bluuebunny/crossref_metadata_2025 huggingface dataset:
OP33 Child And Adolescent Mental Health Care Models: A Scoping Review
Authors: Zechmeister-Koss Ingrid , Jeindl Reinhard , Hofer Viktoria
Published: December 2023

Introduction

Mental illnesses are among the most common health problems in children and adolescents worldwide, and their prevalence has recently increased. At the same time, many countries face gaps in care and a shortage of services. To address these challenges, countries are developing child and adolescent mental health (CAMH) strategies and adopting their models of care. This paper aimed to give an international overview of these strategies and care models to support decision makers and stimulate mutual learning and improved CAMH care.

Methods

We identified core topics within published CAMH strategies and care model documents from seven selected countries within the Global North, which represented different healthcare systems, geographical regions, and public health traditions to maximize variety. We systematically extracted data on the identified topics and summarized them narratively by applying qualitative content analyses.

Results

The documents addressed the following core topics: awareness raising activities; prevention and promotion; detection; treatment; telemedicine; care pathways; transitional psychiatry; vulnerable patient groups; user participation; infrastructure; workforce development; implementation; digital tools for case management; and data acquisition and research. A stand-alone CAMH strategy exists in most countries.

Recommendations on CAMH care often followed a public mental health approach and placed a high priority on mental health promotion and cross-sectional organization and funding of CAMH care services. Key principles of future CAMH care included: increased flexibility of care settings; early intervention; an open and non-judgmental attitude among staff; and strengths orientation instead of focusing on deficits and diagnoses.

Conclusions

Reducing the prevalence of mental illness and current shortcomings in care requires action at the policy level (e.g., developing a CAMH strategy with a focus on mental health promotion and installing cross-sectoral governance), organizational level (e.g., reorganizing treatment settings and pathways of care), and individual level (e.g., strengthening user involvement and workforce development). Applying the recommended approaches in other countries will likely require redesign, ideally with a participatory approach and evaluation alongside piloting.
OP34 Application Of A Case-Mix Analysis In COVID-19 Management At A University Hospital In Malaysia
Authors: Azzeri Amirah , Jaafar Mohd Hafiz , Dahlui Maznah
Published: December 2023

Introduction

The COVID-19 pandemic significantly affected healthcare systems. The most immediate effect was the increased demand for healthcare resources. This study aimed to conduct a case-mix analysis at one of the teaching hospitals in Malaysia to understand the economic implications of the pandemic.

Methods

Admissions related to COVID-19, either as a primary or secondary diagnosis, were extracted and given ICD-10 codes for diagnosis and ICD9-CM codes for procedures. The combined ICD-10 and ICD9-CM codes were imported into a case-mix grouper to generate the case-mix codes. The codes used for COVID-19 were A-4-13-I, A-4-13-II, and A-4-13-III for mild, moderate, and severe disease, respectively. Clinical pathways were collected and healthcare resource utilization was estimated by combining top-down and bottom-up costing approaches. Discounting and inflation were based on guidelines and official rates. The cost data were reported in US dollars (price year 2021).

Results

A total of 4,889 patients with a COVID-19 diagnosis were admitted to the hospital in 2021. Of these, 4,813 patients (98%) had a primary diagnosis of COVID-19. The remaining 76 patients (2%) were admitted for other medical reasons but were found to be positive for SARS-Cov-2 during admission. Therefore, for these patients, infection with the virus was considered a secondary diagnosis during the treatment episode.

Among the 4,813 patients, 3,909 (81%) were admitted with mild COVID-19 (A-4-13-I), 630 (13%) had moderate COVID-19 (A-4-13-II), and 274 (6%) had severe disease (A-4-13-III). More than half (56%) of the patients with a secondary diagnosis of COVID-19 were admitted for elective procedures. The average length of hospital stay (LOS) for mild disease was 9 days, with cumulative hospital costs of USD1,543. The average LOS for both moderate and severe disease was 21 days, and the cumulative hospital costs were USD23,527 and USD26,731 respectively. The total costs incurred for COVID-19 were estimated at USD19,259,153.

Conclusions

COVID-19 has considerable economic implications. This study provided information as part of a health technology assessment in the hospital to inform evidence-based healthcare decisions.
OP46 The Decision Uncertainty Toolkit: Risk Measures And Visual Outputs To Support Health Technology Decision-making During Public Health Crises
Authors: Kirwin Erin , McCabe Chris , Round Jeff
Published: December 2023

Introduction

During public health crises such as the COVID-19 pandemic, decision-makers have relied on infectious disease models to predict and estimate the impact of various health technologies. The difficulties associated with capturing and representing uncertainty using infectious disease models leads to a high risk of making decisions that are misaligned to policy objectives. Even when uncertainty is adequately captured in the analysis, the tools for communicating the risks and harms of making wrong decisions have proved inadequate, which can lead to the suboptimal adoption of critical health technologies including vaccines and antivirals. We aim to adapt and extend health economic methods for the characterization, estimation, and communication of uncertainty to infectious disease modeling.

Methods

Economic and infectious disease models share many features, including the comparison of policy alternatives on outcomes important to decision-makers (such as hospital census, total infections), but each takes a different approach to analysis of uncertainty. We extend best practices from health economics to infectious disease modeling and develop a suite of tools and visualization techniques which represent parameter uncertainty and the risk these unknowns present to decision-makers.

Results

In consultation with decision-makers and infectious disease modeling experts we developed the ‘Decision Uncertainty Toolkit’ of model outputs and visuals. Visual tools for uncertainty are developed to: (i) accurately capture uncertainty in key infectious disease model outputs, and (ii) support intuitive and direct interpretation by infectious disease modelers and decision-makers. We also developed quantitative measures for the downside risk of policy alternatives, specified to capture both the probability and magnitude of losses relative to policy targets for a range of infectious disease model outputs. Together, these outputs can support decision-making by quantifying outcome uncertainty and the risks associated with policy alternatives.

Conclusions

We developed the toolkit visuals and risk measures alongside infectious disease modelers and decision makers. The toolkit is designed to improve decision-maker understanding of decision risk in order to improve outcomes during future public health crises.
OP55 Did It Matter? Developing A Common Framework For Characterizing Impact Of Patient Involvement In Health Technology Assessment
Authors: Gousset Veronica Lopez , Silva Aline Silveira , Holtorf Anke-Peggy , Toledo-Chávarri Ana , Single Ann
Published: December 2023

Introduction

While the impact of health technology assessments (HTAs) is often not evaluated, some HTA bodies measure the impact of patient involvement in their processes. Evaluating how patient involvement is perceived by all stakeholders may help to improve practices and avoid activities that unduly burden patient and HTA communities. Frameworks and tools have been designed to analyze the impact of patient engagement along the healthcare development lifecycle. Reporting on the impact of patient involvement in HTA-specific activities, however, continues to be infrequent, unstandardized, and not comprehensive.

Having a common framework to characterize and report on the impact of patient involvement may enable this practice to be optimized and harmonized across HTA contexts.

Methods

The Patient and Citizen Involvement Interest Group (PCIG) within Health Technology Assessment International set out to contextualize this impact and support reporting. A questionnaire was developed, piloted, and rolled out to collect multistakeholder personal perceptions of the impact of patient involvement in individual HTAs. Questions included: “What difference did you feel patient involvement made in the HTA activity?” and “What would have been missed without patient involvement?”. From January 2019 to September 2021, 24 responses (including one joint submission) were collected through the PCIG’s network from HTA bodies (11), patient representatives (12), and industry representatives (2) from North America (5), South America (3), Europe (13), and Australia (3).

Results

Common themes were extracted from these experiences to characterize the impact of patient participation in HTA processes. Based on these commonalities, a harmonized framework consisting of three “domains” is proposed: impact on the decision-making process; impact on patient stakeholders; and impact on the HTA body. The framework includes a set of items under each domain to support reporting.

Conclusions

By having common language and measures, the HTA community can harmonize processes across jurisdictions to evaluate and communicate the value of patient involvement in HTA. Improving consistent reporting may facilitate more efficient process improvement for meaningful integration of patient stakeholders into HTA decision-making.
OP57 Consumer Engagement In Health Technology Assessment: An Australian Journey To Date
Authors: Watson Jo , Wortley Sally
Published: December 2023

Introduction

Australian health technology assessment (HTA) committees and processes at the national level have needed to consider an increasing focus on patient involvement and interactions within their scope. As various approaches have been explored, the visibility and impacts of patient involvement and consumer representation has evolved markedly over the most recent five years.

Methods

Program activities were developed from review of contemporary HTA models of practice across various HTA sectors internationally alongside input from individual patients and patient groups. Practical application of program pilots was influenced by specific requirements of the Office of HTA, the coordination of HTA Committee procedures and timelines, and involvement of HTA Committee consumer members.

Results

The development of capacity building programs for HTA consumer committee members, the growth of external activity and communications targeting patient groups and networks, and the establishment of an expert position to develop the HTA Consumer Evidence and Engagement Unit within the Department’s Office of HTA, are all examples of the work delivered in this period to better integrate patient evidence and inputs into HTA processes and decision-making. Activities over the next period will seek to establish formal frameworks for meaningful involvement that can demonstrate these contributions to evaluations and decision-making.

Conclusions

Various examples of patient involvement and participation in processes have evolved over time. The scope and impacts of these experiences have illustrated important participatory demands and resourcing needs, as well potential benefits for deliberations. The Australian Government and Departmental frameworks for HTA currently seek to enhance the development of patient evidence inputs and participation. These developments will be informed by the Australian context and comprehensive consultation with Australian patient groups and their networks. Strategies for improvements are anticipated to be relevant across all HTA processes and Committees within HTA areas.
OP58 Challenges In Maintaining Up To Date Health Technology Assessments in Rare Diseases: Lessons From Fabry Disease In Australia
Authors: Nicholls Kathleen , Denaro Charles , Tchan Michel , Ellaway Carolyn , Bratkovic Drago , Palmer Hazel , Campbell Sheridan , Fookes Megan , Thomas Mark
Published: December 2023

Introduction

Fabry disease is a rare, inherited X-linked lysosomal storage disease characterized by a wide spectrum of heterogeneously progressive clinical phenotypes, and which results in progressive kidney disease, cardiomyopathy, cerebrovascular disease, and reduced life expectancy. Disease-specific therapy aims to improve symptoms, stabilize current disease and delay progression. In Australia treatment access requires that patients meet pre-specified criteria, which have been in place for more than 15 years. Patient questions prompted the patient organization, Fabry Australia, to investigate why these criteria had remained unchanged despite significant progress in the understanding and management of Fabry disease.

Methods

A panel comprising two members of Fabry Australia and its Medical Advisory Committee conducted a review of the literature. The aim of this was to inform the clinical quality of the Australian treatment access criteria with reference to international guidelines and contemporary data. The findings from the literature were applied to develop consensus recommendations for classification and Fabry-specific treatment initiation criteria in diagnosed patients.

Results

Evidence supports earlier treatment with reduced barriers to access in some circumstances. Australian access criteria are misaligned with this. They do not distinguish between classical and non-classical Fabry phenotypes, neglect the impact of quality of life and gastrointestinal symptoms, and impose symptom-severity related criteria, which may lead to unnecessary treatment initiation delay. An updated framework is presented. It differentiates phenotypes, facilitates more timely access to Fabry-specific treatment for classical males, and supports relevant organ involvement criteria in classical females and patients with non-classical disease.

Conclusions

A well-performing health technology assessment system facilitates patient access to cost-effective treatments that improve health outcomes. Timely treatment initiation is important to avoid irreversible organ damage in Fabry patients. Patients’ questions about out-dated access criteria has prompted research and uncovered barriers that are no longer clinically valid. The perspectives of the patient as a stakeholder in their disease management should not be overlooked when assessing the value of health technologies in the rare disease setting.
OP60 Community Funding Prioritization Of Drug Treatments: A Discrete Choice Experiment
Authors: Fifer Simon , West Bronwyn , Kong Samson
Published: December 2023

Introduction

Allocating government resources for drug treatments continues to be a challenge in health care, particularly given the increasing number of high-cost personalized drug treatments, finite resources, and aging population. Since taxpayers fund government health budgets, it is important to understand how they think funding should be distributed, considering attributes such as cost of drug treatments, risk of dying, commonality of medical conditions, and quality of life changes with drug treatments. The aim of this study was to understand what attributes of a medical condition and treatment determine a community’s willingness to fund new drug treatments. Two decision-making contexts were explored: the allocation of funds from a health budget and a willingness-to-pay (WTP) perspective.

Methods

A representative sample of 500 Australian adults completed an online survey. The survey comprised two discrete choice experiments (DCEs) with different framing: an allocation of government funds and a WTP for drug treatments. The government allocation DCE allowed a choice between two hypothetical alternatives, each describing the medical condition and the drug treatment, while the WTP for funding showed one hypothetical alternative with an option to not fund the drug treatment. Seven DCE attributes, informed by a literature review, were displayed in each choice set relating to the medical condition (risk of dying, prevalence, and ages affected) and to the drug treatment (change in quality of life, additional life-years, availability of other drug treatments, and cost to the government and the taxpayer).

Results

The resulting DCE model will establish the funding prioritization choices made by the general community. We expect changes in quality of life and risk of dying to be very important attributes. Of key interest for this study is how the difference in the decision-making context impacts preferences. We expect taxpayers to employ a stricter rule set in funding decisions when paying out of pocket (WTP exercise).

Conclusions

The findings from this research have implications for decision makers when aligning funding decisions with community preferences and values.
OP63 Incorporating Machine Learning Methods In Health Economic Evaluations: A Case Study On Depression Prevention
Authors: Lokkerbol Joran
Published: December 2023

Introduction

New methodologies such as machine learning are becoming widely available and are increasingly used. However, more guidance on their role in the context of economic evaluations would be beneficial.

Methods

We developed a machine learning model to predict recurrent depressive episodes and incorporated the model outcomes in a health economic model to assess the cost effectiveness of offering targeted prevention of recurrent depression. We considered the impact on cost effectiveness (defined as cost per quality-adjusted life-year) for machine learning models with different thresholds for classifying a patient as being at risk, resulting in different precision-recall pairs.

Results

Targeted prevention of recurrent depression could enhance the cost effectiveness of depression treatment by preventing a small number of recurrent depressive episodes in patients where the estimated risk of recurrence is relatively high. More depressive episodes could be prevented with the trade-off of less cost effectiveness for the healthcare system.

Conclusions

Health economic modeling approaches can be augmented with machine learning methods, which broadens the areas in which evidence can be generated for policy makers to base their budget allocation. The precision of such predictive machine learning models must be high enough to be able to improve a care-as-usual healthcare system. Machine learning models generally let you set the level of precision acquired, at the cost of a possibly low recall, thereby limiting the impact on the healthcare system as a whole. More and better data for training these machine learning models will allow developed models to better distinguish patients who will and won’t develop a recurrent depressive episode, and for higher recall given a desired precision threshold. This will translate into a more substantial improvement in the treatment of depressive disorders in the healthcare system.
OP74 Analysis Of Literature And Research Foci In Overdiagnosis Based On Citespace
Authors: Yan Juntao , Wei Yan , Yang Yi , Liu Shimeng , Chen Yingyao
Published: December 2023

Introduction

With the rapid development of innovative health technologies, evidence increasingly shows that overdiagnosis is harmful to a person’s health and that it is a global public health issue. This study aimed to analyze the current research status and corresponding foci in the field of overdiagnosis in Chinese and English databases using bibliometric methods.

Methods

A search was conducted in the English Web of Science Core Collection database and the Chinese China National Knowledge Infrastructure database for literature published from inception to 31 December 2021. CiteSpace (version 5.8 R1) software was used to perform bibliometric analysis on the countries, institutions, and keyword clusters of the included literature on overdiagnosis and to draw a corresponding visual knowledge map.

Results

A total of 2,841 English and 43 Chinese publications were included. There was an increasing trend in the annual publication volume of both Chinese and English literature, with the publication volume of English research increasing significantly since 2010. In terms of countries and institutions, the top ten in English research on overdiagnosis were all from high income countries. The cooperation among these countries and institutions was close, unlike in China where the cooperation was relatively limited. Analysis of keyword clustering showed that the potential research foci for English literature on overdiagnosis included breast cancer, thyroid cancer, prostate cancer, lung cancer, and other tumor types, whereas the clustering in Chinese records was relatively scattered and mainly focused on overdiagnosis of thyroid cancer.

Conclusions

The research topics in the Chinese literature on overdiagnosis lag significantly behind English research. It is suggested that more research on overdiagnosis and related fields should be actively promoted and conducted in China in the future.
OP83 Diagnostic Accuracy And Cost Effectiveness Of Automated Ankle Brachial Pressure Index Measurement For Peripheral Arterial Disease In People With Leg Ulceration
Authors: Boyers Dwayne , Cruickshank Moira , Aucott Lorna , Kennedy Charlotte , Manson Paul , Bachoo Paul , Brazzelli Miriam
Published: December 2023

Introduction

Leg ulcers are usually treated with compression therapy unless there is evidence of peripheral arterial disease (PAD). Compression may lead to vascular complications in people with PAD. Timely diagnosis and appropriate treatment are important to ensure the best patient outcomes. PAD is usually assessed using a manual ankle brachial pressure index (ABPI). Automated devices may reduce time to diagnosis and treatment of leg ulcers. This study investigated the diagnostic accuracy, clinical effectiveness, and cost effectiveness of automated ABPI measurement, compared with manual doppler testing, to detect PAD in people with leg ulcers.

Methods

We conducted a systematic review of studies of any design assessing automated devices in any population. Meta-analyses were conducted where possible. A decision tree and Markov model was used to capture lifetime costs, quality-adjusted life-years (QALYs), and cost effectiveness from a UK National Health Service perspective. Sensitivity analyses captured the uncertainty surrounding model assumptions and probabilistic sensitivity analyses described parameter uncertainty. Value of information analysis was conducted to identify future research priorities.

Results

A total of 22 studies provided diagnostic accuracy data for five automated devices, but there were no studies in people with leg ulcers. Meta-analysis of 12 studies demonstrated a pooled sensitivity of 64 percent (95% confidence interval [CI]: 57, 71) and a specificity of 96 percent (95% CI: 92, 98) for detecting PAD. Automated devices were cheaper to complete due to shorter test times, but the increased risk of inappropriate or delayed treatment due to inaccurate test results offset the cost savings and reduced the QALYs for automated devices.

Conclusions

Given the current limited evidence base, automated devices would only be cost effective if they can demonstrate substantial reductions in time to diagnosis in clinical practice. Value of information analysis identified the following research priorities: (i) to determine diagnostic accuracy in a population with leg ulcers; (ii) to determine the impact of test results on ulcer healing times, and (iii) to determine the risk of providing inappropriate treatment based on inaccurate test results.
OP87 A Roadmap for Increasing The Usefulness And Impact Of Patient Preference Studies In Health Technology Assessment (HTA)
Authors: Marshall Deborah A , de Bekker-Grob Esther W , Hauber Brett , Heidenreich Sebastian , Janssen Ellen , Bast Alice , Hanmer Janel , Johnson Laura Lee , Danyliv Andriy , Low Eric , Bouvy Jacoline C , Bridges John F. P.
Published: December 2023

Introduction

The number of patient preference studies in health has increased dramatically. There is growing use of patient preferences in a wide variety of contexts, including health technology assessment. Patient preference studies can help inform decision makers on the needs and priorities of patients and the tradeoffs they are willing to make about health technologies.

Methods

This International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Task Force included international experts, health preference researchers and others from diverse backgrounds, including regulatory, health technology assessment, medicine, patient advocacy, and the pharmaceutical industry. The report underwent two rounds of written reviews by ISPOR Preferences Special Interest Group members until a final consensus was reached. The Task Force focused on developing a roadmap that would: (i) apply to the wide variety of preference methods, (ii) identify key domains to guide researchers and other stakeholders in making patient preference studies more useful to decision makers, and (iii) detail important questions to guide researchers conducting preference studies and those critically appraising them.

Results

This Task Force report provides a novel roadmap that invites patient-preference researchers to work with decision makers, patients and other stakeholders to do even more to ensure that studies are useful and impactful. The ISPOR Roadmap consists of five key elements: (i) Context; (ii) Purpose; (iii) Population; (iv) Method; and (v) Impact. In this report, we define these five elements and provide good practices on how patient-preference researchers can actively contribute to increasing the usefulness and impact of patient preference studies in decision-making. We also present a set of key questions that can support researchers and other stakeholders in assessing efforts that promote preference studies’ intended and unintended impact.

Conclusions

This roadmap can help increase the usefulness and impact of patient preference studies in decision-making by challenging researchers to engage and partner with decision makers, patients and others, and together consider the intended and unintended impacts of patient preference studies on decision-making while actively fostering positive impact.
OP92 The Hidden Burden Of Patients And Families In Rare Diseases: A Scoping Review Of Economic Evaluations
Authors: Currie Gillian , Gerber Brittany , Lorenzetti Diane , MacDonald Karen , Bohach Riley Jewel , Marshall Deborah
Published: December 2023

Introduction

There are more than 7,000 rare diseases (RDs), which are individually rare but have a large collective impact on patients and families, the health system, and society. There are few treatments for RD; where treatments do exist, they are often exceptionally expensive. Understanding the socioeconomic burden (SEB) of RD is crucial to properly valuing these treatments and informing health technology assessment. Our team has developed a framework of cost elements for inclusion in studies of the costs of RDs using an evidence-informed consensus-based approach.

Methods

We conducted a scoping review to identify published economic evaluations studies in RD, searching five electronic databases to identify English language RD studies published 2010-2021. We applied our framework of cost elements to assess studies regarding what cost elements were included.

Results

Of 4,890 records identified, 48 studies were screened for inclusion. Most were from the US (n=27), UK (n=6), and Canada (n=6), and focused on hemophilia (n=14) or cystic fibrosis (n=11). Healthcare system and payer perspectives were most often reported (n=41), with only seven studies reporting a societal perspective. Cost elements most often included were medications (n=41), hospitalizations (n=35), surgery (n=20) medical tests (n=16), and outpatient (unspecified) visits (n=16). Costs to patients, families, and society were less commonly included: productivity (n=5), travel/accommodation (n=3), government benefits (n=2), family impacts (n=0), or other costs relevant to RD (n=1). While unsurprising, given that most analyses focused on healthcare or payer perspectives, this finding illustrates the extent to which the burden of RD is largely unstudied.

Conclusions

Our scoping review demonstrated that most studies are conducted from a healthcare system/payer perspective, and largely consider only medical costs. These studies undercount the hidden burden of rare disease borne by patients and families leading to a gap in our global understanding of the full impact of rare diseases on families. To properly account for the these and value emerging treatments for RD, patient- and family-borne costs must be considered in economic evaluations for health technology assessment.
OP98 Improving Patient Involvement In Health Technology Assessments: Is It Enough To Train Just The Patients?
Authors: Livingstone Heidi , Fitzpatrick Ella , Laura Marsden , Tonkinson Mandy , Taylor Sally
Published: December 2023

Introduction

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.

Methods

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.

Results

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.

Conclusions

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.
OP103 What To Include In A Health Technology Assessment Of Artificial Intelligence-Based Technologies: Results Of A Delphi Expert Survey
Authors: Daugbjerg Signe , Di Bidino Rossella , Cicchetti Americo
Published: December 2023

Introduction

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.

Methods

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.

Results

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%).

Conclusions

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.
OP114 Health Technology Assessment Of Digital Technologies: Results From Two Past Examples And Thoughts For The Future
Authors: Erskine Jamie
Published: December 2023

Introduction

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.

Methods

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”.

Results

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.

Conclusions

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.
OP116 Building Alignment Between Industry, Academia, And Patients On Health Technology Assessment Methods
Authors: Taylor Colman
Published: December 2023

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.

Methods

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

Results

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.

Conclusions

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.
OP119 Collectively Improving The Quality Use Of Highly Specialized Medicines: Starting With Biologics
Authors: Dartnell Jonathan , Brown Sue , Hardy Louise , Barrett Claire , Buchbinder Rachelle , Whittle Sam , Pulver Lisa , Nikolic Nena , Rowett Debra , Hayward Kylee , Hill Catherine
Published: December 2023

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).

Methods

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.

Results

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.

Conclusions

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.
OP121 Cost-Utility Analysis Of A Supervised Exercise Intervention For Women With Early-Stage Endometrial Cancer
Authors: Wang Yufan , McCarthy Alexandra L. , Tuffaha Haitham
Published: December 2023

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.

Methods

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.

Results

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.

Conclusions

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.
OP125 How Can Health Technology Assessment Evolve To Better Consider Benefits For Patients, Their Families, And Carers?
Authors: Gillespie John , Davies Paul
Published: December 2023

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.

Methods

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.

Results

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.

Conclusions

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.
OP126 Clinical And Economic Evaluation Of The Effectiveness Of Cerebrolysin® In Neurological Patients With Post-Stroke Complications In Kazakhstan
Authors: Avdeyev Andrey , Gulyaev Alexander
Published: December 2023

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.

Methods

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.

Results

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).

Conclusions

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.