P45 Are There Any Differences in the Recommendations for Ultra Orphan Medications: An Assessment of NICE, GBA, and HAS
Ultra orphan drugs pose challenges in the health technology assessment (HTA), reimbursement, and pricing due to limited patient population and data, resulting in high decision uncertainty. Considering these challenges, we aim to examine the outcomes for ultra orphan drugs appraised by NICE, GBA, and HAS. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: T. Bhatnagar, M. Siroula, J. Gupta, M.K. Siddiqui, S.K. Nielsen Tags: Differences and New Developments in HTA Around the World Source Type: research
P46 Drivers of Health Technology Assessment Methods and Process Reforms: An International Comparison of Real World Evidence, Patient Involvement and Discounting
Our research aims to understand what drives change in Health Technology Assessment (HTA) agencies ’ methods and processes (M&P), specifically positions on real-world evidence (RWE), patient involvement in HTA, and discounting, in Australia, Belgium, Canada, Denmark, England, France, Germany, Italy, Portugal, Sweden, Singapore, Spain, Taiwan, and the Netherlands. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: E. Bell, P. Cubi-Molla, M. Garau, G. Kumar, J. Pan, P. Radu, C. Baxter, R. Gilardino, M. Goodall, K. Nelson, J. van Bavel Source Type: research
P47 How Do HTA Agencies Manage Uncertainties in the Long-Term Clinical Benefit of Gene Therapies? An Analysis of Recent Gene Therapy HTAs in England, Germany, France, Italy, Canada, and the United States
To date, seven gene therapies (GTs) have European regulatory approval and have been reimbursed at significant upfront costs. Assessing the long-term benefits of one-time GTs is challenging for payers due to the short duration of clinical data at the time of Health Technology Assessment (HTA). This research aims to understand the methods used by HTA agencies to assess the durability of GTs and whether the concept of biological plausibility is considered by payers. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: B. McCormick, C. Sheppard, E. Hutt, S. Schmitz, A. Kumar Source Type: research
P48 New NICE Proportionate Approaches: Are They Any Faster or More Successful?
The National Institute for Health and Care Excellence (NICE) assesses the incremental clinical- and cost-effectiveness of healthcare technologies to inform public reimbursement recommendations in England and Wales. In 2022, NICE implemented a pilot for a new proportionate approach to technology appraisals, aiming to apply faster evaluation processes to simpler, low-risk treatments. This research assesses the impact of this approach on timing and outcomes of NICE recommendations. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: R. Andrews, D. Hall Source Type: research
P49 Comparability of Overall Survival in Real-World and Clinical Trial Data for BRAF+ Advanced Melanoma
External controls based on real-world data (RWD) are increasingly used to complement clinical trial data when assessing effectiveness of treatments in oncology. In the absence of randomization, comparisons between clinical trial and RWD have well-recognized risk of bias, arising from potential differences across these settings in patient populations, diagnostic testing, background therapies and outcome assessments. Suitability of RWD as external controls should be determined on a case-by-case basis considering the RWD source, study outcomes compared, and study design and analysis steps employed to address bias. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: G. Sajeev, K. Chen, G. In, R. Simpson, S. Kalia, D. Christensen, D. Liu, N. Rezai, A. di Pietro, J. Signorovitch Tags: Challenges in Modeling Oncology Outcomes Source Type: research
P50 Joint Modelling of Intermediate Longitudinal Biomarkers to Predict Overall Survival in Patients with Solid Tumors
Joint modelling (JM) of longitudinal and time-to-event data simultaneously can provide an estimated biomarker profile (adjusted for informative dropout due to death) or predictions of the time-to-event outcome conditional upon the longitudinal biomarker profile. This application explores the association between tumor burden and overall survival (OS) in patients with solid tumors, compared to a traditional parametric approach. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: L. Linsell, N. Paracha, J. Grossman, C. Bokemeyer, J. Garcia-Foncillas, A. Italiano, G. Vassal, Y. Chen, B. Torlinska, K. Abrams Source Type: research
P51 Testing a Survival Extrapolation Algorithm for Cancer Immunotherapies: Pass or Fail?
We present a practical demonstration of this algorithm using multiple data-cuts from the CheckMate-649 (CM-649) study. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: N. Latimer, K. Taylor, A. Hatswell, S. Ho, G. Okorogheye, C. Chen, I. Kim, J. Borrill, D. Bertwistle Source Type: research
P52 Validation of an Algorithm to Identify Lines of Therapy Among Adult Patients With Endometrial and Ovarian Cancer in a Real-World Dataset
As electronic health records (EHR) do not explicitly capture lines of therapy (LoT) received by patients, algorithms aim to automatically extract this information. However, LoT algorithms depend on several arbitrary assumptions. We aim to validate a LoT algorithm in patients with a gynaecological cancer diagnosis using EHR. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: S. Luhar, J. Wallis, F. Picariello, F. Tunaru, G. Hatton, L. Carpenter Source Type: research
P53 Description of Polypharmacy and PIM Use Trends in the Elderly From the Perspective of Deprescribing
The issue of polypharmacy and inappropriate medication use has long been recognized. Interventions such as deprescription have been proposed but the uptake of these interventions is mixed. We aimed to explore changes of overall prescription medication pattern and use of potentially inappropriate medication in the elderly and identify predictors of medication use. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: S. Pan, S. Li, S. Jiang, H. Wu, B. Lyu Tags: Medication Use and Its Consequences Source Type: research
P54 Patient-Reported & Health Economic Outcomes for Low-Value Medications in Patients Living with Dementia
Low-value medications (Lvm) provide little or no benefit to patients, can be harmful, and waste healthcare resources and costs. Although there is evidence that people with dementia (PwD) are particularly affected by Lvm, the long-term impact on their patient-reported and health-economic outcomes has rarely been reported. Therefore, we aimed to analyze the impact of Lvm on health-related quality of life (HRQoL), hospitalizations, and healthcare costs over two years. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: M. Platen, A. R ädke, W. Mohr, M. Buchholz, W. Hoffmann, B. Michalowsky Source Type: research
P55 Prevalence and Predictors of Primary Non-Adherence to Medications Prescribed in Primary Care
To study primary nonadherence to medications prescribed in primary care, defined as failure to dispense a new medication or its equivalent within six months of the prescription issue date. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: S. Zeitouny Source Type: research
P56 Trends in Prescription Drug Use Among Adults With Diabetes in the United States From 1999-2018
Diabetes care is associated with substantial medical and financial burden to individuals and society. Understanding the patterns of medication use can help inform clinical and pharmaceutical practice. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: S. Li, S. Pan, S. Jiang, B. Lyu Source Type: research
CO1 Comparative Efficacy of Non-Statin Lipid-Lowering Therapies in Patients With Hypercholesterolemia at Increased Cardiovascular Risk: An Updated Network Meta-Analysis
To update the published network meta-analysis (NMA) from Burnett et al. 2022 comparing the efficacy of non-statin lipid-lowering therapies inclisiran, evolocumab, alirocumab, bempedoic acid, and ezetimibe in patients with hypercholesterolemia and/or increased cardiovascular risk having elevated low-density lipoprotein cholesterol (LDL-C) despite taking maximally-tolerated dose statins. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: H. Burnett, B. Neupane, V. Pierre, K. Fahrbach, A. Cichewicz, H. Natani, D. Bhowmik, A. Reichelt, K. Buesch, R. Jindal Tags: Clinical Outcomes Source Type: research
CO4 Overview of Recent Systematic Literature Reviews on Glucagon-like Peptide-1 Receptor Agonists for Weight Loss in Adults with Obesity
The global prevalence of obesity has drastically increased over the past three decades, increasing the risk of obesity-related morbidity and premature mortality. Lifestyle/behavioral interventions for weight loss have had limited success. Glucagon-like peptide-1 receptor agonists (GLP-1 RAs) are emerging as highly effective anti-obesity medications (AOMs). This research aims to identify key trends in systematic literature reviews (SLRs) on GLP-1 RAs in adults with obesity. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: K.E. Smoyer, L. Moubarak, S. Singh, S. Kandola Source Type: research
CO6 Teduglutide for Treating Short-Bowel Syndrome in Adults: A Meta-Analysis of Real-World Evidence Compared to Clinical Trial Data
Teduglutide is a treatment that allows patients with short bowel syndrome and type 3 intestinal failure (SBS- IF) to reduce their dependence on intravenous nutrition and fluids (parenteral support; PS). A pivotal randomized controlled trial (RCT) of teduglutide showed 63% of patients experienced a ≥20% reduction in weekly PS volume at 24 weeks. Individual real-world evidence (RWE) studies have reported greater and more rapid PS reductions than clinical trials, but whether this is a general trend is unknown. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: J. Eaton, R. Harvey, P. Cain, A. Dallongeville, S. Campbell-Hill Source Type: research
