Flexibility in the Modelling of Comparative Effectiveness in the Absence of Head-to-Head Comparisons in the NICE Single Technology Appraisal of Fenfluramine for Treating Seizures Associated with Lennox-Gastaut Syndrome: An External Assessment Group Perspective.

Several countries now formally include at least one aspect of equity, alongside cost-effectiveness, in health technology assessment (HTA) decisions, often focusing on need. Recent literature suggests public support for prioritising health gains for children over adults. While some agencies may indirectly consider age, none explicitly apply different weights to health gains for children. This study analysed 19 years of data to quantify the additional value the Australian Pharmaceutical Benefits Advisory Committee (PBAC) implicitly places on children compared to adults in drug funding decisions. Logistic regression analysis revealed that after controlling for clinical uncertainty, effect size, cost-effectiveness and government budget impact, interventions for children were more likely to receive a positive funding recommendation. The 'childhood effect' persisted even when accounting for other equity considerations, such as severity, condition rarity, unmet need, and age-related intervention characteristics, including vaccination and affected bodily systems. Our findings provide empirical evidence of a higher social value for health gains in children compared to adults. This is the first study to find a significant 'childhood effect' for public funding recommendations for pharmaceuticals, offering important insights for HTA frameworks that aim to incorporate societal values.

Chronic kidney disease (CKD) is a major global cause of morbidity and mortality. Recent nephroprotective therapies have improved CKD management, yet their cost effectiveness across settings remains uncertain. This review systematically identified and compared cost-effectiveness studies of novel CKD treatments for both broad CKD populations and disease-specific subgroups. A systematic search was conducted in PubMed and the Cochrane Library using terms related to "chronic kidney disease," "cost-effectiveness," "cost-utility," "health technology assessment," "SGLT2 inhibitor," and commercial and generic names of nephroprotective drugs approved since 2013. Eligible studies were full-length articles in English published between January 2015 and September 2025. Costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios were extracted. All monetary values were standardized to 2025 US dollars. The search yielded 172 records, of which 26 met inclusion criteria. A supplementary search identified ten additional studies, resulting in 36 evaluations. Most studies assessed sodium-glucose cotransporter 2 inhibitors or finerenone. Across evaluations, these therapies consistently improved outcomes, with QALY gains reported in all studies (0.012-1.44 QALYs gained). Most concluded that the interventions were cost effective compared with standard of care, and 13 reported cost-saving results. Only three studies reported an incremental cost-efffectiveness ratio above $100,000 per QALY threshold. Cost effectiveness was observed in both general CKD and CKD with diabetes mellitus, although estimates varied by country, time horizon, and analytic perspective. Current evidence indicates that novel nephroprotective therapies for CKD are generally cost effective, and in some settings cost saving. These findings support their value in both general CKD and diabetic populations and highlight the importance of early treatment adoption to delay disease progression and reduce long-term healthcare costs.

Preventing incisional hernia after ileostomy closure: Budget impact analysis of prophylactic biosynthetic mesh use in Italy.

Finding new therapeutic indications for established medicines continues to have important impact. Although drug repurposing (DR) offers the potential for a faster and more affordable complement to de novo development, barriers to successful DR remain pervasive. To prioritize barriers identified earlier in a systematic literature review by the REMEDi4ALL Horizon Europe project, involving multiple stakeholder groups, to create a shortlist of the most important barriers and to examine differences in stakeholders' perceptions. A policy survey was conducted among stakeholder groups involved in DR. Participants rated the barriers based on their impact and actionability, using 5-point categorical scales. A weighted scoring method was used to create the shortlist by combining scores across domains, while ensuring that each stakeholder group's preferences were retained in the final shortlist. 60 individual responses were collected. The final shortlist contained 22 barriers, including 4 barriers related to exclusivity rights for repurposed medicines (RMs), 2 to pricing of RMs, 5 to market authorization of RMs, 2 to perception off-patent RMs, 2 to business case for off-patent RMs, 2 to non-industry funded DR, 2 to health technology assessment of RMs, 2 to ecosystem for non-profit or small-medium sized enterprises-driven DR, and 1 to business case for repurposing on-patent compounds. Prioritizing barriers helps identify solutions by addressing the critical challenges first. Acknowledging that different stakeholder groups may perceive the impact and actionability of these barriers differently is crucial for building a shared, multi-stakeholder perspective when formulating policy recommendations to address the barriers.

• Delphi explored payment models for innovation in IC, where evidence was lacking • Principles were identified to qualify innovations for innovative payment schemes • Technologies with significant therapeutic value should access novel payment models • HTA bodies should guide pricing negotiations and value-based use of innovations • DRG plus P4P was the only model achieving high consensus for technology uptake Payment’s mechanisms that facilitate the uptake of innovative technologies embedded in integrated care (IC) systems have not been described. This study aims to identify the principles that qualify innovations for inclusion in innovative payment schemes, as well as those that facilitate their uptake in IC. A two-round web-based Delphi was conducted to gather consensus among healthcare professionals, health economists, health technology assessment (HTA) professionals, payers, and managers. Statements were rated on a 6-point Likert scale, and consensus was classified as high, moderate, or low based on proportion of agreement-level responses and median score for each statement. Statements with moderate consensus were revised and re-evaluated in round 2. The first-round response rate was 51% (n=92/181); the second was 88% (n=80/92). The involvement of HTA in pricing negotiations along with the identification of the best-value scenarios for the innovation was the principle with the highest level of consensus and agreement. Among the payment models considered, only the combination of diagnostic-related-groups (DRG) and pay for performance (P4P) achieved high consensus. All the others reached moderate consensus (94%, n=15/16); however, most showed a high proportion of agreement-level responses (≥90%). The combination of DRG and P4P emerges as the most accepted pathway to accelerate innovation adoption. Establishing clear preconditions and guidance remains essential for successful implementation and high-quality patient care. Six additional novel schemes may also facilitate adoption. While some have been tested in real-world settings, further cases are required to assess feasibility and effectiveness across diverse healthcare contexts.

Navigating Health Technology Assessment (HTA) processes presents significant challenges for pharmaceutical companies seeking market access in Australia. While previous research has examined HTA decision-making from payer perspectives, pharmaceutical companies' internal decision-making processes remain understudied. To investigate how pharmaceutical companies' market access teams manage uncertainties in the HTA process for medicines in Australia using the ISO 31000 risk management framework to examine decision-making practices. A mixed-methods study was conducted involving an online questionnaire and semi-structured interviews with 18 respondents with recent experience with HTA submissions from 14 pharmaceutical companies, representing approximately 50% of PBS expenditure. Data were analysed using a deductive approach that mapped to ISO 31000 risk management process, supported by thematic analysis and descriptive statistics. Market access teams aim to secure PBS listing by balancing price (45% relative importance), speed (30%), and population coverage (25%) with submission costs. They function as intermediaries between global stakeholders and local market requirements, operating without formal risk management frameworks. Key challenges include managing global-local tensions, navigating increasingly complex stakeholder relationships, addressing rising submission costs (averaging AUD$830,000), and coping with workforce limitations. Risk assessment practices are predominantly informal and experience-based, while communication barriers hinder effective stakeholder engagement. Through mapping to the ISO 31000 framework, this study established the market access processes of pharmaceutical companies, with focus on uncertainty and decision-making. Enhanced communication may reduce risk and improve HTA system efficiency. Addressing these challenges, as well as improving management of uncertainty, would improve patient access to medicines.

Technology assessment for measuring cargo weight and volume in moving trucks

Environmental impacts of medicines are increasingly recognised but rarely embedded in their pricing and reimbursement (P&R) and related assessments. This scoping review mapped how environmental aspects, including antimicrobial resistance (AMR) stewardship, are or could be considered in P&R of medicines and identified challenges for their implementation. Electronic searches from PubMed, Scopus, Web of Science, Edilex, HeinOnline, Westlaw, Google Scholar and official agencies' websites were conducted. Eligible items covered developed country settings and described existing or proposed integration of environmental aspects into medicine pricing or reimbursement. Of 4476 publications, 21 were included. The publications were from 2013 to 2023, most of them addressing ecotoxicological and climate impacts. Six types of means to embed environmental aspects into P&R were identified: health technology assessment (HTA) integration, reimbursement criteria, pricing criteria, environmental classification, regulation and delinkage models. Most proposals were conceptual, whereas implemented or piloted examples focused on AMR-oriented means. Reported challenges for implementation included challenges regarding data availability, comparability and verification; valuation and methodological integration; incentive design and cost distribution; and equity and political feasibility. Environmental aspects are not yet routine in medicine P&R assessments due to a lack of standardised, verifiable product-level data, appraisal methods and equity-sensitive policy design.

Evaluating the efficacy, safety, and clinical effectiveness of IVF add-ons: Methodological challenges and future solutions.

This study characterizes longitudinal changes in health-related quality of life (HRQoL) following radiotherapy in patients with head and neck cancer (HNC) and quantifies associated toxicity burden to inform future health technology assessments (HTA). HNC patients receiving definitive, postoperative radiotherapy, or chemoradiation at the University Medical Center Groningen from March 2007 to September 2022 were included. Health status and utility were assessed using EQ-5D-3 L and EQ-VAS, while patient-reported HRQoL and symptom burden were evaluated with EORTC QLQ-C30 and QLQ-H&N35. Toxicities were rated by physicians using CTCAE v4.0 and the UMCG dysphagia scale. Assessments were conducted before radiotherapy, at 12 weeks, and 6, 12, 18, 24, 30, 36, 48, and 60 months post-treatment. Generalized Estimating Equations (GEE) analyzed health utility values. Among 1,851 patients, HRQoL declined during radiotherapy and partially recovered within the first year, remaining stable up to 60 months. Model-estimated EQ-5D utility decreased during treatment and recovered to above baseline levels by 12 months. Dry mouth and sticky saliva persisted at long-term follow-up. In multivariable GEE models, CTCAE Grade 3 xerostomia and dysphagia were associated with utility decrements of -0.051 and -0.062, respectively, while grade 3 pharyngolaryngeal pain showed the largest reduction (-0.106). Increasing physician-rated toxicity severity was consistently associated with greater disutility. Xerostomia, pharyngolaryngeal pain, oral pain, , and dysphagia significantly impacted EQ-5D-derived utility values. Disutility increased with higher CTCAE-rated toxicities. Symptoms persisted for up to five years post-treatment. These findings can inform QALY-based health economic evaluations and suggest interventions targeting key toxicities to improve patient HRQoL.

Purpose To quantify lung and lobar volumes superimposed on the mediastinum and diaphragm on upright chest radiographs using upright multidetector row CT and to compare them with supine measurements. Materials and Methods In this prospective study, asymptomatic Asian participants underwent both upright and supine multidetector row CT within 2 hours. Lung and lobar volumes were measured in each position. Ray-sum images were reconstructed to delineate mediastinal and diaphragmatic boundaries, and superimposed volumes in the posteroanterior direction were calculated. These CT-derived volumes were considered to approximate lung fields projected over the mediastinum and diaphragm on radiographs. Percentages of superimposed volumes relative to total lung and lobar volumes were determined. Mixed-effects models and multiple linear regression analyses were used. Results The study included 132 participants (mean age ± SD, 47.4 years ± 11.4; 85 female; mean body mass index ± SD, 22.2 ± 3.4 [calculated by dividing weight in kilograms by height in meters squared]). The proportion of lung volume superimposed on the mediastinum and diaphragm was 23.9% ± 3.1 in the upright position, significantly lower than 25.7% ± 3.3 in the supine position (P < .001), despite a 9.8% increase in total lung volume upright. Superimposed percentages of the right upper, middle, and lower lobes and the left upper and lower lobes in the upright position were 10.4% ± 2.5, 7.5% ± 3.2, 28.8% ± 7.9, 12.3% ± 1.9, and 46.0% ± 8.3, respectively. All lobes except the left lower lobe showed significantly lower superimposition upright than supine (all P < .03). Multiple linear regression identified older age, higher body mass index, and male sex as independent predictors of greater superimposition in the upright position (all P < .03). Conclusion Lung volume superimposed on the mediastinum and diaphragm was lower on upright than supine chest radiographs despite greater lung volume in the upright position. University Hospital Medical Information Network Clinical Trials Registry: UMIN000026586 Keywords: CT, Thorax, Lung, Mediastinum, Diaphragm, Anatomy, Comparative Studies, Technology Assessment Supplemental material is available for this article. © The Author(s) 2026. Published by the Radiological Society of North America under a CC BY 4.0 license.

Robust evidence is required to support decision-making about incorporating genomics into healthcare; patient perspectives are crucial. Prior studies centre on people giving research consent for testing, yet significant differences between research and clinical cohorts are well established. We investigated 1690 patients offered genomic testing during clinical care by a range of medical specialists, for rare diseases and cancer. Ninety per cent (1515) accepted testing. Of 74 decliners providing their reasons, 20 gave genomic-specific concerns. Impact and experiences of care were captured using surveys after consent (S1:RR 73%) and return of results (S2:RR 53%). We actively included those often missing from research, e.g. 8% of S2 respondents accepted telephone assistance-typically with interpreters - to complete surveys. Those who spoke English as an additional language were less likely to have received enough information at pre-test counselling (88% v 96%) and less likely to correctly answer questions about potential genomic test results. After receiving results, 10% (52/534) of respondents had moderate-high decision regret; predictors included English as an additional language and not receiving enough information at consent. Value from testing was quantified and compared: those with informative results valued their medical and personal utility; those with uninformative results derived social utility. Perceived personal control increased post-result for those with diagnostic results and decreased for those with uninformative results. Our results expand the evidence base available for genomic health technology assessment. On balance, genomic test results provide more value than harm, but equity issues need to be addressed to ensure all patients can benefit.

Rethinking The Role Of Health Technology Assessment: From Gatekeeper To Health System Shaper.

ABSTRACTThe ProblemTransportability considerations are increasingly important to answer research questions in comparative effectiveness research (CER) to support the transfer of evidence on medicinal products across countries or settings. As drug development costs rise and healthcare systems and professionals face economic and resource pressures, leveraging existing data and evidence generated across borders or settings can improve efficiency and inform decisions in product development and decision‐making (regulatory, health technology assessment [HTA] and clinical care). Differences in population characteristics, healthcare systems, and data availability, including coding discrepancies, may present significant challenges to the transportability of CER evidence. Without rigorous methodological approaches, the utility of transportability analyses is limited.What we DidThis article provides a structured framework for considering transportability exercises in CER analyses. We outlined key methodological principles, when and why to use transportability exercises in CER, feasibility assessments including effect modifier identification and causal inference techniques such as weighting, outcome regression, and combined methods to guide transportability analytical approaches in CER. By synthesizing existing literature and expert insights, we identified opportunities and trade‐offs in applying transportability methods to support decisions across the product lifecycle.Strategies to Disseminate and Facilitate UseTo enhance the adoption of transportability analyses, we proposed best practices for researchers, regulators, HTA bodies, and industry stakeholders. These include early engagement with regulatory agencies and HTA bodies, transparent documentation of data assumptions, quality, fitness, and comparability assessments while ensuring robust analytical approaches. We also emphasized the need for standardized reporting guidelines and cross‐country collaborations to validate transportability methods in real‐world settings and communicate uncertainty in transported evidence.ConclusionsTransportability analyses offer a powerful tool for extending the applicability of CER findings across healthcare systems, improving evidence generation efficiency, and supporting global drug development and evaluation. By implementing best practices that promote a rigorous and transparent approach to the design and conduct of such analyses, stakeholders can maximize the value of transported treatment effects while ensuring scientific rigor and decision‐making relevance. Future research should focus on empirical testing and validation of transportability methods targeting different questions across the product lifecycle and the development of harmonized regulatory and HTA methodological standards. “This manuscript is endorsed by the International Society for Pharmacoepidemiology (ISPE).” Official Endorsement was received on 5/13/26.

Regulatory Approval of New Oncology Drugs, 2020-2024: A Comparative Analysis of FDA, EMA, and ANVISA.

Official Digital Health Technology Assessment Guidelines: An International Comparative Review and Analysis.

An interactive capacity-building workshop for process evaluations of complex interventions in critical care.

Technologies such as AI, autonomous drones, gene-editing tools, and climate engineering are profoundly transforming human life in the third decade of the 21st century. Experience of previous technological leaps points to ambivalent effects that at times may be considered "tragic." This paper examines the relationship between technology and tragedy, contributing to the fields of technology ethics, technology assessment, and the philosophy of the human-environment relationship. The relationship between technology and tragedy is double-edged. On the one hand, technology can serve to eliminate tragedy, e.g. by ensuring safety by design, albeit never with guaranteed success. On the other hand, tragic experiences of technological consequences may result from the discrepancy between initial expectations and actual outcomes. A technological consequence is often framed as "tragic" if it was initially associated with high expectations of existential improvement (first necessary condition). Furthermore, a consequence can be labeled "tragic" in perspective only. Tragic is a personal or cultural narration, experiences that a technology's promise of progress is at least partially reversed to produce the opposite (second necessary condition). As elaborated in this paper, tragic consequences can manifest in two ways: First-order tragedy refers to direct reversals of intended effects (e.g. a technology designed to alleviate hunger exacerbates it). Second-order tragedy involves gradual qualitative changes in life, such as diminished human agency or reduced participation - as if humankind were to become "part of a techno-economical system" and lose the freedom to shape life. This is the "tragic of the machine". Ultimately, some "tragic technologies" narrations may also reflect the one-sided projection of experiences of tragedy onto technology.

Rising hospital prices drive healthcare spending and threaten access, fueled by consolidation, weak regulation, and inefficient procurement. This scoping review applied the "Control Knobs" framework to identify and compare policy options for controlling hospital prices. Following PRISMA-ScR guidelines, PubMed, Web of Science, Scopus, Embase, and Google Scholar were searched for English-language studies (Jan 2013-Jun 2024). Studies without hospital-specific price outcomes were excluded. Two reviewers independently screened and extracted data on study characteristics, policy options, and outcomes, which were synthesized narratively across the five "Control Knobs" domains: financing, payment, organization, regulation, and behavior. Of 5,196 records, 50 studies met inclusion criteria, mostly from the U.S. (n = 30), with others from China, Italy, France, and eight additional countries. Policy options covered all five "Control Knobs" domains. Financing tools including competitive tenders, centralized procurement, and national negotiations, consistently reduced prices, particularly in China, Denmark, and India. Payment reforms, including fixed-rate contracts, price caps, reference pricing, and outcome-based procurement, generally lowered prices, though discounted-charge contracts and regional health technology assessment (HTA) sometimes increased them. Organizational changes had mixed effects. Mergers, Accountable Care Organization (ACO) acquisitions, and trauma center monopolies raised prices, while competition from ambulatory surgery centers (ASCs) and group purchasing organizations (GPOs) reduced them. Regulatory measures including caps, most-favored-nation (MFN) bans, and transparency tools, showed modest to substantial price reductions, whereas Certificates of Public Advantage (COPAs) had variable outcomes. Behavioral approaches, including HTA, biosimilar and generic entry, and public reporting, were generally associated with price declines. Hospital price control relies on the interplay of financing, payment, organizational, regulatory, and behavioral mechanisms, with financing and payment reforms exerting direct short-term effects and organizational and regulatory tools shaping long-term market dynamics. The most effective policy options enhanced competition and purchasing power, including competitive tendering, centralized procurement, and promotion of generics and biosimilars. National price negotiations and merger restrictions also contained prices, while transparency, HTA, and outcome-based contracting had modest or context-dependent effects. Some interventions, such as high-deductible health plans (HDHPs), consolidations, and poorly designed regulations, increased prices. Sustainable price control requires a coordinated, context-specific approach that ensures both affordability and equity.