Oral Presentations
OP83 Diagnostic Accuracy And Cost Effectiveness Of Automated Ankle Brachial Pressure Index Measurement For Peripheral Arterial Disease In People With Leg Ulceration
- Dwayne Boyers, Moira Cruickshank, Lorna Aucott, Charlotte Kennedy, Paul Manson, Paul Bachoo, Miriam Brazzelli
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- 14 December 2023, p. S23
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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.
MethodsWe 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.
ResultsA 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.
ConclusionsGiven 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.
Commentary
Patient and citizen participation at the organizational level in health technology assessment: an exploratory study in five jurisdictions
- Hervé Nabarette, Marie-Hélène Chastenay, Jean-Claude K. Dupont, Isabelle Ganache, Ann N.V. Single
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- Published online by Cambridge University Press:
- 08 August 2023, e51
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Objective
While patient participation in individual health technology assessments (HTAs) has been frequently described in the literature, patient and citizen participation at the organizational level is less described and may be less understood and practiced in HTA bodies. We aimed to better understand its use by describing current practice.
MethodTo elicit descriptive case studies and insights we conducted semi-structured interviews and open-ended questionnaires with HTA body staff and patients and citizens participating at the organizational level in Belgium, France, Quebec, Scotland, and Wales.
ResultsWe identified examples of organizational participation in managerial aspects: governance, defining patient involvement processes, evaluation processes and methods, and capacity building. Mechanisms included consultation, collaboration, and membership of standing (permanent) groups. These were sometimes combined. Participants were usually from umbrella patient organizations and patient associations, as well as individual patients and citizens.
DiscussionAlthough the concept, participation at the organizational level, is not well-established, we observed a trend toward growth in each jurisdiction. Some goals were shared for this participation, but HTA bodies focused more on instrumental goals, especially improving participation in HTAs, while patients and citizens were more likely to offer democratic and developmental goals beyond improving participation processes.
ConclusionOur findings provide rationales for organizational-level participation from the perspectives of HTA bodies and patients. The case studies provide insights into how to involve participants and who may be seen as legitimate participants. These findings may be useful to HTA bodies, the patient sector, and communities when devising an organizational-level participation framework.
Assessment
National health technology assessment in Turkiye after a decade: are key principles followed?
- Tuba Saygın Avşar, Hasan Hüseyin Yıldırım
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- 24 July 2023, e52
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Background
Health technology assessment (HTA) is growing in low- and middle-income countries (LMICs) to ensure optimal use of limited resources. However, the impact of HTAs on decision making in LMICs has been limited. The study aimed to provide an overview of Turkiye’s progress since establishing the first HTA agency in 2012.
MethodsThe web sites of three national HTA agencies in Turkiye were searched for HTA guidelines and national HTA reports. The HTA guidelines were assessed by two researchers independently against the key principles of HTA developed by Drummond et al., and the HTA reports against the national guidelines.
ResultsThe study included one HTA guideline and eight national HTA reports. The guideline included very limited technical guidance. Compliance with the principles was poor to moderate, and significant methodological limitations were identified. The reports were inconsistent regarding the scope and the HTA assessment criteria. The link between HTA findings, HTA decision making, and health policies were not clear.
DiscussionThe inconsistencies between the reports and the methodological limitations demonstrate the need for national HTA guidelines. Improving the characteristics of the HTA might impact implementation. Among the key issues is transparency regarding priority setting, the HTA process, and decision making.
ConclusionEstablishing and adopting national HTA guidelines at international standards is needed. Involving external scientific committees and health economists in the HTA processes might help ensure that the key principles of HTA are followed. The study findings might be helpful for countries that are developing their HTA systems.
Oral Presentations
OP85 Cost Effectiveness Of Prednisolone To Treat Bell’s Palsy In Children: An Economic Evaluation Alongside A Randomized Controlled Trial
- Xiuqin Xiong, Li Huang, David Herd, Meredith Borland, Andrew Davidson, Stephen Hearps, Mark Mackay, Katherine Lee, Stuart Dalziel, Kim Dalziel, John Cheek, Franz Babl
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- 14 December 2023, p. S23
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Introduction
The cost effectiveness of treating Bell’s palsy with prednisolone in children is unknown. This study aimed to assess the cost effectiveness of prednisolone, compared with placebo, in treating Bell’s Palsy in children from a healthcare sector perspective.
MethodsThis economic evaluation was a prospectively planned secondary analysis of a triple-blind randomized superiority trial conducted from 2015 to 2020 that compared prednisolone with placebo. The time horizon was six months after randomization. The 180 participants were aged from six months to 17 years and presented within 72 hours of onset of clinician diagnosed Bell’s palsy. Interventions were oral prednisolone (1 mg per kg daily) or taste-matched placebo administered for ten days. Incremental cost-effectiveness ratios comparing prednisolone with placebo were estimated. Costs included medication costs, doctor visits, and medical tests over the six-month study period. Effectiveness was measured using quality-adjusted life-years (QALYs) derived from the Child Health Utility 9D instrument. Nonparametric bootstrapping was performed to capture uncertainties. Prespecified subgroup analyses by age (12 to 17 years versus <12 years) were performed.
ResultsThe mean cost per patient was USD188 in the prednisolone group and USD121 in the placebo group over the six-month period (difference USD66, 95% confidence interval [CI]: 47, 179). The mean QALYs gained over six months were 0.45 in the prednisolone group and 0.44 in the placebo group (difference 0.01, 95%CI: -0.01, 0.03). Prednisolone was very likely cost effective given a conventional willingness-to-pay threshold of USD 50,000 per QALY gained (the cost per additional QALY gained was USD6,625 using prednisolone compared with placebo). Subgroup analysis suggested that this was primarily driven by the high probability of prednisolone being cost effective in children aged 12 to 17 years (98%), compared with those younger than 12 years (51%).
ConclusionsThis study provides new evidence to stakeholders and policy makers who are considering whether to make prednisolone available for treating Bell’s palsy in children aged 12 to 17 years.
OP87 A Roadmap for Increasing The Usefulness And Impact Of Patient Preference Studies In Health Technology Assessment (HTA)
- Deborah A Marshall, Esther W de Bekker-Grob, Brett Hauber, Sebastian Heidenreich, Ellen Janssen, Alice Bast, Janel Hanmer, Laura Lee Johnson, Andriy Danyliv, Eric Low, Jacoline C Bouvy, John F. P. Bridges
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- Published online by Cambridge University Press:
- 14 December 2023, p. S24
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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.
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.
Assessment
Cost-effectiveness of adding ezetimibe and/or PCSK9 inhibitors to high-dose statins for secondary prevention of cardiovascular disease in Chinese adults
- Yuliang Xiang, Lei Gan, Heyue Du, Qiukui Hao, Bert Aertgeerts, Sheyu Li, Ming Hu
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- 31 August 2023, e53
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Objectives
The latest international guideline recommended the add-on therapy of ezetimibe and PCSK9 inhibitors in selected people for the secondary prevention of cardiovascular diseases (CVDs). However, it remains unclear whether these regimens fit the Chinese healthcare system economically.
MethodsBased on the Chinese context, this simulation study evaluated four therapeutic strategies including the high-dose statin-only group, ezetimibe plus statin group, PCSK9 inhibitors plus statin group, and PCSK9 inhibitors plus ezetimibe plus statin group. The team developed a Markov model to estimate the incremental cost-effectiveness ratio (ICER). With each 1-yr cycle, the simulation subjects could have nonfatal cardiovascular events (stroke and/or myocardial infarction) or death (vascular or nonvascular death event) with a follow-up duration of 20 yr. Cardiovascular risk reduction was gathered from a network meta-analysis, and cost and utility data were gathered from hospital databases and published research.
ResultsFor Chinese adults receiving high-dose statins for secondary prevention of CVDs, the ICER was US$68,910 per quality-adjusted life year (QALY) for adding PCSK9 inhibitors, US$20,242 per QALY for adding ezetimibe, US$51,552 per QALY for adding both drugs. Given a threshold of US$37,655 (three times of Chinese GDP), the probability of cost-effectiveness is 2.9 percent for adding PCSK9 inhibitors, 53.1 percent for adding ezetimibe, and 16.8 percent for adding both drugs. To meet the cost-effectiveness, an acquisition price reduction of PCSK9 inhibitors of 33.6 percent is necessary.
ConclusionIn Chinese adults receiving high-dose statins for the secondary prevention of CVDs, adding ezetimibe is cost-effective compared to adding PCSK9 inhibitors and adding both drugs.
Oral Presentations
OP88 Translating Patient Reported Measure Score Into Specific Outcomes From The Patient Perspective–Example Using Health Assessment Questionnaire-Disability Index
- Chenglong Han, Marcie Strauss, Fareen Hassan, Sarah Alulis, Oluwakayode Adejoro, Adrian Griffin
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- 14 December 2023, p. S24
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Introduction
To facilitate communication of clinical study results among patients, clinicians, and payers, this post-hoc analysis examined the association between achieving the Health Assessment Questionnaire-Disability Index (HAQ-DI) clinical meaningful improvement (CMI) and normalization of individual daily activities using pooled clinical trial data of patients with moderate-severe psoriatic arthritis (PsA).
MethodsPatients with active PsA (n=1,120) who participated in two Phase III trials were administered the HAQ-DI at baseline and weeks (W) 24 and 52. The HAQ-DI is a patient-reported instrument measuring functional status that is validated for rheumatological diseases including PsA. It contains 20-items assessing activities of daily living using a 0-3 Likert scale [0=able to perform activity without difficulty (normalization) through to 3=unable to perform activity]; 0.35 or more improvement in total HAQ-DI represents the CMI in PsA patients. Study participants with baseline item-specific scores more than zero (indicating some level of impairment) who experienced both 0.35 or more improvement in total HAQ-DI and item-specific score equal to zero, were compared over time.
ResultsThe proportion of patients with compromised ability to perform individual activities at baseline ranged from 42.3 percent (Difficulty Turning Faucet) to 84.1 percent (Difficulty Doing Chores). Fourteen of 20 activities with 60 percent or more of patients reporting impairment at baseline decreased to one of 20 activities in patients reaching the CMI at W24. Fourteen of 20 activities with 50 percent or more of patients reporting no impairment at W24 increased to 18 of 20 activities at W52. Notably, the proportion of patients reporting impairment for the two outlier activities at W52 (Reach-Get Down 5lb Item/Doing Chores) decreased during the follow-up period by 28 percent and 30 percent, respectively, despite presenting as very challenging at baseline.
ConclusionsPsA patients achieving a clinically meaningful change in total HAQ-DI over time were more likely to achieve normalization of individual daily activities. Translating changes in patient-reported outcome scores to specific symptom or functional improvements are meaningful to help patients understand clinical trial results, as well as to communicate with payers the value of accessing treatment for specific health conditions.
Policy
Do France, Germany, and Italy agree on the added therapeutic value of medicines?
- Giorgio Casilli, Dario Lidonnici, Claudio Jommi, Marika De Nigris, Armando A. Genazzani
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- Published online by Cambridge University Press:
- 15 August 2023, e54
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Objectives
The Health Technology Assessment (HTA) of medicines is performed separately at the country level with some differences, but Italy, France, and Germany have implemented price and reimbursement systems strongly focused on the Added Therapeutic Value (ATV). This study investigates the level of agreement on ATV assessments by Agenzia Italiana del Farmaco (AIFA), Haute Autorité de Santé (HAS), and Gemeinsamer Bundesausschuss (G-BA).
MethodsA database was created collecting all information about drugs with innovativeness status requests in Italy from July 2017 to December 2022 and populated with the corresponding HAS and G-BA ATV assessments. The primary comparative analysis was conducted by grouping the ATV ratings into “higher added value” and “lower or no added value”, while a secondary analysis considered the Italian innovativeness status as a criterion to include the quality of evidence assessment. The concordance between ATV assessments was investigated through percentage agreement and unweighted Cohen k-value.
Results189 medicines/indications were included. The greatest agreement was found when comparing G-BA versus HAS (82 percent; k = 0.61, substantial agreement). Lower levels of agreements were observed for AIFA versus HAS and AIFA versus G-BA (respectively 52 percent; k = 0.17 and 57 percent; k = 0.25). The secondary analysis led to a reconciliation to moderate agreement for AIFA versus HAS (72 percent; k = 0.45) and AIFA versus G-BA (74 percent; k = 0.47).
ConclusionsA high degree of concordance between HTA organizations is reached when considering jointly ATV and quality of evidence, suggesting that the system is extensively mature to make a Joint Clinical Assessment, avoiding duplications and reducing access inequalities.
Oral Presentations
OP90 Multiple Myeloma: Developing A Benchmark Patient Experience Index In Australia And New Zealand
- Simon Fifer, Bronwyn West, Jenni Godsell, Nella Combe, Todd Stephenson
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- 14 December 2023, p. S25
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Introduction
Within Australia and New Zealand (ANZ) there is limited evidence regarding the experience and satisfaction across the healthcare system of people living with multiple myeloma (MM). We aimed to quantify the patient experience across the healthcare system to help identify potential areas of the healthcare pathway that could be targeted for improvement to maximize patient satisfaction.
MethodsA 30- to 40-minute online survey was completed by adults in ANZ diagnosed with MM. Anchored best-worst scaling (ABWS) is a technique that takes advantage of an individual’s ability to reliably identify extremes (‘best’ and ‘worst’) in sets of items, eliciting discriminating rankings free of scale bias. This study implemented a novel anchoring process to rescale importance and satisfaction best-worst scores for factors across the MM healthcare pathway, which could be compared and combined to form a patient experience index (PEI). There were 15 factors or ‘moments that matter’ (MTM), each describing a different aspect of the patient journey, such as time to diagnosis, treatment logistics, and side effects. The MTM were derived from qualitative research with patients as well as a workshop with key opinion leaders. Additional survey questions were included to help identify potential ways to improve patient satisfaction.
ResultsThe results were based on 62 patients with MM. The overall median PEI score was 63.1. The top three MTM that were most important to patients, but they were least satisfied with (calculated by combining the top four of each most important/least satisfied factor for each participant), were side effects of medication, effectiveness of medication, and medication access.
ConclusionsThe findings from this research contribute to the understanding of patient experiences of treatment and care for MM. The results can inform healthcare decisions for prioritizing interventions that align with patient experiences. In the future, the study could be executed longitudinally to assess shifts in satisfaction within the MM healthcare journey, which would be especially worthwhile if new programs are implemented to improve patient satisfaction.
Policy
An evaluation of managed access agreements in England based on stakeholder experience
- Caroline Farmer, Maxwell S. Barnish, Laura A. Trigg, Samuel Hayward, Naomi Shaw, Louise Crathorne, Thomas Strong, Brad Groves, John Spoors, G. J. Melendez Torres
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- 27 July 2023, e55
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Objectives
The objective of this research was to evaluate managed access policy in England, drawing upon the expertise of a range of stakeholders involved in its implementation.
MethodsSeven focus groups were conducted with payer and health technology assessment representatives, clinicians, and representatives from industry and patient/carer organizations within England. Transcripts were analyzed using framework analysis to identify stakeholders’ views on the successes and challenges of managed access policy.
ResultsStakeholders discussed the many aims of managed access within the National Health Service in England, and how competing aims had affected decision making. While stakeholders highlighted a number of priorities within eligibility criteria for managed access agreements (MAAs), stakeholders agreed that strict eligibility criteria would be challenging to implement due to the highly variable nature of innovative technologies and their indications. Participants highlighted challenges faced with implementing MAAs, including evidence generation, supporting patients during and after the end of MAAs, and agreeing and reinforcing contractual agreements with industry.
ConclusionsManaged access is one strategy that can be used by payers to resolve uncertainty for innovative technologies that present challenges for reimbursement and can also deliver earlier access to promising technologies for patients. However, participants cautioned that managed access is not a “silver bullet,” and there is a need for greater clarity about the aims of managed access and how these should be prioritized in decision making. Discussions between key stakeholders involved in managed access identified challenges with implementing MAAs and these experiences should be used to inform future managed access policy.
HTA capacity building in Asia: towards one goal
- Linda Mundy, Guy Maddern
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- 15 August 2023, e56
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Objectives
The aim of the 2022 Health Technology Assessment International (HTAi) Asia Policy Forum (APF) was to discuss experiences and challenges around health technology assessment (HTA) capacity building for both HTA agencies and companies in the Asia region and to identify possible solutions as part of a capacity building roadmap.
MethodsDiscussions during the 2022 APF, informed by a pre-meeting survey of HTA agencies and industry attendees from the region, form the basis of this paper.
ResultsHTA is an essential element of priority-setting in healthcare; however, the scarcity of skilled technical HTA practitioners is a rate-limiting step in the conduct of HTA. The lack of investment in HTA and the political will to mandate the use of HTA in decision-making may be due to a lack of understanding of the value of the HTA process, and how HTA is interpreted and used in the healthcare decision-making process.
ConclusionsIncreased demand for HTA is created when the value of HTA is recognized. HTA capacity-building challenges may be mitigated by educating stakeholders, particularly policymakers, on the value of, and the need to invest in, HTA as a transparent process to ensure equitable access to health care for all. Investigating a means of funding and implementing an HTA intern program between agencies, in partnership with industry, to facilitate a supportive environment to foster HTA skills and knowledge, build capacity or strengthen existing capacity should be a priority.
Oral Presentations
OP92 The Hidden Burden Of Patients And Families In Rare Diseases: A Scoping Review Of Economic Evaluations
- Gillian Currie, Brittany Gerber, Diane Lorenzetti, Karen MacDonald, Riley Jewel Bohach, Deborah Marshall
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- 14 December 2023, p. S25
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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.
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.
OP93 The Cost Effectiveness Of Antiretroviral Therapy Adherence Interventions In HIV/AIDS Patients: A Systematic Review Of Decision Analytic Models
- Ali Ahmed, Juman Dujaili, Long Le, Saval Khanal, Lay Hong Chuah
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- Published online by Cambridge University Press:
- 14 December 2023, pp. S25-S26
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Introduction
People living with HIV/AIDS (PLWHA) frequently struggle to maintain optimal adherence to antiretrovirals (ARVs). Different adherence-improving interventions have been developed and examined through decision analytic model-based health technology assessments. Therefore, we aimed to conduct a systematic review of all decision analytic models developed to improve adherence in PLWHA.
MethodsThe review protocol was registered on Prospero (CRD42022270039) and relevant studies published from inception to 23 October 2022 were identified through searches of the following databases: PubMed, Embase, the National Health Service Economic Evaluation Database, PsycINFO, the Health Economic Evaluations Database, and EconLit. Studies were included if they were modeling works of full economic evaluations, including cost-effectiveness analyses (CEA), cost-utility analyses, cost-benefit analyses, and cost-consequence analyses. The primary outcome was the cost effectiveness of adherence interventions reported as the incremental cost per additional quality-adjusted life-year (QALY). Study quality was assessed with the Quality of Health Economics Studies instrument. Due to the heterogeneity of the data, a permutation matrix was used for quantitative data synthesis rather than a meta-analysis.
ResultsThe 15 studies identified were conducted in North America (8/15), Africa (4/15), and Europe (3/15). The time horizon was one year in one study, ten years in one study, 20 years in three studies, and a lifetime horizon in ten studies. The types of interventions were smartphone-based (5/15), nurse involved (2/15), directly observed therapy (2/15), case manager involved (1/15), simplification of regimens (1/15), Link4Health (1/15), and others (3/15) that involved multicomponent intervention. The interventions gained higher QALYs with cost savings in all 15 studies and gained QALYs at a higher cost at an acceptable incremental cost-effectiveness ratio in 80 percent (12/15) of studies. The studies were of fair (13%) to high quality (87%).
ConclusionsThis study is the first systematic review of decision analytic model-based CEAs of adherence interventions in the management of PLWHA. Most of the identified studies recently published good quality cost-effectiveness analyses with an adequate timeframe.
Corrigendum
Emerging healthcare interventions: Patient-Centered Outcomes Research Institute’s programmatic initiative – CORRIGENDUM
- Gowri Raman, Meghana Vijaysimha, Emma Kopleff, Kelly Dunham, Greg Martin, Jean Slutsky, William Lawrence
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- Published online by Cambridge University Press:
- 07 September 2023, e57
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Oral Presentations
OP94 Economic Evidence On Hemodialysis Access Creation Procedures In Patients With End-Stage Kidney Disease: A Systematic Literature Review
- Ritu Gupta, Upasna Gaba, Christopher Delaney, George Papadopoulos
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- 14 December 2023, p. S26
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Introduction
It is important to create and maintain durable hemodialysis (HD) access in health systems to reduce morbidity and maintain overall cost control in patients with end-stage kidney disease (ESKD). To evaluate the choice of HD vascular access creation procedures and their related economic costs, we aimed to identify economic evaluations on vascular access (VA) creation procedures in patients with ESKD.
MethodsA systematic literature review was conducted using the Cochrane methodology to identify cost-effectiveness analyses (CEAs), budget impact analyses, and cost analyses of various HD access creation procedures. Eligible publications published from 2012 onwards were retrieved by searching PubMed, Embase, and the Cochrane Library. The Consolidated Health Economic Evaluation Reporting Standards 2022 checklist and ISPOR Task Force guidelines were used to appraise the quality of the economic evaluations and budget impact analyses, respectively. Costs were adjusted for inflation and purchasing power parity and standardized to US dollars.
ResultsA total of 40 economic evaluations met the inclusion criteria, including 28 cost analyses, three budget impact analyses, and nine CEAs. Widely evaluated procedures in the published literature were endovascular and surgical arteriovenous fistula (AVF), arteriovenous graft (AVG), and central venous catheterization (CVC). The results indicated that AVF was the most cost-effective strategy, followed by AVG, and CVC. Three studies showed that endovascular AVF was cost effective, compared with surgical AVF, and resulted in overall cost savings of about USD53 million dollars over a five-year period. Results of the quality assessment showed that budget impact analyses scored 63 percent, while the average score for economic evaluations was 58 percent.
ConclusionsIt was challenging to identify a single effective method of managing vascular access due to the substantial heterogeneity among VA creation techniques. However, most of the included economic evaluations showed that AVF was a cost-effective method of VA creation relative to other identified techniques for patients with ESKD on HD.
Policy
Hospital-based health technology assessment of innovative medical devices: insights from a nationwide survey in France
- Tess Martin, Alessandra Guercio, Hélène Besseau, Laure Huot, Pascale Guerre, Jamal Atfeh, Laurent Piazza, Judith Pineau, Brigitte Sabatier, Isabelle Borget, Nicolas Martelli
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- Published online by Cambridge University Press:
- 21 September 2023, e58
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Objectives
To better understand the process of hospital acquisition of innovative medical devices (MDs) and the hospital-based health technology assessment (HB-HTA) pathways in France, an in-depth study based on a quantitative approach is needed. The aim of the present study was to assess through a national survey how HB-HTA is currently implemented in French hospitals and to identify its level of formalization.
MethodsA quantitative online survey was conducted among hospitals performing HB-HTA in France, with a focus on the acquisition of innovative MDs for individual use. The survey, conducted between March and June 2022, was developed by a scientific board composed of members of the French-speaking Society for HB-HTA.
ResultsSixty-seven out of 131 surveyed hospitals with HB-HTA activities responded, including 29 university hospitals, 24 nonprofit private hospitals, and 14 local hospitals. Sixty-one respondents (91 percent) reported the existence of a process dedicated to evaluating innovative MDs; of these, 16 declared that their hospitals had a formalized unit with HB-HTA activity. These units were more frequently found in larger hospitals with more than 500 inpatient beds (n = 16, p = 0.0160) and in university hospitals (n = 12, p = 0.0158). No hospital reported any collaboration with HAS, the French national HTA agency.
ConclusionA diverse range of HB-HTA organizations with different structural levels exist in France for MD procurement linked to the category of hospitals. The study highlights the need for recognition of HB-HTA activity at the regulatory level in France and for direct collaboration between HTA activities performed at local and national levels.
Oral Presentations
OP95 A Systematic Review Of The Cost And Cost Effectiveness Of Immunoglobulin Treatment In Patients With Hematological Malignancies
- Sara Carrillo De Albornoz, Khai Li Chai, Alisa M. Higgins, Dennis Petrie, Erica M. Wood, Zoe K. McQuilten
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- Published online by Cambridge University Press:
- 14 December 2023, pp. S26-S27
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Introduction
Patients with hematological malignancies are likely to develop hypogammaglobulinemia (HGG) and subsequent infections. Immunoglobulin (Ig) replacement is commonly given to prevent infections, but the total costs and cost effectiveness of its use are unknown.
MethodsA systematic review was conducted following PRISMA guidelines to assess evidence on the costs and cost effectiveness of Ig replacement, administered intravenously (IVIg) or subcutaneously (SCIg), in adult patients with hematological malignancies. This review was registered with PROSPERO (CRD42022321908).
ResultsSix studies were included out of a total of 3,612 citations. A narrative synthesis was conducted because of the high level of heterogeneity across the included studies. Two economic evaluations were identified: one cost-utility analysis (CUA) of IVIg versus no Ig and one comparing IVIg with SCIg. The quality of the evidence was low, with most studies having small patient numbers and a high risk of bias. Compared with no treatment, Ig replacement reduced the hospitalization rate in patients with hematological malignancies.
One study reported no change in hospitalization rates following a program to reduce IVIg use, and an observational study comparing IVIg with SCIg found more hospitalizations with SCIg but lower total costs per patient. The CUA comparing IVIg with no IVIg suggested that IVIg treatment was not cost effective, but this study was published in 1991 and had significant limitations. The other CUA found that home-based SCIg was more cost effective than IVIg, but model inputs were derived from unpublished data in a very small patient cohort with HGG and different malignancies.
ConclusionsOur review highlights key gaps in the literature. The cost effectiveness of Ig replacement in patients with hematological malignancies is still very uncertain. Despite the increasing use of Ig replacement there are limited data regarding its direct and indirect costs, and its optimal use and implications for healthcare resources remain unclear. Given the paucity of data on the cost and cost effectiveness of Ig treatment in this population, further health economic research is warranted.
Assessment
Economic evaluation of remote patient monitoring and organizational analysis according to patient involvement: a scoping review
- Myriam Le Goff-Pronost, Isabelle Bongiovanni-Delarozière
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- Published online by Cambridge University Press:
- 26 September 2023, e59
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Background
A literature review concerning the economic evaluation of telemonitoring was requested by the authority in charge of health evaluation in France, in a context of deployment of remote patient monitoring and identification of its financing. Due to the heterogeneity of existing telemonitoring solutions, it was necessary to stratify the evaluation according to patient involvement. Three levels of patient involvement are considered: weak (automated monitoring), medium (monitoring supported by a professional), and strong (active remote participation).
ObjectivesWe performed a scoping review to provide a comprehensive overview of different systems of telemonitoring and their reported cost-effectiveness.
MethodsFollowing PRISMA-ScR guidelines, a search was performed in four databases: PubMed, MEDLINE, EMBASE, and Cochrane Library between January 1, 2013 and May 19, 2020. Remote patient monitoring should include the combination of three elements: a connected device, an organizational solution for data analysis and alert management, and a system allowing personalized interactions, and three degrees of involvement.
ResultsWe identified 61 eligible studies among the 489 records identified. Heart failure remains the pathology most represented in the studies selected (n = 24). The cost-utility analysis was chosen in a preponderant way (n = 41). Forty-four studies (72 percent) reported that the intervention was expected cost-effective. Heterogeneity has been observed in the remote monitoring solutions but all systems are reported cost-effective. The small number of long-term studies does not allow conclusions to be drawn on the transposability.
ConclusionsRemote patient monitoring is reported to be cost-effective whatever the system and patient involvement.
Oral Presentations
OP96 Adapting Patient Involvement For Fast Track Appraisals
- Mark Rasburn, Helen Crosbie, Laura Marsden
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- Published online by Cambridge University Press:
- 14 December 2023, p. S27
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Introduction
The National Institute for Health and Care Excellence (NICE) is piloting a new innovative approach to the way digital products, devices, and diagnostics that most reflect system need and demand are assessed. This early value assessment (EVA) approach will allow a more rapid assessment to enable patients to benefit from promising technologies sooner. Involving patients in the health technology assessment (HTA) lifecycle is a core principle at NICE, but established methods are not suitable for a rapid timeframe. NICE needs to adapt the approach to ensure that patients are supported to participate in EVAs and that their involvement is meaningful.
MethodsDue to the rapid timeframe, it was important to ensure patient contributors were not overloaded with information and that contact points were aligned. NICE reviewed the standard induction, support documents, and contact points to adapt the support provided. This included:
• updating recruitment documents to communicate the role of the committee and the EVA process;
• combining induction meetings between various NICE teams and providing recorded presentations;
• organizing earlier peer support with experienced lay members; and
• advising which of NICE’s nine online modules were most relevant.
ResultsSupport for patient contributors has been an important part of the HTA process, so enabling people to prepare and confidently deliver content at a committee meeting is vital. There has been some variation in the processes for different topics, but the feedback received from patient contributors indicated that their involvement was meaningful and valued. This was attributed to their close working relationship with the project team. NICE is collecting feedback from all patient contributors using an online survey. The findings of this survey and the evaluation of the support mechanisms will be presented.
ConclusionsDespite shorter timeframes, patient involvement has not been compromised. NICE will use the feedback from patient contributors to review and adapt the induction process and support offered. This will support patient contributors and enable NICE to allocate appropriate resources in the shortened timeframe.
Assessment
Proposing a health-specific organizational impact framework to evaluate organizational impacts in health technology assessments
- Caryn Mathy, Christophe Pascal, Isabelle Bongiovanni-Delarozière, Lauriane Favez
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- Published online by Cambridge University Press:
- 11 October 2023, e60
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Objectives
Health technology assessments (HTAs) have traditionally included clinical and cost-effectiveness evaluation of new health technologies (HTs). However, new HTs can generate important organizational impacts (OIs) that influence their overall value. OIs are currently not clearly identified and evaluated in HTA procedures and tools are limited. To address this issue, a comprehensive framework that allows to assess OIs of new HTs in HTAs is proposed.
MethodsA working and methodological group identified the Oslo Manual 2018, 4th edition, OECD/Eurostat, on the objectives and outcomes of commercial innovations as the basis for the OIs framework for HTAs. The Oslo Manual was translated to the healthcare sector and adapted to HTA procedures through a three-step process.
ResultsThe framework is composed of three main parts. Part I tackles the context of the evaluation, Part II the categories of impacts and the specific impacts – in total, 16 OIs were identified – and Part III the stakeholders involved. The central part of the framework is Part II, and consists of three categories of impacts: (i) on the care process, (ii) on the stakeholders’ capabilities and skills, and (iii) on society or the community.
ConclusionsThis framework provides a comprehensive and structured basis to document OIs of new HTs. It thus contributes to the extension of HTA evaluation criteria to other dimensions than clinical and economic aspects, that is, organizational aspects. Some of its intrinsic limitations and the questions they raise in the field for policy-makers, practitioners, and researchers are discussed.