bluuebunny/crossref_metadata_2025 huggingface dataset:
PP75 Treatment Of Rheumatoid Arthritis In The Single Health System: Profile Of Medication Use And Expenses
Authors: Moreira Adson José , Prata Wallace Mateus , de Assis Acurcio Francisco , Guerra Augusto , Alvares-Teodoro Juliana
Published: January 2025

Introduction

Rheumatoid arthritis (RA) is a chronic inflammatory disease. Treatment aims to improve functional capacity and quality of life. Biological disease-modifying antirheumatic drugs (DMARDs) and oral inhibitor of Janus kinases (JAKs) used in treatment are high cost. The objective of this study was to analyze the profile of use and expenses of these medicines used in Brazil’s Unified Health System (SUS).

Methods

This is a retrospective cohort of users who received at least one biological DMARDs or oral inhibitor of Janus kinases (JAKs) registered in the Open Room on Health Intelligence—Sabeis/Datasus. The valuation of the 11 drugs standardized by the Clinical Protocols and Therapeutic Guidelines (PCDT) was carried out based on public purchases registered in the Price Bank in Health and an active search with the Ministry of Health, between 2017 and 2021. Expenses were calculated based on the number of High Complexity Procedure Authorizations (APACs) registered in the SIA/SUS database.

Results

Evaluation of 195,163 patient dispensing records was undertaken. Spending on medicines was BRL408,146,527 (USD79,012,759) in 2017, BRL237,615,290 (USD46,002,748) in 2018, BRL213,790,222 (USD41,350,815) in 2019, BRL195,626,575 (USD37,837,644) in 2020, and BRL140,096,142 (USD27,109,717) in 2021. It was found that in 2017, adalimumab (33%), etanercept (28%), and golimumab (14%) were the most used biological DMARDs. It is worth noting that in 2017, adalimumab and etanercept represented more than 50 percent of the costs of treating RA in the SUS. In 2021, it was found that 61 percent of total expenses were distributed between the drugs etanercept (23%), golimumab (18%), certolizumab (14%), adalimumab (11%), and tofacitinib (11%).

Conclusions

In the period from 2017 to 2021, changes can be observed in the profile of use and expenditure of biological DMARDs and oral inhibitor JAKs, resulting from the incorporation of new drugs and the entry of biosimilars in the Brazilian market. However, adalimumab, one of the first biological DMARDs incorporated, is the most consumed and least expensive medicine.
PP78 Data Extrapolation With Survival Curves: An Alternative Approach With Aggregated Data
Authors: Zimmermann Ivan Ricardo , Borges Solange
Published: January 2025

Introduction

Recently, there has been considerable emphasis on survival curves for data extrapolation, especially in the field of economic evaluation in oncology. Common methods for adjusting survival curves are complex and heavily reliant on individual patient data (IPD), which may not be feasible for health technology assessment (HTA). We propose an alternative method for survival curve extrapolation with direct adjustment to aggregated data.

Methods

Common parametric survival analysis models were tested: exponential, Weibull, log-normal, log-logistic, generalized gamma, and Gompertz. We had access to the IPD from a published randomized clinical trial (n=694) testing therapies (anastrozole and fulvestrant) for metastatic breast cancer with 10 years of follow-up on progression-free survival (PFS) and overall survival (OS) outcomes. After adjusting the original IPD, we sought to fit models to published aggregated data (Kaplan–Meier curves) using nonlinear regressions and optimization algorithms. Both methods were compared in terms of visual inspection and statistical fit quality (Akaike information criterion [AIC] and Bayesian information criterion [BIC]).

Results

Survival curves directly adjusted to aggregated data showed a visually similar profile compared to IPD adjustments. According to AIC/BIC values, Weibull and generalized gamma distributions best fit OS data, both in individualized and aggregated approaches. For PFS, log-logistic and log-normal curves were the best choices for the anastrozole arm, and for fulvestrant, the best choices were log-normal and generalized gamma for individualized data, and Gompertz and generalized gamma for the aggregated method. The proposed R language code proved to be reproducible and amenable to automation in future HTA applications.

Conclusions

Directly adjusting survival curves to aggregated data is a simple and useful alternative in situations where access to IPD is not feasible.
PP81 The Importance Of Networking To Produce Better Health Technology Assessment Evaluations: An HTA Guideline For Orthoses And Prostheses In Brazil
Authors: Martins Ketinlly Yasmyne , Branco Rodolfo Castelo , Galdino Katia , Medeiros Lauriston , Oliveira Eduardo Jorge V , de Souza Monica Vinhas
Published: January 2025

Introduction

Health technology assessment (HTA) studies focused on equipment have particularities. The evaluation of orthoses (ORT) and prostheses (PRT) is even more challenging and tends to present more difficulties when compared to technologies such as medicines. Furthermore, in Brazil there are different manufacturing and dispensing processes for these technologies throughout the country; this heterogeneity could also affect the sustainability of the system itself.

Objectives

(i) evaluate and disseminate the elements necessary to carry out adequate and sustainable assessments of non-implantable ORT and PRT and; (ii) produce a guideline on HTA assessment for non-implantable ORT and PRT.

Methods

Visits were made to ORT and PRT centers throughout Brazil to collect data on the production chain, dispensing, adaptation, and evaluation of the patient’s clinical results. At the same time, systematic literature reviews were carried out on two topics: (i) elements that must be considered in the manufacture, dispensation, and adaptation of non-implantable ORT and PRT; and (ii) HTA guidelines/examples focused on ORT and PRT.

Results

Eight of a total of 12 centers throughout different Brazilian regions were visited. A scoping review was carried out on body-sizing methods for manufacturing non-implantable ORT and PRT, and the differences and particularities of each method. Another systematic review on existing HTA guidelines and the HTA assessment of ORT and PRT is ongoing. The data of the three phases will be analyzed and translated in a Brazilian guideline of HTA for non-implantable ORT and PRT.

Conclusions

With the network of regional and national centers and the sum of different actors (such as government agencies, universities, and health centers), working together for common ground and the sustainability of the health system in complex areas such as HTA of ORT and PRT would be viable. Even with this effort, the work to be done and the challenges are significant.
PP82 Prevention And Intervention Software Applications For People At Risk Of Suicide: Effectiveness And Safety
Authors: Rivero-Santana Amado , Duarte-Díaz Andrea , Álvarez-Pérez Yolanda , Linertová Renata , Torres-Castaño Alezandra , González-Pacheco Himar , Guirado-Fuentes Carmen , Sacks Analía Abt , García Vanesa Ramos , Toledo-Chávarri Ana , Acosta-Artiles Francisco Javier , Serrano-Pérez Pedro , Carrion Carme , Guillén Marta , Perestelo-Pérez Lilisbeth
Published: January 2025

Introduction

Suicide poses a severe public health challenge worldwide, impacting individuals, families, work, and society. The multifaceted nature of suicide demands a complex approach involving psychological, biological, social, cultural, and environmental factors. Recognizing suicide’s status as the leading external cause of death in Spain, prevention increasingly incorporates technology, specifically mobile and software applications.

Methods

A systematic review of the effectiveness and safety of mobile and software applications was conducted (MEDLINE, Embase, CINAHL, and PsycINFO databases). Outcome variables included: suicide; suicidal behavior; suicidal intent; suicidal ideation/thinking; self-perceived suicide risk; using/seeking mental health services; associated mental symptoms; mental health-related quality of life; satisfaction of the user and the health professional; adverse events related to the app, as defined in the included studies. Studies that do not include suicidal behavior, intention, or ideation were excluded. Where available data allowed, a meta-analysis was conducted for each outcome variable.

Results

One systematic review and 13 randomized controlled trials (n=2,952) were analyzed. No significant differences were found in deaths by suicide or suicide attempts. At post-intervention, small but significant reductions were observed in suicidal ideation, hopelessness, depression, and worry, with anxiety reduction slightly above statistical significance. At follow-up (8 to 52 weeks), these variables also obtained significant results, except depression and suicidal ideation. Regarding safety, there was no significant difference in safety phone calls for participants with suicidal ideation.

Conclusions

The evidence on suicide prevention app effectiveness is of low quality, precluding conclusive findings. Attempt reduction is suggested at 21 percent, but the confidence interval includes a potential 60 percent increase. Evidence on suicide-related psychological variables (suicide ideation, depression, hopelessness, and anxiety) is of higher quality (low–moderate), but effects are small and clinically uncertain. Safety findings are uncertain, impacting risk/benefit balance.
PP86 Development Of A Tool To Assist In The Identification Of Study Designs For The Purposes Of Health Technology Assessment
Authors: di Ruffano Lavinia Ferrante , Reddish Katie , Bishop Emma , Watkins Deborah , Edwards Mary , McCool Rachael
Published: January 2025

Introduction

As the most internally rigorous design, randomized controlled trials (RCTs) are the gold standard for assessing the efficacy and safety profile of interventions. Increasingly, health technology assessment (HTA) considers evidence from non-randomized studies. Guidance recommends synthesizing different study designs separately due to their different inherent biases/limitations. But when authors or reviewers misclassify studies, this could affect which studies are included and therefore have an impact on review results.

Methods

We are conducting a methods project to (i) identify a clear study design classification system, (ii) explore whether its use produces consistent study design categorizations among reviewers, and (iii) iteratively improve the classification system. We performed a pragmatic web-based search for study design categorization tools and used the resulting schemas to develop a clear algorithm for use by reviewers of all levels of experience, specifically in reviews of treatment interventions. Next, we tested tool consistency and user experience by web-based survey in a small internal sample of reviewers, each independently using the system to categorize 18 published studies.

Results

A median of seven reviewers (range four to eight) categorized each study. Rater agreement using the chart varied widely, with 100 percent agreement on the designs of three studies (17%), and at least 75 percent of reviewers agreeing on one design for nine studies (50%). The most common agreement was reached on RCTs and non-randomized controlled trials. The most common sources of disagreement were between different types of cohort studies and between case series and controlled cohort studies, largely due to inconsistent reporting. We also identified several improvements: the wording of prompt questions, the ordering of designs, and the addition of new elements.

Conclusions

The classification system as initially designed led to too much variation in study design categorization to be useful. Consequently, we present a revised version that we now aim to evaluate in a larger sample of reviewers. Further research will also investigate whether using the tool would change the results of systematic reviews, using a small sample of published reviews.
PP89 Artificial Intelligence And Health Technology Assessment: Playing Catch-Up
Authors: England Clare , Washington Jenni
Published: January 2025

Introduction

The use of artificial intelligence (AI) in health care has the potential to improve clinical and patient outcomes and to reduce rising costs. There is an exponential increase in health technologies that use AI. We present a health technology assessment (HTA) case study demonstrating that the rapid rise in publications presents challenges for HTA bodies seeking to provide robust, timely assessments.

Methods

We conducted an HTA of AI-assisted endoscopy in the detection and characterization of lower gastrointestinal (GI) cancer. Searches were conducted up to October 2023. Unusually, the search targeted only the intervention: artificial intelligence and lower-GI-tract endoscopy (including colonoscopy, proctoscopy, etc.). The search strategy was peer reviewed. MEDLINE, Embase, KSR Evidence, CINAHL, Cochrane Library, and the INAHTA HTA database were searched, as well as ongoing trial registers and key websites. A date limit of 2010 onwards was applied, as Xbox Kinect launched in 2010 and was the first mainstream device used for healthcare imaging.

Results

Two network meta-analyses, 15 meta-analyses, one systematic review of meta-analyses, and three systematic reviews published since 2020 were identified. One review conducted searches to January 2020, identifying three randomized controlled trials (RCTs); a review that searched up to February 2023 identified 21 RCTs. There was substantial overlap regarding included primary studies, but not all reviews included the same outcomes. An additional seven RCTs were published in 2023. We also identified 12 cohort studies published between 2021 and 2023. We prioritized higher quality meta-analyses of clinical RCTs to include all outcomes of interest and updated the meta-analyses for primary outcomes.

Conclusions

This case study of an HTA of an AI-related technology demonstrates how rapidly the field is moving. It is necessary to use well targeted but not overly exclusive search strategies, limit by date, and prioritize inclusion of identified evidence according to quality and availability of outcomes. Time should be allowed to update existing meta-analyses.
PP97 Conduction System Pacing Implantation Through Electroanatomic Mapping-Guided Versus Fluoroscopy In Patients With Severe Bradyarrhythmias
Authors: Infante-Ventura Diego , León-salas Beatriz , Hernández-Yumar Aránzazu , Linertová Renata , García Javier García , López Raúl Quirós , Ramos Estefania Herrera , Trujillo-Martín Mar
Published: January 2025

Introduction

Electroanatomic mapping (EAM) has been shown to be an alternative procedure to fluoroscopy for the implantation of conduction system pacing (His-bundle pacing [HBP] and left bundle branch pacing [LBBP]) in patients with severe bradyarrhythmias, mainly those vulnerable to ionizing radiation. However, the evidence of its beneficial and harmful effects has not been assessed in a systematic review (SR).

Methods

An SR of the available scientific literature was conducted on the safety, effectiveness, and cost-effectiveness of implantation of the HBP and LBBP using EAM system versus fluoroscopy in patients with bradycardia with an indication for pacing therapy. Cochrane methodology and PRISMA statement for reporting were followed. A partial economic evaluation was carried out to compare the costs of both pacemaker implantation strategies from the perspective of the Spanish National Health System. A budget impact analysis was also conducted with a five-year horizon.

Results

Seven comparative observational studies (N=259) analyzing the use of EAM versus fluoroscopy were selected. Statistically significant differences were observed in total fluoroscopy time: −9.87 minutes (95% confidence interval [CI]: −14.20, −5.53; p<0.01; I2=95%; k=7; n=231); His-lead fluoroscopic time: −8.08 minutes (95% CI: −10.36, −5.81; p<0.01; I2=0%; k=2; n=50); and His-lead radiation dose: −17.21 mGy (95% CI: −24.08, −10.34; p<0.01; k=1; n=20). No differences in total radiation dose, procedural success, immediate procedure-related complications, electrode revision, or device infection were found. The use of EAM represents an increase of EUR1,397.81 (USD1,513.88) per patient and a net budget impact of EUR1.63 million (USD1.77 million).

Conclusions

No differences between EAM and fluoroscopy in terms of procedure success and safety were found. Therefore, EAM is a valuable alternative for patients who should not be exposed to ionizing radiation. The inclusion of EAM systems, for the indication under study, in routine clinical practice would mean an increase in costs for the Spanish National Health System.
PP104 Utility Of Chemiluminescence Detection Of Anti-Domain
1 β2 Glycoprotein I Antibodies In Antiphospholipid Syndrome
Authors: Pino-Sedeño Tasmania del , Hernández-Yumar Aránzazu , Valcárcel-Nazco Cristina , De Armas-Castellano Aythami , González-Hernández Yadira , Infante-Ventura Diego , Martín Sánchez Juan Ignacio , Trujillo-Martín Mar
Published: January 2025

Introduction

Patients with antiphospholipid syndrome (APS) present a significant risk of thrombotic events or morbidity during pregnancy, associated with the presence of persistently positive antiphospholipid antibodies (aPL). The risk stratification is crucial to adopt primary prophylaxis measures. With this objective, assays for the in vitro detection of anti-domain
1 β2 glycoprotein I (aD1 IgG) antibodies by chemiluminescence immunoassay (CLIA) have been developed.

Methods

We conducted a systematic review with meta-analyses on the effectiveness of incorporating aD1 IgG detection by CLIA for the identification of patients with APS at high risk of thrombosis or morbidity during pregnancy. A cost analysis was also conducted using a decision tree model from the perspective of the Spanish National Health System and a time horizon of three months. We ran extensive sensitivity analyses. Twelve diagnostic performance studies with a total of 3,570 patients were selected.

Results

The sensitivity and specificity of aD1 IgG added to the conventional aPL criteria for obstetric and thrombotic manifestations ranged from 15 to 16 percent, and from 94 to 97 percent, respectively. The sensitivity of the detection of aD1 IgG in isolation for thrombotic and obstetric manifestations was 29 and 48 percent, and specificity was 60 percent, respectively. The GRADE quality of evidence was very low due to the risk of bias and indirectness issues. The incorporation of the aD1 IgG detection involves a higher cost per patient than the usual clinical practice, with an incremental difference of EUR24.04 (USD26.01).

Conclusions

Despite its potential, the incorporation of the aD1 IgG detection by CLIA as an additional test to the determination of classical aPL for the identification of patients with APS at high thrombotic or obstetric risk may not be feasible due to the very low available evidence on diagnostic performance and increased costs.
PP105 Assessment Of Blood Biomarkers For The Diagnosis Of Alzheimer’s Disease: From Promise To Reality
Authors: Sanchez-Piedra Carlos , Cardaba-Arranz Mario , García-Carpintero Esther Elena , Imaz-Iglesia Inaki
Published: January 2025

Introduction

The objective of this assessment was to assess diagnostic effectiveness of phosphorylated isoforms of the tau protein (p-tau) in blood samples (plasma and/or serum) in Alzheimer’s disease diagnosis compared to clinical criteria, cerebrospinal fluid (CSF) biomarkers, positron emission tomography (PET), or post-mortem pathology imaging.

Methods

A systematic review of MEDLINE, Cochrane, and Embase was undertaken from 1 January 2000 to 1 September 2022. QUADAS-2 was used to assess the quality of the studies. The selection and quality assessment was carried out independently by two reviewers.

Results

A total of 1,811 references were identified after eliminating duplicates, selecting five: two studies evaluated p-tau181, two others evaluated p-tau217, and one evaluated both isoforms. The studies provided information on diagnostic accuracy using ROC curves. However, none of the studies provided information on sensitivity and/or specificity. The ROC curve values ranged from 83.3 percent to 99.4 percent for p-tau181 and 84 percent to 94 percent for p-tau217. Information was presented in an unclear and heterogeneous way, so our PICO (population, intervention, comparator, outcome) questions were left unanswered. The heterogeneity was due to factors such as the type of isoform, the laboratory platform, and the classification criteria considered.

Conclusions

Despite technology presenting as promising in several studies, the available evidence did not provide high-quality information about its diagnostic accuracy. Diagnostic technologies are often launched and marketed without enough high-quality evidence. The generation of evidence in diagnostic studies is often based on designs that do not provide adequate health technology assessment information.
PP107 Effectiveness And Safety Of Cytoreductive Surgery And Heated Intraperitoneal Chemotherapy For Pediatric Peritoneal Carcinomatosis: A Living Evidence Synthesis
Authors: Roteta Nora Ibargoyen , García Josune Domínguez , Jausoro-Zubiaga Ainhoa , Galnares-Cordero Lorea , Gutierrez-Ibarluzea Iñaki , Rojas Maria X. , Auladell-Rispau Ariadna , Verdugo-Paiva Francisca
Published: January 2025

Introduction

The evidence synthesis developed to inform decision-making on the use of cytoreductive surgery (CRS) and heated intraperitoneal chemotherapy (HIPEC) for pediatric peritoneal carcinomatosis showed that currently available evidence is of very low quality. As new evidence could arise within the following months, we adopted a rigorous living evidence synthesis (LES) approach to provide a timely update and favor decision-making based on actual evidence.

Methods

This LES started with a baseline synthesis about the effects of CRS and HIPEC on pediatric peritoneal carcinomatosis. On 31 August 2023, we set up the evidence monitoring for up to 12 months. Following the Living Evidence to Inform Health Decisions (LE-IHD) framework, we planned and developed the evidence monitoring, supported by technological enablers. We searched for ongoing studies in trial registries every three months. New eligible studies were assessed following a systematic and reproducible process to decide on their incorporation in the evidence summary. This process was periodically reviewed to determine the continuation/withdrawal of the living mode.

Results

The baseline synthesis identified one systematic review suggesting that CRS and HIPEC could increase overall survival in pediatric peritoneal carcinomatosis (very low-quality evidence), but no comparative data could be obtained against usual care. To date, the evidence monitoring has not identified new relevant studies on the impact of CRS and HIPEC in overall and disease-free survival, morbidity, or quality of life in pediatric peritoneal carcinomatosis. At the time of the conference, we will report on nine months of monitoring and regular updates including key messages on any changes in the evidence synthesis conclusions.

Conclusions

For HTA reports based on very low-quality evidence (uncertain results), the LE approach allows for timely updating of conclusions, adding value in decision-making. The LE-IHD framework facilitates HTA developers’ tasks for planning and conducting LE synthesis to inform health decisions.
PP108 A Systematic Review Of Reactogenicity And Safety Of Recombinant Zoster Vaccine For Prevention Of Herpes Zoster In Adults
Authors: Reece Emma , Jenkins Orla , Bergin Aoife , Reidy Ellen , Byrne David , Mc Loughlin Carol , Quigley Joan , Teljeur Conor , Harrington Patricia , Ryan Máirín
Published: January 2025

Introduction

Herpes zoster (HZ), also known as shingles, is characterized by a vesicular skin rash, often associated with acute pain and itching. The safety profile of the recombinant zoster vaccine (RZV) in adults aged 50 years and older and in adults aged 18 and older who are at increased risk of HZ was assessed in this systematic review.

Methods

A comprehensive electronic search was performed in Embase, MEDLINE, the Cochrane Library, and clinical trials registries. Searches were limited to the period from 2008 to July 2023. Article screening and data extraction were carried out by two independent reviewers. Risk of bias was assessed using the Cochrane revised Risk of Bias 2 (RoB2) tool for randomized controlled trials (RCTs). The Risk of Bias in Non-Randomized Studies of Interventions (ROBINS-I) tool was used to assess the quality of non-randomized studies. An adapted version of the Newcastle–Ottawa Scale was used for the appraisal of quality of non-comparative studies.

Results

Eighteen RCTs, four observational cohort studies, seven single-arm trials, and 11 single-arm observational studies were identified. Compared with placebo, solicited local (RZV: 74.1 to 84.0%; placebo: 7.9 to 11.9%) and systemic reactions (RZV: 53.0 to 66.1%; placebo: 6 to 11.4%) were more common in the vaccinated cohorts. Reactions were generally transient and mild to moderate in intensity. The most frequent reactions reported were pain at the reaction site, fatigue, and myalgia. The incidence of potential immune-mediated diseases (pIMDS), serious adverse events (SAEs), and fatalities was similar in vaccine and placebo groups. No SAEs, pIMDs, or deaths were reported as vaccine related.

Conclusions

The available data on RZV shows that while local and systemic adverse events are common with RZV, these are typically transient, and SAEs are uncommon in both the general population and those at increased risk of HZ.
PD01 Budget Impact Analysis Of Expanding Newborn Inherited Metabolic Diseases Screening In Shanghai, China
Authors: Xiao Dunming , Zhou Shanyan , Chen Yingyao
Published: January 2025

Introduction

China has a high incidence of birth defects. Tandem mass spectrometry (MS/MS) screening enables rapid detection of multiple inherited metabolic disorders and has been widely promoted globally. This study aimed to conduct a budget impact analysis of replacing phenylketonuria screening with MS/MS by calculating the financial impact of reimbursing the costs of MS/MS screening.

Methods

An Excel-based budget impact analysis model for MS/MS screening was developed. The number of newborns in Shanghai from 2024 to 2026 was estimated using the birth rate trend among the permanent population of Shanghai over the past decade. By integrating clinical screening data, along with the corresponding screening costs and diagnostic fees for the gold standard test, the financial impact of replacing phenylketonuria screening with MS/MS screening was calculated. The screening data for this study was extracted from a tertiary hospital in Shanghai. Demographic data were obtained from statistical websites, while cost data were derived from literature and a tertiary hospital in Shanghai.

Results

The fiscal expenditures for phenylketonuria screening were CNY1.75 million (USD0.25 million), CNY1.65 million (USD0.23 million), and CNY1.56 million (USD0.22 million) for 2024, 2025, and 2026, respectively. In contrast, the corresponding fiscal expenditures for MS/MS were CNY25.23 million (USD3.54 million), CNY23.78 million (USD3.33 million), and CNY22.41 million (USD3.14 million). The additional fiscal expenditure for MS/MS, compared with phenylketonuria screening, was CNY23.48 million (USD3.29 million), CNY22.13 million (USD3.10 million), and CNY20.85 million (USD2.92 million), showing a yearly decreasing trend.

Conclusions

The financial impact of MS/MS screening was controllable. It was recommended that the cost of MS/MS screening in Shanghai be covered by government funding. The promotion of newborn screening using MS/MS deserves priority consideration and publicity in Shanghai, China.
PD08 Clinical And Cost Effectiveness Of Selected Diabetes Medicines In Ghana - An Adaptive Health Technology Assessment
Authors: Asante Hannah , Owusu Richmond , Asare Brian , Amankwah Ivy , Reuda Sergio Torres , Kazibwe Joseph , Ruiz Francis
Published: January 2025

Introduction

There is a need to consider whether treatments included in essential medicine lists, standard treatment guidelines, and health benefits packages are cost effective, improve financial sustainability, and increase equitable access to health care. This assessment evaluated the cost effectiveness of selected antidiabetic medicines for inclusion on Ghana’s Essential Medicines List and updated standard treatment guidelines, and reimbursement by the National Health Insurance Authority.

Methods

This study was produced in line with the broad steps of the Ghana health technology assessment (HTA) process guideline using an adaptive HTA (aHTA) approach and following the process used by the National Cancer Grid of India. High quality HTA evidence was sourced from four HTA agencies based on the population (children aged two years or older and adults older than 18 years using antidiabetics), intervention, comparator, and outcomes framework using an adaptability checklist developed by the researchers. A price benchmarking analysis was conducted to generate context relevant evidence on medicine prices in terms of local value for money.

Results

The study found that all medicines evaluated (sitagliptin, vildagliptin, saxagliptin, insulin detemir, insulin degludec, insulin glargine, insulin glusiline, insulin aspart, and insulin lispro) were efficacious. The price benchmark analysis showed that insulin detemir, glargine, and degludec had higher price ratios than their comparators, and an annual drug cost per patient that was approximately two to four times higher. Insulin lispro, aspart, and glulisine had price ratios of 0.22 to 0.44 and an estimated annual cost of GHS1,894 to GHS3,552 (USD163.3 to USD306.2), which was two to five times higher per patient than the comparators. The cost of saxagliptin and vildagliptin were four and three times lower than those in the benchmark country (the UK).

Conclusions

The study revealed that all medicines included are efficacious and potentially cost effective. The price benchmark analysis showed that, except for gliclazide 80 mg, Ghana is paying less for antidiabetic medications than the UK. Cost effectiveness may not be a sufficient basis to include or exclude medicines for reimbursement because they have a potentially significant budget impact for the payer.
PD37 Development Of A Tool For Quality Assessment Of Health Economic Evaluations
Authors: Schneider Nayê Balzan , Migliavaca Celina Borges , Stein Cinara , Gräf Débora Dalmas , Escher Gabrielle Nunes , Decker Sérgio , Falavigna Maicon , Polanczyk Carisi
Published: January 2025

Introduction

Health economic analyses compare the necessary investments and health outcomes for two or more technologies, assisting in resource allocation. How these analyses are conducted directly affects the results obtained. Therefore, it is essential to consider their quality during decision-making. The aim of this study was to develop a domain-based tool for the critical assessment of cost-effectiveness and cost-utility studies.

Methods

We conducted a scoping review to identify tools available for the critical assessment of health economic analyses and extracted their recommendations. Based on the tools’ items and the discussions of a working group, we identified domains related to the methodological quality of health economic analyses for inclusion in the new tool. The items extracted during the scoping review were classified according to the previously defined domains and were used to identify complementary aspects that should be included in the new tool.

Results

We identified 21 tools, all of which were checklists containing seven to 80 items. The following four quality domains were established for the new tool: (i) applicability of the research question; (ii) model structure; (iii) model parameters; and (iv) precision of the results. Assessment of each domain was guided by signaling questions. The first domain assessed the applicability of the research question to the desired setting; the second evaluated whether the model adequately represents the complexity of the clinical condition; the third assessed the quality (certainty) of the key parameters used in the model; and the fourth evaluated the certainty of the incremental cost-effectiveness or cost-utility ratio.

Conclusions

The tool was developed to integrate critical aspects that affect the methodological quality of health economic analyses, which are often missing in other tools. The quality of reporting was not included as a domain because it is already covered by existing tools. A multidisciplinary panel with different key stakeholders is being organized to review and refine the first version of the tool.
PD38 Ensuring Study Validity To Inform Health Technology Assessments Globally
Authors: Devine (Emily) Beth , Whiting Penny , Mallett Sue , Wolff Robert , Savovic Jelena
Published: January 2025

Introduction

Assurance that supporting evidence is based on valid and unbiased assessments, evaluated using rigorously developed risk of bias (validity assessment) tools, is fundamental to good decision-making. Among those available, selecting and correctly using the best tool that is fit-for-purpose is challenging. Collaboration across the global evidence synthesis and health technology assessment (HTA) communities promotes best practices and harmonizes tool use across jurisdictions.

Methods

We have established the LATITUDES Network (https://www.latitudes-network.org/), a publicly available website library of validity assessment tools and resources to guide decision-makers in selecting and applying tools appropriate for particular contexts, including informing HTA reimbursement decisions, clinical guideline development, and stand-alone evidence synthesis projects. The internationally representative leadership team comprises five evidence synthesis experts who have been supported by a competitively awarded academic innovations grant. The 23-member advisory panel representing five continents provided expertise to finalizing criteria for tool inclusion, identifying key tools, and suggesting inclusion of additional tools. Formal launch took place at the 2023 Cochrane Colloquium.

Results

Officially launched in September 2023, the LATITUDES Network indexes validity assessment tools developed for healthcare studies in an online library. To date, 10 key tools are featured to help reviewers identify the optimal tool for their use. Nineteen additional tools have met all screening criteria and are also recommended. Information characterizing each tool (e.g., citation and training materials) is provided. Seven tools are currently under development. A mechanism for users to suggest new tools is provided. Additional tools and information on toolkits and online training materials, as well as links to courses and events, will be added over time.

Conclusions

LATITUDES aims to be the primary resource that provides key information to reviewers conducting validity assessments for evidence synthesis, clinical guideline development, and HTA decision-making. It is intended to increase the robustness of evidence synthesis by improving the process of validity assessment, helping scientists use tools more effectively and efficiently, promoting best practices, and harmonizing validity assessment across the globe.
PD40 Pilot Healthcare Programs - Bridging The Evidence Gap For Innovative Technologies
Authors: Gruszka Jarosław , Sejbuk-Rozbicka Katarzyna , Koperny Magdalena , Dzik Maciej , Pelczarska Aleksandra , Syta Joanna , Malinowska Kamila
Published: January 2025

Introduction

The scarcity of high quality evidence is a common constraint on the willingness to publicly fund innovative technologies. Our aim was to prepare a Methods and Process Guide to support the development of pilot healthcare programs in Poland. Such guides play a pivotal role in enhancing the quality of pilot programs and confidence in public funding decisions in health care.

Methods

We reviewed guidelines for pilot healthcare programs published by the World Health Organization (WHO) and other healthcare organizations and analyzed the pilot healthcare programs in Poland. The Ministry of Health in Poland and the general public will be invited to provide feedback on the Guide.

Results

Pilot programs serve as valuable testing grounds for healthcare solutions in low risk, small-scale clinical practice settings. A pilot program may be considered for interventions with proven safety and effectiveness, and when the intervention is complex, its implementation requires testing, or the intervention is considered high cost. Our Methods and Process Guide defines key elements of pilot healthcare programs, including objectives, starting criteria, conducting conditions, and monitoring rules. Public consultation on the Guide is underway.

Conclusions

The publicly available Methods and Process Guide should enhance the methodological rigor of pilot healthcare programs in Poland. Well-designed pilot programs are expected to provide high quality real-world data that will facilitate public funding decisions for innovative technologies.
PD42 Should Antibiotics Be Used To Treat Recurrent Otitis Media In Children? Updating A Recommendation
Authors: Ruiz-Ramos Alba , Sabalete-Moya Trinidad , Carmona-Hidalgo Beatriz , Rosario Lozano María Piedad , Fernández-Urrusuno Rocío , Blasco-Amaro Juan Antonio
Published: January 2025

Introduction

Acute otitis media (AOM) is one of the most common childhood infections. Recurrent AOM (rAOM) is defined as the presence of three or more AOM episodes in a period of six months. We describe the methodology used to update the recommendation of the 2018 Spanish National Antimicrobial Therapeutic Guide on the use of antibiotic treatments for rAOM in children.

Methods

We followed the GRADE-ADOLOPMENT approach to update the recommendation on antibiotic treatment for rAOM. Firstly, the research question was framed in a Population, Intervention, Comparison, and Outcome format. A comprehensive search strategy was developed, the results of which were screened according to the inclusion criteria. The selected studies were reviewed, and the quality of the evidence was assessed. Subsequently, an Evidence to Decision (EtD) framework was created and the new evidence was presented to the Guideline Development Group (GDG), which updated the recommendation on rAOM treatment in children.

Results

Among the 1,934 references identified by the database searches, only one guideline from the National Institute for Health and Care Excellence (NICE91, updated in 2022) met our inclusion criteria. This CPG included five individual studies comparing antibiotic treatments for rAOM. None of the studies demonstrated a significant advantage for any treatment. The overall quality of the evidence for these comparisons was considered low. A GRADE EtD framework was elaborated using the NICE91 recommendations but contextualized to the Spanish National Health System. Based on the evidence, the GDG did not modify the current recommendation provided in 2018.

Conclusions

The overall quality of the available evidence regarding antibiotic use for rAOM in children was considered low. Further research is therefore needed to resolve the controversy and increase confidence in the appropriateness of using antibiotics in the treatment of rAOM, thereby improving the quality of life of children with this condition.
PD52 Evidence Review Of Universal Ultrasound Screening For Developmental Dysplasia Of The Hip In Infants
Authors: Maher Arielle , Jordan Karen , Finnegan Eanan , Spillane Susan , Neill Michelle O’ , Ryan Máirín
Published: January 2025

Introduction

Developmental dysplasia of the hip (DDH) is a congenital disease in which there is abnormal development of the hip in infancy. Ultrasound screening has the potential to enable earlier identification and diagnosis of DDH, facilitating earlier and less invasive treatment. Ultrasound screening programs can be selective or universal, but the optimal method is unclear.

Methods

The aim of this review was to examine the comparative effectiveness of universal and selective ultrasound screening for DDH in infants. The domains of the Health Technology Assessment Core Model® selected for assessment were consistent with a rapid relative effectiveness assessment approach (i.e., focusing on the clinical benefit of the intervention) and included the following: (i) the health problem; (ii) a description of the technology; and (iii) clinical effectiveness and safety outcomes. An expert advisory group comprising nominated representatives from key stakeholder groups was convened for the purposes of quality assurance and to assist in interpreting the evidence.

Results

DDH severity can range from mild dysplasia to complete dislocation, with incidence varying internationally. Ultrasound screening can result in unnecessary treatment given the potential for spontaneous correction of hip instability. Furthermore, treatment may give rise to complications. Appropriate governance of a screening program and associated training may reduce the risk of unnecessary treatment. Limited high quality evidence from four studies was identified. This evidence suggested that increased rates of non-surgical intervention were associated with universal ultrasound screening, compared with selective screening, without a corresponding reduction in the incidence of late DDH or requirement for surgical intervention.

Conclusions

The relative benefit of universal ultrasound screening, compared with selective screening, remains unclear. Screening all infants has the potential to lead to unnecessary treatment, with the risk of clinically significant consequences. Consideration could be given to implementing a selective ultrasound screening program, with appropriate governance, end-to-end care, quality assurance, and outcome monitoring.
PD54 Impact Of Experiencing Vaso-Occlusive Crisis In Patients With Sickle Cell Disease: Systematic Review And Meta-Analysis Of Prognostic Studies
Authors: Ascef Bruna , Marques Mariana Oliveira , Kashiura Diego , Bertinato Juliano , De Oliveira Haliton Alves
Published: January 2025

Introduction

Sickle cell disease (SCD) is characterized by recurrent painful ischemic vaso-occlusive episodes (VOEs). Acute episodes of pain, often termed sickle cell pain crises or vaso-occlusive crises (VOCs), are one of the most common and debilitating manifestations of SCD. Here we present a systematic review of 31 trials of 202,758 patients with SCD.

Methods

This study followed the guidelines of Riley et al. for conducting a systematic review and meta-analysis of prognostic factor studies and was reported according to PRISMA guidelines. Literature searches were conducted in the PubMed, Embase, and LILACS databases. The websites of the Annual Academy for Sickle Cell and Thalassemia Conferences, the European Hematology Association, and the American Society of Hematology were also searched. Additionally, manual searches were conducted in Google Scholar, Epistemonikos, and the reference lists of included studies. The searches were performed on 22 March 2023.

Results

In total, 31 studies were included in this systematic review. There was considerable heterogeneity in the definition of prognostic factors of interest across the included studies. Findings revealed a link between VOCs and reduced health-related quality of life (HRQoL), severe pain, and a high rate of hospitalization. Although VOCs were related to an increased mortality risk, the mortality rate remained relatively low, with acute chest syndrome being a common cause of death. Despite the study heterogeneity, consistent evidence highlighted the impact of VOCs on SCD-related hospitalizations (pooled hospitalization rate due to VOEs of 47%, 95% confidence Interval: 33, 61; 16 studies, 139,752 participants).

Conclusions

This study suggests that VOCs reduce HRQoL, cause severe pain, and lead to high rates of hospitalization in patients with SCD. Furthermore, VOEs were related to an increased mortality risk. Future research should prioritize more well-designed comparative prospective studies and standardization of the definition of VOCs. A better understanding of VOCs as a prognostic factor could enhance HRQoL, alleviate healthcare system burden, and inform more effective interventions for patients with SCD.
PD58 The Repellent Effects Of Cymbopogon Nardus On Aedes Aegypti Mosquitoes: A Rapid Review
Authors: Rocha Medeiros Ana Célia , Galdino Allancleriston Alves , Gurjão Edmar Candeia , Camargo Erika Barbosa
Published: January 2025

Introduction

Arboviruses transmitted by the A. aegypti mosquito are a public health problem in Brazil. Citronella, known for its repellent properties, has been suggested as a possible sustainable and natural preventive measure against the arbovirus triad. This study aimed to carry out a rapid review of the efficacy and safety of the repellent properties of citronella for the A. aegypti mosquito.

Methods

The rapid review followed methods proposed by the Joanna Briggs Institute. Searches were conducted in the following literature databases: PubMed, LILACS, Embase, Scopus, Web of Science, and the Cochrane Library. Quality assessment was carried out using the AMSTAR-2 tool. The review aimed to determine the efficacy and safety of citronella (C. nardus) as a repellent for the A. aegypti mosquito, compared with usual methods.

Results

Citronella repels A. aegypti mosquitoes for between 12 and 480 minutes, depending on the concentration and formulation of the product. Considering its protection time, reapplying the product doubles the protective effect. Adding vanillin to the formula reduces the product’s volatility. Citronella is not absorbed through the skin like DEET products, making it less toxic.

Conclusions

Citronella-based products can be used as a complementary measure of protection against arboviruses. Additional investigations are needed on the percentage of essential oil present in homemade formulations. Studies addressing the safety of citronella are imperative for its use in the public health system. Controlled studies evaluating its degree of repellency are also needed.