CO63 Systematic Literature Review of Health-Related Quality of Life in Advanced or Metastatic Esophageal Cancer
Patients with esophageal cancer (EC), the eighth most diagnosed cancer worldwide, face poor health-related quality of life (HRQoL) due to multiple factors related to disease progression and treatment side-effects. The aim of our systematic literature review was to evaluate health-related quality of life (HRQoL) in this vulnerable patient group in the last ten years. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: A. Pandey, R. Tomer, S. Sharma, N. Sharma, D. Jain, N. Fischer, S. Sharma Source Type: research
CO64 Challenges Associated with Surrogate Endpoints for Healthcare Decision Making in Oncology
With the continuous emergence of new technologies filling high unmet medical needs in oncology, accelerated time to market for earlier patient access has gained importance. Increasing costs of drug development and intense competition between treatment options have enhanced the desire for early market access using surrogate endpoints. No study has recently assessed the use of surrogate endpoints in oncology and how this affects a new product in development. This research aims to identify the key challenges associated with surrogate endpoints in oncology from a payer perspective and provide recommendations to ensure an optim...
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: S. Ferguson, S. Boukouvalas Source Type: research
CO65 Modelled Impact of Nirsevimab for All Infants in the Prevention of Respiratory Syncytial Virus (RSV): Related Hospitalizations and Its Predicted Cost to the Brazilian Public Healthcare System
Respiratory syncytial virus (RSV) is a major cause of lower respiratory tract infection (LRTI) in young children and poses a significant burden for Brazil's public healthcare system (SUS). Palivizumab is the sole preventive intervention for early preterm infants ( (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: M. Falavigna, S.F. Watanabe, J. Santoro, K. Ribeiro, A. Tolardo, N. Schneider, M. Safadi, R.T. Stein, A. Kieffer Source Type: research
CO66 Improvements in the Peabody Developmental Motor Scale, Second Edition (PDMS-2) and Correlation With Bayley-III Scores and Motor Milestones in Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd)
AADCd is a rare genetic disorder of monoamine neurotransmitter synthesis that typically manifests by one-year of age. Patients with AADCd have minimal functional motor movement and rarely meet developmental milestones. PDMS-2 assesses motor function in young children, and Bayley-III could identify developmental delays, including non-motor (cognitive, social/emotional) delays. Clinical trials of the gene therapy eladocagene exuparvovec (EE) assessed changes in motor function of AADCd patients using PDMS-2. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: W.L. Hwu, H.M. Lee, J. Peipert, R. Zhang, J.R. Sierra, T. OConnell, J. Woolley, M. Crowell, A. Wang, I. Tomazos Source Type: research
CO67 Development of an Investigator Global Assessment of Severity in Propionic Acidemia
To develop an Investigator Global Assessment of Severity (IGA-S) in propionic acidemia (PA). (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: N. Longo, V. Sikirica, S. Grunewald, O. Moshkovich, C. Dionisi-Vici, J. Vockley, M.A. Bellenger, J. de las Heras, G. Banerjee, E.J. Schwartz Source Type: research
CO68 Gastrointestinal Manifestations and Incontinence Products in the Real World Among Patients with Rett Syndrome in the United States
This study describes GI manifestations and DME incontinence products among female patients with RTT in the United States (US). (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: D. May, K. Kponee-Shovein, M. Mahendran, L. Cosand, N. Downes, P. Lefebvre, W.Y. Cheng Source Type: research
CO69 Evidence Gap Analysis of the Burden of Illness and Treatment of Bullous Pemphigoid
To identify evidence gaps in the literature on the burden of illness and treatment of bullous pemphigoid (BP) to support the launch of efgartigimod to treat this rare disease. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: L. Gildea, K. Copley-Merriman, C. Arvin-Berod, K. Vande Walle, P. Verheesen, I. Stoykov Source Type: research
CO70 COPD Exacerbations Pre, During, and Post COVID-19 Pandemic in the US
To evaluate the indirect impact of Coronavirus Disease 2019 (COVID-19) pandemic on patients with chronic obstructive pulmonary disease (COPD) in the US by describing changes in the rate of exacerbations over time. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: G. Requena, S.G. Noorduyn, L. Lee, A. Steffens, T. Bancroft, K. Rothnie, S. Gelwicks, H.J. Birch, C. Compton, D. Leather, R. Paczkowski, A. Ismaila Source Type: research
CO71 Faricimab Versus Standard of Care in Naive Neovascular Age-Related Macular Degeneration Patients: An Indirect Treatment Comparison Using Individual Level Data
To evaluate the potential benefit of faricimab, a new targeting vascular endothelial growth factor A (VEGF-A) and Angiopoietin-2, versus the current intravitreal (IV) a-VEGF SoC in nAMD patients. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: P. Lanzetta, M.C. Parravano, M. Nicol ò, G. Staurenghi, F. Viola, C. Galeone, L. Bianchino, E. Sicari, G. Villa, S. Vujosevic Source Type: research
CO72 Vitamin D as an Adjunct Therapy in the Treatment of Atopic Dermatitis: A Targeted Review
Atopic dermatitis (AD) is one of the most prevalent dermatological conditions with autoimmune elements. Vitamin D has been found to modulate the immune system, and hence may be a beneficial adjunct therapy in the treatment of AD. This review aimed to identify literature assessing vitamin D supplementation and its effect on AD. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: O. Borecka, J. Lawrence, S. Llewellyn Source Type: research
CO73 Overview of COA Measures in SLE Clinical Trials and Label Claims
The objective of this research is to review the landscape of COAs that were included in SLE clinical trials and FDA and EMA drug labels. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: N. Suminski, Z. Zupan, K. Rudell Source Type: research
CO75 Non-Pharmacological Strategies for Chemotherapy-Induced Peripheral Neuropathy: A Comprehensive Review
This study aimed to assess the effectiveness of non-pharmacological interventions in the management of Chemotherapy-Induced Peripheral Neuropathy (CIPN). (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: S. Jangid, A. Verma, J. Pruthi, R. Prasanna, M.K. Rai Source Type: research
CO76 Does Next Generation Sequencing (NGS) Improve Outcome in Non-Small Cell Lung Cancer (NSCLC)? A Real-World Data (RWD) Example
The objective of this analysis was to evaluate the impact of sequencing versus point mutation testing on outcomes in non-small cell lung cancer (NSCLC) using real-world data (RWD). Over time, there has been a shift in recommendations from single-gene or small-panel testing to broader genomic profiling with next-generation sequencing (NGS). In 2018, The Centers for Medicare and Medicaid Services (CMS) established the first national coverage for NGS for NSCLC. Since then, both CMS and compendial recommendations have continued to evolve, with increasing emphasis on NGS. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: S. Gendy, D. Kumar, J. Maskin, T. Obradovic, L. Turk, S. Viaggi Source Type: research
CO77 Impact of Imetelstat Treatment on PROs and Health Care Resource Utilization in Heavily Transfused Non-Del(5Q) Lower-Risk Myelodysplastic Syndromes Relapsed/Refractory to Erythropoiesis Stimulating Agents: IMerge Phase 3 Trial
Imetelstat, a first-in-class telomerase inhibitor, showed high rates of durable transfusion independence (TI) across all lower-risk myelodysplastic syndrome (LR-MDS) patient subtypes in the phase 3 IMerge trial. Here, the impact of imetelstat on patient-reported outcomes (PROs) and health care resource utilization (HCRU) was explored. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: M. Sekeres, V. Santini, M. Diez-Campelo, R. Komrokji, P. Fenaux, M. Savona, Y. Madanat, D. Valc árcel-Ferreiras, T. Illmer, A. Jonášová, P. Bělohlávková, A. Regnault, K. Creel, N. Sengupta, L. Sherman, T. Berry, S. Dougherty, S. Shah, Q. Xia, L. Su Source Type: research
CO78 Time to Diagnosis in Hypertrophic Cardiomyopathies and the Association With Adverse Outcomes Post-Diagnosis
Hypertrophic cardiomyopathy (HCM) is a chronic, and often genetic, condition characterised by unexplained left ventricular hypertrophy often impacting patients ’ quality of life. This retrospective study aims to analyse time to diagnosis (TTD) from referral and its association with time to adverse clinical outcomes post-diagnosis. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: V.G. Lim, C. Murphy, J. Ayisatu, E. Shofoluwe, P. Kimani, T. Lemmer, C. Sadlowski, B. Sandler, C.L. Zema, K.G. Pollock, M. Hurst, F. Osman Source Type: research
