The aim of this study was to assess the effect of the wearable cardioverter-defibrillator (WCD) on patient-reported outcomes (PRO) in adult patients with high risk for sudden cardiac arrest.

We performed a systematic literature search in Medline (via PubMed) and Cochrane Library in February 2022 and included studies with a study population ≥18 years and prescribed WCD. PRO include health-related quality of life (QoL), symptoms, utilities, or satisfaction ratings. Study selection was done by two reviewers independently using predefined inclusion and exclusion criteria. Quality assessment of studies as well as data extraction was performed by one author and approved by a second author. Results of the included studies are presented quantitatively.

One randomized controlled trial (RCT), one comparative non-randomized trial, and three single-arm trials were included. QoL was assessed in four studies, but with different assessment tools. One study additionally evaluated the change in depressive symptoms and anxiety and one study focused on acceptability of WCD but evaluated items that are closely related to QoL. Results of the RCT show no statistically significant difference in QoL assessed by SF-36 and EQ-5D comparing WCD and Guideline-Directed Medical Therapy (GDMT) versus GDMT alone. One comparative study reports an improvement in depressive symptoms and anxiety within groups but no significant difference between groups. Further, one single-arm study reported improvement in QoL between baseline and day 90 and day 180.

The available evidence demonstrates that the usage of WCD is not affecting PRO, like QoL, depressive symptoms or anxiety negatively.

The management of non-metastatic castration-resistant prostate cancer (nmCRPC) is rapidly evolving; however, little is known about the direct healthcare costs of nmCRPC. We aimed to estimate the cost-of-illness (COI) of nmCRPC from the Italian National Health Service perspective.

Structured, individual qualitative interviews were carried out with clinical experts to identify what healthcare resources are consumed in clinical practice. To collect quantitative estimates of healthcare resource consumption, a structured expert elicitation was performed with clinical experts using a modified version of a previously validated interactive Excel-based tool, EXPLICIT (EXPert eLICItation Tool). For each parameter, experts were asked to provide the lowest, highest, and most likely value. Deterministic and probabilistic sensitivity analyses (PSA) were carried out to test the robustness of the results.

Ten clinical experts were interviewed, and six of them participated in the expert elicitation exercise. According to the most likely estimate, the yearly cost per nmCRPC patient is €4,710 (range, €2,243 to €8,243). Diagnostic imaging (i.e., number/type of PET scans performed) had the highest impact on cost. The PSA showed a 50 percent chance for the yearly cost per nmCRPC patient to be within €5,048 using a triangular distribution for parameters, and similar results were found using a beta-PERT distribution.

This study estimated the direct healthcare costs of nmCRPC in Italy based on a mixed-methods approach. Delaying metastases may be a reasonable goal also from an economic standpoint. These findings can inform decision-making about treatments at the juncture between non-metastatic and metastatic prostate cancer disease.

Based on a real-world collaboration with innovators in applying early health economic modeling, we aimed to offer practical steps that health technology assessment (HTA) researchers and innovators can follow and promote the usage of early HTA among research and development (R&D) communities.

The HTA researcher was approached by the innovator to carry out an early HTA ahead of the first clinical trial of the technology, a soft robotic sock for poststroke patients. Early health economic modeling was selected to understand the potential value of the technology and to help uncover the information gap. Threshold analysis was used to identify the target product profiles. Value-of-information analysis was conducted to understand the uncertainties and the need for further research.

Based on the expected price and clinical effectiveness by the innovator, the new technology was found to be cost-saving compared to the current practice. Risk reduction in deep vein thrombosis and ankle contracture, the incidence rate of ankle contracture, the compliance rate of the new technology, and utility scores were found to have high impacts on the value-for-money of the new technology. The value of information was low if the new technology can achieve the expected clinical effectiveness. A list of parameters was recommended for data collection in the impending clinical trial.

This work, based on a real-world collaboration, has illustrated that early health economic modeling can inform medical innovation development. We provided practical steps in order to achieve more efficient R&D investment in medical innovation moving forward.

Alternative options to hospital care like home care or local health centers (LHCs) are being advocated. However, no study has measured citizens’ preferences (who will finance these services via taxation) for these options.

We measured (i) citizens’ preferences for these services, that is, respondents stated where they would like to get the treatment; (ii) the strength of their preference.

A computerized survey composed of (i) a decision aid to inform respondents about the three options; (ii) three scenarios, from light-to-heavy care, that respondents should rank from the most to the least preferred option of care. (iii) a contingent valuation survey (CVS) to assess how much respondents were willing to pay for their preferred option (except for hospital care if chosen, because it is the default option and free). (iv) a socio-demographic questionnaire.

Data were collected from a representative sample of citizens living in the Rhône–Alps Region (n = 800). The heavier the care was, the more respondents preferred hospital care. Willingness to pay for additional taxation per household/month varied from €13.9 for light care in LHC to €19.1 for heavy home care. The small number of protesting respondents and outliers, and the close correlation between preferences, income, and WTP supports the validity of the CVS.

In France, for cancer, not all citizens would prefer to be treated at home rather than in a hospital. Only less than a quarter would prefer LHC. These results show the mismatch between public health policies and the citizens’ preferences.

Adherence to preventative inhaled therapies in people with cystic fibrosis (CF) is low, resulting in potentially avoidable health losses and the need for costly rescue therapies.

To estimate the cost-effectiveness of the CFHealthHub (CFHH) intervention to support adherence to inhaled medications.

A state transition model was developed to assess the cost-effectiveness of the CFHH intervention versus usual care from the perspective of the UK National Health Service and Personal Social Services over a lifetime horizon. Costs and health outcomes were discounted at a rate of 3.5 percent per annum. Costs were valued at 2021/22 prices. The model structure includes health states defined by survival status, level of lung function, and transplant history. Treatment effects were modeled by changing the probabilities of transitioning between lung function states and reducing exacerbation rates. Model parameters were informed by the CFHH trial, CF Registry data, routine cost databases, literature, and expert opinion. Deterministic and probabilistic sensitivity analyses were undertaken to assess uncertainty.

The CFHH intervention is expected to generate additional health gains and cost savings compared with usual care. Assuming that it is delivered for 10 years, the CFHH intervention is expected to generate 0.17 additional quality-adjusted life years and cost savings of GBP 1,600 (EUR 1,662) per patient.

The CFHH intervention is expected to dominate usual care, irrespective of the duration over which the intervention is delivered. The modeled benefits and cost savings are smaller than initially expected and are sensitive to relative treatment effects on lung function.

Numerous studies have shown that arthroscopic partial meniscectomy (APM) is not (cost-) effective in patients with symptoms attributed to a degenerative meniscus tear. We aimed to assess the budget impact of reducing APM in routine clinical practice in this population.

A patient-level state transition model was developed to simulate patients recently diagnosed with a degenerative meniscus tear. Three strategies were compared: “current guideline” (i.e., postpone surgery to at least 3 months after diagnosis), “APM at any time” (i.e., APM available directly after diagnosis), and “nonsurgical” (i.e., APM no longer performed). Total societal costs over 5 years were calculated to determine the budget impact. Probabilistic and deterministic sensitivity analyses were conducted to address uncertainty.

The average cost per patient over 5 years were EUR 5,077, EUR 4,577, and EUR 4,218, for the “APM at any time,” “current guideline,” and “nonsurgical” strategy, respectively. Removing APM from the treatment mix (i.e., 30,000 patients per year) in the Netherlands, resulted in a reduction in health care expenditures of EUR 54 million (95 percent confidence interval [CI] EUR 38 million–EUR 70 million) compared to the “current guideline strategy” and EUR 129 million (95 percent CI EUR 102 million–EUR 156 million) compared to the “APM at any time” strategy. Sensitivity analyses showed that uncertainty did not alter our conclusions.

Substantial costs can be saved when APM is no longer performed to treat symptoms attributed to degenerative meniscus tears in the Netherlands. It is therefore recommended to further reduce the use of APM to treat degenerative meniscus tears.

This study aimed to provide a structured description of the commonalities and differences in healthcare structures across Africa to establish a reliable basis for the health technology assessment (HTA) of nutrition and nutrition interventions. A ranking of current nutrition conditions in the general population of the participating countries was included to gain a better understanding of the factors influencing hospital malnutrition (HMN), which will inform future multi-country research.

A questionnaire on the structure of the health systems was distributed among ten African countries. Subsections were included that inquired about the drivers or barriers to using principles of HTA to assess nutritional care. Analysis and ranking of malnutrition data were based on data from the Global Hunger Index report and two poverty indicators used by the World Bank.

The health system structure of each country was identified and described, whereas questions about HTA could not always be analyzed due to a lack of adequate in-depth knowledge and skills in most countries. Early experience from some countries demonstrates a conceivable route ahead for African countries in strengthening the capacity for and implementing HTA in accordance with distinct national healthcare contexts and social determinants of health.

Problems related to nutritional care represent one of the major priorities in the surveyed countries. A future HMN multi-country study will provide valuable insight into the potential of low-cost primary prevention orientations.

Out of 185 orphan medicinal products (OMPs) registered in 2015–2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia.

The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients’ organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial.

We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of “value-based” decision making.

To report the processes used to design and implement an assessment tool to inform funding decisions for competing health innovations in a tertiary hospital.

We 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.

We 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.

We 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.

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.

An 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.

A 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.

It 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.

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.

A 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.

Seven 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.

There 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.

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.

A 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).

NPH 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).

There 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.

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.

Previous 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.

The 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.

Anchoring 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.

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.

To 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.

A 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.

A 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).

Functional, 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.

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.

A survey was undertaken of patient group (PG) representatives and clinicians who participated in PACE meetings, and SMC committee members.

PACE 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.

In 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.

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.

The 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.

Three 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.

All 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.

This 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.