, and long-term follow-up through data linkage and datasets. We partnered with a university to explore what information could be gained from an analysis of linked administrative patient data, with a view to addressing current evidence gaps and/or limitations. The findings can be used as a source of local data to define patient populations, estimate actual costs of care, and enable more comprehensive economic modeling to inform medical device HTA.

The University-developed New South Wales Cardiovascular Cohort dataset, comprising person-level longitudinal NSW administrative data for all patients admitted to hospital with a cardiovascular diagnosis from 2001 onwards, linked to national Medicare Benefits Schedule and Pharmaceutical Benefits Scheme claims data, was interrogated.

Working with RWE is resource intensive in terms of time and costs. The potential of these data was revealed as the research progressed. It was possible to continually refine the data analyzed and reported,as well as expand the data requested. Varied expertise is required to accurately analyze the administrative datasets, particularly clinical classification skills and expertise in methods for causal inference using observational data. Findings from this study will enable the refinement of information for MSAC submissions, including identifying the most relevant patient population and reporting comprehensive costs, beyond an admitted hospital setting. The data will enhance engagement with clinicians and refine messaging, for example regarding patient risk factors.

RWE enhances Australian HTA applications. Local data, extended periods of time and insights not apparent from a focus on admitted hospital episodes can be revealed. Data can be refined during the process for specificity and applicability.

In order to increase the value of its services and activities, the Agency for Health Quality and Assessment of Catalonia (AQuAS) has incorporated in its strategic plan the commitment to improve the processes, quality and people, while ensuring transparency, independence, rigor and efficiency following the guidelines of the European Foundation for Quality Management. We aim to present the standardization processes to improve the efficiency in elaborating health technology assessment (HTA) reports at AQuAS.

Process standardization has been developed in seven stages: (i) definition of scope, objectives and creation of working groups; (ii) mapping and analysis of all ongoing processes to determine whether they needed improvement or were already optimal; (iii) creation of new processes by evaluating the inclusion of automatic tools and their possible digitization; (iv) creation of the process map; (v) communication to the team for its implementation after training; (vi) dissemination on corporate website; (vii) monitoring and evaluation of their impact.

The creation of AQuAS’ HTA report development process map has involved 14 people over the past two years. After an initial two-day workshop, the team was organized in working groups of two to three people, with regular monitoring and the creation of a specific knowledge management unit led by the AQuAS’ evaluation manager. The process map was configured based on three axes. The strategic axis contains legal frameworks, ethical principles, good practices and methodological frameworks (29 specific for HTA reports). The key axis presents in an integrated way the process and methodology followed from the request of the HTA report to the final product’s dissemination. The support axis includes, among other, the management of 31 requests, information specialist processes (37 searches), training (51 courses) and 6 internal procedures with their corresponding 26 templates and 27 tools.

With a process reengineering approach, HTA reporting has become more efficient. We believe this approach can help other agencies improve their internal processes and subsequently improve team and customer satisfaction.