bluuebunny/crossref_metadata_2025 huggingface dataset:
OD04 The EQ-5D-5L Value Set For Ghana
Authors: Addo Rebecca , Mulhern Brendan , Norman Richard , Owusu Richmond , Viney Rosalie , Nonvignon Justice
Published: January 2025

Introduction

Ghana’s reference case, developed to guide the conduct of economic evaluation as part of health technology assessment (HTA) guidelines, recommends the conduct of cost–utility analysis using outcomes such as quality-adjusted life years (QALYs). There is no national value set available for the Ghanaian population to be used in estimating QALYs. This study aimed to develop a value set for Ghana using the EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L) instrument.

Methods

Face-to-face preference data were collected from 300 adults across three regions of Ghana using the adapted version of the EuroQol Valuation Technology (EQ-VT) standardized valuation protocol developed specifically for EQ-5D-5L valuation studies using composite time-trade-off (cTTO) and discrete choice experiments (DCEs). Different preference models were generated using both the cTTO and DCE data, individually or together to provide complementary results on respondents’ utility preferences. Models explored include generalized least squares, tobit, heteroskedastic, logit, and hybrid. The best-fitting model was selected for the value set based on its logical consistency, ability to account for left-censored and heteroskedasticity data, and statistical significance of parameters.

Results

The 300 interviews provided 4,500 cTTO responses and 4,200 DCE responses. The demographic characteristics of respondents were representative of the Ghanaian population for religious background, level of education, and marital status. The preferred model chosen for the Ghana value set was hybrid tobit, random effect heteroskedastic, constrained model. The predicted value for the worst attainable health on the EQ-5D-5L (i.e., health state 55555) was −0.493 and that of the best health state (11112; except full health) was 0.969. The largest decrement was registered for level five mobility (0.369) followed by pain/discomfort (0.312), self-care (0.273), anxiety/depression (0.271), and usual activities (0.268).

Conclusions

This is the first Ghanaian EQ-5D-5L value set based on social preference derived for a nationally representative sample in Ghana. The value set will play a key role in the institutionalization of HTA in Ghana and the use of economic evaluation studies to inform priority setting where different health technologies can be compared. A planned findings dissemination to stakeholders is underway.
OD12 Large Language Models As Powerful Tools In Health Technology Assessment
Authors: Segur-Ferrer Joan , Moltó-Puigmartí Carolina , Lleixà Berta Mestre , Pla Anna Godo , Peiró Roland Pastells , Llinàs-Mallol Laura , Sabadell Maria-Dolors Estrada , Vivanco-Hidalgo Rosa Maria
Published: January 2025

Introduction

In recent years, large language models (LLMs) have emerged as powerful tools in natural language processing, presenting diverse opportunities across various sectors. In this context, the Agency for Health Quality and Assessment of Catalonia (AQuAS) is actively investigating how LLMs can enhance the development of health technology assessment (HTA) reports.

Methods

To assess the potential of LLMs in the development of HTA reports, our initial step involved a comprehensive review of technical literature to understand the functionalities of existing LLM tools. This effort was followed by a systematic identification of specific HTA report development tasks that these models could potentially facilitate. We then rigorously evaluated the performance of these tools in executing the delineated tasks, with a focus on meticulously recording the applied prompts and areas of high and low effectiveness, alongside any encountered errors and identified risks.

Results

Among a range of commercial tools, ChatGPT and Microsoft Copilot were selected for exploration due to their accessibility, ease of use, and versatility. Both tools demonstrated acceptable performance in certain tasks (e.g., data extraction), while their efficacy was notably lower in others (e.g., information search). Additionally, a range of risks requiring mitigation were identified (e.g., hallucination or authority bias). Consequently, we drafted a proposal for an internal guide with directives for the technical staff of the HTA unit on utilizing these two tools. Additionally, we constituted a working group.

Conclusions

LLMs have emerged as promising tools in the field of HTA. Over the past months, AQuAS has been investigating the potential of these models to improve the HTA report development process, targeting enhanced efficiency and improved quality. This exploration has led to the identification of numerous opportunities and associated risks within this innovative application.
OD14 Harmonization Of HTA For Digital Health Technologies: The EU-Funded European Digital Health Technology Assessment Project
Authors: Tsiasiotis Emmanouil , Di Bidino Rossella , Sacchini Dario , Cicchetti Americo
Published: January 2025

Introduction

The European Digital Health Technology Assessment (EDiHTA), an EU-funded project, aims to deliver the first European digital HTA framework co-created by all relevant stakeholders, validated and ready to use. It responds to the need to harmonize tools, methods, guidelines, and frameworks that are already adopted or under development. All types of digital health technologies (DHTs) and Technology Readiness Levels (TRLs) are covered.

Methods

The EDiHTA project adopts a multistakeholder, multidomain, and modular approach to reach a consensus on a common HTA framework for DHTs (e.g., mApp, telemedicine, artificial intelligence). The currently available HTA frameworks refer to different DHTs (e.g., MAST, DiGA, MAS-AI), include different HTA domains, respond to the needs of different stakeholders, and are applicable in specific local and national contexts. In the majority of cases, they are applicable only in advance TRL. In a few cases, they are already adopted in regulatory/HTA processes. The lack of coordination and harmonization has significant implications for all stakeholders, manufacturers and patients included.

Results

The EDiHTA project adopts a holistic approach involving all relevant stakeholders for consensus building at national and European levels. Cross-border collaboration among stakeholders will be reinforced by the definition of a common taxonomy based on consensus building, and the development and validation of common methods and the HTA framework. Evidence requirements will be defined and made more transparent to increase support for health technology manufacturers. In addition, the harmonization effort, together with the development of a digital platform for the HTA framework, will allow faster and safer access to DHTs by patients/citizens.

Conclusions

The goal of the EDiHTA project is to provide the first European digital HTA framework for DHTs that will be adopted by all relevant stakeholders along the lifecycle of digital technologies to promote sustainable health systems. A consortium comprising 15 partners from eight countries, including all relevant stakeholders, works to reach these goals within the next four years.
OD16 Assessing Technical Aids In Europe: Are There Any Specific Health Technology Assessment Methods To Assess Technical Aids?
Authors: Bonamour Julie , Marguerite Camille , Bouaraba Feyriel , Fernandez Judith , Galmiche Hubert
Published: January 2025

Introduction

Technical aids (TAs) are health devices (e.g., hearing aids, prostheses, wheelchairs) used to restore a body function or compensate for disability. In France, TAs are assessed based on clinical data, as are any medical devices (MDs). Considering that TAs are special MDs (no curative action), specific assessment methods might be proposed. The methods used in Europe were explored.

Methods

To learn about TA evaluation practices in Europe, the French National Authority for Health (HAS) sent a survey to nine identified European health technology assessment (HTA) agencies. The questionnaire was specific to TAs, and the questions asked were about: (i) the reimbursement of TAs (reimbursed or not, at national or local level), (ii) the HTA process (HTA process or not, HTA process description), and (iii) the methods used (any specific methods for assessing TAs other than HTA methods to assess “classic” MDs).

Results

All the European HTA agencies contacted provided a response. All these countries have a healthcare system that allows TAs to be reimbursed. In Austria, Ireland, Italy, the Netherlands, Norway, Portugal, Spain, and the United Kingdom, TAs must be CE-marked and comply with technical standards to be reimbursed; no clinical data are required. However, the National Institute for Health and Care Excellence provides recommendations for certain TA categories. In Germany, the CE-marking and the respect of technical standards are mandatory, and certain TAs need to show a medical benefit (e.g., orthoses); nevertheless, there are no specific methods to assess TAs.

Conclusions

Overall, the feedback obtained does not reveal any specific evaluation method applied to TA assessment in the various European countries analyzed. France stands out from these European countries regarding the assessment of TAs because of its one-stop-shop system for health technology developers and the HAS requirements for technical and clinical data.
OD18 Surrogate Measures And The Health Technology Assessment Of Cancer Drugs In Ireland: A Retrospective Analysis, 2017 to 2022
Authors: Assi Alexandre , Karbanova Tereza , Algawas Lama , Darren Lim Yue Xiang , Trela-larsen Lea , Usher Cara , Barry Michael , Walsh Caroline
Published: January 2025

Introduction

Surrogate endpoints are increasingly being used in the pivotal trials of cancer drugs to underpin (conditional) regulatory approval. We examined the relationship between the use of surrogate measures in pivotal trials underpinning cancer drug approvals by the European Medicines Agency (EMA) between 2017 and 2022 and health technology assessment (HTA) recommendations made by the National Centre for Pharmacoeconomics in Ireland (NCPE).

Methods

A previously published methodology was used to identify cancer drug indications that received (conditional) marketing authorization between 2017 and 2022, inclusive. EMA-approved cancer drugs were categorized using the following benefit categories, based on pivotal trial endpoints: overall survival (OS), progression-free survival (PFS), disease response (DR), and single-arm trials (SATs). The NCPE website was searched to identify indications that had undergone, at least, a rapid review (RR) assessment. The NCPE recommendation for each assessment was recorded. Additional data including the incremental quality-adjusted life years (QALY) gain reported in cost-effectiveness analyses were extracted for indications that had undergone a full HTA.

Results

One hundred and eight cancer drug indications were identified, comprising 68 cancer drugs. In 2017, OS, PFS, and SAT benefit underpinned equal proportions of approvals (28.6% each). In 2022, SAT underpinned the largest proportion of approvals (53.6%). As of June 2023, 77 indications (71.3%) had undergone at least a RR assessment; 31 indications had completed a full HTA appraisal. All of the indications underpinned by SAT evidence (n=7) received a conditional negative recommendation. Indications with SAT evidence had a mean incremental QALY gain of 1.88 (standard deviation [SD] 1.20), whereas indications with an OS benefit had a mean incremental QALY gain of 0.81 (SD 0.36).

Conclusions

The proportion of cancer drug indications receiving regulatory approval on the basis of SAT evidence, where no direct comparative evidence is available, is increasing. This results in additional uncertainty in the comparative benefit of cancer drugs supported by SAT evidence. The study is limited by the sample size of HTA appraisals included. Further in-depth analysis of factors influencing NCPE recommendations is needed.
OD32 Single Nucleotide Polymorphism Marker Analysis In Donor-Derived Cell-Free DNA For Solid Organ Transplantation (Next-Generation Sequencing): Systematic Review
Authors: Kim Yoojin , Im Jiae , Lee Wurlsook , Choi Jieun
Published: January 2025

Introduction

Single nucleotide polymorphism marker analysis in donor-derived cell-free (dd-cf
) DNA for solid organ transplantation is a technique for post-transplantation monitoring, including early detection of injury to the transplanted organ, signs of infection, and treatment decision-making, by measuring dd-cf-DNAs as a percentage of the total cf-DNAs in the patient’s body in donors and recipients of kidney, heart, lung, or liver transplants.

Methods

The assessments were performed via a systematic review. Searching five databases (KoreaMed, Ovid MEDLINE, Ovid Embase, and Cochrane) yielded 1,619 related studies. Two reviewers independently assessed the quality of these studies using a Scottish Intercollegiate Guidelines Network checklist, and the assessment results were described based on the results of the quality appraisal and level of evidence.

Results

The population of the included studies was patients who underwent kidney, heart, lung, or liver transplantation. Medical outcomes in kidney transplantation patients were reported in 15 studies. The index test was reported to have an area under curve (AUC) of 0.68 to 0.99 and a sensitivity of 0.24 to 1.00. Four studies reported effectiveness data for the index test for lung transplantation patients. The diagnostic accuracy of the index test for acute cell-mediated rejection was reported have an AUC of 0.72. Sensitivity was reported as 0.44 and specificity as 0.80 for heart transplant patients, and sensitivity as 0.73 to 1.00 and specificity as 0.60 to 0.95 for liver transplant patients.

Conclusions

This is a safe and effective technique for post-transplantation monitoring, including the early detection of injury to the transplanted organ and signs of infection, and for treatment decision-making, by measuring dd-cf-DNA as a percentage of total cf-DNA in the patient’s body in recipients and donors of kidney, heart, lung, or liver transplantation (level of evidence: C).
OD39 Evaluation Of Sustainability And Environmental Impact Measures In Health Technology Assessment Of Medical Technologies In Europe: Starting An Important Journey
Authors: Orozco María Álvarez , Monje Julen , Hallas Natalie , Vogt-Humberg Ilona , Borghetti Francesca , Mauratille Constance , Strachan Liesl
Published: January 2025

Introduction

Reducing environmental impact and ensuring sustainable practices are important to the medical technology (MedTech) industry. However, it is unclear how these factors will be formally addressed by health technology assessment (HTA). The objective of this research was to understand what sustainability measures are required and included in HTA reports on medical technologies in five European countries (France, England, Germany, Italy, and Spain).

Methods

HTA guidelines, framework papers, and published HTA reports for devices were searched from January 2022 to November 2023 for inclusion of environmental aspects. Search terms included environmental sustainability, carbon footprint, greenhouse gas, and waste generation. Data were extracted into standardized templates and analyzed both quantitively and qualitatively for the inclusion of sustainability and environmental impact measures.

Results

HTA guidance on inclusion of sustainability and environmental measures is lacking. While some countries request the inclusion of these aspects in the HTA process, there is little detail on evidence requirements and how it will be evaluated. Of the over 450 HTA reports examined, less than 10 percent included environmental aspects, with most sustainability benefit claims made related to the reduction in waste or less resources used. Very few claims were supported by published evidence. This resulted in the exclusion of potential environmental benefits in final HTA recommendations. Costs associated with environmental impact of medical technologies were rarely assessed.

Conclusions

Current inclusion and evaluation of sustainability measures in MedTech HTA in Europe is nascent. While countries like England [National Institute for Health and Care Excellence (NICE)] and France [Haute Autorité de santé (HAS)] have very recently made public statements regarding the importance of environmental impact, there is a growing need for detailed guidance on environmental evidence requirements and how these will be incorporated into the evaluation and decision-making processes.
OD45 Evaluating Quality In Health Economics: Quality Appraisal Checklist For Systematic Reviews Of Studies Eliciting Health State Utility Values
Authors: Muchadeyi Muchandifunga T. , Hernandez-Villafuerte Karla , Di Tanna Gian Luca , Eckford Rachel , Feng Yan , Meregaglia Michela , Peasgood Tessa , Petrou Stavros , Ubels Jasper , Schlander Michael
Published: January 2025

Introduction

The reliability of cost–utility analyses depends on the quality of health state utility values (HSUVs). Given the increasing number of studies eliciting HSUVs, systematic reviews (SRs) are vital to economic evaluations. Nevertheless, a universally acceptable quality appraisal (QA) tool specific to the SRs of HSUV studies is lacking—this study aimed to develop one and fill this gap.

Methods

We employed a mixed-method approach, starting with a rapid review to identify QA dimensions, QA items, and terminology in the SRs of HSUV-eliciting studies. This informed a modified Delphi process with a seven-member international expert panel, aiming to define key terms, refine the QA tool dimensions, and establish relevant signaling questions. The experts participated in two anonymous online survey rounds interspersed with structured feedback, enabling iterative refinement of their views. Following these surveys, a virtual face-to-face meeting was held to resolve outstanding issues. Consensus was defined a priori at all stages of the modified Delphi process.

Results

The rapid review identified three QA dimensions and 16 initial items, noting the diverse terminologies in defining QA. Response rates to the first- and second-round questionnaires and the virtual consensus meeting were 100, 86, and 71 percent, leading to a consensus on the definitions of scientific quality, QA, the three QA dimensions (reporting, methodological limitation, and risk of bias and relevance), and scope of the QA tool. The number of QA items was refined to 14: all relevant to reporting, six to relevance, and 11 to methodological limitations and bias risk dimensions. The QA tool underscores distinct evaluations for each dimension.

Conclusions

We present the first version of a QA checklist designed to provide SR authors with a tool to appraise the quality of HSUV-eliciting studies comprehensively. The QA tool aims to (i) facilitate QA in SRs of HSUV elicitation studies, (ii) promote consistency in the appraisal process, and (iii) emphasize the importance of differentiating between reporting quality, methodology, and relevance.
PP03 Investigating Technological Strategies In The Hospital Setting: Insights From The Dutch Context
Authors: Pinelli Maria , Lettieri Emanuele , Tummers Marcia , Grutters Janneke
Published: January 2025

Introduction

Rapid advancements in technology are significantly impacting the healthcare system, and decision-making regarding technology adoption occurs at multiple decentralized levels within hospitals. National bodies seek to standardize this process, yet differing visions and strategies hinder centralization. This study explores the relationship between technological innovation and hospital strategies, focusing on scanning and assessment, aiming to assess the feasibility of centralized decision-making.

Methods

To do this, we performed a qualitative analysis through 23 semistructured interviews in seven hospitals in the Netherlands, a country characterized by strong healthcare innovation and decentralization. We interviewed different actors involved in technological innovation, on different levels in the organization: CEOs, medical doctors, medical physicists or similar roles, and innovation managers. Ethics approval was obtained, and interviews were conducted, recorded, transcribed, and shared with participants for accuracy confirmation. Thematic analysis via grounded theory methodology and ATLAS.ti software generated insights on technological innovation’s relationship to hospitals’ strategies. Initial codes were refined into themes relevant to the research question.

Results

Hospitals primarily aim to provide optimal patient care, with academic hospitals emphasizing research and education. Some hospitals aspire to be pioneers in adopting new technologies, while patient-centric healthcare is a shared goal. Technological strategies are not precisely designed in hospitals, being shaped by factors like people, financial constraints, or external environments. Hospitals’ scanning of technologies lacks systematization, and evaluations before and after technology adoption are not univocally performed. The need for systematic scanning and assessment practices is recognized by some interviewees, while others emphasize the importance of experimenting without the constraint of evaluation, perceiving it as a hurdle delaying innovation.

Conclusions

Centralization could represent a benefit for hospitals, allowing them more streamlined decision-making, but it could also be perceived as a barrier. Involving hospitals’ stakeholders in centralization would be crucial to achieve it through a joint effort. Suggestions for future research could include focusing on a specific hospital, involving more stakeholders, and exploring other decentralized healthcare systems.
PP04 Assessing The Utility Of Natural Language Processing In Generating A Granular Estimated Indication For A Horizon Scanning Database
Authors: Choitir Caitríona Ní , Harte Marie , Duell Daisy , Odnoletkova Irina , Christensen Anna Bergkvist , Persson Marie , Bräuner Helle , Guardian Marcus , Tuckova Alzbeta Alzbeta , Wilkinson Brian , Kuzmak Beth , Adams Roisin , Shaw John , Boyce Craig , Palakollu Prashanth , Erinoff Eileen
Published: January 2025

Introduction

Detailed, precise information on a pharmaceutical’s projected therapeutic use is required for horizon scanning. Inferring an estimated indication from trial protocols is a key skill of horizon scanners. The International Horizon Scanning Initiative (IHSI) database utilizes semi-automated data collection. This pilot aimed to verify that the extraction of relevant word sets to generate an estimated indication could be semi-automated.

Methods

Ten drugs approved in Europe in 2021 were selected as the pilot test set. The test set included drugs approved for the treatment of rare diseases (n=4), haemato-oncology (n=3), and non-oncology conditions (n=3). Eight of the drugs were approved based on phase III trials. The assessment comprised a review of the pivotal trial that supported product registration for these drugs. We undertook a comparison between a human curator and a natural language processing (NLP) algorithm in generating granular tags relating to key aspects of the drugs’ estimated indication (stage of disease, patient-specific subgroup, and place in treatment).

Results

In 50 percent of cases, the NLP accurately tagged a word or word set related to stage of disease, patient-specific subgroup, or place in treatment, which was also tagged by human curators. In 50 percent of cases, the NLP did not identify words or word sets tagged by human curators. Where relevant, the NLP successfully tagged the same word sets relating to stage of disease for all drugs in the test set. The same word sets relating to patient-specific subgroup were successfully tagged for three drugs in the set. NLP successfully tagged word sets relating to place in treatment for two drugs.

Conclusions

The NLP algorithm is successful in extracting relevant word sets, which can be used to generate an estimated indication in an automated or semi-automated process. The pilot highlighted that further testing is required to advance the sensitivity of the algorithm. Further piloting exploring both unsupervised and supervised modeling approaches (named entity recognition and deep neural networks, respectively) is planned.
PP12 Projecting The Potential Impact Of Disease-Modifying Therapies For Alzheimer’s Disease On The Carbon Emissions From Hospital Bed-Days in the UK
Authors: Trepel Dominic , Edwards Sophie , Ritchie Craig , Hahn-Pedersen Julie Hviid , Kettle Jamie , Chan Mei Sum , Bray Benjamin D , Clark Alice , Ivkovic Milana , Wichmann Christian Ahmad , Evans Marc
Published: January 2025

Introduction

Disease-modifying therapies (DMTs) for Alzheimer’s disease (AD) are emerging treatment options. This study aimed to estimate the potential health system and associated environmental impacts of DMTs by modeling future bed-days and carbon dioxide equivalent (CO2e) emissions for the UK population under various scenarios for access to and efficacy of DMTs.

Methods

A cohort Markov model was developed to predict the UK population distribution from 2020 to 2040 across five health states—cognitively unimpaired and four stages of AD (mild cognitive impairment, and mild, moderate, severe dementia). These distributions were estimated using national population projections, AD prevalence data, and stage-specific transition rates. Annual bed-days per person for each state and associated CO2e emissions from published literature were applied to estimate total bed-days and emissions. Modeled scenarios combined ranges of DMT efficacy estimates (20 to 30%) and access levels (25 to 58% eligible patients receiving treatment) elicited from expert opinion to explore the extent of potential DMT impacts.

Results

Without DMT access, annual bed-days across the four AD stages were projected to increase from 5.5 million to 8.6 million from 2020 to 2040, with cumulative bed-days totaling 140 million. Associated annual emissions increased from 0.7 Mt to 1.1 Mt CO2e, reaching 17 Mt CO2e cumulatively from 2020 to 2040. Under the various high-access (58% eligible patients treated) DMT efficacy scenarios, relative to no DMT access, annual reductions of 430 thousand to 650 thousand bed-days and 54 kt to 81 kt CO2e were estimated by 2040, and cumulative emissions decreased by 419 kt to 633 kt CO2e. Decreasing DMT access to 25 percent, assuming 25 percent DMT efficacy, reduced annual bed-days by 230 thousand by 2040, and annual emission savings decreased to 29 kt CO2e.

Conclusions

DMTs for AD may contribute to efforts by healthcare systems to reduce the carbon emissions from hospital inpatient care. Environmental sustainability should be considered as part of a holistic value proposition when assessing the benefits of new medicines.
PP13 Cost-Effectiveness Analysis Of Antiandrogen Therapies For Metastatic Castration-Sensitive Prostate Cancer: The Brazilian Healthcare System Perspective
Authors: Gargano Ludmila , Oliveira Layssa , De Oliveira Haliton Alves , Lucchetta Rosa
Published: January 2025

Introduction

Brazilian Unified Health System (SUS) Clinical Practice Guidelines (CPG) for prostate cancer recommend the use of androgen deprivation therapy (ADT) ± docetaxel for metastatic castration-sensitive patients (mCSPC). However, new medications have been developed since the last CPG update. We assessed the cost-effectiveness of abiraterone, apalutamide, darolutamide, and enzalutamide for mCSPC as part of the CPG update process.

Methods

A cost–utility analysis was built, with partitioned survival model composed of three states: progression-free, evidence of radiological progression, and death. The overall survival (OS) and progression-free survival (PFS) curves from docetaxel STAMPEDE trial were extrapolated using different distributions for a 20-year horizon. Visual inspection, clinical plausibility, and Akaike information criterion (AIC)/Bayesian information criterion (BIC) tests were considered to choose the best fit, which were adjusted by hazard ratios (HR) from indirect comparisons of interventions with docetaxel. Utility and disutility values were identified from the literature. Direct costs of medications and pre- and post-progression disease monitoring were considered. We considered a BRL120,000/quality-adjusted life year (QALY) (i.e., USD24,511) cost-effectiveness threshold.

Results

When compared to docetaxel+ADT, interventions with higher effectiveness gains were abiraterone+docetaxel and darolutamide+docetaxel (0.94 and 0.99 QALY, respectively). Apalutamide and enzalutamide showed the highest incremental cost (BLR637,342 and BLR516,313, [i.e., USD130,187 and USD105,465], respectively). Abiraterone monotherapy or +docetaxel presented the lowest incremental cost–utility ratio (ICUR) (BLR84,960 and BLR79,428/QALY gained [i.e., USD17,354 and USD16,224], respectively) and may be cost effective for the SUS. Apalutamide, enzalutamide, and darolutamide may not be cost effective for the SUS as ICUR were higher than the cost-effectiveness threshold. Probabilistic sensitivity analyses or using different distributions for survival curves confirmed the direction of the findings.

Conclusions

Recent studies have demonstrated that adding docetaxel or antiandrogen drugs to ADT in men with mCSPC can improve OS and PFS compared with ADT alone. However, in this analysis, only abiraterone was cost effective, and the incorporation of apalutamide, darolutamide, and enzalutamide would require substantial price reductions to be cost effective for the SUS.
PP15 Cost–Benefit Analysis Of An Antimicrobial Stewardship Program In A Cancer Setting In Qatar
Authors: Abushanab Dina , Moustafa Diala Alhaj , Sankar Bhagyasree Adampally , Nasr Ziad , Al Hail Moza , Hamad Anas , Abdul Rouf Palli Valappila , Elkassem Wessam , Thomas Binny , Al-Badriyeh Daoud
Published: January 2025

Introduction

The inappropriate use of antimicrobial agents has been associated with increased healthcare costs and the spread of multidrug-resistant organisms. We aimed to estimate the economic impact of the developed antimicrobial stewardship program (ASP), after five years of implementation, versus the preliminary ASP, upon implementation, in the cancer setting at the National Center for Cancer Care and Research (NCCCR) in Qatar.

Methods

The research investigated the economic benefits of employing a preliminary ASP versus a developed ASP from the perspective of a public healthcare hospital. Preliminary ASP was defined as the 12 months following the establishment of the ASP (i.e., 1 May 2015 to 30 April 2016), while developed ASP was defined as the most recent 12 months of ASP implementation (i.e., 1 February 2019 to 31 January 2020). Patient records were retrospectively reviewed. The total economic benefit of ASP maturity was calculated as the sum of the cost savings and the cost avoidance associated with the service, minus the operational cost.

Results

A total of 1,000 patients were included in the study. The developed ASP was associated with substantial reduction in antimicrobial consumption and resource utilization. Total cost of resources to avoid during the developed ASP period was USD1,634,658, in contrast to USD4,923,024 during the preliminary ASP period, yielding a positive cost avoidance of USD3,288,366. Developed ASP incurred lower operating costs than preliminary ASP, resulting in positive change in operational cost of USD3,428. The benefit-to-cost ratio was 640. The net benefit due to ASP maturity was USD3,624,875. The robustness of the results is demonstrated by the sensitivity analysis.

Conclusions

This study underscores the benefits of ASP development in a cancer setting. The observed reductions in antimicrobial costs from reduced antimicrobial use and the added value of cost avoidance signify the positive impact of a well-developed ASP. These findings lend support to the broader implementation of comprehensive ASPs, which contribute not only to patient-care optimization but also to cost containment.
PP17 Navigating Health Technology Assessment (HTA) Requirements During Development Through Early HTA Scientific Advice: Insights From Companies’ Strategies, Challenges, And Priorities
Authors: Wang Tina , McAuslane Neil
Published: January 2025

Introduction

Pharmaceutical companies have been actively taking early scientific advice from health technology assessment (HTA) agencies during development, focusing on study design to understand the HTA evidentiary requirements. The evolving advice landscape, including multistakeholder and international collaborations, highlights proactive engagement’s importance. This opinion survey assessed international pharmaceutical companies’ current experiences in seeking early HTA and explored strategies for forward-looking actions and considerations.

Methods

An opinion survey was designed and conducted in 2023 as a cross-sectional questionnaire consisting of multiple-choice questions. The questionnaire provided a qualitative assessment of companies’ current strategies and experiences in taking early HTA advice as well as future considerations. Eligible survey participants were the senior management of Global HTA/market access departments at 22 top international pharmaceutical companies.

Results

Responses were received from 13 companies. The National Institute for Health and Care Excellence (NICE) or the Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA) were utilized mostly for early HTA advice (92% respondents). Past European Medicines Agency HTA experience exists (50%), but there is no current engagement in Joint Scientific Consultation (JSC). However, JSC is deemed a top priority (83%) given the Regulation (EU) 2021/2282 on health technology assessment. Challenges in seeking advice include agency availability and internal resource and timing constraint. Advice-seeking actions mostly occurred during phase II trials but were frequently limited by agencies’ availability (84% respondents). Divergences were identified regarding eligibility criteria to seek advice and internal practices. Five success indicators were identified with the top-rated being the impact on development plan.

Conclusions

This survey assessed companies’ practices in seeking early HTA advice, with most engaging national agencies like NICE and G-BA. The lack of current JSC participation, despite companies’ prioritization, highlights the agencies’ need to enhance capacity and resources. Survey results underscored the importance of companies’ adaptive strategies in the evolving environment, which can be supported by active measures of advice success.
PP26 Systematic Literature Review Of Funding Models For The Access To Drugs For Patients With Rare Diseases
Authors: Parada Constanza Vargas
Published: January 2025

Introduction

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.

Methods

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.

Results

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.

Conclusions

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.
PP37 Guidance On Using Hospital-Based Real-World Evidence In Health Technology Assessments For Oncology
Authors: Al-khayat Zainab , Franzen Nora , Retèl Valesca P , van Harten Wim H
Published: January 2025

Introduction

Real-world evidence (RWE) is increasingly used in healthcare research to address evidence gaps, reduce uncertainty about medical technology benefits, and provide real-world insights. Efforts to integrate RWE in regulatory and health technology assessment (HTA) processes are growing. However, variations among countries pose challenges. The objective is to analyze and compare various (inter)national RWE guidelines, focusing on real-world hospital data utilization.

Methods

We conducted a review to identify RWE guidance published from 2016 to 2023, with a focus on the EU5 nations (UK, France, Germany, Italy, and Spain) and the ONCOVALUE consortium affiliates (Finland, the Netherlands, Denmark, Italy, and Portugal). To ensure a comprehensive overview, we also investigated Canada, the European Medicines Agency (EMA), ISPOR, and the European Society for Medical Oncology (ESMO). We conducted in-depth interviews with HTA experts of all included countries, focusing on real-world hospital data within the European HTA context. The interviews underwent thematic analysis related to the utilization of RWE in HTA.

Results

We identified nine guidance reports: six focused on HTA-RWE (Medicinrådet/Denmark, NICE/UK, AQuAS/Spain, HAS/France, IQWiG/Germany, CADTH/Canada), one from EMA, and two international (ISPOR, ESMO). Only NICE, IQWiG, and CADTH offered recommendations covering hospital data, emphasizing the data curation process. HAS addressed considerations in choosing secondary data sources, while IQWiG established robust criteria for registries to ensure data quality. Regarding patient-reported outcomes data, only HAS and NICE provided recommendations in their guidance. The HTA experts acknowledged the value of hospital data but expressed caution due to its unstructured nature, noting that the use of hospital-based RWE is more accepted in descriptive studies.

Conclusions

Guidances prioritize the clinical domain, emphasizing transparency, fitness for purpose, reproducibility, robustness, bias minimization, and generalizability. Notably, there’s a lack of comprehensive source-specific guidance for real-world data sources, including registries, hospitals, claims, and wearables. Enhanced guidance on the total data generation process, (data mapping, data federation), cost data, quality of life, and cross-border data usage would strengthen hospital-based RWE assessments.
PP46 Economic Value Of Minimally Invasive Procedures: Examining Revolutionary And Evolutionary Devices In Aortic Stenosis And Benign Prostatic Hyperplasia
Authors: Lim Schezn , Swan Georgia , Evans Jennifer Sara , Lim Andrew , Fujita Natsumi , Lo Justin , Priest Virginia
Published: January 2025

Introduction

Minimally invasive procedures (MIPs) shorten procedural, hospital, and patient recovery time, providing patient benefits and reducing healthcare resource use (HCRU). This study explored the economic impact of two MIPs as discussed in published literature: transcatheter aortic valve replacement (TAVI), a step-change in cardiac clinical practice (“revolutionary”); and water vapor thermal therapy (WVTT), a progressive innovation in benign prostatic hyperplasia (BPH) (“evolutionary”).

Methods

Two pragmatic literature reviews were conducted to identify studies reporting comparative HCRU for TAVI versus surgical aortic valve replacement (SAVR) in aortic stenosis (AS), and WVTT versus other procedures in BPH. Searches were conducted on 20 October 2023 in the MEDLINE and MEDLINE plus Embase databases for the AS and BPH reviews, respectively. Studies from Asia-Pacific (AS review) and all geographies (BPH review) were included. HCRU data reported in the literature were identified and extracted.

Results

Forty eligible studies were included (14 AS, 26 BPH). Commonly reported outcomes were hospital length of stay (LOS) (AS, BPH studies), intensive care unit (ICU) LOS (AS studies), and procedure duration (BPH studies). In AS studies, hospital LOS was shorter for TAVI (8.00 to 19.96 days) than SAVR (13.09 to 29.50 days). ICU LOS was shorter with TAVI (0.00 to 6.40 days) than SAVR (1.00 to 8.13 days). In BPH, WVTT had shorter hospital LOS (0.00 to 1.10 days) than other procedures (1.00 to 3.00 days). Of five studies reporting procedure duration, four showed shorter procedure time with WVTT (4.00 to 30.00 mins) than other procedures (5.20 to 148.00 mins).

Conclusions

MIPs, whether “revolutionary” or “evolutionary,” could offer notable time-savings (both in terms of procedure duration and length of stay) at the hospital level, which may also lead to cost savings. This highlights the importance of establishing mechanisms in payer-level economic evaluations or health technology assessments that can account for the time-saving advantages of MIPs at the hospital level.
PP48 Cost-Effectiveness Of Mepolizumab In Children And Adolescents With Refractory Severe Eosinophilic Asthma In Brazil
Authors: Zimmermann Ivan Ricardo , Elias Flávia Tavares , da Silva EricaTatiane
Published: January 2025

Introduction

Asthma affects individuals of all ages and is the most common chronic disease among children. The Brazilian Unified Health System (SUS) reimburses the use of mepolizumab as an additional maintenance treatment for severe eosinophilic asthma in adults. This study aimed to evaluate the cost-effectiveness of expanding the reimbursement of mepolizumab to patients aged six years and older.

Methods

The model included patients aged six years and older refractory to inhaled corticosteroid (ICS) plus long-acting beta-agonist (LABA) treatment. Baseline characteristics and outcomes were based on clinical trials and national hospitalization data. A Markov model with monthly cycles considered the health states without exacerbation, with exacerbation (including the need for oral steroids, emergency room admission, or hospitalization), and death. Direct medical costs (rate: USD1=BRL4.93) associated with all treatments were estimated from public health reimbursement sources. Quality-adjusted life years (QALY) was the primary outcome. Deterministic (Tornado) and probabilistic (Monte Carlo simulations) sensitivity analyses were conducted using Microsoft Excel.

Results

Considering the current mepolizumab price (USD964.49) reimbursed by the Ministry of Health (MOH), the incremental cost-effectiveness ratio (ICER) was USD137,384.92/QALY (95% confidence interval: USD92,625.64, USD208,542.44), exceeding the USD24,333.86 threshold proposed by the National Committee for Health Technology Incorporation (Conitec). Using the average price from local public purchases (USD417.74), the ICER was USD96,673.51/QALY. Considering the past proposed discount from the manufacturer (USD390,93) and dose fractionation in children up to 12 years, the ICER was estimated in USD50,574.06/QALY. The most impactful parameters were the utility without exacerbation, mepolizumab cost, and relative reduction in exacerbation rate.

Conclusions

Considering current reimbursement prices, mepolizumab use in children and adolescents with refractory severe eosinophilic asthma is not cost effective in Brazil. Alternative scenarios with price discounts and dose fractionation may alter these conclusions.
PP50 Facilitating Academic Life Science Innovation With Early Health Technology Assessment: A Survey Of Potential User Needs And Perceptions
Authors: Dragojlovic Nick , Nagase Fernanda , Kopac Nicola , Yi (Grace) Meng Fan , Akinsoji Toluwase , Lynd Larry D.
Published: January 2025

Introduction

Academic life scientists often struggle to develop and commercialize concrete medical products based on their discoveries. Early health technology assessment (eHTA) can help innovators to define target product profiles (TPPs) with strong value propositions. To understand how eHTA can best help facilitate the clinical translation of university-based inventions, we conducted a survey of stakeholders in the life science innovation ecosystem.

Methods

Our 10-minute online survey includes questions on respondents’ location, organizational affiliations, experiences in health technology development, and awareness and perceptions of eHTA. eHTA is broadly defined as the use of tools from health economics, epidemiology, management, and related disciplines to assess the potential value of a medical product candidate for patients, payers, providers, manufacturers, and other stakeholders. The survey is being advertised using social media and email, and it will be followed up with semistructured interviews. Data on 51 complete responses were summarized using frequency tables and cross-tabulations, and the statistical significance of subgroup differences was evaluated using Fisher’s exact test.

Results

Of 51 respondents, a majority lived in Canada (38/51; 75%) and had an academic affiliation (39/51; 76%). A “lack of commercialization skills among academic life science teams” was identified as a barrier to clinical translation by 41 percent (21/51), though this varied by academic affiliation (33% vs 67%; p=0.051) and industry experience (65% vs 29%; p=0.033). While 31 percent (16/51) reported familiarity with eHTA, this also varied by academic affiliation (23% vs 58%; p=0.033). Only 20 percent (10/51) had previously used eHTA, but a majority expressed an interest in learning more (39/51; 76%) and in using eHTA in the future (31/51; 61%).

Conclusions

Making eHTA more accessible for academic life scientists who lack commercialization experience may mitigate an important barrier to clinical translation of university-developed health technologies. While awareness of eHTA is relatively low in this group, they are interested in learning more about and using eHTA, and efforts should be made to integrate eHTA with existing product development tools like the TPP.