bluuebunny/crossref_metadata_2025 huggingface dataset:
OP40 Circumstances That Unlock Payer Acceptability Of Innovative Trial Designs: A Case Study And Primary Research-Based Global Analysis
Authors: Sundaram Sudha , Sutharsan Vesshnu , Kielar Catherine , Nørtoft Emil
Published: January 2025

Introduction

Innovative designs have been used to enhance trial efficiency and enable access to novel treatments when traditional randomized controlled trials (RCTs) were considered operationally challenging to implement. Besides demonstrating scientific and regulatory rigor, innovative approaches also need to be perceived favorably by payers. This work delves into payer perspectives on innovative designs, identifying circumstances that contribute to higher payer acceptability.

Methods

Using targeted searches, we mapped clinical trials since 2010 with novel design elements, including adaptive, master protocol, hybrid, enrichment, and innovative endpoints. Sixty-two asset-indication examples using these designs were identified across different therapeutic areas. Based on the availability of health technology assessment (HTA) reports and the innovative element’s impact on final HTA outcome, 17 of these identified examples were developed as case studies to highlight the designs’ implications for access. Interviews with eight payer-experts across US, France, Germany, and Japan were conducted to further validate the research, explore scenario analyses, and clarify how circumstances impacted payer acceptability of the studied innovative trial designs.

Results

While oncology has historically spearheaded innovative trial designs, other therapeutic areas are now incorporating innovative elements. Nonetheless, published payer guidance on innovative designs remains limited. We identified seven circumstances impacting payer acceptance of innovative designs: disease prognosis, eligible patient population size, type of treatment, mode of action, availability of treatment alternatives, comparative benefit versus standard of care, and launch sequence. Patient population size had the greatest impact on payer decisions, followed by comparative benefit and type of treatment; this suggests that payers may be inclined to accept innovative trial designs for small populations with high unmet need or therapies with transformative clinical benefits.

Conclusions

Innovative trials trade some scientific validity for increased practicality compared to traditional RCTs. Stakeholders need to align on appropriateness and scientific validity of innovative designs for access decisions, while implementing measures to minimize uncertainties. Collaboration across stakeholders including regulators, payers, and manufacturers is needed to refine innovative methodologies and guide policies to improve the relevance of innovative trials for decision-making.
OP43 Incorporating Mathematical Modeling To Improve Accuracy Of Budget Impact Analysis: Using Screening For Hepatitis C As An Example
Authors: Wong William
Published: January 2025

Introduction

The majority of those infected with chronic hepatitis C (CHC) are asymptomatic. Population screening has proven to be both effective and cost effective. When considering whether to implement screening or not, the uncertainty of the budget impact plays an important role. This study aims to develop methods that improve the accuracy of budget impact analysis for a one-time CHC screening program.

Methods

We developed a back-calculation mathematical model that employs a Markov chain Monte Carlo algorithm to estimate the prevalence and proportion of undiagnosed CHC. Subsequently, we utilized a state-transition model to assess the budget impact of two strategies: (i) no screening; and (ii) screen-and-treat with direct-acting antiviral (DAA) for individuals born between 1945 and 1965 (“baby-boomer” birth cohort). Model data were gathered from published literature. Our analysis adopted a Canadian provincial payer perspective, employed a 10-year time horizon, and followed best-practice recommendations by not applying discounting.

Results

For individuals born between 1945 and 1965, the estimated prevalence of CHC was 1.74 percent (95% confidence interval [CI]: 1.52, 2.30) with an undiagnosed proportion of 15.72 percent (95% CI: 11.27, 18.54). The initial budget impact analysis indicated an additional cost of CAD61.5 million (USD45.0 million) over 10 years for screening related individuals for CHC in Ontario. With these updated prevalence and undiagnosed proportion estimates, our projection suggests a 29.6 percent reduction in the budget impact, now estimated at CAD43.3 million (USD31.7 million).

Conclusions

By comparing the budget impact of the CHC screening strategy with other recommended health services and technologies in Ontario, we have concluded that CHC screening may be considered affordable. To enhance the accuracy of budget impact analysis for population-level screening decision-making, it is crucial to develop precise methodologies for estimating the underlying prevalence and undiagnosed proportions.
OP45 Incorporation Of The Tetravalent Dengue Vaccine In The Brazilian Public Health System: A Cost–Benefit Analysis
Authors: Gomes dos Santos Ana Zulmira , Barros Gabriella Santos , Gonzalez Mosegui Gabriela Bittencourt , Martins de Freitas Gabriel Rodrigues
Published: January 2025

Introduction

Dengue, a mosquito-borne disease, is prevalent in Brazil, ranking third in the Americas. The country has confirmed all four serotypes (DENV-1, DENV-2, DENV-3, and DENV-4) of the virus. In 2023, a tetravalent vaccine, approved for individuals aged four to 60 years, opens the possibility of integration into the public health system.

Methods

The invested amount for purchasing two doses of the vaccine to immunize the entire eligible population in the year 2023 was calculated. A comprehensive analysis of direct and indirect costs was conducted regarding the integration of the tetravalent dengue virus vaccine into Brazil’s public healthcare system. Direct costs encompassed treatment expenses for mild, moderate, and severe cases, while indirect costs involved workdays lost and mortality-related expenses. Direct and indirect costs were compared in two scenarios (with and without vaccination), followed by a cost–benefit ratio calculation.

Results

The investment for procuring vaccines for over 168 million Brazilians amounted to approximately EUR17.9 billion, resulting in savings of about EUR193.5 million. Indirect costs were particularly significant when compared to the non-vaccinated population. Considering herd immunity and reducing the vaccinated population to 70 percent of the eligible populace, the invested amount was approximately EUR12.5 billion, while savings reached EUR214.2 million. A cost-benefit ratio calculation revealed a return of one centavo (EUR0.0019) for every BRL1.00 invested (EUR0.0019), and considering herd immunity, the cost–benefit ratio was approximately 0.02.

Conclusions

Despite Brazil being one of the countries with the highest prevalence of dengue in the Americas, the availability of the tetravalent dengue virus vaccine in the country’s public system does not seem to be a sustainable option, given the unfavorable cost–benefit analysis for such implementation. Nevertheless, it is imperative to conduct this analysis with due consideration for alternative scenarios.
OP63 A European Value Assessment Framework For Next-Generation Sequencing And Comprehensive Genomic Profiling Oncology Diagnostics
Authors: Augustovski Federico , Chavez Danitza , Haig Madeleine , Main Caitlin , Argento Fernando , Colaci Carla , Mills Mackenzie , Alfie Verónica , Pichon-Riviere Andres , Alcaraz Andrea , Kanavos Panos
Published: January 2025

Introduction

Advances in next-generation sequencing/comprehensive genomic profiling (NGS/CGP) diagnostics require a value framework (VF) for accurate assessment within cancer care in Europe. Building on a previously established VF for diagnostic technologies in Latin America by the Institute for Clinical Effectiveness and Health Policy (IECS), this study synthesizes insights from a systematic literature review and stakeholder perspectives to inform the co-creation of an NGS/CGP diagnostic framework for healthcare decision-making.

Methods

The study utilized a mixed methods approach including a systematic literature review (SLR) and web-Delphi panel. The SLR identified and mapped existing VFs against the IECS VF, extracting non-overlapping criteria relevant to NGS/CGP in Europe. A Delphi panel further adapted and validated the framework, ensuring comprehensive stakeholder contribution and engagement. The Delphi´s first round was qualitative and involved open-ended feedback from participants to adapt the indicators and domains. Rounds two through four involved Likert-scale judgments of importance of each indicator. In rounds three and four, participants were shown the distribution of responses across stakeholders and could reconsider their answers.

Results

The SLR revealed 42 VFs with an 83 percent criterion overlap with the IECS VF, resulting in 46 indicators forming the literature-adapted framework. Thirty-four participants completed the Delphi. In round one, 14 indicators and 22 descriptions were adapted, 11 indicators were merged, 14 were deleted, and one was kept the same resulting in 29 indicators for scoring in round two. The final VF has 27 indicators: 23 essential and four complementary; two indicators did not reach consensus. The domains included clinical impact, test performance, scientific evidence quality, non-clinical impact, health system integration, economic aspects, ethical/governance concerns, and health system priorities.

Conclusions

This approach has yielded a robust, stakeholder co-created VF for NGS/CGP diagnostics in oncology, tailored to the European setting. It offers a comprehensive set of criteria that extends beyond traditional health technology assessments, incorporating novel aspects like post-test data governance. This framework sets the stage for improving patient access to high-value technologies by aligning European stakeholder values across health systems.
OP65 Focusing On What Matters Most: A Public Dialogue On How NICE Should Prioritize Topics In Health Technology Assessment
Authors: Murray Alice , Shah Koonal
Published: January 2025

Introduction

To meet the needs of an evolving health and care system, the National Institute of Health and Care Excellence (NICE) is changing its approach to topic prioritization so it can focus on what matters most. To support this, NICE ran a public dialogue to gather informed opinion on how it should select topics for guidance, including for some technology evaluation programs.

Methods

Fifty-five general public participants from across England took part in two face-to-face and three online deliberative workshops (each lasting two or three hours, held over four weeks in 2023). Participants were asked to consider the following criteria in the context of prioritization: health and care need, evidence availability, system impact, budget impact, health inequalities, and environmental sustainability. The workshops were designed to understand whether any aspects were more important than others and explore the reasons why. They used deliberative engagement methods and included trade-off exercises, role-play, group discussion, ranking tasks, and interactions with specialists.

Results

Emerging findings show that the participants think NICE should consider several aspects when prioritizing topics for guidance. Health and care need was of primary importance for people, followed by evidence availability, budget impact, and system impact. Health inequalities and environmental sustainability were generally considered to be less important, though participants still felt these were areas that should inform NICE’s prioritization decisions. Participants identified relevant interactions between the criteria, suggesting that each criterion cannot be considered in isolation. Full results will be available to present at HTAi 2024.

Conclusions

Deliberative public engagement is a meaningful way to involve the public in complex policy decisions with a social value element. Broad public agreement was found with the criteria NICE has proposed to consider when prioritizing topics for guidance, and some criteria are more important to people than others. The findings will feed into NICE’s new approach to topic prioritization.
OP80 Generating Patient Preference Evidence For Health Technology Assessment: A Sustainable Roadmap
Authors: Fifer Simon , Joshi Maya , Keen Brittany
Published: January 2025

Introduction

Research with members of health technology assessment (HTA) bodies has uncovered key barriers to integrating patient preference (PP) data into HTA, including concerns about resources/time constraints and a lack of clarity around who is responsible for data generation. We sought to develop a roadmap that addresses these issues, outlining the roles and responsibilities of different stakeholders to foster more sustainable PP data generation.

Methods

Based on a forthcoming article to be published in The Patient, this roadmap consists of a step-by-step approach for PP evidence generation. Real-world case studies and literature will be used to illustrate each stage, from identifying priority treatment areas and evidence gaps, forming a steering committee and engaging HTA members, to securing syndicated funding and disseminating results with full transparency.

Results

In contrast to standard approaches to data generation, this roadmap focuses on proactive data collection, collaborating with those who will ultimately use the data (HTA), and pooling resources to mitigate costs and the risk of bias. The roadmap can be applied to all preference-sensitive treatment areas and across health systems/countries. It is designed to be a continuous process, whereby preferences are regularly updated to align with changes to the treatment landscape. A graphic summary of the roadmap is available for viewing at this link: https://cappre.info/images/HTAprocess.pdf

Conclusions

Patient preference data has the potential to make healthcare decision-making more informed, socially legitimate, transparent, and accountable to the patient community. However, current approaches to capturing PP data can be resource intensive with narrow applicability in their findings. The present roadmap offers an alternative, sustainable solution.
OD01 Delays In Funded Access To Medicines: A Global Perspective
Authors: Laka Mah , Gao Yuan , Carter Drew , Merlin Tracy
Published: January 2025

Introduction

There are significant delays in the funded access to medicines. Studies indicate that in many countries it takes more than a year for patients to have funded access to medicines after market authorization. This study aimed to understand the disparities in timelines for funded access to medicines across different countries and to identify underlying reasons for this access gap.

Methods

We conducted a scoping review to examine the nature of health technology assessment (HTA) processes, current methods, and policies for medicines in ten jurisdictions. The jurisdictions included in this study are Australia, Canada, France, Germany, South Korea, the Netherlands, United Kingdom (divided into England, Scotland and Wales), and United States of America. The information was extracted from the websites of International Network of Agencies for Health Technology Assessment (INAHTA) member agencies in the selected jurisdictions, grey literature from governments’ websites, and peer-reviewed literature.

Results

Overall median time from submission of the evidence dossier to HTA recommendations for most jurisdictions is 22 weeks. Although there are similarities in the time taken to reach a funding decision, there are considerable variations in the time taken for patients to have funded access to medicines after HTA recommendations. Only a few countries mentioned a specific timeline within which medicines approved for funding should be listed. Time taken for price negotiations and other arrangements (i.e., risk-sharing agreements) may contribute to varying timelines for listing medicines for funding. Mostly, such negotiations are confidential and may not be time limited.

Conclusions

There was surprising consistency, globally, in the time it takes for funding decisions after medicines registration. The causes of delays in the medicines’ listing decisions are multifactorial and mostly occur after HTA recommendations. The parallel regulatory-assessment process and prioritization tend to reduce the time to a funding decision. However, transparency is needed in the listing process to improve overall timeliness.
OD09 How Are Population/Intervention/Comparator/Outcomes Criteria For Pharmaceutical Assessments Determined Around The World?
Authors: Newton Skye , Hill Hayley , Merlin Tracy
Published: January 2025

Introduction

Defining the population, intervention, comparator, outcomes (PICO) criteria is an essential step prior to performing a health technology assessment (HTA), but variations exist in how this step is performed.

Methods

A scoping review was performed to compare the processes and guidance provided for developing the PICO criteria for the assessment of new medicines across Australia, the UK, Canada, the US, European Union (as a single jurisdiction), Germany, France, the Netherlands, South Korea, and Taiwan. The websites of HTA agencies in these jurisdictions were searched for methodological guidance, and PubMed, Embase, and the HTA database were also searched for published literature on the topic of the process or methods for developing the PICO criteria.

Results

Two main approaches are used for developing the PICO criteria. In the UK, US, and European Union, a separate scoping process is used; in the remaining countries, the pharmaceutical manufacturer defines the PICO criteria as part of developing their dossier for submission. Guidance on PICO elements were similar in content but highly varied in the degree of guidance provided. The largest differences were in whether outcomes for people beyond the treated individual were recommended to be assessed.

Conclusions

A separate scoping phase allows stakeholder input into the criteria, which is important with the shift to incorporating more patient input into each phase of HTA. It can come at the cost of timeliness, so requires manufacturers to engage with the HTA systems earlier in the process.
OD15 Multiparametric Magnetic Resonance Imaging In Biopsy-Naive Patients: A Real-World Evidence For Decision-Making In Prostate Cancer Pathway
Authors: Asselin Genevieve , Tremblay-Boily Melanie , Fradet Yves , Rhainds Marc
Published: January 2025

Introduction

Use of multiparametric magnetic resonance imaging (mpMRI) in the prostate cancer (PCa) diagnostic pathway could reduce prostate biopsy (Bx) in Bx-naive patients. The objective was to assess the feasibility of implementing a new PCa diagnostic pathway with the addition of mpMRI in a real care environment.

Methods

Following an HTA report published in 2019 by our team, a committee involving stakeholders (e.g., urologists, radiologists, hospital managers) was created to review the PCa diagnostic pathway including mpMRI in Bx-naive patients and both systematic and targeted 3DTRUS-MRI fusion Bx when Bx was recommended. Data in the new PCa diagnostic pathway were collected between September 2021 and June 2022. The comparison group is a cohort of 629 men who underwent an initial systematic transrectal ultrasound Bx in 2017 when prostate mpMRI was not available. Clinically significant PCa (csPCa) was defined as Grade Group ≥2.

Results

In 2021 and 2022, 1,336 Bx-naive patients were referred to a urologist. Recommendations were: 703 (53%) for follow-up in six to 12 months, 254 (19%) directly to systematic Bx, and 379 (28%) to mpMRI. Overall, csPCa was diagnosed in 246/427 (58%) patients referred to mpMRI or Bx in the 2021 to 2022 cohort compared to 274/629 (44%) patients in the 2017 cohort (p<0.05). The new diagnostic pathway prevented 33 percent of patients from having Bx. Shorter delays between initial consultation with urologists and transmission of Bx results were observed for patients referred directly for prostate Bx compared to mpMRI before Bx (mean: 2.8 vs 9.1 months).

Conclusions

Experimentation in a real care setting has highlighted the added value of the early involvement of urologists in the diagnostic pathway of prostate cancer for the triage of patients. Integration of mpMRI was associated with a lower number of patients referred for prostate Bx and a higher csPCa detection rate.
OD24 Scalability Analysis Of Multimodal Prehabilitation For Frail Elderly Patients Before Elective Surgery In Germany
Authors: Felgner Susanne , Eckhardt Helene , Weber Zoe , Quentin Wilm , Rombey Tanja
Published: January 2025

Introduction

Multimodal prehabilitation, including interventions like physiotherapy, combined with frailty screening and a shared decision-making conference for frail elderly patients before elective surgery is an innovative approach currently under investigation (PRAEP-GO RCT, NCT04418271). The PRAEP-GO intervention aims to enhance postoperative outcomes and prevent care dependency. Our aim is to systematically assess the scale-up potential of PRAEP-GO within the German healthcare system.

Methods

We are conducting a scalability analysis using the Intervention Scalability Assessment Tool (ISAT). The ISAT questionnaire comprises two parts: (A) “Setting the scene,” describing the current health service situation (e.g., intervention characteristics, political context), and (B) “Intervention implementation planning,” outlining future requirements (e.g., workforce, infrastructure), with open-ended questions and a scalability readiness assessment using a scale. Our analysis involves three stages: (i) health economists from the PRAEP-GO research team individually answering ISAT questions, (ii) trialists from the PRAEP-GO research team interviewed in a group, and (iii) external experts representing relevant stakeholders for future implementation interviewed in an advisory board meeting.

Results

Data collection for stage (i) and (ii) has been completed, while data collection for stage (iii) is expected to be completed in February 2024. The preliminary findings for part (A) highlight the need for a sustainable approach to manage an aging and increasingly frail patient population requiring surgery. There is no clinical guideline available for the management of this population group. Regarding part (B), the current infrastructure (e.g., therapy facilities) and personnel structures might need to be adapted and should be expanded for large-scale application. Employing professionals to coordinate the patient pathway was recommended, along with adjustments to reimbursement structures.

Conclusions

In PRAEP-GO, we are pursuing a multidisciplinary process with the aim of supporting health decisions that promote an equitable, efficient, and high-quality healthcare system meeting the challenges of an aging population due to demographic change. The PRAEP-GO trial is currently exploring this approach on a small scale. Existing infrastructure and personnel structures would need to be adapted and expanded for scale-up.
OD37 How The PICOTS-ComTeC Framework For Digital Health Interventions Can Be Useful For Health Technology Assessment
Authors: Zrubka Zsombor , Champion Annette , Di Bidino Rossella , Holtorf Anke-Peggy , Earla Jagadeswara R , Boltyenkov Artem , Tabata-Kelly Masami , Asche Carl , Burrell Anita
Published: January 2025

Introduction

The diffusion of digital health interventions (DHIs) requires agreement on a minimum information framework to define them. The ISPOR Digital Health (DH) Special Interest Group (SIG) developed the PICOTS-ComTeC framework based upon a systematic review and a Delphi study. It is an enriched version of the traditional PICOTS widely adopted in health technology assessment (HTA). ComTeC stands for communication, technology, and context.

Methods

The PICOTS-ComTeC is based upon a review that included the Shannon–Weaver model of communication, AHRQ Quality Measures, technology, geography, and the World Health Organization classification of DHIs followed by a Delphi panel. The development process adhered to the EQUATOR guidelines. The PICOTS-ComTeC aims to be a flexible and versatile tool tested on different DHIs. The results of the testing are discussed from the HTA perspective considering the tool’s additional value, utility for and applicability to HTA. The additional value is strictly linked to the actual need for a dedicated PICOTS for DHIs and its implications for HTA assessments of DHIs.

Results

The PICOTS-ComTeC was tested internally and externally to the ISPOR DH-SIG on four DHIs for breast cancer surgery/management/patient education, one DHI for obesity, and one DHI for patients with heart failure. The testing phase demonstrated the level of detail required to use the tool, hitherto available evidence to cover all domains, and opened up discussion on implications of the PICOTS-ComTeC framework for HTA related activities (i.e., scoping, literature search, comparator selection). It emerged that there is a diffuse lack of homogeneity and details when DHIs are defined in the literature with significant implications for conducting appropriate HTAs.

Conclusions

The diffuse adoption of the PICOTS-ComTeC for patient-facing DHIs will promote a greater level of detail in order to define homogenous DHI groups. The implications for HTA range from the definition of relevant research questions to the selection of the most appropriate comparator so that assessments are geared to fulfill the needs of decision-makers.
OD41 Conducting Systematic Reviews On Rare Diseases: Lessons Learned From The European Reference Networks Guidelines Programme
Authors: Martín-Gómez Carmen , Isabel-Gómez Rebeca , García Guillermo Pérez , Vicente Edo Maria José , De Armas-Castellano Aythami , Trujillo-Martín Mar , Pla Anna Godo , Estrada Sabadell Maria Dolors , García Josune Domínguez , Jausoro Ainhoa
Published: January 2025

Introduction

A consortium of five Spanish health technology assessment (HTA) agencies conducted the European Reference Networks Guidelines Programme for the development, appraisal, and implementation of clinical practice guidelines aiming to support clinical decision-making in the field of rare diseases (RDs). In response to this objective, methodologists and information specialists conducted systematic reviews (SRs). This study aims to explore the barriers/facilitators they encountered.

Methods

A survey was designed to elicit HTA agencies’ experience in developing SRs on RDs. Information was collected on the number of SRs conducted and the types of RDs and clinical questions addressed. In addition, they were asked to identify barriers and facilitators for each stage of the review (from the definition of PICO [population, intervention, comparator, outcome] components of the question to the issuing of recommendations). Finally, they were asked for process improvement suggestions. The survey was distributed by email and completed online. A thematic analysis was conducted to identify the issues identified at each stage of SR.

Results

A total of 111 SRs were conducted on 35 RDs. Most clinical questions were about diagnosis and treatment. The main barriers identified were lack of MesH (Medical Subject Headings) terms associated with the conditions, non-representative abstracts and keywords, lack of relevant information in the body of the articles, and reported data not allowing for quantitative syntheses or recommendations to be made. Facilitating aspects included Orphanet’s specific source of RD documents and having expert clinicians in the working groups who were also involved in all steps of the SR.

Conclusions

Conducting SRs in the field of RDs is challenging. Authors of primary studies are encouraged to be more exhaustive in reporting the results. More research focused on the SR methodology in RDs is necessary to address their particular characteristics and obtain robust results. It is crucial to collaborate with reference networks to address RDs, where the evidence is scarce.
PP16 Vial Sharing And Wastage Are Familiar Concepts, But What About Pack Sharing And Wastage?
Authors: Wilkes Sarah Kate , Turner Emily Eaton , Jiandani Neha , Thomson David
Published: January 2025

Introduction

The National Institute for Health and Care Excellence (NICE) conducts health technology assessment to assess cost-effectiveness and budget impact. For treatments provided in vials, NICE often considers how treatments are dispensed and adjusts the economic modeling costs accordingly. Vial sharing and wastage are likely familiar concepts to stakeholders, but the same consideration is not consistently given to tablet packs.

Methods

Using anonymized examples, NICE assessed potential implications for cost-effectiveness and budget impact of different methods for modeling oral treatments. Firstly, the cost-effectiveness and budget impact were calculated based on a cost per milligram (mg) of treatment. The per mg cost was multiplied by the number of mg for each dose and did not account for the number of tablets or packs required. Using the same example, the cost per tablet was calculated by rounding each dose to the nearest whole tablet mg dose. Finally, the example was costed based on whole packs, which included the cost for any wasted tablets.

Results

The anonymized examples showed that costing per mg versus per tablet versus per pack can have a significant impact on cost-effectiveness and budget impact. One example showed that treatment costs per 28 days could increase by over GBP1,000 (USD1,271) when costing per whole pack compared to per mg. This led to a difference in the incremental cost-effectiveness ratio (ICER) of nearly GBP10,000 (USD12,716). Another example demonstrated a potential increase in budget impact of nearly GBP1 million (USD1.27 million) per year. This magnitude of impact on cost-effectiveness and budget has the potential to change health technology assessment decisions and affordability in the United Kingdom.

Conclusions

NICE is assessing an increasing number of oral treatments provided as tablet packs, not vials. This highlights the need to consider how pack sharing and wastage should be consistently considered in economic modeling. Developing standardized methods for modeling oral treatments would help ensure consistency of cost calculations and better reflect how treatments are dispensed in clinical practice.
PP23 Machine-Learning-Based Evaluation Of Community Resilience Through Social Media During The First Post-COVID-19 Reopening In China
Authors: Zhang Shouchuang , Zhang Lanyue , Weng Jiayi , Yang Yiying , Chen Zefeng , Jian Weiyan
Published: January 2025

Introduction

The Chinese government lifted most COVID-19 pandemic restrictions in December 2022, triggering a spike in confirmed cases and higher demand for medications. Consequently, a significant number of residents resorted to social media to seek assistance. This study aimed to evaluate community resilience by leveraging Weibo user datasets, coupled with interpretable machine learning (ML)-based techniques, to identify important resilience characteristics.

Methods

Datasets geotagged from the Sina Weibo social media platform between 8 December 2022 and 7 January 2023 were crawled using search terms of “help-seeking” and the keywords of conventional drugs. This study utilized natural language processing (NLP) to label COVID-19-related posts to identify the type of posts, stakeholders’ behaviors, and other information. We built a comprehensive evaluation model, and five ML-based algorithms were compared for analyzing community resilience. Local interpretable model-agnostic explanations (LIME) was employed to verify five models and the XGBoost algorithm showed optimal effects. Shapley Additive Explanations (SHAP) elucidated the best model’s outputs and estimated contributions for key resilience characteristics.

Results

For this study, 199,709 posts were collected. Out of these, 48,425 posts were identified as help-seeking posts, with more than two-thirds receiving responses from community level. The area under curve (AUC) of the XGBoost model was 0.82 (95% confidence interval [CI]: 0.82, 0.83), and the values of accuracy and F1 score were 0.72 and 0.80, respectively. This result demonstrated that the model can successfully evaluate community resilience and subsequently identify the features driving this outcome. Collective efficacy in providing aid, support from official rescue guidelines, and residents’ rapid response to rescue information were identified as the most important characteristics for evaluating community resilience.

Conclusions

This study is the first to harness social media data to quantify community resilience in China based on a framework we developed. Five updated ML-based algorithms were developed to evaluate community resilience, and XGBoost showed optimal effects. Three characteristics of community resilience were found as potential predictors that can enhance decision-making support to reshape health emergency rescue activities.
PP28 The Final Arguments To Incorporate Or Not Incorporate Technologies For Ultra-Rare Diseases Into The Brazilian Public Healthcare System
Authors: de Paula de Sordi Monica Aparecida , Machado-Rugolo Juliana , Thabane Lehana , Santos Marisa , Komoda Denis Satoshi , Modelli de Andrade Luis Gustavo , da Silva Pereira Curado Daniel , Theresa Weber Silke Anna , de Almeida Cardoso Marilia Mastrocolla
Published: January 2025

Introduction

The decision-making process for health technology assessment (HTA) in ultra-rare diseases is a global challenge. Establishing a comprehensive analytical framework for these unique diseases poses difficulties. This study aims to descriptively analyze arguments reported by the Brazilian National Committee for Health Technology Incorporation (Conitec) in deciding whether to include technology for ultra-rare diseases.

Methods

Data from recommendation reports (2012 to 2022) were analyzed. Diseases with a prevalence of fewer than one per 50,000 inhabitants were classified as ultra-rare. Extracted variables included preliminary and final recommendation results and justifications by Conitec. Six argument categories were created (method-related issues; evidence; cost; technology effectiveness or safety; context; innovation). Word clouds were generated based on word frequency in each category to present the data.

Results

In the analysis of 45 reports, the word clouds highlight frequent terms in favorable arguments, emphasizing evidence quality, cost reduction, and applicability in the healthcare system. Conversely, unfavorable arguments also revolve around evidence quality and cost impact. The analysis of the arguments according to categories, 16 arguments were identified: seven concern evidence issues, five cite methodological problems in presented studies, four relate to costs, and three pertain to technology effectiveness or safety. Unfavorable arguments primarily stem from evidence-related concerns. In favorable arguments, cost (seven) and safety (six) are prominent, with innovation (one) and context (three) being additional categories not found in the unfavorable group.

Conclusions

While technology assessment processes for ultra-rare diseases have evolved, the justifications for recommending or not incorporating new technologies remain unchanged. Over time, reports have become more detailed, focusing on evidence and methodological specifics. This highlights the importance of scrutinizing evidence characteristics and determining relevant criteria and data types for this unique context.
PP32 Health Technology Assessment Regulation In Europe: Implications For A National Authority
Authors: Usher Cara , Fogarty Emer , Barry Michael , Adams Roisin
Published: January 2025

Introduction

The Regulation (EU) 2021/2282 on health technology assessment (HTAR) for medicines will come into effect in January 2025; initially, new oncology medicines and advanced therapy medicinal products will be assessed at EU level. How will this work in practice? What does this mean for national HTA bodies, such as the National Centre for Pharmacoeconomics (NCPE) Ireland that uses a cost-effectiveness framework to inform decision-making for medicines?

Methods

Joint work to be conducted under the Regulation includes joint clinical assessments (JCA), joint scientific consultations (JSC), and production of procedures and methodological guidance. A review was undertaken of key areas that will be impacted by the HTAR in the Irish HTA process for medicines, including timing, evidence synthesis structure, capacity building, and resource implications.

Results

The HTAR will alter the current process for medicines assessment in Ireland, from early scientific advice to cost-effectiveness assessment post-authorization. JSCs will represent an additional step. Significant training and capacity implications are associated with the JCA, which will require earlier engagement with stakeholders. The NCPE’s pragmatic HTA early triaging process, the “Rapid Review,” may be delayed due to the non-duplication clause in the HTAR. The availability of high-quality comparative effectiveness evidence may help avoid full HTAs in some cases. The benefit of the JCA will be realized if the results can directly inform treatment-effectiveness estimates in cost-effectiveness modeling.

Conclusions

The HTAR will significantly impact on medicines reimbursement procedures in Ireland. For the HTAR to be effective in achieving its aims, sufficient resources will need to be built into the EU HTA system. The balance between the extra resources needed and the resources spared will depend on the quality of the comparative effectiveness evidence available for the JCA.
PP38 Assessment Outcome By Type Of Available Evidence: Retrospective Analysis Of Oncological Early Benefit Assessment Reports
Authors: Goretzko Jonas , Pusch-Klein Annette , Wieseler Beate
Published: January 2025

Introduction

For early benefit assessment in Germany, drugs must be assessed at market entry as to whether they provide an added benefit versus the current standard therapy. This retrospective analysis focuses on assessment reports prepared by the independent Institute for Quality and Efficiency in Health Care (IQWiG). The objective was to describe the relationship between available evidence and assessment outcome.

Methods

Data were retrospectively extracted from IQWiG’s early benefit assessment reports of oncological drugs that entered the market between June 2021 and December 2021. The underlying evidence for the benefit assessments was divided into three groups: direct comparison (based on a randomized controlled trial [RCT]), indirect comparison, and other sources (e. g., only a single-arm trial was submitted). Afterwards, the outcome of the assessment was described by type of available evidence.

Results

In the period under review, IQWiG worked on 27 oncology projects addressing 40 different oncological research questions resulting in 46 conclusions. About half of the available evidence in the benefit assessments was a direct comparison with standard therapy based on an RCT (n=22/46). From this level of evidence, an added benefit was derived for the drug under assessment in 64 percent of cases. Indirect comparisons (n=7/46) mostly led to an added benefit not proven (86%). If only other sources were submitted (n=17/46), the added benefit was assessed as not proven in all projects examined.

Conclusions

This retrospective analysis highlights the pivotal role of RCTs in providing strong evidence for health technology assessment. Conversely, single-arm and observational studies fail to yield substantial evidence. In the future, RCTs should be made simpler, faster, and cheaper; for example, by integrating them into existing infrastructures in routine practice (e.g., registry-based RCTs) or by conducting randomized adaptive platform trials.
PP42 Implementing A Sustainable Enhanced Recovery From Surgery Pathway After Hip And Knee Arthroplasty: A Budget Impact Analysis
Authors: Lloyd Melanie , Ackerman Ilana , Harris Ian , Naylor Justine , Lewis Peter , de Steiger Richard , Wan Anthony , Buchbinder Rachelle , Ademi Zanfina
Published: January 2025

Introduction

Increasing resources are devoted to osteoarthritis surgical care in Australia annually, with significant expenditure attributed to hip and knee arthroplasties. Safe, efficient, and sustainable models of care are required. This study aimed to determine the impact on healthcare costs of implementing an enhanced short-stay model of care (ESS-MOC) for arthroplasty at a national level.

Methods

Budget impact analysis was conducted for hospitals providing arthroplasty surgery over the years 2023 to 2030. Population-based sample projections obtained from clinical registry and administrative datasets of individuals receiving hip or knee arthroplasty for osteoarthritis were applied. The ESS-MOC assigned 30 percent of eligible patients to a shortened acute-ward-stay pathway and outpatient rehabilitation. The remaining 70 percent received a current practice pathway. The primary outcome was total healthcare cost savings post-implementation of the ESS-MOC, with return on investment (ROI) ratio and hospital bed-days utilized also estimated. Costs are presented in Australian dollars (AUD) and United States dollars (USD), at 2023 prices.

Results

Estimated hospital cost savings for the years 2023 to 2030 from implementing the ESS-MOC were AUD641 million (USD427 million) (95% CI: AUD99 million [USD66 million] to AUD1,250 million) [USD834 million]). This corresponds to a ROI ratio of 8.88 (1.3 to 17.9) dollars returned for each dollar invested in implementing the care model. For the period 2023 to 2030, an estimated 337,000 (261,000 to 412,000) acute surgical ward bed-days, and 721,000 (471,000 to 1,028,000) rehabilitation bed-days could be saved. Total implementation costs for the ESS-MOC were estimated at AUD72 million (USD46 million) over eight years.

Conclusions

Implementation of an ESS-MOC for eligible arthroplasty patients in Australia would generate significant cost and healthcare resource savings. This budget impact analysis demonstrates a best practice approach to comprehensively assessing value, at a national level, of implementing sustainable models of care in high-burden healthcare contexts. Findings are relevant to other settings where hospital stay following joint arthroplasty remains excessively long.
PP43 Budget Impact Analysis Of Next-Generation Sequencing Coverage In Taiwan’s National Health Insurance
Authors: Chang Hui-Ru , Li Ying Huang Grace , Lin Shou-Yu , Lin Shyr-Yi
Published: January 2025

Introduction

The exploration of molecular characteristics has emerged as a prominent trend to advance precision medicine. The utilization of genetic testing to guide therapy is integral to precision medicine. This study aims to investigate the potential patient populations for the reimbursement of next-generation sequencing (NGS) and assess the budget impact from the perspective of Taiwan’s single insurer, the National Health Insurance Administration.

Methods

To comprehend the scope for medicines with companion diagnostics (CDx) involved, we analyze the U.S. Food and Drug Administration-approved/cleared diagnostic tests, conduct a literature review to identify medicines approved by the European Medicines Agency that require a CDx, and identify the medicines with CDx involved covered by the National Health Insurance (NHI) in Taiwan. Subsequently, we explore the potential reimbursement indications for NGS testing and conduct a budget impact analysis to evaluate the expected financial impact for the NHI over a five-year period. Furthermore, sensitivity analyses are conducted to deal with uncertainty.

Results

We have compiled 13 cancer types for which NGS can serve as a companion diagnostic. These encompass non-small-cell lung cancer, colorectal cancer, breast cancer, ovarian cancer, biliary tract cancer, acute myeloid leukemia, acute lymphoblastic leukemia, melanoma, cholangiocarcinoma, prostate cancer, pancreatic cancer, gastrointestinal stromal tumor, and thyroid cancer/medullary thyroid cancer. The implementation of NGS reimbursement in NHI will benefit 25,000 to 30,000 patients undergoing targeted therapies. The projected incremental budget impact ranges from TWD570 million to TWD650 million (USD19 million to USD22 million) over five years.

Conclusions

This study focuses on evaluating the financial impact of incorporating NGS testing into NHI reimbursement for relevant cancer drug indications. The findings can serve as references for the planning of reimbursement policies. However, with the advancement of precision medicine, it is foreseeable that there will be a broader range of applications for NGS, and its cost will gradually decrease.
PP54 Towards Equitable Pricing: Unraveling The Complexities Of External Reference Pricing In Medical Devices
Authors: Tibet Birol , Kilic Ayse Gokcen , Atashi Zahra Ahangar , Kockaya Guvenc
Published: January 2025

Introduction

External reference pricing (ERP) serves as a widely adopted method for establishing authorization and reimbursement prices for pharmaceuticals in Europe. However, its application within the medical device (MD) industry remains shrouded in uncertainty. This study seeks to investigate the feasibility of integrating ERP into the realm of MDs, identify pertinent challenges and ambiguities, and propose solutions to bolster affordability and accessibility.

Methods

The methodology encompassed a systematic search across diverse databases (Google Scholar, Google, ResearchGate, PubMed), utilizing combinations of relevant keywords, like “medical device,” “external reference pricing,” “price,” “reimbursement,” “affordability,” “challenge,” and “implement.” Articles were thoroughly evaluated by each researcher independently, followed by a consensus-based approach for final assessment. Rigorous scrutiny of relevance, potential insights, study design, and methodology ensured a comprehensive review, maintaining a high standard of research quality. This meticulous process guaranteed the inclusion of pertinent literature on ERP and MD pricing, enhancing the study’s depth and breadth.

Results

Global ERP application in the MD sector exhibits significant variability in country selection, pricing methods, and adaptations to market changes. Pricing and reimbursement policies across international contexts contribute to this diversity, involving authorization procedures, regulatory oversight, technology appraisals, and state financing limitations. Challenges in establishing equitable pricing frameworks underscore the need for clear guidelines, regulatory empowerment, regular audits, and international collaborations. Transparent methodologies for reference price calculation are essential. Addressing observed shortcomings involves negotiating lower prices, implementing subsidies, promoting generic competition and local production, and tailoring reimbursement policies to treatment needs, enhancing affordability and accessibility on a global scale.

Conclusions

Ambiguities in implementing ERP for MDs underscore the necessity of equitable pricing frameworks. The highlighted multidimensional strategies and consideration of market dynamics are deemed essential for the feasibility of ERP in MDs. These insights emphasize the cruciality of a structured and transparent roadmap for optimizing ERP in the realm of MDs, holding significant potential for ensuring affordability and accessibility.