Early Health Technology Assessment Research Articles

ESR Essentials: how to get to valuable radiology AI: the role of early health technology assessment-practice recommendations by the European Society of Medical Imaging Informatics.

AI tools in radiology are revolutionising the diagnosis, evaluation, and management of patients. However, there is a major gap between the large number of developed AI tools and those translated into daily clinical practice, which can be primarily attributed to limited usefulness and trust in current AI tools. Instead of technically driven development, little effort has been put into value-based development to ensure AI tools will have a clinically relevant impact on patient care. An iterative comprehensive value evaluation process covering the complete AI tool lifecycle should be part of radiology AI development. For value assessment of health technologies, health technology assessment (HTA) is an extensively used and comprehensive method. While most aspects of value covered by HTA apply to radiology AI, additional aspects, including transparency, explainability, and robustness, are unique to radiology AI and crucial in its value assessment. Additionally, value assessment should already be included early in the design stage to determine the potential impact and subsequent requirements of the AI tool. Such early assessment should be systematic, transparent, and practical to ensure all stakeholders and value aspects are considered. Hence, early value-based development by incorporating early HTA will lead to more valuable AI tools and thus facilitate translation to clinical practice. CLINICAL RELEVANCE STATEMENT: This paper advocates for the use of early value-based assessments. These assessments promote a comprehensive evaluation on how an AI tool in development can provide value in clinical practice and thus help improve the quality of these tools and the clinical process they support. KEY POINTS: Value in radiology AI should be perceived as a comprehensive term including health technology assessment domains and AI-specific domains. Incorporation of an early health technology assessment for radiology AI during development will lead to more valuable radiology AI tools. Comprehensive and transparent value assessment of radiology AI tools is essential for their widespread adoption.

Early health technology assessment of gene silencing therapies for lowering lipoprotein(a) in the secondary prevention of coronary heart disease

Olpasiran and pelacarsen are gene-silencing therapies that lower lipoprotein(a). Cardiovascular outcome trials are ongoing. Mendelian randomisation studies estimated clinical benefits from lipoprotein(a) lowering. Our study estimated prices at which olpasiran and pelacarsen, in addition to standard-of-care, would be deemed cost-effective in reducing risk of recurrent coronary heart disease (CHD) events in the Australian healthcare system. We developed a decision tree and lifetime Markov model. For olpasiran, participants had CHD and lipoprotein(a) 260 nmol/L at baseline and three-monthly injections, profiled on OCEAN(a) Outcomes trial (NCT05581303). Baseline risks of CHD, costs and utilities were obtained from published sources. Clinical trial data were used to derive reductions in lipoprotein(a) from treatment. Mendelian randomisation study data were used to estimate downstream clinical benefits. Annual discounting was 5 %. For pelacarsen, participants had CHD and lipoprotein(a) 226 nmol/L at baseline and one- monthly injections, profiled on Lp(a) HORIZON (NCT04023552) trial. Olpasiran in addition to standard-of-care saved 0.87 discounted quality-adjusted life years (QALYs) per person. Olpasiran in addition to standard-of-care would be cost- effective at annual prices of AU$1867 (AU$467 per dose) at threshold AU$28,000 per QALY. Pelacarsen would be cost-effective at annual prices of AU$984 (AU$82 per dose). For ICER threshold AU$50,000 per QALY, olpasiran and pelacarsen would be cost-effective at annual prices AU$4207 and AU$2464 respectively. This early health technology assessment model used inclusion criteria from clinical trials. Olpasiran and pelacarsen would be cost-effective if annual treatment prices were AU$1867 and AU$984 respectively, from the Australian healthcare perspective.

A study on the formative usability testing for modular powered wheelchair

The increasing prevalence of mobility impairments underscores the urgent need for accessible and affordable mobility aids. To overcome the mobility limitations of people with disabilities, there is an increasing need for the development of lightweight and portable powered wheelchairs that can be easily loaded. This study aimed to perform an early health technology assessment and a formative usability evaluation on a modular (detachable) powered wheelchair. It aimed to gauge device satisfaction among users, pinpoint areas for improvement, and detect any unforeseen errors to inform future development. Engaging 16 participants, including powered wheelchair users, healthcare professionals, and caregivers, the research evaluated the wheelchair’s functionality in various scenarios, emphasizing safety, effectiveness, and convenience. Statistical analyses of task performance and satisfaction surveys highlighted that, while powered wheelchair users successfully completed tasks focusing on driving and power control, healthcare professionals and caregivers encountered difficulties with the wheelchair’s assembly and disassembly. Despite general positivity, the surveys indicated mixed satisfaction levels regarding safety, validity, and convenience, with specific issues related to frame durability, seat comfort, and control mechanisms. These findings suggest that refining the wheelchair’s design and addressing user concerns could significantly enhance satisfaction and mobility services. Future efforts will include a thorough review of an advanced prototype and further satisfaction assessments.

Toward a Useful and Cost-Effective Workup in Chronic Polyneuropathy: The EXPRESS Study Protocol.

Knowledge gaps exist about the usefulness and extent of blood tests and nerve conduction studies in the workup of polyneuropathy. We hypothesize that a limited workup improves costs spent on diagnostics without loss of diagnostic reliability or disadvantageous effect on treatment choice in many patients with a clinical diagnosis of chronic polyneuropathy. We aim to determine which investigations are necessary in the workup of patients with suspected chronic polyneuropathy clinically diagnosed by neurologists in an outpatient clinic and will perform an early health technology assessment. This is a prospective multicenter quality in healthcare evaluation. We compare two diagnostic strategies, both performed on all participants: the standard care by each patient's neurologist and the proposed (limited) workup by the study panel members consisting of neurologists with experience in neuromuscular diseases. The primary outcome is the effectiveness of a limited workup expressed as concordance between the patient's neurologist diagnosis and the panel diagnosis. This will be related to differences in costs and impact on treatment or patient management otherwise. Other outcomes are burden/gain for the patient in terms of number of investigations, time to diagnosis, hospital visits, sick leave, loss of productivity, expenses, experienced quality of care. This multicenter prospective observational study on quality in health care will provide improved evidence about the components of a cost-effective workup for patients with chronic polyneuropathy.

HTA57 Uptake of Early Health Technology Assessment in the United States (US): A Literature Review of Empirical Studies

HTA57 Uptake of Early Health Technology Assessment in the United States (US): A Literature Review of Empirical Studies

The value of artificial intelligence for the treatment of mechanically ventilated intensive care unit patients: An early health technology assessment

PurposeThe health and economic consequences of artificial intelligence (AI) systems for mechanically ventilated intensive care unit patients often remain unstudied. Early health technology assessments (HTA) can examine the potential impact of AI systems by using available data and simulations. Therefore, we developed a generic health-economic model suitable for early HTA of AI systems for mechanically ventilated patients. Materials and methodsOur generic health-economic model simulates mechanically ventilated patients from their hospitalisation until their death. The model simulates two scenarios, care as usual and care with the AI system, and compares these scenarios to estimate their cost-effectiveness. ResultsThe generic health-economic model we developed is suitable for estimating the cost-effectiveness of various AI systems. By varying input parameters and assumptions, the model can examine the cost-effectiveness of AI systems across a wide range of different clinical settings. ConclusionsUsing the proposed generic health-economic model, investors and innovators can easily assess whether implementing a certain AI system is likely to be cost-effective before an exact clinical impact is determined. The results of the early HTA can aid investors and innovators in deployment of AI systems by supporting development decisions, informing value-based pricing, clinical trial design, and selection of target patient groups.

Artificial intelligence support in MR imaging of incidental renal masses: an early health technology assessment

ObjectiveThis study analyzes the potential cost-effectiveness of integrating an artificial intelligence (AI)–assisted system into the differentiation of incidental renal lesions as benign or malignant on MR images during follow-up.Materials and methodsFor estimation of quality-adjusted life years (QALYs) and lifetime costs, a decision model was created, including the MRI strategy and MRI + AI strategy. Model input parameters were derived from recent literature. Willingness to pay (WTP) was set to $100,000/QALY. Costs of $0 for the AI were assumed in the base-case scenario. Model uncertainty and costs of the AI system were assessed using deterministic and probabilistic sensitivity analysis.ResultsAverage total costs were at $8054 for the MRI strategy and $7939 for additional use of an AI-based algorithm. The model yielded a cumulative effectiveness of 8.76 QALYs for the MRI strategy and of 8.77 for the MRI + AI strategy. The economically dominant strategy was MRI + AI. Deterministic and probabilistic sensitivity analysis showed high robustness of the model with the incremental cost-effectiveness ratio (ICER), which represents the incremental cost associated with one additional QALY gained, remaining below the WTP for variation of the input parameters. If increasing costs for the algorithm, the ICER of $0/QALY was exceeded at $115, and the defined WTP was exceeded at $667 for the use of the AI.ConclusionsThis analysis, rooted in assumptions, suggests that the additional use of an AI-based algorithm may be a potentially cost-effective alternative in the differentiation of incidental renal lesions using MRI and needs to be confirmed in the future.Clinical relevance statementThese results hint at AI’s the potential impact on diagnosing renal masses. While the current study urges careful interpretation, ongoing research is essential to confirm and seamlessly integrate AI into clinical practice, ensuring its efficacy in routine diagnostics.Key Points• This is a model-based study using data from literature where AI has been applied in the diagnostic workup of incidental renal lesions.• MRI + AI has the potential to be a cost-effective alternative in the differentiation of incidental renal lesions.• The additional use of AI can reduce costs in the diagnostic workup of incidental renal lesions.

The added value of risk assessment and subsequent targeted treatment for epileptic seizures after stroke: An early-HTA analysis

IntroductionThe development of post-stroke epilepsy (PSE) is related to a worse clinical outcome in stroke patients. Adding a biomarker to the clinical diagnostic process for the prediction of PSE may help to establish targeted and personalized treatment for high-risk patients, which could lead to improved patient outcomes. We assessed the added value of a risk assessment and subsequent targeted treatment by conducting an early Health Technology Assessment. MethodsInterviews were conducted with four relevant stakeholders in the field of PSE to obtain a realistic view of the current healthcare and their opinions on the potential value of a PSE risk assessment and subsequent targeted treatment. The consequences on quality of life and costs of current care of a hypothetical care pathway with perfect risk assessment were modeled based on information from a literature review and the input from the stakeholders. Subsequently, the maximum added value (the headroom) was calculated. Sensitivity analyses were performed to test the robustness of this result to variation in assumed input parameters, i.e. the accuracy of the risk assessment, the efficacy of anti-seizure medication (ASM), and the probability of patients expected to develop PSE. ResultsAll stakeholders considered the addition of a predictive biomarker for the risk assessment of PSE to be of value. The headroom amounted to €12,983. The sensitivity analyses demonstrated that the headroom remained beneficial when varying the accuracy of the risk assessment, the ASM efficacy, and the number of patients expected to develop PSE. DiscussionWe showed that a risk assessment for PSE development is potentially valuable. This work demonstrates that it is worthwhile to undertake clinical studies to evaluate biomarkers for the prediction of patients at high risk for PSE and to assess the value of targeted prophylactic treatment.

PP127 Early Health Technology Assessment (HTA) Of Medical Technologies To Inform Subsidy Decision-making In Singapore

IntroductionMedical technologies are evolving rapidly, with many new and expensive technologies entering the market constantly, challenging the sustainability of the public healthcare system. Early health technology assessment (HTA) to inform subsidy decision for innovative medical technologies, before they diffuse into the public healthcare system, may drive appropriate early adoption or curtail inappropriate use. This abstract describes the Agency for Care Effectiveness (ACE)’s experience in conducting early HTAs and key challenges faced.MethodsDuring ACE’s 2021 topic prioritization exercise, ACE took a proactive approach by considering medical technologies identified from horizon scanning (HS) for subsidy evaluation. Two topics were shortlisted from HS. Standard HTA evaluation framework and local clinician consultation were used to define the evaluation scope and clinical pathways. Literature search and appraisal were conducted for safety, effectiveness, and economic evidence. Budget impact estimations and organizational feasibility assessment were additional domains considered for subsidy decision-making by the Ministry of Health Medical Technology Advisory Committee (MTAC).ResultsMTAC did not recommend subsidy for the two technologies due to weak evidence base, largely due to a lack of comparative evidence, small samples, short-term follow-ups, or heterogeneity of population. Additional considerations included potentially high budget impact or organizational feasibility issues such as substantial capital and maintenance cost and infrastructure reconfiguration required. During the evaluation, key challenges of assessing such technologies in their early diffusion within the healthcare system were: (i) differing clinical opinions on whether the technology meets an unmet need; (ii) uncertain place in the clinical management algorithm for the relevant indications; (iii) sparse and weak evidence; (iv) uncertain financial implications to the healthcare system due to a lack of available local costs.ConclusionsEarly HTA on medical technologies identified from HS can be a useful tool to guide subsidy decisions; however, several challenges exist. Careful selection of technologies and timing of evaluation are critical. Seeking stakeholder inputs earlier would ensure shortlisting appropriate technologies with greater clinical need for HTA.

OP104 Pilot Implementation Of Health Technology Assessment Topic Prioritization In The Philippines: Lessons And Plans For Moving Forward

IntroductionThe Health Technology Assessment (HTA) Council in the Philippines carried out its process tracks while the implementing guidelines were being finalized in 2020, due to the urgent need to respond to COVID-19. Two years later, as mandated by the Universal Healthcare (UHC) law, we opened the nominations for the HTA Council’s topic priority list, which will be assessed to inform government financing decisions.MethodsWe adopted the former Philippine National Formulary System (PNFS) but set the prioritization criteria according to the decision framework stipulated by the UHC law and allowed industry submissions. We streamlined dossier completion for topics with numerous proponents, supplemented dossier deficiencies, and adjusted the timelines of crucial steps for better reach, while accounting for possible setbacks during the time periods. We satisfied the prioritization criteria using a Delphi technique at the HTA Council subcommittee and Core Committee levels in conjunction with consultations with the Department of Health and the national payer. We also shared evidence base and topic prioritization criteria scores with stakeholders during the public consultation.ResultsIn the pilot implementation, we processed a total of 140 nominations (88 complete submissions) and released the priority list in five months. After processing 31 appeals from all key stakeholder groups, the 2022 priority list covered 31 assessments based on topics from the Department of Health, the national payer, industry, hospitals, and medical societies. Although we found gaps in the set timelines, inadequacy in the prioritization criteria parameters, and the need to increase exposure of the public to the process, we were able to accommodate all stakeholder concerns and maintain flexibility in the process.ConclusionsWe need to update our HTA process guidelines, accept joint dossier submissions, and review our topic prioritization process. The changing health system landscape and transitioning of national health priorities require coordination with the Philippine Food and Drug Administration for horizon scanning, early HTA, and managed entry agreements. Finally, there is a need to create special pathways for rare disease and innovative technologies.

OP66 Adoption Of The World Health Organization Algorithm For Essential Medicines In The Philippine National Formulary Listing Process

IntroductionThe Philippine National Formulary (PNF) System preceded the health technology assessment (HTA) process in the Philippines, which was institutionalized in 2019. The transition led to previously prioritized topics of expert bodies overseeing the PNF System being endorsed to the HTA Council. However, the advent of COVID-19 forced the HTA Philippines to focus on emergency assessment needs and financing recommendations for the national government, resulting in limited capacity to assess non-public health emergency topics. To address this and improve patient access to medicines, we adopted the World Health Organization (WHO) process for evaluating and selecting medicines in the National Essential Medicines List (NEML).MethodsIn assessing the pre-pandemic topics, we matched the population, intervention, comparator, and outcomes of the WHO clinical evidence reviews with those scoped with relevant stakeholders and performed local costing analyses to ensure applicability of findings to the Philippine setting. When needed, we subjected the topics to price negotiation or conducted qualitative assessments.ResultsWe found the method efficient in expediting the decision-making process of the HTA Council. However, given the limited internal capacity of the HTA Philippines to conduct assessments for all ongoing HTA tracks, some of the topics responsive to Universal Health Care will be outsourced to the HTA Research Network, which is yet to be established. There is also a need to improve alignment among the topics being assessed, since the priorities of the proponents, national health program, and national payer have already evolved.ConclusionsIt is important to identify the priority areas for stakeholders as part of the topic nomination process, account for analytic capacity when setting the number of topics for HTA, establish mechanisms to allow proponents to conduct HTAs based on the HTA Council’s methodological standards, and proactively work with the national regulatory agency on horizon scanning and early HTA. We also recommend efficient monitoring, evaluation, and updating of the Philippine HTA guidelines so that they are more responsive to the needs of the healthcare system and the Filipino people.

Comparison of Elicitation Approaches in Early Stage HTA Applied on Artificial Thymus for Patients with DiGeorge Syndrome.

This paper focuses on research in expert elicitation as a part of the early stage health technology assessment (eHTA). The current state of affairs is analysed and two elicitation approaches are compared-the four fixed intervals method and the histogram method-as applied to an example of early assessment of clinical effectiveness of artificial thymus for patients with DiGeorge syndrome. A survey was carried out consisting of four questions concerning the topic, with the aim to apply the elicitation methods. Eight experts answered the questions using both elicitation methods. Based on their answers, the methods were compared visually and by means of statistical tests. In order to compare the perception of the two elicitation methods, the survey also included questions regarding the experts' subjective preferences. The results of the comparison of the two elicitation approaches did not clearly confirm which method was more beneficial and better; however, it was possible to indicate which of the two methods is better suited for different types of experts. Before selecting an elicitation method as a part of eHTA, it is advisable to effectively consider the technology to be assessed and the type of experts to be invited to share their opinion.

Early practice of use of video consultations in rehabilitation of hand injuries in children and adults: Content, acceptability, and cost-effectiveness

BackgroundVideo consultation was implemented as a new service in a hospital hand therapy setting. PurposeTo describe the first year’s practice of video consultations in the rehabilitation of upper extremity injuries, evaluate the acceptability, and investigate economic effects. Study DesignIterative design including economic evaluation. MethodsUsing the framework early health technology assessment, 13 hand therapists described characteristics of 99 video consultations, under predefined headlines: the patients’ municipally, adult vs child, time use, technical, content, and usefulness compared to physical consultations. The text was coded and categorized according to 22 techniques or tools used by hand therapists, and challenges were identified. Acceptability was assessed on a three-graded adjectival scale. To illustrate the costs associated with video vs physical consultations, we drafted different scenarios based on the data and stakeholder insights. ResultsOf 99 planned video consultations (16 with children), 88 were completed. Techniques or tools most frequently used were the performance of exercises (n = 55), orthoses (n = 26), and daily activities (n = 23). Technical challenges were common, and observation of children could be difficult. Eleven of the completed consultations were rated as not acceptable and 77 as acceptable and as either useful (n = 28) or very useful (n = 49). Four drafted scenarios showed cost savings of video consultations for the health institution and society, highest at longer travel distances and in other cases where the patient could claim refunds related to travel and time away from work and home. ConclusionsThe results show therapeutic possibilities and points to areas for improvements and illustrate settings where the use of video may save costs for the health institution and society.

Development and validation of Medical Device Key Evidence Tool ('MeDKET'): An evidence-based framework to explain success in selected European and US companies.

Innovating in Medical Device (MD) industry is challenging. This study aims to develop and validate an evidence-based framework that helps innovators of small and large enterprises (SEs and LEs) assess their readiness for successful MD development and deployment. We conducted a key-informant process (stage 1) where 25 international experts identified a list of emergent Health Technology Assessment (HTA) themes they believed were essential to company success. A sample of 22 European and US selected companies (13 SEs and 9 LEs) then reached a consensus on a list of key themes through a robust Delphi process (stage 2). Finally, we constructed (stage 3) and validated (stage 4) the checklist for SEs and LEs. The checklist for SEs and LEs included 21 and 15 items (i.e., fundamental Yes/No questions) with nine overlapping criteria for both SEs and LEs. In both groups, MD success was driven by three major item categories: (i) R&D assessment strategy; (ii) device-outcome measures; (iii) company profiling. Alongside the retrospective validation study, we collected 40 case studies on MDs (23 successes and 17 failures) across the selected enterprises. The retrospective validation provided the proportion of successful and failed case studies that met the 'MeDKET' criteria. We discovered that early HTA plays a pivotal role in MD industry success with different implications based on enterprise size. This study is the first of its kind to provide a holistic picture of the perceived role of early-stage HTA in MD industry success.

IMproving facial PRosthesis construction with contactlESs Scanning and Digital workflow (IMPRESSeD): study protocol for a feasibility crossover randomised controlled trial of digital versus conventional manufacture of facial prostheses in patients with orbital or nasal facial defects

BackgroundFacial prostheses can have a profound impact on patients’ appearance, function and quality of life. There has been increasing interest in the digital manufacturing of facial prostheses which may offer many benefits to patients and healthcare services compared with conventional manufacturing processes. Most facial prosthesis research has adopted observational study designs with very few randomised controlled trials (RCTs) documented. There is a clear need for a well-designed RCT to compare the clinical and cost-effectiveness of digitally manufactured facial prostheses versus conventionally manufactured facial prostheses. This study protocol describes the planned conduct of a feasibility RCT which aims to address this knowledge gap and determine whether it is feasible to conduct a future definitive RCT.MethodsThe IMPRESSeD study is a multi-centre, 2-arm, crossover, feasibility RCT with early health technology assessment and qualitative research. Up to 30 participants with acquired orbital or nasal defects will be recruited from the Maxillofacial Prosthetic Departments of participating NHS hospitals. All trial participants will receive 2 new facial prostheses manufactured using digital and conventional manufacturing methods. The order of receiving the facial prostheses will be allocated centrally using minimisation. The 2 prostheses will be made in tandem and marked with a colour label to mask the manufacturing method to the participants. Participants will be reviewed 4 weeks following the delivery of the first prosthesis and 4 weeks following the delivery of the second prosthesis. Primary feasibility outcomes include eligibility, recruitment, conversion, and attrition rates. Data will also be collected on patient preference, quality of life and resource use from the healthcare perspective. A qualitative sub-study will evaluate patients’ perception, lived experience and preference of the different manufacturing methods.DiscussionThere is uncertainty regarding the best method of manufacturing facial prostheses in terms of clinical effectiveness, cost-effectiveness and patient acceptability. There is a need for a well-designed RCT to compare digital and conventional manufacturing of facial prostheses to better inform clinical practice. The feasibility study will evaluate key parameters needed to design a definitive trial and will incorporate early health technology assessment and a qualitative sub-study to identify the potential benefits of further research.Trial registrationISRCTN ISRCTN10516986). Prospectively registered on 08 June 2021, https://www.isrctn.com/ISRCTN10516986.

Cost-Effectiveness Analysis of CT-Based Finite Element Modeling for Osteoporosis Screening in Secondary Fracture Prevention: An Early Health Technology Assessment in the Netherlands.

For postmenopausal Dutch women who have been referred to the FLS, direct access to CT2S may be cost-effective compared with DXA for age groups 70s and 80s, when considering the ICER threshold of the Netherlands. This study positions CT2S as a potential novel osteoporosis-screening tool for secondary fracture prevention in the clinical setting.A shorter screening interval of 2 y increases the effectiveness of both screening strategies, but the ICER of CT2S compared with DXA also increased substantially, which made CT2S no longer cost-effective for the 70s age group; however, it remains cost-effective for individuals in their 80s.Annual zoledronic acid treatment with better adherence may contribute to a lower cost-effectiveness ratio when comparing CT2S to DXA screening and the treat all strategies for all age groups.

The value of maintaining cognition in patients with mild cognitive impairment: The innovation headroom and potential cost-effectiveness of roflumilast.

Early health-technology assessment can support discussing scarce resource allocation among stakeholders. We explored the value of maintaining cognition in patients with mild cognitive impairment (MCI) by estimating: (1) the innovation headroom and (2) the potential cost effectiveness of roflumilast treatment in this population. The innovation headroom was operationalized by a fictive 100% efficacious treatment effect, and the roflumilast effect on memory word learning test was assumed to be associated with 7% relative risk reduction of dementia onset. Both were compared to Dutch setting usual care using the adapted International Pharmaco-Economic Collaboration on Alzheimer's Disease (IPECAD)open-source model. The total innovation headroom expressed as net health benefit was 4.2 (95% bootstrapinterval: 2.9-5.7) quality-adjusted life years (QALYs). The potential cost effectiveness of roflumilast was k€34 per QALY. The innovation headroom in MCI is substantial. Although the potential cost effectiveness of roflumilast treatment is uncertain, further research on its effect on dementia onset is likely valuable.

A first exploration of the economic consequences of an autonomous surgical robot for lateral skull base surgery: an early health technology assessment.

Lateral skull base procedures, such as translabyrinthine approach (TLA), are challenging. An autonomous surgical robot might be a solution to these challenges. Our aim is to explore in an early phase the economic consequences of an autonomous surgical robot compared with conventional TLA. An early decision analytic model was constructed in order to perform a step-wise threshold analyses and a sensitivity analysis to analyze the impact of the several factors on the incremental costs. Using surgical robot results in incremental costs - EUR 5,562 per procedure - compared to conventional TLA. These costs are most reduced by higher number of procedures, followed by lower price of the robot, saved operation time, and reduced risk of complication, respectively. The incremental costs of using an autonomous surgical robot can be decreased by choosing applications with a high turnover rate, a long operation time, and a high complication rate.

Using early health economic modeling to inform medical innovation development: a soft robotic sock in poststroke patients in Singapore.

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.

Patient-based evidence: its role in decision making on end-of-life, orphan, and ultra-orphan medicines.

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.