Medical devices cause significant spending in healthcare systems and provide methodological challenges for health technology assessment (HTA). This poster presents the results of a dissertation on the HTA of prosthetic limb technology and care, with a focus on the ethical implications of this aspect of health care. It is also a case study for ethics in HTA more generally, which will contribute to methodological discussions in this field.

The methodology was based on Hofmann´s Socratic approach and empirical ethics. Literature reviews of HTA reports and ethics and social science literature on limb prosthetics and care were supplemented by semi-structured interviews with 16 prosthesis wearers and 18 stakeholders (e.g., insurance payers and orthopedic technicians) from Germany. This material served to identify and prioritize ethical issues and dive deeper into the causes of unequal access to prosthetic limbs. Beauchamp and Childress´ principlism and Norman Daniels’ theory of just health were used to describe ethical requirements and conflicts and to discuss the limits of normative recommendations that HTA can provide for decision-makers.

There were 42 ethical aspects related to limb prosthetic technology and care identified, reflecting also general challenges for HTA like artificial intelligence in health and resource scarcity in times of multiple crises. The perspectives of patients and stakeholders provided evidence for unequal access to limb prosthetics that was dependent on socioeconomic status, age, and living region. This is due to a combination of legal framework conditions (not supporting evidence-based reimbursement), socioeconomic success factors in interaction with gatekeepers, non-optimal quality of care (due to lack of data use and scarcity of professionals), and political non-willingness to address rationing–as well as the lack of HTAs.

Access issues in prosthetic care and their implications for patient wellbeing, efficiency, and sustainability may be generalizable to a certain extent to other medical device types and healthcare systems. Governments should provide resources, and synergies with health services research could be leveraged to enable HTA to address the challenges of medical device and ethical assessment.

An evidence synthesis developed to inform decision-making on the use of vitamin D for preventing and treating COVID-19 showed that current available evidence is of low to very low quality. We set up a rigorous living evidence to inform health decisions (LE-IHD) approach to provide timely updates of this health technology assessment (HTA) report and aid decision-making.

Following the LE-IHD framework, we developed a baseline synthesis and evidence monitoring on the effects of high-dose vitamin D for the prevention and treatment of severe COVID-19 on all-cause mortality, COVID-19-related hospitalization, intensive care unit admission, length of hospital stay, quality of life, adverse events, and long COVID-19. The evidence identification, screening, and selection processes were supported by Epistemonikos technological enablers and the Living Overview of Evidence platform. We searched for ongoing studies in trial registries every three months. New eligible studies were assessed using a systematic and reproducible process to update the HTA report.

For the baseline synthesis we identified nine randomized control trials (RCTs) assessing high dose vitamin D2, vitamin D3, and their metabolites, which provided very low quality evidence on all the outcomes of interest. Up to date evidence monitoring identified seven studies reporting on all-cause mortality and intensive care unit admission, eight studies reporting on length of hospital stay, and six studies reporting on adverse events. The living evidence synthesis has been updated twice. At the time of the conference, we will report on 10 months of monitoring results and any substantial updates to the HTA report.

For HTA reports based on low and very low quality evidence (uncertain results), the living evidence approach allows for timely updating of conclusions. The LE-IHD framework facilitates the planning and execution of living evidence syntheses to inform health decisions. This living evidence synthesis is being developed as part of a project to strengthen decision-making capacity in the Spanish health system.

Diabetes has been shown to influence the individual’s work productivity in terms of both presenteeism (reduced productivity while at work) and absenteeism (absence from work because of illness). We sought to estimate the potential productivity gains associated with the modification of cardiovascular disease (CVD) risk in type 2 diabetes (T2D) over the next 10 years in Australia, from 2023 to 2032.

Dynamic models were structured to estimate productivity-adjusted life years (PALYs) lived by Australians aged 20 to 69 years. The model simulation was first undertaken assuming currently expected trends in the incidence of myocardial infarction (MI) and stroke in T2D (original cohort), as calculated using the 2013 PCE-ASCVD algorithm. Subsequent models were then re-simulated using hypothetical scenarios that reflected the potential benefits of CVD reduction using published trials. The model was also repeated assuming that the original cohort has no CVD. Differences in PALYs lived by the “original cohort” and the different cohorts with reduced CVD risk reflected the PALYs gained. Sensitivity analyses were conducted.

Using data from published studies, the model assumed a reduction of 50 percent in systolic blood pressure (SBP), a reduction of 50 percent in smoking, 50 percent increase in high-density lipoprotein cholesterol (HDL), and a reduction of 35 percent in incidence of T2D. Over the working lifetime, from 2023 to 2032, reducing SBP, smoking, and incidence of T2D led to the gain of 140,105, 333,127, and 998,805 PALYs, respectively. Further, increasing HDL and assuming the original cohort with T2D has no CVD are expected to lead to the gain of 71,623 and 889,455 PALYs, respectively. Sensitivity analyses confirmed the robustness of study findings.

The impact of CVD as a complication from T2D on work productivity is significant. Screening and prevention strategies tailored early in life are likely to exert a positive impact on health and work productivity.

The diagnosis intervention packet (DIP) is a health insurance payment method with Chinese characteristics. Because the DIP is easy to understand and operable, it has become the main payment method promoted by China’s National Health Insurance Bureau. The development and reform of the DIP policy, which was introduced in 2020, is worth evaluating.

Policy texts were selected using conceptual sampling and expert interviews. The basic framework of policy evaluation was determined using text mining and statistical analysis methods. Word frequency analysis of the DIP payment policy content was undertaken using NVivo and Gephi software to compare the scope of concern before and after policy implementation. Quantitative evaluation of representative DIP health insurance payment policy content at the national level in China before and after policy implementation (2020 to 2022) was conducted by constructing a text mining and policy modeling consistency (TM-PMC) index model containing nine primary variables and 38 secondary variables.

Policy content analysis using text mining tools revealed that DIP-related policy themes were relatively concentrated, primarily focusing on disease types, medical institutions, and directories. These themes continue to be consistently updated. The quantitative results of the TM-PMC index model showed that the overall design of the policies was reasonable, but there was a noticeable variation in differentiation between the policies. Out of the eight policies analyzed, five were rated as excellent, two as good, and one as acceptable.

The DIP policy is subject to continuous supplementation and optimization. The main factors that influence the value of the TM-PMC index for the policy include the following: policy objectives, policy objects (the groups for which the policy is implemented), policy tools, and policy perspectives. Therefore, various reforms related to collection and payment should be carried out in a locally adapted and standardized manner.

Use of multiparametric magnetic resonance imaging (mpMRI) in the prostate cancer (PCa) diagnostic pathway could reduce prostate biopsy (Bx) in Bx-naive patients. The objective was to assess the feasibility of implementing a new PCa diagnostic pathway with the addition of mpMRI in a real care environment.

Following an HTA report published in 2019 by our team, a committee involving stakeholders (e.g., urologists, radiologists, hospital managers) was created to review the PCa diagnostic pathway including mpMRI in Bx-naive patients and both systematic and targeted 3DTRUS-MRI fusion Bx when Bx was recommended. Data in the new PCa diagnostic pathway were collected between September 2021 and June 2022. The comparison group is a cohort of 629 men who underwent an initial systematic transrectal ultrasound Bx in 2017 when prostate mpMRI was not available. Clinically significant PCa (csPCa) was defined as Grade Group ≥2.

In 2021 and 2022, 1,336 Bx-naive patients were referred to a urologist. Recommendations were: 703 (53%) for follow-up in six to 12 months, 254 (19%) directly to systematic Bx, and 379 (28%) to mpMRI. Overall, csPCa was diagnosed in 246/427 (58%) patients referred to mpMRI or Bx in the 2021 to 2022 cohort compared to 274/629 (44%) patients in the 2017 cohort (p<0.05). The new diagnostic pathway prevented 33 percent of patients from having Bx. Shorter delays between initial consultation with urologists and transmission of Bx results were observed for patients referred directly for prostate Bx compared to mpMRI before Bx (mean: 2.8 vs 9.1 months).

Experimentation in a real care setting has highlighted the added value of the early involvement of urologists in the diagnostic pathway of prostate cancer for the triage of patients. Integration of mpMRI was associated with a lower number of patients referred for prostate Bx and a higher csPCa detection rate.

Since April 2017, National Health Service England (NHSE) has been granted new powers to negotiate directly with pharmaceutical companies offering innovative, high value medicines. This research systematically evaluated all innovative therapies that have undergone commercial discussions with NHSE.

NHSE press releases for the period from 1 April 2017 to 10 November 2023 were screened for drug reimbursement decisions and the corresponding National Institute for Health and Care Excellence (NICE) assessments were identified. Key information was extracted, including reimbursement decision, date of decision, and type of commercial deal.

NHSE announced the conclusion of commercial discussions for 36 therapies (four in 2017, three in 2018, nine in 2019, two in 2020, nine in 2021, seven in 2022, and two in 2023). Of these, 27 NHSE commercial discussions were associated with positive NICE guidance; 12 of the 27 preceded the NICE final appraisal determination. In addition, three of the 36 therapies were associated with negative NICE appraisals (not recommended or non-submissions), one had an ongoing NICE assessment, and five did not go through NICE (subject to clinical commissioning policies). The specific type of agreement was not typically stated, but two were outcomes-based agreements, three were budget neutral, one was a portfolio-wide agreement, and one was a population health agreement.

NHSE is becoming an increasingly active and important stakeholder in medicines access. The UK may become an increasingly important early launch market for certain therapies, as evidence by the Medicines and Healthcare products Regulatory Agency joining Project Orbis and the Access Consortium and Casgevy (the first CRISPR-based gene therapy) receiving its first global marketing authorization in the UK.

Humanitarian aid, including food aid, has increasingly shifted towards provision of cash assistance over in-kind benefits. This paper examines whether food security mediates the relationship between receipt of humanitarian cash transfers and subjective wellbeing among Syrian refugee youth in Jordan.

Secondary analysis of the 2020-21 Survey of Young People in Jordan, which is nationally representative of Syrian youth aged 16-30. We employ stepwise model building and structural equation models.

Jordan.

Syrian refugee youth aged 16-30 (n = 1,572).

While 92% of Syrian households with youth received cash transfers from a United Nations agency, 78% of households were food insecure using the Food Insecurity Experience Scale. Fifty-one percent of youth suffered from poor wellbeing using the WHO-5 subjective wellbeing scale. Household food insecurity was associated with poorer youth wellbeing. Receiving larger cash transfer amounts was associated with better wellbeing among Syrian youth in unadjusted models. The relationship between receipt of cash transfers and youth wellbeing was not mediated by food security.

We do not find support for the hypothesis that food security is a mediator of the association between cash transfers and subjective wellbeing for this population.

There is no comprehensive framework that considers the various features of current funding models on drug accessibility for rare diseases; such a framework would assist policymakers to more effectively meet the challenges of these patients. This article reviews the funding models implemented worldwide to facilitate this access.

The PRISMA guidelines were used to conduct a systematic literature review. The following databases were searched: Ovid (Embase/MEDLINE), Cochrane database, Web of Science, EconLit, the National Institute for Health and Care Research (NIHR), Centre for Review and Dissemination (CRD), and International Network of Agencies for Health Technology Assessment (INAHTA). Two independent reviewers screened all titles and abstracts, and one reviewer did the full-text review and data extraction. Data were collected on general study characteristics, general aspects of rare diseases, source of funding, allocation of resources, and pricing strategies.

A total of 3,815 unique citations were screened, and 148 were included for data extraction. Each funding model was characterized based on its unique features specific to rare diseases, focusing on process, methods applied, and consideration of attributes. Sixty funding models were identified in 41 countries, categorized as separate processes (42%), exceptions to standard processes (32%), standard processes with no changes (23%), and alternative pathways (3%). More than one funding model was available for 29 percent of countries. Funding models varied in their approach to HTA, source of funding, consideration of uncertainty, and pricing strategies.

The diversity of funding models highlights the complexity of addressing access to treatments for rare diseases. Special considerations towards rare diseases generally targeted the greater uncertainty in the clinical evidence. Despite the existing platform that enables access for drugs for rare diseases, only 10 percent of rare diseases have an available treatment and fewer patients can access these technologies.

In 2022, a group of health technology assessment (HTA) bodies from Australia, Canada, and the UK announced a collaboration to identify solutions to common challenges. This collaboration was later expanded to include agencies from New Zealand and Quebec, Canada. Since one possible activity of the consortium is joint assessments, we compared the methodologies of the agencies on 11 topics to assess the feasibility of this.

We reviewed the methodological guidelines of the Canadian Agency for Drugs and Technologies in Health (CADTH), L’Institut national d’excellence en santé et services sociaux (INESSS), the National Institute for Health and Care Excellence (NICE), the Pharmaceutical Benefits Advisory Committee (PBAC), the Pharmaceutical Management Agency (Pharmac), and the Scottish Medicines Consortium (SMC). The topics considered were real-world evidence, consideration of health effects, economic reference case, survival analysis, surrogate endpoints, patient involvement, uncertainty, orphan pathways, clinical evidence requirements, carer perspective, and decision modifiers. We analyzed the level of alignment across the collaborating agencies using information from the guidelines, supplemented by published literature where necessary.

Three topics exhibited high alignment: consideration of health effects, clinical evidence requirements and surrogate endpoints. The topics of orphan pathways and carer perspective had low alignment. The remaining topics had moderate alignment. Regarding orphan pathways, NICE and the SMC had separate processes for ultra-orphan drugs, CADTH and INESSS implicitly consider rarity, and PBAC and Pharmac do not appear to consider rarity. Since carer perspective is not commonly accepted in HTA, NICE was the only agency with relevant guidance on this topic. INESSS required the societal perspective as standard, while the PBAC and Pharmac explicitly excluded it. CADTH may consider carer perspective in some circumstances, whereas the SMC guidance was ambiguous.

While there is good alignment on most topics, there are several areas where agencies would need to resolve divergences in preferred methodology if joint assessments are going to be carried out in the future. All relevant stakeholders should be part of this process, including patient groups and industry.

Deployment of an electronic automated advisory vital signs monitoring and notification system to signal clinical deterioration is associated with significant improvement in clinical outcomes. This study aimed to estimate the incremental cost per quality-adjusted life-year (QALY) gained with an electronic automated advisory notification system, compared with standard care.

A decision analytic model was developed to estimate the cost effectiveness of an electronic automated advisory notification system, compared with standard care, in adults admitted to a district general hospital. Analyses considered the following: (i) cost effectiveness (cost/event avoided) based on a before-and-after study (n=3,787) that recorded rates of acute myocardial infarction, pulmonary embolism, acute pulmonary edema, respiratory failure, stroke, severe sepsis, acute renal failure, cardiopulmonary arrest, admission to the intensive care unit, and death; and (ii) the cost utility (cost per QALY) over a lifetime horizon extrapolated using published data. The analysis was conducted from the perspective of the National Health Services (NHS) in the UK.

The automated notification system was more effective (2.7 fewer events per 100 patients) and provided cost savings of −GBP12.17 [−EUR14.07] per patient admission (95% CI: −GBP182.07 [−EUR211.20], GBP154.80 [EUR179.57]). The automated notification system was dominant over a lifetime horizon, demonstrating a positive incremental QALY gain (0.0287 QALYs, equivalent to approximately 10 days of perfect health) and a cost saving of −GBP55.35 (−EUR64.02). At a threshold of GBP20,000 per QALY (EUR23,126), the probability of automated monitoring being cost effective in the NHS was 0.81. The increased use of cableless sensors may reduce cost-savings, but the intervention remained cost effective at 100 percent usage (incremental cost-effectiveness ratio GBP3,107 per QALY [EUR3,594 per QALY]).

An automated notification system for adult patients admitted to general wards appears to be a cost-effective strategy in the NHS. The analysis suggests that adopting this technology could be good use of scarce resources. The impact of automated monitoring solutions on staffing warrants further exploration and may show additional value in adopting such technology.

Budget impact analyses for the treatment of rare diseases are especially important for the sustainability of health systems due to high treatment costs and uncertainties in target population estimates. The objective of this work is to analyze the elements that influence discrepancies between predicted and observed budget impacts for enzyme replacement therapies for rare diseases in Brazil’s public health system.

All enzyme replacement therapies for rare diseases evaluated by the National Committee for Health Technology Incorporation in the Brazilian Public Health System (Conitec) and with at least one year of use were included. For each technology, the following were identified: number of patients, median patient weight, annual quantity of medication, unit price, and budget impact. The attributes were compared between previous estimates and real-world observation after use. The data sources were publicly accessible administrative databases and Conitec technical reports.

Five technologies were selected: elosulfase alfa, alglucosidase alfa, idursulfase, laronidase, and galsulfase. In the first year, the difference between the estimated and the observed number of patients treated was up to 15 percent lower or higher for four technologies, but with monthly fluctuation throughout the year. The median weight of users was between 23 percent and 468 percent higher for three technologies. The observed price was as expected, with variations between three percent lower and 14 percent higher. The quantity of medicines used was lower (between 39% and 46%) than expected for all technologies. The observed budget impact was 37 percent to 47 percent lower than estimated.

Real-world budget impact was lower than expected for all technologies. The main cause of discrepancies was the estimate of the annual amount of medication, which did not consider gradual adherence and discontinuation of treatment. This highlights the need to review the budget impact methodology for rare diseases, forecasting monthly market share and treatment discontinuation rate.

Transcatheter aortic valve replacement (TAVR) has become a leading treatment for patients with severe aortic stenosis (AS). Recent studies comparing TAVR outcomes with balloon-expandable valves (BEVs) and self-expandable valves (SEVs) show generally similar results, although BEVs have lower rates of moderate-to-severe aortic regurgitation and pacemaker implantation. This study aimed to compare the clinical outcomes of SEVs and BEVs in Taiwan.

The Taiwan National Health Insurance Research Database (NHIRD) is a representative claims database capturing 99.9 percent of residents. We identified patients who underwent TAVR with either a SEV or BEV in 2021 using the NHIRD. The outcomes were six-month survival, length of hospital stay (LOS) and intensive care unit (ICU) stay, postoperative complications, and healthcare expenditure. We used inverse probability of treatment weighting (IPTW) based on age, gender, and Charlson Comorbidity Index score to identify the effect of TAVR valve type on LOS and ICU stay, postoperative complications, and healthcare expenditure. Differences between SEVs and BEVs for IPTW-weighted Kaplan-Meier curves of overall survival were measured with the log rank test.

Among the patients identified who underwent TAVR, 366 received a SEV and 132 received a BEV. The mean ages were 82.70 (standard deviation [SD] 8.08) years and 82.25 (SD 7.53) years, respectively. The hazard ratio for six-month mortality rate for SEVs compared with BEVs was 2.78 (95% confidence interval 1.52, 5.09). The six-month mortality rate was also significantly higher for SEVs than for BEVs (13.11% versus 4.55%). For clinical outcomes, the mean total LOS (14.78 [SD 12.19] versus 14.45 [SD 12.96] days), mean ICU stay (5.91 [SD 9.78] versus 6.23 [SD 8.04] days), rate of complications (<3%) and in-hospital healthcare costs (USD43,285 [SD 11,993] versus USD42,920 [SD 13,931]) were similar for both groups. The results were also similar after weighting.

Patients in Taiwan who underwent TAVR with BEVs had better survival outcomes than those who received SEVs, while other clinical and cost outcomes were comparable between the valve types.

The use of real-world evidence (RWE) by health technology assessments to evaluate emerging technologies has increased. Although traditionally recognized as the gold standard of evidence, randomized controlled trials (RCTs) may be challenging to conduct, especially in surgical settings. The aim of this analysis is to synthesize and compare results from RWE with those from RCTs for robotic-assisted surgery (RAS).

A systematic review was performed according to PRISMA methods. RWE and RCT studies published between 1 January 2010 and 31 December 2022 and comparing RAS, laparoscopic, or open surgery across seven procedures were included. Perioperative outcomes of interest were operative time, length of stay (LOS), conversion to an open procedure, estimated blood loss (EBL), blood transfusions, readmissions, reoperations, postoperative complications, and mortality. A meta-analysis was performed using R software.

Thirty-three RCTs and 121 RWE studies were included. For RAS versus laparoscopy, RCTs and RWE were concordant for conversions (RCT:OR=0.56 [0.42, 0.74], p<0.01; RWE:OR = 0.41 [0.36, 0.47], p<0.01) and LOS in favor of RAS (RCT:WMD = −0.66 [−1.12, −0.20], p<0.01; RWE:WMD = −0.50 [−0.63, −0.36], p<0.01), while operative time was longer for RAS (RCT:WMD = 27.89 [12.66, 43.12], p<0.01; RWE:WMD = 28.89 [15.56, 42.22], p<0.01). RWE complemented RCTs on blood transfusions and mortality, showing RAS favored over laparoscopy. For RAS versus open surgery, RCTs and RWE agreed RAS had significantly lower EBL (RCT:WMD = −260.42 [−515.16, −5.67], p = 0.05; RWE: WMD = −328.27 [−474.08, −182.47], p<0.01), lower postoperative complications (RCT:OR = 0.70 [0.50, 0.97], p = 0.03; RWE:OR = 0.56 [0.50, 0.62], p<0.01), shorter LOS (RCT:WMD = −1.88 [−3.12, −0.64], p<0.01; RWE:WMD = −1.95 [−2.20, −1.70], p<0.01), and longer operative time (RCT:WMD = 35.38 [2.14, 68.61], p=0.04; RWE:WMD = 38.80 [24.62, 52.97], p<0.01). For the remaining outcomes, RCTs showed no difference, while RWE provided complementary results in favor of RAS.

RWE confirmed many of the results shown in RCTs and complemented findings for perioperative outcomes. Based on these results, RWE can supplement the findings from RCTs in the literature, provide more generalizability, and offer a more comprehensive landscape of the evidence on robotic-assisted surgery.

Congenital heart disease (CHD), the most common congenital malformation affecting fetuses and infants, poses a significant and rapidly emerging global challenge in children’s health. Prenatal and newborn screening are very important for preventing CHD. Therefore, this study aimed to analyze the status and corresponding foci of articles on CHD screening in the Chinese or English language using bibliometric methods.

Publications on prenatal or newborn screening for CHD were included. The Web of Science Core Collection (WoS) and China National Knowledge Infrastructure (CNKI) databases were searched to identify literature published from inception to 31 March 2023. CiteSpace was used to perform bibliometric analysis on the number of publications, institutions, authors, and keywords to generate corresponding knowledge maps.

A total of 582 publications were retrieved from the WoS and 233 from the CNKI databases. There was an increasing trend in the number of English and Chinese articles published, with the trend beginning in 2010 for Chinese language articles and in 2007 for English language articles. In English language publications, GR Martin was the most influential author, and the top five institutions were from high-income countries. Among the Chinese language publications, Wenhong Ding was the most influential author and the Hunan Province Maternal and Child Health Care Hospital was the most commonly reported institution. Keyword analysis revealed that the most frequently occurring terms in both language publications were as follows: antenatal diagnosis, cardiac auscultation, and fetal echocardiography in English language articles and screening, prenatal screening, and fetal in Chinese language publications.

Increasingly, articles on CHD screening have been listed in the WoS and CNKI databases. This bibliometric study provides the status and trends in the research on screening for CHD and may help researchers identify hot topics and explore new research directions in this field.

Patients diagnosed with low- to intermediate-1-risk myelodysplastic syndrome (LR-MDS) encounter symptom burden that diminishes their health-related quality of life (HRQoL). Erythropoiesis-stimulating agents (ESAs) remain an option to alleviate anemia-related symptoms. However, existing HRQoL studies show limited evidential support. This study assesses the impact of ESAs on LR-MDS patients’ EuroQol 5-dimension questionnaire (EQ-5D) scores compared to not using ESA as initial therapy.

The European MDS Registry (EUMDS) collects information including ESA usage, covariates, and EQ-5D scores at six-month intervals. Estimating average treatment effect (ATE) from observational data requires adjusting for several sources of bias. Our study controls for baseline and time-varying confounding by using inverse probability of treatment weights. Employing a methodology based on marginal structural models, we are able to estimate robust ATEs. A two-part mixed-effects beta model was used to calculate ATE during a four-year follow-up period. We compare ESA therapy every six months versus clinical management not involving the use of ESAs.

Our results show an overall positive ESA effect on EQ-5D over the four-year follow-up period. The majority of time points have a positive ESA effect after adjustment, though a few time points show no effect. The estimated ATE at four years is small: 0.046 (−0.031, 0.114).

We found that use of ESAs over a four-year follow-up period produces mostly positive treatment effect estimates after adjusting for time-varying variables and confounders. Our robust results can be used to inform more reliable treatment decision-making.

Personalized precision medicine (PPM) is an innovative approach to disease diagnosis, prognosis, and treatment of individual or group characteristics using diverse data sources. While omics technologies are integral to PPM, they pose challenges. Therefore, developing an appropriate methodology to assess these technologies is crucial for patient safety, resource efficiency, and clinical decision-making within the Spanish National Health System.

This health technology assessment (HTA) methodology procedure was developed by combining three different approaches: a systematic review (SR); a survey targeting experts in omics technology, ranging from basic science researchers to clinicians, and patient associations; and, finally, a consensus method (RAND Appropriateness Method [RAM]).

Through data extraction and evidence synthesis of the 38 studies included in SR, 30 existing frameworks for evaluating omics technologies were identified, as well as the elements needed to assess these technologies, leading to the first version of the framework. Two surveys were performed to integrate the perspectives of omics technology experts and patients. Subsequently, this framework version was further developed by a RAM consensus panel of experts from HTA agencies to ensure a rigorous evaluation of gathered data. The final framework was categorized into 94 elements divided into sections, categories, domains, and subdomains.

A methodological guide, including the assessment framework, was developed for the Spanish HTA network. The framework is divided into several sections addressing evidence-gathering, provision models, organizational elements, economic evaluation, and ethical and social implications. Compared to other available frameworks, our proposal included aspects such as bioinformatics, technological maturity level, and the patient perspective with the personal utility domain.