Oral Presentations
OP115 Scoping Meta-Review On Methods Used To Assess Artificial Intelligence-Based Medical Devices For Heart Failure
- Rossella Di Bidino, Davide Piaggio, Ernesto Iadanza, Leandro Pecchia, Martina Andellini, Raza Zeeshan, Beatriz Merino-Barbancho, Laura López Pérez, Giuseppe Fico, Melody Ni, Simone Borsci, Tianhui Zhu
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- 14 December 2023, p. S32
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Introduction
The International Federation of Medical and Biological Engineering created a multidisciplinary working group to discuss assessments of artificial intelligence and machine learning (AI/ML) applications in health care. Engineers, clinicians, and economists identified evidence generation as a critical topic. Heart failure (HF) was selected to investigate the available evidence on the clinical effectiveness and safety of AI/ML applications. Attention was paid to transparency of AI/ML methods and their data sources.
MethodsA scoping review was conducted on AI/ML algorithms developed for the management of HF. A search for systematic reviews, scoping reviews, and meta-analyses published from 1976 to October 2022 was conducted in Embase, MEDLINE, and Scopus.
ResultsOf 456 relevant publications, 21 papers were included in the final analysis. Most papers (10 systematic reviews, five meta-analyses, and six non-systematic or scoping reviews) included studies conducted in North America. No study was conducted in Africa. The healthcare setting was not clearly stated in approximately half of the studies. A lack of agreement was noticed regarding the quality assessment tools used among the reviews. The most common data source for AI/ML algorithms was electronic health records, but in some cases data sources were not reported. While deep learning emerged as the most common adopted methodology, covariates were not always included in the algorithm development. The review demonstrated that comparative assessment of algorithms requires further investigation, given the high variability in the comparator used (e.g., clinical gold standard, other AI/ML algorithms, or other statistical methods). The main investigated endpoints were the incidence of HF and the number of hospital admissions.
ConclusionsWhen assessing innovative health technologies such as AI/ML applications in health care, evidence is among the main challenges. Our scoping review, focusing on algorithms developed to manage HF, showed that the biggest challenges relate to the quality of the studies, the adoption of a comparative approach, and transparency of methods.
Assessment
Priority setting for health technology adoption at the national level: Lessons learned over 25 years’ experience
- Osnat Luxenburg, Tal Morginstin, Vicki Myers, Mor Saban, Joshua Shemer, Rachel Wilf-Miron
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- Published online by Cambridge University Press:
- 06 November 2023, e71
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Background
Limited health budgets and continual advancement of health technologies require mechanisms for prioritization. Israel, with a publicly funded health service basket, has implemented and optimized such a health technology assessment process since 1999.
We describe the process of evaluating technologies according to the Israeli model, analyze its outputs and benefits over two decades of implementation, and compare its key features with international experience.
MethodsRetrospective data were collected between 1998 and 2023, including work processes, committee composition, number of applications submitted and approved by a clinical domain, and yearly cost of the basket. Features were evaluated within the evidence-informed deliberative process (EDP) framework.
ResultsThis national model involves relevant stake holders in a participatory and transparent process, in a timely manner, and is accepted by the public, health professionals, and policy makers, facilitating early adoption of the newest medical technologies. Between 11 and 19 percent of applications are approved for reimbursement annually, mostly pharmaceuticals. On average 26 percent of approved technologies are added to the list without additional budget. Major domains of approved technologies were oncology, cardiology, and neurology.
ConclusionsIsrael created a unique model for the expansion of the health service basket. Despite an increasing number of applications and rising costs, the mechanism enables a consensus to be reached on which technologies to fund, while remaining within budget constraints and facilitating immediate implementation. The process, which prioritizes transparency and stake holder involvement, allows just a resource allocation while maximizing the adoption of novel technologies, contributing to an outstanding national level of health despite relatively low health spending.
Policy
Dynamic HTA for digital health solutions: opportunities and challenges for patient-centered evaluation
- Jan B. Brönneke, Annika Herr, Simon Reif, Ariel D. Stern
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- Published online by Cambridge University Press:
- 17 November 2023, e72
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Objectives
Germany’s 2019 Digital Healthcare Act (Digitale-Versorgung-Gesetz, or DVG) created a number of opportunities for the digital transformation of the healthcare delivery system. Key among these was the creation of a reimbursement pathway for patient-centered digital health applications (digitale Gesundheitsanwendungen, or DiGA). Worldwide, this is the first structured pathway for “prescribable” health applications at scale. As of October 10, 2023, 49 DiGA were listed in the official directory maintained by Germany’s Federal Institute for Drugs and Medical Devices (BfArM); these are prescribable by physicians and psychotherapists and reimbursed by the German statutory health insurance system for all its 73 million beneficiaries. Looking ahead, a major challenge facing DiGA manufacturers will be the generation of the evidence required for ongoing price negotiations and reimbursement. Current health technology assessment (HTA) methods will need to be adapted for DiGA.
MethodsWe describe the core issues that distinguish HTA in this setting: (i) explicit allowance for more flexible research designs, (ii) the nature of initial evidence generation, which can be delivered (in its final form) up to one year after becoming reimbursable, and (iii) the dynamic nature of both product development and product evaluation. We present the digital health applications in the German DiGA scheme as a case study and highlight the role of RWE in the successful evaluation of DiGA on an ongoing basis.
ResultsWhen a DiGA is likely to be updated and assessed regularly, full-scale RCTs are infeasible; we therefore make the case for using real-world data and real-world evidence (RWE) for dynamic HTAs.
ConclusionsContinous evaluation using RWD is a regulatory innovation that can help improve the quality of DiGAs on the market.
Oral Presentations
OP116 Building Alignment Between Industry, Academia, And Patients On Health Technology Assessment Methods
- Colman Taylor
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- 14 December 2023, p. S33
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Introduction
With the announcement of the health technology assessment (HTA) review in Australia, a HTA Summit was organized by ISPOR Australia in November 2022. The aim of the Summit was to provide a forum for industry, academia, and patients to share ideas and find common ground with respect to HTA policy, processes, and methods. Topics were determined by a Steering Committee and included: managing uncertainty; patient engagement; second order effects; genetics, genomics and precision medicines; conditional listing; and real-world evidence. Presentations on each topic were conducted by industry and non-industry experts. Breakout sessions led by facilitators were also held for each topic with members of the audience.
MethodsDiscussions were recorded during the event and a thematic analysis was performed.
ResultsThe following themes were identified from the event.
• There was a strong sentiment that participants enjoyed the opportunity to discuss ideas and work toward solutions.
• There was a consistent theme that many of the issues arising in HTA were due to a lack of communication between sponsors, evaluators, patients, and decision makers.
• It was noted that HTA encompasses several technical terms that have different meanings among various stakeholders.
• There was a clear consensus that patients should be involved in HTA earlier and throughout the process.
• HTA reform can help drive better access to real-world evidence.
• To improve the efficiency of the process, uncertainty could be reframed as risk management, which incorporates the effect of uncertainty in the funding decision.
• HTA includes policies, processes, and methods and is used as a tool by decision makers to make informed funding decisions. It was noted on several occasions during the Summit that funding decisions have a political element that should be separated from the HTA process.
ConclusionsIt is possible to achieve better collaboration between industry, academic, and patient groups with respect to HTA reform. To promote more collaborative work a consistent conflict of interest definition would be helpful.
OP119 Collectively Improving The Quality Use Of Highly Specialized Medicines: Starting With Biologics
- Jonathan Dartnell, Sue Brown, Louise Hardy, Claire Barrett, Rachelle Buchbinder, Sam Whittle, Lisa Pulver, Nena Nikolic, Debra Rowett, Kylee Hayward, Catherine Hill
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- Published online by Cambridge University Press:
- 14 December 2023, p. S33
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Introduction
In contrast to high-volume medicines prescribed by general practitioners, low-volume highly specialized medicines have not been supported by national quality use of medicine (QUM) programs in Australia. The first area addressed has focused on optimizing use of biological disease-modifying antirheumatic drugs (bDMARDs).
MethodsThe program was designed, developed and implemented in partnership with nine consortium member organizations and four affiliate organizations representing consumer and clinical audiences, program development expertize and implementation capability. The common agenda for the collective impact approach was to achieve better health outcomes for people with inflammatory arthritis, inflammatory bowel disease and plaque psoriasis. Multidisciplinary expert working groups reviewed formative QUM research and agreed on objectives, audiences, messages and interventions. Interventions were selected based on identified barriers, enablers and behavioral drivers, informed by the Theoretical Domains Framework. Interventions were co-designed and tested with end-users. Marketing and promotion activity supported implementation of all interventions through consortium channels and networks. Evaluation includes process, impact and outcome measures, and a realist evaluation of the academic detailing.
ResultsProgram objectives were to optimize: (i) first-line therapy before bDMARD use; (ii) first-choice bDMARDs; (iii) biosimilar prescribing and dispensing; (iv) bDMARD dosage; (v) glucocorticoid and analgesic use. Over 60 interventions supporting key messages for each objective were developed for audiences: consumers; rheumatologists, gastroenterologists, dermatologists; pharmacists; drug and therapeutic committees. Interventions implemented between September 2020 and September 2022 included: consumer decision aids, action plans, fact sheets, lived experience videos; living guidelines and evidence summaries; guidance/position statements for hospitals, podcasts, webinars, online learning; prescribing feedback reports; and academic detailing. Uptake of interventions has largely met targets and surveys have demonstrated shifts in specialist and consumer knowledge and behavior in line with key messages and objectives. Realist and outcome evaluation is ongoing.
ConclusionsOur experience demonstrates the value of a consortium of stakeholder organizations, with different expertise and interests but agreed goals and roles, working together to progress the quality use of highly specialized drugs.
Policy
Real-world evidence: experiences and challenges for decision making in Latin America
- Sebastián García Martí, Andrés Pichón-Riviere, Federico Augustovski, Manuel Espinoza
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- 18 December 2023, e73
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Objective
The Health Technology Assessment (HTA) process aims to optimize health system funding of technologies. In recent years there has been an increase in what is known as Real-World Evidence (RWE) as a complement to clinical trials. The objective of Health Technology Assessment International’s Latin American Policy Forum 2022 was to explore the utility of incorporating RWE into HTA and decision-making processes in the region.
MethodsThis article is based on a background document, survey, and the deliberative work of the country representatives who participated in the Forum.
ResultsThere is a growing interest in the use of Real-World Data / Real-World Evidence in HTA processes in Latin America, although currently there are no specific local guidelines for RWE use by HTA agencies. At present, its use is limited to certain areas such as adding context to HTA reports, the evaluation of adverse events, or cost estimation.
Potential future uses of RWE were identified, including the creation of risk-sharing agreements, the assessment of technology performance in routine practice, providing information on outcomes that are not so easily evaluated in clinical trials (e.g., the identification of specific subpopulations or quality of life), and the estimation of input parameters for economic evaluations.
ConclusionsThe participants agreed that there are several areas presenting significant potential to expand the application of RWD/RWE and that the development of normative frameworks for its use could be helpful.
Oral Presentations
OP120 Developing A Call To Action For Patient Involvement In Health Technology Assessment (HTA) In Southern Africa
- Lauren Pretorius, Debjani Mueller, Anke Peggy Holtorf
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- 14 December 2023, p. S34
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Introduction
In building health technology assessment (HTA) and related decision processes in Southern Africa, institutions and stakeholders face region-specific challenges such as disease prevalence and population makeup. These can be addressed by collaboratively discussing patient engagement solutions that fit the local culture and systems and serve to ensure equitable and sustainable access to patient-relevant health technologies. Our aim is to initiate a collaboration for driving patient involvement (PI) suitable for the Southern African context and Sub-Saharan patient communities. In addition, we explore current experiences in PI, including the stakeholder expectations, gaps, limitations, and new opportunities.
MethodsA one-day hybrid multi-stakeholder PI in HTA workshop was held in Johannesburg, South Africa. Co-created by the participants, the outputs are a call to action and a concept draft for the vital success criteria for PI in the region. The content of the call to action is gathered from pre-workshop surveys, interviews, and outcomes from historic meetings held in conjunction with the Health Technology Assessment International (HTAi) PI workstream as well as facilitated discussion from the actual workshop.
ResultsThe workshop was attended by 42 participants from nine countries, representing diverse stakeholder groups. The attendees represented multiple PI stakeholder groups. The workshop survey was completed by 44 respondents, while 12 participants completed the post-event survey. A workshop outcomes document highlighting a high level of alignment and identifying seven key success factors was developed. A workshop proceeding detailing the outcomes is now being drafted.
ConclusionsOver 95 percent of respondents to pre-and post-surveys indicated an interest in contributing to a more in-depth description of PI in their country. While the majority of participants were from South Africa, participants from Tanzania, Ethiopia, Zambia, and Zimbabwe emphasized that trans-African-engagement for HTA will provide an additional opportunity for HTA in Africa and patient and community participation in HTA and healthcare decision-making. Hence, a collaborative platform could help all African countries to advance and benefit from improved healthcare decision processes.
Method
A framework for local-level economic evaluation to inform implementation decisions: health service interventions to prevent hospital-acquired hypoglycemia
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- Jodi Gray, Tilenka R. Thynne, Vaughn Eaton, Brianna Reade, Rebecca Larcombe, Linda Baldacchino, Jessica Gehlert, Paul Hakendorf, Jonathan Karnon, for the Southern Adelaide Local Health Network’s (SALHN) Hypoglycaemia Clinical Working Group
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- 20 December 2023, e74
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Objectives
Published evidence on health service interventions should inform decision-making in local health services, but primary effectiveness studies and cost-effectiveness analyses are unlikely to reflect contexts other than those in which the evaluations were undertaken. A ten-step framework was developed and applied to use published evidence as the basis for local-level economic evaluations that estimate the expected costs and effects of new service intervention options in specific local contexts.
MethodsWorking with a multidisciplinary group of local clinicians, the framework was applied to evaluate intervention options for preventing hospital-acquired hypoglycemia. The framework included: clinical audit and analyses of local health systems data to understand the local context and estimate baseline event rates; pragmatic literature review to identify evidence on relevant intervention options; expert elicitation to adjust published intervention effect estimates to reflect the local context; and modeling to synthesize and calibrate data derived from the disparate data sources.
ResultsFrom forty-seven studies identified in the literature review, the working group selected three interventions for evaluation. The local-level economic evaluation generated estimates of intervention costs and a range of cost, capacity and patient outcome-related consequences, which informed working group recommendations to implement two of the interventions.
ConclusionsThe applied framework for modeled local-level economic evaluation was valued by local stakeholders, in particular the structured, formal approach to identifying and interpreting published evidence alongside local data. Key methodological issues included the handling of alternative reported outcomes and the elicitation of the expected intervention effects in the local context.
Oral Presentations
OP121 Cost-Utility Analysis Of A Supervised Exercise Intervention For Women With Early-Stage Endometrial Cancer
- Yufan Wang, Alexandra L. McCarthy, Haitham Tuffaha
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- Published online by Cambridge University Press:
- 14 December 2023, p. S34
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Introduction
Cardiovascular disease (CVD) is the leading cause of death in women diagnosed with endometrial cancer. There is clinical evidence that exercise significantly reduces the risks of CVD and cancer recurrence; however, it is unclear whether there is value for money in integrating exercise into clinical cancer care for this population. This paper aimed to assess the long-term cost effectiveness of a 12-week supervised exercise intervention, compared with standard care, for women diagnosed with early-stage endometrial cancer.
MethodsA cost-utility analysis was conducted from the Australian health system perspective for a time horizon of five years using a five percent discount rate. A Markov cohort model was designed with six mutually exclusive health states: no CVD; post-stroke; post-coronary heart diseases; post-heart failure; post-cancer recurrence; and death. The model was populated from the best evidence available in the literature. The incremental cost-effectiveness ratio (ICER) and net monetary benefit were reported. Uncertainty in the results was explored using deterministic and probabilistic sensitivity analyses.
ResultsOver the time horizon of five years, the incremental cost of supervised exercise versus standard care was AUD358 (USD236.74) and the incremental quality-adjusted life-year (QALY) was 0.079, resulting in an ICER of AUD5,148 (USD3,404) per QALY gained. The incremental net monetary benefit was AUD3,589 (USD2,373.24) and the likelihood that the supervised exercise intervention was cost effective at a willingness-to-pay threshold of AUD50,000 (USD33,062.75) per QALY was 99.5 percent.
ConclusionsThis is the first economic evaluation of exercise for endometrial cancer survivors. The results suggest that exercise is cost effective in this population. Given the low uncertainty in the outcomes, efforts should focus on implementation of exercise as part of clinical cancer care.
Policy
The value and impact of health technology assessment: discussions and recommendations from the 2023 Health Technology Assessment International Global Policy Forum
- Rebecca Trowman, Antonio Migliore, Daniel A. Ollendorf
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- 22 December 2023, e75
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Health technology assessment (HTA) programs inform decision making about the value and reimbursement of new and existing health technologies; however, they are under increasing pressure to demonstrate that they are a cost-effective use of finite healthcare resources themselves. The 2023 HTAi Global Policy Forum (GPF) discussed the value and impact of HTA, including how it is assessed and communicated, and how it could be enhanced in the future. This article summarizes the discussions held at the 2023 HTAi GPF, where the challenges and opportunities related to the value and impact of HTA were debated. Core themes and recommendations identified that defining the purpose of value and impact assessment is an essential first step prior to undertaking it, and that it can be done through the use and expansion of existing tools. Further work around aligning HTA programs with underlying societal values is needed to ensure the long-term value and impact of HTA. HTA could also have a role in assessing the efficiency of the wider health system by applying HTA methods or concepts to broader budgetary allocations and organizational aspects of health care. Stakeholders (particularly patients, industry, and clinicians but also payers, wider society, and the media) should ideally be actively engaged when undertaking the value and impact assessment of HTA. More concerted efforts in communicating the role and remit of HTA bodies would also help stakeholders to better understand the value and impact of HTA, which in turn could improve the implementation of HTA recommendations and application to future actions in the lifecycle of technologies.
Oral Presentations
OP122 Economic Evidence To Support Expanding Use Of Existing Positron Emission Tomography Technology As A Diagnostic Tool For High-Risk Cancer Patients
- Michelle Tew, Abby Douglas, Jeff Szer, Ashish Bajel, Simon Harrison, Shio Yen Tio, Leon Worth, Rodney Hicks, David Ritchie, Monica Slavin, Karin Thursky, Kim Dalziel
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- Published online by Cambridge University Press:
- 14 December 2023, pp. S34-S35
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Introduction
In Australia, 18F-fluorodeoxyglucose positron emission tomography with low-dose computed tomography (FDG-PET/CT) is currently only funded for cancer staging-related indications. A recent multicenter randomized trial demonstrated that FDG-PET/CT, compared with standard of care computed tomography (CT) imaging, improved antimicrobial management and the outcomes of patients with persistent and recurrent neutropenic fever. There is potential value in expanding the use of FDG-PET/CT as a diagnostic tool for this high-risk population. We conducted an economic evaluation from a healthcare perspective alongside the randomized trial and compared FDG-PET/CT with standard CT up to 6 months after the scans.
MethodsCase report forms were used to collect resource utilization data and length of hospitalization. Effectiveness was measured as the number of patients with antimicrobial rationalization and quality-adjusted life-years (QALYs) derived from patient-reported trial-based health-related quality of life. Generalized linear models (GLM) were used to analyze costs and outcomes. Incremental cost-effectiveness ratios (ICERs) for each of the outcomes were calculated and interpreted as the cost per patient with antimicrobial rationalization and cost per QALY gained. To account for sampling, we performed bootstrapping with 1,000 replications using the recycled predictions method.
ResultsThe adjusted healthcare costs were lower in the FDG-PET/CT group (mean AUD49,563, 95% confidence interval [CI]: 36,867, 65,133; equivalent to USD34,268, 95% CI: 25,490, 45,033) compared with the standard CT group (mean AUD57,574, 95% CI: 44,837, 73,347; equivalent to USD39,807, 95% CI: 31,000, 50,712). The magnitude of differences in QALYs between the two groups was small (0.001; 95% CI: -0.001, -0.001). When simulated 1,000 times, our analysis showed that across both outcomes FDG-PET/CT was the dominant strategy as it was cheaper and had better outcomes than standard CT in 74 percent of simulations.
ConclusionsFDG-PET/CT is cost effective when compared with standard CT for investigating persistent or recurrent neutropenic fever in high-risk patients. Aligning economic evaluations with clinical studies is key to an integrated evidence generation approach for supporting funding for FDG-PET/CT in this patient group.
Assessment
Advancing hospital-based health technology assessment: evaluating genomic panel contracting strategies for blood tumors through a multimethodology
- Susana Afonso, Ana C.L. Vieira, Carla Pereira, Mónica D. Oliveira
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- Published online by Cambridge University Press:
- 22 December 2023, e76
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Introduction
The adoption of genomic technologies in the context of hospital-based health technology assessment presents multiple practical and organizational challenges.
ObjectiveThis study aimed to assist the Instituto Português de Oncologia de Lisboa Francisco Gentil (IPO Lisboa) decision makers in analyzing which acute myeloid leukemia (AML) genomic panel contracting strategies had the highest value-for-money.
MethodsA tailored, three-step approach was developed, which included: mapping clinical pathways of AML patients, building a multicriteria value model using the MACBETH approach to evaluate each genomic testing contracting strategy, and estimating the cost of each strategy through Monte Carlo simulation modeling. The value-for-money of three contracting strategies – “Standard of care (S1),” “FoundationOne Heme test (S2),” and “New diagnostic test infrastructure (S3)” – was then analyzed through strategy landscape and value-for-money graphs.
ResultsImplementing a larger gene panel (S2) and investing in a new diagnostic test infrastructure (S3) were shown to generate extra value, but also to entail extra costs in comparison with the standard of care, with the extra value being explained by making available additional genetic information that enables more personalized treatment and patient monitoring (S2 and S3), access to a broader range of clinical trials (S2), and more complete databases to potentiate research (S3).
ConclusionThe proposed multimethodology provided IPO Lisboa decision makers with comprehensive and insightful information regarding each strategy’s value-for-money, enabling an informed discussion on whether to move from the current Strategy S1 to other competing strategies.
Oral Presentations
OP124 Cost Effectiveness Of End-Stage Renal Disease Treatment Methods In Türkiye
- Mustafa Kılıç, Vahit Yigit
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- 14 December 2023, p. S35
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Introduction
Chronic kidney disease is an important public health problem and is a leading cause of morbidity and mortality worldwide. Hemodialysis (HD), peritoneal dialysis (PD), and kidney transplantation (Tx) are the main treatments for this disease. The aim of this research was to determine the cost effectiveness of treatments for end-stage renal disease from the perspective of a reimbursement institution in Türkiye.
MethodsA Markov model was developed to measure costs and health outcomes in terms of quality-adjusted life-years (QALYs). The model parameters were based on a six percent discount rate, lifetime time horizon, and a reimbursement agency perspective. The main outcome measures were the incremental cost-effectiveness ratio (ICER) and the cost per QALY. One-way and probabilistic sensitivity analyses were performed to determine parameter uncertainty.
ResultsThe lifetime costs of HD, PD, and Tx were USD26,883, USD37,672, and USD31,227, respectively. The lifetime QALYs gained with HD, PD, and Tx were 5.21, 6.77, and 9.73, respectively. The cost per QALY of HD, PD, and Tx were USD5,161, USD5,567, and USD3,211, respectively. Compared with Tx, the ICERs for HD and PD were USD961 and USD2,178, respectively.
ConclusionsCost differences have occurred between the treatment options for end-stage renal disease due to the increase in drug costs in Türkiye in recent years. As seen in the Markov model in this research, HD, PD, and Tx are complementary rather than rival treatments. This study found that the cost effectiveness of Tx is higher than HD or PD. However, the rate of Tx, which has a higher quality of life compared with HD, is around 22 percent in Türkiye; the rate for PD is four percent. It is therefore recommended that a health policy be developed to encourage kidney donation and promote PD as a superior alternative to HD for eligible patients.
OP125 How Can Health Technology Assessment Evolve To Better Consider Benefits For Patients, Their Families, And Carers?
- John Gillespie, Paul Davies
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- 14 December 2023, pp. S35-S36
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Introduction
In Australia, technical guidelines for the health technology assessment (HTA) of medical technologies do not formally include broader societal benefits in the base case economic evaluation; they are considered supplementary analyses. If what matters to patients is relevant and valuable, then why shouldn’t these broader benefits play a more important role? This presentation will consider the challenges and opportunities for HTA guidelines to change to allow this, and the broader implications for decision makers.
MethodsA targeted literature review was undertaken to assess whether economic evaluation methods and their application in HTA are well positioned to assess what matters to patients. Practical challenges for this will be considered, particularly from the perspective of decision makers having a full understanding of broader societal benefits.
ResultsPreliminary findings from the literature review suggested that taking a broader societal perspective in economic evaluations used in HTA has the potential to enable more informed decisions for policy makers. However, there are practical considerations regarding consistent approaches to assessing broader societal and patient benefits.
ConclusionsFor decision makers to be fully informed on the impact of their decisions beyond healthcare budgets alone, explicit consideration of a societal perspective is necessary. However, for decisions to be equitable across different patient groups, there must be consistency in methodological approaches. Fixing this current limitation should not prevent HTA from giving what matters to patients a central role now, and refining methods on an ongoing basis.
OP126 Clinical And Economic Evaluation Of The Effectiveness Of Cerebrolysin® In Neurological Patients With Post-Stroke Complications In Kazakhstan
- Andrey Avdeyev, Alexander Gulyaev
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- 14 December 2023, p. S36
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Introduction
Medical rehabilitation, one of the main components in the care of patients after stroke, is currently not specified in Kazakhstan, even though neurological disorders are a frequent and potentially disabling consequence of a stroke. The study aimed to evaluate the clinical and economic effectiveness of using Cerebrolysin in patients with post-stroke complications in the Republic of Kazakhstan.
MethodsAn annual cost per patient Markov model was developed to compare the use of Cerebrolysin with placebo in the medical rehabilitation of adult patients after acute ischemic stroke. Outcomes and costs were assessed at day 90. Secondary analysis was performed at the end of one year. The primary criterion for effectiveness was change in Action Research Arm Test (ARAT) scale – Hand Function Assessment Test scores. The modified Rankin Scale (mRS) was used as a secondary measure of effectiveness.
ResultsThe results of the cost-effectiveness analysis showed a pharmacoeconomic advantage in using Cerebrolysin, in comparison with placebo, in the early rehabilitation of patients after stroke. Cerebrolysin resulted in a better ratio of the main cost-effectiveness ratio (CER) parameters and a negative incremental cost-effectiveness ratio (ICER), regardless of which effectiveness criterion was used. For the ARAT scale, the CER was USD63.33 versus USD148.07 and the ICER was -USD27.71; for the mRS, the CER was USD45.95 versus USD158.54 and the ICER was -USD14.93. The annual budget impact per patient of funding Cerebrolysin is expected to be an increase in the cost of purchasing the drug (an additional USD343.85) and an overall cost saving in the Cerebrolysin group due to accelerated patient rehabilitation (USD1,944.30 versus USD2,354.37).
ConclusionsNew evidence has emerged on the effectiveness and safety of Cerebrolysin in patients after stroke, which has served as the basis for including this drug in many international clinical recommendations. The pharmacoeconomic advantages of Cerebrolysin make it possible to recommend its use in the medical rehabilitation of patients after stroke in Kazakhstan.
OP127 The Cost Effectiveness Of Anti-Vascular Endothelial Growth Factor Treatments For Age-Related Macular Degeneration In The Italian Healthcare Setting
- Michele Basile, Eugenio Di Brino, Filippo Rumi, Americo Cicchetti
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- 14 December 2023, p. S36
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Introduction
Age-related macular degeneration (AMD) is a common condition that affects the middle part of a patient’s vision. Typically, it first appears in people in their 50s and 60s. While it does not cause total blindness, it can make everyday activities, such as reading and recognizing faces, more difficult. This analysis aimed to define the resource absorption and cost-effectiveness profiles of the anti-vascular endothelial growth factor therapies currently available in the Italian healthcare context.
MethodsA questionnaire was prepared to gather information on specific drivers involved in the provision pathway. The economic analysis was conducted according to activity-based costing methods. A cost-effectiveness analysis was carried out to provide information on the sustainability profile of the treatments available in the Italian setting. Results were reported in terms of the incremental cost-effectiveness ratio (ICER).
Deterministic and probabilistic sensitivity analyses were carried out to test the robustness of the results.
ResultsThe average absorption of resources per patient along the whole clinical pathway for aflibercept, bevacizumab, ranibizumab, and brolucizumab was EUR6,858, EUR1,420, EUR7,930, and EUR5,667, respectively. Brolucizumab was characterized by an unacceptable cost-effectiveness profile (ICER EUR43,454) versus bevacizumab, considering a willingness-to-pay threshold of EUR40,000 per quality-adjusted life-year (QALY). Compared with ranibizumab, brolucizumab was associated with lower costs (EUR22,368 versus EUR29,333) and higher QALYs (12.8 versus 12.6). Brolucizumab had a higher level of QALYs (12.8 vs 12.7) and lower resources absorbed than aflibercept, with a saving of EUR4,222. Therefore, brolucizumab was a dominant alternative to ranibizumab and aflibercept.
ConclusionsThe analysis underlined how brolucizumab is a cost-saving strategy, compared with aflibercept and ranibizumab, and is likely to be cost-effective relative to bevacizumab in the Italian healthcare context.
OP128 Uncertainties In The Cost-Effectiveness Analysis Of Onasemnogene Abeparvovec For Spinal Muscular Atrophy Type 1
- Stefani Borges, Brígida Fernandes, Fernanda Rodrigues, Hérica Núbia Cirilo, Bárbara Krug, Livia Probst, Ivan Zimmermann
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- Published online by Cambridge University Press:
- 14 December 2023, p. S37
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Introduction
Nusinersen and risdiplam are available in the Brazilian Unified Health System (SUS) for the treatment of spinal muscular atrophy (SMA) type 1. Onasemnogene abeparvovec, a promising gene therapy, was approved in 2020 in Brazil. Given the high cost of this therapy and its promise of a lifetime effect, the objective of this study was to evaluate the cost effectiveness of onasemnogene abeparvovec, compared with nusinersen and risdiplam, in the treatment of SMA type 1 from the perspective of SUS in different scenarios.
MethodsA Markov model was adapted from one originally developed for the USA that considers five states of health. Short-term data were obtained from pivotal clinical trials and long-term survival curves were extracted from published reports from the USA. Maintenance of motor function milestones achieved at the end of follow up in clinical trials was considered until death. Costs and quality-adjusted life-years (QALYs) were discounted at five percent per year over a baseline lifetime time horizon. Alternative scenarios were evaluated for horizons of five and ten years, with and without a discount.
ResultsOnasemnogene abeparvovec resulted in an incremental cost of BRL742,890 (USD297,156) per QALY and an increase of 3.32 QALYs in relation to the alternatives over a lifetime time horizon. In the same time horizon, but without the discount, onasemnogene abeparvovec resulted in an incremental cost-effectiveness ratio (ICER) of BRL166,539 (USD66,615) per QALY. In a five-year time horizon, considering the discount rate, the therapy resulted in an ICER of BRL12,527,667 (USD5,011,066); in ten years the ICER was BRL3,384,793 (USD1,353,917).
ConclusionsSince the benefits of onasemnogene abeparvovec mainly occur in the long term, decision makers need to consider the uncertainty of assumptions of sustained effectiveness in view of the high initial cost of the technology.
OP129 A Cost-Utility Analysis Of Denosumab (Prolia®) For Treating Osteoporosis In Postmenopausal Women: A Swiss Healthcare Payer Perspective
- Danielle Stringer, Magdalena Ruth Moshi, Konstance Nicolopoulos, Mathias Jenal, Thomas Vreugdenburg, Ning Ma
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- Published online by Cambridge University Press:
- 14 December 2023, p. S37
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Introduction
Osteoporosis is characterized by decreased bone mass and density, increasing skeletal fragility, and the risk of fragility fracture. Fragility fractures are associated with a high economic burden. Denosumab (Prolia®) is a pharmacological therapy used to treat osteoporosis and reduce the risk of fragility fracture. This study aimed to assess the cost effectiveness of denosumab, compared with other pharmacological therapies (oral bisphosphonates, intravenous [IV] ibandronate, zoledronate, raloxifene, and bazedoxifene) and no treatment, for treating postmenopausal women with osteoporosis.
MethodsA discrete event simulation model was developed using a lifetime time horizon. A Swiss healthcare payer perspective was adopted. Time-to-fracture distributions were derived from Swiss-specific Fracture Risk Assessment Tool (FRAX®) probabilities. Reductions in the risk of vertebral and nonvertebral fractures due to treatment were informed by a Bayesian network meta-analysis. Cost-effectiveness frontier analysis was utilized. Pairwise incremental cost-effectiveness ratios (ICERs) between denosumab and each comparator were also estimated. Sensitivity analyses were conducted to identify key drivers and explore the overall certainty of findings.
ResultsAt a hypothetical willingness-to-pay (WTP) threshold of CHF100,000 (EUR101,630), IV ibandronate was the most cost-effective therapy in women aged 60 years who had a very high risk of fracture, and in women aged 70 or 80 years of any risk level. In women aged 60 years with a lower risk level, zoledronate was the most cost-effective option. Nevertheless, ICERs from pairwise comparisons between denosumab and some comparators (no treatment, bazedoxifene, raloxifene, and/or zoledronate depending on the cohort’s age and risk profile) were below the hypothetical WTP threshold. Higher intervention costs, smaller reductions in the risk of hip fracture, and shorter duration of residual benefit associated with denosumab contributed to the high ICER values seen in pairwise comparisons with oral bisphosphonates (as a class) and IV ibandronate.
ConclusionsThe present evaluation supported the cost effectiveness of denosumab against some, but not all, comparators. Nevertheless, these results should be interpreted cautiously in light of uncertainty in the true effect of treatments on fracture risk.
OP130 Estimating The Economic Burden Of HIV/AIDS In Türkiye Towards The Next Decade
- Yaren Erkut, Mustafa Kurnaz, Ismail Balik, Birol Tibet, Güvenc Kockaya
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- Published online by Cambridge University Press:
- 14 December 2023, p. S38
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Introduction
Approximately 34.8 million people globally are affected by HIV, 34,000 of whom are in Türkiye, and the impact of HIV continues to grow. Not providing the necessary treatment eventually leads to life threatening AIDS-related consequences. In this study, a descriptive analysis of the published official data on HIV/AIDS was undertaken to assess existing statistics and raise social awareness. The aim was to provide data that will help decision makers in future planning by forecasting the possible number of cases and treatment costs to 2030 using available statistics in Türkiye.
MethodsThe forecast of HIV-infected patients, deaths, and healthcare costs to 2030 were calculated using linear regression based on data published between 1985 and 2022 in the literature or by official authorities. The proportion of past deaths caused by AIDS was used to forecast deaths, and the number of patients living with HIV was estimated indirectly using this forecast. Possible treatment expenditures related to HIV/AIDS in Türkiye were estimated based on published healthcare cost data and the number of living patients obtained by considering the possible increase in HIV/AIDS cases, future inflation predictions from the Turkish Central Bank, existing literature, and information from statista.com.
ResultsEstimates suggested that 3,002 new cases of HIV/AIDS in 2021 will become 5,709 among a total of 74,227 living patients in 2030. The 598 deaths reported between 1985 and 2022 are predicted to rise to 1,256 by 2030. Estimation of the total treatment cost of HIV/AIDS was TRY1,051,026,183 (USD118,683,580) in 2021, which was estimated to reach TRY15,432,842,049 (USD296,450,696) by 2030. This consisted of outpatient visits (TRY246,710,838 [USD4,739,088]), laboratory tests (TRY2,072,489,815 [USD39,810,623]), inpatient visits (TRY1,352,192,479 [USD25,974,373]), management of complications (TRY3,371,126,960 [USD64,756,247]), and antiretroviral therapy drugs (TRY8,390,321,957 [USD161,170,365]).
ConclusionsThe prevalence of HIV/AIDS is expected to rise by 137 percent by 2030, with the economic burden increasing 14.7 times in TRY (2.5 times in USD) from 2021 to 2030 in Türkiye. This proves the severity of the situation and the need for relevant policy measures for society.
OP133 COVIDIAGNOSTIX: Health Technology Assessment For COVID-19 Serological Tests As Companion Diagnostics To Vaccination
- Rossella Tomaiuolo, Chiara Di Resta, Pietro Derrico, Matteo Ritrovato, Giuseppe Banfi
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- Published online by Cambridge University Press:
- 14 December 2023, p. S38
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Introduction
In scenarios of vaccine scarcity or the context of organizational complexity, it is necessary to define prioritization strategies for allocating vaccine in compliance with the criteria of equity and efficiency of health resources. The COVIDIAGNOSTIX project, based on health technology assessment (HTA), assessed the role of SARS-CoV-2 serological tests as a companion diagnostic in the definition of strategies for vaccine administration. To guarantee evidence support for health policy choices, two different strategies were analyzed: one based on administering the vaccine booster dose to the entire population (VACCINE strategy) and the other based on allocation criteria (TEST&VACCINE strategy).
MethodsAn Italian multidisciplinary team conducted a decision-oriented HTA using a combination of the EUnetHTA Core Model and the multicriteria decision analysis model based on the Analytic Hierarchy Process.
Moreover, the Department of HTA method was integrated with the Susceptible-Exposed-Infectious-Recovered model, appropriate modelling techniques, simulation, and quantification of uncertainty that considered the ability to reduce deaths and to contain the pandemic. After identifying the evaluation elements and the decision-making structure, the weights of the evaluation areas and key performance indicators were calculated. This is a constituent part of the mathematical model of data processing, as the Analytic Hierarchy Process was based on a structured questionnaire that compared the relative importance of the two elements on a qualitative scale (1=equal importance; 9=more important).
ResultsThe processing of the scores attributed to the key performance indicators concerning all the evaluation domains resulted in a performance of 94 percent for the TEST&VACCINE strategy and 84 percent for the VACCINE strategy. The TEST&VACCINE strategy was the most advantageous in various scenarios due to the greater speed of response from an operational and economic point of view.
ConclusionsThe assessment schemes defined by COVIDIAGNOSTIX (i.e., technologies, intended use, and settings), which highlight the characteristics that differentiate the tests from each other and guarantee a timely and appropriate evaluation, can be adapted to respond to similar health policy management situations.