Patient involvement is a core principle of the National Institute for Health and Care Excellence (NICE) and we continually strive to improve patient involvement in health technology assessments (HTAs) of medicines. We iteratively surveyed and reviewed how patient involvement can be improved with patient organizations, patient experts, NICE HTA decision-making committees, and staff. We re-examined feedback that we collect on an ongoing basis, as well as one-off evaluations, to check how we can improve patient involvement.

Improvements ranged from support for and how we work with patient stakeholders to training the various stakeholders who take part in the HTA process to build up a comprehensive and evolving training package and stimulate a cycle of continually improving patient involvement.

We reviewed the outcomes and recommendations from the following larger projects:

• • Review of public involvement across NICE 2015;

• • Improving meaningful patient involvement in HTAs 2019;

• • Improving patient expert involvement in committee meetings 2019; and

• • The value of patient expert input 2022.

Feedback from monthly surveys of patient experts and organizations was also reviewed.

The results included recommendations about:

• • Changing the culture so that patient involvement at NICE is everybody’s business;

• • The key role of the committee chair in including patient experts;

• • The importance of committee culture and behavior in including and valuing patient input;

• • The need for a greater understanding of how NICE technical teams can best support and obtain the most meaningful evidence from patients; and

• • What additional support and training patient organizations and experts want from NICE’s public involvement team.

We concluded that not only patients need training, but also everybody included in the NICE medicines HTA process. Over time we have gradually added to our training portfolio for patient organizations and experts as well as NICE staff and independent committees. We now run patient involvement as part of the induction program for all staff, technical staff, medicines committee chairs, and NICE committees and lay members.

We also provide monthly training for patient organizations and patient experts.

The importance of patient centricity in healthcare decision making has been recognized and advocated for decades. However, approaches for including the patient perspective are diverse, and progress varies among countries. Some reimbursement bodies acknowledge the importance of patient preferences in health technology assessment (HTA) and funding decision processes. However, patients’ perspectives are not yet systematically and transparently included in value assessment frameworks globally, and even less so in the Asia-Pacific (APAC) region. This systematic review aimed to investigate how patients’ perspectives are used to inform pricing and reimbursement decisions in the APAC region.

A systematic review is ongoing that utilized a search of 12 databases, including MEDLINE and Embase, to identify publications on the consideration of patient perspectives in health policy decision-making published to November 2022. Conference abstracts published in the last five years from ISPOR and Health Technology Assessment International (HTAi) were screened, along with gray literature and government websites from Australia, China, Japan, Malaysia, New Zealand, the Philippines, Singapore, South Korea, Taiwan, and Thailand. Publications were included if the impact of either one or more of the following on HTA decision-making was assessed: active participation of patients or patient advocacy groups; type, extent, and evolution of patient-reported outcomes; health-related quality of life or quality of life tools; and themes where the impact of patients’ perspectives on value assessment was the primary outcome. Countries were characterized into archetypes based on similarities or differences in the weight and value assigned to patient perspectives in decision-making.

A total of 6,438 retrieved citations will undergo the systematic review process. Additionally, 758 conference abstracts from ISPOR, 1,312 from HTAi conferences and 73 records from gray literature will be screened.

The results of the systematic review will be consolidated into country archetypes, examples, and learnings. Gaps and opportunities will also be identified.

The research will provide recommendations to increase shared decision-making and support the development of decision-making frameworks that systematically incorporate patients’ perspectives in value assessment across APAC countries.

The mission of the Department of Health (DoH) of Abu Dhabi in the United Arab Emirates is to provide its population with a healthy life and world leading preventive and curative services. While the DoH has regulations in place to grant market approval to new health technologies, there is a need to develop a clear overall framework for reimbursement and disinvestment decisions. Establishing a structured health technology assessment (HTA) framework is critical for informing decisions on health technologies that offer value for money, with the aim of improving equitable access to health care, financial risk protection, and, ultimately, better health outcomes.

During 2022, the DoH collaborated with the Radboud University Medical Center to explore the feasibility of applying an evidence-informed deliberative process (EDP) HTA approach through workshops and interviews involving all stakeholders in the ecosystem, such as policy makers, principal investigators, providers, patients and public groups, product manufacturers, payers, and purchasers. A situational analysis was conducted to collect stakeholders’ views and build EDPs. Based on this analysis, a structured roadmap was developed.

The comprehensive five-year roadmap to implement a holistic HTA framework in Abu Dhabi consisted of five major elements, starting with the establishment of an appropriate HTA policy framework as a foundation. Abu Dhabi should firmly establish its HTA structure and program (in one to two years), and at the same time invest in developing and retaining HTA training capacity so that over time (within three to five years) the country can build up its own expertise to sustain the program. This needs to be accompanied by continuous awareness raising among all relevant stakeholders.

This roadmap is the first and most important step toward implementing a holistic HTA framework in Abu Dhabi. Technical work needs to be complemented by continuously raising awareness and involving all relevant stakeholders. Looking at the initial results and international benchmarks, HTA will significantly contribute to achieving a sustainable, high quality healthcare system.

Clinicians are increasingly relying on artificial intelligence (AI) generated technologies for support in diagnosis, therapeutic decision-making, and prediction. Despite the increased focus on AI in health, an agreed HTA model for AI technologies, including consensus on new domains and topics to be assessed, is lacking.

A Delphi survey was sent to a multidisciplinary expert panel asking about the importance of including the nine domains and associated topics presented in the EUnetHTA Core Model, as well as 20 additional topics identified through literature reviews, when assessing AI-supported health technologies. The Delphi survey was repeated twice among the same panelists and a nine-point Likert scale was used to identify the perceived relevance of each domain and topic.

The survey was sent to 87 various experts, with a total 47 of experts completing both Delphi rounds. The majority of panelists was knowledgeable of HTA (80%), familiar with the EUnetHTA Core Model (61%), and had adequate or high-level knowledge of AI (65%). The EUnetHTA domains most often indicated as “critical to include” were clinical effectiveness (82%), ethical aspects (81%), and cost effectiveness (77%), whereas organizational (59%) and social aspects (63%) were less often perceived as critical to assess. For the additional 20 topics identified through literature reviews, bias in data, accuracy in the AI model, appropriateness, and trustworthiness emerged as some of the new topics deemed critical to include in HTAs (all above 85%), whereas there was a lack of agreement on the relevance of including environmental (51%) and social sustainability (55%).

The study investigated in detail which issues should be included in an AI HTA core model. Current models need some adjustment and revision. At the same time, it is essential to open the discussion on including new domains and topics.

The Health Technology Assessment (HTA) Council in the Philippines carried out its process tracks while the implementing guidelines were being finalized in 2020, due to the urgent need to respond to COVID-19. Two years later, as mandated by the Universal Healthcare (UHC) law, we opened the nominations for the HTA Council’s topic priority list, which will be assessed to inform government financing decisions.

We adopted the former Philippine National Formulary System (PNFS) but set the prioritization criteria according to the decision framework stipulated by the UHC law and allowed industry submissions. We streamlined dossier completion for topics with numerous proponents, supplemented dossier deficiencies, and adjusted the timelines of crucial steps for better reach, while accounting for possible setbacks during the time periods. We satisfied the prioritization criteria using a Delphi technique at the HTA Council subcommittee and Core Committee levels in conjunction with consultations with the Department of Health and the national payer. We also shared evidence base and topic prioritization criteria scores with stakeholders during the public consultation.

In the pilot implementation, we processed a total of 140 nominations (88 complete submissions) and released the priority list in five months. After processing 31 appeals from all key stakeholder groups, the 2022 priority list covered 31 assessments based on topics from the Department of Health, the national payer, industry, hospitals, and medical societies. Although we found gaps in the set timelines, inadequacy in the prioritization criteria parameters, and the need to increase exposure of the public to the process, we were able to accommodate all stakeholder concerns and maintain flexibility in the process.

We need to update our HTA process guidelines, accept joint dossier submissions, and review our topic prioritization process. The changing health system landscape and transitioning of national health priorities require coordination with the Philippine Food and Drug Administration for horizon scanning, early HTA, and managed entry agreements. Finally, there is a need to create special pathways for rare disease and innovative technologies.

Given time and resource constraints, health technology assessments (HTAs) should avoid duplicating prior HTAs. This abstract describes the experience of the Agency for Care Effectiveness (ACE) with adapting and building on published HTAs of medical technologies to support efficient decision-making.

Upon defining the scope of an evaluation topic, searches were performed on reference agency websites and HTA networks, such as the International Network of Agencies for Health Technology Assessment, to identify relevant published HTAs. Identified reports were included for evidence synthesis if the inclusion criteria were met. If multiple relevant HTAs were identified, the most recent or comprehensive HTA identified was used as the foundational evidence base and supplemented by additional primary or secondary literature as needed. When deemed necessary, de novo economic models were developed. Where available, reimbursement decisions or recommendations from published HTAs and their applicability to Singapore’s context were also considered. Following ACE’s evaluations, the Ministry of Health Medical Technology Advisory Committee (MTAC) made subsidy recommendations.

To date, ACE has completed 54 HTA evaluations of medical technologies, 47 (87%) of which incorporated published HTA evidence. Although some MTAC recommendations were aligned with published HTAs, discrepancies were observed that were mainly attributed to evolution of the technology or evidence since publication of HTAs; different local clinical needs or management algorithms; and different local costs of the intervention and comparator. Key challenges in adapting published HTAs included differences in the target population and intervention or comparator in published HTAs, mixed recommendations from published HTAs, and non-English language reports, which may underscore the need to adapt published HTAs to Singapore’s context. To overcome some of these challenges, close consultation with stakeholders is critical to understand local clinical need and care pathways and to gather key information such as costs.

Published HTAs, when used as an evidence base and supplemented by evidence updates and local contextualization, allow for efficient and robust HTA to support decision-making.

Pursuant to the Universal Health Care Act of 2019, the Health Technology Assessment Council (HTAC) in the Philippines was mandated to make recommendations for government financing of health technologies, including medical devices. The development of a Philippine Essential Medical Device List (PEMDL) will serve as a guide for the procurement of medical devices and supplies in government health facilities and as the basis for creating a price reference index.

The HTAC and a team of medical device experts (the Expert Advisory Committee) developed a streamlined pathway and guidelines using a Delphi technique with the Philippine Food and Drug Administration (FDA) and the Department of Health (DOH) to determine the necessary criteria for fast tracking the inclusion of medical devices. The pathway was also revised throughout a pilot processing of commonly procured medical devices (e.g., consumables) that were either already covered by existing national health insurance packages or funded by DOH programs, among others.

The final pathway consisted of validating whether the device was already approved by the Philippine FDA and whether it was already part of standard of care. Similar to the usual HTA nomination requirement, the local FDA approval attested to the safety and quality of the device. Meanwhile, the inclusion of the device in the standard of care guaranteed that it was essential in the healthcare setting. This can be written in the World Health Organization technical documents and databases such as the Medical Devices Information System, health facility listing requirements, or locally adopted clinical practice guidelines. The initial PEMDL for release contained a total of 307 medical devices across 16 categories.

Unlike the mandate for the Philippine National Formulary, government health facilities are not yet required to base their procurement on the PEMDL because the list is still in its infancy. Moving forward, the list will include big ticket items and will be updated through consultations with specialty centers and hospitals.

Our objective was to conceptualize and assess the potential value of a disease management approach to health technology assessment (HTA) in Canada.

We conducted 18 semi-structured interviews between April 2022 and October 2022 to elicit informant views on potential opportunities for re-conceptualizing the decision-problem in HTA as a disease management problem versus a technology management problem. Participants were purposefully sampled from national and provincial HTA agencies and related organizations in Canada to achieve representation across the disease pathway including prevention, screening, and treatment, and the decision-making pathway including HTA organizations, expert committee members, and decision-makers. Data were analyzed using thematic organizations (based on the interview guide) and manual line by line coding of the data. Ethics approval was received from the Health Sciences Research Ethics Board at the University of Toronto.

Three key features of a disease management approach to HTA (i.e., disease-based, multi-interventional, and dynamic) emerged from informants that differed from traditional HTA processes in Canada. The concept was generally not perceived to be a new idea – some informants indicating that it was implicit in the HTA analysis framework. There was general support for an explicit disease management approach to HTA if the impact of the approach could be demonstrated, if the assessment could be completed within an appropriate time frame, and if the assessment could include the equity, ethical and implementation domains of HTA. Informants indicated that the reconceptualization of HTA could lead to effective and efficient decision-making throughout a technology’s lifecycle, help breakdown system silos, and offer a platform for greater consideration of non-drug alternatives and upstream interventions. The impact of this approach was anticipated to contribute to a proactive health system that could improve population health, enhance the patient experience, and ensure appropriate stewardship of health care resources.

A disease management approach to HTA has international relevance as an approach that could promote integrated, proactive, sustainable, and resilient health systems.

Assessments self-administered via smart phone-based apps can gather patient wellness data; however, data regarding app utilization in clinical trials are limited. This study evaluated engagement with a self-administered wellness app as a supplemental tool to assess quality-of-life (QoL) in study BGB-3111-215 (NCT04116437), which included patients with B-cell malignancies treated daily with oral zanubrutinib.

This exploratory analysis evaluated the feasibility of using a voluntary device-based, self-administered activity and QoL questionnaire app (Medable, California, USA) in clinical trial patients. Feasibility was measured by rates of consent, utilization, and compliance. Consent was assessed by the percentage of patients who agreed to use the app. Utilization was assessed by the percentage of patients who engaged with the app. Compliance was assessed by actual versus scheduled engagements. The app also included passive activity tracking and a six-minute walk test.

As of 1 September 2022, 78 patients had enrolled in the study (median age 71 years) and 20 had consented to participate (26% consent rate). Eleven patients (median age 65 years) engaged with the app at least once (14% utilization rate; n=11 for the questionnaire; n=7 for the walk test). Engagement occurred a median two times (range 1 to 17) for the questionnaire and a median seven times (range 1 to 15) for the walk test. Four patients engaged with the questionnaire and six patients engaged in the walk test beyond week 12. Passive activity was collected for eight patients beyond week 12. Among patients who engaged with the QoL questionnaire and walk test, compliance rates were 18 and 21 percent, respectively.

This initial study showed that a subset of patients was willing to participate in the self-administered QoL questionnaire and activity tracker. Further comparison of app results and clinical trial findings are ongoing to explore factors affecting utilization and compliance rates.

Digital health care is a rapidly growing and increasingly diverse area of medicine and social care. Health technology assessment (HTA) agencies around the world are engaging with digital health technologies and developing methods for appropriately assessing their value. Given our experience in HTA for pharmaceuticals and how methodologies must differ when assessing medical devices and diagnostics, we must be proactive in rapidly developing useful methods for assessing a variety of digital health technologies.

We will first present the results of two completed HTAs from the UK and contrast their methods and results, while looking at the UK approach to assessing digital health care in general. Next, we will present preliminary results from an ongoing consensus conference study of HTA’s for integrated digital technologies. This study will use a modified Delphi methodology to develop statements for discussion at a consensus conference. The primary research question is “how digital technologies can help build resilient healthcare systems and how HTAs can assess their value”.

An HTA conducted in the UK of an artificial intelligence-enabled ambulatory electrocardiogram device resulted in a recommendation for further real-world evidence generation. Another HTA of a digital therapeutic app for insomnia, which was also conducted in the UK, resulted in a positive recommendation but the process was lengthy and needed to be flexible. The preliminary results of an upcoming consensus conference study will also be presented. The consensus panel will consist of a range of stakeholders from digital experts, HTA practitioners, clinicians, health economists, industry representatives, and policy makers.

Digital technology is now a broad field with a wide range of very different technologies included in its definition. Questions remain as to whether digital technologies should be assessed using a traditional pharma-based framework for HTA, a MedTech model, or something else entirely. Given our past experience of developing flexible methods for HTA, we have an opportunity to develop useful recommendations and best practices from the ground up.

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.

A 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.

Of 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.

When 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.

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.

Discussions were recorded during the event and a thematic analysis was performed.

The 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.

It 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.

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).

The 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.

Program 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.

Our 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.

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.

A 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.

The 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.

Over 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.

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.

A 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.

Over 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.

This 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.

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.

Case 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.

The 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.

FDG-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.

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.

A 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.

The 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.

Cost 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.

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.

A 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.

Preliminary 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.

For 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.

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.

An 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.

The 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).

New 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.

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.

A 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.

The 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.

The 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.