bluuebunny/crossref_metadata_2025 huggingface dataset:
OP56 Hospital-Based Health Technology Assessment: Barriers And Facilitators In France, Hungary, Italy, Kazakhstan, Poland, Switzerland, And Ukraine
Authors: Di Bidino Rossella , Lipska Iga , Kukla Monika , Nemeth Bertalan , Hren Rok
Published: January 2025

Introduction

The adoption and development of health technology assessment (HTA) in the hospital setting (HB-HTA) differs among countries, but in all cases, it encounters barriers and facilitators. An analysis was conducted to promote international cooperation to develop strategies both to enforce common facilitators and overcome common barriers. HTA specialists from seven countries (at least two per country) contribute to the study.

Methods

HTA experts from countries in Western Europe (three), Central and Eastern Europe (three), and Central Asia (one) voluntarily participated in the project. They provided a description of the scenario that HB-HTA faces in their country, then a two-round Delphi study was conducted. The survey was based on twelve statements that were categorized into external and internal barriers and external and internal facilitators. Next, panel experts ranked statements on a seven-point Likert scale with a median agreement score greater than or equal to six and an interquartile range (IQR) greater than or equal to one accepted as reaching consensus. The goal was to identify similarities and differences in the HB-HTA scenarios among countries.

Results

Fifteen experts from France, Hungary, Italy, Kazakhstan, Poland, Switzerland, and Ukraine contributed to the analysis. Among the twelve statements, six were ranked as reaching consensus (two barriers, four facilitators). One external and one internal barrier, which reached consensus, were (i) lack of formal recognition of the role of HB-HTA in national/regional legislations and (ii) limited human resources. Two external facilitators were (iii) creation of a network among hospitals performing HB-HTA and (iv) dissemination of HB-HTA methods and activities, while two internal facilitators were (v) top hospital management support in evidence-based decision-making and (vi) training initiatives dedicated to HB-HTA.

Conclusions

The analysis showed a consensus on the barriers and facilitators for HB-HTA. This creates the opportunity for internationally developed strategies to enforce facilitators and support the adoption and sustainability of HB-HTA. Future research should extend the analysis to other countries and include the results of the HB-HTA survey conducted in 2023 by the HB-HTA Interest Group.
OP61 Target Trial Emulation To Determine The Population-Level Cost-Effectiveness Of Multigene Panel Sequencing For Advanced Melanoma
Authors: Krebs Emanuel , Weymann Deirdre , Pollard Samantha , Regier Dean
Published: January 2025

Introduction

Compared to single-gene BRAF testing to guide targeted treatment with BRAF/MEK inhibitors for advanced melanoma, multigene panels can identify additional gene mutations with known therapeutic or prognostic relevance. Implementation of multigene panels remains uneven across healthcare systems given an uncertain clinical and economic evidence base. We determined the population-level cost-effectiveness of multigene panels compared to single-gene BRAF testing for advanced melanoma.

Methods

Our population-based retrospective study emulated a hypothetical pragmatic trial comparing multigene panel sequencing to single-gene BRAF testing. We drew on comprehensive patient-level clinical and health administrative data between September 2016 and December 2018 in British Columbia, Canada. To emulate random treatment assignment, we 1:1 matched multigene panel patients to contemporaneous single-gene tested controls using genetic algorithm-based matching. We estimated three-year overall survival and healthcare costs (2021 CAD), and incremental net monetary benefit (INMB) for life years gained (LYG) using inverse probability of censoring weighted linear regression and nonparametric bootstrapping. We also estimated overall survival using Weibull regression and Kaplan–Meier survival analysis.

Results

We matched 147 patients with advanced melanoma receiving multigene panel sequencing to contemporaneous single-gene-tested controls, achieving good balance for all 15 baseline clinical and sociodemographic covariates. After matching, mean incremental costs were CAD19,447 (USD14,217) (95% confidence interval [CI]: −CAD18,517 [−USD13,537], CAD76,006 [USD55,565]; p=0.41) and mean incremental LYG were 0.22 (95% CI: −0.05, 0.49; p=0.12). We found uncertain differences on overall survival using Kaplan–Meier (stratified Log-rank test p=0.11) and Weibull regression (HR: 0.73 [95% CI: 0.51, 1.03]; p=0.07) survival analysis. Cost differences were driven by systemic therapy (∆C: CAD8,665 [USD6,334]; 95% CI: −CAD36,387 [−USD26,600], CAD53,716 [USD39,268]; p=0.71). The INMB at CAD100,000(USD73,104)/LYG was CAD2,646 (USD1,934) (95% CI: −CAD30,044 [−USD21,963], CAD43,416 [USD31,739]; p=0.89), with a 52.8 percent probability of being cost effective.

Conclusions

There were clinically relevant but uncertain differences in improved survival associated with multigene panel sequencing for advanced melanoma, and the cost-effectiveness of panel-based testing was finely balanced. This real-world evidence generated using randomized trial design principles can support jurisdictions’ deliberations on the reimbursement of precision oncology interventions.
OP73 Navigating The Advanced Therapy Medicinal Products (ATMPs) Conundrum: Insights From ATMPs Withdrawn In The European Market
Authors: Leong Katherine , Macaulay Richard
Published: January 2025

Introduction

Advanced therapy medicinal product (ATMP) is a classification used by the European Medicines Agency (EMA) for cell-, gene-, and tissue-engineered therapies. Since the first ATMP received market authorization in 2009, a substantial proportion of these highly innovative therapies have been withdrawn from the European market. This research investigates the key reasons underlying these withdrawals.

Methods

ATMPs with withdrawn EMA marketing authorizations were identified from the EMA website. A targeted review of relevant company press releases was undertaken and key information extracted. Health technology assessment (HTA) outcomes in France, Germany, and England were extracted from their respective websites (1 Sep 2023).

Results

Thirty-two ATMPs have received EMA marketing authorizations, with 22 percent (7/32) withdrawn. One of these seven withdrawals was driven by unfavorable clinical results versus six out of seven due to commercial reasons. Of the six withdrawals driven by commercial reasons, four were associated with negative HTA and/or reimbursement issues (Chondro-Celect, Glybera, and Zalmoxis’s Service Médical Rendu (SMR, actual medical benefit) insufficient, driven by data-related issues in France; Glybera’s challenge to obtain reimbursement by insurance funds with only one sale reported, and Zynteglo’s price agreement not reached in Germany). One of the six withdrawals (Chondro-Celect) was also associated with hospital exemption (continued production without marketing authorization). One of six (Provenge) was also associated with chemistry-, manufacturing-, and controls (CMC)-related issues.

Conclusions

The ATMP landscape has rapidly evolved in the past decade. While ATMPs offer the promise of long-term benefits, the unique manufacturing, clinical, and especially the reimbursement challenges they face can lead to commercial failure. To ensure ATMP patient access and commercial success, manufacturers should engage early with payers, understand potential reimbursement challenges, and proactively plan to mitigate these.
OP75 Pharmaceutical Innovativeness Index: Evaluation Of Pharmaceutical Innovation In Lung Cancer Treatment Drugs Approved Between 2011 And 2021
Authors: Freitas Isabela , Gargano Ludmila , André Ariane , de Assis Acurcio Francisco , Alvares-Teodoro Juliana , Guerra Augusto
Published: January 2025

Introduction

Lung cancer (LC) ranks second globally in neoplasia diagnoses and exhibits the highest oncological mortality rate. Oncology has seen a marked increase in U.S. Food and Drug Administration (FDA) approvals for novel drugs, notably in the case of LC. This study aims to assess the innovativeness of recently FDA-approved LC treatments compared to existing options, spanning the 2011 to 2021 period.

Methods

The innovativeness of drugs was assessed through the Pharmaceutical Innovativeness Index (PII). The methodology considers population health needs and aggregated clinical benefits, while weighing methodological quality and the adequacy of available evidence. New drugs are analyzed considering available therapeutic alternatives. The assessment assigns an innovativeness score to the drugs, with 1.0 representing the highest degree of innovation. PII was applied to all drugs with their first FDA indication for LC treatment. The evaluation considered specific clinical indications (e.g., treatment of ALK-positive LC), and data from FDA clinical review reports and pivotal clinical trials were considered.

Results

Eighteen new drugs were identified during the period. The drugs were evaluated with a mean PII of 0.615, ranging from 0.474 to 0.811. No discernible trends in innovativeness were observed within specific indications. Overall, the evaluations indicated that these medications were approved to address significant therapeutic needs. However, the added therapeutic value ranged from absent to moderate. Most supporting evidence for these approvals was derived from clinical studies with low risk of bias. Nevertheless, these studies often featured inadequate designs due to the absence of a comparator arm, limiting the assessment of added therapeutic value.

Conclusions

Pharmaceutical innovations in recent years for LC treatment have demonstrated limited clinical benefits when compared to existing alternatives, highlighting a substantial therapeutic need. The application of the PII can assist in healthcare decision-making, guide investments and research efforts, as well as inform pricing and reimbursement of new technologies, thereby contributing to the sustainability of healthcare systems.
OD05 Frameworks For Synthesizing Qualitative Evidence In Health Technology Assessment: A Scoping Review
Authors: de Almeida Cardoso Marilia Mastrocolla , Marques Raphael Thomaz , Machado-Rugolo Juliana , Thabane Lehana , Püschel Vilanice , Theresa Weber Silke Anna , Duque Graciela Paula , Almeida Rosimary Terezinha , Rodrigue Clarice , Guimarães Drumond Sybelle Luzia , De Paula Cristiane , Lopes Luciane , Gabriel Mariana , Vanston Meredith
Published: January 2025

Introduction

Health technology assessment (HTA) agencies and researchers recognize the necessity of evidence-based methodologies beyond quantitative data to assess feasibility, appropriateness, meaningfulness, patient values, preferences, acceptability, and equity. Despite existing guidelines for synthesizing qualitative data, the HTA framework requires clarification. This review aims to describe the frameworks, tools, and processes used to synthesize qualitative evidence and assess the quality of HTA.

Methods

Using the JBI methodology, the authors accessed databases such as MEDLINE, LILACS, CINAHL, Embase, Web of Science, Scopus, PsycINFO, Cochrane Library, JBI Database, and ScienceDirect. Grey literature searches included ProQuest, OpenGrey, CADTH’s Grey Matters, Google Scholar, and HTA agencies’ websites. Inclusion criteria focused on synthesizing qualitative evidence frameworks, methods for evidence synthesis, and quality rating. The review had a global scope, without specific population and time restrictions. Data, encompassing fundamental concepts, frameworks, methods, subjects, and objectives, were presented in tables and figures.

Results

Out of 2,054 articles, 31 were included, mainly from Europe, with a predominant “guide” authored by an HTA agency and university. The majority of documents did not originate from agencies. Only three agencies developed specific documents. A surge in publications occurred in 2018/2019. Qualitative data in HTA were justified for opinions, acceptability, feasibility, and equity. SPICE was the most cited acronym; RETREAT was the preferred framework. Thematic synthesis was the most cited method, CASP for quality assessment. GRADE-CERQual graded evidence quality, and ENTREQ was cited for reporting qualitative research. The GRADE EtD framework was the sole tool mentioned for recommendations.

Conclusions

This review highlights a growing trend in including qualitative evidence in HTA. While various proposals suggest instruments and methods, few documents cover all necessary steps, resulting in diverse recommendations. Standardizing processes can improve decision-making by guiding the integration of qualitative evidence, potentially enhancing recommendation quality. This ensures evidence on feasibility, appropriateness, significance, patient values, preferences, acceptability, and equity are considered.
OD07 Disaggregation Of The Costs Of Pharmaceutical Research And Development
Authors: Fabian Daniel , Wild Claudia
Published: January 2025

Introduction

Costs for pharmaceutical products are increasing. Pharmaceutical companies claim that high research and development (R&D) costs are the reason for the steep price increase of new products. However, there exists little data to support such claims; there is a lack of transparency in R&D cost reporting. This research intends to analyze and to disaggregate the costs of pharmaceutical R&D.

Methods

Studies on the costs of introducing new medications to the market can differ substantially in their methodology, their origin of data, and their results. A scoping review was conducted on costs of R&D for pharmaceutical products using Embase, PubMed, and EconLit, using a combination of the terms “drug research and development” and “costs” or “drug research and development” and “expenditure.” Additionally, semi structured interviews with 16 experts from non-governmental organizations (NGOs), pharmaceutical companies, academic researchers, and not-for-profit pharmaceutical companies were conducted to identify the main driving factors for rising costs of new drugs.

Results

Out of 24 studies that analyzed mixed therapeutic fields, the five highest cost estimates had affiliations with industry or received funding from pharmaceutical companies. Non-affiliated researchers are unable to reproduce studies that use confidential data and therefore cannot check the validity of the results. Additionally, different definitions for R&D make analyzing costs challenging. The interviewees emphasized that driving factors influencing costs for pharmaceutical R&D are therapeutic indication, drug complexity, number of patients in clinical trials, length of the development process, and attrition rates.

Conclusions

Due to the diverse nature of drug development and the confidential information held by pharmaceutical companies, it is a challenge to provide an exact assessment of the average costs of pharmaceutical R&D. Transparency policies with well-defined definitions for R&D are necessary to level the information asymmetries between the private and the public at price negotiations.
OD17 Future Health And Economic Burden Of Cardiovascular Disease In Type 2 Diabetes In Australia From 2021 To 2031
Authors: Abushanab Dina , Marquina Clara , Morton Jedidiah I , Al-Badriyeh Daoud , Lloyd Melanie , Magliano Dianna J , Liew Danny , Ademi Zanfina
Published: January 2025

Introduction

Diabetes is a key risk factor for cardiovascular disease (CVD), with a two-to-four-times higher risk than that in non-diabetes. The health burden associated with CVD imposes a major economic concern on healthcare systems and on society. We sought to estimate the future burden of CVD in terms of health and economic outcomes in type 2 diabetes (T2D) from 2022 to 2031.

Methods

From Australian public healthcare and societal perspectives over the next decade, two dynamic models with annual cycles were designed to predict myocardial infarction (MI) and stroke in patients aged 40 to 90 years. First events at risk of CVD people were estimated using the 2013 pooled cohort equation, while recurrent events in existing CVD were acquired from the global Reduction of Atherothrombosis for Continued Health registry. Costs and utilities were obtained using public sources. Outcomes were fatal and non-fatal MI and stroke, years of life lived, quality-adjusted life years (QALYs), total direct and indirect costs. An annual discount rate of 5 percent was used.

Results

Over the next 10 years, the model projected a total of 83,618 non-fatal MIs (95% confidence interval [CI]: 83,170, 84,053) and 58,774 non-fatal strokes (95% CI: 58,458, 59,013). In terms of health outcomes, total years of life lived and QALYs were 9,549,487 (95% CI: 9,416,423, 9,654,043) and 6,632,897 (95% CI: 5,065,606, 7,591,679), respectively. In terms of economic outcomes, total direct and indirect were AUD9.59 billion (USD6.38 billion) (95% CI: AUD1.90 billion [USD1.26 billion], AUD30.45 billion [USD20.26 billion]) and AUD9.07 billion (USD6.03 billion) (95% CI: AUD663.53 million [USD441.61 million], AUD33.19 billion [USD21.96 billion]), respectively. The chronic costs of CVD contributed most of the total direct cost, while morbidity contributed most of the total indirect costs.

Conclusions

Our study shows that from 2022 to 2031, CVD as a complication from T2D will cause a significant impact on the Australian healthcare system and society. These estimates can be used to explore different strategies to optimize the control of risk factors for the prevention and management of CVD in T2D in Australia.
OD22 Innovating Patient Involvement In Health Technology Assessments To Enable More Sustainable Involvement From Patient Stakeholders
Authors: Livingstone Heidi , Fitzpatrick Ella , Tonkinson Mandy , Crosbie Helen , Rasburn Mark , Taylor Sally , Wigmore Janine
Published: January 2025

Introduction

The National Institute for Health and Care Excellence (NICE) heard from small organizations how resource intensive and difficult it is for them participate in medicines health technology assessments (HTA) since the COVID-19 pandemic. To provide additional support for these organizations or to provide alternative patient input, NICE explored implementing surveys directly with patients to share with patient stakeholder organizations and NICE’s HTA medicines committees.

Methods

Patient organizations and colleagues at NICE were included in the background investigation. Informal interviews were conducted with the HTA bodies in Wales and health technology colleagues in NICE about their experience of this method of patient input.

Two approaches were piloted:

(i) Developing the online questionnaire using the Summary of Information for Patients.

(ii) Developing a jointly branded questionnaire collaboratively with the patient organization and implementing a data-sharing agreement to share the raw data.

The survey was distributed by the patient organization, analyzed by NICE, and shared with the patient organization to inform their submission to NICE.

Results

The results of the background investigation showed that the option to include this additional method of input could provide valuable support for patient organizations and has the potential to increase the amount and quality of patient input to an HTA committee.

Both pilots were successful in:

•

Supporting patient organizations’ input into a medicines HTA

•

Reducing the resources required from patient organizations.

The second pilot added more value due to:

•

Collaboration, relationship, and building trust

•

Joint development of the survey

•

Data sharing and potential to add to patient evidence about a disease and treatments.

Conclusions

Surveys conducted directly with patients can help patient organizations participate in medicines HTAs, but they are only one element of developing more innovative and sustainable patient involvement in the process. HTA bodies need to innovate and work collaboratively with patient stakeholders to produce a menu of options for involvement so that it can be tailored to stakeholders’ resources.
OD26 Comparing Health Technology Developers’ Proposed Indication To An Estimated Indication Generated By An International Horizon Scanning Database
Authors: Harte Marie , Choitir Caitríona Ní , Eames Heather , Adams Roisin , McCullagh Laura , Tilson Lesley , Duell Daisy , Odnoletkova Irina , Christensen Anna Bergkvist , Persson Marie , Bräuner Helle , Guardian Marcus , Tuckova Alzbeta Alzbeta , Wilkinson Brian , Kuzmak Beth , Shaw John , Boyce Craig , Palakollu Prashanth , Erinoff Eileen
Published: January 2025

Introduction

Detail on a technology’s projected therapeutic use is required for horizon scanning. The International Horizon Scanning Initiative (IHSI) database will utilize natural language processing (NLP) augmented by human curation to generate an estimated indication for technologies in development. We compared the estimated indication, generated as a test-set for NLP, with health technology developers’ (HTDs) proposed indications identified from Ireland’s horizon scanning system (HSS).

Methods

Eight oncology technologies common to both Ireland’s HSS and the IHSI database were analyzed. The analysis included unlicensed technologies in late-stage development that have not submitted a European marketing authorization application. Ireland’s HSS receives data on proposed indications for technologies from HTDs. IHSI database curators extract and convert terms from clinical trials into structured inputs (condition, combination therapy, stage of disease, place in treatment, patient/disease-specific subgroups) to produce an estimated indication for a technology. We sought to identify, by structured input, the degree of alignment between HTDs’ proposed indications with the IHSI database’s estimated indication.

Results

There was 100 percent alignment between the HTD’s proposed indication and the estimated indication generated in the IHSI database for five of the eight included technology records. There was 83 percent alignment for two records and 67 percent alignment for one record. Across all records there was full alignment on condition, combination therapy details, patient-specific subgroup, disease-specific subgroup, and place in treatment. Stage of disease was the only element where data was either not generated for the IHSI database’s estimated indication, not aligned with the HTD’s proposed indication, or reported in an incorrect field.

Conclusions

There is a high degree of alignment between an HTD-proposed indication and the IHSI-estimated indication. The processes for generating an estimated indication will involve both NLP-generation and human co-curation. The current (curator-selected) elements are being used to train the NLP engine. Thereafter, the engine will process clinical trial data to surface tags for human selection to generate the structured inputs.
OD27 Estimating The Causal Effect Of Adaptive Treatment Strategies Using Longitudinal Observational Data
Authors: Zhang Yingying , Kreif Noemi , Bennett Alastair , Manca Andrea
Published: January 2025

Introduction

Real-world data can help inform policymaking in health care by facilitating the evaluation of realistic treatment protocols. To generate robust evidence, analysts must address time-dependent confounders—variables influenced by past treatment decisions and affecting future treatment. Double-robust methods can help in eliminating bias by modeling both the treatment and the outcome mechanisms, using machine learning to improve model specification.

Methods

Longitudinal targeted minimum loss-based estimation (LTMLE) is a double-robust method that handles time-varying confounding, currently with only a few applications on real-world data. We demonstrate the use of LTMLE to evaluate realistic treatment protocols by applying it on longitudinal registry data to compare various treatment protocols that involve the use of erythropoiesis-stimulating agents (ESA) for myelodysplastic syndromes patients. We define dynamic regimes that trigger initiating ESA when relevant criteria (e.g., low hemoglobin levels) are met and require continuing/stopping ESA based on the response to treatment. We estimate the effect of these protocols on survival and EuroQol 5-dimension questionnaire (EQ-5D) scores.

Results

We study static treatment regimes where we compare patients always on treatment with patients always not on treatment, and we find the average effects of always administering ESA versus never administering it are positive but not significant on patients’ EQ-5D scores or on survival probabilities across all treatment time periods. We also study dynamic treatment regimes where decisions to initiate and continue/discontinue treatment over time depend on changing patient characteristics and responses to treatment. We find that patients following dynamic treatment regimes are predicted to score higher in EQ-5D and have longer survival probabilities than patients under static treatment regimes.

Conclusions

The paper provides a tutorial and case study demonstration of the LTMLE model that can evaluate realistic treatment protocols using longitudinal observational data. It accounts for time-varying confounding in estimating treatment effects and can incorporate machine learning in improving accuracy of outcome prediction. The model has been applied in the setting of long follow-up times and gradually reduced sample size.
OD38 Real-World Analysis Of First-Line Maintenance Treatment For Patients With Nonsquamous Advanced/Metastatic Non-Small-Cell Lung Cancer
Authors: Tapan Umit , Bell Kelly F. , Aziez Amine , Sun Jiaqian , Du Mandy , Du Ella Xiaoyan , Hua Qi , Yang Hongbo , Shah Manasee
Published: January 2025

Introduction

Pemetrexed and immunotherapies (e.g., pembrolizumab) are approved for first-line maintenance (1LM) treatment of nonsquamous advanced/metastatic non-small-cell lung cancer (NSCLC), but real-world data on their use are limited. The objective of this study was to assess 1LM clinical outcomes, safety, and treatment patterns of immunotherapy versus immunotherapy+pemetrexed among patients with advanced/metastatic NSCLC from the EU4 (France, Germany, Italy, Spain)+UK.

Methods

Data from patients in the US, Canada, and EU4+UK with nonsquamous advanced/metastatic NSCLC without targetable mutations were collected via electronic case report form. Physician-identified patients (≥18 y) in the EU4+UK were eligible for this subgroup analysis if they achieved stable disease or complete or partial response with first-line platinum-based chemotherapy+immunotherapy (January 2019 to March 2021) and received 1LM immunotherapy or immunotherapy+pemetrexed. Patients were followed from index (1LM initiation) until last physician contact or death. Outcomes were overall survival (OS), progression-free survival (PFS), treatment patterns and duration, and adverse events.

Results

Among the selected 367 patients (male, 71.9%; mean±StDev age, 63.4±7.2 y; current/former smokers, 85.8%), 203 (55.3%) received immunotherapies, most commonly pembrolizumab (n=173; 85.2%), and 164 (44.7%) received immunotherapy+pemetrexed. Patients receiving immunotherapy had longer median adjusted OS and PFS compared to those receiving immunotherapy+pemetrexed (OS hazard ratio [HR]: 0.63; 95% confidence interval [CI]: 0.36, 0.90; PFS HR: 0.58; 95% CI: 0.38, 0.79). Patients receiving immunotherapy versus patients receiving immunotherapy+pemetrexed had longer median treatment duration (14.0 vs 10.3 mo; p<0.001) and were less likely to experience anemia (19.7% vs 33.5%; p<0.01). Results were similar in the overall study population.

Conclusions

In this real-world study, among the selected patients with nonsquamous advanced/metastatic NSCLC who achieved stable disease or complete or partial response with first-line therapy, the addition of pemetrexed to immunotherapy in 1LM did not appear to confer a clinical benefit. Identifying treatments that can improve clinical outcomes for these patients remains an area of unmet need.
OD43 The Growing Clinical And Economical Burden Of Chronic Kidney Disease In Brazil: An IMPACT CKD Analysis
Authors: Moreira Ricardo , Regis Cassio , Grizolli Beatriz , Montenegro Cinthia , Gobbi Talita , Rao Naveen , Priest Stacey , Brown Stephen , Guiang Hannah , Moura Ana Flavia , Zhao Ming-Hui , Chadban Steven
Published: January 2025

Introduction

The growing burden of chronic kidney disease (CKD) in Brazil is increasingly evident, marked by its significant contributions to mortality rates and healthcare costs. Managing CKD, especially through renal replacement therapy (RRT), demands substantial resources. To enhance healthcare decision-making, a thorough examination of the relationship between the rising prevalence of CKD and its clinical and economic impacts is crucial.

Methods

We developed a patient-level simulation model to project the natural history of CKD, defined as the IMPACT CKD. This model integrated factors such as acute kidney injury, cardiovascular events, and comorbidities, and aimed to assess CKD’s clinical, humanistic, and economic impact on the healthcare system. It forecasted the burden of CKD over the next decade (2023 to 2032). This projection is pivotal to derive the burden of CKD for health technology assessment (HTA) evaluations. Validation was conducted against Brazil’s demographic data and cross-validated with the Inside CKD model.

Results

The IMPACT CKD forecast a rapid increase of CKD population in Brazil, outpacing the growth of the general population. Specifically, there is an expected 6.9 percent increase in stages 3 to 5 CKD, leading to a higher demand for dialysis (projected 370,000 cases in 2032) and transplants (projected 115,000 cases in 2032). A significant increase in cardiovascular CKD-related events (+100.6%) and mortality (+67.8%) is expected. In 2032, it is projected 15 million CKD patients will be in stages 1 to 2, and 12.7 million in stages 3 to 5. CKD-related healthcare costs will represent 25.7 percent of Brazil’s healthcare budget, and dialysis will reach USD2.7 billion in annual costs.

Conclusions

IMPACT CKD predicts an increasing CKD prevalence and an alarming rise in stages 3 to 5 and RRT, including thousands of premature deaths, and a substantial economic burden on the Brazilian healthcare system. This data could be informative for healthcare decision-makers when choosing strategy to reduce the impact of CKD in Brazil.
PP01 Early Health Technology Assessments Of Health And Well-Being Returns On Investment In The Biobanks
Authors: Marquina Clara , Lloyd Melanie , Ng Wayne , Hess Jonas , Evans Sue , Ademi Zanfina
Published: January 2025

Introduction

Human tissue biobanks provide vital infrastructure to support both basic science and clinical research, but their economic value in terms of attributable population health gains is unclear. We evaluated the population health returns from investment in the Victorian Cancer Biobank (VCB). The VCB comprises five hospital-integrated sample repositories and a central lead agency located in Melbourne, Australia.

Methods

This evaluation assigned monetary values to the health gains attributable to VCB-supported public-funded research. These were then compared to the total investment in VCB infrastructure since inception (2006 to 2022) to determine the return on investment (ROI). A time lag of 40 years was incorporated, recognizing the delay from investment to impact in scientific research. Health gains were therefore measured for the years 2046 to 2066, with a three percent discount rate applied. Health gains were measured in terms of disability-adjusted life years (DALYs) attributable to VCB-associated research, with monetary cost assigned via the standardized value of a statistical life year (AUD227,000 [USD149,883]).

Results

The age-standardized DALY rate attributable to cancer was modeled for two standpoints: (i) extrapolating the current decreasing trajectory and (ii) assuming nil future improvement from current rates, with 33 percent of the difference attributed to scientific innovation. The proportion of the aggregate health gain attributable to VCB-supported research was estimated from the number of VCB-credited scientific publications as a proportion of total oncology publications over the same period. The AUD32,628,016 [USD21,554,571] of public funding invested in VCB activities over the years 2006 to 2022 generated AUD84,561,373 [USD55,868,539] total savings. Return on investment was AUD1.59 [USD1.05] for each AUD1 [USD0.66] invested.

Conclusions

The VCB offers a strong return on investment in terms of population health impacts, justifying the use of public funds and supporting the use of biobanks to advance scientific research. Future health technology assessments could capture the total impact of research on the role of the biobanks attributed to research outputs.
PP02 The Application Of Care Pathway Analysis And Economic Modeling In Early Health Technology Assessment: Learnings From Two Projects
Authors: Gregg Emily , Butler Karin , McCool Rachael , Graziadio Sara
Published: January 2025

Introduction

Early health technology assessment (eHTA) can help to explore the potential value of a technology in the early stages of development. Care pathway analysis (CPA) is a method to identify and map clinical decisions in the current and new care pathways (including the new intervention). This work provides examples of applying CPA within the context of eHTA for medical interventions.

Methods

CPA usually involves a pragmatic review to identify and synthesize national/international guidelines that describe the care pathway for the condition of interest. This is typically followed by a qualitative evaluation that can include semistructured interviews with thematic analysis. Interviews with experts are undertaken to understand where (and why) real-world practices differ from published guidance and to validate the care pathway. They also help to evaluate the strengths/weaknesses of the new technology, potential population and role in the pathway, and barriers/facilitators to adoption. The CPA forms the basis of economic modeling that helps assess the monetary value of the new technology.

Results

The application of CPA from two recent projects will be presented: an innovative diagnostic test for respiratory tract infections and a medical device for treating cataracts. Additionally, the value of CPA in eHTA will be described from the technology developers’ perspective. In both projects, CPA was used to inform the potential value propositions of the new technology and its positioning in the care pathway. It also helped to optimize the structure of the early economic model and to identify evidence generation needs. The early model identified the pathway that was more likely to be cost effective in the future.

Conclusions

CPA is a valuable method within the context of eHTA. Alongside identifying the potential role and positioning of the new technology, test developers found the assessment useful for informing internal strategy decisions and discussions with potential external investors. The developers were able to demonstrate the clinical perspective around the value of the test, elicited through an independent and rigorous methodology.
PP10 Addressing The Challenge Of Environmental Sustainability Between Management And Health Technology Assessment At The Hospital Level
Authors: Bobini Michela , Di Bidino Rossella , Lipska Iga , Cicchetti Americo
Published: January 2025

Introduction

Several organizations specializing in health technology assessment (HTA), at various levels, are exploring avenues to integrate the dimension of environmental sustainability (ES) within their assessments. However, evidence remains scant on how best to do this. Across the different levels, hospital-based HTA (HB-HTA) also needs to comply with the leadership and strategies of the hospital and adapt to the existing resources and established partnerships.

Methods

The purpose of this study is threefold: to provide an overview of the progress achieved by hospitals with respect to the integration of ES into HB-HTA; to detect consistencies between HB-HTA activities and hospital ES strategies; and to explore possible approaches and methods for integrating ES into HB-HTA. The methodology is structured in two steps: (i) a literature review on HB-HTA and environmental impact/sustainability to identify emerging approaches and methods for integrating environmental sustainability into HB-HTA; (ii) data collection by means of a questionnaire submitted to hospitals/organizations based on their membership of Health Technology Assessment International.

Results

Preliminary results highlight a widespread awareness of the importance of integrating ES issues into HTA at the different levels at which it is performed. The incorporation of ES issues into HB-HTA is still in its infancy. There are some factors hindering the development of methods, such as the absence of scientific evidence on emerging approaches and the scarce availability of data or difficulty in tracing data back to a specific technology.

Conclusions

The integration of the dimension of ES into HB-HTA still appears to be very immature. Coordination between the different actors in the system seems necessary to overcome the obstacles identified.
PP11 Evaluating Environmental Sustainability In Health Technology Assessment: A Multisectoral Systematic Review
Authors: Pegg Melissa , Bouttell Janet , Taylor Matthew
Published: January 2025

Introduction

Research widely reports healthcare technologies contribute substantial environmental impacts. Timely health technology assessment (HTA) environmental sustainability (ES) framework development is critical. A lack of multisectoral expertise, resource constraints, and consensus to assess environmental impact is delaying methodological progression. The study objective was to identify and critically evaluate methods supporting ES in HTA and formulate recommendations to underpin the development of an ES framework in HTA.

Methods

This multisectoral systematic review followed PRISMA guidelines. Maximizing the opportunity to evaluate applied environmental assessment methods, the databases Web of Science, Embase, PubMed, IEEE Xplore, EBSCOhost GreenFILE, Cochrane Library, and International Network of Agencies for Health Technology Assessment (INAHTA) were searched between 2008 and 2023. Full-text published studies applying both qualitative and quantitative methods to evaluate technology ES were included. Frameworks were critiqued for their comprehensiveness based on sustainability scope and in challenging barriers to decision-making. Studies were ranked according to how transparent and feasible frameworks were in their ability to assess technology ES and alignment with HTA principles.

Results

A total of 10 studies were identified and ranked in order of suitability in assessing technology ES in HTA. All studies applied a combination of methods to overcome issues such as data and resource constraints, expert knowledge, consensus in decision-making, and multiple criteria trade-off. Ranked highest, the One Health “extended” life cycle assessment (LCA) framework that utilized SimaPro LCA advanced software addressed the greatest methodological needs for HTA. Ranked second, the circular economy (CE) framework used in conjunction with an analytical hierarchy process (AHP) makes use of weighting and expert consultation to support multiple-criteria decision-making (MCDM).

Conclusions

This is the first systematic review to identify multidisciplinary frameworks, supporting evaluation of ES and decision-making in HTA. Providing valuable insight into potential methodological solutions where there is limited research, this study facilitates ES policy development in HTA. This study challenges limitations to methodological development highlighted by previous research. Further research should apply these recommendations in an HTA setting.
PP20 Health Technology Assessment To Sustain The Health Insurance Scheme: A Case Study On Anemia Treatment In Ghana
Authors: Amankwah Ivy , Heupink Lieke , Mensah Annan Ruby Aileen , Asare Brian , Owusu Richmond , Abassah-Konadu Emmanuella , Asante Hannah , Chola Lumbwe , Frønsdal Katrine
Published: January 2025

Introduction

In Ghana, the reimbursement of iron polymaltose complex (IPC) for iron-deficiency anemia treatment raises concerns as a potential cost driver for the National Health Insurance Scheme (NHIS). Prioritizing value for money is crucial for NHIS sustainability. With Ghana’s established health technology assessment (HTA) framework, we provide insights and preliminary findings in this case study on anemia treatment.

Methods

To further support institutionalization, we follow the Ghana HTA process guidelines and Ghana HTA reference case. Simultaneously, to build wider capacity for HTA in Ghana, relatively new trained staff was engaged while anchoring the team at the HTA Secretariat from the Ministry of Health. We started a situational analysis, which includes desktop review and key-stakeholder interviews. An umbrella review was commenced simultaneously to identify systematic reviews assessing clinical effectiveness of IPC compared to other medicines on the essential medicine lists for all population groups. We will assess cost-effectiveness to ultimately inform coverage decisions and possible implications for organizational structures.

Results

Anemia is a persistent public health issue in Ghana, impacting children under five, young women, and expectant mothers. The essential medicine list includes various treatments, but ambiguity exists in the standard treatment guidelines regarding when to administer IPC versus alternative medicines. Under the NHIS, the intravenous formulation of IPC stands out as the most expensive treatment. A systematic review identified three papers, focusing on infants, children, adults, and pregnant women. Preliminary findings suggest weak overall evidence supporting IPC’s superiority, although adults may exhibit higher tolerance for IPC compared to alternative treatments.

Conclusions

Conducting an HTA on anemia treatments, despite the low treatment costs, is still significant as technical efficiencies can be achieved with high-volume drugs by analyzing prescription patterns and generating evidence to support potential changes. Findings from this HTA can be used elsewhere, especially in resource-constrained settings. Next steps involve finalizing the HTA and disseminating results to stakeholders.
PP21 Examining Clinical Evidence: Incorporation Of Medications For Ultra-Rare Diseases – Descriptive Analysis Of Conitec Reports (2012 to 2022) – Brazil
Authors: Machado-Rugolo Juliana , da Silva Pereira Curado Daniel , Komoda Denis Satoshi , Modelli de Andrade Luis Gustavo , de Paula De Sordi Mônica Aparecida , Fonseca Alan Francisco , Coneglian de Almeida Juliana Tereza , Lima Silvana Molina , Theresa Weber Silke Anna , Santos Marisa , Tarride Jean-Eric , Thabane Lehana , de Almeida Cardoso Marilia Mastrocolla
Published: January 2025

Introduction

The decision-making process for health technology assessment (HTA) in ultra-rare diseases faces a significant challenge for agencies worldwide. This study sought to offer an analytical overview of the clinical evidence outlined in the recommendations of the Brazilian National Committee for Health Technology Incorporation (Conitec) in ultra-rare diseases.

Methods

Data were extracted from recommendation reports for the ultra-rare diseases evaluated between 2012 and 2022. To classify a disease as ultra-rare, the epidemiological criterion or a consultation with the Orphanet platform was used (prevalence of ≤1/50,000 inhabitants). The extracted variables included the type of evidence synthesis, type of studies, instrument, the result of the assessment of the methodological quality of the studies, the format of evidence synthesis presentation, whether the evidence was graded, and the result.

Results

Among 53 analyzed reports, 70 percent relied on randomized controlled trials, followed by systematic reviews (SR), and observational studies. Reports with positive recommendations based on SR comprised 63 percent. GRADE applied to 27 reports and indicated low or very low results for the first two outcomes (62% and 65%). No clear link between evidence quality and final recommendations was observed. Meta-analysis-based reports had 83 percent positive recommendation rate, compared to 55 percent without meta-analysis. Surrogate outcomes were predominant. Clinical characteristics significantly influenced final decisions, especially when new data emerged in public consultation or had the potential to alter disease progression, reduce severe events, or enhance survival.

Conclusions

Ultra-rare diseases pose challenges in evidence quantity and quality. Traditional HTA frameworks seem inadequate, lacking robust evidence for these conditions. The difficulties in ultra-rare disease HTA underscore the need for specialized frameworks. This analysis acknowledges limitations, notably the heterogeneity in older report structures compared to recent ones, reflecting evolving HTA methodologies in Brazil.
PP24 Creating A Comprehensive Open-Access Health Technology Assessment (HTA) Policy Research Database Through Automated Data Extraction From HTA Reports
Authors: Versteeg Jan-Willem , Bloem Lourens , Leopold Christine , De Bruin Marieke , Mantel-Teeuwisse Aukje , Goettsch Wim
Published: January 2025

Introduction

Findable, structured, and understandable data from health technology assessment (HTA) reports is the core of HTA policy research. Available databases with this information, such as the International Network of Agencies for Health Technology Assessment (INAHTA) database, may be incomplete and their common manual data collection is time-consuming. Automated data extraction may offer a solution by creating a standardized, real-time-updating, comprehensive, open-access HTA database.

Methods

In this research, we explore the possibilities of automated data extraction in the context of creating a standardized and comprehensive HTA policy research database. Data points were extracted from publicly available guidance reports of the National Institute for Health and Care Excellence (NICE) using different text extraction techniques such as natural language processing (NLP) and generative pre-trained transformers (GPTs). Future efforts are aiming to expand the database to other HTA bodies and link it to the European Medicines Regulatory Database (EMRD) that is also being developed.

Results

Preliminary results of our research show that it is possible to use existing text extraction techniques to extract relevant information from publicly available HTA recommendations. Scaling the system to include more HTA bodies and data points is challenging as extraction based on document structure is complicated by heterogeneity in document structure within HTA bodies and between HTA bodies. Future results will focus on finding the best data extraction approach for each data point and on validating the system.

Conclusions

Using automated data extraction to extract data from HTA reports can be a viable option for creating a comprehensive database that can be used to enhance comparative HTA policy research. Challenges remain in scaling the system to include more HTA bodies and data points. Results regarding best-performing extraction techniques and data validation of the system are expected soon.