bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP159 Quality Of Evidence For Clinical Benefit Of Cancer Medicines Assessed For Funding In Australia
Authors: Vitry Agnes , Caird Cathy 
Published: December 2023

IntroductionThis study aimed to describe the type of evidence available for and the clinical benefit of cancer medicines assessed for funding in Australia by the Pharmaceutical Benefits Advisory Committee (PBAC). The evidence was assessed with the European Society of Medical Oncology Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS).MethodsAll data on applications submitted to PBAC between 2010 and 2020 were independently extracted in duplicate from PBAC Public Summary Documents available online. Any disagreements were resolved through discussion. ESMO-MCBS ratings were retrieved from the ESMO-MCBS website. Substantial benefit for the ESMO-MCBS was defined as a grade A or B for (neo)adjuvant intent and four or five for palliative intent.ResultsIn the study period, 182 cancer indications for 100 cancer medicines were examined by PBAC, including 124 (68%) for solid tumors (116 in the palliative setting) and 58 (32%) for hematological cancers. A total of 138 (76%) indications were recommended for public funding, 40 (22%) were rejected, and four (2%) were deferred. Randomized controlled trials (RCTs) were the main source of evidence in 154 indications (85%) and single-arm studies in 27 (15%) indications. RCTs were available in 113 (91%) and 41 (71%) of the solid tumor and hematological cancer indications, respectively. In submissions with RCTs, mature overall survival (OS) was reported in 81 (53%) indications. For indications with a statistically significant improvement in OS, the median gain was 3.0 months (range 0.9 to 17.0) for solid tumors and 8.2 months (range 1 to 9.1) for hematological cancers. The ESMO-MCBS score was available for 99 solid tumor indications. For indications in the palliative setting, 47 (52%) had substantial clinical benefit according to ESMO-MCBS v1.1, including 35 (51%) indications recommended by PBAC and six (35%) indications that were rejected.ConclusionsThese results show that only a minority of cancer medicine indications considered by PBAC are supported by a good level of evidence and provide a modest extension of patient survival.
OP162 Making Local Economic Evaluation More Relevant: Using Expert Elicitation To Adjust Published Intervention Effects To Reflect Local Context
Authors: Gray Jodi , Karnon Jonathan 
Published: December 2023

IntroductionExpert elicitation is often used in economic analyses to estimate uncertain or unobserved parameters for decision models. However, it has rarely been used in the context of local decision-making. A pragmatic elicitation process was used during a local economic evaluation to prompt local experts to assess the relevance of the published evidence to their setting, and to adjust the published effect estimates to better reflect the intervention effect expected in their setting.MethodsElicitation was undertaken for two interventions that targeted the prevention of hospital-acquired hypoglycemia. Six clinical experts from within the Southern Adelaide Local Health Network (SALHN) were systematically presented with information on the setting of the published evaluation and their local setting. This included information on the hospital and quality of care, patient characteristics, and the research context. After comparing the settings, the experts were asked to estimate the most realistic, most pessimistic, and most optimistic intervention effects for their local context.ResultsThe local intervention effect was estimated to be smaller than the published estimate for both interventions. For one intervention, this was driven by the lower complexity of the local patient cohort. For the other intervention, it was driven by differences in the scope of implementation, with hospital-wide local implementation expected to reduce staff buy-in relative to the targeted implementation used in the published evaluation. The elicited local intervention effects were used in a cost-consequence analysis to estimate the likely costs and effects of the interventions if they were implemented locally.ConclusionsThe pragmatic elicitation process provides a feasible and acceptable way to assess and transparently adjust the published effect estimates to better reflect the expected intervention effect in the local setting.Including this step in local economic evaluations can increase the relevance of these evaluations to local decision makers. Further development and application of these methods may facilitate greater use of economic evaluation in local settings.
OP174 Health Technology Assessment And Economic Evaluations For A Genomic Strategy In Italy
Authors: Di Brino Eugenio , Antonini Debora , Falasca Giulia , Basile Michele , Rumi Filippo , Cicchetti Americo 
Published: December 2023

IntroductionThe challenge to health systems is sustainability, not only in the economic and financial sense of compatibility of spending with allocated resources, but also in terms of equity in access to services and care, quality, safety, innovation and research, that is, in terms of the effectiveness of the right to health. Some countries, such as Italy, do not have formal health technology assessment (HTA) or other similar processes that take into account views outside the decision maker.MethodsIn the Italian national context, there is currently no single tariff for the reimbursability and pricing of genomic technologies. In fact, although genomic tests have been in clinical practice for many years now, to date they have not yet been included in the LEA (Minimum healthcare provision), especially in view of the fact that a defined and transparent process for updating the Essential Levels of Care has been operational since 2018. With the goal of structuring guidelines for the adaptability of economic evaluations to currently available and developing genomic technologies, a literature review was conducted.ResultsThe literature review showed that there are some methodological and practical issues that need to be carefully considered when designing and conducting economic evaluations of genomic tests. In more detail, five key concepts were identified in order to implement the most comprehensive economic evaluation of the technologies under study: the PICO model, the survey perspective, the costs included in the analysis, the effectiveness analysis, the time horizon, and the discount rate.ConclusionsAdequate definition of these concepts appears to be of paramount importance in view of the fact that genomic testing may have important consequences for future generations as well. For the purpose of sustainability of access of genomic technologies, the use of Budget Impact Analysis (BIA) is recommended in all analysis settings being essential for the regulator to tie access to its available budget capacity.
PP17 Rare Diseases: An Analysis Of Assessments Carried Out By The National Committee For Health Technology Incorporation In Brazilian Public Health System (Conitec)
Authors: Rodrigues Fernanda , Borges Stefani , Pires Wolney , da Costa Nathalia , Louly Priscila , Prado Clementina , Santos Vania 
Published: December 2023

IntroductionThe treatment of rare diseases has been a challenge for the Brazilian Unified Health System. In addition to the high costs of treatments, the characteristics inherent to this type of disease bring weaknesses to the scientific evidence of efficacy and safety. The National Committee for Health Technology Incorporation In Brazilian Public Health System (Conitec) is formed by a plenary of experts who monthly assess demands for incorporation into the public health system.MethodsThis exploratory, descriptive, and retrospective study aims to gather qualitative and quantitative data on criteria considered by healthcare decision-makers from Conitec and analyzes which rare diseases were benefited by the commission recommendations. Data from June 2012 to November 2022 were collected from the Conitec website to a specific extraction form and analyzed using descriptive statistics.ResultsA total of 763 technologies were evaluated from June 2012 to November 2022, with 158 being drugs for rare diseases. Among these, those with the highest number of diseases benefited were multiple sclerosis 13.3 percent (n=21), cystic fibrosis 6.3 percent (n=10) and pulmonary hypertension 5.7 percent (n=9). About 70 (44.3%) technologies were incorporated into the Unified Health System to treat rare diseases. In these incorporations, 25 technologies initially had an unfavorable recommendation, and only after the public consultation they were recommended for incorporation. Reasons that contributed to this change in recommendation were the new scientific evidence presented (64%), new negotiation of the drug price (28%), and new budgetary impact by revising the calculation of the target population (8%).ConclusionsThe criteria for evaluating technologies for rare diseases are similar to those adopted for other clinical conditions. However, it is important to adopt specific criteria in analysis of drugs targeted at diseases considered rare for the population. Negotiating prices with industry is an important factor that was highlighted, potentially favoring access to new treatments that can modify the progress of these diseases.
PP21 Are We Ready For It? Developing Criteria To Include Artificial Intelligence Medical Devices (AI-MDs) For Health Technology Assessment
Authors: See-Toh Rachel , Ooi Lydia , Ng Zhen Long , Soon Swee Sung , Ju Hong , Ng Kwong 
Published: December 2023

IntroductionThe increasing pace at which artificial intelligence medical devices (AI-MDs) or digital health technologies (DHTs) have been introduced and integrated in healthcare has not been matched with appropriate selection criteria for health technology assessment (HTA) to inform funding decision-making. To align with international best practice and local regulatory guidance, the Agency for Care Effectiveness (ACE) developed criteria to include AI-MDs as part of its 2022 topic prioritization process for medical technologies. This abstract describes ACE’s approach to develop the inclusion criteria.MethodsTo develop key principles for including AI-MDs in ACE’s topic prioritization process, relevant information from overseas HTA agencies, local regulatory guidelines, and ACE’s existing topic selection criteria were reviewed. A search of international HTA agency websites was conducted in September 2022 to identify relevant information on inclusion of AI-MDs in healthcare for reimbursement recommendations.Additionally, local regulatory guidelines for AI-MDs in healthcare were also identified. The inclusion criteria were then piloted with AI-MDs identified from ACE’s horizon scanning workstream to examine their feasibility for HTA topic selection.ResultsOne overseas framework on DHTs from the National Institute for Health and Care Excellence (NICE) and two local regulatory guidelines were identified. Based on the key finding that the purpose of AI-MD use in guiding clinical management and its associated risks were important considerations, the following criteria were developed: (i) full registration with the regulatory body;(ii) device characteristics should be interventional, have direct impact on patient safety, or support accurate diagnosis or treatment which is critical to avoid death and serious health deterioration; and (iii) the AI algorithm should be fixed as opposed to adaptable as per regulatory requirements. Using this inclusion criteria, eight AI-MDs surfaced from horizon scanning were screened with the above criteria and deemed suitable for HTA topic selection.ConclusionsAs AI technologies are increasingly used to replace or supplement current clinical practice, continuous adaptation of HTA method is needed to ensure appropriate topic selection.
PP30 The Fast Track In Drug Registration By ANVISA – Brazil And Possible Consequences
Authors: Kelles Silvana , Pereira Camila , Martins Carina , Reis Daniel , Azevedo Ernesto , Ribeiro Geraldo , Zocrato Karina , Carvalho Lélia , Freitas Marcela , Horta Maria , Barbosa Mariana , Talim Mariza , Borin Marcus 
Published: December 2023

IntroductionThe fast track in drug registration by the Brazilian Health Regulatory Agency, ANVISA, began in 2017 and is intended to prioritize analysis related to drugs relevant to public health. This process is appropriate in situations where there are no therapeutic alternative available or for technologies show significant improvement in safety, efficacy, or adherence to treatment.MethodsThe Brazilian public administration has a tool for accessing information called Transparency Portal. Thus, data on the number of drugs approved by fast track between 2018 and 2021 were requested through this tool and evaluated.ResultsThe data received by the Brazilian transparency portal shows that the number of requests for fast track had an increase from one in 2018 to 32 in 2021. There is an important increase of registrations, being drugs with phase II trials and with single-arm clinical trials. With registration based on a phase II trial, these patients are in fact receiving drugs in a context similar to clinical trials, but with funding from the healthcare system. Given that phase II studies are conducted in a limited population, there has been an increase in the registration of drugs with suboptimal efficacy and safety concerns. This scenario of uncertainty leads to non-adherence to treatment and a discrepancy in real-world outcomes in comparison to the clinical trial. About 70 percent of phase II trials, show no benefit, and only 30 percent proceed to later phases. It is noteworthy that about 50 percent of the studies that move on to later phases fail to show benefits. Nowadays the number of drugs approved by the fast track has increased, many probably with phase II studies and no comparator group.ConclusionsGiven the uncertainties in the efficacy and safety of a drug registered via fast track, often based on phase II studies, implementing provisional registration with real-world evaluation of outcomes, and coupled with financing based on risk-sharing agreements, may be a sustainable alternative for health systems.
PP61 Cost-Effectiveness Analysis Of Trastuzumab Deruxtecan Versus Chemotherapy For Previously Treated HER2-Positive Gastric Cancer In Singapore
Authors: Koh Ivan , Tan Ling Eng , Abdul Aziz Mohamed Ismail , Lin Liang , Ng Kwong 
Published: December 2023

IntroductionThe phase two DESTINY-Gastric-01 trial demonstrated that trastuzumab deruxtecan (T-DXd) improved overall survival in Asian patients with human epidermal growth factor receptor 2 (HER2)-positive, advanced gastric or gastroesophageal adenocarcinoma that had progressed following two or more treatments, compared with chemotherapy (irinotecan or paclitaxel monotherapy). Considering the high cost of T-DXd, we assessed the cost-effectiveness of T-DXd versus chemotherapy from the Singapore healthcare system’s perspective.MethodsA partitioned survival model with three health states (progression-free, progressed disease and death) was developed, with a five-year time horizon. Survival curves from DESTINY-Gastric-01 were extrapolated beyond the trial duration using parametric functions. Health state utilities were obtained from published literature and direct costs were sourced from public healthcare institutions in Singapore. Utility decrements for adverse events such as interstitial lung disease was incorporated into the model for the differences in safety profiles. A discount rate of three percent was applied to costs and outcomes. One-way deterministic sensitivity analyses (OWSA) and scenario analyses were conducted to assess parameter and model uncertainties.ResultsTreatment with T-Dxd, compared to chemotherapy, had a high base case incremental cost-effectiveness ratio (ICER) of over SGD450,000 (USD334,900) per quality-adjusted life-year gained. The cost of T-DXd greatly influenced the results according to OWSA. Seventy-three percent of the total costs accrued in the T-DXd arm was due to the cost of the drug, compared to seven percent in the chemotherapy arm. The ICER was also sensitive to the assumptions around extrapolation of the survival curves, but when tested across all scenario analyses, the results remained unfavorable.ConclusionsAt the current cost, T-DXd does not represent good value compared to chemotherapy for previously treated HER2-positive gastric cancer in Singapore. The findings from our cost-effectiveness analysis, alongside other considerations, will be useful to inform policy makers on funding decisions.
PP75 Using Real World Evidence To Support The Reimbursement Of Proton Therapy Across A Broad Range Of Rare Cancers
Authors: Arora Nimita , Tilden Dominic , Penfold Scott 
Published: December 2023

IntroductionThe Australian Bragg Centre for Proton Therapy and Research in Adelaide will be Australia’s first center with the capacity to deliver proton beam therapy (PBT). PBT uses energy from protons to target cancer cells while minimizing damage to surrounding healthy tissue, including vital organs. Compared to X-ray (photon) radiation therapy (PRT), PBT reduces the risk of serious and long-term complications.To improve access to PBT, the South Australian Health and Medical Research Institute (SAHMRI) submitted an application to the Medical Services Advisory Committee (MSAC) including a cost utility analysis (CUA) comparing PBT with PRT to treat specific pediatric/adolescent and young adult (AYA) and rare adult tumors.MethodsA systematic review identified 28 comparative, mostly real-world studies to support the conclusion that PBT has superior safety and non-inferior efficacy to PRT in the requested indications. The key challenge for the CUA was to quantify the cost and quality of life implications of the superior safety profile across a wide range of indications with a limited comparative evidence base. A simple lifetime decision analytic model was developed which modeled the rates, costs and utilities associated with relevant toxicities. The complications of radiotherapy are often chronic and included secondary malignancies, visual impairments, endocrine dysfunction, dysphagia, hearing loss and intellectual disability. Some of these toxicities are only applicable to patients with cranial cancers. Therefore, the event rates applied in the evaluation were adjusted to account for the proportion of patients within each population estimated to have extracranial cancers.ResultsWhen results in the adult and pediatric/AYA populations were weighted across the expected utilization of PBT (34% adults, 66% pediatric/AYA) in each population, PBT was dominant relative to PRT.ConclusionsIn November 2020, MSAC recommended funding PBT in specific populations at high risk of long-term side effects from PRT. To address uncertainties around the evidence base, MSAC further requested the following:•All patients receive comparative photon/proton plans to determine eligibility•A national registry is established for patients treated with PBT.
PP76 From Hospital-based Health Technology Assessment (HTA) To Treatment Decision: What Decisions Actually Result After HTA In A Resource Constrained Environment?
Authors: Fitzgerald Anita , Fevre James Le , Morgan Gina , Streat Stephen , Munn Stephen 
Published: December 2023

IntroductionThe Northern Region Clinical Practice Committee (NRCPC) conducts hospital-based health technology assessments (HTA) to provide advice to hospital managers regarding both the implementation of new technologies and the configuration of existing services. To assist in the comparison of dissimilar health technologies applied across different disciplines and different hospitals, the NRCPC developed a prioritization tool. This abstract reports the use of the tool over the 17-year period that the committee has been in operation.MethodsThe score given to each HTA depends on cost-utility, predicted health improvements and the quality of evidence. In addition to the scoring tool, editorial notes are provided to contextualize the agreed score and to explain the NRCPCs interpretation of the evidence.ResultsMost of the time hospital managers have made decisions concordant with the recommendations of the NRCPC; submissions are recommended to be implemented, declined, or receive interim approval with data collection. The latter often occurs when there are uncertainties about efficacy, but no (or very few) safety concerns, or where there are uncertainties about whether the proposed costs are reproducible in the hospital setting. In these cases, management responses often require submitters to undertake a limited number of cases and collect data for audit over a one-to two-year period. Low-scoring submissions are often declined, whereas high-scoring submissions have not been declined to date. The interim approval (with data collection) strategy has had variable outcomes based on the willingness of the implementing clinicians to collect accurate data about both costs and outcomes. From 2005 to 2022, the NRCPC received 146 submissions. This poster reports graphical representations of the decisions made over the NRCPCs period of operation.ConclusionsThe NRCPC scoring tool has been successful to date in providing a framework for decision makers to allow consistent, unbiased and objective assessments of dissimilar technologies. Prioritization tools in hospital-based HTA are beneficial to decision makers in hospital settings.
PP82 Comparison Between Informal Caregiver Burden Of Patients With Alzheimer’s Disease Versus Other Chronic Diseases
Authors: Demirbas Murat , Hahn-Pedersen Julie Hviid , Jørgensen Henrik Løvendahl 
Published: December 2023

IntroductionAlzheimer’s disease (AD) is a neurodegenerative disease with progressive neuropsychiatric symptoms. Patient care is often provided by informal caregivers similarly to various other chronic diseases. This targeted literature review assessed the difference in burden experienced by caregivers of people with AD in comparison to other chronic diseases.MethodsTwo separate search strings were developed to identify (i) caregiver burden in AD and (ii) caregiver burden in other chronic diseases using PubMed. Studies published in English (January 2012-October 2022) were included. Comparison of the caregiver burden was done using the weighted mean values (MV) of several questionnaires including the Zarit Burden Interview (ZBI), a 22-item self-report questionnaire for caregivers ranging from 0 to 88 points. ZBI is stratified into four categories of caregiving burden: Little or no burden (0 to 21), mild to moderate burden (22 to 40), moderate to severe burden (41 to 60) and severe burden (61 to 88).ResultsZBI was the most frequently used questionnaire; 13 studies reported data on caregiver burden in AD and 39 studies reported data on 20 other chronic diseases. The caregiver burden ranged from 18 to 48 in AD, measured by ZBI. The MV of AD burden was 36 based on a total of 1,703 participants. The caregiver burden in other chronic diseases ranged from MV of 5 (chronic musculoskeletal pain) to 59 (bipolar disorder).Measured by ZBI, AD burden on caregivers (MV: 36, range: 18-48) was greater than heart failure (MV: 27, range: 16-29) and type 2 diabetes (MV: 26, range: Not reported) but lesser than schizophrenia (MV: 56, range: 52-65) and bipolar disorder (MV: 59, range: Not reported).ConclusionsAD has a significant burden on caregivers. When assessing the value of interventions targeting AD, the impact of AD on caregivers should be considered in addition to the impact of AD on patients. Further studies are required to assess the informal care burden in AD and other chronic diseases.