bluuebunny/crossref_metadata_2025 huggingface dataset:
OP14 Cost-Utility Analysis Of Regorafenib For Patients With Hepatocellular Carcinoma Who Progressed On Sorafenib Treatment
Authors: Singh Ambrish , Hussain Salman
Published: December 2023

Introduction

In the RESORCE trial, regorafenib was shown to provide overall survival (OS) benefit for patients with hepatocellular carcinoma (HCC) that has progressed on sorafenib treatment. Subsequently, it was approved by the Therapeutic Goods Administration for the treatment of patients with HCC who were previously treated with sorafenib; however, regorafenib is still not recommended by the Pharmaceutical Benefits Advisory Committee in Australia. We aimed to assess the cost effectiveness of regorafenib as a second-line therapy for patients with HCC who progressed on sorafenib from an Australian healthcare perspective.

Methods

We developed a Markov model to compare the cost effectiveness of regorafenib with best supportive care (BSC) as a second-line therapy for HCC after treatment with sorafenib. The health outcomes of life-years and quality-adjusted life-years (QALYs) were derived from the RESORCE trial. Survival benefits sourced from the RESORCE trial were fitted with the parametric model to estimate survival beyond the follow-up period. Drug costs and costs associated with adverse events (AEs) were sourced from published literature and the Independent Health and Aged Care Pricing Authority cost report. Model validity was verified using probabilistic sensitivity analyses.

Results

The incremental monthly cost of treatment with regorafenib was AUD19,273 (USD13,374), with an incremental life-year gain of 0.38, compared with BSC. The incremental QALYs gained with regorafenib were 0.24, resulting in a base-case incremental cost-effectiveness ratio (ICER) of AUD80,511 (USD55,872) per QALY. In the probabilistic sensitivity analyses across scenarios, the ICER remained above the conventional threshold of AUD50,000 (USD34,698) per QALY, with a zero probability of being cost effective at this willingness-to-pay threshold.

Conclusions

At the current price, second-line treatment with regorafenib in patients with HCC that has progressed on sorafenib was not cost effective at the conventional willingness-to-pay threshold from an Australian health-system perspective.
OP19 Exploring The Environment/Capacity Of South African Citizen Actors To Contribute To Health Technology Assessment Processes, Policy Development And Institutionalization
Authors: Pretorius Lauren , Muller Debjani
Published: December 2023

Introduction

Several overarching health policy reform processes are currently underway in South Africa (SA), providing an opportunity to establish health technology assessment (HTA) and value-based assessment (VBA) frameworks that foster patient and citizen involvement (PCI). A mapping of the capacity, knowledge, and skill of SA PCI advocacy actors and understanding of the ‘middle-ground’ and influencing relationships that influence advocacy strategies for PCI in HTA, will allow us to determine the needs of PCI actors to entrench PCI principles in the emerging institutionalization of HTA in SA.

Methods

An analysis of national and international legislative and policy frameworks indicates current gaps and opportunities for PCI institutionalization in HTA in SA. A survey was conducted to determine SA patient and citizen advocacy actors’ capacity, knowledge, and skill across multiple disease areas. An analysis of decision maker’s opinions and positions about PCI in HTA and VBA policy, and their potential influence on the PCI process was undertaken.

Results

The legislation and policy review indicate that engagement initiatives are positioned at the ‘involvement’ or ‘consultation’ stages of the engagement continuum, rather than higher-level engagement. Five percent of patient advocacy groups (PAGs) interviewed have formalized PCI HTA advocacy strategies. Few PAGs indicated employing processes to actively monitor the HTA and PCI-related activities of decision-makers.

The majority of PAGs stated that collaborative efforts within larger networks would generate more success, if they engaged in PCI in HTA advocacy. Over eighty percent of civil society stakeholders face capacity constraints, such as lack of knowledge of the legislative framework and theory of HTA, funding and manpower to engage in PCI. The majority of HTA processes undertaken by funders in SA do not actively include PAGs or formalized PCI.

Conclusions

Existing legislative and policy frameworks do not include PCI capacity-building strategies. This is impacted by the lack of coordination amongst patient and consumer groups, the willingness of existing HTA structures to formalize PCI, and the resources of the country’s PCI advocate actors to influence existing HTA processes.
OP52 Treating Osteoporosis In Postmenopausal Women With Denosumab (Prolia®): A Systematic Review And Network Meta-Analysis
Authors: Moshi Magdalena Ruth , Nicolopoulos Konstance , Stringer Danielle , Ma Ning , Jenal Mathias , Vreugdenburg Thomas
Published: December 2023

Introduction

It is estimated that osteoporosis affects over 200 million people globally. Postmenopausal women (PMW) have an increased risk of developing osteoporosis due to low estrogen levels. This study assessed the safety and effectiveness of denosumab (Prolia®) relative to placebo, selective estrogen receptor modulators (SERMs) (bazedoxifene and raloxifene), and bisphosphonates (alendronate, ibandronate, risedronate, and zoledronate) for the treatment of osteoporosis in PMW.

Methods

Systematic searches were conducted in three biomedical databases (PubMed, the Cochrane Library, and Embase) to identify randomized controlled trials (RCTs) of PMW with osteoporosis allocated to denosumab, placebo, bisphosphonates, or SERMs. The Cochrane Risk of Bias 2.0 tool was used to critically appraise included RCTs. Pairwise and Bayesian network meta-analyses were performed on the following predetermined outcomes: fractures (vertebral and nonvertebral); adverse events (AEs); mortality; serious AEs (SAEs); withdrawals due to AEs; bone mineral density (BMD); and AEs caused by denosumab discontinuation.

Results

The analyses included 12 RCTs (22 publications, 25,879 participants). Denosumab, ibandronate, alendronate, zoledronate, and risedronate produced a statistically significant improvement in total hip (TH) and lumbar-spine (LS) BMD, compared with placebo. Similarly, ibandronate, risedronate, and alendronate showed a statistically significant improvement in femoral neck (FN) BMD. Risedronate produced a statistically significant decrease in nonvertebral fractures (risk ratio 0.20, 95% confidence interval: 0.00, 0.97) relative to placebo. However, there were no significant differences between any of the interventions for rates of vertebral fractures, AEs, SAEs, withdrawals due to AEs, or mortality, compared with placebo.

None of the included trials reported evidence on AEs caused by denosumab discontinuation.

Conclusions

Denosumab was associated with significant improvements in both LS and TH BMD relative to placebo. Similarly, compared with placebo, denosumab was not associated with significant changes in nonvertebral or vertebral fractures. Denosumab was not associated with significant changes in safety outcomes relative to placebo. Given that some of the analyses suffered from statistical imprecision, these findings should be interpreted with caution. Regarding policy, continued funding of denosumab needs to be reviewed.
OP70 Treating Patients With Hormone-Sensitive Cancer On Endocrine Therapy With Denosumab (Prolia®): A Systematic Review And Network Meta-Analysis
Authors: Nicolopoulos Konstance , Moshi Magdalena Ruth , Stringer Danielle , Ma Ning , Jenal Mathias , Vreugdenburg Thomas
Published: December 2023

Introduction

Patients receiving endocrine therapy for hormone-sensitive cancers, such as men with prostate cancer (MPC) on hormone ablation therapy (HAT) and women with breast cancer (WBC) on adjuvant aromatase inhibitor therapy (AAIT), have an increased risk of developing osteoporosis. The aim of this study was to compare the safety and effectiveness of denosumab (Prolia®) with selective estrogen receptor modulators (SERMs) (raloxifene and bazedoxifene), bisphosphonates (zoledronate, ibandronate, alendronate, and risedronate), and placebo for the treatment of osteoporosis in patients receiving endocrine therapy for hormone-sensitive cancer.

Methods

Systematic literature searches were conducted in three biomedical databases (PubMed, the Cochrane Library, and Embase) to identify randomized controlled trials (RCTs). Only RCTs that investigated MPC on HAT or WBC on AAIT allocated to denosumab, SERMs, bisphosphonates, or placebo were included. RCTs were appraised using the Cochrane Risk of Bias 2.0 tool. Frequentist network and pairwise meta-analyses were performed on predetermined outcomes of vertebral or nonvertebral fractures, treatment-related adverse events (AEs), bone mineral density (BMD), mortality, withdrawal due to treatment-related AEs, and serious AEs.

Results

A total of 14 RCTs (15 publications, 6,463 participants) were included. Relative to placebo, denosumab was found to prevent vertebral fractures in cancer patients receiving endocrine therapy. Moreover, denosumab, alendronate, and zoledronate increased femoral neck (FN) and lumbar spine (LS) BMD in MPC on HAT, compared with placebo, whereas denosumab, risedronate, and ibandronate improved LS and total hip BMD in WBC on AAIT. Similarly, denosumab and risedronate increased trochanteric BMD in WBC on AAIT, compared with placebo. In WBC on AAIT, only denosumab increased FN BMD relative to placebo.

Conclusions

Denosumab was more effective than placebo in preventing vertebral fractures and improving BMD at the LS and FN in MPC on HAT, and in preventing vertebral fractures and improving FN, trochanteric, total hip, and LS BMD in WBC on AAIT. From a policy perspective, the continued reimbursement of denosumab needs to be reviewed.
OP79 Gene Expression Profiling In The Diagnosis Of Aggressive Large B Cell Lymphoma: An Early Exploratory Economic Evaluation
Authors: Bouttell Janet , Hawkins Neil , Masson Rachel , Goodlad John
Published: December 2023

Introduction

The addition of gene expression profiles (GEP) to the current clinicopathological diagnosis of aggressive large B cell lymphomas may lead to the reclassification of patients, treatment changes and improved outcomes. A GEP test is in development using TempoSeq technology to distinguish Burkitt Lymphoma (BL) and Primary Mediastinal Large B Cell lymphoma (PMBCL) from Diffuse Large B Cell Lymphoma (DLBCL). This study aims to inform developers about the potential impact of the test on costs and health outcomes, and pricing and evidence generation strategies.

Methods

Decision models compared current diagnosis with current plus GEP signatures over a lifetime horizon using a UK health and social care perspective. Inputs were taken from the literature and based on assumptions. Threshold estimates were made of the maximum price of the test and impact of incorrect disease classification using a threshold of GDP30,000 (USD37,155) per Quality Adjusted Life year (QALY). One way sensitivity analysis was conducted.

Results

At base case values the BL signature delivers incremental QALYs of 0.0249 at an additional cost per patient of GBP508 (USD629). This results in a net monetary benefit (NMB) of GBP239 (USD296). The PMBCL signature delivers 0.0011 QALYs, a cost saving of GBP202 (USD250) and an NMB of GBP236 (USD292). The maximum threshold price for a combined test to be cost effective is GBP776 (USD961) (base case GBP400 (USD495)). Results are sensitive to cost differences in first line treatments and impact of false diagnoses.

Conclusions

A combined test could be cost-effective in a UK context at a price around GBP750 (USD929). The developers can use this estimate to inform return on investment calculations. The number of patients who were reclassified as a result of the addition of GEP in our model was taken from small retrospective studies and the impact of false diagnoses was based on limited evidence. If the developers choose to proceed with the development, these aspects should be incorporated in evidence generation strategies.
OP82 Positron Emission Tomography Combined With Computed Tomography Using 18F-Sodium Fluoride
Authors: Aimanova Tansolpan , Akhmetova Makpal , Okesh Makhabbat , Avdeyev Andrey , Shanazarov Nasrulla , Albayev Rustam , Saduakassova Aigul , Lyugay Yekaterina , Tleulessova Indira , Turar Olzas , Bariyeva Gulzada
Published: December 2023

Introduction

Positron emission tomography combined with computed tomography (PET/CT) using 18F-sodium fluoride (18F-NaF) is used for functional imaging in diseases to detect abnormally altered osteogenic activity, such as benign and malignant bone diseases and inflammatory or traumatic changes in skeletal bones.

Methods

A systematic search of literature using keywords in the MEDLINE database was conducted to identify literature on the clinical and cost effectiveness of using PET/CT with 18F-NaF-based radiopharmaceuticals in the diagnosis of bone and cartilage cancer. The search retrieved 323 publications. The analysis included 11 publications that met the selection criteria, including one meta-analysis and ten literature reviews.

Results

The pooled sensitivity, specificity, diagnostic odds ratio (DOR) and area under the receiver operating characteristic curve of 18F-NaF-based PET/CT for the detection of bone metastases were 0.98 (95% confidence interval [CI]: 0.95, 0.99), 0.90 (95% CI: 0.86, 0.93), 123.2, and 0.97, respectively. 18F-NaF-based PET/CT was highly effective in detecting bone metastases during staging and restaging of patients with high-risk prostate cancer. The effectiveness of 18F-NaF-based PET/CT was superior to bone scintigraphy with technetium-99m and single-photon emission computed tomography (SPECT) and was comparable to diffusion-weighted imaging.

Conclusions

PET/CT with 18F-NaF is a more accurate method of localizing and characterizing malignant bone lesions than SPECT. This method has improved clinical accuracy and provides greater convenience for patients and referring physicians. PET/CT with 18F-NaF in malignant neoplasms is a more specific, sensitive study than 18F-fluorodeoxyglucose PET/CT. These results were submitted to the Ministry of Health for a decision on the inclusion of 18F-NaF-based PET/CT in the state reimbursement system.
OP90 Multiple Myeloma: Developing A Benchmark Patient Experience Index In Australia And New Zealand
Authors: Fifer Simon , West Bronwyn , Godsell Jenni , Combe Nella , Stephenson Todd
Published: December 2023

Introduction

Within Australia and New Zealand (ANZ) there is limited evidence regarding the experience and satisfaction across the healthcare system of people living with multiple myeloma (MM). We aimed to quantify the patient experience across the healthcare system to help identify potential areas of the healthcare pathway that could be targeted for improvement to maximize patient satisfaction.

Methods

A 30-
to 40-minute online survey was completed by adults in ANZ diagnosed with MM. Anchored best-worst scaling (ABWS) is a technique that takes advantage of an individual’s ability to reliably identify extremes (‘best’ and ‘worst’) in sets of items, eliciting discriminating rankings free of scale bias. This study implemented a novel anchoring process to rescale importance and satisfaction best-worst scores for factors across the MM healthcare pathway, which could be compared and combined to form a patient experience index (PEI). There were 15 factors or ‘moments that matter’ (MTM), each describing a different aspect of the patient journey, such as time to diagnosis, treatment logistics, and side effects. The MTM were derived from qualitative research with patients as well as a workshop with key opinion leaders. Additional survey questions were included to help identify potential ways to improve patient satisfaction.

Results

The results were based on 62 patients with MM. The overall median PEI score was 63.1. The top three MTM that were most important to patients, but they were least satisfied with (calculated by combining the top four of each most important/least satisfied factor for each participant), were side effects of medication, effectiveness of medication, and medication access.

Conclusions

The findings from this research contribute to the understanding of patient experiences of treatment and care for MM. The results can inform healthcare decisions for prioritizing interventions that align with patient experiences. In the future, the study could be executed longitudinally to assess shifts in satisfaction within the MM healthcare journey, which would be especially worthwhile if new programs are implemented to improve patient satisfaction.
OP93 The Cost Effectiveness Of Antiretroviral Therapy Adherence Interventions In HIV/AIDS Patients: A Systematic Review Of Decision Analytic Models
Authors: Ahmed Ali , Dujaili Juman , Le Long , Khanal Saval , Chuah Lay Hong
Published: December 2023

Introduction

People living with HIV/AIDS (PLWHA) frequently struggle to maintain optimal adherence to antiretrovirals (ARVs). Different adherence-improving interventions have been developed and examined through decision analytic model-based health technology assessments. Therefore, we aimed to conduct a systematic review of all decision analytic models developed to improve adherence in PLWHA.

Methods

The review protocol was registered on Prospero (CRD42022270039) and relevant studies published from inception to 23 October 2022 were identified through searches of the following databases: PubMed, Embase, the National Health Service Economic Evaluation Database, PsycINFO, the Health Economic Evaluations Database, and EconLit. Studies were included if they were modeling works of full economic evaluations, including cost-effectiveness analyses (CEA), cost-utility analyses, cost-benefit analyses, and cost-consequence analyses. The primary outcome was the cost effectiveness of adherence interventions reported as the incremental cost per additional quality-adjusted life-year (QALY). Study quality was assessed with the Quality of Health Economics Studies instrument. Due to the heterogeneity of the data, a permutation matrix was used for quantitative data synthesis rather than a meta-analysis.

Results

The 15 studies identified were conducted in North America (8/15), Africa (4/15), and Europe (3/15). The time horizon was one year in one study, ten years in one study, 20 years in three studies, and a lifetime horizon in ten studies. The types of interventions were smartphone-based (5/15), nurse involved (2/15), directly observed therapy (2/15), case manager involved (1/15), simplification of regimens (1/15), Link4Health (1/15), and others (3/15) that involved multicomponent intervention. The interventions gained higher QALYs with cost savings in all 15 studies and gained QALYs at a higher cost at an acceptable incremental cost-effectiveness ratio in 80 percent (12/15) of studies. The studies were of fair (13%) to high quality (87%).

Conclusions

This study is the first systematic review of decision analytic model-based CEAs of adherence interventions in the management of PLWHA. Most of the identified studies recently published good quality cost-effectiveness analyses with an adequate timeframe.
OP94 Economic Evidence On Hemodialysis Access Creation Procedures In Patients With End-Stage Kidney Disease: A Systematic Literature Review
Authors: Gupta Ritu , Gaba Upasna , Delaney Christopher , Papadopoulos George
Published: December 2023

Introduction

It is important to create and maintain durable hemodialysis (HD) access in health systems to reduce morbidity and maintain overall cost control in patients with end-stage kidney disease (ESKD). To evaluate the choice of HD vascular access creation procedures and their related economic costs, we aimed to identify economic evaluations on vascular access (VA) creation procedures in patients with ESKD.

Methods

A systematic literature review was conducted using the Cochrane methodology to identify cost-effectiveness analyses (CEAs), budget impact analyses, and cost analyses of various HD access creation procedures. Eligible publications published from 2012 onwards were retrieved by searching PubMed, Embase, and the Cochrane Library. The Consolidated Health Economic Evaluation Reporting Standards 2022 checklist and ISPOR Task Force guidelines were used to appraise the quality of the economic evaluations and budget impact analyses, respectively. Costs were adjusted for inflation and purchasing power parity and standardized to US dollars.

Results

A total of 40 economic evaluations met the inclusion criteria, including 28 cost analyses, three budget impact analyses, and nine CEAs. Widely evaluated procedures in the published literature were endovascular and surgical arteriovenous fistula (AVF), arteriovenous graft (AVG), and central venous catheterization (CVC). The results indicated that AVF was the most cost-effective strategy, followed by AVG, and CVC. Three studies showed that endovascular AVF was cost effective, compared with surgical AVF, and resulted in overall cost savings of about USD53 million dollars over a five-year period. Results of the quality assessment showed that budget impact analyses scored 63 percent, while the average score for economic evaluations was 58 percent.

Conclusions

It was challenging to identify a single effective method of managing vascular access due to the substantial heterogeneity among VA creation techniques. However, most of the included economic evaluations showed that AVF was a cost-effective method of VA creation relative to other identified techniques for patients with ESKD on HD.
OP100 Patient Perspectives In Value Assessment Frameworks: The Asia Pacific Perspective
Authors: Best Alex , Tsai I-Ching , Tan Jin Yu , Wu David bin-chia , Yu DaeYoung , Keetley Alison , Wong-Rieger Durhane , Jain Ritu
Published: December 2023

Introduction

The importance of patient centricity in healthcare decision making has been recognized and advocated for decades. However, approaches for including the patient perspective are diverse, and progress varies among countries. Some reimbursement bodies acknowledge the importance of patient preferences in health technology assessment (HTA) and funding decision processes. However, patients’ perspectives are not yet systematically and transparently included in value assessment frameworks globally, and even less so in the Asia-Pacific (APAC) region. This systematic review aimed to investigate how patients’ perspectives are used to inform pricing and reimbursement decisions in the APAC region.

Methods

A systematic review is ongoing that utilized a search of 12 databases, including MEDLINE and Embase, to identify publications on the consideration of patient perspectives in health policy decision-making published to November 2022. Conference abstracts published in the last five years from ISPOR and Health Technology Assessment International (HTAi) were screened, along with gray literature and government websites from Australia, China, Japan, Malaysia, New Zealand, the Philippines, Singapore, South Korea, Taiwan, and Thailand. Publications were included if the impact of either one or more of the following on HTA decision-making was assessed: active participation of patients or patient advocacy groups; type, extent, and evolution of patient-reported outcomes; health-related quality of life or quality of life tools; and themes where the impact of patients’ perspectives on value assessment was the primary outcome. Countries were characterized into archetypes based on similarities or differences in the weight and value assigned to patient perspectives in decision-making.

Results

A total of 6,438 retrieved citations will undergo the systematic review process. Additionally, 758 conference abstracts from ISPOR, 1,312 from HTAi conferences and 73 records from gray literature will be screened.

The results of the systematic review will be consolidated into country archetypes, examples, and learnings. Gaps and opportunities will also be identified.

Conclusions

The research will provide recommendations to increase shared decision-making and support the development of decision-making frameworks that systematically incorporate patients’ perspectives in value assessment across APAC countries.
OP115 Scoping Meta-Review On Methods Used To Assess Artificial Intelligence-Based Medical Devices For Heart Failure
Authors: Di Bidino Rossella , Piaggio Davide , Iadanza Ernesto , Pecchia Leandro , Andellini Martina , Zeeshan Raza , Merino-Barbancho Beatriz , Pérez Laura López , Fico Giuseppe , Ni Melody , Borsci Simone , Zhu Tianhui
Published: December 2023

Introduction

The International Federation of Medical and Biological Engineering created a multidisciplinary working group to discuss assessments of artificial intelligence and machine learning (AI/ML) applications in health care. Engineers, clinicians, and economists identified evidence generation as a critical topic. Heart failure (HF) was selected to investigate the available evidence on the clinical effectiveness and safety of AI/ML applications. Attention was paid to transparency of AI/ML methods and their data sources.

Methods

A scoping review was conducted on AI/ML algorithms developed for the management of HF. A search for systematic reviews, scoping reviews, and meta-analyses published from 1976 to October 2022 was conducted in Embase, MEDLINE, and Scopus.

Results

Of 456 relevant publications, 21 papers were included in the final analysis. Most papers (10 systematic reviews, five meta-analyses, and six non-systematic or scoping reviews) included studies conducted in North America. No study was conducted in Africa. The healthcare setting was not clearly stated in approximately half of the studies. A lack of agreement was noticed regarding the quality assessment tools used among the reviews. The most common data source for AI/ML algorithms was electronic health records, but in some cases data sources were not reported. While deep learning emerged as the most common adopted methodology, covariates were not always included in the algorithm development. The review demonstrated that comparative assessment of algorithms requires further investigation, given the high variability in the comparator used (e.g., clinical gold standard, other AI/ML algorithms, or other statistical methods). The main investigated endpoints were the incidence of HF and the number of hospital admissions.

Conclusions

When assessing innovative health technologies such as AI/ML applications in health care, evidence is among the main challenges. Our scoping review, focusing on algorithms developed to manage HF, showed that the biggest challenges relate to the quality of the studies, the adoption of a comparative approach, and transparency of methods.
OP128 Uncertainties In The Cost-Effectiveness Analysis Of Onasemnogene Abeparvovec For Spinal Muscular Atrophy Type 1
Authors: Borges Stefani , Fernandes Brígida , Rodrigues Fernanda , Cirilo Hérica Núbia , Krug Bárbara , Probst Livia , Zimmermann Ivan
Published: December 2023

Introduction

Nusinersen and risdiplam are available in the Brazilian Unified Health System (SUS) for the treatment of spinal muscular atrophy (SMA) type 1. Onasemnogene abeparvovec, a promising gene therapy, was approved in 2020 in Brazil. Given the high cost of this therapy and its promise of a lifetime effect, the objective of this study was to evaluate the cost effectiveness of onasemnogene abeparvovec, compared with nusinersen and risdiplam, in the treatment of SMA type 1 from the perspective of SUS in different scenarios.

Methods

A Markov model was adapted from one originally developed for the USA that considers five states of health. Short-term data were obtained from pivotal clinical trials and long-term survival curves were extracted from published reports from the USA. Maintenance of motor function milestones achieved at the end of follow up in clinical trials was considered until death. Costs and quality-adjusted life-years (QALYs) were discounted at five percent per year over a baseline lifetime time horizon. Alternative scenarios were evaluated for horizons of five and ten years, with and without a discount.

Results

Onasemnogene abeparvovec resulted in an incremental cost of BRL742,890 (USD297,156) per QALY and an increase of 3.32 QALYs in relation to the alternatives over a lifetime time horizon. In the same time horizon, but without the discount, onasemnogene abeparvovec resulted in an incremental cost-effectiveness ratio (ICER) of BRL166,539 (USD66,615) per QALY. In a five-year time horizon, considering the discount rate, the therapy resulted in an ICER of BRL12,527,667 (USD5,011,066); in ten years the ICER was BRL3,384,793 (USD1,353,917).

Conclusions

Since the benefits of onasemnogene abeparvovec mainly occur in the long term, decision makers need to consider the uncertainty of assumptions of sustained effectiveness in view of the high initial cost of the technology.
OP149 How To Improve The Impact Of Health Technology Assessment: Stakeholders’ Perspectives In Spain
Authors: Triñanes Yolanda , Gómez Patricia , Faraldo-Vallés María J , Pérez-Ríos Mónica , Ruano-Raviña Alberto , lema Leonor Varela
Published: December 2023

Introduction

Health technology assessment (HTA) agencies in Spain have an important role in informing decisions about the introduction and use of health technologies in the Spanish National Health System. However, although different approaches have been taken to measure and improve their impact, no study to date has explored the perceived impact of HTA products at the national level. The aim of this study was to explore the perspectives of macro-, meso-, and micro-level decision makers on how to improve the impact of HTA.

Methods

Three online focus groups were conducted with policy makers, healthcare managers, clinicians, and patients. The transcripts were evaluated using a deductive thematic analysis based on a multidimensional framework to explore mechanisms of impact.

Results

Four key themes were identified:

(i)

Timeliness and use of HTA assessments: Although the quality of the reports was recognized, the time taken for the elaboration and extension of reports negatively affected their use. Participants considered that reports should be tailored to the needs of end users (e.g., briefer versions available for meso-and micro-level use);

(ii)

Effective engagement and external communications: The engagement of multiple stakeholders (policy makers, manufacturers, clinicians, and patients) in the elaboration process was considered crucial to improve HTA impact and ensure adequate communication of results;

(iii)

Good institutional reputation and fit within the healthcare and policy making system: Stakeholders agreed on the need to strengthen collaboration at the national level and increase public understanding of the value of HTA and its use in healthcare decision-making; and

(iv)

Effective implementation of policy change regarding health technologies: Stakeholders were very receptive to the results and recommendations of HTA reports when new technologies are demanded, but the identification and selection process should be improved to guarantee that these reports are available on time.

Conclusions

This study has identified different proposals and mechanisms that could improve the impact of HTA in Spain.
OP165 Changing Decision Criteria: An Analysis Of Health Technology Assessment Reports To The Brazilian Unified Health System
Authors: Borges Stefani , Rodrigues Fernanda , Pires Wolney , da Costa Nathalia , Louly Priscila , Prado Clementina , Santos Vania
Published: December 2023

Introduction

In Brazil, the National Committee for Health Technology Incorporation of the Brazilian Public Health System (Conitec) defines the criteria for incorporating technologies into the public health system. However, all recommendations are submitted to public consultations to ensure transparency and public participation.

Contributions from the public consultation are analyzed and entered into Conitec’s final report and sometimes change the preliminary recommendation.

Methods

This descriptive, retrospective exploratory study aimed to gather qualitative and quantitative data on criteria considered by healthcare decision makers from Conitec and to identify which criteria most changed after reports were submitted to public consultation. Data from 2012 to November 2022 were collected from the Conitec website, entered into a specific extraction form, and analyzed using descriptive statistics.

Results

Medicines (77%) were the most frequently evaluated type of technology, followed by procedures (15%), and products (8%). A total of 763 recommendations were analyzed. Of these, 70 percent did not change the decision criteria from the preliminary to the final published report, 9 percent did change, and 20 percent were simplified analyses of technologies of relevant public interest that were of low cost and low budget impact (these were not submitted to public consultation or public hearing). As pharmaceutical companies usually propose cost reductions or discounts during the Conitec public consultations, most of the recommendations (45%) were modified in the final report, with incremental budgetary reduction being the main criterion. This study identified that most of the changes occurred in neurology and were related to the high incremental budgetary impact of technologies for the care of rare diseases like spinal muscular atrophy.

Conclusions

Although different criteria are considered for Conitec recommendations, some of these are regarded as crucial for decision-making. Unfortunately, as there is no definition for the weight of each of these criteria, it is difficult to understand their influence on the recommendations made. Therefore, it is crucial to establish standardized criteria for proper decision-making.
OP171 Canadian Disease Registry Inventory: Environmental Scan Of The Literature
Authors: Hodgson Amanda , Loshak Hannah , Lachaud James , Bernard Amélie , Slade Teri , Mák Geneviève , Fowler Heather Smith
Published: December 2023

Introduction

In consideration of the lessons learned from other jurisdictions and other ongoing work in the disease registry data space, an opportunity existed to investigate the current Canadian landscape and identify opportunities for a Canadian registry list. Previously, no national-level inventory of registries existed in Canada that could provide the necessary information to support awareness and use of available data for decision-making.

Methods

A literature search was conducted on key resources, including MEDLINE and a focused internet scan. No methodological filters were applied to limit retrieval by publication type. The search was limited to documents published in English or French.

Results

Core characteristics of the identified registries were extracted and contextual information on the current landscape of disease registries in Canada was explored. A literature review and draft inventory list has been produced.

Conclusions

A CADTH environmental scan was undertaken to collect and report on existing Canadian disease registries and to identify key features, characteristics, and intersections. This information and analysis increase the potential of Canadian registries to inform decision-making and identifies opportunities for the optimal use of registry data in Canada more broadly.
PP11 Patient Involvement In Drug Evaluations To Inform Funding Decisions: A Singapore Case Study
Authors: Tan Ping-Tee , Pearce Fiona , Quek Shawn , Teo Sok Huang
Published: December 2023

Introduction

Patient involvement has become increasingly integral to health technology assessment (HTA) processes globally due to greater recognition of the important contribution patients make to address uncertainties in the scientific evidence base and interpret results for real-world implementation. To align with best practices and encourage meaningful patient input in HTAs in Singapore, patient involvement processes were established by the Agency for Care Effectiveness (ACE) in 2021. This presentation discusses how patient groups were identified in Singapore and describes the new patient involvement processes.

Methods

In the absence of a centralized database, a stakeholder mapping exercise was undertaken in 2021 to identify all relevant patient and volunteer groups in Singapore. A comprehensive search of the Singapore Charity Portal, hospital websites, standard search engines and social media platforms was conducted.

Identified groups were screened in line with specific inclusion criteria and contacted via email and cold calling to find out more about their remits. Plain English resources, targeted training materials and a process guide to encourage patient involvement in ACE’s work were co-developed with local patient organizations by drawing upon best practices from overseas HTA agencies contextualized to local patients’ needs. Supporting resources and processes were revised in 2022 based on ACE’s experience receiving inputs from patients and caregivers to inform drug HTAs.

Results

One hundred and six patient groups covering 20 conditions were identified including registered organizations and informal support groups. In the first half of 2022, ACE received responses from 82 patients from ten patient organizations to inform seven drug HTAs for cancer, diabetes, HIV, and other conditions in line with the new patient involvement processes. Patient organizations viewed the opportunity to submit testimonials of their lived experience with different conditions as a meaningful and important activity for their members.

Conclusions

Patient involvement processes have improved the legitimacy and acceptance of ACE’s work and will be continuously revised to ensure that they remain relevant and meet patients’ expectations and needs.
PP14 The Role Of Health Technology Assessment In The Public Hospitals Of The National Health Service Of Catalonia
Authors: Mestre-Lleixà Berta , Llinàs-Mallol Laura , Gallastegui-Calvache Edurne , Ramos-Masdeu Laia , Ruiz-Baena Jessica , Vivanco-Hidalgo Rosa Maria
Published: December 2023

Introduction

Hospitals play an essential role to facilitate the appropriate introduction of health technologies into the National Health System. We aimed to analyze if the decision-making at the hospital level is based on health technology assessment (HTA).

Methods

We developed a seventeen-question survey to assess the decision-making mechanisms for the introduction of health innovation into the public hospitals of the National Health Service (NHS) of Catalonia. All questions were related to the field of HTA and health innovation. We asked about the process of new health technology acquisition, the existence of HTA units, and the impact those units might have within the center. We invited the three main public healthcare providers (n= 68 centers). We performed a quantitative analysis grouping the results by type of healthcare provider.

Results

Thirty-five different health providers (51.5%) remitted forty-two responses. We identified two differential mechanisms for the introduction of health innovation in those centers: 51.4 percent used a directive committee for decision-making, whereas 37.1 percent employed a technical commission. Some centers (8.6%) used both options. Both the directive committees and technical commissions predominantly considered the professional experience or scientific evidence provided by the healthcare professionals of the center (80%) or external HTA reports or clinical practice guidelines (65.7%) to guide their decision-making. Using HTA products developed within the center was less frequent (34.3%). Only 57.1 percent of the healthcare providers had an HTA unit, but 75 percent of them declared that HTA products had a direct and quantifiable impact on their decision-making regarding the introduction of innovation. Healthcare providers without HTA units manifested their willingness to adopt HTA practices into their decision-making processes.

Conclusions

HTA at the hospital level is not a common practice when deciding to introduce innovative health technologies in the NHS of Catalonia. However, the main healthcare providers agreed that the introduction of HTA is necessary for improving their decision-making and that the HTA agency, Agency for Health Quality and Assessment of Catalonia (AQuAS) will play a key role (training, promoting, and assessing).
PP38 Designing A Training And Capacity Building Pathway In Patient Involvement
Authors: Triñanes Yolanda , Faraldo-Vallés María J. , Gómez Patricia , Cantero-Muñoz Paula , Vicente-Edo María J. , Reviriego-Rodrigo Eva , Novella Blanca , Toledo-Chávarri Ana
Published: December 2023

Introduction

The Patient Involvement (PI) Interest Group of the Spanish Network of Health Technology Assessment Agencies (RedETS) was set up in 2017 by a group of health technology assessment (HTA) researchers interested in PI. Since its inception, training and capacity-building to support PI and patient-based evidence in HTA processes has been one of its main aims. The objective of this work was to identify the needs and priorities related to training and capacity building activities to be developed within the framework of the PI Interest Group.

Methods

The PI Interest Group met on November 14, 2022, for its Annual Meeting. The group discussed the needs, priorities and possibilities on training, and carried out a prioritization exercise. For this purpose, a self-reported and anonymous questionnaire was used, which included 16 training activities. Every item was scored with a Likert-type scale ranging from 0 to 10.

Results

The questionnaire was answered by twenty participants. The most highly rated training activities (mean less than or equal to 8) were: qualitative evidence synthesis (8.75); PI case studies (basic (8.65) and advanced (8.56) level); quality assessment tools for qualitative evidence (8.37); and qualitative research (8.11). Other proposals scoring above 7 points were: ethical aspects related to PI, evaluation of patient participation and impact, identification and recruitment procedures, and discrete choice experiments. The group agreed to organize bi-monthly webinars and three structured training activities for the whole RedETS network on: Qualitative Evidence Synthesis, Qualitative Research and PI Case Studies.

Conclusions

The prioritization of training activities according to PI Interest Group members allowed planning a tailored capacity-building program adapted to the needs of RedETS.
PP41 Using Medicare Claims Data To Support Reimbursement Of A Novel Leadless Pacing System For The Management Of Bradycardia
Authors: Makino Koji , Mudge Mia , Zaunmayr Chelsea , Tilden Dom
Published: December 2023

Introduction

The Micra Transcatheter Pacing System (Micra TPS) is a single-chamber transcatheter leadless pacemaker (LPM). LPMs do not require leads or a subcutaneous pocket, which represent the primary sources of device-related complications with conventional transvenous pacemakers (TVPMs). Complications such as infections and lead dislodgements cause significant patient burden, which have significant economic consequences. Running a randomized controlled trial (RCT) to estimate risk differences of infrequent events requires large sample sizes and long follow-up periods. Real-world observational data, while informative, requires an appropriate study design and statistical adjustments to control for potential biases.

Methods

The Micra Coverage with Evidence Development (CED) study was a cohort study of LPM versus TVPM based on US Medicare claims data of 16,431 patients with 2-year follow up (LPM: n=6,219; TVPM: n=10,212). Propensity score matching (PSM) was applied to account for differences in baseline characteristics. As no RCT was identified in the literature, this study was presented to the Australian payer as the primary source of clinical evidence, upon which a cost-utility analysis was conducted.

Results

After PSM, the CED study demonstrated significantly more complications with TVPM versus LPM with adjusted rates of 6.5 percent and 4.6 percent (p<0.001). Significant differences favoring LPM (p<0.01) were observed in device breakdown (1.4% vs 2.0%), dislodgment (0.4% vs 1.2%) and infection (<0.1% vs 0.6%). Based on these findings, a claim of superior safety was accepted by Medical Services Advisory Committee (MSAC) to support reimbursement. In making this decision, MSAC considered that the large sample size and propensity weighting overcame some of the potential biases and the magnitude of the benefit supported cost-effectiveness relative to TVPM.

Conclusions

The lack of a sufficiently powered RCT with an extended follow-up period can mean the impact and benefits of new technologies that reduce clinically important adverse events of relative infrequency are not formally incorporated into payer decision making, particularly where RCTs are a requirement. A well-designed observational study can provide valuable, real-world evidence to support a HTA for reimbursement decisions.
PP50 Early Diagnosis Effect Of Newborns With Critical Congenital Heart Disease Using National Health Insurance Data In South Korea
Authors: Choi Miyoung , Kim Jimin , Park Chanmi , Choi Byung Min , Ha Gisu , Shin Hong Joo , Kim Sujin , Shin Jeonghee , Choi Eui Kyung
Published: December 2023

Introduction

Critical congenital heart disease (CCHD) refers to a group of heart defects that cause serious, life-threatening symptoms in the neonatal period and requires timely surgical or catheter interventions. We tried to explore current status of CCHD burden and the effect of early diagnosis of CCHD to mortality using the Korean national health insurance (NHI) data.

Methods

We analyzed the national health insurance (NHI) data from 2014 to 2018. We identified CCHD patients using the diagnosis codes and intervention codes from the claim data and the prevalence, mortality and medical expenditure of CCHD were analyzed. We linked neonatal data with their mother’s medical claim data and developed retrospective cohort data set for analyzing the effect of early diagnosis to mortality and related outcomes of CCHD treatment.

Results

The annual prevalence of neonatal CCHD in Korea was 0.144% percent. A total of 2,241 CCHD neonates, 1,546 (69.0%) underwent cardiac ultrasound within three days after birth, and mothers of 419 neonates had a record of prenatal fetal ultrasound (18.7%). In our comparison of neonates diagnosed with CCHD within three days of birth with those diagnosed with CCHD on or after day 4 of birth, the probability of early diagnosis increased for preterm infants and infants with low birth rate. Regarding mortality rate, most types of CCHD showed a significantly higher mortality rate in the early diagnosis group.

Conclusions

The reason for the high mortality rate despite a high early diagnosis rate pertains to the high percentage of patients with severe conditions that induce a serious heart rate within three days of birth. More than half of the neonates with CCHD were found to have not undergone a prenatal fetal ultrasound, rendering this an important policy target.