bluuebunny/crossref_metadata_2025 huggingface dataset:
PP22 A Dashboard To Support The Value Assessment Of Digital Health Technologies For Health Technology Assessment Agencies
Authors: Sandhu Gurmit , Mohamed Ghazali Izzuna Mudla , Laurenzi Emanuele , Miho Enkelejda
Published: January 2025

Introduction

There needs to be more awareness and agreement on value criteria important to digital health stakeholders (DHSs), including health technology assessment agencies, especially in low- and middle-income countries (LMICs). To overcome these challenges, we focused on the feasibility of a dashboard to improve low confidence and assessment readiness of digital health technologies (DHTs) among DHSs in Malaysia.

Methods

“Artificial Intelligence Embedded X-ray for Lung Cancer Screening” from the Malaysian Health Technology Assessment Section (MaHTAS) was selected as a DHT use case. Representatives from MaHTAS, health informatics, research and innovation agencies, the digital health industry, patient advocates, and bioethicists attended an information briefing session and workshops. Participants completed a value assessment checklist questionnaire on the completeness of the information for the use case. Data and insights from these online workshops were incorporated into the development of Figma dashboard mock-up. Feedback from participants, other Malaysian DHSs, additional LMICs, and high-income countries was incorporated into the mock-up.

Results

For the use case, average ratings from participants on the completeness of information on the seven value domains (VDs) were 3.4 and 3.1 for “healthcare system challenges and current use of technology” and “effectiveness” out of a maximum rating of 4 for “sufficient.” Both VDs were classified as “partially sufficient.” Ratings for the remaining VDs were 2.9 on “technical product information and use,” 2.8 on “safety,” 2.5 on “cost and economic impact,” 2.2 for “usability and accessibility,” and “ethical aspects.” These VDs were classified as “not sufficient.” The dashboard and its use were evaluated positively by 17 of 22 DHSs, including participants.

Conclusions

It was feasible to develop a useful dashboard applying a value assessment framework for DHTs on a use case with clear visualizations and a systematic approach. Our results have potential for applications in early scientific advice. It is to be considered that workshop participants’ profiles may create a bias regarding their subjectivity in rating completeness of information on a DHT.
PP25 Artificial Intelligence In Healthcare Decision-Making: Addressing Challenges, Ethical Considerations, And Bias
Authors: Bergemann Rito , Sharma Sugandh , Vanderpuye-Orle Jackie , Poirrier Jean-Etienne
Published: January 2025

Introduction

Artificial intelligence (AI) is transforming healthcare decision-making, particularly in evidence evaluation and health technology assessment (HTA). This research explores challenges and ethical considerations associated with AI implementation, and biases. It highlights the need for diverse stakeholder perspectives and collaboration to ensure responsible AI use. Through transparency, accountability, and bias mitigation, AI has the potential to revolutionize decision-making and improve patient care while promoting equitable outcomes.

Methods

Literature research was conducted, including peer-reviewed studies and grey literature, using the PEARL search strategy. Relevant articles from various databases and sources were screened and selected based on their alignment with the research objectives. The selected articles were then analyzed to identify key findings and insights related to the integration of AI in healthcare decision-making, ethical considerations, bias mitigation, and stakeholder perspectives.

Results

The literature research revealed that AI in healthcare decision-making holds great promise. AI algorithms can efficiently analyze diverse healthcare data sources, improve evidence evaluation, and streamline decision-making processes. Ethical considerations, patient privacy and transparency are crucial. Bias in AI algorithms emerged as a significant challenge, requiring diverse and representative data, bias testing, and explainable AI. Stakeholder engagement plays a vital role in responsible AI implementation. Strategies for ongoing monitoring, collaboration, and training were identified to ensure fair and ethical decision-making in healthcare. The results emphasize the need for a balanced approach to harness the potential of AI while addressing its challenges.

Conclusions

Integration of AI in healthcare decision-making offers promising opportunities but also presents challenges that need to be carefully navigated. By addressing ethical considerations and mitigating bias, AI can revolutionize decision-making, improve patient outcomes, and ensure the responsible and ethical use of AI in healthcare. The results provide valuable insights and recommendations for researchers working in the field of HTA.
PP36 Real-world Effectiveness Of Umeclidinium And Umeclidinium/Vilanterol For Chronic Obstructive Pulmonary Disease: A Singapore Database Study
Authors: Ng Shannon , Ong Benjamin , Ng Kwong
Published: January 2025

Introduction

Long-acting muscarinic antagonist (LAMA) monotherapy and LAMA in combination with a long-acting beta2 agonist are preferred over inhaled corticosteroid/long-acting beta2-agonist (ICS/LABA) therapy for Global Initiative for Chronic Obstructive Lung Disease (COPD) Group B and C patients. This study assesses impact of the subsidy decision of umeclidinium (umec) and umeclidinium/vilanterol (umec/vil) on Singapore’s healthcare system.

Methods

A retrospective cohort study was conducted using national health record databases. Maintenance-naive COPD patients, with no concurrent asthma, initiated on umec, umec/vil, or ICS/LABA from 2016 to 2020, were included. Patient demographics, comorbidities, and clinical characteristics were balanced using propensity score matching. Primary outcomes measured were the rate of severe or moderate COPD exacerbation and pneumonia hospitalization within one-year follow-up. Effect size was estimated using incidence rate ratio (IRR) with 95% confidence intervals (CIs) from Poisson regression. Markov model extrapolated the number of exacerbations and pneumonia hospitalizations avoided arising from the initiation of umec or umec/vil over ICS/LABA.

Results

Patients initiated on umec (n=1,019) were less likely to experience severe (IRR 0.649; 95% CI: 0.438, 0.961) and moderate exacerbations (IRR 0.713; 95% CI: 0.569, 0.892) than ICS/LABA. Similarly, umec/vil-treated patients (n=1,206) had lower rates of severe (IRR 0.713; 95% CI: 0.517, 0.985) and moderate exacerbation (IRR 0.778; 95% CI: 0.642, 0.942). Both therapies were safer than ICS/LABA, with fewer pneumonia hospitalizations for umec (IRR 0.719; 95% CI: 0.532, 0.973) and umec/vil (IRR 0.781; 95% CI: 0.623, 0.980). Coupled with reduced drug cost from value-based pricing, subsidy potentially resulted in SGD53 million (USD39 million) cost savings over 10 years.

Conclusions

As the largest real-world study conducted among COPD patients in Singapore, our findings contribute to the limited real-world evidence in the region. Compared to ICS/LABA, umec or umec/vil were associated with better COPD control and reduced rates of pneumonia hospitalization. This confirms the importance of appropriate prescribing of COPD therapies and validates the subsidy decision.
PP40 The Use Of “Softer” Outcomes In The NICE Health Technology Assessment Process: The Case Of Multiple Sclerosis
Authors: Kritikou Persefoni , Rose Alison
Published: January 2025

Introduction

Multiple sclerosis (MS) is characterized by a significant humanistic burden, as this is a chronic progressive disease associated with high levels of disability and need for caregiver support. Our objective was to explore to what extent “softer” outcome measures are considered as part of the health technology assessment (HTA) process of medications evaluated for the management of MS.

Methods

We performed a review of all Technology Appraisal Guidance (TAs) available at the National Institute for Health and Care Excellence (NICE) website, relating to the management of MS, in order to identify outcomes used either by the manufacturer or discussed by the Committee, that were beyond the typical ones around the clinical evidence, side effects, and costs associated with each technology under evaluation. The review focused on outcomes relating to the caregiver burden, the ease of use of the medication under evaluation, and the fatigue reported by patients.

Results

Fifteen TAs were identified. Eight (53%) of the TAs included information on the caregiver burden, where the manufacturers applied caregiver disutilities in the cost-effectiveness analyses, which were deemed appropriate by the Committee. Six (40%) of the TAs further included a discussion on the ease of use for specific medications where the Committee concluded that the benefits of the mode of administration may not have been captured in the cost-effectiveness analysis. Fatigue was discussed in two TAs; the Committee provided conflicting conclusions around this measure, but in the most recent TA, it recognized that fatigue was an important outcome measure.

Conclusions

Based on the above findings, it appears that “softer” outcome measures are deemed relevant in the HTA process for treatments aimed for MS, on top of the measures typically used in the HTA process; this highlights the importance of recording the humanistic burden of disease in a holistic way.
PP49 Prescribing Trends And Economic Impact Of Somatostatin Analogues In Acromegaly: A Threat To Brazil’s Public Health System Sustainability
Authors: Porto Dantas Lara Benigno , Mendes de Farias Fellipe Miguel , de Sousa Alves da Silva Thizzah Cecília , Naves Luciana Ansaneli , Zimmermann Ivan Ricardo
Published: January 2025

Introduction

First-generation somatostatin analogues (SSA) are indicated as a first-line medical treatment for acromegaly in patients with persistent disease after surgery or who are not eligible for surgery. These drugs are the largest contributor to the direct medical cost of acromegaly management worldwide. We analyze the patterns of prescription and costs of SSA for the treatment of acromegaly in Brazil.

Methods

The first-generation SSA, octreotide LAR (OCT-LAR) and lanreotide autogel (LAN-ATG), are available in Brazil to treat acromegaly through the Specialized Component of Pharmaceutical Assistance policy. The public records of the nationwide database (DATASUS) were accessed to identify the drug use patterns during the year 2022. The current values of the drug acquisition costs of each medication in 2022 were consulted at the national prices database (BPS). Results were converted in purchase power parity (PPP) dollars according to the World Bank rates.

Results

The acquisition cost of each octreotide (OCT-LAR) ampoule was USD492.33 (10 mg), USD537.04 (20 mg), and USD703.79 (30 mg); for lanreotide (LAN-ATG), each ampoule was USD394.74 for the 60 mg dosage, and USD418.48 for the 90 mg and 120 mg dosages. The average annual cost of a monthly 30 mg dosage of OCT-LAR would be estimated as USD8,445,48 against an average annual cost of USD5,021.76 for a monthly 120 mg dosage of LAN-ATG. Thus, we estimate that USD15,520,747.59 was spent on first-generation SSA to treat acromegaly in Brazil’s Unified Health System (SUS) in the year 2022; of this, 79 percent was attributed to OCT-LAR.

Conclusions

Life-long treatment with SSA is related with a high economic burden in Brazil. There is a predominance of OCT-LAR prescription, where expanding the use of LAN-ATG may help reduce costs to the SUS. Nevertheless, studies and investments in other treatments, such as pituitary surgery and radiotherapy access, at a national level are also essential to improve acromegaly treatment costs.
PP52 Pharmaceutical Innovativeness Index – FDA-Approved Prostate Cancer Medications (2011 To 2021): A Case Study
Authors: André Ariane , Gargano Ludmila , Freitas Isabela , de Assis Acurcio Francisco , Alvares-Teodoro Juliana , Guerra Augusto
Published: January 2025

Introduction

Prostate cancer (PC) constitutes 13.5% of all cancer cases globally. Treatment is individualized and depends on various factors. In recent years, there has been an increase in the approval of drugs for the disease by the U.S. Food and Drug Administration (FDA). The study aimed to assess the innovativeness of FDA-approved drugs for PC treatment from 2011 to 2021.

Methods

A search was conducted in the FDA database to identify medications approved from 2011 to 2021 and their supporting studies for approval. The assessment of the value of innovations was performed through the Pharmaceutical Innovativeness Index (PII), a methodology that quantitatively evaluates innovations in four domains: Therapeutic Need and Added Therapeutic Value of the new medication (graded on five levels from important to absent), and Study Design and Methodological Quality (graded on three levels) of the pivotal studies used as a data source for evaluation. Medications are assessed for a specific clinical indication and compared to available therapeutic alternatives.

Results

Seven medications were identified for the treatment of PC, targeting different stages of the disease. The drugs were evaluated with a score ranging from 0 to 100, measuring the degree of innovativeness across the four assessed domains. Five (70%) medications scored above 50.0. The majority of the medications addressed a significant Therapeutic Need (n=4; 56%). The Added Therapeutic Value was assessed based on the survival gain compared to the comparator, with five medications considered poor (70%) and two moderate (30%). Regarding the quality of evidence, most studies showed a low risk of bias and a partially adequate design.

Conclusions

In recent years, there has been an increase in the development of drugs for prostate cancer. However, the Added Therapeutic Value shows a small to moderate increase in survival compared to existing treatments. Many studies used a placebo instead of comparing new medications with available therapies. The PII indicates that, despite advancements, new technologies are needed to improve patient survival.
PP53 Potential Cost-Effectiveness And Innovation Headroom Of A More Accurate Companion Diagnostic For PD-L1 In Non-Small-Cell Lung Cancer
Authors: Akinsoji Toluwase , Dragojlovic Nick , Darviot Cécile , Meunier Michel , Lynd Larry D.
Published: January 2025

Introduction

While targeted therapies have substantially improved survival rates for non-small-cell lung cancer (NSCLC), it remains the leading cause of cancer mortality in the US. Companion diagnostics (CDx) measuring programmed death-ligand 1 (PD-L1) expression help inform NSCLC treatment but have limited accuracy. We assessed the potential value of a more accurate PD-L1 CDx for multiple stakeholders.

Methods

We developed decision tree models to assess the potential cost-effectiveness of a hypothetical new CDx for atezolizumab, a PD-L1 inhibitor used as a first-line therapy for metastatic NSCLC and as an adjuvant therapy for stage II–IIIa NSCLC patients. The sensitivity and specificity of current PD-L1 assays as well as cost and health payoffs were estimated based on data extracted from the scientific literature. We calculated incremental cost-effectiveness ratios (ICERs) for both indications, conducted headroom and threshold analyses, and used model outputs to estimate the size of the US serviceable addressable market (SAM) for a new PD-L1 CDx in the adjuvant indication.

Results

Approximately five percent of metastatic and seven percent of stage II–IIIa NSCLC patients currently tested for PD-L1 expression receive false negative results. An equivalently priced 100 percent accurate PD-L1 CDx would add an average of 0.04 quality-adjusted life years (QALYs) and cost USD6,069 more per metastatic NSCLC patient (ICER: USD144,512/QALY gained; 95% confidence interval [CI]: USD74,178, USD206,937). It would add 0.08 QALYs and cost USD3,682 more for stage II–IIIa NSCLC patients (ICER: USD49,031/QALY gained; 95% CI: USD47,104, USD50,064). The maximum value-based price in stage II–IIIa NSCLC (assuming a USD100,000/QALY willingness-to-pay threshold) would be ˜USD4,000/patient. At a USD500 price per unit, the SAM would be ˜USD6.5M/year.

Conclusions

Because existing PD-L1 assays for NSCLC are optimized for specificity, the cost-effectiveness of a more accurate CDx reflects that of the immunotherapy drug it is paired with. Stage II–IIIa NSCLC has much greater innovation headroom for a new PD-L1 CDx than metastatic NSCLC. Early cost-effectiveness modeling can identify CDx use cases with higher value headroom and help inform market sizing.
PP56 National Immunization Technical Advisory Groups Are Essential For Successful Implementation Of The European Health Technology Assessment Regulation
Authors: Wolters Sharon , Marapin Ramesh , Beekman Jasmijn , Gurgel Gabriel , Viceré Anna
Published: January 2025

Introduction

Starting from 2030, vaccines in the European Union (EU) require joint clinical assessments (JCAs), and joint scientific consultations (JSCs) can be requested from 2025. Involvement of national immunization technical advisory groups (NITAGs) is crucial to address vaccine specificities. However, NITAGs are currently not considered in the EU health technology assessment (HTA) framework. This study highlights potential risks for non-applicability of vaccine JCA reports nationally.

Methods

This study examined where in the JCA and JSC implementation process NITAGs could play an important role and highlighted the practical challenges of incorporating vaccine JCA reports into already very heterogeneous and complex vaccine access pathways across EU Member States. The EU HTA Regulation process was tested for three countries with different vaccine market access characteristics. This study was conducted using JCA guidance documents, NITAG vaccine assessment guidelines, and communications with the respective decision-making bodies.

Results

The EU HTA Regulation framework requires adjustments for vaccine-specific considerations. NITAGs across the EU vary in experience, resources, expertise, and influence on decision-making regarding vaccine assessment and inclusion in the national immunization program. In addition to JCA and JSC, the EU HTA framework also covers horizon scanning, which several NITAGs are currently involved with at national level.. However, the EU HTA framework currently lacks explicit requirements for NITAG input in horizon scanning, JSC, and JCA processes. This could lead to unnecessary duplication of work, further complexity of the processes, and lengthening of population time to access to new vaccines.

Conclusions

The EU HTA framework of vaccines aims to avoid duplicate efforts and enhance patient access, but current processes that will be introduced may not achieve this optimally. Early and systematic inclusion of NITAGs in the horizon scanning, JSC, and JCA processes is pivotal to mitigate the risk of non-applicability and to successfully realize the objectives of the EU HTA framework.
PP84 Developing The Network For The Future Of Healthcare Through Telemedicine-Driven Diagnostic Innovation
Authors: Galvan Pedro , Ortellado José , Rivas Ronald , Grossling Benicio , Hilario Enrique
Published: January 2025

Introduction

The healthcare digital landscape has evolved and is crucial in shaping strategies for fortifying the health system, specifically in building a network for the future of health services. There is also considerable interest in digital health to facilitate innovation and access to health services in underserved hospitals. This study has evaluated the results of a telemedicine-driven diagnostic network in remote Paraguayan hospitals.

Methods

This is a descriptive study, where the results using a digital telemedicine-driven diagnosis innovation in remote public hospitals were evaluated as a tool to improve equity and accessibility of specialized diagnostic services countrywide. For these purposes, the type and frequency of diagnosis performed through a digital telemedicine platform was determined.

Results

During the study, a futuristic telemedicine-driven diagnostic innovation was implemented in 67 hospitals countrywide. The digital telediagnosis network facilitated tele-electrocardiography (ECG), teletomography, tele-electroencephalography (EEG), tele-Holter and tele-ABPM (ambulatory blood pressure monitoring). The implemented digital telemedicine network has performed 828,073 tests in total between 2013 and 2023. The most performed diagnoses were ECG (543,815 tests) followed by teletomography (266,750 tests), EEG (17,418 tests), Holter (43 tests), and ABPM (28 tests).

Conclusions

According to our results, the telemedicine-driven diagnostic innovation network facilitates faster and more equitable access to tertiary-level diagnostic services for patients in remote underserved public hospitals in Paraguay. A widespread use-assessment is necessary before this platform is implemented.
PP92 Enhancing Patient Engagement In Health Technology Assessment: Identification Of Barriers, Facilitators, And Proposals For Improvement
Authors: Triñanes Yolanda , Gómez-Salgado Patricia , Molina-Linde Máximo , Cantero-Muñoz Paula , Martín-Gómez Carmen , Granja-Domínguez Anabel , Toledo-Chávarri Ana
Published: January 2025

Introduction

Patient engagement in the health technology assessment (HTA) field is crucial but faces significant challenges. This study focuses on identifying the barriers and facilitators surrounding the incorporation of patients in HTA while proposing solutions from the perspective of the Patient Interest Group (GIP) within the Spanish Network of Agencies for Health Technology Assessment and Services of the National Health System (RedETS).

Methods

An ad hoc questionnaire with four open-ended questions about barriers, facilitators, and proposals was developed and distributed among the GIP members through an email survey methodology. The obtained results were discussed in an open in-person meeting, and a thematic analysis of the data was conducted.

Results

Fifteen people answered the questionnaire (at least one HTA researcher from each agency), and forty people participated in the face-to-face discussion. Main barriers included the perception of a lack of institutional support, insufficient participative culture, lack of time and experience, and difficulties in planning. Facilitators included activities organized within the GIP and methodological procedures and documents published. The main proposals for improvement were focused on the training and capacity building of HTA researchers and patients, institutional commitment to patient involvement, promotion of forums and activities with patient entities, and improvement of communication channels with patients and dissemination.

Conclusions

Despite current challenges in patient involvement, this study highlights facilitators and proposals for improvement. Specialized training and education, along with the promotion of a participative culture, emerge as relevant strategies.
PP96 Evaluating The Quality-Of-Life Impact Of Erythropoiesis-Stimulating Agents For Lower Risk (Low To Intermediate-1) Myelodysplastic Syndrome Patients
Authors: Bennett Alastair , Manca Andrea
Published: January 2025

Introduction

Patients diagnosed with low- to intermediate-1-risk myelodysplastic syndrome (LR-MDS) encounter symptom burden that diminishes their health-related quality of life (HRQoL). Erythropoiesis-stimulating agents (ESAs) remain an option to alleviate anemia-related symptoms. However, existing HRQoL studies show limited evidential support. This study assesses the impact of ESAs on LR-MDS patients’ EuroQol 5-dimension questionnaire (EQ-5D) scores compared to not using ESA as initial therapy.

Methods

The European MDS Registry (EUMDS) collects information including ESA usage, covariates, and EQ-5D scores at six-month intervals. Estimating average treatment effect (ATE) from observational data requires adjusting for several sources of bias. Our study controls for baseline and time-varying confounding by using inverse probability of treatment weights. Employing a methodology based on marginal structural models, we are able to estimate robust ATEs. A two-part mixed-effects beta model was used to calculate ATE during a four-year follow-up period. We compare ESA therapy every six months versus clinical management not involving the use of ESAs.

Results

Our results show an overall positive ESA effect on EQ-5D over the four-year follow-up period. The majority of time points have a positive ESA effect after adjustment, though a few time points show no effect. The estimated ATE at four years is small: 0.046 (−0.031, 0.114).

Conclusions

We found that use of ESAs over a four-year follow-up period produces mostly positive treatment effect estimates after adjusting for time-varying variables and confounders. Our robust results can be used to inform more reliable treatment decision-making.
PP109 Which Review Is Right For You? Choosing A Review Methodology
Authors: Edwards Mary , di Ruffano Lavinia Ferrante
Published: January 2025

Introduction

While systematic reviews (SRs) are regarded as the gold standard in healthcare evidence reviewing (and a requirement of many health technology assessments [(HTAs)]), other types of review also play an important role throughout a product’s lifecycle. Drawing on more than thirty years’ experience in conducting reviews, we present key points to consider when deciding which review type might be required.

Methods

SRs are recommended when a comprehensive search and synthesis approach is required, for example HTAs. They have highly structured methods, emphasizing bias minimization, transparency, and replicability. “Rapid,” “pragmatic,” or “targeted” reviews are increasingly popular due to their accelerated timelines and reduced costs, with methodological shortcuts possible at various stages. Scoping reviews explore what is known about a topic and typically have a broad research question. “Reviews of reviews” or “overviews” identify existing SRs on an established topic. Finally, “living reviews” follow the same process as an SR or rapid review but incorporate new evidence on a continual or regular basis.

Results

Rapid reviews may be appropriate when flexibility exists regarding the scope and review methods. Any limitations due to methodological shortcuts must be acknowledged in a transparent manner. Scoping reviews are useful for pioneering research ahead of an SR, or early in a product’s development phase, when an overall understanding of the evidence base is required. Reviews of reviews are particularly useful when the size of the primary study literature means that a review of primary studies would be unfeasible. Living reviews are best suited to topics where the evidence base is changing rapidly, or the best information is needed quickly.

Conclusions

When considering conducting or commissioning a review, organizations should consider the intended audience for the review, the resources, time, and budget available, and the size of the existing literature. Although SRs remain the gold standard, a rapid review, scoping review, or review of reviews may offer a more suitable way to approach a given research question.
PD20 Economic Assessment And Budget Impact Model Of Injectable Ganciclovir For Cytomegalovirus Infection In Immunosuppressed Patients In Brazil
Authors: Alvernaz dos Santos Bárbara Rodrigues , Martins Álex , Silva Mendes Ana Clara , Pereira Camila , Nassif Livia , Gargano Ludmila , Freitas Isabela , Muniz-Júnior Roberto , de Assis Acurcio Francisco , Alvares-Teodoro Juliana , Guerra-Junior Augusto
Published: January 2025

Introduction

Cytomegalovirus (CMV), a prevalent human herpes virus, has long periods of latency. It can reactivate following immunosuppression causing diverse clinical manifestations. In immunocompetent individuals, CMV infections are usually asymptomatic. However, in immunosuppressed individuals, such as patients with human immunodeficiency virus, CMV infections can cause serious complications. Antiviral treatment is crucial for minimizing health risks and preventing severe outcomes, such as retinitis and colitis, in this population.

Methods

A cost-effectiveness analysis was conducted using a decision tree model to estimate the incremental cost-effectiveness ratio (ICER) of ganciclovir (1 mg/mL) for people with acquired immunodeficiency syndrome (AIDS) affected by CMV-related retinitis or colitis. The model compared ganciclovir therapy to no ganciclovir, with the outcome being progression of CMV disease. Budget impact was considered over a five-year time frame with 100 percent diffusion among patients with AIDS and CMV-related colitis.

Results

In patients with AIDS and CMV-related retinitis, use of ganciclovir incurred an incremental cost of BRL29,378.95 (USD5,188.70) with an incremental effectiveness of 0.51 in the therapeutic response rate and an ICER of BRL57,605.79 (USD10,173.93). For those with AIDS and CMV-related colitis, ganciclovir incurred an incremental cost of BRL10,040.71 (USD1,773.32), with an incremental effectiveness of 0.26 and an ICER of BRL38,618.11 (USD6,820.45). The estimated cumulative budget impact, considering 100 percent ganciclovir diffusion over five years, was BRL1,124,800,751.17 (USD198,654,342.23) and BRL571,223,309.75 (USD100,885,415.26) for patients with AIDS CMV-related retinitis and colitis, respectively.

Conclusions

The application of economic evaluation and budget impact tools is crucial for maintaining health system sustainability. Introducing treatments with high ICERs and budget impact can jeopardize the health system and access to treatments. Upholding criteria, such as the willingness-to-pay threshold, is essential for ensuring the sustainability and continuity of health care.
PD24 Innovation In Chronic Obstructive Pulmonary Disease Care: Cost Effectiveness Of A New Self-Management Maintenance Program In The United Kingdom
Authors: Gumber Anil , Khan Amir J , Richardson Matthew , Hong Annabel , Nolan Claire , Alqahtani Khaled , Man William , Singh Sally , Smart Freda , Hedger Chris , Houchen-Woolloff Linzy , Szczepura Ala
Published: January 2025

Introduction

Innovation is needed for the growing number of patients with chronic obstructive pulmonary disease (COPD). Pulmonary rehabilitation (PR) is effective in improving exercise tolerance and quality of life, but these benefits do not appear to be sustained. This highlights the need for cost effective methods to maintain benefits on completion of therapy. The findings of a large trial from the UK are reported.

Methods

A two-center randomized controlled trial of patients discharged from PR compared the costs and benefits of PR maintenance with standard care. National Health Service (NHS) resource use, personal expenditure, and societal costs were recorded over one year, and bottom-up costing was undertaken for the PR maintenance program. Changes in health-related quality of life were recorded using the EQ-5D-5L, and differences were compared with the level identified as significant for COPD. A cost utility analysis was undertaken from an NHS perspective; uncertainties in cost and outcome data were incorporated into a sensitivity analysis. Cost-effectiveness ratios and cost-effectiveness acceptability curves (CEACs) were computed.

Results

The study included 116 patients who had finished PR within the last four weeks. The economic analysis showed that mean healthcare costs per patient for PR maintenance were approximately GBP139.72 (EUR165.57) lower than for usual care. The observed 0.118 advantage in mean quality-adjusted life-years (QALYs) (p<0.05) was above the threshold (0.051) for COPD significance. CEACs indicated there was a 97 percent chance of achieving GBP20,000 (EUR23,699.80) per QALY (NICE acceptance level ≤GBP30,000 (EUR35,549.70). Patient and societal costs increased this percentage. It was estimated that if patients with COPD completed a maintenance program following PR, the NHS could save up to GBP28.6 million (EUR33.89 million).

Conclusions

Our findings confirm that a structured PR maintenance program is highly cost effective in extending the benefits of short-term PR. The trial, undertaken during COVID, also signals the potential for emerging digital innovations to provide future transformative change in delivering self-management programs to sustain health and reduce NHS costs for people living with chronic conditions.
PD30 How Is Sustainability Incorporated Into Healthcare Systems? A Progress Report Card For The EU5
Authors: Marlow Laurie , Webb Sarah , Sulanowska Joanna
Published: January 2025

Introduction

In 2020, the World Health Organization (WHO) released guidance for climate-resistant and environmentally sustainable healthcare facilities. Implementation of sustainability into healthcare policy is at varying stages, and its impact on technology appraisal and procurement is expected to differ across countries. The objective of this research was to review healthcare sustainability policy and implementation progress across the EU5.

Methods

Healthcare sustainability policies in the EU5 (France, Germany, Italy, Spain, and the UK) were reviewed. Policies were identified via government websites (e.g., the Ministere de la sante et de la prevention) and through review of climate and sustainability policy news on the WHO website. Common goals featured in the policies included carbon reduction plans, reduction of single use plastics, ethical and environmental considerations, and self-sustainability. These four goals were selected to form sustainability categories for comparisons across markets. The policies were reviewed for each of the categories with respect to level of inclusion, targets, and anticipated implementation dates.

Results

The UK had clear policies focusing on carbon and emission reduction. Implementation of these policies began in April 2023 and will become a requirement for all new procurements from April 2024. France, Italy, and Spain mentioned carbon emission reduction and environmental impact in policies, but targets and guidelines have yet to be set. Italy and the UK had policies relating to reduction of single-use plastics, whereas the other markets did not. Germany had limited policies mandating changes geared toward increased environmental sustainability, focusing instead on self-sustainability and resilience in the market.

Conclusions

As expected, incorporation of sustainability plans into healthcare policy is variable. Within markets with established policies, such as the UK, there is progress toward mandating the inclusion of sustainability policy in procurement, the impact of which may be seen in early 2024. Although the progress of implementing sustainability in other markets is slower, countries are moving toward environmentally sustainable healthcare policies.
PD41 Quality Of Diagnostic Test Accuracy Systematic Reviews In Neglected Diseases Registered In PROSPERO: Meta-Epidemiological Review Of Methodological Compliance
Authors: Komoda Denis Satoshi , de Almeida Cardoso Marilia Mastrocolla , Fernandes Brígida Dias
Published: January 2025

Introduction

Diagnostic test accuracy (DTA) systematic reviews (SR) provide acknowledged challenges for health technology assessment (HTA) due to insufficiency of trials and a paucity of methodological frameworks (compared with interventional SRs). Additionally, research in neglected tropical diseases (NTDs) is scarce. Assessing the methodological compliance of a SR protocol in this context is of utmost importance for developing robust HTA in NTD.

Methods

A search strategy was conducted in PROSPERO in November 2023 to identify protocols of SRs on diagnostic test accuracy using the following terms: “diagnostic accuracy test”, “diagnostic test”, and “diagnostic accuracy”. Deduplication was performed using Excel’s “remove duplicates” functionality. Eligible studies were SRs with predicted meta-analyses on DTA in any NTD (as per the World Health Organization list). in vitro studies, those with non-human populations, and methodological studies were ineligible. The variables of interest included registry characteristics (author, year, country), protocol status, and methodological characteristics (pre-defined statistical analyses, sensitivity analyses, and heterogeneity analyses). The results were presented using descriptive statistics and narrative analysis of methodological issues.

Results

From 7,931 registries, 106 protocols were selected that anticipated conducting a meta-analysis of DTA in the context of NTDs. The number of registry entries has grown steadily from one protocol in 2012 to 17 in 2023. Twenty-three NTDs were identified, the three most common being dengue (n=23; 22%), leishmaniasis (n=18; 17%), and syphilis (n=12; 11%). Only 14 protocols were reported as published. Regarding quality of reporting and methods, half (n=54) of the reports explicitly stated a meta-analytical approach in their title. Only 16 mentioned a sensitivity analysis and 32 did not mention an analysis of heterogeneity.

Conclusions

The meager number of protocols reported as published could mean a lack of updates or publications, or both. The absence of essential methodological descriptions in protocol titles and body text is worrisome and may ultimately be reflected in the number of publications. Further analysis should assess the specifics of methodological quality based on well-established methodological frameworks for quality of reporting and methods.
PD60 The Importance Of Systematic Reviews Addressing Questions Of Prevalence In Health Technology Assessment
Authors: Migliavaca Celina Borges , Barker Timothy Hugh , Stein Cinara , Colpani Verônica , Munn Zachary , Falavigna Maicon
Published: January 2025

Introduction

The use of systematic reviews (SRs) of interventions is commonplace in health technology assessment (HTA). However, SRs synthesizing other data types, such as prevalence, are rarely used. These SRs may complement the HTA process by gathering complementary evidence essential for developing trustworthy recommendations. We aimed to discuss the importance and application of SRs of prevalence in the context of HTA.

Methods

A methodological working group, the Prevalence Estimates Reviews – Systematic Review Methodology Group (PERSyst), was created to provide guidance on how to improve the development of SRs and meta-analyses of prevalence. As part of the group’s work, a guide for HTA developers regarding the value of SRs of prevalence was developed.

Results

There are many benefits to including SRs of prevalence in the process of HTA. These include providing data for estimating burden of disease; helping to set priorities regarding technology assessment; informing the absolute impact on health outcomes from association measures (e.g., relative risk) reported in clinical studies; and providing data for estimating resource requirements for and feasibility of implementing health technologies under consideration. Within the GRADE framework, prevalence estimates are necessary to assess the quality of diagnostic test accuracy evidence and to support decision-making using the Evidence to Decision framework.

Conclusions

Although not commonly used, SRs of prevalence are an important tool in the process of HTA. There is a need for standardization of methodologies and guidance on how to use these reviews in the HTA process.
PD61 Thromboprophylaxis After Major Orthopedic Surgeries: Health Technology Assessment To Promote Access To Oral Anticoagulants
Authors: Ribeiro Mayra Carvalho , Santos Fidelis Giovana Fernanda , Silveira Correa Carlos Roberto , de Oliveira Conterno Lucieni , de Oliveira Motta Maia Flávia
Published: January 2025

Introduction

While implementing an evidence-based guideline for venous thromboembolism (VTE) prophylaxis in a Brazilian tertiary hospital, we identified an unmet need for patients undergoing major orthopedic surgery. The Brazilian Unified Health System (SUS) does not provide access to direct oral anticoagulants (DOACs) or enoxaparin. Therefore, an assessment of the efficacy, safety, and budgetary impact of these medications from a hospital perspective is warranted.

Methods

Our Health Technology Assessment Center performed an overview of systematic reviews (SR) to compare the efficacy and safety of DOACs with enoxaparin. The Cochrane Library, Embase, and MEDLINE databases were searched in May 2023. The relative risks of symptomatic VTE, clinically relevant bleeding, and mortality were collected. The AMSTAR-2 tool was used to assess the methodological quality of included SRs. Treatment costs and estimates of the number of patients undergoing knee or hip arthroplasty were derived from historical institutional data.

Results

Of the 32 SRs included in the analysis, seven performed a network meta-analysis. All SRs had at least one flaw in a critical methodological domain, mainly in not providing the list of excluded studies. Regarding mortality rates, most SRs did not detect any differences between the treatments. The risk of experiencing VTE was lower with DOACs in eight SRs for hip arthroplasty and in five SRs for knee arthroplasty. The risk of major bleeding was similar between the treatments in all but two SRs. Substituting enoxaparin for DOACs led to a cost reduction of BRL490 (USD98) per patient, which could save BRL29,890 (USD6,038) per year.

Conclusions

Patients undergoing hip and knee arthroplasties are at high risk for the occurrence of VTE. Our overview of SRs showed that the efficacy and safety of DOACs are well recognized. DOACs reduce the risk of VTE, but to date patients in Brazil do not have access to these medicines through the SUS. By providing DOACs, hospitals could ensure adequate prophylaxis without increasing costs.
PD65 How Methods Innovation In Health Technology Assessment Missed The Opportunity To Include Health Equity In The Value Puzzle
Authors: Sarri Grammati , Radhakrishnan Amruta , Muir Jeffrey , Stillman Ipek Ozer
Published: January 2025

Introduction

Reconstructing the value puzzle in health technology assessment (HTA) of new technologies is an ongoing discussion among different stakeholders. Little progress has been made toward consistently and transparently incorporating additional value elements, such as health equity, or moving the focus beyond the traditional value elements of clinical benefit and economic cost associated with the introduction of new technologies.

Methods

The objective was to conduct a series of pragmatic reviews of recent HTA guidance documents, international organizations, and previous systematic reviews to answer the following research questions.

•

Would increased familiarity with real-world evidence, advanced analytics, and expanded forms of economic modeling create the forum for HTA bodies to reconsider their processes and include health equity when assessing new products?

•

Which methods innovations would facilitate changes in HTA methods and processes to consistently and transparently assess health equity value elements?

Results were documented and qualitatively synthesized by outlining missed opportunities and highlighting potential barriers to integrating equity-informed HTA processes.

Results

Our findings were grouped into three main parts: HTA guidance summaries, trends summarized by key organizations (HTAi, ISPOR, and others), and peer reviewed publications. HTA bodies have increasingly emphasized health equity concerns and the importance of standardizing methods to support health equity considerations but have not recommended explicit quantitative methods. Our database search found previous systematic literature reviews explicitly referring to methods of integrating real-world evidence into comparative effectiveness assessments, whereas modeling techniques such as distributional, augmented, or cost-effectiveness analyses, and multicriteria decision-making can integrate health equity effects for both patients and healthcare systems.

Conclusions

Our research showcases the gap between recognizing health equity as a missing element in HTA and incorporating methods to implement such considerations into real-life decision-making. Greater familiarity with health equity methods may move the discussion from “whether” to “how” additional value elements such as health equity can inform decision-making.
PD76 Cost-Effectiveness Analysis Of An Influenza Vaccination Program In Algeria, Egypt, Saudi Arabia, Turkey, and the United Arab Emirates
Authors: Jones Arun , Mongan Suzanna , Saleh Ahmed Abdou , Rachida Khelafi , Idrees Majdy , Tag El Din Mohamed Awad , Ergönül Önder , Kim Kyoo , Aouida Nesrine , Kondratenko Alona , Kim Dr Hansoo
Published: January 2025

Introduction

Influenza causes seasonal epidemics and is a significant burden. Annual vaccination is an effective way to prevent influenza and its complications. However, vaccination coverage remains low in North Africa and the Middle East. Quadrivalent vaccines offer protection but require monetary investments. This study aimed to quantify the costs and benefits of subunit quadrivalent influenza vaccination among healthy adults in the selected countries.

Methods

A decision tree was used to model the costs and outcomes of treatment with an inactivated subunit quadrivalent vaccine, compared with no vaccine, in healthy adults from the societal and payer perspectives. Outcomes measured include productive days gained through reduced absenteeism and the proportion of the population acquiring influenza. Each country’s expected health resource utilization costs for influenza were determined through literature searches and consultations with clinical experts. Deterministic and probabilistic sensitivity analyses were undertaken to characterize the effects of parameter uncertainty on our results.

Results

We found that in every country the proportion of people who acquired influenza was 2.4 times lower in vaccinated individuals than in the unvaccinated. This resulted in between 0.9 and 1.5 more productive days worked for vaccinated individuals. The average cost savings from the societal perspective ranged from USD12.36 to USD276.85 per person. Deterministic sensitivity analysis indicated that influenza prevalence was the strongest driver of the model results. A probabilistic sensitivity analysis of over 1,000 simulations resulted in cost savings 54.4 to 99.3 percent of the time.

Conclusions

Vaccination with a subunit quadrivalent influenza vaccine results in cost savings when compared with no vaccination in every country studied. We therefore conclude that, in terms of cost effectiveness, vaccination with a subunit quadrivalent influenza vaccine is dominant over no vaccination in Algeria, Egypt, Saudi Arabia, Turkey, and the United Arab Emirates.