bluuebunny/crossref_metadata_2025 huggingface dataset:
PP29 Social Preferences In Health Technology Assessments For Rare Diseases: A Systematic Literature Review Of New Analytic Approaches
Authors: Vasquez Paola , Hall Lisa , Merlo Gregory
Published: January 2025

Introduction

Rare diseases (RD) can be severe and can dramatically reduce life expectancy and quality of life. RD therapies, mostly orphan drugs (OD), fail to meet the standard criteria for public reimbursement due to uncertainty in cost-effectiveness estimates limiting healthcare access. To address this issue, experts have suggested the integration of social preferences into health technology assessments (HTAs) by implementing different methods.

Methods

This systematic literature review, performed in 2021, aimed to explore worldwide experiences of social preferences integration into HTAs for RD and OD through the implementation of multiple-criteria decision analysis (MCDA), discrete choice experiments (DCE), and person trade-off (PTO), among other methods. A systematic search of the literature was conducted using PubMed, Cochrane, Embase, and Scopus databases. The PRISMA approach was used for the review phases. Finally, the Promoting Action on Research Implementation in Health Services (PARIHS) framework was used to discuss the implementation of these instruments in the RD context.

Results

Thirty-three articles met the inclusion criteria. The studies measured social preferences for RD and OD as part of HTA using MCDA (n=17), DCE (n=8), and PTO (n=4), among other methods (n=4). These found that patients and clinicians do not prioritize funding based on rarity. The public is willing to allocate funds only if OD demonstrates effectiveness and improves the quality of life (QoL), considering as relevant factors disease severity, unmet needs, and QoL. Conversely, HTA agency experts preferred their current approach and placed more weight on cost-effectiveness and evidence quality even though they expressed concern about the fairness of the drug review process.

Conclusions

MCDA, PTO, and DCE, among others, are helpful and transparent methods for assessing social preferences in HTAs for RD and OD. However, their methodological limitations, such as arbitrary criteria selection, subjective scoring methods, framing effects, weighting adaptation, and value measurement models, could be hurdles to implementation. Further research is needed to tailor these methods’ applicability and impact in different social contexts.
PP30 The Health System Impact Of Returning Rare Disease Variants As Secondary Findings From Genomic Sequencing: A Population-Based Model
Authors: Dragojlovic Nick , Kopac Nicola , Llorian Elisabet Rodriguez , Lehman Anna , Lynd Larry D.
Published: January 2025

Introduction

The American College of Medical Genetics and Genomics (ACMG) recommends that pathogenic variants linked to 37 genetic diseases be disclosed as secondary findings (SFs) to patients undergoing genome-wide sequencing (GWS), including rare diseases (RDs) treated with enzyme replacement or gene silencing/replacement therapies. We estimated the potential budget impact of treating these RD patients in the US and Canada.

Methods

A population-based model was used to estimate the number of patients that would be identified in a given jurisdiction for the 11 diseases on the ACMG’s SFv3.2 list classified as “inborn errors of metabolism” or “miscellaneous phenotypes.” Genetic and clinically diagnosed prevalence was estimated for each gene-phenotype pair based on the scientific literature. Demographic and GWS utilization data were obtained from government websites and payer reports. Drug costs were obtained from the literature and payer websites and reported in 2023 US dollars. A range of one-way sensitivity and scenario analyses were conducted, and province- and state-level estimates were also generated.

Results

Based on an estimated current annual GWS utilization rate of 77 per million in the US, 237 RD SFs would be identified annually (0.9% of 25,663 patients tested), out of which 107 (45%) would be variants linked to hereditary transthyretin amyloidosis (hATTR). In Canada (GWS utilization rate of 169 per million), 45 patients would be identified per year, of which 11 (24%) would be hATTR. Treating 50 percent of hATTR SF patients with patisiran would cost approximately USD26.0 million in the USA and USD2.8 million in Canada annually. In contrast, no cases of RPE65-related retinopathy would be detected at current utilization rates.

Conclusions

The addition of hATTR (with an estimated genetic prevalence of one in 240 in the US) to the ACMG SF list may result in a significant number of additional cases diagnosed per year. While there is an ongoing debate about initiating presymptomatic treatment, reimbursement requests for high-cost drugs like patisiran are likely to increase if GWS utilization continues to grow.
PP39 Co-Creation Of A European Digital Health Technology Assessment Framework: The EDiHTA EU-Funded Project
Authors: Tsiasiotis Emmanouil , Di Bidino Rossella , Sacchini Dario , Cicchetti Americo
Published: January 2025

Introduction

The widespread adoption of digital health technologies (DHTs) brings new methodological challenges to health technology assessment (HTA). Different tools, guidelines, and methods have been defined by different stakeholders at national/international levels. In this scenario, the real thread for HTA is the lack of coordination and harmonization in the efforts to finalize the definition of an HTA framework for DHTs.

Methods

The European Digital Health Technology Assessment (EDiHTA) project aims to provide the first framework co-created by all stakeholders along the value chain for DHTs. The consortium is comprised of 15 partners from nine countries (Italy, the Netherlands, Denmark, Spain, Germany, Norway, UK, Belgium, and Poland). Stakeholders included are HTA agencies, regulators, hospitals, patient/citizen representatives, academics/universities, notified bodies, and developers. The project covers all types of DHTs and Technology Readiness Levels (TRLs). A multistakeholder, multidomain, and modular approach is adopted with the final goal to provide a validated, ready-to-use digital HTA framework for DHTs for the European ecosystem.

Results

The EDiHTA is articulated in eight working packages and covers four years. Starting from the mapping of the current scenario, after the definition of a common taxonomy, EDiHTA will develop a DHT-dedicated HTA framework. A common digital platform to support the assessment will be developed. Both the HTA framework and platform will be tested, refined, and validated through a piloting process involving disparate DHTs (mApp, telemedicine, artificial intelligence, and others) with different TRLs, available in different countries, with the participation of all stakeholders. As a consequence, evidence requirements will be defined and made more transparent; this will support health technology.

Conclusions

The EDiHTA project will deliver the first European digital HTA framework able to support and inform decision-making (at the macro [policy], meso [management providers], and micro [clinicians] level) towards faster and safer access to DHTs of added value, taking European regulations and legal requirements into account and assessing all HTA domains.
PP63 Perspectives Of Physical And Organic Disability Organizations On Health Technology Assessment Processes
Authors: Lorenzo Laura Quintas , Suárez Isabel González , Gómez-Salgado Patricia , Faraldo Vallés María José
Published: January 2025

Introduction

Effective participation of individuals with disabilities in health technology assessment (HTA) processes is paramount. Aware of the reality of people with physical and organic disabilities, COGAMI (a not-for-profit umbrella organization of disability associations) conducted an internal study to gather perspectives on the participation of people with disabilities in HTA processes.

Methods

An ad hoc questionnaire of four open-ended questions was designed and distributed via email to COGAMI’s socio-health commission, representing 23 entities and 4,000 people in Galicia. A thematic analysis of the responses obtained was carried out.

Results

Consensus underscores the fundamental role of individuals with disabilities and their representative organizations in HTA processes, though currently, only those with greater resources actively participate. The participants found that insufficient information reaching patient organizations hinders participation (e.g., lack of awareness in proposal submission), complicating their involvement. Additional challenges include accessibility and the digital divide. Proposed solutions involve enhancing communication channels and information accessibility, establishing collaborative frameworks nationally, and actively considering the disability condition to ensure a fair and equitable implementation.

Conclusions

This study suggests the need for concrete actions to enhance the participation of individuals with disabilities in HTA processes. Recommendations include improving communication channels, capacity building, and recognizing disability as a key element in HTA.
PP67 From Insight To Foresight: Singapore’s Experience In Horizon Scanning Of Medical Technologies
Authors: Ng Zhen Long , Ju Hong , Ng Jaryl , Soon Swee Sung , Ng Kwong
Published: January 2025

Introduction

The Agency for Care Effectiveness (ACE) in Singapore has established a horizon scanning (HS) system to provide advance notice of new and emerging medical technologies (MedTechs) to inform service planning since 2020. To remain fit for purpose, HS needs to evolve over time to better serve its stakeholders. This abstract aims to share ACE’s early HS experience and future opportunities.

Methods

To gather insights into ACE’s HS successes and potential gaps, a mixed-methods approach was used. A review of key HS activities was conducted, including different HS outputs produced, stakeholder engagements conducted, and dissemination strategies employed. Feedback from key stakeholders was gathered to understand the potential utility of our outputs. Lastly, potential gaps and opportunities were identified to inform future initiatives.

Results

As of July 2023, 20 HS reports were developed, comprising three high-level overviews and 17 in-depth briefs. Output of briefs increased five-fold from 2021 (n=2) to 2023 (n=10). Efforts to drive impact included industry submission of pipeline MedTechs to supplement identification sources; report dissemination to over 100 policymakers and healthcare professionals for early service planning; and participation in network activities and conferences to share knowledge. Initial stakeholder feedback was positive, citing the reports’ relevance for clinical practice. The review recommended diversifying HS outputs to include environmental scan and trends reports, strengthening patient involvement and formal impact assessment through stakeholder surveys.

Conclusions

ACE’s HS has made significant progress in achieving its intended objectives to enable service planning for new and emerging MedTechs. However, this remains an iterative process with new opportunities for growth. Future initiatives were identified, such as impact assessment, enhancing stakeholder engagement to include patients, and broadening outputs to meet stakeholder demands.
PP87 A Descriptive Study Of The Use Of Data Visualization In Full Health Technology Assessment Reports: 2021 To 2023
Authors: Ramos Estefania Herrera , Rodríguez-López Rocío , Isabel-Gómez Rebeca , Rosario Lozano María Piedad , Casal-Acción Beatriz , Peiró Roland Pastells , Galnares-Cordero Lorea
Published: January 2025

Introduction

Data visualization is a powerful communication tool to facilitate the understanding of a complex process or data. Data visualization has been used in health technology assessment (HTA) reports for a long period (e.g., PRISMA flow diagrams, critical assessments, or GRADE). This study aims to investigate the number and manners in which HTA reports have been using descriptive data visualization for the years 2021 to 2023.

Methods

The international HTA database was used to identify and download the HTA reports from the years 2021 to 2023. We applied the database limits: full HTA and completed reports. The records were imported into a Microsoft Excel spreadsheet and screened by eight independent researchers applying the inclusion criteria: full HTA (according to methodological definition), access to the full text, and use of data visualization with a descriptive purpose (we excluded PRISMA flow diagrams, forest plots, and others). The data were observed with the software Power BI. Our analysis included variables such as agency name, country, section, type of visualization, and software.

Results

From the international HTA database, 1,128 records were exported: 89 records were directly excluded from this set as they were tagged as ongoing or other types of reports in the database; 1,039 records were screened. Around 30 percent of records were included for the data visualization screening criteria after fulfilling our inclusion criteria (full HTA and available full text). Finally, 12 percent of the reports included data visualizations for a descriptive purpose of their results or conclusions. The countries with a higher number of records included in this analysis were Germany (28%), Canada (18%), the UK (16%), and Spain (11%).

Conclusions

In our sample, we observed that data visualization is not widely used so far to describe outcomes and/or conclusions from HTA reports. An additional finding was the number of records tagged as full HTA in the international HTA database that were excluded as non-full HTA from our study.
PD02 Budget Impact Analysis: A Challenge To Incorporating Medications For Ultrarare Diseases In The Brazilian Healthcare System
Authors: Santos Marisa , da Silva Pereira Curado Daniel , Machado-Rugolo Juliana , Komoda Denis Satoshi , de Paula De Sordi Mônica Aparecida , Fonseca Alan Francisco , Coneglian de Almeida Juliana Tereza , Lima Silvana Molina , Theresa Weber Silke Anna , Tarride Jean-Eric , Thabane Lehana , de Almeida Cardoso Marilia Mastrocolla , Modelli de Andrade Luis Gustavo
Published: January 2025

Introduction

The decision-making process for incorporating technologies for ultrarare diseases (URD) has been a challenge for health technology assessment agencies worldwide. These challenges have been presented in debates about the budget impact of incorporating technologies for URD. This is an important issue because there are other dimensions of the economic and social impact of URD that require consideration.

Methods

Data were extracted from National Committee for Health Technology Incorporation (CONITEC) reports (2012 to 2022) on technologies for the treatment of URD in Brazil. Diseases were classified using an epidemiological criterion or Orphanet consultation (prevalence ≤1 per 50,000 inhabitants). Variables included eligible patient count, population estimation method, incremental impact values for one and five years, and diffusion rate in the first and fifth year. Univariate logistic regression was used to adjust the relationship between the budget impact analysis and the final recommendation, considering factors associated with incorporation in univariate regression and p-values less than 0.10 in a multivariate regression.

Results

Among 53 reports, 48 percent exclusively employed the epidemiological approach for incremental impact assessment population estimation, rising to 69.5 percent when combined with measured demand. Population data were nearly evenly sourced from national and international platforms, with the UK, the USA, and multicenter studies being the most cited internationally. Notable differences were found between favorable and unfavorable CONITEC recommendations, with lower values being associated with incorporation. Market share diffusion rates favored the option of 100 percent diffusion in both the first year and the cumulative five years. The analysis highlighted the influence of demand characteristics and technology type on the budget impact value over one and five years.

Conclusions

The study found that budget impact data significantly influenced the final recommendation for technology incorporation, indicating a criterion favoring technologies with a lower budget impact. However, requester characteristics and technology type also played a role in the decision-making process, suggesting that additional factors influence recommendations.
PD03 Exploring Expenditure On State Subsidized Medicines in Ireland Between 2018 And 2022: Special Focus On Cancer Drug Expenditure
Authors: Walsh Caroline , Trela-Larsen Lea , Adams Roisin
Published: January 2025

Introduction

The Department of Health in Ireland published a review of expenditure on state subsidized medicines in August 2021. Detailed analysis indicated exponential growth of expenditure on cancer drugs administered in the community. However, expenditure by drug group across all state subsidized schemes, including hospitals, was not explored.

Methods

Using national reimbursement claims data, total medicines expenditure on community drug schemes (CDS) was analyzed annually for the years 2018 to 2022. The total drug expenditure stratified by anatomical therapeutic class (ATC) code was calculated. Expenditure on cancer drugs (ATC code L) between 2018 and 2022, including hospital data, was further explored. Cancer drugs with the highest expenditure were identified, and trends in their expenditure were analyzed. Dates of European regulatory approval, completion of health-technology assessments by the National Centre for Pharmacoeconomics in Ireland, and reimbursement by the Health Service Executive for the identified cancer drugs were collected.

Results

The total expenditure on drugs on CDS rose from EUR1.74 billion (EUR1.61 billion excluding value-added tax [VAT]) in 2018 to EUR2.2 billion (EUR2.03 billion excluding VAT) in 2022. Expenditure on antineoplastic and immunomodulatory agents (ATC code L) rose from 34 percent in 2018 to 38 percent in 2022. The cancer drugs with the highest cumulative expenditure on hospital schemes were pembrolizumab (EUR98.41 million excluding VAT), nivolumab (EUR75.15 million), daratumumab (EUR54.05 million), and trastuzumab (EUR41.48 million). All the aforementioned drugs demonstrated year-on-year increases in annual expenditure apart from nivolumab. Expenditure on pembrolizumab increased by 48 percent in 2022, compared with 2021.

Conclusions

Expenditure on state subsidized medicines is increasing annually, although confidential discounts may reduce budget impact. Increasing expenditure may be attributed to the expansion of existing indications and increasing patient volume. Expenditure on monoclonal antibodies is substantially larger than expenditure on other drugs. Pembrolizumab was reimbursed for six additional indications in 2021, contributing to the sharp increase in expenditure between 2021 and 2022.
PD06 Automated Systems For Hospital Inpatient Safety: A Cost-Utility Analysis
Authors: Holmes Emily , Williams Huw Lloyd , Hughes Dyfrig , Naujokat Elke , Duller Bernd , Subbe Christian
Published: January 2025

Introduction

Deployment of an electronic automated advisory vital signs monitoring and notification system to signal clinical deterioration is associated with significant improvement in clinical outcomes. This study aimed to estimate the incremental cost per quality-adjusted life-year (QALY) gained with an electronic automated advisory notification system, compared with standard care.

Methods

A decision analytic model was developed to estimate the cost effectiveness of an electronic automated advisory notification system, compared with standard care, in adults admitted to a district general hospital. Analyses considered the following: (i) cost effectiveness (cost/event avoided) based on a before-and-after study (n=3,787) that recorded rates of acute myocardial infarction, pulmonary embolism, acute pulmonary edema, respiratory failure, stroke, severe sepsis, acute renal failure, cardiopulmonary arrest, admission to the intensive care unit, and death; and (ii) the cost utility (cost per QALY) over a lifetime horizon extrapolated using published data. The analysis was conducted from the perspective of the National Health Services (NHS) in the UK.

Results

The automated notification system was more effective (2.7 fewer events per 100 patients) and provided cost savings of −GBP12.17 [−EUR14.07] per patient admission (95% CI: −GBP182.07 [−EUR211.20], GBP154.80 [EUR179.57]). The automated notification system was dominant over a lifetime horizon, demonstrating a positive incremental QALY gain (0.0287 QALYs, equivalent to approximately 10 days of perfect health) and a cost saving of −GBP55.35 (−EUR64.02). At a threshold of GBP20,000 per QALY (EUR23,126), the probability of automated monitoring being cost effective in the NHS was 0.81. The increased use of cableless sensors may reduce cost-savings, but the intervention remained cost effective at 100 percent usage (incremental cost-effectiveness ratio GBP3,107 per QALY [EUR3,594 per QALY]).

Conclusions

An automated notification system for adult patients admitted to general wards appears to be a cost-effective strategy in the NHS. The analysis suggests that adopting this technology could be good use of scarce resources. The impact of automated monitoring solutions on staffing warrants further exploration and may show additional value in adopting such technology.
PD07 Can Commencing Colorectal Cancer Screening At Age 45 Years Be A Good Investment? An Individual-Level Simulation Analysis In Germany
Authors: Lwin Min Wai , Cheng Chih-Yuan , Calderazzo Silvia , Schramm Christoph , Schlander Michael
Published: January 2025

Introduction

The effectiveness and cost saving advantages of colorectal cancer (CRC) screening have gained widespread scientific consensus. However, the rising incidence of early-onset CRC has challenged Germany’s current screening program, which focuses on individuals aged 50 years or older. This study evaluated the potential cost effectiveness of initiating CRC screening in Germany at the age of 45 years.

Methods

The cost-effectiveness analysis utilized a validated discrete-event-simulation model, DECAS, which incorporates both adenomatous and serrated polyp pathways in CRC development. This model has been validated using German CRC epidemiological data and simulates the effects of screening interventions. It was used to compare four new CRC screening strategies starting at age 45 years (10-yearly colonoscopy, annual or biennial fecal immunochemical testing [FIT], or both) with the current screening strategy starting at age 50 years. The simulation, assuming perfect adherence, included a cohort of 100,000 individuals with an average CRC risk from age 20 to 90 years or death, applying a three percent discount with costs in 2023 Euros.

Results

The model outcomes included quality-adjusted life-years (QALYs) gained and total incremental costs, considering both CRC treatment and screening costs. Initiating 10-yearly colonoscopy only or FIT plus colonoscopy strategies at age 45 years yielded incremental gains of seven to 28 QALYs, with incremental costs of EUR28,360 to EUR71,759 per 1,000 individuals, compared with the current strategy. The incremental cost-effectiveness ratios varied between EUR1,029 and EUR9,763 per QALY gained. The FIT-only strategy was dominated by the current screening strategy. These findings remained consistent throughout the probabilistic sensitivity analyses.

Conclusions

The cost-effectiveness findings support initiating CRC screening at age 45 years with either colonoscopy alone or colonoscopy plus FIT, demonstrating substantial gains in QALYs and a modest increase in costs. Our findings emphasize the importance of implementing CRC screening five years earlier than the current practice to achieve more significant health and economic benefits.
PD12 Cost Effectiveness Analysis Of Nivolumab Plus Ipilimumab For First-Line Treatment Of Advanced Non-Small Cell Lung Cancer In China
Authors: Xiao Dunming , Chen Yingyao , Yang Yi
Published: January 2025

Introduction

Non-small cell lung cancer (NSCLC) is the most prevalent malignant tumor in China. This study aimed to compare the cost effectiveness of combined nivolumab and ipilimumab with chemotherapy as a first-line treatment for advanced NSCLC. The findings will contribute to the economic evidence for making clinical and health policy decisions.

Methods

Taking a healthcare system perspective, this study used a partitioned survival analysis model to simulate the disease trajectory of advanced NSCLC during first-line treatment over a model cycle of three weeks. The simulation extended over a span of 12 years. A five percent discount was incorporated for both costs and health outcomes. Published clinical efficacy and cost data were extracted from the CheckMate 9LA study (NCT03215706) and drug pricing information was gathered from the YaoZhi website. Utility values were derived from 13 tertiary hospitals in five provinces of China. Base case and sensitivity analyses were also conducted.

Results

The combination of nivolumab and ipilimumab resulted in a lifetime cost of CNY850,068 (USD119,127) and 1.796 quality-adjusted life-years (QALYs), whereas chemotherapy incurred a lifetime cost of CNY276,313 (USD38,722) and a gain of 1.206 QALYs. The incremental cost-effectiveness ratio (ICER) for combination therapy was CNY971,955 (USD136,208) per QALY gained, which was more than three times the average gross domestic product per capita in China (CNY85,698 [USD12,010] in 2022) and indicated that the therapy was not cost effective. Probabilistic sensitivity analysis indicated that the likelihood of nivolumab plus ipilimumab being cost effective, compared with chemotherapy, was 0.02.

Conclusions

Nivolumab plus ipilimumab demonstrated enhanced health outcomes for patients with advanced NSCLC, compared with standard chemotherapy, but the ICER exceeded the acceptable threshold, suggesting that the treatment is not cost effective.
PD13 Cost Effectiveness Of Imipenem-Cilastatin-Relebactam Compared With Colistina-Imipenem For Treating Confirmed Carbapenem Non-Susceptible Gram-Negative Bacterial Infections
Authors: Paoletti Martina , Marcellusi Andrea , Yang Joe , Mennini Francesco Saverio
Published: January 2025

Introduction

Antimicrobial resistance is an important public health problem with a strong epidemiological, economic, and social impact. The objective of this analysis was to compare the cost effectiveness of imipenem-cilastatin-relebactam (IMI/CIL/REL) therapy with colistin-imipenem for the treatment of hospitalized patients with Gram-negative bacterial infection caused by imipenem-resistant pathogens.

Methods

The population comprised patients with hospital-acquired bacterial pneumonia or ventilator-associated bacterial pneumonia that was complicated with an intra-abdominal or urinary tract infection caused by carbapenem-resistant Gram-negative pathogens. The model started with a short-term decision tree describing possible treatment routes and outcomes for patients during hospitalization. After treatment, patients were classified as cured, not cured, or dead. Patients who had not responded to the initial treatment received another line of therapy. Successfully treated patients were entered into the long-term Markov model, which captured follow-up costs and health-related quality of life over their lifetimes.

Results

The analysis was conducted on a hypothetical cohort of 1,000 patients and demonstrated that IMI/CIL/REL therapy was advantageous in terms of diagnosis and treatment in the short term as well as cost effectiveness. In fact, IMI/CIL/REL therapy was dominant, compared with colistin-imipenem, from the National Health System and the societal perspective, providing an average saving of EUR2,800.15 and EUR3,174.63, respectively, and gains of 4.76 years of life and 4.12 quality-adjusted life-years per patient.

Conclusions

Thanks to its economic and societal value, IMI/CIL/REL therapy represents an investment in health that is lifesaving in critically ill patients and is a valuable public health tool in the fight against antimicrobial resistance.
PD15 Cost Effectiveness Of The Wolbachia Method For Dengue Control In Brazil
Authors: Zimmermann Ivan Ricardo , Fernandes Ricardo , da Costa Márcia Gisele , Pinto Márcia , Peixoto Henry Maia
Published: January 2025

Introduction

Dengue virus is a significant public health threat in Brazil. It is transmitted by Aedes aegypti mosquitoes and causes severe symptoms such as hemorrhagic fever and dengue shock syndrome. This study explored the economic evaluation of Wolbachia mosquito replacement as a promising dengue virus control strategy.

Methods

The model considered Wolbachia mosquito replacement in seven Brazilian cities: Belo Horizonte, Campo Grande, Fortaleza, Goiânia, Manaus, Niterói, and São Paulo. A mathematical microsimulation model tracked 23 million residents over 20 years and considered transitions between five health states (susceptible, asymptomatic, ambulatory, hospitalized, and death). The current dengue control strategy and the incorporation of Wolbachia mosquito replacement were analyzed from both the health sector (public and private) and the Unified Health System perspectives. Direct costs included local dengue control program resources, Wolbachia replacement implementation, and care of patients with dengue virus. The primary outcome was disability-adjusted life-years (DALYs) averted. Sensitivity analyses were also performed.

Results

The model projected 1,762,688 dengue cases over 20 years without Wolbachia replacement. Implementing Wolbachia replacement would prevent at least 1,295,566 cases, demonstrating a high benefit in all simulated cities. Except for Manaus and São Paulo, Wolbachia replacement was dominant (lower costs and higher effectiveness) over current control strategies. Nevertheless, estimated incremental cost-effectiveness ratios for Manaus and São Paulo, which ranged from BRL1,747.11 to BRL5,072.21 (USD309.51 to USD898.56), were well below the Brazilian cost-effectiveness threshold of BRL120,000 (USD21,258.50) for neglected diseases. Furthermore, all incremental net monetary benefit values remained positive for both scenarios in all cities—from BRL41.37 to BRL1,852.42 (USD7.33 to USD328.16).

Conclusions

Wolbachia replacement is a highly cost-effective option in the Brazilian context, aligning with previous international and diverse perspective studies. Sensitivity analysis and alternative scenarios confirmed the robustness of the findings.
PD25 Innovation In Facial Palsy Treatment: The Costs And Benefits Of Telerehabilitation Introduced Into Physical Therapy Pathways
Authors: Szczepura Ala , Khan Amir J , Nduka Charles , Neville Catriona , Martin Helen , Johnson Karen , Oxford Samuel W , Palmer Shea , Holliday Nikki , Bark Chris , Thomson David , Mistry Hema
Published: January 2025

Introduction

The incidence of facial palsy has been rising worldwide, with recent evidence emerging of links to COVID-19 infection. To date, guidance on cost-effective treatments is limited to medication (prednisolone). In terms of physical therapy, neuromuscular retraining (NMR) to restore balanced facial function has been most widely evaluated, but not in terms of cost effectiveness. The added value of telerehabilitation is unknown.

Methods

A multistage technology assessment was conducted, which included the following:

•

a national survey of current therapy pathways in the UK and patients’ and clinicians’ views on the benefits and challenges of telerehabilitation;

•

a systematic review of clinical effectiveness trials evaluating facial NMR therapy;

•

calculation of long-term morbidity costs (national economic burden) based on incidence, patient recovery profiles, health-related quality of life, and national facial palsy treatment costs (valuation of clinical improvements in monetary terms was provided by a national Delphi panel); and

•

evaluation of the cost effectiveness of telerehabilitation (remote monitoring wearables) added to current face-to-face NMR delivery.

Results

Nationally, approximately five percent of patients with facial palsy (17% of unresolved cases) are referred for facial NMR. The long-term economic burden associated with unresolved cases is estimated to range from GBP351 (EUR417) to GBP584 (EUR692) million, indicating substantial savings if long-term recovery can be improved. Medical treatment costs are GBP86.34 (EUR102) million per annual cohort, and physical and psychological therapy costs are GBP643,292 (EUR762,561). Economic modeling showed that telerehabilitation was cost effective, producing a health gain and a cost-saving of GBP468 (EUR555) per patient. If scaled to the national level for all patients who do not recover fully, an annual saving of GBP3.075 (EUR3.65) million is possible.

Conclusions

Economic modeling indicates that NMR could improve patient outcomes and reduce costs. The national survey demonstrated that access to NMR therapy services is limited, so introduction of telerehabilitation could improve access for currently underserved populations. Future clinical trials need to incorporate economic evaluations to help inform decision-making.
PD26 Seeking Sustainability For The Brazilian Public Healthcare System: Cost-Utility Analysis Of Pembrolizumab For Advanced Non-Small Cell Lung Cancer
Authors: Gargano Ludmila , Farinasso Cecilia , De Oliveira Junior Haliton Alves , Lucchetta Rosa
Published: January 2025

Introduction

Pembrolizumab is used as a monotherapy or in addition to chemotherapy for patients with non-small cell lung cancer (NSCLC) as an alternative to platinum-based chemotherapy. The objective was to conduct a cost-utility analysis for subgroups of patients with NSCLC using the Brazilian Unified Health System (SUS) threshold of BRL120,000 (USD24,458) per quality-adjusted life-year (QALY).

Methods

We built a partitioned survival model with three health states based on overall survival (OS) and progression-free survival (PFS) curves for three scenarios according to programed death ligand-1 (PD-L1) expression as follows: (i) pembrolizumab monotherapy for at least 50 percent PD-L1; (ii) pembrolizumab monotherapy for at least one percent PD-L1; and (iii) pembrolizumab plus chemotherapy for one to 49 percent PD-L1. The outcome of interest was QALYs, so utility values were derived from pre- and post-progression states according to the treatment received. Survival curves were extrapolated for 20 years using different distributions. The best fitted distribution was selected by visual inspection, clinical plausibility, and Akaike and Bayesian information criterion tests. Direct costs were also considered.

Results

Pembrolizumab provided incremental gains of 1.23, 0.35, and 1.10 QALYs when compared with platinum-based chemotherapy for scenarios one, two, and three, respectively. The subgroup with the best incremental cost-utility ratio (ICUR) was pembrolizumab monotherapy for at least 50 percent PD-1 (BRL201.366 [USD41,041] per QALY gained), followed by pembrolizumab with chemotherapy for one to 49 percent PD-L1 (BRL267.216 [USD54,463] per QALY gained). When assessed for all PD-L1 positive patients, the ICUR reached BRL571.425 (USD116,465), which was 4.5 times the cost-effectiveness threshold. Sensitivity analysis showed that pembrolizumab must cost a maximum of BRL4.761 (USD970) per vial to be cost effective for the SUS, which is a 40.7 percent reduction in the base case price.

Conclusions

Evidence shows that pembrolizumab alone or in combination with chemotherapy is more effective than platinum-based chemotherapy for treating patients with NSCLC, especially those with a high expression of PD-L1. However, price reduction is essential for pembrolizumab to be cost effective for treating these patients in the SUS.
PD31 The Significant Societal And Environmental Impact Of Chronic Kidney Disease Over The Next Decade In Brazil
Authors: Gobbi Talita , Moreira Ricardo , Montenegro Cinthia , Toledo-Chávarri Ana , Rao Naveen , Priest Stacey , Brown Stephen , Guiang Hannah , Zhao Ming-Hui , Chadban Steven
Published: January 2025

Introduction

Chronic kidney disease (CKD) is a progressively worsening condition that is often overlooked in its early stages. In Brazil, factors such as population aging and rising comorbidities are expected to shift CKD prevalence toward more advanced stages, leading to greater socioeconomic and environmental impacts. The significant burden of renal replacement therapy (RRT) suggests the need to prioritize preventive and early detection strategies.

Methods

We developed a patient-level simulation model to estimate the impact of CKD in Brazil over 10 years (from 2023 to 2032) on clinical, patient, health system, environmental, productivity, and societal outcomes. Validation was conducted against Brazilian demographic data and cross-validated with the Inside CKD model. We estimated productivity losses by multiplying CKD-related workdays missed by daily costs for patients and caregivers.

Results

The number of Brazilians with CKD was projected to increase by 7.2 percent (approximately 27.7 million) over the next 10 years, mainly among patients with late-stage disease, while the number of patients undergoing dialysis was projected to increase by 170.8 percent (approximately 233,000) over the same period. CKD was projected to result in BRL198 billion (USD 38 billion) of lost income. From an environmental standpoint, freshwater consumption, fossil fuel depletion, and carbon dioxide emissions due to patients with CKD were projected to increase by 40 percent by 2032. RRT was projected to require the equivalent annual water usage of approximately 370,000 households and the annual power of approximately 11 million lightbulbs and will produce annual carbon dioxide emissions equivalent to approximately 1.5 million cars.

Conclusions

While the overall number of patients with CKD will increase by 7 percent (from 25.8 million in 2022 to 27.7 million in 2032), the distribution toward later stages of CKD will cause significant impacts in terms of the healthcare system (resource use and costs), patients and caregivers, society, and the environment.
PD45 A Rapid Evidence Synthesis Method For Cancer Screening Recommendations In A Hospital Setting
Authors: Muñoz-Montecinos Carlos , González-Browne Catalina , Maza Felipe , Quirland Camila
Published: January 2025

Introduction

International agencies advocate for population-based cancer screening to prevent cancer-related deaths. The Arturo Lopez Perez Oncology Institute is interested in implementing screening programs, but international recommendations differ on program details such as screening tests, target population, age range, and frequency. A review of international evidence-based recommendations is essential for advising stakeholders on the effective implementation of screening programs.

Methods

A rapid scoping review was performed to identify international recommendations on cancer screening programs. Evidence-based recommendations derived from the World Health Organization and the European Union were analyzed. We also searched for evidence-based recommendations from the following health technology assessment agencies with specific sections for evaluating screening strategies: the Canadian Agency for Drugs and Technologies in Health (Canada), the Institute for Quality and Efficiency in Health Care (Germany), the Medical Services Advisory Committee (Australia), and the National Institute of Health and Care Excellence (UK). Additionally, we explored international cancer screening programs implemented by health systems in the aforementioned countries or in countries with implemented screening programs. Finally, we searched for recommendations from scientific societies on cancer screening strategies. This iterative process was repeated for five different cancers.

Results

We found a total of 32 favorable or unfavorable recommendations for breast, cervical, colorectal, lung, gastric, and prostate cancer screening. Breast and cervical cancer had the highest number of favorable recommendations, with complete agreement on the type of test and only small differences regarding age range and periodicity. On the other hand, we found some recommendations against population-based screening for prostate and gastric cancer and limited agreement for both test type and target population. Direct comparisons between the recommendations served as a guide to elaborate a cancer screening program based on the most recommended strategies.

Conclusions

This rapid scoping review allowed us to assess the consistency of cancer screening recommendations. Major differences were found mainly between recommendations from international agencies and scientific societies. As a result, a cancer screening program was designed based on the most recommended strategies.
PD49 Developing Evidence-Based Optimal Testing Strategies To Monitor Long-Term Conditions In Primary Care
Authors: Elwenspoek Martha , O’Donnell Rachel , Charlwood Katie , Malpass Alice , Ward Mary , Thom Howard , Banks Jonathan , Thomas Clare , Jones Hayley , Sterne Jonathan , Palma Francesco , Stokes Christina , Hay Alastair , Watson Jessica , Whiting Penny
Published: January 2025

Introduction

Most patients with long-term conditions (LTC) receive regular blood tests to monitor disease progression and response to treatment and to detect complications. There is currently no robust evidence to inform recommendations on monitoring. Creating this evidence base is challenging because the benefits and harms of testing are dependent on what is done in response to the test results.

Methods

We identified a list of commonly used tests. We defined a series of filtering questions to determine whether there was evidence to support the rationale of monitoring, such as “Can the general practitioner do anything in response to an abnormal test result?” Through a series of rapid reviews we identified evidence to answer each question. The evidence was presented at a consensus meeting where clinicians and patients voted for inclusion, exclusion, or further analysis. A process evaluation was performed alongside this. Further analyses were performed using routinely collected healthcare data and by performing incidence analyses, emulating randomized controlled trials (RCTs), and modeling disease progression.

Results

We tested this methodology on three common LTCs: chronic kidney disease (CKD), type 2 diabetes mellitus (T2DM), and hypertension. We found sufficient evidence to include hemoglobin A1C and estimated glomerular filtration rate (eGFR) for monitoring patients with T2DM; hemoglobin and eGFR for patients with CKD; and eGFR for patients with hypertension. The consensus panel excluded four tests, while 10 tests were selected for further analysis. The emulated RCTs will investigate the effect of regular monitoring with certain tests on health outcomes among routinely monitored patients. In addition, we will investigate the signal-to-noise ratio of each test over time using a modeling approach.

Conclusions

The cost effectiveness of the evidence-based testing panels needs to be tested in clinical practice. We are currently developing an intervention package and are planning to run a feasibility trial. This program of work has the potential to change how LTCs are monitored in primary care, ultimately improving patient outcomes and leading to more efficient use of healthcare resources.
PD56 Long-Term Use Of Lisdexamfetamine For Attention-Deficit/Hyperactivity Disorder: What Does The Evidence Say?
Authors: Ramos Maíra Catharina , Silva Elias Flávia Tavares
Published: January 2025

Introduction

Attention-deficit/hyperactivity disorder (ADHD) is a neuropsychiatric disorder that can interfere with school and academic life, work, and even personal relationships. One of the alternative medications is lisdexamfetamine (LDX), a prodrug amphetamine preparation that lasts an average of 13 hours due to its gradual conversion. Since LDX is used continuously, it is necessary to evaluate its long-term efficacy and safety.

Methods

A rapid health technology assessment (HTA) was performed. Searches were conducted in the PubMed, Embase, Web of Science, and Cochrane Library databases using descriptors and their respective synonyms to identify studies on the long-term efficacy and safety of LDX in people with ADHD. Interventional and control group studies with a follow-up period of more than five weeks were included. Secondary studies were excluded. The reference lists of included studies were screened to identify potentially eligible publications that were not found in the database searches. Study selection was carried out in two stages, with screening of titles and abstracts and then assessment of full-text articles for eligibility.

Results

This rapid HTA included 32 studies. The population included patients aged five to 55 years, and the longest follow-up was 108 weeks. In general, the literature reported a decrease in symptoms in the first five to six weeks of treatment, stabilizing thereafter. After 108 weeks, the mean change in ADHD Rating Scale-IV hyperactivity/impulsivity was -25.8 (95% confidence interval [CI]: -27.0, -24.5; p<0.001) and -13.1 (95% CI: -13.8, -12.4; p< 0.001) for the ADHD Rating Scale-IV inattention subscale. However, psychiatric disorder system organ class adverse events were frequent, including irritability, anxiety, and aggression, in addition to suicide attempts in severe cases.

Conclusions

It appears that long-term use of LDX has been associated with good clinical results in the treatment of ADHD, with treatment effectiveness remaining stable during the time observed (two-year follow-up). However, adverse events, especially psychiatric disorders, require attention as they can be confused with symptoms of the disease.
PD62 Judicialization Of Health In A Brazilian University Hospital: How Can We Reduce The Budget Impact?
Authors: Ribeiro Mayra Carvalho , Santos Fidelis Giovana Fernanda , Lopes Claudio , Silveira Correa Carlos Roberto , de Oliveira Conterno Lucieni , de Oliveira Motta Maia Flávia , Campello Carvalheira José Barreto
Published: January 2025

Introduction

Public hospitals in São Paulo can be held financially responsible for costs related to medications prescribed outside the recommendations of the Brazilian Unified Health System (SUS). The objective of this study was to describe these expenses in a public hospital and the measures implemented and evaluated to reduce this problem.

Methods

In January 2023, the Health Technology Assessment Center collected data on legal proceedings filed against a tertiary teaching hospital from January 2021 to November 2023. The data were obtained from monthly reports sent by the São Paulo State Department of Health (SES). The proceedings were categorized according to the type of technology and its availability in the SUS, costs, and the prescribing specialties. The indicators developed were used to plan improvement actions to guide care teams, negotiate with the SES, and resolve current legal proceedings.

Results

The cost of legal proceedings for 136 patients was BRL4,410,278 (USD890,965). Four medicines for six patients constituted 56 percent of the total cost. A group created an informative folder explaining how to access the National List of Essential Medicines, prescribe medicines from the high-cost program, and make administrative requests. Other related actions were the creation of a standardized process for requesting medicines, monthly assessment of judicialization data, clinical discussions with prescribers, and educational activities with residents. The interventions reduced average monthly costs from BRL177,268 (USD35,811) to BRL85,493 (USD17,271) in the last trimester.

Conclusions

Knowledge and measurement of judicialization costs allowed the hospital to implement improvements to help avoid new legal proceedings and to understand the demands of medical specialties regarding situations not covered by SUS guidelines. The Health Technology Assessment Center’s work with managers made it possible to identify opportunities for improving the education of professionals regarding the procedures and technologies available in the SUS.