bluuebunny/crossref_metadata_2025 huggingface dataset:
OP22 Productivity Benefits Of Modifying Cardiovascular Disease Risk In Type 2 Diabetes In Australia: A 10-Year Prediction Dynamic Model
Authors: Abushanab Dina , Al-Badriyeh Daoud , Marquina Clara , Morton Jedidiah I , Lloyd Melanie , Zomer Ella , Talic Stella , Liew Danny , Ademi Zanfina
Published: January 2025

Introduction

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

Methods

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

Results

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

Conclusions

The impact of CVD as a complication from T2D on work productivity is significant. Screening and prevention strategies tailored early in life are likely to exert a positive impact on health and work productivity.
OP23 Key Challenges For The Appraisal Of Disease-Modifying Dementia Drugs
Authors: Joshi Roshni , Dawoud Dalia , Salih Fatima , Avsar Tuba Saygin , Longson Carole , Crabb Nick
Published: January 2025

Introduction

Disease-modifying dementia treatments (DMDTs) target amyloid beta or tau proteins and have the potential to change disease progression, representing a step change in the management and treatment of Alzheimer’s disease. Given the novel mechanism of action and impact on health care, the NICE Health Technology Assessment Innovation Laboratory (HTA Lab) sought to identify and contextualize the key issues for future appraisals.

Methods

We reviewed published assessment reports of DMDTs from international HTA agencies and conducted a scoping review of published economic models of pharmacological treatments to understand the challenges associated with evaluating the cost-effectiveness of dementia treatments. The HTA Lab held an engagement workshop with 27 external stakeholders, including expert clinicians, implementation partners, health economists, and representatives from international agencies to discuss and confirm the key issues and considerations likely to emerge during an appraisal of DMDTs.

Results

Key clinical and cost-effectiveness issues were identified and discussed. We concluded that consideration needs to be given to the diagnostic methods to identify the DMDT-eligible population in the UK, the validity of the surrogate outcomes used in the DMDT clinical trials, treatment effectiveness in different populations, and the incidence of DMDT-associated adverse events. Economic considerations include the type of economic model used in the appraisal, modeling the natural history of the disease, paucity of quality-of-life data in the treatment population, the inclusion of societal impact, treatment duration, stopping rules, and long-term effectiveness beyond the clinical trials.

Conclusions

DMDTs could have the potential to transform Alzheimer’s disease care. With multiple treatments on the horizon, the appropriateness and acceptability of the new mechanism of action underpinning these treatments should be considered. We have identified areas of uncertainty that are likely to arise during an appraisal process, to facilitate the timely approval of these medicines and patient access.
OP33 Advancing Patient Experience Data Implementation In Reimbursement Decision-Making: Insights On Challenges And Opportunities From Multistakeholder Interviews
Authors: Vanneste Alice , Wens Io , Adriaenssens Tom , Janssens Rosanne , Barbier Liese , Huys Isabelle
Published: January 2025

Introduction

Patient experience data (PED), encompassing patient preferences (PP), patient-reported outcomes (PROs), and patient input, play a pivotal role in understanding patient needs and informing healthcare decision-making, including reimbursement decisions. This study aimed to assess the current barriers hindering the integration of PED into practice and its particular challenges, opportunities, and concrete policy actions for the systematic implementation of PED.

Methods

Semistructured interviews (n=38) were conducted with industry (n=12), non-profit organizations and academia (n=4), regulatory authorities (n=6), health technology assessment (HTA) bodies and reimbursement agencies (n=6), and patient organizations (n=10) in Europe. A thematic analysis was conducted to explore stakeholders’ perspectives and to gain a comprehensive understanding of challenges and opportunities related to the systematic implementation of PED. Interview transcripts were analyzed using the thematic framework analysis to extract and elucidate the insights provided by the diverse stakeholders.

Results

HTA and reimbursement interviewees agreed on the value of including quality-of-life data, particularly when assessed using validated PRO measures. Despite acknowledging the potential of PP, there remained reluctance to integrate PP into reimbursement decision-making. Participants expressed divergent opinions regarding who should collect PED, with some regulators favoring industry, while HTA and reimbursement agencies emphasized transparency and independent PED collection. Limited experience in assessing PED also contributed to hesitancy, underscoring the need for more guidelines, especially at the national reimbursement level. Stakeholders endorsed collaboration through joint scientific consultations, expressing optimism about the impact of the Regulation (EU) 2021/2282 on health technology assessment (HTAR).

Conclusions

This study emphasizes the high potential of PED in informing reimbursement decision-making, fostering a more patient-centered approach. Stakeholder disparities highlight the complexity, necessitating more guidance, scientific robustness, transparency, and collaboration. In light of these stakeholder considerations, the upcoming HTAR holds promise to enhance the systematic implementation of PED, aligning healthcare decision-making with patients’ needs and preferences.
OP41 Pharmaceutical Technologies Conditionally Approved By The National Institute For Health And Care Excellence: A Critical Analysis
Authors: Perez Yankier Pijeira , Hughes Dyfrig
Published: January 2025

Introduction

There are concerns with the National Institute for Health and Care Excellence (NICE) conditional approval process with respect to the quality and reliability of the methods of evidence generation, analysis, and reporting; and on the increasing focus on OwR (only with research) in preference to OiR (only in research). Our study critically appraises the methods, quality, and risk of bias of the evidence generated in response to NICE conditionally approved technologies.

Methods

Our study reviewed pharmaceutical technologies appraised by NICE: technology appraisals approved between March 2000 and September 2022 and highly specialized technologies approved up to October 2023. From those, we identified appraisals with OiR and OwR conditional recommendations, and summarized the evidence requested by NICE as part of conditional approval. Then, evidence resubmitted to NICE for reappraisal was analyzed for its compliance with NICE’s initial recommendations for further research and assessed using the Cochrane Collaboration’s tools for risk of bias in randomized trials and the ROBINS-I tool for the non-randomized evidence.

Results

NICE made 54 conditional recommendations relating to technology appraisals (TAs) (13 OiR and 41 OwR) and five highly specialized technologies (HSTs). From those, 16 TAs presented additional evidence for reappraisal [nine OiR and seven OwR] and three HSTs [three OwR]. Two of the nine reappraised TAs with OiR recommendation and four of the seven OwR fully complied with NICE’s request for further evidence. All three HSTs complied in full. However, the majority of reappraised TAs and HSTs included evidence that was deemed to be at serious, high, moderate, or unclear risk of bias.

Conclusions

The quality of evidence presented to NICE following conditional approval varies considerably. There is often widespread noncompliance with requests for further research when conditionally approving a pharmaceutical technology. Complying with NICE’s requests, however, does not necessarily guarantee high-quality evidence when the technology is reappraised. Evidence generated in response to NICE conditional approval recommendations should be subject to quality standards.
OP44 Cost-Of-Illness And Cost–Consequence Analysis Of Dementia In Italy
Authors: Mennini Francesco Saverio , Bini Chiara , Sciattella Paolo , Vanacore Nicola
Published: January 2025

Introduction

In Italy, there is a lack of evidence regarding the care and management of patients with dementia, as well as the associated costs. This study aims to fill this informational gap by utilizing data from both literature and national surveys.

Methods

A prevalence-based cost-of-illness (COI) model was developed to assess dementia-related costs from a societal perspective. The resources utilization for management and treatment of patients with dementia was derived from both the literature and the analysis of surveys conducted by the National Institute of Health on the social-health structures dedicated to dementia. Indirect costs from informal caregiving were evaluated through a human capital approach. Additionally, a cost–consequence analysis (CCA) was conducted to assess the economic impact of healthcare resource utilization changes.

Results

Based on an estimated 1,150,691 dementia cases in Italy, with approximately 12 percent institutionalized, the COI model estimated an annual expenditure of around EUR23.6 billion (USD25.7 billion) for dementia patient management, with 63 percent attributed to out-of-pocket expenses. CCA indicated that if all affiliated with Centers for Cognitive Disorders and Dementia (CDCD) received nonpharmacological interventions (versus the surveyed 25.5 percent), there would be a direct cost increase of approximately EUR4.3 million (USD4.7 million).

Conclusions

This analysis provides an updated overview of current dementia patient management in Italy, offering valuable insights for decision-makers to prioritize health policies and interventions for patients and their caregivers.
OP46 A New Modular Approach To Updating National Institute For Health And Care Excellence Manuals
Authors: Walker Lizzie , Flight Laura , Prescott Carl , Shah Koonal
Published: January 2025

Introduction

Methods and processes for health technology assessment (HTA) must be sufficiently flexible to reflect emerging best practice and adapt to changes in the health and care landscape. They must also be clearly documented and subject to review, assessment, and consultation. This requires a framework to flexibly update HTA manuals, while providing stability and predictability for stakeholders.

Methods

The National Institute for Health and Care Excellence (NICE) is introducing a modular approach to updating its manuals. Previously, NICE completed full, but infrequent, updates to its manuals. A modular update is a review of the process and/or methods informing NICE’s guidance in a specific subject area, which can then be updated in the appropriate manual(s) if required. We are developing a modular updates framework that sets out NICE’s approach for identifying and prioritizing modular updates to review, reviewing the evidence and proposing changes to the manual(s), engaging and consulting with stakeholders, and implementing the modular updates.

Results

The proposed approach allows external stakeholders to contribute to topic identification and the content of updates, helping to ensure that the manuals meet the needs of users. The first modular update to the NICE manual for health technology evaluations was published in October 2023. This included updated processes for carrying out cost comparisons, streamlining committee decision-making and handling confidential information. Potential future modular updates may include updated methods to consider health inequalities.

Conclusions

The introduction of a modular approach will enable NICE to be more agile and responsive in monitoring, reviewing, and improving our methods and processes, making sure they remain cutting edge as the health and care landscape continues to evolve.
OP50 NICE Listens: Engaging The Public On How Environmental Sustainability Should Be Considered In Health Technology Assessment
Authors: Murray Alice , Shah Koonal
Published: January 2025

Introduction

Involving the public is essential to building trust in health technology assessment (HTA) organizations. The National Institute for Health and Care Excellence (NICE) runs a deliberative public engagement program, NICE Listens. It was used to explore informed public opinion on how environmental sustainability should be taken into account in HTA.

Methods

Twenty-three general public participants from across England took part in three iterative online workshops (each lasting two or three hours, held three weeks apart in 2022). The workshops included trade-off exercises, role-play, group discussion, and video clips from interviews with sustainable healthcare experts.

Results

Strong support was found for NICE taking action to make healthcare more environmentally sustainable. Support increased as participants learned that sustainable healthcare offers co-benefits, such as reduced burden on the National Health Service through better self-management of conditions. Participants did not want health outcomes to be compromised in pursuit of sustainability. We identified some circumstances where they found it acceptable to consider the environmental impact of interventions in decision-making: when effective treatments already exist; when the condition is not severe; when the alternative is equally cost effective; and when greener options are marginally higher in cost but as clinically effective as the alternative.

Conclusions

The findings demonstrate that environmental sustainability is clearly considered a relevant element of value. They also offer insight into how the environmental impact of health interventions should be considered in HTA. Further research should focus on methods for consistent measurement of the environmental impacts of health interventions and the incorporation of those impacts into decision-making.
OP52 How Will European Joint Clinical Assessment Impact National Decision-Making?
Authors: Müller Elvira , Neeser Kurt
Published: January 2025

Introduction

In 2025, oncology drugs with new active substances and advanced therapy medicinal products will undergo joint clinical assessment (JCA). The comparative analysis of the clinical evidence as defined in the Regulation (EU) 2021/2282 on health technology assessment (HTAR) will save national/regional submissions of the same evidence. JCA will be available early supporting appraisal and decision-making, which remains within the responsibility of member states (MS).

Methods

Targeted searches on JCA and statements from stakeholders were performed and analyzed. We conducted interviews with current and former national payers, as well as members of HTA agencies, across Germany, France, Italy, and Eastern Europe to explore their perspectives on the anticipated implications of JCA on decision-making processes and reimbursement strategies in Europe. Focus was on reduced/additional effort for authorities and health technology developers (HTDs), required national amendments, and potential discrepancies between JCA outcome and MS benefit evaluations.

Results

Stakeholders appreciate the standardized methodology and guidance on HTA, which, especially in countries without an established HTA system, could enhance patients’ access to new treatments by considering JCA in decision-making. The comprehensive evidence compilation may also save resources in pursuing national/regional submissions. On the other hand, country-based appraisals within the MS could lead to diverse conclusions, and there is uncertainty as to which extent national authorities will adopt JCA and how its integration into decision-making will be handled. Some stakeholders challenge an impact on local patients’ access as reimbursement and pricing processes remain within MS responsibility.

Conclusions

JCA is a long-desired achievement and will set the groundwork for timely access of new treatments in the MS. However, presently there are several uncertainties on how JCA will impact decision-making and whether MS appraisal could lead to contradictory value conclusions for a given treatment. Future adjustments to national/regional procedures and refinement of the JCA framework are expected.
OP62 Economic Evaluations Submitted For Reimbursement To The Brazilian Unified Health System: A Meta-Epidemiological Study
Authors: Falavigna Maicon , Dorneles Gilson , Araujo Cintia , Parahiba Suena , Stein Cinara
Published: January 2025

Introduction

Allocating financial resources efficiently poses a significant challenge for health systems worldwide. In this context, economic evaluations (EE) are pivotal for the health technology assessment (HTA) process, particularly in standardizing approaches to enhance decision-making accuracy. This study focuses on delineating methodological nuances of EE submissions within Brazil’s Unified Health System (SUS).

Methods

This meta-epidemiological study analyzed dossiers submitted to the National Committee for Health Technology Incorporation (Conitec) for drug reimbursement from January 2022 to May 2023. We selected dossiers that included complete EE studies such as cost–utility or cost-effectiveness analyses. Our evaluation involved extracting data on the characteristics of the studies, reimbursement decisions, elements of the base case scenario, and parameters used in utility and sensitivity analyses. [Protocol: DOI 10.17605/OSF.IO/JVYEC]

Results

We included 56 dossiers, leading to 11 favorable reimbursement decisions. EE study methods were cost-effectiveness (17) and cost–utility analyses (17), with some employing both (16). Markov chain models were used in 27 dossiers, primarily utilizing quality-adjusted life years as the health outcome measure. Additional outcomes included life years gained and frequency of avoided events. While utility assessment was reported in 33 dossiers, only six adjusted for age and seven accounted for disutility from adverse events. Thirty-two conducted deterministic or probabilistic sensitivity analyses, but only four presented parameters like credibility intervals, and 17 presented acceptability curves.

Conclusions

This study highlights the need for more standardized and refined methods in the EE submitted to Conitec. The existing EE methodological guidelines, dating from 2014, are currently under revision. This update is crucial to integrate recent advancements in health technology assessment and to better address contemporary requirements, particularly in light of the newly defined willingness-to-pay threshold.
OP64 Risk-Based Prioritization In Patients Referred For Transcatheter Aortic Valve Implantation: A Simulation Study
Authors: Miranda Rafael , Austin Peter , Naimark David , Wijeysundera Harindra
Published: January 2025

Introduction

Demand for transcatheter aortic valve implantation (TAVI) has increased in the last decade and has outpaced system capacity, impacting wait times and bringing undesirable health outcomes such as waitlist mortality and number of urgent procedures. Risk-based prioritization can improve equitable access to patients. In this study, we assess the impacts of different classifications and wait times for each risk group on health outcomes.

Methods

We developed decision-analytic models that simulate the patient trajectory from referral to completion of TAVI. Using prediction models that can classify patients based on their risk of adverse events on the waitlist, we assessed the impacts of (i) the number of risk groups, (ii) size of the risk groups, and (iii) recommended wait times for each risk group, on waitlist mortality, hospitalization, and the proportion of urgent TAVIs. All scenarios were modeled under the same resource constraints, allowing us to explore the trade-offs between faster access to prioritized patients and deferred access to nonprioritized groups.

Results

Increasing the number of risk groups from two to three, increasing the sizes of the higher-risk groups from five percent to 30 percent of the cohort each, and providing faster access to the higher-risk groups (five to three weeks for high-risk and 11 to five weeks for medium-risk) achieved the greatest reductions in mortality, hospitalizations, and urgent TAVIs (relative reductions of up to 29%, 23%, and 38%, respectively). However, this occurs at the expense of excessive wait times in the nonprioritized group (up to 25 weeks). The reduction in adverse events was lower when the nonprioritized group had more reasonable wait times.

Conclusions

When developing and implementing waitlist prioritization strategies, it is important to consider the resource constraints of the system and the patient profile, as the benefits of providing faster access to prioritized patients can lead to unreasonable wait times for nonprioritized ones. In settings with long wait times, prioritization initiatives must be followed by expansion of supply to achieve optimal improvements in health outcomes.
OP70 Implementation Of An Online Consultation Hub To Facilitate Consumer Engagement In Health Technology Assessment Processes
Authors: Trowman Rebecca , Watson Jo , Beach-Mills Bella
Published: January 2025

Introduction

In 2021, the Australian Government Department of Health and Aged Care’s Consumer Evidence and Engagement Unit (CEEU) launched an online consultation hub to provide a centralized pathway for consumer engagement in health technology assessment (HTA) processes. The hub enables consumers (patients, carers, health professionals, and citizens) to provide commentary on items being considered by HTA committees for subsidization.

Methods

A survey was developed by the CEEU, committee members, and consumer representatives to facilitate consultation on applications assessed by the Pharmaceutical Benefits Advisory Committee (PBAC)—a principal Australian HTA committee. Questions were designed as simple and engaging, including guidance on the type of information required. Responses are summarized thematically by efficacy, safety, accessibility, and quality-of-life impacts of the proposed health technology. New surveys are launched to coincide with each PBAC meeting agenda publication and allow a ten-week consultation period. Awareness of consultations is supported by the CEEU’s HTA Engage e-newsletter, which alerts the public and targeted stakeholders.

Results

The hub surveys have enabled streamlined consumer commentary to be provided for PBAC considerations. It has also allowed increased time for quality consultation with stakeholders. The success of the hub is further demonstrated in the current development of a similar survey for another principal committee, the Medical Services Advisory Committee (MSAC), to transition its consultation processes to the hub. Of note, while consumers’ feedback on the hub is positive, there remains a desire for educational resources and face-to-face interactions to support consumer engagement in HTA processes. The CEEU are developing materials to address and further support this need.

Conclusions

In a time when technological communication can be optimized to complement face-to-face conversations, it is vital consumer engagement in HTA processes follows suit. To facilitate continued engagement that is sustainable for both present and future Australians, the CEEU continues to evolve a strategy regarding virtual consultations to increase consumer awareness and education and promote effective participation in Australian HTA.
OP71 Patient Disease Strategy: A New Operational Framework For Collecting And Applying Patient Experience Data Into Clinical Development Programs
Authors: Coulouvrat Catherine , DiBiaso Victoria , Bascle Stephanie , Lucats Laurence , Largeron Nathalie , Tomme Sophie Van , Arnould Benoit
Published: January 2025

Introduction

Understanding patient experience and needs is crucial to develop high-value therapies. Patient experience data (PED) inform trial design and evidence generation plans. The U.S. Food and Drug Administration’s roadmap to patient-focused outcome measurement advocates integrating PED into product development. We adapted this theoretical framework to include the health technology assessment (HTA) perspective and operationalized it as a patient disease strategy (PDS) framework.

Methods

The PDS framework is a methodology that systematically integrates patient-informed activities to reflect the patient health value of a new treatment. A PDS is developed per indication, initiated in the preclinical phase, applied in clinical development, and continuously adapted throughout the product development lifecycle. The three PDS phases include: (i) development of patient profile, including epidemiology, demographics, patient journey, disease, and treatment burden for patients and caregivers; (ii) PED gap analysis, focusing on identification of patient priorities, unmet needs, preferences, and expectations for new therapies; and (iii) translation into actions, such as diversity and inclusion (D&I) plans and outcomes strategy.

Results

Out of 58 indications, 31 percent have endorsed PDS and 67 percent are in progress. Patient-relevant label opportunities increased by over 50 percent. Each indication was informed on average by patients from three different countries. The PDS framework helped to identify factors that impacted health outcomes for integration into trial designs and D&I plans. Early understanding of heterogeneous patient populations, unmet needs, benefit/risk trade-offs, and patient experiences ensured development programs measured the most meaningful outcomes while also addressing evidence gaps. Early understanding of patient priorities and barriers to participation optimized the studies by reducing burden and identifying proactive support needed to complete the trial.

Conclusions

The PDS framework systematically identified health value opportunities for a target population and integrated the patient needs into the overall development plan. PED informs clinical trial design and endpoint strategy optimization, including factors that influence diversity and data integrity. We anticipate that the PDS will enable HTA decisions to reflect patients’ health value and ultimately improve access to innovative therapies.
OP79 Innovations In Measuring And Valuing Outcomes: Generating Preferences For The EQ Health And Wellbeing Short
Authors: Mukuria Clara
Published: January 2025

Introduction

The value of health technologies may not be fully reflected in existing generic preference-based measures (GPBMs). The nine-dimension EQ Health and Wellbeing Short (EQ-HWB-S) is a new GPBM developed to assess health, informal carer, and social-care-related quality of life. The objective was to compare standard and new approaches used to generate preferences for the EQ-HWB-S.

Methods

Three feasibility studies that included qualitative work have valued the EQ-HWB-S with members of the public in the United Kingdom: (i) videoconferencing interviews using time trade-off (TTO) and discrete choice experiments (DCEs) from the EuroQol Valuation Technology (EQ-VT) protocol (n=600); (ii) online DCEs using the Potentially All Pairwise RanKings of all possible Alternatives (PAPRIKA) method (n=300), an adaptive DCE with a binary search to locate “dead”; and (iii) Online elicitation of Personal Utility Functions (OPUF) (n=300), which is a compositional method with dimension weighting, response level rating and anchoring on dead. Participant/interviewer feedback, data quality, and the weights were compared.

Results

Self/interviewer reported understanding was high (>70%) across all studies. Qualitative findings indicated misunderstanding for some OPUF steps (e.g., the anchoring on dead). Inconsistencies or illogical answers were small in the EQ-VT study (7%) and OPUF (16%). The PAPRIKA study had a priori exclusions criteria (e.g., time taken) that resulted in 44 percent exclusions. Pain, activity, mobility, and sadness/depression were the most important in all the studies. The value of the worst state was −0.384, −0.51, and −0.15 for EQ-PVT, PAPRIKA, and OPUF, respectively, and there were differences in dimension weights (e.g., PAPRIKA gave less weight to mobility but more to cognition).

Conclusions

EQ-HWB-S is a GPBM that provides an innovative approach to measuring and valuing outcomes. Standard and new approaches to eliciting preferences are feasible, but there are differences in the resultant weights. PAPRIKA and OPUF may improve attribute attendance and be more cost effective as they are administered online, but there is scope for improvement to ensure understanding and engagement.
OP81 Experience Matters: A Discrete Choice Experiment Exploring Patient Preferences For Heart Valve Procedures
Authors: Fifer Simon , Keen Brittany , Guilbert‐Wright Polo , Murdoch Dale
Published: January 2025

Introduction

Treatment for heart valve diseases (HVD) typically involves surgery, but less invasive procedures are becoming more common. Although the two procedures have similar outcomes, the risk–benefit profiles differ, indicating patients should be included in treatment decisions so they align with their values/preferences. This study aimed to determine patients’ preferences for HVD procedures, and the relative importance of treatment attributes.

Methods

An online survey with discrete choice experiment (DCE) was disseminated to patients with aortic stenosis, mitral valve regurgitation, and tricuspid valve regurgitation. Participants were presented with several choice sets, each comprising two hypothetical treatment procedures (labeled “invasive procedure” and “minimally invasive procedure”) as well as an opt-out. DCE attributes were selected based on a literature review, qualitative interviews with patients and specialist doctors, and steering committee consultation (patients, patient organization representatives, and cardiac physicians). Responses were collected via healthcare recruiters, online panels, and patient organizations. DCE data from 143 Australian patients was analyzed using a mixed multinomial logit (MMNL) model.

Results

Results indicate an “experience effect” whereby patients preferred the same type of treatment they had undergone previously. For example, patients who had undergone a transcatheter procedure were more likely to choose the minimally invasive procedure in the experiment and vice versa for those who had undergone invasive procedures like open-heart surgery. Patients were willing to switch procedures based on its risk–benefit profile, and most patients preferred the minimally invasive procedure when it reflected the profile of transcatheter aortic valve replacement (TAVI), even if they had previous invasive procedures experience. Key attributes driving choice were valve durability and regaining independence.

Conclusions

There is a great deal of heterogeneity in HVD patient preferences, even when treatment outcomes appear similar. Patients preferred a minimally invasive procedure over an invasive procedure, irrespective of prior treatment experience with valve durability and independence driving choice. These results can inform healthcare decision-makers about what features of HVD procedures patients value most, taking into consideration patients’ prior experiences.
OD02 Developing Components For A National Strategy For Heart Valve Disease In Canada
Authors: Sproule John M , Warkentin Lindsey , Messika-Zeitoun David
Published: January 2025

Introduction

The research included a rapid review of current literature to describe epidemiology, management, and system impact of heart valve disease (HVD) in adult populations. Key issues were identified in consultation with expert focus groups and were framed across the continuum of care and systemic policy issues. The groupings were adapted and adjusted during deliberations.

Methods

A rapid literature review was conducted on HVD key interventions and evidence of effectiveness along with expert interviews to identify high-level themes for reform. This served as the evidentiary backgrounder. Two virtual policy engagements with clinical leaders, patients, and health system managers were conducted. The focus was their collective drafting of recommendations. These workshops identified nine thematic areas and developed associated recommendations for action under each theme. The success of the process is evident as the report has been taken up as a roadmap for ongoing research and policy work.

Results

A comprehensive grouping of recommendations for improving HVD detection, management, and treatment in Canada was produced. It was designed to be comprehensive to then allow more targeted work to proceed under an evidence-informed and clinically endorsed agenda.

Conclusions

Heart valve conditions are increasingly treatable, especially if detected early. Innovation in treatments as well as detection and management to address gaps in care were identified as the most urgent priorities. The key result was formation of formal working groups with a professional society to explore spoke–hub-and node models for care delivery and to launch awareness programs for early detection.
OD08 Health Technology Assessment Timelines And Outcomes Of Common Compounds In France, Germany, Sweden, And Poland From 2014 To 2022
Authors: Sola-Barrado Belen , Wang Tina , McAuslane Neil
Published: January 2025

Introduction

The Regulation (EU) 2021/2282 on health technology assessment (HTAR) takes effect in January 2025 and aims to improve and accelerate patients’ access to new health technologies. We examine HTA timelines and outcomes for new active substances (NASs) commonly appraised in all of France, Germany, Poland, and Sweden from 2014 to 2022, providing insights into the European landscape before HTAR.

Methods

Public data was extracted from the HTA reports from the corresponding health authorities from France (HAS), Germany (IQWiG), Poland (AOTMiT), and Sweden (TLV) published between 1 January 2014 to 31 December 2022. NASs appraised by HTA in the four jurisdictions were referred to as common compounds. Time to recommendation was calculated as European Medicines Agency approval to HTA recommendation in the corresponding country. Differences in time parameters were assessed using the Kruskal–Wallis test. HTA recommendations were categorized as positive, positive with restrictions, and negative.

Results

A total of 97 common compounds (75% chemical entities and 25% biotechnology products) were identified (388 HTA appraisals). Notably, 47/97 (48%) NASs were associated with anticancer drugs (ATC-code=L). The median (interquartile range) time to recommendation differed by jurisdiction: 128 (113, 169) days in Germany; 203 (151, 308) days in Sweden; France, 209 (162, 294) days; Poland, 479 (330, 738) days (p-value <2.2e-16). In addition, 62/97 (63.9%) products received their initial HTA recommendation in Germany, followed by 25/97 (25.8%) products in Sweden, and 10/97 (10.3%) in France. Only 7/97 (7%) of products exhibited unanimous HTA outcomes across jurisdictions.

Conclusions

Discrepancies in the time to HTA recommendation were identified across four prominent European jurisdictions, and consensus in HTA outcomes was notably limited across countries, underscoring the intricate landscape preceding the enforcement of the HTAR. The future implementation of HTAR holds the potential to address these disparities, fostering greater harmonization in HTA processes and outcomes across the European healthcare landscape.
OD11 Application Of Natural Language Processing To Predict Final Recommendation Of Brazilian Health Technology Assessment Reports
Authors: de Almeida Cardoso Marilia Mastrocolla , Machado-Rugolo Juliana , Thabane Lehana , da Rocha Naila Camila , Barbosa Abner , Almeida Juliana Tereza , da Silva Pereira Curado Daniel , Komoda Denis Satoshi , Theresa Weber Silke Anna , Modelli de Andrade Luis Gustavo
Published: January 2025

Introduction

Several countries established health technology assessment (HTA) processes to support decision-making. Considering the high volume of submissions processed by HTA agencies, approaches to determine factors associated with the approval would be beneficial. This study aimed to predict the final recommendation of the National Committee for Health Technology Incorporation (Conitec) using a natural language processing (NLP) algorithm for text extraction.

Methods

Conitec’s 2012 to 2022 reports (n=389) were split into 75 percent training and 25 percent testing data. Tokenization enabled NLP models: Least Absolute Shrinkage and Selection Operator (LASSO), logistic regression, support vector machine (SVM), random forest, neural network, and Extreme Gradient Boosting (XGBOOST). Evaluation criteria included accuracy, area under the receiver operating characteristic curve (ROC AUC) score, precision, and recall. Cluster analysis with k-modes identified two clusters (group 0 = approved, group 1 = rejected).

Results

The neural network model demonstrated the best accuracy metrics with a precision of 0.815, accuracy of 0.769, ROC AUC of 0.871, and a recall of 0.746. Some tokenization identified that linguistic markers could contribute to the prediction of incorporation decision by the Brazilian HTA Committee, such as international HTA agencies’ experience and the government as the main requester. Cluster and XGBOOST analysis identified similar results with approved technologies with a predominance of drugs assessment, mainly requested by the government, and not approved mostly assessing drugs, the industry as the main requester.

Conclusions

The NLP model could identify predictors for the final decision process on the incorporation of health technologies in Brazil’s Unified Health System, opening paths for future work using HTA reports coming from other agencies. This model could potentially improve the throughput of HTA systems by supporting experts with prediction/factors/criteria for approval or nonapproval as an earlier step.
OD29 From Listening To Lift Off: Developing A Three-Year Public Involvement And Engagement Strategy For NICE
Authors: Rasburn Mark , Crosbie Helen , Fitzpatrick Ella , Livingstone Heidi , Morgan Clare , Norburn Laura , Taylor Sally , Tonkinson Mandy , Wigmore Janine
Published: January 2025

Introduction

Involving patients in the health technology assessment (HTA) lifecycle is a core principle at the National Institute for Health and Care Excellence (NICE). To achieve this, NICE has adopted a mixed approach to patient and public involvement and engagement (PPIE) spanning the entire appraisal process. To ensure the PPIE approach enables meaningful involvement, NICE engaged with stakeholders to review its effectiveness and identify areas for improvement.

Methods

In 2023, an independent consultant reviewed NICE’s PPIE approach and engaged with NICE staff and external stakeholders from patient organizations, individual patient contributors, and engagement leads at national health and social care organizations. The engagement included interviews with NICE staff (n=19) and external stakeholders (n=13), and an online survey that received 83 responses from patient organizations and patient contributors. Using this feedback, NICE’s patient and public involvement program conducted four focus groups to develop a framework of improved methods and processes for PPIE with NICE staff, patient organizations, and patient contributors.

Results

The engagement identified many positives in NICE’s approach to PPIE, including:

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lay members sitting on each HTA committee as equal members

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patient organizations providing written evidence to HTA committees

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patient experts providing written and verbal testimony to HTA committees

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support provided by NICE.

The engagement also identified areas where PPIE could have a greater impact, including:

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improved methods for collecting patient evidence and insight

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strengthening the role of lay members

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collating and reusing previously collected patient evidence

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taking a proportionate approach to involving small organizations

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allocating staff resources to focus on impactful PPIE practices.

Conclusions

NICE has developed a draft framework for an improved approach to increase the impact of PPIE in HTA decision-making. In 2024, NICE will publicly consult with NICE staff and external stakeholders to review the framework, agree the strategic aims, and develop metrics for measuring success. Following this consultation, the findings and NICE’s updated approach to PPIE will be presented.
OD31 Value-Based Healthcare And Health Technology Assessment: Opportunities For Implementing A Colorectal Cancer Patient-Centered Care System
Authors: Asselin Genevieve , L’Espérance Sylvain , Nourissat Alice , Rhainds Marc
Published: January 2025

Introduction

The value-based healthcare (VBHC) concept links dollars spent to outcomes that matter to patients, rather than to the volume of services. A health technology assessment (HTA) was carried out to determine if the VBHC model could be effective to improve care delivery in colorectal cancer at the CHU de Québec-Université Laval.

Methods

A systematic review on VBHC models implemented in oncology was conducted in indexed databases and grey literature between January 2000 and January 2022. Semistructured interviews were conducted from various key informants in our hospital (n=19), including three patients, to characterize the colorectal cancer care organization. HTA was conducted with an interdisciplinary group including surgeons, nurses, hospital managers, and patients.

Results

Key factors to consider in the implementation of VBHC include organizing care around the same medical condition, measuring outcomes and costs for every patient, and tracking data through information technology platforms. Results from case studies suggest that VBHC may have promising effects on clinical, patient-reported, and process outcomes measures. Several issues regarding the degree of agreement with the VBHC principles have been identified in our facility, including a non-uniform care trajectory, a lack of an integrated interdisciplinary team, and the absence of efficient information technologies.

Conclusions

It was recommended that the CHU de Québec-Université Laval initiates an organizational transformation in colorectal cancer care to implement a VBHC model. An effective care transformation will require a significant culture change with impacts on organization and practices but also with positive spin-offs by creating value and patient-centered
care.
OD44 Consolidated Health Economic Evaluation Reporting Standards For Interventions That Use Artificial Intelligence (CHEERS-AI)
Authors: Avsar Tuba Saygin , Elvidge Jamie , Hawksworth Claire , Knies Saskia , Zemplenyi Antal , Petykó Zsuzsanna , Siirtola Pekka , Chandra Gunjan , Srivastava Divya , Denniston Alastair , Chalkidou Anastasia , Delaye Julien , Nousios Petros , Gomes Manuel , Wang Junfeng , Petrou Stavros , Dawoud Dalia
Published: January 2025

Introduction

Progress and innovation in artificial intelligence (AI)-based healthcare interventions continue to develop rapidly. However, there are limitations in the published health economic evaluations (HEEs) of AI interventions, including limited reporting on characteristics and development of algorithms. We developed an extension to the existing Consolidated Health Economic Evaluation Reporting Standards (CHEERS) to improve consistency, transparency, and reliability of the reporting of HEEs of AI interventions.

Methods

The Delphi method was used, following a prespecified study protocol. A steering group with expert oversight was formed to guide the development process. A long list of potential items was defined based on two recent systematic reviews of HEEs of AI-based interventions. The steering group identified and invited 119 experts to the three-stage survey. Participants were asked to score each item on a nine-point Likert scale, and they were also able to provide free-text comments. The final checklist was piloted on a random sample of nine HEEs of AI-based interventions.

Results

Three stages of the Delphi survey were completed by 58, 42, and 31 multidisciplinary respondents, respectively, including HTA specialists, health economists, AI experts, and patient representatives. The CHEERS-AI extension includes 18 AI-specific reporting items. Ten are entirely new items, including considerations about user autonomy, validation of the AI component, and AI-specific uncertainty. In addition, elaborations on eight existing CHEERS items were added to emphasize important AI-specific nuances. Some participants highlighted that CHEERS-AI can provide key benefits; for example, it could clarify the misconception that the predictive algorithms supporting AI-driven healthcare interventions are available for use without cost.

Conclusions

CHEERS-AI can aid in improved reporting quality for researchers, editors, and reviewers conducting or assessing HEEs of AI interventions.