bluuebunny/crossref_metadata_2025 huggingface dataset:
PP58 Enhancing Health Technology Assessment Understanding Through Targeted Educational Programs
Authors: Chitan Elena , Timotin Alina , Anisei Angela , Volovei Ilie , Lozan Oleg
Published: January 2025

Introduction

As health technology assessment (HTA) becomes vital in healthcare decision-making, the demand for specialized education grows. A new HTA course, tailored to the specific needs of the Republic of Moldova’s health system , was launched under a joint project—a collaboration between the World Bank, the Swiss Cooperation Office, Radboud University Medical Center, and the School of Public Health Management. The course targets first- and second-year master’s students, and health professionals enrolled in continuing education.

Methods

The course aimed to introduce the fundamentals of HTA. Participants included 49 master’s students and 26 health professionals. A pre- and post-test model was employed, with participants completing a 10-(multiple) question HTA knowledge assessment at both the start and end of the course.

Results

Initial pre-test results showed an average score of 30 percent, reflecting limited prior HTA knowledge. Following course completion, the post-test average escalated to 80 percent. This 50 percent increase in knowledge was consistent across student and professional groups. While a significant 79 percent of participants accurately answered questions about the use of HTA reports from other jurisdictions, identification of stakeholders, and the elements of establishing PICO (population, intervention, comparator, outcome), they found it more challenging to understand the deeper aspects of HTA. Notably, about 72 percent had difficulties with questions related to the main goals of HTA, evaluating the broader impacts of health technologies, and starting the evidence-based deliberative decision-making process.

Conclusions

The HTA course successfully covered basic concepts, yet it also highlighted the need for more comprehensive teaching of complex topics. The participants showed varying levels of understanding. This underscores the necessity for an HTA curriculum that equally emphasizes fundamental knowledge and in-depth analysis, preparing future healthcare professionals for complex decision-making in their roles.
PP59 Do Patient Contributions Matter? A Thematic Document Analysis Of NICE Ultra-Rare Disease Appraisals
Authors: Gentilini Arianna , Rana Alina
Published: January 2025

Introduction

Patient organizations play a crucial role in health technology assessment (HTA), especially for rare diseases. Despite their important role, the contributions and impact of patient organizations have been overlooked in the literature. This study aims to address this gap by examining the contributions of patient organizations and their nominated experts in National Institute for Health Care and Excellence (NICE) highly specialised technology (HST) appraisals.

Methods

We thematically analyzed the scope and frequency of contributions from patient organizations and experts associated with 10 NICE HST appraisals completed between January 2022 and September 2023. First, to allow for a representation of what patient contributions focus on, their written submissions were categorized according to themes following a deductive/inductive approach, employing a tiered system for disease-, technology-, or submission-specific themes. Second, we compared the themes identified in written submissions with those found in the final NICE recommendations, included in the final evaluation determination (FED), to understand whether and to which extent patients’ contributions were considered.

Results

From 2013 to 2023, 22 drugs underwent HST assessment, with nearly half appraised during 2022 and 2023. All technologies received positive recommendations. A total of 475 unique patient contributions—from both patient organizations and their nominated experts—were identified in their written submission in support of the 10 HST appraisal assessed, predominantly emphasizing disease-specific themes (53%), such as quality of life. While 42 percent of raised themes aligned with FED content, 52 percent did not. When looking at individual appraisals, the share of themes mentioned in patients’ written submission explicitly considered in the FEDs ranged from nine percent to 73 percent, with a median of 50 percent.

Conclusions

Despite progress in integrating patient inputs into HTA, this study reveals a discrepancy between patient priorities and explicit consideration in NICE’s final recommendations. While NICE consistently adheres to its methodology, certain patient-raised aspects are overlooked. Further research is important to discern the optimal areas and timing for patient contribution, refining NICE’s involvement strategies in their decision-making processes.
PP68 Successfully Streamlining New Medicines Assessments In Scotland: A Stakeholder Evaluation Of The Scottish Medicines Consortium’s Abbreviated Submission Process
Authors: Henriquez Alex , McGuire Pauline , Hepburn Christine
Published: January 2025

Introduction

The Scottish Medicines Consortium (SMC) assesses new medicines for NHSScotland, based on company submissions. The abbreviated process previously applied to new formulations of medicines with anticipated low net budget impact. During the COVID-19 pandemic, SMC extended the abbreviated process to new medicines with alternatives within the same therapeutic class already approved in Scotland. This evaluation explored stakeholder perspectives on this process

Methods

A mixed-method approach, with focus groups and online surveys, was utilized to collect data from stakeholders (n=56) including Patient Group Partners (n=14), Public Partners (n=2), pharmacists employed in local health boards and in national procurement (n=17), pharmaceutical industry representatives (n=10), SMC senior clinical assessors (n=6), and the SMC Executive Team (n=7). All participants were asked a series of open and semi-open questions about specific aspects of the process, its advantages, disadvantages, and potential areas for improvement. A thematic analysis, based on initial color coding and grouping of responses into themes, was employed to analyze the qualitative data

Results

Overall, the abbreviated process was commended for its time and resource efficiency, particularly benefiting the SMC assessment teams, decision-making committees, and the pharmaceutical industry. The decision-making committees experienced a decrease in workload and capacity gains, which allowed them to focus on more innovative new medicines. Overall, stakeholders welcomed rapid access to new medicines, and resource savings for pharmaceutical companies, patient groups, and the SMC assessment teams. Suggestions for improvement included strengthening patient group involvement in the process, improving transparency of the decision-making process, in particular the budget impact assessment, and providing further guidance on the definitions of “same therapeutic class.”

Conclusions

Regulatory agencies are licensing more new medicines, in terms of volume and complexity. HTA bodies must consider innovative ways to undertake proportionate assessments. Extension of SMC’s abbreviated process was well commended by stakeholders, primarily due to resource-savings benefits and expedited access to new medicines. Evaluation of the change identified further areas for improvement.
PP74 Are Commonly Used Cost-Effectiveness Thresholds Too Low? Empirical Evidence From Economic Studies On The Value Of Life
Authors: Schlander Michael , Hernandez Diego , Schwarz Oliver , Schaefer Ramon
Published: January 2025

Introduction

In health technology assessment (HTA), “value for money” is frequently conceptualized as incremental cost-effectiveness (CE), with effectiveness measured as (quality-adjusted) life years gained (LYG). Commonly used CE thresholds have been subject to controversial debate. We reviewed and analyzed the worldwide economic literature on the value of a statistical life (VSL), which reported empirical estimates during the last 25 years.

Methods

We conducted an extended systematic literature search in the EconBiz and EconLit databases, spanning the period from January 1995 to December 2020. We transformed VSL data into the implied values of a statistical life year (VSLY) and analyzed the results by regional origin, elicitation method, and annual gross domestic product (GDP) per capita. Then, we performed a regression analysis using the ordinary least squares (OLS) model after log-transforming the VSLY estimates.

Results

We identified 156 empirical economic studies with 169 unique VSL estimates. Overall, the median VSLY was EUR168,367 (mean, EUR256,701) or 6.3 times annual GDP/capita. The median VSLY (expressed as multiples of GDP/capita) differed by regional origin (North America: EUR288,994; 7.2; Europe: EUR168,367, 5.2; Asia: EUR45,260, 4.5) and by elicitation method (revealed preference/wage risk studies: EUR274,625, 9.1; stated preference/contingent valuation studies: EUR113,246, 4.4; stated preference/discrete choice experiments: EUR178,130, 5.2). Regression analyses confirmed that studies with data originating from North America resulted in significantly higher VSLY estimates. The difference remained statistically significant after adjusting for GDP/capita.

Conclusions

The empirical economic literature, while showing heterogeneity by elicitation method, suggests that the adoption of a preference-based or “demand-side” perspective leads to willingness-to-pay estimates for a LYG that exceed commonly used CE thresholds in the context of HTA.
PP76 “It Is Better For Me To Die Than To Be Disgraced”: Perceptions Of Worse-Than-Death Health States In Ghana
Authors: Essel Adwoa , Akweongo Patricia , Addo Rebecca , Nonvignon Justice , Affram Akosua Adjeiwa
Published: January 2025

Introduction

Many cultures across the world have varying conceptions about death and dying. Perceptions about health states considered “worse than death” also vary based on sociocultural norms as well as health system capacity. We explore worse-than-death health states in Ghana as well as reasons for opting for death in those health states.

Methods

We interviewed 28 participants from three regions in Ghana to understand the contextual “value of life” in Ghana and factors influencing respondents’ decision to opt to die rather than live in a particular health state. Written consents were sought from all participants to partake in the study. Interviews were conducted in either Twi or English based on each participant’s preference and lasted for an average of 30 to 35 minutes. Interviews were transcribed verbatim and stored in NVivo software. Data were analyzed thematically.

Results

We identified that health states perceived as worse than death were those associated with impairment in mobility, anxiety/depression, and pain/discomfort. Participants preferred death under these circumstances because they wanted to avoid the financial burden on themselves and family, time spent in caregiving by family, loss of personhood, and loss of social status. Decisions regarding health states worse than death hold considerable importance, particularly in a context where culture and societal norms play a role in shaping how quality of life is assessed.

Conclusions

An understanding of the value Ghanaians attach to health states perceived as worse than death provides useful information for patient-centered care. Findings from the study can provide evidence on healthcare resource allocation and aid policymakers and clinicians in making informed decisions on which treatments to prioritize, and how to maximize the overall health and well-being of individuals.
PP79 Challenges With Integrating Early-Stage Cancer Trial Endpoints Into Economic Models: Review Of Canadian And International HTA Recommendations
Authors: Beca Jaclyn , Chan Cherry , Ting Eon
Published: January 2025

Introduction

Therapies for early-stage cancers demonstrate clinical benefit with early endpoints that measure delayed or avoided disease recurrence, and survival benefits take years to confirm. Economic evaluations for health technology assessment (HTA) require assumptions about long-term benefits to project lifetime disease trajectories. We examined economic modeling approaches used in HTA for adjuvant/neo-adjuvant therapies to understand challenges, explore patterns, and identify opportunities for methodological improvements.

Methods

We included drug indications with Canadian Agency for Drugs and Technologies in Health (CADTH) reimbursement recommendations as of November 2023 for adjuvant/neoadjuvant treatment of early-stage solid tumors. We collected recommendation outcomes and details of submitted clinical and economic evidence. Adapting prior work and focusing on threats to validity arising from equivocal methodological challenges, we classified issues raised in the economic review, particularly those related to surrogacy, treatment pathways, and long-term benefit assumptions (extrapolation, duration of benefit, and cure). We made comparisons with respect to the approaches to long-term benefit assumptions and treatment pathways for drug indications that also had appraisals by the National Institute for Health and Care Excellence (NICE, UK) and the Pharmaceutical Benefits Advisory Committee (PBAC, Australia).

Results

Seventeen drug indications for adjuvant/neoadjuvant treatment of solid tumors were included. Reimbursement was recommended in Canada for 83 percent of drug indications, all were reviewed and recommended in the UK, and 11/15 (73%) have been recommended in Australia. Assessments described overall survival (OS) as immature, but interim OS data appeared to be supportive. There was considerable variability in approaches to long-term benefit assumptions by both submitters and reviewers. Modifications to the assumptions were made in two-thirds of reviews before acceptance by HTA. There was notable inconsistency in approaches to handling treatment-waning, while cure assessment time greater than or equal to five years from initiation was consistently considered appropriate by reviewers.

Conclusions

While all assessments recognized immature OS data, positive reimbursement recommendations were common. There was important variability in application of methods to estimate long-term treatment effectiveness, particularly determining appropriate, evidence-informed assumptions for duration of benefit and cure. This research can support guidance, methodological advancements, and consensus-building to appropriately capture benefits and assess uncertainties for treatments in early-stage cancers with more consistency.
PP83 Building Resilient Capacity For The Diagnosis Of Cardiac Pathologies Through Telemedicine: Pilot Study
Authors: Galvan Pedro , Ortellado José , Rivas Ronald , Grossling Benicio , Hilario Enrique
Published: January 2025

Introduction

The evolution of technology in medicine and information and communication technology (ICT) has been an important step to introduce innovative/resilient specialized health services. The emergence of telemedicine offers opportunities to enhance specialized diagnosis services. This study has evaluated the feasibility of using tele-Holter and tele-ABPM (ambulatory blood pressure monitoring) to build a resilient diagnosis network for cardiac pathologies in Paraguayan remote public hospitals.

Methods

This observational descriptive multicenter feasibility study is based on a telemedicine-driven approach for specialized cardiac diagnostic services using Holter and ABPM devices in remote and underserved public hospitals in Paraguay. A telemedicine platform was used to send records of Holter and ABPM devices from the remote hospitals to the cardiologist to screen cardiac pathologies.

Results

During the pilot study, 52 cardiac diagnostic tests were carried out using the tele-Holter and tele-ABPM approach in 10 regional hospitals countrywide. Cardiac diagnosis was performed in 24 patients using Holter and 28 patients using ABPM. The most frequent findings using tele-Holter were normal (91.6%), not sustained ventricular tachycardia (4.2%), and atrial fibrillation (4.2%). Regarding tele-ABPM, the diagnoses performed were arterial hypertension (50.0%), uncontrolled arterial hypertension (40.0%), and normal (10.0%). Overall, an average of 90.0 percent of diagnosed patients suffered high blood pressure and 8.4 percent suffered heart disorder.

Conclusions

According to our results, the use of a telemedicine-driven approach to build a resilient diagnosis network for cardiac pathologies in remote underserved public hospitals in Paraguay is feasible. A widespread use-assessment should be analyzed before this tool is adopted.
PP88 Breaking Boundaries In Health Technology Assessment: Quantifying Non-Traditional Value Of Medical Technology Through ExpertLink Remote Connectivity Solutions
Authors: Wong Callix , Patel Dinesh , Cheng Michael , Khor Tze Wei , Manoharan Devindran , Sothilingam Selvalingam , Tan Hui Meng , Swan Georgia
Published: January 2025

Introduction

A diverse range of services often supplements procedures that involve medical technologies and adds value along patient care pathways. However, these novel elements of value are often not captured in traditional assessment frameworks. ExpertLink is one such example. ExpertLink uses digital solutions to connect clinical experts worldwide, enabling remote training and collaboration, while maintaining the highest standard of patient care.

Methods

Rezum™ is a minimally invasive therapy for patients with symptomatic benign prostatic hyperplasia (BPH). It is a quick day procedure with proven safety, effectiveness, and durability in clinical outcomes. Leveraging ExpertLink, an expert in Sydney, Australia, remotely proctored 11 Rezum™ procedures in Malaysia in November 2022, supporting five urologists in five hospitals across five states within five hours. Efficient and straightforward procedures such as Rezum™ are well suited to remote proctorship. Through this case study, we quantify the sustainability, equity, and access benefits, illustrating the additional value ExpertLink brings across the healthcare continuum and beyond.

Results

For a proctor traveling from Australia to Malaysia, over 6,500 kilometers and 17 hours travel time is saved, equating to an estimated 1,700-kilogram reduction in CO₂ emissions. Without ExpertLink, a proctor may be away from practice for up to a week. ExpertLink allows for continuity of practice, including consultations and procedures, during this time. For five doctors traveling from Malaysia to Australia for training, an estimated 7,400-kilogram reduction in CO₂ emissions and approximately 85 hours travel time is saved. ExpertLink provided 11 geographically dispersed patients with timely access to treatment and expedited the physician learning curve.

Conclusions

This case study illustrates the value for just one technology on one day. ExpertLink embodies novel elements of value that are not captured in traditional value assessment frameworks. Collaborative effort between stakeholders is needed to broaden the view of value in healthcare, incorporate additional elements of value in existing assessment frameworks, and appropriately recognize this often-uncounted value in decision-making.
PP94 Chronic Obstructive Respiratory Disease Patients’ Involvement In The Evaluation Of Transcutaneous Capnography In Primary Care
Authors: Rodríguez-Ortiz-de-Salazar Begoña , Cardaba-Arranz Mario , Palma-Ruiz Matilde , Hass D Nicole , Sanchez-Gomez Luis-Maria , González-Enríquez Jesús
Published: January 2025

Introduction

Patient participation in health technology assessment (HTA) plays an increasingly relevant role due to increased recognition of its essential contribution to addressing uncertainties in evidence and its real-world application. The objective is to analyze and describe how patients with chronic obstructive pulmonary disease (COPD) participate in the evaluation of transcutaneous capnography (TC) in the primary care setting.

Methods

The Spanish Association of Patients with Chronic Obstructive Pulmonary Disease facilitated contact with three COPD expert patients. A face-to-face video interview was conducted with each patient to know about their knowledge of the technology, real-life experiences, and expectations. Patients were informed of the objective of the evaluation and signed confidentiality and conflict of interest forms. All interviews were conducted in April 2023 by two researchers. Expert patients with COPD were able to participate in the review of the protocol and in the final version of the report. Literature searches were also conducted on patient perceptions of TC compared to arterial blood CO2 measurement.

Results

All patients were male, older than 60 years and were ex-smokers with greater than 30 years of tobacco consumption. Patients highlighted the relevance of early detection of COPD to facilitate the planning and organization of treatment as their clinical situation progresses. Likewise, they also emphasized the importance of the implementation of less invasive tests, and the proximity and accessibility of primary care. Two studies reported greater patient satisfaction and less pain with TC than with arterial blood gases.

Conclusions

Expert patients show high expectations for the technology, as it is less invasive than arterial blood gases; they also acknowledged its proximity in primary care settings and the potential for detecting complications of oxygen therapy. Patient participation in HTA adds intangible value, as they provide “disease-specific knowledge” and real-life applicability of the technology.
PP100 A Study On Health-Related Quality Of Life Among Patients With Advanced Gastric Or Gastro-esophageal Junction Adenocarcinoma
Authors: Jin Doyeon , Ahn Jeonghoon , Lee Hyorim , Seo Hyerin
Published: January 2025

Introduction

Health-related quality of Life (HRQoL) is reported as a strong predictor of survival. However, little is known about the relationship between HRQoL and influencing factors of gastric cancer among Korean patients. This study aims to investigate the HRQoL of Korean patients with advanced gastric cancer and identify factors influencing their HRQoL.

Methods

A total of 156 patients with advanced gastric cancer were 1:1 matched for demographics and clinical characteristics between a ramucirumab/paclitaxel combination group and a chemotherapy group. The objective response of the subjects was divided into four categories: partial response (PR), stable disease (SD), progressive disease (PD), and no complete response/no progressive disease (non-CR/non-PD). The six-month changes in HRQoL measured by EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L) were estimated and further investigated for associations with demographic and clinical factors.

Results

The average changes in HRQoL were highest in PR (0.086±0.057), followed by non-CR/non-PD (0.078±0.076), SD (0.035±0.097), and PD (no change). The HRQoL by age group was 0.176±0.088 in subjects 40 years or younger, 0.035±0.071 in the 50s, 0.067±0.072 in the 60s, and −0.003±0.029 in those who were 70 years or older. The HRQoL of females was 0.145±0.136, and for males it was 0.047±0.066. The HRQoL of the non-diabetic group was 0.072±0.095, and for the diabetic group was 0.044±0.057. The HRQoL of the non-hypertensive group was 0.097±0.097, and for the hypertension group was 0.020±0.048.

Conclusions

For advanced gastric cancer patients, the increase of HRQoL was diminished as cancer progresses, age increases, for males, and when comorbidity of diabetes or hypertension exists. The reported HRQoL data is valuable for health technology assessment of advanced gastric cancer agents such as ramucirumab, as this study is a publicly funded neutral study.
PD14 The Cost Effectiveness Of Robot-Assisted Gait Training For Patients In Japan With Subacute Hemiplegic Stroke
Authors: Morii Yasuhiro , Abiko Kagari , Ohashi Kazuki , Osanai Toshiya , Tani Yuji , Tanikawa Takumi , Takamiya Soichiro , Fujiwara Kensuke , Ogasawara Katsuhiko
Published: January 2025

Introduction

Although it is reported that robot-assisted gait training (RAGT) with conventional physiotherapy for stroke patients increases the odds of walking independence at the end of the intervention, compared with conventional physiotherapy alone, it is not clear how long the benefit lasts or whether it is cost effective. This research aimed to clarify the duration of benefit of RAGT and its cost effectiveness.

Methods

A cost-utility analysis was conducted that compared RAGT plus conventional physiotherapy with conventional physiotherapy alone from a public healthcare payer’s perspective. The population comprised patients with subacute hemiplegic stroke who had a modified Rankin Scale of three to five (severe) and were treated in Japanese hospitals. The time horizon was half a year after admission to a convalescent hospital, since no additional benefit has been proved beyond that point. A decision tree model was used for the analysis. The effect of RAGT on walking independence and the durability of the benefit were estimated based on a literature review of randomized controlled trials (RCTs) and a meta-analysis. Costs and utility values were estimated from the literature.

Results

The literature review identified 14 RCTs. A meta-analysis of RCTs with more than two months’ follow-up after the intervention showed a significantly higher rate of independence in walking for the RAGT group at the end of follow-up (risk ratio 1.52, 95% confidence interval: 1.20, 1.93), whereas there was no significant difference between the groups more than three months after treatment. The incremental quality-adjusted life-years (QALYs) was 0.004 and the incremental cost was -USD287, indicating that RAGT with conventional physiotherapy was dominant. The incremental cost-effectiveness ratio was USD5,509 per QALY when it was assumed that the length of stay was not reduced by achieving early independence in walking.

Conclusions

The results showed excellent cost effectiveness for RAGT plus conventional physiotherapy in patients with subacute severe hemiplegic stroke in the Japanese setting when considering a reference value of USD34,000 (JPY5,000,000) per QALY in the cost-effectiveness evaluation. Although the incremental QALY gains were relatively small, cost savings could be expected from achieving early independence in walking.
PD18 Cytomegalovirus Prophylaxis With Valganciclovir For HIV-Immunosuppressed Patients: Cost Effectiveness And Budget Impact Analysis From The Brazilian Healthcare System Perspective
Authors: Gargano Ludmila , André Ariane , Freitas Isabela , de Assis Acurcio Francisco , Alvares-Teodoro Juliana , Guerra Augusto
Published: January 2025

Introduction

Cytomegalovirus (CMV) infections are usually mild but can lead to serious consequences in immunocompromised patients, such as those infected with human immunodeficiency virus (HIV). Valganciclovir, which is approved for treating CMV, may be a cost-effective prophylactic for CMV disease in immunocompromised patients. Cost-effectiveness and budget impact analyses were conducted to compare valganciclovir prophylaxis with no prophylaxis in immunosuppressed patients with HIV.

Methods

Immunosuppressed patients with HIV who were seropositive for CMV were modeled in a one-year horizon decision tree. Outcomes were occurrence of CMV end-organ disease (EOD)–retinitis and gastroenteritis (85 and 15 percent of cases, respectively)–and survival probability after EOD. Direct medical costs related to prophylaxis (induction and maintenance phases) and treatment of EOD were considered. The budget impact analysis (BIA) used the clinical and cost parameters of the cost-effectiveness analysis to compare scenarios with valganciclovir prophylaxis and no prophylaxis in the public health system over a five-year time horizon. The annual market share adoption rate used was 10 percent. The number of eligible patients was calculated from HIV and acquired immunodeficiency syndrome cases reported in the country and epidemiological estimates.

Results

Valganciclovir prophylaxis had an incremental cost of BRL31,781 (USD6,531) and added 0.69 percent in one-year survival, resulting in an incremental cost-effectiveness ratio (ICER) of BRL4,610,022 (USD947,414) per death avoided in one year. The annual budget impact ranged from BRL35,660,931 (USD7,328,743) in the first year to BRL457,707,796 (USD94,064,365) in the fifth year, totaling BRL1,230,400,576 (USD252,861,870) over five years. In sensitivity analyses, the lack of a maximum treatment time for the maintenance phase with valganciclovir (50 to 365 days per year) resulted in a wide variation in the ICER and BIA results.

Conclusions

Despite being recommended for the treatment of CMV retinitis, the use of valganciclovir in immunocompromised patients with HIV who are seropositive for CMV but have not developed CMV disease has a higher ICER when compared with no prophylaxis. For these patients, appropriate use of standard antiretroviral therapy to improve the immune system may be more appropriate.
PD22 Economic Evaluation Of Polygenic Risk Scores In Clinical Practice: A Systematic Review
Authors: Isonne Claudia , Iera Jessica , Pierri Francesco , Migliara Giuseppe , Baccolini Valentina , Sciurti Antonio , Siena Leonardo Maria , Leone Immacolata , Marzuillo Carolina , Villari Paolo
Published: January 2025

Introduction

Implementation of polygenic risk scores (PRS) may improve disease prevention and management, but its benefits and costs are still to be quantified. The review aimed to summarize evidence on the economic evaluation of PRS, exploring how their use impacts healthcare resource utilization, cost outcomes, and overall cost-effectiveness in order to help guide healthcare decision-making and resource allocation.

Methods

The PubMed, Scopus, and Web of Science databases were searched. The search terms were constructed to identify full economic evaluations of healthcare programs containing PRS aimed at assessing the risk of diseases or other health outcomes in any target population. The search strategy was adapted to fit each database’s research criteria. The literature search was supplemented by scanning the reference lists of retrieved articles. No language or date restriction was applied. The database search was rerun just before the final analysis. This research was supported by the European Commission and the Ministry for Universities and Research under the National Recovery and Resilience Plan (M4C2-I1.3 Project PE_00000019 “HEAL ITALIA”).

Results

Of the 1,891 articles screened, seven economic evaluations were included. They were conducted between 2020 and 2022 in Asia (n=1), Australia (n=1), Europe (n=2), and the USA (n=3). The field of application was ophthalmology (n=2), oncology (n=1), cardiovascular (n=2), nephrology (n=1), and endocrinology (n=1). The time horizon was lifetime (57%) or between five and 40 years (43%). The most frequent viewpoint was the health system perspective (n=5). The target populations included healthy people or individuals at risk for a specific disease. Performing PRS tests was found to be a dominant strategy, with lower costs and better health outcomes in all studies.

Conclusions

Although the integration of PRS in clinical practice seems to be a cost-effective strategy, the small number of studies available and the heterogeneity in the field of application and the target populations limits the generalizability of results. A health technology assessment approach could be useful for summarizing the evidence on all domains of PRS implementation.
PD23 Economic Evaluation Of Prophylactic Immunoglobulin Versus Prophylactic Antibiotics In Hematological Malignancies: Results From The RATIONAL Feasibility Trial
Authors: De Albornoz Sara Carrillo , Higgins Alisa , Petrie Dennis , Irving Adam , Fanning Laura , Weinkove Robert , Crispin Philip , Dendle Claire , Gilbertson Michael , Johnston Anna , Keegan Anastazia , Pepperell Dominic , Pullon Humphrey , Reynolds John , Tonder Tina van , Trotman Judith , Waters Neil , Wellard Cameron , Weston Helen , Morrissey Orla , Wood Erica , McQuilten Zoe
Published: January 2025

Introduction

Patients with hematological malignancies are at high risk of infections due to both the disease and the associated treatments. The use of immunoglobulin (Ig) to prevent infections is increasing in this population, but its cost effectiveness is unknown. This trial-based economic evaluation aimed to compare the cost effectiveness of prophylactic Ig with prophylactic antibiotics in patients with hematological malignancies.

Methods

The economic evaluation used individual patient data from the RATIONAL feasibility trial, which randomly assigned 63 adults with chronic lymphocytic leukemia, multiple myeloma, or lymphoma to prophylactic Ig or prophylactic antibiotics. The following two analyses were conducted to estimate the cost effectiveness of the two treatments over the 12-month trial period from the perspective of the Australian health system:

(i) a cost-utility analysis (CUA) to assess the incremental cost per quality-adjusted life-year (QALY) gained using data collected with the EuroQol 5D-5L questionnaire; and

(ii) a cost-effectiveness analysis (CEA) to assess the incremental cost per serious infection prevented (grade ≥3) and per infection prevented (any grade).

Results

The total cost per patient was significantly higher in the Ig arm than in the antibiotic arm (difference AUD29,140 [USD19,000]). There were non-significant differences in health outcomes between the treatment arms: patients treated with Ig had fewer QALYs (difference −0.072) and serious infections (difference −0.26) than those given antibiotics, but more overall infections (difference 0.76). The incremental cost-effectiveness from the CUA indicated that Ig was more costly than antibiotics and associated with fewer QALYs. In the CEA, Ig costed an additional AUD111,262 (USD73,000) per serious infection prevented, but it was more costly than antibiotics and associated with more infections when all infections were included.

Conclusions

These results indicate that, on average, Ig prophylactic treatment may not be cost effective compared with prophylactic antibiotics for the group of patients with hematological malignancies recruited to the RATIONAL feasibility trial. Further research is needed to confirm these findings in a larger population and over the longer term.
PD27 The Cost Effectiveness Of Non-Hospital Cardiac Rehabilitation: A Systematic Review
Authors: Guirado-Fuentes Carmen , Duarte-Díaz Andrea , Ramos Estefania Herrera , Sacks Analía Abt , García-Pérez Lidia
Published: January 2025

Introduction

Cardiac rehabilitation (CR) with physical exercise is crucial for the secondary prevention of myocardial infarction and heart failure. However, according to published studies there are differences in access to hospital-based CR depending on sex, age, ethnicity, and geographical region. An alternative is CR in non-hospital settings such as primary care, the patient’s home, or another place by means of telerehabilitation.

Methods

We conducted a systematic review of full economic evaluations where non-hospital CR was compared with hospital CR in patients with ischemic heart disease or heart failure. Other eligibility criteria were model-based or clinical trial-based evaluations; studies reporting quality-adjusted life-years, years gained, or other clinical outcomes relevant to CR; and studies published in English or Spanish. Searches were conducted in June 2023 in various literature databases, including MEDLINE, Embase, CINAHL, Web of Science, INAHTA, PEDro, the Cost-Effectiveness Analysis Registry, and others. Study selection, data extraction, quality assessment, and evidence synthesis were conducted by one economist and checked by a second reviewer.

Results

Nine studies were selected from the 673 references identified. Another study was identified through previous systematic reviews. Ten randomized clinical trials were included in the review. None of the studies found differences in effectiveness between hospital CR and non-hospital CR. Two studies found that non-hospital CR was less costly than hospital CR, whereas the remainder did not find any differences in costs between the two groups or were unable to demonstrate the statistical significance of any differences observed. The best conducted studies concluded that non-hospital CR was as effective as and less costly than hospital CR.

Conclusions

Non-hospital CR was as cost effective as hospital CR for low- to-moderate risk patients. Based on the evidence, CR can be recommended in non-hospital settings. However, any form of CR should be evaluated after implementation because its complexity limits the generalizability of results across regions.
PD32 Incorporating Environmental Factors Into Health Technology Assessment Submissions: A Scoping Review
Authors: Briggs Andrew , Germeni Evi , Chen Jieling
Published: January 2025

Introduction

A scoping review was conducted to understand which environmental factors are currently being discussed as candidates for inclusion in health technology assessments (HTAs). By mapping the current literature, the results of the scoping review will inform future work to assess the importance policymakers place on aspects of environmental concern.

Methods

The scoping review involved literature searches across three electronic databases (MEDLINE, CINAHL, and PsycINFO), encompassing articles published up to the end of 2023, to identify a starting set of articles. Backward and forward citation searching of relevant studies was then employed to expand the scoping review, with inclusion criteria established to select studies based on the specific focus on HTA. Screening was conducted independently by two reviewers in two stages: title and abstract and then full-text screening. Data extraction involved a structured approach, collating relevant information for thematic analysis.

Results

While the scoping review is ongoing, emerging issues included: methods for incorporating environmental pollution externalities into HTAs; the impact of climate change on health outcomes; intergenerational equity of health outcomes and definitions of sustainability; environmental epidemiology; and the potential for technologies to mitigate the impact of climate change. The ongoing thematic analysis will synthesize the results of the scoping review using mapping and categorization to identify knowledge gaps.

Conclusions

The threat of climate change is the greatest issue faced by HTA in a generation, but this challenge is intersectoral and demands an intersectoral response. This paper identifies environmental concerns that might, in principle, be included within the HTA process. Ultimately, the HTA community must do its part to address the environmental crisis we face and move toward a more sustainable future.
PD33 Using Environmental Impact Data To Support Health Technology Assessment At The National Institute for Health and Care Excellence: An Options Appraisal
Authors: Elvidge Jamie , Kenny Juliet , Shah Koonal
Published: January 2025

Introduction

The National Health Service (NHS) in England aims to be carbon neutral by 2045, acknowledging the link between planetary and human health. By 2028, NHS suppliers will need to report carbon footprint data for their products, including the medicines and technologies that the National Institute for Health and Care Excellence (NICE) appraises. Additionally, company level data are already being reported. This type of data may provide NICE with an opportunity to support system environmental sustainability goals.

Methods

The NICE Science Policy and Research team conducted an options appraisal to consider the feasibility and acceptability of different ways NICE might engage with environmental impact data (EID). We held discussions with NICE teams that could be affected by the collection and use of EID. Discussions examined the array of potential options for NICE—from being a simple conduit for EID to incorporating EID into its decision-making—and sought to identify those that were practical for further consideration. We then discussed these options with key external stakeholders (NHS England, industry, and commissioners) to understand their expected usefulness and practicality.

Results

Several options for NICE to use EID were identified as suitable for further consideration. Using company level data, NICE could encourage companies to engage with NHS sustainability goals by citing their carbon reduction plans alongside guidance or using them to prioritize its activities. Using product level data, NICE could pilot an evaluation comparing the environmental outcomes of competing health technologies that have no differential direct health outcomes. NICE could also publish environmental impact assessment tools to help commissioners consider EID in procurement decisions. Better data and methodological standards are needed before NICE might consider embedding product-level EID in its usual decision-making frameworks.

Conclusions

Our options appraisal has identified several ways that NICE might start to engage with EID to have a positive impact on NHS sustainability goals and wider planetary health. Future work will scope out how these activities should proceed. The identified options are not necessarily mutually exclusive and may evolve as the data and methods around sustainable health care continue to advance.
PD34 Ethics In Health Technology Assessment – A Case Study Of Prosthetic Care And Proposal For An Empirical Approach
Authors: Baumann Martina
Published: January 2025

Introduction

Medical devices cause significant spending in healthcare systems and provide methodological challenges for health technology assessment (HTA). This poster presents the results of a dissertation on the HTA of prosthetic limb technology and care, with a focus on the ethical implications of this aspect of health care. It is also a case study for ethics in HTA more generally, which will contribute to methodological discussions in this field.

Methods

The methodology was based on Hofmann´s Socratic approach and empirical ethics. Literature reviews of HTA reports and ethics and social science literature on limb prosthetics and care were supplemented by semi-structured interviews with 16 prosthesis wearers and 18 stakeholders (e.g., insurance payers and orthopedic technicians) from Germany. This material served to identify and prioritize ethical issues and dive deeper into the causes of unequal access to prosthetic limbs. Beauchamp and Childress´ principlism and Norman Daniels’ theory of just health were used to describe ethical requirements and conflicts and to discuss the limits of normative recommendations that HTA can provide for decision-makers.

Results

There were 42 ethical aspects related to limb prosthetic technology and care identified, reflecting also general challenges for HTA like artificial intelligence in health and resource scarcity in times of multiple crises. The perspectives of patients and stakeholders provided evidence for unequal access to limb prosthetics that was dependent on socioeconomic status, age, and living region. This is due to a combination of legal framework conditions (not supporting evidence-based reimbursement), socioeconomic success factors in interaction with gatekeepers, non-optimal quality of care (due to lack of data use and scarcity of professionals), and political non-willingness to address rationing–as well as the lack of HTAs.

Conclusions

Access issues in prosthetic care and their implications for patient wellbeing, efficiency, and sustainability may be generalizable to a certain extent to other medical device types and healthcare systems. Governments should provide resources, and synergies with health services research could be leveraged to enable HTA to address the challenges of medical device and ethical assessment.
PD35 A Decade In Review: Assessing The Quality Of Clinical Evidence For Oncology Drugs Submitted To A National Health Technology Assessment Agency
Authors: Eames Heather , Leahy Joy , Trela-larsen Lea , McCullagh Laura
Published: January 2025

Introduction

To ensure accurate and objective comparisons between interventions, the best available clinical evidence must be used. This updated analysis sought to review the quality of clinical evidence submitted by applicant companies to a national health technology assessment (HTA) agency in Ireland. This analysis aimed to assess the association between the quality of clinical evidence and the reimbursement recommendation.

Methods

A retrospective analysis of the clinical evidence supporting all oncology HTAs submitted to the National Centre for Pharmacoeconomics (NCPE) from May 2012 to December 2023 was conducted. NCPE recommendations were classed as “conditional-positive/positive” or “conditional-negative/negative” for the purposes of this analysis. Data extraction of key clinical evidence characteristics relating to the main clinical trial and indirect treatment comparison (ITC), if applicable, was informed by EUnetHTA guidance. Trends in clinical evidence characteristics were assessed over time. Potential associations between the quality of clinical evidence and reimbursement recommendations were investigated using a hierarchy of chi-squared tests and multivariate regression analysis.

Results

This analysis included 101 completed HTAs, 84 of which were supported by traditional randomized controlled trials (RCTs). The proportion of HTAs supported by single-arm trials (SATs) increased to 55.6 percent in 2023. HTAs supported by RCTs were more likely to receive a “conditional-positive/positive” recommendation, compared with those supported by SATs (42.8% versus 17.6%). Most of the RCTs were open-label studies (n=49), however recommendations were similar among HTAs supported by blinded and open-label studies. The overall survival data maturity was variable. ITCs were required in 76 HTAs, mostly supported by anchored ITCs. SATs supported by unanchored ITCs were more likely to receive a “conditional-positive/positive” recommendation than naïve comparisons.

Conclusions

Reliance on SATs and open-label studies has increased over the past decade. This analysis proposes that HTAs supported by RCTs remain the gold standard for HTA. A key limitation of this study is that the quality of evidence submitted is only one domain of the HTA evaluation process. No tests were statistically significant, which was partly explained by the small sample sizes.
PD36 Assigning GRADE Levels To An Overview Of Reviews Using General Principles Identified From Current GRADE Guidelines
Authors: Dullea Andrew , O’Sullivan Lydia , O’Brien Kirsty , Harrington Patricia , McGarry Maeve , Ahern Susan , McGarry Maeve , Walsh Kieran A. , Smith Susan , Ryan Máirín
Published: January 2025

Introduction

Existing guidelines on overviews of reviews and umbrella reviews recommend an assessment of the certainty of evidence but provide limited guidance on how to apply GRADE to such a complex evidence synthesis. We present one approach to applying GRADE to an overview of reviews developed using general principles derived from current GRADE guidelines.

Methods

The methods were developed in an iterative and exploratory fashion following discussion with 11 methodologists and health services researchers. Key principles were distilled on the five GRADE domains (risk of bias, inconsistency, imprecision, indirectness, and publication bias) from the relevant GRADE guidelines, particularly those on test accuracy.

Results

A ‘general principles’ approach of applying the five domains of GRADE to an overview of reviews and arriving at an overall summary judgment for outcomes was developed. These methods were successfully applied to an overview of reviews on 18F-prostate specific membrane antigen positron emission tomography and computed tomography in the staging of patients with high-risk or recurrent prostate cancer.

Conclusions

Our approach distilled key principles from relevant GRADE guidelines and allowed us to apply GRADE to a complex body of evidence. Such an approach may be of interest to other researchers working on overviews of reviews or umbrella reviews.