Assessment
A decision-support tool for funding health innovations at a tertiary academic medical center
- Yiying Cai, Nuraini Nazeha, Shamira Perera, Alexandre H. Thiery, Michaël J.A. Girard, Chen E. Lee, Weiwei Hong, Nicholas Graves
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- Published online by Cambridge University Press:
- 13 February 2023, e11
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Objectives
To report the processes used to design and implement an assessment tool to inform funding decisions for competing health innovations in a tertiary hospital.
MethodsWe designed an assessment tool for health innovation proposals with three components: “value to the institution,” “novelty,” and “potential for adoption and scaling.” The “value to the institution” component consisted of twelve weighted value attributes identified from the host institution’s annual report; weights were allocated based on a survey of the hospital’s leaders. The second and third components consisted of open-ended questions on “novelty” and “barriers to implementation” to support further dialogue. Purposive literature review was performed independently by two researchers for each assessment. The assessment tool was piloted during an institutional health innovation funding cycle.
ResultsWe used 17 days to evaluate ten proposals. The completed assessments were shared with an independent group of panellists, who selected five projects for funding. Proposals with the lowest scores for “value to the institution” had less perceived impact on the patient-related value attributes of “access,” “patient centeredness,” “health outcomes,” “prevention,” and “safety.” Similar innovations were reported in literature in seven proposals; potential barriers to implementation were identified in six proposals. We included a worked example to illustrate the assessment process.
ConclusionsWe developed an assessment tool that is aligned with local institutional priorities. Our tool can augment the decision-making process when funding health innovation projects. The tool can be adapted by others facing similar challenges of trying to choose the best health innovations to fund.
Oral Presentations
OP14 Cost-Utility Analysis Of Regorafenib For Patients With Hepatocellular Carcinoma Who Progressed On Sorafenib Treatment
- Ambrish Singh, Salman Hussain
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- 14 December 2023, p. S5
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Introduction
In the RESORCE trial, regorafenib was shown to provide overall survival (OS) benefit for patients with hepatocellular carcinoma (HCC) that has progressed on sorafenib treatment. Subsequently, it was approved by the Therapeutic Goods Administration for the treatment of patients with HCC who were previously treated with sorafenib; however, regorafenib is still not recommended by the Pharmaceutical Benefits Advisory Committee in Australia. We aimed to assess the cost effectiveness of regorafenib as a second-line therapy for patients with HCC who progressed on sorafenib from an Australian healthcare perspective.
MethodsWe developed a Markov model to compare the cost effectiveness of regorafenib with best supportive care (BSC) as a second-line therapy for HCC after treatment with sorafenib. The health outcomes of life-years and quality-adjusted life-years (QALYs) were derived from the RESORCE trial. Survival benefits sourced from the RESORCE trial were fitted with the parametric model to estimate survival beyond the follow-up period. Drug costs and costs associated with adverse events (AEs) were sourced from published literature and the Independent Health and Aged Care Pricing Authority cost report. Model validity was verified using probabilistic sensitivity analyses.
ResultsThe incremental monthly cost of treatment with regorafenib was AUD19,273 (USD13,374), with an incremental life-year gain of 0.38, compared with BSC. The incremental QALYs gained with regorafenib were 0.24, resulting in a base-case incremental cost-effectiveness ratio (ICER) of AUD80,511 (USD55,872) per QALY. In the probabilistic sensitivity analyses across scenarios, the ICER remained above the conventional threshold of AUD50,000 (USD34,698) per QALY, with a zero probability of being cost effective at this willingness-to-pay threshold.
ConclusionsAt the current price, second-line treatment with regorafenib in patients with HCC that has progressed on sorafenib was not cost effective at the conventional willingness-to-pay threshold from an Australian health-system perspective.
OP18 Laying The Foundation For Sustainable Health Technology Assessment Training Program In Ukraine
- Wietske Kievit, Jip Janssen, Wija Oortwijn, Anton Voitenko, Oresta Piniazhko, Rabia Sucu
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- Published online by Cambridge University Press:
- 14 December 2023, pp. S5-S6
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Introduction
Since 2017, health technology assessment (HTA) has been included in the Ukrainian Health Law fundamentals and its implementation has accelerated since it became mandatory in 2020. SAFEMed has been supporting the Ministry of Health in integrating HTA into the decision-making ecosystem and building capacity in HTA. In this 2022 to 2023 project, we aimed to create and conduct HTA training for doers, users, and trainers based on a developed model curriculum for an HTA master’s program, and to identify sets of criteria for successful training and training centers.
MethodsFirst, we reviewed websites and documents of current academic HTA master’s and advanced programs worldwide. Second, we performed an assessment of the training needs of HTA doers, users, and trainers in Ukraine using an online survey that captured level of experience and knowledge gaps. Third, we reviewed the capacity and quality requirements of existing academic centers that provide HTA training.
ResultsWe identified seven HTA master’s programs globally, which covered five HTA domains: (i) health problem and current use of the technology; (ii) description and technical characteristics; (iii) safety; (iv) clinical effectiveness; and (v) costs and economic evaluations. Other aspects of HTA, such as ethical, legal, social, and cultural aspects were also covered, but not in all programs. The needs assessment was completed by 40 doers (53%), users (43%), and potential trainers (5%) of HTA in Ukraine. Specific knowledge gaps included: comparative effectiveness, health economics, qualitative evidence synthesis, patient and public involvement, and ethical issues. The proposed program addresses these gaps and includes an introduction to HTA that is in line with the new HTA definition. We also generated a minimum set of quality assurance criteria to ensure successful training and to develop efficient training centers for delivering HTA programs.
ConclusionsOur study provides a strong foundation for planning and conducting sustainable HTA training for current and future doers, users, and trainers in Ukraine, which can be an example for other countries wishing to increase HTA capacity.
Assessment
Stakeholder preferences for attributes of digital health technologies to consider in health service funding
- Amy von Huben, Martin Howell, Sarah Norris, Kam Cheong Wong, James Tang, Samia Kazi, Liliana Laranjo, Clara K. Chow, Kirsten Howard
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- Published online by Cambridge University Press:
- 14 February 2023, e12
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Objectives
Health service providers are currently making decisions on the public funding of digital health technologies (DHTs) for managing chronic diseases with limited understanding of stakeholder preferences for DHT attributes. This study aims to understand the community, patient/carer, and health professionals’ preferences to help inform a prioritized list of evaluation criteria.
MethodsAn online best–worst scaling survey was conducted in Australia, New Zealand, Canada, and the United Kingdom to ascertain the relative importance of twenty-four DHT attributes among stakeholder groups using an efficient incomplete block design. The attributes were identified from a systematic review of DHT evaluation frameworks for consideration in a health technology assessment. Results were analyzed with multinomial models by stakeholder group and latent class.
ResultsA total of 1,251 participants completed the survey (576 general community members, 543 patients/carers, and 132 health professionals). Twelve attributes achieved a preference score above 50 percent in the stakeholder group model, predominantly related to safety but also covering technical features, effectiveness, ethics, and economics. Results from the latent class model supported this prioritization. Overall, connectedness with the patient’s healthcare team seemed the most important; with “Helps health professionals respond quickly when changes in patient care are needed” as the most highly prioritized of all attributes.
ConclusionsIt is proposed that these prioritized twelve attributes be considered in all evaluations of DHTs that manage chronic disease, supplemented with a limited number of attributes that reflect the specific perspective of funders, such as equity of access, cost, and system-level implementation considerations.
Oral Presentations
OP19 Exploring The Environment/Capacity Of South African Citizen Actors To Contribute To Health Technology Assessment Processes, Policy Development And Institutionalization
- Lauren Pretorius, Debjani Muller
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- 14 December 2023, p. S6
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Introduction
Several overarching health policy reform processes are currently underway in South Africa (SA), providing an opportunity to establish health technology assessment (HTA) and value-based assessment (VBA) frameworks that foster patient and citizen involvement (PCI). A mapping of the capacity, knowledge, and skill of SA PCI advocacy actors and understanding of the ‘middle-ground’ and influencing relationships that influence advocacy strategies for PCI in HTA, will allow us to determine the needs of PCI actors to entrench PCI principles in the emerging institutionalization of HTA in SA.
MethodsAn analysis of national and international legislative and policy frameworks indicates current gaps and opportunities for PCI institutionalization in HTA in SA. A survey was conducted to determine SA patient and citizen advocacy actors’ capacity, knowledge, and skill across multiple disease areas. An analysis of decision maker’s opinions and positions about PCI in HTA and VBA policy, and their potential influence on the PCI process was undertaken.
ResultsThe legislation and policy review indicate that engagement initiatives are positioned at the ‘involvement’ or ‘consultation’ stages of the engagement continuum, rather than higher-level engagement. Five percent of patient advocacy groups (PAGs) interviewed have formalized PCI HTA advocacy strategies. Few PAGs indicated employing processes to actively monitor the HTA and PCI-related activities of decision-makers.
The majority of PAGs stated that collaborative efforts within larger networks would generate more success, if they engaged in PCI in HTA advocacy. Over eighty percent of civil society stakeholders face capacity constraints, such as lack of knowledge of the legislative framework and theory of HTA, funding and manpower to engage in PCI. The majority of HTA processes undertaken by funders in SA do not actively include PAGs or formalized PCI.
ConclusionsExisting legislative and policy frameworks do not include PCI capacity-building strategies. This is impacted by the lack of coordination amongst patient and consumer groups, the willingness of existing HTA structures to formalize PCI, and the resources of the country’s PCI advocate actors to influence existing HTA processes.
Perspective
Environmental impact assessment in health technology assessment: principles, approaches, and challenges
- Michael Toolan, Sarah Walpole, Koonal Shah, Juliet Kenny, Páll Jónsson, Nick Crabb, Felix Greaves
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- Published online by Cambridge University Press:
- 23 February 2023, e13
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To reduce harm to the environment resulting from the production, use, and disposal of health technologies, there are different options for how health technology assessment (HTA) agencies can consider environmental information. We identified four approaches that HTA agencies can use to take environmental information into account in healthcare decision making and the challenges associated with each approach. Republishing data that is in the public domain or has been submitted to an HTA agency we term the “information conduit” approach. Analyzing and presenting environmental data separately from established health economic analyses is described as “parallel evaluation.” Integrating environmental impact into HTAs by identifying or creating new methods that allow clinical, financial, and environmental information to be combined in a single quantitative analysis is “integrated evaluation.” Finally, evidence synthesis and analysis of health technologies that are not expected to improve health-related outcomes but claim to have relative environmental benefits are termed “environment-focused evaluation.”
Method
Health technology assessment of diagnostic tests: a state of the art review of methods guidance from international organizations
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- Lavinia Ferrante di Ruffano, Isobel M. Harris, Zhivko Zhelev, Clare Davenport, Sue Mallett, Jamie Peters, Yemisi Takwoingi, Jon Deeks, Chris Hyde
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- Published online by Cambridge University Press:
- 21 February 2023, e14
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Objectives
To identify which international health technology assessment (HTA) agencies are undertaking evaluations of medical tests, summarize commonalities and differences in methodological approach, and highlight examples of good practice.
MethodsA methodological review incorporating: systematic identification of HTA guidance documents mentioning evaluation of tests; identification of key contributing organizations and abstraction of approaches to all essential HTA steps; summary of similarities and differences between organizations; and identification of important emergent themes which define the current state of the art and frontiers where further development is needed.
ResultsSeven key organizations were identified from 216 screened. The main themes were: elucidation of claims of test benefits; attitude to direct and indirect evidence of clinical effectiveness (including evidence linkage); searching; quality assessment; and health economic evaluation. With the exception of dealing with test accuracy data, approaches were largely based on general approaches to HTA with few test-specific modifications. Elucidation of test claims and attitude to direct and indirect evidence are where we identified the biggest dissimilarities in approach.
ConclusionsThere is consensus on some aspects of HTA of tests, such as dealing with test accuracy, and examples of good practice which HTA organizations new to test evaluation can emulate. The focus on test accuracy contrasts with universal acknowledgment that it is not a sufficient evidence base for test evaluation. There are frontiers where methodological development is urgently required, notably integrating direct and indirect evidence and standardizing approaches to evidence linkage.
Oral Presentations
OP21 Patient Values Project (PVP): Patient Preferences For Cancer Treatments To Inform A Framework Incorporating Patient Values Into Health Technology Assessment
- Deborah A Marshall, Karen V MacDonald, Barry Stein
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- Published online by Cambridge University Press:
- 14 December 2023, pp. S6-S7
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Introduction
The methodology for explicitly incorporating patient preferences by expert committees engaged in deliberative health technology assessment (HTA) processes for drug reimbursement recommendations is a relatively unexplored area despite the growing emphasis on patient-reported outcomes and patient engagement. The Patient Values Project (PVP) aims to improve patient input to expert review committees and promote a better understanding of the patient perspective using quantitative data to support the rationale in assessing new cancer drugs. Using colorectal cancer as a starting point, the PVP aims to develop a framework to objectively incorporate quantitative patient values and preferences into Canada’s cancer drug HTA decision-making process. We report on results from the first phase.
MethodsIn the first phase, we developed a bilingual survey informed by qualitative focus groups, literature review and feedback from clinicians, patients and experts. The survey includes background questions, general and cancer specific quality-of-life tools, two discrete choice experiments (DCE) and a best worst scaling (BWS) experiment. After pre-testing and pilot testing, the survey was administered across Canada to metastatic and non-metastatic colorectal cancer patients and caregivers, in addition to adults from the general population. In the next phases, we will use vignettes to explore how patient preferences could be incorporated explicitly into decision-making, and what approach to use in HTA submissions.
ResultsDCE1 survey results (˜n=1,000) reflect trade-offs between health-related quality-of-life and survival; DCE2 results reflect trade-offs between treatment regimens, side effects and survival/risk of recurrence; BWS results ranked and weighted the tolerability of 25 possible side effects of treatment. We observed differences in preferences amongst the general population, patients with metastatic cancer, non-metastatic cancer and caregivers.
ConclusionsPatients have unique perspectives and preferences about what is important and of value to them, which may impact patient adherence to treatment. In the next phases, we will explore how this evidence from patient preferences can be translated into values that could potentially be incorporated as an explicit element of the deliberative process for HTA decision-making.
Policy
Health technology assessment 2025 and beyond: lifecycle approaches to promote engagement and efficiency in health technology assessment
- Rebecca Trowman, Antonio Migliore, Daniel A. Ollendorf
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- Published online by Cambridge University Press:
- 23 February 2023, e15
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Lifecycle considerations have always been part of health technology assessment (HTA). However, the concept of taking a fuller, more holistic “lifecycle approach” is gaining interest in the HTA community. The 2022 HTAi Global Policy Forum (GPF) discussed how adopting a lifecycle approach could promote stakeholder engagement and robust evidence generation, and whether it could enhance information sharing and transparency across stakeholder groups. This article summarizes the discussions held at the 2022 HTAi GPF and subsequent HTAi Annual Meeting panel session that debated some of the key challenges and opportunities, with particular focus on the pre- and postmarket and disinvestment phase activities. Core themes and recommendations identified that collaboration and patient involvement are happening but still needs to be strengthened, and moving to disease-based approaches may help, although individual contexts still need to be considered. Appropriately developed and mandated core outcome sets may help with information sharing and efficiency in all lifecycle activities. Further, methods for the appropriate use of big data and digital data collection should be developed and driven by the HTA community. The value of lifecycle activities should be reviewed; in particular, scientific advice appears valuable, but the magnitude of effect is somewhat unknown due to the challenges around the confidential nature of these activities. Not all lifecycle activities can be conducted for every technology, and while there is a move away from disinvestment phase activities, more structured prioritization criteria are required. This article ends with suggested next steps to bring forward some of the priority recommendations.
Oral Presentations
OP22 Benchmarking Of Population-Based Childhood Cancer Survival By Toronto Stage: Know The Differences To Propose Effective Interventions
- Rosalia Ragusa, Dott Fabio Didonè, Laura Botta, Antonina Torrisi, Maria Alessandra Bellia, Gemma Gatta, BENCHISTA Italy working group
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- Published online by Cambridge University Press:
- 14 December 2023, p. S7
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Introduction
Pediatric cancers are rare tumors, heterogeneous in location and biologically very different from adult cancers. Documented survival variation across European countries and Italian regions shows that there is still room for further improvement by reducing inequalities. We aim to understand why there are differences in survival. The BENCHISTA-ITA project (National Benchmarking of Childhood Cancer Survival by Stage at diagnosis), that is the Italian twin project of the International BENCHISTA, collects stage at diagnosis of solid pediatric tumors, according to the Toronto Guidelines. We will compare how far the cancer has spread at diagnosis and test if differences in tumor stage explain any survival differences between Italian regions.
MethodsThe project study involved the stage distribution and the survival of 9 pediatric solid tumors diagnosed between 2013 and 2017 in Italy. All patients therefore had at least 3 years of follow-up in 2021 for life-stage definition. The study involves the identification of all new diagnoses of cancer, evaluation of the clinical documentation of cases eligible for research, and international classification and coding. Analyses of stage distribution and survival rates for each tumor type will be described.
ResultsData from 35 population-based cancer registries from 18 out of 20 Italian regions were collected covering about 84 percent of the Italian child population. In particular, data on: imaging/examination performed before any treatment; source used for staging; primary treatment defined as given within one year from diagnosis; relapse/ recurrence/ progression; follow up and status of life. The study tested the applicability of the Toronto Guidelines as a tool to obtain population-level comparable stage information for childhood cancers. There were 1,343 cases collected (242 Neuroblastoma, 124 Wilms Tumour, 145 Medulloblastoma, 148 Osteosarcoma, 135 Ewing sarcoma, 115 Rhabdomyososarcoma, 54 Ependymoma, 47 Retinoblastoma, 333 Astrocytoma). Toronto stage could be assigned in more than 90 percent in the majority of tumors. Tumors in which it was more difficult to assign the stage using the Toronto staging guidelines were ependymoma, astrocytoma, and retinoblastoma. It was easier to retrieve data for patients in the 0-14 years of age range than adolescents (14-18 years). Differences in stage distribution and survival differences between regional grouping were presented.
ConclusionsThe Italian BENCHISTA project, improving the connection between pediatric cancer registries, aims to improve care of children with cancer across the nation, reducing possible disparities.
The wide adoption of the Toronto Guidelines will facilitate international comparative incidence studies, strengthen the interpretation of survival data, and contribute to more appropriate solutions to improve childhood cancer outcomes.
Method
Clinical effectiveness reporting of novel cancer drugs in the context of non-proportional hazards: a review of nice single technology appraisals
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- David Salmon, G. J. Melendez-Torres
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- 08 March 2023, e16
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Objectives
The hazard ratio (HR) is a commonly used summary statistic when comparing time to event (TTE) data between trial arms, but assumes the presence of proportional hazards (PH). Non-proportional hazards (NPH) are increasingly common in NICE technology appraisals (TAs) due to an abundance of novel cancer treatments, which have differing mechanisms of action compared with traditional chemotherapies. The goal of this study is to understand how pharmaceutical companies, evidence review groups (ERGs) and appraisal committees (ACs) test for PH and report clinical effectiveness in the context of NPH.
MethodsA thematic analysis of NICE TAs concerning novel cancer treatments published between 1 January 2020 and 31 December 2021 was undertaken. Data on PH testing and clinical effectiveness reporting for overall survival (OS) and progression-free survival (PFS) were obtained from company submissions, ERG reports, and final appraisal determinations (FADs).
ResultsNPH were present for OS or PFS in 28/40 appraisals, with log-cumulative hazard plots the most common testing methodology (40/40), supplemented by Schoenfeld residuals (20/40) and/or other statistical methods (6/40). In the context of NPH, the HR was ubiquitously reported by companies, inconsistently critiqued by ERGs (10/28), and commonly reported in FADs (23/28).
ConclusionsThere is inconsistency in PH testing methodology used in TAs. ERGs are inconsistent in critiquing use of the HR in the context of NPH, and even when critiqued it remains a commonly reported outcome measure in FADs. Other measures of clinical effectiveness should be considered, along with guidance on clinical effectiveness reporting when NPH are present.
Oral Presentations
OP23 Early Detection Tools For Emotional Distress In Adult Cancer Patients In Spain: A Health Technology Assessment Report
- Patricia Gomez-Salgado, Yolanda Triñanes, Beatriz Casal-Acción, María José Faraldo-Vallés
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- Published online by Cambridge University Press:
- 14 December 2023, p. S7
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Introduction
Patient-reported outcome measures are being increasingly considered both in clinical practice and in the field of health technology assessment. Although emotional distress is currently recognized as the sixth vital sign in cancer care, its early detection and screening is not yet included in routine clinical practice in Spain. The main objective of this study was to assess the psychometric properties and diagnostic accuracy of validated tools for the early detection of distress among adults with cancer in the Spanish context, at the request of the Spanish National Health System (NHS) Cancer Strategy.
MethodsA systematic review was carried out to analyze development and validation studies. The Quality Assessment of Diagnostic Accuracy Studies tool (QUADAS-2) was used for the risk of bias assessment, and a multicriteria global assessment was used for the tests. Ethical and organizational aspects were also addressed.
ResultsFifteen validation studies were included, corresponding to seven tests. The tools considered were the Distress Thermometer (DT), the Brief Symptom Inventory-18 (BSI-18), the Edmonton Symptom Assessment System-revised (ESAS-r), the Hospital Anxiety and Depression Scale (HADS), the Visual Analog Scale for Anxiety and Depression (VAS-AD), the Detection of Emotional Distress (DED) scale, and the Psychosocial and Spiritual Needs Evaluation (ENP-E) scale. Evidence of validity, reliability (internal consistency), and diagnostic accuracy (sensitivity, specificity, and area under the receiver operating characteristic curve) were summarized. Three scales were rated as poor (VAS-AD, BSI-18, and ESAS-r), the ENP-E scale was rated as acceptable, and three scales were rated as moderate (DT, DED, and HADS).
ConclusionsThe DT (single-item measure) stands out as an appropriate tool for early detection of emotional distress in the Spanish NHS. The use of this scale could be considered a first stage, to be combined later with a longer scale to improve screening specificity. The HADS scale could be utilized for this purpose. The use of these tools should be framed within a structured screening program that ensures further evaluation and subsequent psychological care when needed.
Policy
The added value of applying a disinvestment approach to the process of health technology assessment in Italy
- Chiara Cadeddu, Luca Regazzi, Eugenio Di Brino, Michele Basile, Fidelia Cascini, Andrea Paladini, Filippo Rumi, Americo Cicchetti, Walter Ricciardi
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- Published online by Cambridge University Press:
- 02 March 2023, e17
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Objectives
The objective of the present policy analysis was to understand how a disinvestment approach to the process of health technology assessment (HTA), applied to the field of medical devices, might help Italian policymakers to properly spend the resources in healthcare.
MethodsPrevious international and national experiences in disinvestment for medical devices were reviewed. Precious insights for the rational expenditure of the resources were derived by assessing the evidence available.
ResultsThe disinvestment of ineffective or inappropriate technologies or interventions with an inadequate value-for-money ratio has become a growing priority for National Health Systems. Different international disinvestment experiences of medical devices were identified and described through a rapid review. Although most of them have a strong theoretical framework, their practical application remains difficult. In Italy, there are no examples of large and complex HTA-based disinvestment practices, but their importance is becoming increasingly acknowledged, especially given the need to prioritize the funds provided by Recovery and Resilience Plan.
ConclusionsAnchoring decisions on health technologies without reassessing the current technological landscape through a robust HTA model might expose to the risk of not ensuring the best employment of the resources available. Thus, it is necessary to develop a strong HTA ecosystem in Italy through adequate consultation with stakeholders to enable a data-driven and evidence-based prioritization of resources toward choices characterized by high value for both patients and society as a whole.
Oral Presentations
OP24 Preferences Of Depressed And Depression-Prone Groups With Regard To Antidepressants In China: A Best-Worst Scaling Survey
- Shimeng Liu, Yanfeng Ren, Jing Liu, Yan Wei, Yingyao Chen
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- 14 December 2023, p. S8
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Introduction
Antidepressants are one of the main treatment approaches for depression, and previous evidence suggests that consideration of patient preferences can improve their adherence to medication regimens. The objective was, therefore, to evaluate the preferences of depressed and depression-prone groups in China with respect to antidepressant medications.
MethodsAn online survey with best-worst scaling choices was administered in depressed and depression-prone patients. The balanced independent block design generated 13 choice task profiles for each participant to answer, with each choice set consisting of four alternatives out of 13 antidepressant-specific attributes. Count analysis and a conditional logit model were used to estimate the relative importance of the 13 attributes and preference heterogeneity.
ResultsThe analytical sample included 210 participants, comprising 49 individuals who had previous experience with depression and 161 who were depression prone. Participants in both groups preferred medications with a low risk of liver or kidney damage, headache or dizziness, and recurrence. There were significant differences in both groups regarding out-of-pocket costs and duration of medication. The K-means clustering further proved preference heterogeneity among the patients.
ConclusionsOur study revealed patient preferences for antidepressant medication choices in China. Healthcare decision makers should consider and discuss patient preferences in the treatment decision-making process to improve patient adherence to and satisfaction with medications, and to ultimately improve patient outcomes.
Method
Health technology assessment for oral health in the past decade: a scoping review
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- Sonal Bhatia, Vikrant Mohanty, Aswini Y. Balappanavar, Puneet Chahar, Kavita Rijhwani, Radhika Gupta
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- Published online by Cambridge University Press:
- 27 March 2023, e18
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Background
Health technology assessment (HTA) is the systematic evaluation of various properties and effects of a health technology. HTA can serve as a bridge between the world of knowledge and that of decision making, offering decision makers the best summary of scientific evidence. Scoping HTA reports in the context of dentistry can help researchers identify grey areas; help practitioners make evidence-based decisions and further initiate better policy making.
AimTo provide an overview on HTAs pertaining to oral health and dentistry in the past decade, map the extension and scope of the methodological practices, key findings, and limitations.
MethodologyA scoping review was conducted using the Joanna Briggs Institute framework. A comprehensive search for HTA reports was done through the International Network of Agencies for Health Technology Assessment Database from January 2010 to December 2020. Consecutively, electronic databases (PubMed and Google Scholar) were searched. Finally, thirty-six reports were included in this review and analyzed.
ResultsA total of 709 articles were initially identified, of which thirty-six met the inclusion criteria. Reviewed HTAs focused on various specialties of dentistry worldwide. Maximum number of reports (N = 5) were related to “prosthodontics and dental implants” and technologies related to preventive dentistry were most commonly assessed (N = 4).
ConclusionFunctional, appropriate, and evidence-based information provided through HTA pertaining to oral health on a regular basis will enable decision makers to have enough data to make decisions on the future use of new technology, modify existing policies, accelerate its translation into practice, and ensure provision of robust dental healthcare services.
Oral Presentations
OP27 Impact Of Generic Entry Of Pharmaceuticals On Drug Prices In Australia
- Arun PG-Jones, Victoria Serra-Sastre, Hansoo Kim
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- Published online by Cambridge University Press:
- 14 December 2023, p. S8
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Introduction
Policy makers are keen to introduce cost containment measures in medicine spending due to aging populations and fiscal pressure. A major price reform was applied to the Australian Pharmaceutical Benefits Scheme (PBS) in 2014. This aimed to stimulate price reductions by increasing competition among generics, amidst growing evidence at the time of unnecessarily high generic medicine prices in Australia. The aim of this study was to estimate the effect of patent expiration and generic competition on drug prices, while controlling for other determinants of drug prices in Australia.
MethodsA dataset from publicly available sources was constructed using monthly data on the price of drugs listed on the PBS from October 2014 to July 2022. The information included the generic drug name, item code, date, approved ex-manufacturer price, dispensed price per maximum quantity, and brand names. This was supplemented with monthly government spending and number of prescriptions filled per item code. A fixed effects regression model was used to estimate the effect of patent expiration and generic competition on dispensed drug prices.
ResultsThe model estimated that introducing generics in Australia led to an 18 percent decrease in prices, excluding further decreases resulting from other controlled variables. The price elasticity of total prescriptions filled was estimated to be -0.6, suggesting that a one percent increase in prescriptions filled resulted in drug prices being lowered by 0.6 percent. This reflects the fact that, on average, firms reacted by reducing prices to increase market share when faced with an increase in quantity demanded. Each extra competitor was estimated to result in a reduction in prices of roughly 1.8 percent.
ConclusionsThese results show that entry of generics into the Australian pharmaceutical market resulted in a significant reduction in drug prices. However, this alone does not provide empirical support for the effectiveness of these price reforms in generating savings by inducing generic competition, especially over other forms of pharmaceutical regulation.
OP29 Impact Of New Drug Indications After Initial Registration By The Agência Nacional de Vigilância Sanitária (ANVISA) in Brazil
- Silvana Kelles, Camila Pereira, Carina Martins, Daniel Reis, Ernesto Azevedo, Geraldo Ribeiro, Karina Zocrato, Lélia Carvalho, Marcela Freitas, Maria Horta, Mariana Barbosa, Mariza Talim, Marcus Carvalho Borin
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- Published online by Cambridge University Press:
- 14 December 2023, pp. S8-S9
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Introduction
Most drugs have data only from clinical trials focused on a specific population at the time of first registration, so their indications for use are restricted to this population. In Brazil, the prices of new drugs for clinical conditions with no therapeutic alternatives are relatively high. When these drugs expand to other indications their prices are not reviewed, which can have a major financial impact. This study aimed to evaluate the financial impact of expanding the indication for trastuzumab deruxtecan.
MethodsWe calculated the annual cost to treat all Brazilian patients with the indications listed for trastuzumab deruxtecan at first registration, and then all additional indications. Populations were estimated from epidemiological data from National Committee for Health Technology Incorporation reimbursement documents and a clinical trial comparing trastuzumab deruxtecan with trastuzumab emtansine. Costs were calculated using the ANVISA Câmara de Regulação do Mercado de Medicamentos price value and a patient weight of 70 kg.
ResultsIn January 2022, trastuzumab deruxtecan was introduced in Brazil for patients with human epidermal growth factor receptor (HER2) positive metastatic or unresectable breast cancer who had received two or more anti-HER2 treatment regimens. In June 2022, the indication was expanded to patients with HER2-positive metastatic or unresectable breast cancer who had received one anti-HER2 treatment regimen. In November 2022, the indication was further expanded to patients with metastatic or unresectable low-expression HER2 breast cancer who had received prior systemic therapy. The number of patients estimated to be eligible for the drug increased from 383 to 23,000, with an increased total cost from BRL467,970,786 (USD90,621,763) to BRL26,048,234,160 (USD5,044,197,164).
ConclusionsThe expansion of indications for trastuzumab deruxtecan may substantially increase its financial impact and compromise the sustainability of health systems. In Brazil, the lack of monitoring of drug prices means that the only change in prices occurs due to regulated annual inflation adjustments. Regulation is needed to reduce drug prices according to new indications, changes in therapeutic options for the same condition, and obsolescence.
Method
Patient-based evidence: its role in decision making on end-of-life, orphan, and ultra-orphan medicines
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- Sharon Hems, Louise Taylor, Jan Jones, Eileen Holmes
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- 11 April 2023, e19
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Objectives
The Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of NHSScotland. Assessment of end-of-life (EoL), orphan, and ultra-orphan medicines includes a process to gather evidence from patients and carers during Patient and Clinician Engagement (PACE) meetings. The output of PACE meetings is a consensus statement describing the medicine’s added value from the perspective of patients/carers and clinicians. The PACE statement is used by SMC committee members in decision making. This study compared how PACE participants and SMC committee members rate the importance of information in PACE statements for these medicines.
MethodsA survey was undertaken of patient group (PG) representatives and clinicians who participated in PACE meetings, and SMC committee members.
ResultsPACE participants who responded (26 PG representatives and 14 clinicians) rated health benefits and ability to take part in normal life as important/very important. Convenience of administration and treatment choice received the lowest rating. Hope for the future received the most diverse response. PACE participants generally rated the importance of quality of life themes higher than committee members (n = 20) but the rank order was similar. Differences between the proportion of PACE participants and committee members who rated themes as important/very important were greatest for treatment choice and hope for the future.
ConclusionsIn general, PACE themes and subthemes that were rated highly by PACE participants were also considered important by SMC committee members, indicating that information captured during PACE meetings is relevant when making decisions on EoL, orphan, and ultra-orphan medicines.
Oral Presentations
OP33 Child And Adolescent Mental Health Care Models: A Scoping Review
- Ingrid Zechmeister-Koss, Reinhard Jeindl, Viktoria Hofer
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- 14 December 2023, p. S9
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Introduction
Mental illnesses are among the most common health problems in children and adolescents worldwide, and their prevalence has recently increased. At the same time, many countries face gaps in care and a shortage of services. To address these challenges, countries are developing child and adolescent mental health (CAMH) strategies and adopting their models of care. This paper aimed to give an international overview of these strategies and care models to support decision makers and stimulate mutual learning and improved CAMH care.
MethodsWe identified core topics within published CAMH strategies and care model documents from seven selected countries within the Global North, which represented different healthcare systems, geographical regions, and public health traditions to maximize variety. We systematically extracted data on the identified topics and summarized them narratively by applying qualitative content analyses.
ResultsThe documents addressed the following core topics: awareness raising activities; prevention and promotion; detection; treatment; telemedicine; care pathways; transitional psychiatry; vulnerable patient groups; user participation; infrastructure; workforce development; implementation; digital tools for case management; and data acquisition and research. A stand-alone CAMH strategy exists in most countries.
Recommendations on CAMH care often followed a public mental health approach and placed a high priority on mental health promotion and cross-sectional organization and funding of CAMH care services. Key principles of future CAMH care included: increased flexibility of care settings; early intervention; an open and non-judgmental attitude among staff; and strengths orientation instead of focusing on deficits and diagnoses.
ConclusionsReducing the prevalence of mental illness and current shortcomings in care requires action at the policy level (e.g., developing a CAMH strategy with a focus on mental health promotion and installing cross-sectoral governance), organizational level (e.g., reorganizing treatment settings and pathways of care), and individual level (e.g., strengthening user involvement and workforce development). Applying the recommended approaches in other countries will likely require redesign, ideally with a participatory approach and evaluation alongside piloting.
Policy
Regulatory, health technology assessment and company interactions: the current landscape and future ecosystem for drug development, review and reimbursement
- Ting Wang, Neil McAuslane, Wim G. Goettsch, Hubert G.M. Leufkens, Marie L. De Bruin
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- 11 April 2023, e20
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Background
Multi-stakeholder interactions have evolved at product and policy levels. There is a need to assess the current and future landscape of interactions between companies, and regulatory and HTA agencies to address challenges and identify areas for improvement.
ObjectivesThe aims of this study were to review the current interactions within and across regulatory and HTA agencies, and companies’ experiences in engaging in these activities; to assess the added value of interactions as well as limitations; to explore the future ecosystem for stakeholder interactions.
MethodThree separate questionnaires were developed for companies, regulators and HTA agencies, respectively, to assess their experiences and perceptions. The responses were analyzed using descriptive statistics and discussed at a multi-stakeholder workshop. Key outcomes from the surveys and workshop discussion were reported.
ResultsAll seven regulators and seven HTA agencies in the survey indicated that they had stakeholder interactions. More formal collaboration occurred with regulators compared with HTA agencies. All nine companies have taken early advice but indicated the need for future prioritization. Success indicators can be built at the product and therapy levels, with the added value of faster patient access. Four principles were proposed for the future ecosystem: separate remit and functions between regulators and HTA; align processes; converge evidence requirements where possible; increase transparency.
ConclusionsThis research brought together regulators, HTA agencies, companies to examine how they interact with one another. We propose measures of value and make recommendations on future evolution to enable better evidence generation and improve regulatory and HTA decision-making.