Demonstrating meaningful overall survival (OS) treatment benefit in high-risk localized or locally advanced prostate cancer (HR-LPC/LAPC) remains challenging due to the prolonged natural disease evolution. There is growing interest in validating intermediate clinical endpoints (ICEs) for OS. We evaluated the predictive value of ICEs for OS in HR-LPC/LAPC using data from randomized controlled trials (RCTs) and non-randomized comparative studies (non-RCTs).

We conducted a systematic literature review using Embase, MEDLINE, CENTRAL, and gray literature (22 March 2024) to identify RCTs and non-RCTs investigating therapeutic options for adults (≥18 years) with HR-LPC/LAPC. Eligible studies reporting treatment effects (hazard ratios [HR]) for OS (HROS) or ICEs were included. Event-free survival (EFS), metastasis-free survival (MFS), no evidence of disease (NED), and pathological complete response (pCR) were ICEs of interest. Correlation of each ICE with OS was evaluated using Bayesian bivariate random-effects meta-analysis (BRMA) with an uninformative prior distribution. The predictive value of each ICE was determined by assessing observed versus predicted treatment effects of OS via leave-one-out cross validation (LOOCV).

We selected 137 unique studies (89 RCTs and 48 non-RCTs) for meta-analysis: EFS-OS, k=85 studies (n=53,965 patients); MFS-OS, k=79 (n=51,746); NED-OS, k=100 (n=56,187); pCR-OS, k=112 (n=70,228). We observed moderate correlation estimates of 0.53 (95% credible interval [Crl]: −0.23, 0.96), 0.69 (95% CrI: −0.35, 0.98), and −0.53 (95% CrI: −0.95, 0.39) for EFS-OS, MFS-OS, and NED-OS, respectively. pCR-OS showed a weak correlation (0.06, 95%Crl: −0.89, 0.94). None of the correlation estimates were statistically significant. An RCT-only sensitivity analysis showed consistent results. The LOOCV had good predictive accuracy, with 95 percent prediction intervals capturing the observed HROS for 89.5, 87.5, 100, and 83.3 percent of studies, for respective ICEs. No ICEs met the Institute for Quality and Efficiency in Health Care (IQWiG) criteria for strong correlation.

Despite consistent direction of associations between ICEs and OS using rigorous BRMA, we could not meet the stringent surrogacy thresholds used by health technology assessment (HTA) agencies. Our findings highlight a need for further analysis to prove correlations, including analyses using real-world data and consideration of how surrogacy evidence is assessed by HTA agencies in early-stage prostate cancer.

Second-line treatment for Philadelphia-positive chronic myeloid leukemia (Ph+CML) in the Brazilian Unified Health System (SUS) currently involves the tyrosine kinase inhibitors (TKIs) dasatinib and nilotinib. Choice of TKIs should be made considering existing comorbidities and the risk of adverse events. Our objective was to estimate the proportion of patients with cardiovascular comorbidities receiving TKIs in the SUS.

A retrospective administrative database analysis was conducted of cases recorded from October 2013 to May 2024. The International Classification of Diseases, 10th Revision codes used for chronic cardiovascular diseases were identified. Two databases of the outpatient information system were analyzed, the oncology database and the individualized outpatient production report, to identify patients receiving dasatinib or nilotinib as a second-line treatment for Ph+CML who had at least one outpatient procedure with an International Classification of Diseases, 10th Revision code of cardiovascular disease. To achieve this, an inner join procedure using the cryptographed identifier was implemented. Cases were characterized by age, sex, disease stage, and geographic region.

A total of 6,556 unique patients receiving second-line treatment for Ph+CML were analyzed. Their mean age was 49.4 (standard deviation 16.2). There was a predominance of males (55.6%) and white (47.6%) people. Cases were concentrated in the southeast (48.4%), the most populous region of the country. Most cases were at the chronic phase (70.8%), followed by the accelerated (17.7%) and blast phases (11.5%). Dasatinib was the most used treatment, representing 52 percent of cases, followed by 40.5 percent using nilotinib. Other regimens included chemotherapy and combined treatments. Patients with cardiovascular disease represented 10.9 percent of the total sample; 9.8 percent of patients treated with dasatinib and 13 percent of patients receiving nilotinib.

Real-world administrative data provided information on patient profile, treatment patterns, and disease severity. Moreover, the proportion of patients with Ph+CML who had cardiovascular comorbidities was estimated at the national level. These elements are necessary to assess unmet needs and treatment patterns not compliant with the recommendations from guidelines, to underpin the design of optimized strategies of care.

CONITEC plays a central role in health technology assessment (HTA), particularly in advancing the evaluation of medical devices (MDs). Tools such as horizon scanning (HS) and the Committee on Products and Procedures enhance its methodologies. This study examined the evolution of MD evaluation over time.

The requests submitted to CONITEC for analysis were carefully identified, quantified, and analyzed in detail. Special attention was given to requests that were specifically directed to the evaluation and recommendation processes carried out by the Commission’s various committees. These requests were thoroughly reviewed to ensure that all relevant aspects were considered. Subsequently, the MD-related proposals were carefully examined, with a particular focus on the incorporation recommendations provided by CONITEC. In addition, the evaluations that led to the creation of HS analyses were closely analyzed, providing insights into the decision-making process and the development of these important evaluations.

Over the last 12 years, CONITEC received 1,082 HTA requests, with 660 originating internally and 422 externally. Of these, 832 were related to medicines, 174 to procedures, and 76 to products. Of the product and procedure proposals, 159 were recommended for incorporation, 35 were not recommended, five resulted in exclusion, one resulted in non-exclusion, and six were closed by CONITEC’s decision. Approximately 18 percent of the MD-related requests led to the creation of HS analyses, particularly in 2023 and 2024 when all demands in this area resulted in HS analyses, reflecting improvements in the evaluation process.

The evaluation of MDs is a rapidly growing field within HTA, requiring the adoption of specific methods for progress. These methods include tools and databases focused on HS as well as tailored methodological guidelines. CONITEC has continuously developed tools to enhance decision-making on the incorporation of MDs into the Unified Health System.

Choosing the optimal ulcerative colitis treatment is complex, given the range of medical and surgical options with varying side effects and effectiveness. Decision aids can improve patient choices, but current tools lack personalization. To address this, we developed a personalized decision tool using a discrete choice experiment (DCE) to help patients make informed decisions about medical or surgical treatments.

An online DCE survey was developed containing competing treatment profiles described using all important aspects of the treatment (effectiveness, side effects, family planning). Patients (n=300) with ulcerative colitis were asked to consider the benefits and disadvantages of each treatment profile and select the treatment that they would choose. The DCE data were analyzed using mixed logit and latent class models. The model results were integrated into an online decision aid using a Shiny application.

R Shiny was successfully used to enable the real-time personalization of DCE results. The developed decision aid contained two aspects of personalization. First, attribute importance scores showed the treatment characteristics that mattered most to patients based on their DCE choices. Second, a “best-match” treatment that aligned with their preferences was provided from uptake rate calculations. User testing of the developed decision aid is ongoing. However, initial feedback from patients has been positive.

A key challenge in developing personalized decision aids is providing real-time, tailored recommendations based on individual preferences. This study demonstrated the feasibility of integrating DCE methods into personalized decision aids for ulcerative colitis. By tailoring treatment recommendations to individual patient preferences, this tool has the potential to empower patients, reduce decisional conflict, and enhance shared decision-making between patients and clinicians.

Health technology assessment (HTA) of Class III implantable medical devices (MDs) in Europe is conducted at the national or regional level. Despite common clinical evidence, sometimes appraisal results and recommendations vary significantly, affecting patient access to innovative technologies. An adaptive approach to HTA for MDs will need to be consistent with current expert thinking on adaptive approaches in HTA methodology.

The websites of HTA agencies across 13 European countries were searched for HTA reports published between 1 January and 31 December 2023 on high-risk cardiovascular MDs used to treat structural heart disease. The goal was to identify the classes of technologies assessed by different HTA agencies and compare the submitted evidence and decisions. Since assessments are sometimes repeated across different years, we reviewed all available submissions for the selected innovations from the CE mark up to 2023 to follow the evolution of appraisals and decisions on the same technology.

Eighty reports from 11 countries were identified, with 23 on structural heart disease from Austria, France, Italy, and Spain. The main diseases involved heart valves, septal defects, and heart failure. In 2023, two agencies reassessed technologies for mitral valve regurgitation (MVR). Austria’s HTA gave negative recommendations to the first device CE marked in 2008 in fourth consecutive assessments, despite three randomized controlled trials (RCTs). A fifth reassessment is planned for 2026. France has conducted 26 assessments since 2015 on MVR devices (due to design changes, indication expansions, generation changes, economic assessments, new devices entering the market, and life cycle reassessment), with a positive recommendation in 2015, which was supported by two single-arm studies and a registry. A second device, CE marked in 2019, was recommended by France in 2023 based on a single-arm trial, an RCT, and two registries.

The number of HTAs performed on MDs remains low, particularly for high-risk device classes. There was no consistency in the number of HTAs performed between countries, nor in the evidence required for a successful assessment. These differences lead to unequal access to specialized treatment for patients in different countries and highlight the need for a unified European HTA approach.

The Cancer Drugs Fund (CDF) is a type of managed entry agreement used in England to allow patient access while undertaking evidence generation followed by re-evaluation to address uncertainty. We assessed the impact of changes to the National Institute for Health and Care Excellence (NICE) health technology assessment (HTA) methods in 2022 plus the publication of a national commercial framework on recommendation outcomes.

NICE staff, in conjunction with clinical stakeholders, assess all cancer medicines for their suitability for entry into the CDF. Cancer topics looked at by NICE from 2016 to 2024 were identified, and the type of evidence uncertainties and the final recommendation outcomes were recorded. Types of evidence uncertainty were assigned to one of nine different categories: survival data, relevant patient population, lack of relevant comparators, trial design, quality-of-life data, cost estimates, treatment duration, cost-effectiveness model, and adverse events. Recommendation outcomes included entry to the CDF, full reimbursement, reimbursement for optimized population, no reimbursement, or research only.

The types of uncertainties identified for appraisals using pre-2022 methods (n=103) and post-2022 methods (n=35) showed the same trends in most common uncertainties. Immature survival data was the most common, with lack of relevant comparators the second most common. There was an increase in the average number of uncertainties seen in post-2022 topics (2.05), compared with pre-2022 topics (1.75). The number of CDF recommendations has been decreasing in recent years, with a peak between 2017 and 2019. For appraisals that did not enter the CDF, the likelihood of a positive recommendation was lower in topics where entry to the CDF had been considered a possibility.

There were no clear changes in trends of uncertainties seen in cancer topics following the 2022 methods changes, though there was a small increase in overall uncertainty. There was a decline in CDF entries, which correlates to payers offering a more flexible commercial environment giving alternative reimbursement options to evidence generation with re-evaluation via the CDF.

Instituto Dara, a Brazilian non-profit organization, supports vulnerable families through multidimensional interventions addressing poverty and chronic illness. While validated quality-of-life instruments are essential for program evaluation, few target disadvantaged populations with chronically ill children in Brazil. This study evaluated the psychometric properties of the EuroQol Health and Wellbeing Short Version (EQ-HWB-S) questionnaire, including its convergent validity and test-retest reliability.

The study included 99 individuals representing families supported by Instituto Dara. Data collection used face-to-face interviews with EQ-HWB-S and additional validated instruments (EuroQol-5D and the Warwick-Edinburgh Mental Wellbeing Scale [WEMWBS]) to assess convergent validity. Test-retest reliability was assessed in 45 participants over a two-week period. Statistical analyses included descriptive methods and correlation analyses to summarize EQ-HWB-S applicability and performance in this population.

Physical domains (mobility and activities) showed minimal impairment, with most participants at level one (>75%). In contrast, psychosocial domains showed significant burden: over 50 percent reported moderate to extreme levels of exhaustion, loneliness, and anxiety or depression. Test-retest reliability showed moderate agreement (r=0.568; p<0.001). The EQ-HWB-S yielded an overall score of 0.707. Construct validity analyses demonstrated moderate correlations with established measures (r=0.33 to 0.45), particularly with the physical well-being domains of the EQ-5D and alignment with the WEMWBS for psychological dimensions.

The results highlighted the significance of EQ-HWB-S in assessing domains such as loneliness, exhaustion, and control. The instrument demonstrated robust psychometric properties with moderate test-retest reliability and strong convergent validity, though its discriminatory ability warrants further investigation. These findings support its use for assessing quality-of-life outcomes in vulnerable populations with chronically ill children.

During the health technology assessment (HTA) process it is possible to identify unmet medical needs (UMN) that can be used to guide research and development investments. This study aimed to develop a methodology to identify and prioritize research and development themes using UMN identification in the HTA process of a teaching hospital.

We reviewed and looked for UMN in all the HTA technical reports related to technology incorporation solicitations submitted between 2018 and 2023. The UMN definition used was based on that published by the US Food and Drug Administration in 2014. A prioritization process was adopted using criteria based on the World Health Organization and the Brazilian Unified Health System list of priorities for the year. The UMN were categorized by characteristics such as indication (diagnostic, therapeutic, preventive, rehabilitation) and type of diseases.

Thirty-seven Structured Technical Reports—formal documents submitted for evaluation of health technologies—were reviewed by two investigators. Among these, 37 UMN were identified, most of which were characterized as therapeutic needs (59.5%) or related to neurological diseases (16.2%). According to established criteria, 81.1 percent of the UMN warranted investment in research and technology development.

The proposed methodology can reliably identify UMN by analyzing technical reports. Further improvements can make it possible to fit the process to diverse HTA systems to support research and development decisions and investments.

The objective of this assessment was to determine the benefit of using a next-generation sequencing (NGS) gene panel for the clinical management of gastrointestinal stromal tumor (GIST) among patients in routine clinical practice. The aim was to assess the clinical utility of this procedure, the somatic molecular alterations of specific interest, and to define its role in the therapeutic care of patients with GIST.

The method used for this fast-track assessment were based on: (i) a critical analysis of systematic reviews, meta-analyses, and clinical practice guidelines identified by a systematic literature search; (ii) identification of the level of evidence of molecular alteration clinical actionability as set out by the European Society for Medical Oncology Scale, of targeted therapies included on the list of reimbursable drugs assessed by the French National Authority for Health, or drugs that have compassionate use authorizations issued by the French National Agency for Medicines and Health Products Safety; and (iii) stakeholder consultations and observations by public health institutions.

Assessment of the evidence and data demonstrated that NGS gene panel testing has: (i) superior diagnostic performance for detecting KIT and PDGFRA molecular alterations, compared with Sanger sequencing; (ii) superior diagnostic performance for detecting NTRK1/2/3 fusion, compared with immunohistochemistry; and (iii) evidence of clinical utility of the targeted gene panel considering the benefits provided by targeted therapies.

The composition of the NGS gene panel may be subject to change, in accordance with favorable assessments of new gene alterations. New assessments will be conducted in a dynamic manner in response to developments in scientific knowledge.

The French National Authority for Health deemed that funding the NGS gene panel for GIST was justified for: (i) KIT and PDGFRA genes in cases of locally advanced or metastatic GIST at an intermediate or high risk of recurrence, and in cases of suspected GIST when histology is inconclusive for diagnosis; and (ii) the NTRK1/2/3 gene in cases of refractory or relapsed locally advanced or metastatic pediatric wild-type GIST.

Diffuse large B-cell lymphoma (DLBCL) is the most common hematological neoplasm in adults globally and in Colombia, but there are limited data on its local economic impact. This study analyzed the relationship between clinical variables and healthcare resource utilization of patients with DLBCL in a Colombian health maintenance organization.

This analytical observational study included patients with histopathologically confirmed DLBCL treated at the Luis Carlos Sarmiento Angulo Cancer Treatment and Research Center (CTIC) between July 2022 and September 2025. We reported an interim analysis with a data cut-off in July 2024. Clinical data from CTIC’s institutional registry were merged with the information provided by the administrative database, covering healthcare resource use and costs for drugs, procedures, and medical supplies. A multivariate linear regression estimated the effect of prognosis (revised International Prognostic Index [R-IPI] score) on total costs, adjusting for confounding (e.g., sex, comorbidities, and history of neoplasm).

Sixty-two patients were included (mean 66 years [standard deviation 14]; 58% men). R-IPI scores at baseline indicated that 34 percent were at high-intermediate risk, 21 percent had low and low-intermediate risk, and 23 percent were at high risk. R-IPI scores at baseline showed that 17.7 percent patients had very good prognosis, while 24.2 and 41.9 percent had good or poor prognosis, respectively (16.1% missing). The R-CHOP chemotherapy regimen was the most common first-line (1L) treatment (73%), and fifteen percent of patients had disease progression. The total 1L treatment cost was COP1,386,178,104 (USD320,500) per patient. Thirteen patients received second-line (2L) treatment, costing COP3,203,033,513 (USD740,577) per patient. Regression analysis showed that patients with poor prognosis, per R-IPI score, incurred 8.9 times higher costs than those with very good R-IPI scores.

Our study showed the association between clinical variables and the cost of treatment in patients with DLBCL. Regression analysis indicated that higher R-IPI scores and advanced disease stages significantly increased costs. The cost per patient for 2L treatment was 2.3 times higher than for 1L treatment, highlighting the importance of timely detection and treatment.

Hemophilia A (HA) results from mutations in the F8 gene, leading to a deficiency in factor VIII (FVIII) and consequent spontaneous bleeds. Treatment options include bleeding prophylaxis with FVIII and, more recently, emicizumab (EMI), a FVIII-mimetic bispecific antibody. EMI was introduced into the Brazilian Unified Health System in 2019 for people with HA and inhibitors (PwHAi). This study compared EMI costs with prior prophylactic methods and evaluated its budgetary impact against government projections.

The National Registry of People with HA Using Emicizumab in Brazil (EMCase) is a prospective observational study initiated in 2020, conducted in 16 Brazilian hemophilia treatment centers. The analysis included consumption of FVIII, bypassing agents, and EMI. Treatment costs for the pre-EMI period and the first year of EMI use were calculated using prices paid by the Ministry of Health, as reported in the Brazilian Health Price Database. Yearly average treatment costs per kilogram of body weight were compared with government budget projections.

A total of 56 moderate to severe PwHAi were included. Before EMI treatment, the actual treatment costs were BRL31,145.19 (USD5,190.87) per kg per year. During the first year of EMI prophylaxis, the actual treatment costs were BRL22,583.79 (USD3,763.96) per kg per year, representing approximately 27.5 percent reduction in costs, compared with the previous period. In addition, the government projected cost for 2019, adjusted for inflation, was estimated at BRL19,736.84 (USD3,289.47) per kg per year for the first year of EMI use, resulting in a 14.4 percent increase in the projected budget impact.

Although the actual cost during the first year of EMI prophylaxis was higher than the government project cost, a cost reduction was observed in comparison with the pre-EMI period cost. The study highlighted the importance of real-world data-based economic analyses in the evaluation of health technologies, complementing government projections to inform decision-making.

Rare diseases, including Gaucher disease and spinal muscular atrophy, impose significant challenges in access to treatments and equitable healthcare policies. In Ecuador, patients and caregivers lack representation in health technology assessment (HTA) processes. This study aimed to amplify their voices, exploring their experiences to develop inclusive methodologies that promote equity and transparency in healthcare decision-making.

This study employed a mixed-methods approach combining qualitative and quantitative data comprising perspectives of patients with Gaucher disease and spinal muscular atrophy (as well as the perspectives of their caregivers), in-depth interviews, surveys, and observation. Collaboration occurred with patient organizations. Qualitative analysis focused on thematic coding of experiences, while quantitative surveys assessed access to treatment and socioeconomic burdens. Data triangulation ensured robust findings.

Preliminary results indicated significant challenges in equitable access to treatments for rare diseases in Ecuador. Patients and caregivers reported high emotional and financial burdens, with limited systemic support. Early findings suggested disparities in treatment availability and awareness between urban and rural settings. Engagement workshops demonstrated a strong willingness among stakeholders to participate in developing inclusive HTA processes.

Preliminary findings highlighted the urgent need for inclusive HTA processes in Ecuador. Amplifying patient and caregiver voices can improve equity in rare disease management and inform policy changes. Future research will focus on refining methodologies for participatory HTA and addressing systemic gaps in access to treatment, with emphasis on rural and vulnerable populations.

Since 2023, the Agency for Care Effectiveness (ACE) has included lived experiences from patients and caregivers in health technology assessments (HTAs) for wearable or home-based medical devices. This presentation discusses the impact of patient experience data on the HTA and subsequent subsidy recommendations for continuous glucose monitoring (CGM) systems for type 1 diabetes mellitus (T1DM) in Singapore.

All local patient organizations with an interest in CGM systems or T1DM were invited to share their views using a structured, qualitative survey or simple free-text patient journey form. Testimonials were analyzed by ACE to extract key themes and insights and incorporated into the HTA report alongside clinicians’ input and clinical and economic evidence to inform subsidy recommendations made by the Ministry of Health Medical Technology Advisory Committee. The impact of patient experience data on the Committee’s deliberations and recommendations for CGM systems was determined by reviewing the HTA report, minutes from Committee meetings, and the published HTA guidance document.

Eighty testimonials were received from patients and caregivers about the impact T1DM has on their lives, and their experiences using different blood glucose monitors. They also confirmed real-world CGM usage across different age groups, informing assumptions for budget impact calculations. Testimonials highlighted benefits and drawbacks of different monitoring methods, unmet needs, patient preferences, and expectations for new technologies to be more affordable and less invasive. While scientific evidence for children with T1DM was limited, testimonials confirmed CGM systems could improve outcomes for both adults and children, which influenced the subsidy criteria recommended by the Committee to cover both age groups.

Incorporating patients’ lived experiences in ACE’s HTAs validated and strengthened the scientific evidence, addressed uncertainties by filling data gaps, and led to patient-centered subsidy recommendations for CGM systems, demonstrating the value of systematic patient involvement. Post-recommendation, ACE co-developed a factsheet on CGM systems with local patient organizations to explain the subsidy recommendations in plain language and encourage HTA guidance adoption.

Monitoring elderly health via wearable electronic devices offers significant benefits but faces ethical and regulatory challenges. While these devices are useful in emergencies, such as falls, concerns about user privacy and data confidentiality persist. This study aimed to develop recommendations for developers of wearable electronic devices used in elderly health monitoring, as well as for other stakeholders, focusing on privacy and confidentiality.

A scoping review was performed by searching databases including MEDLINE (PubMed), Embase, Scopus, the Cochrane Library, Web of Science, Epistemonikos, and the Virtual Health Library through to October 2023. Studies were included if they addressed all the following criteria: (i) wearable electronic devices; (ii) elderly individuals; (iii) health monitoring; and (iv) privacy, confidentiality, or data protection. Studies that did not include recommendations for developers or other stakeholders aimed at enhancing user privacy protection and data confidentiality were excluded. The results were presented as a narrative synthesis.

A total of 1,037 records were identified in the databases, and 12 studies published between 2014 and 2022 were included. The main findings were addressed in topics, including: (i) privacy and confidentiality in monitoring the health of elderly individuals using wearable electronic devices; (ii) barriers and facilitators for the adoption of wearable devices in monitoring the health of elderly individuals; and (iii) recommendations for developers of wearable devices in monitoring the health of elderly individuals and for other stakeholders.

The use of wearable electronic devices for health monitoring in elderly individuals presents complex ethical, regulatory, and technological challenges, particularly regarding privacy and confidentiality. Collaboration between developers and stakeholders is essential for implementing robust security measures and establishing effective regulatory standards that ensure comprehensive user protection.

Scientific advice (SA) in the process of developing medical devices and technologies (MedTechs) allows manufacturers to target the profile of their production and gain a better understanding of the requirements for approval and reimbursement. The aim of this study was to evaluate the SA framework developed by National Evidence-based Healthcare Collaborating Agency (NECA) in Korea.

We developed the SA program by reviewing all available information regarding the service offered, processes, duration (in weeks), stage of development, types of technologies, and fees. The SA program supports a total of 60 MedTechs per year. By the end of 2023, a total of 150 technologies had participated. We also evaluated participant satisfaction with the SA program after their participation.

The NECA SA program offers Evidence Preview, Evidence Search Education, Evidence Development Plans with regulatory advice, and Clinical Expert Advice for clinical trial design. The process is flexible, depending on the applicant’s needs, and is completed within 24 weeks, with a final report issued after completion. Stage of development and type of technology are mainly focused on early stages of development (110/150 technologies) and innovative technologies (87/150 technologies). Participant satisfaction with the SA program was more than 90 percent for the past three years. The SAs offered by NCEA are optional, free of charge, confidential, and non-binding.

This framework helps manufacturers understand the evidence requirements for NECA and other payers. Considering that most MedTechs are in the early stages of development, it is important to develop a target technology profile (TTP) tool to prepare guidelines for MedTech development plans. In addition, there is growing international collaboration between industry and regulatory authorities. It may also be beneficial to engage with specialized consulting institutions.

Therapeutic ranking (TR) is a process used in health technology assessment (HTA) by the Institute for Health Technology Assessment (IETS) in Colombia to optimize drug prescription through analyses of effectiveness, safety, costs, and post-marketing surveillance. However, decision-making relying on clinical expert preferences may introduce biases, since they are often influenced by subjective values. This study presented an alternative methodology by IETS that emphasizes a structured, evidence-based approach to improve decision-making in TR.

The standard TR process involved forming an interdisciplinary group to assess the effectiveness, safety, costs, and post-marketing surveillance of medications, followed by evidence synthesis and informed voting using the Borda method to prioritize technologies based on consensus-driven preferences. To enhance informed voting, a methodological strategy was introduced that incorporated structured comparative tables and relative position scales derived from specific quantitative analyses for each parameter (e.g., surface under the cumulative ranking curve [SUCRA], costs, number of adverse events). This approach visually displayed performance patterns for integrated analysis by clinical experts.

This adaptation to TR consolidated the results for effectiveness, safety, costs, and economic evaluations of the assessed technologies. The structured format facilitated the identification of performance patterns by technology and enabled the integration of these results into a visual framework that supported deliberations during informed voting. This methodology promoted the integration of multiple evaluated dimensions, ensuring that inputs from epidemiology, pharmacology, health economics, and other specialized analyses were not overshadowed by the clinical experience of the expert panel. Instead, each input contributed to the collective decision-making process.

This methodological approach enhanced TR as an HTA process by fostering evidence-based decisions. Optimizing the process could involve assigning weights to each analysis component, including clinical expertise, to balance their influence on prioritization. Explicit weighting would improve transparency and reproducibility. This model is replicable in other settings, maximizing HTA’s impact on global health systems.

Neonatal sepsis remains a major cause of morbidity and mortality in neonatology. While blood culture is considered the gold standard for detecting sepsis, its low sensitivity significantly limits its utility. The identification of biomarkers, such as procalcitonin (PCT), is essential for improving diagnostic accuracy. This Scientific Technical Opinion critically evaluated the available evidence on the role of PCT in diagnosing neonatal sepsis and clinical practice.

This study followed Joanna Briggs institute and Cochrane Collaboration guidelines and was registered with PROSPERO. Systematic searches were conducted in Embase and MEDLINE. Eligible studies included randomized, quasi-randomized, cohort, case-control, observational, and cross-sectional designs that evaluated PCT diagnostic accuracy for neonatal early or late sepsis, microbiologically or clinically documented. Studies had to perform testing at the onset of sepsis symptoms, prior to antimicrobial therapy. Data collected included true positive, true negative, false positive, and false negative results. Sensitivity and specificity meta-analyses were performed, and summary receiver operating characteristic curves were generated using Meta-DiSc 2.0 software, with results reported as 95 percent confidence intervals (CIs).

A total of 111 studies were analyzed, involving 19,175 neonates. The sensitivity of PCT in diagnosing sepsis was 0.787 (95% CI: 0.757, 0.815) and the specificity was 0.853 (95% CI: 0.822, 0.879). In the “time of collection” group analysis, we obtained the following results: at zero hours, sensitivity was 0.753 (95% CI: 0.695, 0.802) and specificity was 0.803 (95% CI: 0.739, 0.854); up to 12 hours, sensitivity was 0.912 (95% CI: 0.748, 0.973) and specificity was 0.827 (95% CI: 0.643, 0.927).

The findings suggested that PCT demonstrates high diagnostic accuracy for neonatal sepsis. Thus, incorporating PCT could enhance diagnostic precision and help minimize the unnecessary use of antimicrobials, ultimately supporting better outcomes in antimicrobial stewardship programs within neonatal intensive care units.