bluuebunny/crossref_metadata_2025_split huggingface dataset:
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

IntroductionThe 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.MethodsThis 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.ResultsThis 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.ConclusionsThis 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

IntroductionThere 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.MethodsWe 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.ResultsOf 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.ConclusionsOur 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

IntroductionPatient 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.MethodsWe 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.ResultsThe 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.ConclusionsWe 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

IntroductionClinicians 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.MethodsA 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.ResultsThe 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%).ConclusionsThe 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

IntroductionDigital 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.MethodsWe 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”.ResultsAn 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.ConclusionsDigital 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

IntroductionWith 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
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

IntroductionIn 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.
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

IntroductionCardiovascular 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.
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

IntroductionIn 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
Authors: Avdeyev Andrey , Gulyaev Alexander 
Published: December 2023

IntroductionMedical 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.