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Assessment
Economic evaluation guidelines in low- and middle-income countries: a systematic review
- Caroline Daccache, Rana Rizk, Jalal Dahham, Silvia M. A. A. Evers, Mickael Hiligsmann, Rita Karam
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- 21 December 2021, e1
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Objectives
To systematically identify the latest versions of official economic evaluation guidelines (EEGs) in low- and middle-income countries (LMICs) and explore similarities and differences in their content.
MethodsWe conducted a systematic search in MEDLINE (Ovid), PubMed, EconLit, Embase (Ovid), the Cochrane Library, and the gray literature. Using a predefined checklist, we extracted the key features of economic evaluation and the general characteristics of EEGs. We conducted a comparative analysis, including a summary of similarities and differences across EEGs.
ResultsThirteen EEGs were identified, three pertaining to lower-middle-income countries (Bhutan, Egypt, and Indonesia), nine to upper-middle-income countries (Brazil, China, Colombia, Cuba, Malaysia, Mexico, Russian Federation, South Africa, and Thailand), in addition to Mercosur, and none to low-income countries. The majority (n = 12) considered cost–utility analysis and health-related quality-of-life outcome. Half of the EEGs recommended the societal perspective, whereas the other half recommended the healthcare perspective. Equity considerations were required in ten EEGs. Most EEGs (n = 11) required the incremental cost-effectiveness ratio and recommended sensitivity analysis, as well as the presentation of a budget impact analysis (n = 10). Seven of the identified EEGs were mandatory for pharmacoeconomics submission. Methodological gaps, contradictions, and heterogeneity in terminologies used were identified within the guidelines.
ConclusionAs the importance of health technology assessment is increasing in LMICs, this systematic review could help researchers explore key aspects of existing EEGs in LMICs and explore differences among them. It could also support international organizations in guiding LMICs to develop their own EEGs and improve the methodological framework of existing ones.
Oral Presentations
OP01 Convergent Validity Between Discrete Choice Experiment And Other Stated Preference Methods: A Multistudy Comparison
- Jorien Veldwijk, Tommi Tervonen, Esther de Bekker-Grob, Brett Hauber, Catharina G.M. Groothuis-Oudshoorn
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- 23 December 2022, p. S1
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Introduction
To assess convergent validity of stated preference methods in studies where they were used to elicit patient preferences for informing medical product decisions.
MethodsIn four studies, two stated preference methods were used to elicit preferences of patients with neuromuscular diseases (NMD; n = 140, Discrete Choice Experiment [DCE] and Best-Worst Scaling [BWS] case 2), diabetes (n = 495, DCE and swing weighting [SW]), myocardial infarction (MI; n = 335, DCE and BWS case 1), and rheumatoid arthritis (RA; n = 982, DCE and probabilistic threshold technique [PTT]). In each study, results of the two methods were compared using a normalized preference measure for which confidence intervals (CIs) were estimated using nonparametric bootstrapping of 500 samples. Normalized preference measures comprised of mean relative attribute importance weights (NMD and diabetes studies), attribute uptake probability (MI study), or maximum acceptable risk (RA study).
ResultsIn all four studies, attribute ranking showed similar patterns between DCE and other methods for the most important attributes. The same attribute had highest importance in three out of four studies. Significant differences were found in ranges of normalized preference measures of each study between DCE and the other methods: 4.1–43.4 versus 8.9–24.7 for DCE and BWS case 2 in NMD; 3.8–49.7 versus 11.9–16.8 for DCE and SW in diabetes; 2.0–85.5 versus 0.2–69.0 for DCE and BWS case 1 in MI; -3.5–49.2 versus 1.1–18.1 for DCE and PTT in RA.
ConclusionsPreferences differed significantly between DCE and other preference methods implying limited convergent validity. The substantially larger ranges in normalized outcome measures in DCE compared to other methods, are likely due to differences in mechanics and bias related to the methods. Since none of the methods is considered the golden standard for measuring stated preferences as true preferences are unknown, further studies are necessary to compare stated preference methods, determine internal validity and data quality, and potentially measure external validity.
Assessment
A review of audiovisual telemedicine utilization and satisfaction assessment during the COVID-19 pandemic
- Raphael Agbali, Andrew E. Balas, Francesco Beltrame, Gianluca De Leo
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- 20 December 2021, e2
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Introduction
The use of telemedicine has broadened as technology that both restores continuity of care during disruptions in healthcare delivery and routinely provides primary care alone or in combination with in-person care. During the Covid-19 outbreak, the use of telemedicine as a routine care modality further accelerated.
MethodsA review of scientific studies that used telemedicine to provide care from December 2019 to December 2020 is presented. From an initial set of 2,191 articles, 36 studies are analyzed. Evidence is organized and evaluated according to the country of study, the clinical specialty, the technology platform used, and satisfaction and utilization outcomes.
ResultsThirty-one studies reported high patient satisfaction scores. Eight studies reported satisfaction from both providers and patients with no uniformly accepted assessment instrument. Eight studies conducted a descriptive analysis of telemedicine use and patient adoption patterns. Less than one-third of studies were controlled before/after studies. Most studies were conducted in the USA followed by Europe.
ConclusionsReported satisfaction rates are high, consistent with previously documented research, whereas utilization rates increased significantly compared with the prepandemic period. Future work in developing standardized uniform assessment instruments, embedded with each telemedicine system, would increase versatility and agility in the assessment, boosting statistical power and the interpretation of results.
Oral Presentations
OP02 The Use Of Discrete Choice Experiments For Measuring Patient Preferences In Health Technology Assessment
- Michael Strauss, Gavin George, Yael Hirsch-Moverman, Joanne Mantell, Miriam Rabkin, Elizabeth Kelvin
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- 23 December 2022, pp. S1-S2
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Introduction
Understanding patient preferences and the demand for healthcare interventions and technology is critical for health technology assessment (HTA). New health technologies have potential for savings and increased efficiency but even the most cost-effective and efficacious interventions can fail if patient preferences are not properly accounted for. Patient preferences in HTA are primarily limited to representation in appraisal committees; however, more robust methods are available and should be incorporated into the assessment of interventions.
MethodsUsing data from three discrete choice experiments (DCEs), we reflect on the importance of patient preferences in the design of healthcare interventions. We draw insights from three studies which investigated preferences relating to HIV self-testing amongst long distance truck drivers in Kenya; differentiated antiretroviral therapy services amongst stable HIV patients in Zimbabwe; and tuberculosis preventive therapy for children in Eswatini.
ResultsWe highlight three key findings. First, understanding patient preferences is crucial when designing services, and providers sometimes underestimate behavioural barriers and overestimate the extent to which people are motivated simply by health benefits. Optimism is often driven by evidence showing high acceptability, but when preference structures are incorporated in intervention design, there are important insights into how patients plan to utilize services. Second, trade-offs matter in determining which characteristics are perceived to be most important to patients – a key strength of the DCE methodology. Understanding of these trade-offs can help prioritize which characteristics of interventions to target. Finally, disentangling the effect of different characteristics of service delivery models on preferences is important for rethinking how interventions are delivered. If services are designed to better align with preferences, implementers can ensure new interventions have the desired effect on health and economic outcomes.
ConclusionsThese findings highlight the value of behavioural economic approaches for investigating preferences for health interventions and providing insights into the demand for services, which must feed into the HTA analyses. Incorporating DCEs into HTA is inexpensive and provides robust data for improving HTA.
Article Commentary
Mentoring as a potential means to assist consumer representatives contribute effectively to the assessment of health technologies in Australia: a pragmatic review
- Christine Walker, Jan Donovan, Jo Watson
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- 20 December 2021, e3
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Objectives
This project was implemented on behalf of the Health Technology Assessment (HTA) Consumer Consultative Committee (CCC) to explore training to support and retain new consumer representatives to participate effectively in HTA committees. These committees are key parts of the Australian Government's health system. Currently, there is no training available to them, specific to their roles in HTA committees. Hypothesizing that mentoring is appropriate, the project team undertook a literature review to identify definitions of mentoring, its benefits, skills requirements, resources, examples of best practice, and how mentoring might support consumer representatives in formal health technology assessment committee structures.
MethodsA rapid review was commenced by the project team and fifty-seven articles were identified and read independently. Following discussion, the team revised its approach as there was little evidence to assess and drew upon thirty-five articles where elements of mentoring were described. Discussion was followed by a thematic qualitative analysis exploring mentoring models.
ResultsThe project team agreed that features of mentoring programs were necessary to design a mentoring program under the headings of definitions, mentors’ qualities, benefits of training, resources, other considerations, and evaluation. These assist the design of a pilot project to test mentoring's effectiveness.
ConclusionMentoring may assist consumers working in the health technology area to develop their skills and competencies and contribute to representing the needs of health consumers in the approval of applications. A pilot mentoring program is currently being designed and will run with one mentor and one mentee in an HTA committee.
Oral Presentations
OP03 Patient Characteristics Affect Their Treatment Choice: A Discrete Choice Experiment With Breast Cancer Patients In Six European Countries
- Eugena Stamuli, Sorcha Corry, Petter Foss
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- 23 December 2022, p. S2
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Introduction
The evolution of breast cancer treatments over the last decade has resulted in tailored therapies for the different types and stages of breast cancer. Each treatment has a profile of benefits and adverse effects which are taken into consideration when planning a treatment pathway. The objective of this study is to examine whether patients’ preferences are in line with what is considered important from policy-makers viewpoint.
MethodsAn online discrete choice experiment (DCE) was conducted in six European countries (France, Germany, Ireland, Poland, Spain, UK) with breast cancer (BC) patients. The DCE comprised of six attributes: overall survival (OS), hyperglycaemia, rash, pain, functional well-being (FWB) and out-of-pocket payment (OOP). Sixteen choice sets with two hypothetical treatments and a “no treatment” option were presented. Sociodemographic and disease related data were collected. Heteroscedastic conditional and mixed logistic models accounted for scale and preference heterogeneity between countries and patients respectively. Latent class analysis categorized patients in classes. Marginal rates of substitution (MRS) were estimated for OOP versus the rest of attributes to establish the ranking of preferences for each attribute.
ResultsTwo hundred and forty-seven patients with advanced or metastatic BC and 314 with early-stage BC responded. Forty-nine percent of patients were less than 44 years old and 65 percent had completed university education. The MRS of the analysis demonstrated that “severe pain” is the highest dis-preferred attribute level, followed by “severe impairment in FWB” and OS. Four classes of patients as “decision-makers” were identified. Additionally, there is sensitivity in preferences for both levels of pain and FWB depending on the stage of the disease.
ConclusionsThis study suggests that there is heterogeneity in treatment preferences of breast cancer patients depending on their sociodemographic and disease related characteristics. In combination with clinical guidelines, patient preferences can support the selection and tailoring of treatment options.
Method
Opportunities to improve reporting of rapid response in health technology assessment
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- Matheus O. de Almeida, Thais Montezuma, Haliton A. de Oliveira Júnior, Cleusa Pinheiro Ferri
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- 20 December 2021, e4
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Introduction
Mini health technology assessment (HTA) reports have been used to support policy makers and health systems by providing a timely summary of scientific evidence. The objective of this meta-epidemiologic study was to evaluate the quality of reporting of mini-HTA reports published in Brazil.
MethodsAn electronic search for all mini-HTA reports published between 2014 and March 2019 was conducted in the SISREBRATS and CONITEC databases. The study selection and data extraction were performed by two independent assessors. The following data were extracted: bibliographic data; research question; characteristics of the population, health technologies and outcomes assessed; eligibility criteria; information about searches and study selection; risk of bias assessment; quality of evidence assessment; synthesis of results; and recommendation about the technology evaluated. A descriptive analysis was used to summarize the information retrieved from all the included mini-HTA reports.
ResultsWe included 103 mini-HTA reports, the great majority of which (92.3 percent) focused on the coverage of the technologies in the healthcare system, with more than 60 percent being about drugs. Only five mini-HTA reports (4.8 percent) gave reasons for the choice of outcomes, and fifteen (14.5 percent) discriminated between primary and secondary outcomes. All mini-HTAs reported the databases searched and 99 percent of them reported using Medline. Sixty percent of the mini-HTA reported assessing the risk of bias, and 52 percent reported assessing the quality of evidence.
ConclusionThe quality of reporting of the mini-HTA reports performed in Brazil is insufficient and needs to be improved to guarantee transparency and replicability.
Oral Presentations
OP04 Methodological Challenges Of Assessing An Evolving Technology: The Cochlear Implant For Deaf People
- Hugo Nijmeijer, Hans Groenewoud, Emmanuel Mylanus, André Goedegebure, Wendy Huinck, Gert Jan van der Wilt
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- 23 December 2022, p. S2
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Introduction
When Cochlear implants (CI) were first introduced, only postlingually, profoundly hearing impaired individuals were implanted unilaterally. As experience grew, eligibility was followed by prelingual deaf children, and a second contralateral CI was being considered. Due to surgical and technological improvements, eligibility criteria for CI are now shifting, encompassing patients with more residual hearing. We aimed to explore, ex ante, whether such shift is warranted.
MethodsA dynamic, population-based Markov modeling study was conducted. Model parameters were based on available evidence, expert opinion, and calibration. The model mimics Dutch demographic development in three age categories over a period of 20 years. Impact of changing eligibility was explored in terms of number of CI recipients, costs, quality of life and cost-effectiveness from a societal perspective.
ResultsIf those with severe hearing loss would qualify and opt for CI similar to those with profound hearing loss, this would lead to a fourfold increase of CI recipients (from 8,815 to 35,630) over a 20 year period, resulting in an increase in costs (EUR 550 million) and QALYs (54,000), with an Incremental Cost Utility Ratio of EUR 10,771/QALY (2.5–97.5 percentiles: 1,252–23,171).
ConclusionsResults suggest that expected health gains could be such, that the investment may be considered cost-effective against the backdrop of currently prevailing criteria. However, for this, a substantial increase in operating capacity, follow-up care and rehabilitation are required. Further inquiries are needed to investigate whether such increased capacity can be achieved, to ensure equitable access to those services.
Assessment
Exploring facilitators and barriers to introducing health technology assessment: a systematic review
- Christian Suharlim, Ritu Kumar, Julian Salim, Meenakshi Mehra, Colin Gilmartin, Ana Amaris Caruso, Hector Castro
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- 21 December 2021, e5
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Objective
This study aims to identify and codify the facilitators and barriers to help implementing partners institutionalize health technology assessment (HTA) successfully and navigate complex systems for health-related policy making.
MethodsWe searched for peer-reviewed and gray literature articles examining HTA programs globally using six databases. Keywords used as a guide for capturing articles included “health technology assessment,” “barrier,” and “facilitator” and their synonyms. Search results were scrutinized for duplicates and screened through a review of titles and abstracts. A full-text review was conducted exploring articles’ coverage of twenty-seven evaluation criteria across four primary areas of interest: barriers/facilitators, motivations, guidelines, and institutional frameworks.
ResultsA total of 18,599 records were identified for duplication check, title, and abstract review. A total of 1,594 articles underwent full-text review, leading to a final synthesis of 262 studies. We found that ninety-seven articles discussed barriers/facilitators, with fifty-three of those discussing local capacity and unavailable human resources. Out of the sixty-six articles discussing motivations, forty-two cited the interest in supporting the decision-making process for, and promoting, appropriate resource allocation. Of the sixty-one articles that discussed guidelines and institutional framework, twenty-one articles described HTA as an independent national unit, and sixteen described their HTA unit as a unit within the Ministry of Health (MOH).
ConclusionsThis systematic review unpacks the dynamic and relevant contexts for understanding the HTA institutionalization process to help policy makers and practitioners achieve tangible progress in confronting the most critical issues facing priority setting and HTA institutionalization.
Oral Presentations
OP05 The Role Of Conditional Reimbursement In The Lifecycle Approach
- Hedi Schelleman, Simone van Montfort, Lenneke Sicking, Daniëlle Haasnoot-Volker
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- 23 December 2022, p. S3
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Introduction
In 2012, the Netherlands introduced a conditional reimbursement (CR) program to give patients earlier access to promising treatments (i.e., drugs and medical devices). After completion of the CR-study the results are used to (re)assess whether the treatment fulfills the criteria for reimbursement.
MethodsPromising treatments were identified via a bottom-up (physicians/researchers initiated) or top-down process (after the Dutch National Health Care Institute (Zorginstituut Netherland; ZIN) concluded that the intervention did not fulfill the criteria for reimbursement but the initial results seemed promising). A CR-grant was only approved if a committee determined the treatment had an added (health, social, ethical or organizational) benefit compared to Standard of Care (SoC) and the grant proposal was of good quality and fit for purpose. After approval from the Dutch Ministry of Health, all Dutch insurance companies were obliged to reimburse the treatment for patients participating in the CR-study. Researchers could also apply for a research grant (maximum EUR 400,000) from the Netherlands Organization for Health Research and Development (ZonMw), if there was no ‘wealthy’ manufacturer.
ResultsCurrently, there are 23 (ongoing and completed) CR-studies. All of them are closely monitored by ZIN, ZonMw, and stakeholders. The results of all completed CR-studies (n = 11) have been used in a (re)assessment. ZIN concluded that five treatments were not effective compared to SoC. Six interventions were effective and cost-effective compared to SoC and are now reimbursed. In most cases (>80%) the physician and patient groups agreed with the conclusion about reimbursement. In some cases there were additional requirements to maintain the clinical effectiveness and cost-effectiveness in clinical practice (such as training of new physicians).
ConclusionsData from CR-studies are important for reassessments. Factors with a positive influence are: a maximum duration for a CR-study, close monitoring, possibility to adapt the study design (only with approval from ZIN and ZonMw), and active involvement of stakeholders (physician and patient groups). A negative influence was: the legal requirements to ensure only reimbursement for patients participating in a CR-study.
Policy
Bridging health technology assessment and healthcare quality improvement using international consortium of health outcomes measurement standard sets
- Rachel R. J. Kalf, Marloes Zuidgeest, Diana M. J. Delnoij, Marcel L. Bouvy, Wim G. Goettsch
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- 21 December 2021, e6
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Objective
Although health technology assessment (HTA) and healthcare quality improvement are distinct processes, a greater level of alignment in outcome measures used may increase the quality and efficiency of data collection. This study evaluates the agreement in outcome measures used in oncology for healthcare quality improvement and HTAs, and how these align to the International Consortium for Health Outcomes Measurement (ICHOM) standard sets.
MethodsWe conducted a cross-sectional comparative analysis of ICHOM sets focusing on oncological indications and publicly available measures for healthcare quality and HTA reports published by the National Health Care Institute from the Netherlands and the National Institute for Health and Care Excellence from the United Kingdom.
ResultsAll ICHOM sets and HTAs used overall survival, whereas quality improvement used different survival estimates. Different progression estimates for cancer were used in HTAs, ICHOM sets, and quality improvement. Data on health-related quality of life (HRQoL) was recommended in all ICHOM sets and all HTAs, but selectively for quality improvement. In HTAs, generic HRQoL questionnaires were preferred, whereas, in quality improvement and ICHOM sets, disease-specific questionnaires were recommended. Unfavorable outcomes were included in all HTAs and all ICHOM sets, but not always for quality improvement.
ConclusionsAlthough HTA and quality improvement use outcome measures from the same domains, a greater level of alignment seems possible. ICHOM may provide input on standardized outcome measures to support this alignment. However, residual discrepancies will remain due to the different objectives of HTA and quality improvement.
Oral Presentations
OP06 Percutaneous Transforaminal Endoscopic Discectomy: From Insufficient Evidence To Reimbursement
- Hedi Schelleman, Ingrid de Groot, Daniëlle Haasnoot-Volker, Petra Jellema
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- 23 December 2022, p. S3
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Introduction
The standard surgical technique for lumbosacral radicular syndrome in the Netherlands is open microdiscectomy (OM). An alternative technique, preferred by some Dutch physicians, is percutaneous transforaminal endoscopic discectomy (PTED). However, in 2006 the Dutch National Health Care Institute (Zorginstituut Netherland [ZIN]) concluded that the available evidence was insufficient, and a high quality randomized controlled trial (RCT) was needed to assess the cost-effectiveness of PTED compared to OM. The relevant physician group agreed with this conclusion, but they were unable to perform this RCT due to lack of funding and high treatment costs.
MethodsIn 2012, the Netherlands introduced a conditional reimbursement (CR) program to give patients earlier access to promising treatments. Researchers, in collaboration with physicians and patients, submitted a grant proposal and in 2016 the Dutch Ministry of Health approved the CR of PTED. Due to this decision, insurance companies were obliged to reimburse PTED for patients participating in the PTED-study (NCT02602093). The Netherlands Organization for Health Research and Development (ZonMw) also provided a research grant to fund the PTED-study. In total, 682 adult patients with greater than 10 weeks of radiating pain, or greater than 6 weeks of excessive radiating pain and an indication for surgery were included. After 4 years and 5 months the PTED-study was completed.
ResultsOutcomes of published studies and the unpublished PTED-study were used in the HTA reassessment. Results showed that PTED was noninferior to OM with regards to leg pain (Visual Analogue Scale: mean difference (MD) -0.73; 95% confidence interval [CI] -5.04, 3.59), functional status (Oswestry Disability Index: MD -2.07; 95% CI -3.61, -0.53), and rate of complications (relative risk 0.45; 95% CI 0.18, 1.12) after 6 months (GRADE level ‘moderate’). Furthermore PTED was, after the surgeons’ learning-curve, cost-effective.
ConclusionsThis CR project was successful and PTED is now reimbursed as part of the Dutch healthcare package. However, in order to maintain high quality care in clinical practice, safeguards should be developed (including the appropriate training of surgeons). This example shows that CR programs are essential for promising treatments without ‘wealthy’ manufacturers. Additionally, all stakeholders are needed to make a CR-study successful.
Perspective
Toward a broader concept of societal value: family spillovers in Alzheimer's disease
- Jenny Yeonhee Park, Zachary A. Marcum, Louis P. Garrison
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- 20 December 2021, e7
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Recognizing that the “healthcare sector perspective” can be too limited in some situations, the National Institute of Health and Care Excellence (NICE), Institute for Clinical and Economic Review (ICER), and the U.S. Second Panel on Cost-Effectiveness in Health and Medicine all recommend a “societal” perspective in “reference case” cost-effectiveness analyses (CEAs). Although costs of informal caregiving are sometimes included in the CEAs of Alzheimer's Disease (AD) drugs, the benefits and disutility to family members, referred to as “family spillovers” by the U.S. Second Panel, are usually omitted. We estimate that the aggregate cost of family spillovers could be substantial in the USA—on the order of USD 57 billion or over 10 percent of the total economic burden of AD in 2020. Incorporation of family spillovers in AD value frameworks and HTAs is important for comprehensively defining, rewarding, and providing high-value care in AD.
Method
A framework for action to improve patient and public involvement in health technology assessment
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- Aline Silveira Silva, Karen Facey, Stirling Bryan, Dayani Galato
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- 20 December 2021, e8
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Background
Patient and public involvement (PPI) in the Brazilian Health Technology Assessment (HTA) process occurs in response to a legislative mandate for “social participation.” This resulted in some limited patient participation activities, and, therefore, a more systematic approach was needed. The study describes the development of a suggested framework for action to improve PPI in HTA.
MethodsThis work used formal methodology to develop a PPI framework based on three-phase mixed-methods research with desktop review of Brazilian PPI activities in HTA; workshop, survey, and interviews with Brazilian stakeholders; and a rapid review of international practices to enact effective patient involvement. Patient partners reviewed the draft framework.
ResultsAccording to patient group representatives, their involvement in the Brazilian HTA process is important but could be improved. Different stakeholders perceived barriers, identified values, and made suggestions for improvement, such as expansion of communication, capacity building, and transparency, to support more meaningful patient involvement. The international practices identified opportunities for earlier, more active, and collaborative PPI during all HTA stages, based on values and principles that are relevant for Brazilian patients and the public. These findings were synthesized to design a framework that defines and systematizes actions to support PPI in Brazil, highlighting the importance of evaluating these strategies.
ConclusionsSince the publication of this framework, some of its suggestions are being implemented in the Brazilian HTA process to improve PPI. We encourage other HTA organizations to consider a systematic and planned approach with regular evaluation when pursuing or strengthening involvement practices.
Oral Presentations
OP07 Dealing With Uncertainty In Early Health Technology Assessment: An Exploration Of Methods For Decision-Making Under Deep Uncertainty
- Mirre Scholte, Vincent Marchau, Jan Kwakkel, Maroeska Rovers, Janneke Grutters
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- 23 December 2022, pp. S3-S4
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Introduction
In early stages, the consequences of innovations are often unknown or deeply uncertain. This complicates health economic modelling. The field of decision-making under deep uncertainty (DMDU) uses exploratory modelling (EM) to help decision-makers make sound decisions under conditions of deep uncertainty (i.e., when stakeholders do not know, or cannot agree on, the system model, the probability distributions to place over the inputs to these models, which consequences to consider, and their relative importance). The aim of this research was to evaluate the potential of EM for the early evaluation of health technologies.
MethodsEM and early health economic modelling (EHEM) were applied to an early evaluation of minimally invasive surgery (MIS) for acute intracerebral hemorrhage, and were compared to derive differences, merits, and drawbacks of EM.
ResultsThe approaches fundamentally differ in the way uncertainty is handled. Where in EHEM the focus is on the value of the technology, while accounting for the uncertainty, EM focuses on the uncertainty. EHEM aims to assess whether the innovative strategy is potentially cost-effective, often using assumptions. EM on the other hand focuses on finding robust strategies (i.e., strategies that give relatively good outcomes over a wide range of plausible futures). This was also reflected in our case study. For example, EHEM provided cost-effectiveness thresholds for MIS effectiveness, assuming fixed MIS costs. EM showed that a strategy with a population in which most patients had severe intracerebral hemorrhage was most robust, regardless of MIS effectiveness, complications, and costs.
ConclusionsEM seems most suited in the very early phases of innovation (i.e., when a problem is signaled). Here, it can explore the robustness of many potential strategies under uncertainty. When potential strategies are selected, EHEM seems useful to optimize these strategies. Yet, EM methods are complex and might only be fully effective when a policy window exists that facilitates flexible research and adoption strategies.
Assessment
Application of a health technology assessment framework to digital health technologies that manage chronic disease: a systematic review
- Amy von Huben, Martin Howell, Joseph Carrello, Sarah Norris, Sally Wortley, Angus Ritchie, Kirsten Howard
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- 20 December 2021, e9
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Background
As health services increasingly make investment decisions in digital health technologies (DHTs), a DHT-specific and comprehensive health technology assessment (HTA) process is crucial in assessing value-for-money. Research in DHTs is ever-increasing, but whether it covers the content required for HTA is unknown.
ObjectivesTo summarize current trends in primary research on DHTs that manage chronic disease at home, particularly the coverage of content recommended for DHT-specific and comprehensive HTA.
MethodsMedline, Embase, Econlit, CINAHL, and The Cochrane Library (1 January 2015 to 20 March 2020) were searched for primary research studies using keywords related to DHT and HTA domains. Studies were assessed for coverage of the most frequently recommended content to be considered in a nine domain DHT-specific HTA previously developed.
ResultsA total of 178 DHT interventions were identified, predominantly randomized controlled trials targeting cardiovascular disease/diabetes in high- to middle-income countries. A coverage assessment of the cardiovascular and diabetes DHT studies (112) revealed less than half covered DHT-specific content in all but the health problem domain. Content common to all technologies but essential for DHTs was covered by more than half the studies in all domains except for the effectiveness and ethical analysis domains.
ConclusionsAlthough DHT research is increasing, it is not covering all the content recommended for a DHT-specific and comprehensive HTA. The inability to conduct such an HTA may lead to health services making suboptimal investment decisions. Measures to increase the quality of trial design and reporting are required in DHT primary research.
Oral Presentations
OP08 Early Access To New Direct-Acting Antiviral: A Journey On Introduction Of Ravidasvir For Hepatitis C Treatment In Malaysia
- Nazatul Syima Idrus, Faridah Aryani Md Yusof, Rosliza Lajis
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- Published online by Cambridge University Press:
- 23 December 2022, p. S4
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Introduction
Access to affordable direct-acting antiviral (DAA) remains limited in developing countries, often due to high treatment cost. This study aimed to elaborate the initiatives taken by the Ministry of Health Malaysia (MoHM) to provide early access to ravidasvir, a new DAA for hepatitis C treatment, in Malaysia.
MethodsMoHM collaborated with Drugs for Neglected Diseases initiative (DNDi) to develop ravidasvir, a new chemical entity of oral non-structural protein 5A (NS5A) inhibitor. MoHM co-sponsored and participated the DNDi-led Phase II/III study (STORM-C-1 trial) to assess the efficacy and safety of ravidasvir-sofosbuvir combination therapy. Agreement was signed between Pharmaniaga, Pharco Pharmaceuticals and DNDi to register and supply affordable hepatitis C treatment in Malaysia and South-East Asia. MoHM and Pharmaniaga mutually worked on the registration of ravidasvir in Malaysia. Series of pre-submission meetings took place, rolling submission was allowed and conditional registration pathway was used. As a separate initiative, MoHM partnered with the Foundation for Innovative New Diagnostics (FIND) to implement decentralization and test-and-treat strategies for screening of hepatitis C virus (HCV).
ResultsFirst stage of the STORM-C-1 trial reported that the combination of ravidasvir-sofosbuvir was highly effective across all genotypes and safe. The Drug Control Authority (DCA) Malaysia has granted a conditional registration for ravidasvir hydrochloride 200mg tablet (Ravida®) in June 2021, making Malaysia as the first country in the world to approve ravidasvir. Registration process expedited and took place within 15 months. The supply of Ravida® in Malaysia is expected in near future. Meanwhile, MoHM also implemented nationwide HCV screening using rapid diagnostic test kit in private hospitals, community clinics, prisons and rehabilitation centers which previously was done only in hospitals for outreach to the targeted group.
ConclusionsRavidasvir-sofosbuvir has potential as a tool to eliminate hepatitis C in Malaysia by 2030, the WHO’s global elimination targets. This alternative new drug development model was successful due to strong leadership, public-private partnership and collaborative strategies. This could also be exercised in other disease area.
OP10 Outcomes Of Expanded Access To Transcatheter Aortic Valve Implantation In Ontario: A Model-Based Analysis
- Rafael N Miranda, John Peel, David Naimark, Harindra Wijeysundera
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- Published online by Cambridge University Press:
- 23 December 2022, pp. S4-S5
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Introduction
Transcatheter aortic valve implantation (TAVI) is a minimally invasive therapy for patients with severe aortic stenosis. In Ontario, increases in capacity have not matched the rapidly growing demand for TAVI. As a result, wait-times for TAVI in Ontario exceed guideline targets, and waitlist morbidity is consequently considerable. The objective of this study was to evaluate the clinical implications of expanded TAVI capacity.
MethodsWe performed a decision analysis using an open, parallel, resource-constrained microsimulation from the Ontario Ministry of Health perspective. Simulated patients entered the model during a five-year period, and stayed in the model until death or end of time horizon. Referral numbers increased annually according to historical trends. The additional capacity required to meet wait-time benchmarks in five years was identified by a sensitivity analysis. Clinical outcomes were estimated for three strategies: (i) current practice with annual capacity increases; (ii) accelerated capacity increases achieving benchmarks after five years; and (iii) no increase in capacity. Outcomes included pre-procedural mortality and hospitalization, and the proportion of TAVIs performed urgently.
ResultsOver the five years, we estimated that TAVI referrals would increase from 1,980/year to 3,268/year. To achieve wait-time benchmarks during this period, TAVI rates must be increased by approximately 6.3 percent annually, for a total of 12,220 procedures performed over the 5 years. Compared to current TAVI capacity increase, an accelerated increase in capacity achieving wait-time benchmarks led to a reduction of 29.36 percent in pre-procedural deaths, as well as 26.38 percent in pre-procedural hospitalizations and 30.31 percent in nonelective TAVIs.
ConclusionsIncreases in TAVI capacity in Ontario must be accelerated to meet wait-time benchmarks in five years. Expansion of TAVI care in Ontario would be associated with considerable reductions in mortality and hospitalizations. Without intervention, both wait-times and adverse outcomes on the waitlist are expected to continue increasing. Prioritization strategies to mitigate the adverse effects of long wait-times must be used until wait-time targets are achieved.
Assessment
Optimizing red blood cell transfusion practices in the intensive care unit: a multi-phased health technology reassessment
- Lesley J.J. Soril, Tom W. Noseworthy, Derek R. Townsend, Sean M. Bagshaw, Henry T. Stelfox, David A. Zygun, Fiona M. Clement
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- Published online by Cambridge University Press:
- 20 December 2021, e10
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Background
Health technology reassessment (HTR) is a process to manage existing health technologies to ensure ongoing optimal use. A model to guide HTR was developed; however, there is limited practical experience. This paper addresses this knowledge gap through the completion of a multi-phase HTR of red blood cell (RBC) transfusion practices in the intensive care unit (ICU).
ObjectiveThe HTR consisted of three phases and here we report on the final phase: the development, implementation, and evaluation of behavior change interventions aimed at addressing inappropriate RBC transfusions in an ICU.
MethodsThe interventions, comprised of group education and audit and feedback, were co-designed and implemented with clinical leaders. The intervention was evaluated through a controlled before-and-after pilot feasibility study. The primary outcome was the proportion of potentially inappropriate RBC transfusions (i.e., with a pre-transfusion hemoglobin of 70 g/L or more).
ResultsThere was marked variability in the monthly proportion of potentially inappropriate RBC transfusions. Relative to the pre-intervention phase, there was no significant difference in the proportion of potentially inappropriate RBC transfusions post-intervention. Lessons from this work include the importance of early and meaningful engagement of clinical leaders; tailoring the intervention modalities; and, efficient access to data through an electronic clinical information system.
ConclusionsIt was feasible to design, implement, and evaluate a tailored, multi-modal behavior change intervention in this small-scale pilot study. However, early evaluation of the intervention revealed no change in technology use leading to reflection on the important question of how the HTR model needs to be improved.