Cost-effectiveness of the McGill interactive pediatric oncogenetic guidelines in identifying Li-Fraumeni syndrome in female patients with osteosarcoma.

BACKGROUND: Li-Fraumeni syndrome (LFS) is a penetrant cancer predisposition syndrome (CPS) associated with the development of many tumor types in young people including osteosarcoma and breast cancer (BC). The McGill Interactive Pediatric OncoGenetic Guidelines (MIPOGG) decision-support tool provides a standardized approach to identify patients at risk of CPSs. METHODS: We conducted a cost-utility analysis, from the healthcare payer perspective, to compare MIPOGG-guided, physician-guided, and universal genetic testing strategies to detect LFS in female patients diagnosed at an age of less than 18 years with osteosarcoma. We developed a decision tree and discrete-event simulation model to simulate the clinical and cost outcomes of the three genetic referral strategies on a cohort of female children diagnosed with osteosarcoma, especially focused on BC as subsequent cancer. Outcomes included BC incidence, quality-adjusted life-years (QALYs), healthcare costs, and incremental cost-utility ratios (ICURs). We conducted probabilistic and scenario analyses to assess the uncertainty surrounding model parameters. RESULTS: Compared to the physician-guided testing, the MIPOGG-guided strategy was marginally more expensive by $105 (-$516; $743), but slightly more effective by 0.003 (-0.04; 0.045) QALYs. Compared to MIPOGG, the universal testing strategy was $1333 ($732; $1953) more costly and associated with 0.011 (-0.043; 0.064) additional QALYs. The ICUR for the MIPOGG strategy was $33,947/QALY when compared to the physician strategy; the ICUR for universal testing strategy was $118,631/QALY when compared to the MIPOGG strategy. DISCUSSION: This study provides evidence for clinical and policy decision-making on the cost-effectiveness of genetic referral strategies to identify LFS in the setting of osteosarcoma. MIPOGG-guided strategy was most likely to be cost-effective at a willingness-to-pay threshold value of $50,000/QALY.

Long-Term Sequelae and Health-Related Quality of Life Associated With Lyme Disease: A Systematic Review.

Lyme disease (LD) is the most commonly reported vector-borne disease, but its clinical consequences remain uncertain. We conducted a systematic review of the long-term sequelae and health-related quality of life (HRQoL) associated with LD in North America and Europe. We performed searches in 6 electronic databases up to December 2018 following Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, including observational studies reporting long-term sequelae, HRQoL, and prognostic factors. We included 46 studies, published between 1994 and 2019. Based on 21 studies reporting attributable outcomes, higher proportions of sequelae reported from exposed patients were: neck pain, myalgia, arthralgia, paresthesia, sleep disorder, poor appetite, and concentration difficulties. Patients with PTLDS reported impaired HRQoL compared to the general US population. Included studies were highly heterogeneous in terms of study design, settings, patient characteristics, and quality. Patients with LD are more likely to report nonspecific long-term sequelae, especially those experiencing persistent symptoms posttreatment. Opportunities exist for prospective longitudinal studies to better understand LD outcomes.

Cost-Utility of Rhegmatogenous Retinal Detachment Repair With Pars Plana Vitrectomy, Scleral Buckle, and Pneumatic Retinopexy: A Microsimulation Model.

PURPOSE: To assess the cost-effectiveness of primary noncomplex rhegmatogenous retinal detachment (RRD) repair, comparing 3 different strategies, pars plana vitrectomy (PPV), scleral buckle (SB), and pneumatic retinopexy (PnR) from the health care payer perspective over a lifetime. DESIGN: Model-based cost-utility analysis. METHODS: A simulated cohort of 100,000 adult patients (≥18 years old) requiring primary noncomplex RRD repair in theoretical surgical centers in the United States. Quality-adjusted life years (QALYs), lifetime costs (2022 United States dollars), and the incremental cost-effectiveness ratio (ICER) of the 3 interventions were projected over a lifetime horizon, with a cost-effectiveness threshold of ≤$50,000 per gained QALY. RESULTS: Based on inputted parameters, the primary anatomical success was highest for PPV (95.00%) compared to SB (91.76%) and PnR (63.41%). The QALYs associated with PPV, SB, and PnR were (11.87, SD 1.62), (11.84, SD 1.63), and (11.59, SD 1.72), respectively. The incurred lifetime costs of RRD repair and associated postoperative surgeries for PPV, SB, and PnR were $4445.72 (SD 655.75), $4518.04 (662.92), and $3978.45 (728.50), respectively. Parameter-level simulations suggested that PPV was most likely to be the most cost-effective therapy compared to SB and PnR beyond a threshold of $3000/QALY. The incremental cost-effectiveness ratio for PPV compared to PnR was $1693.54. SB was dominant in all scenarios. Threshold analyses indicated that the success rate of PnR would have to be 100% and/or the cost would have to be $2000 or less over lifetime for it to be more cost-effective than PPV. CONCLUSIONS: This study found PPV to be the most cost-effective primary procedure for RRD repair at a threshold of $50,000/QALY gained over a lifetime horizon from the health care payer perspective.

The PSY-SIM Model: Using Real-World Data to Inform Health Care Policy for Individuals With Chronic Psychotic Disorders.

BACKGROUND AND HYPOTHESIS: Few microsimulation models have been developed for chronic psychotic disorders, severe and disabling mental disorders associated with poor medical and psychiatric outcomes, and high costs of care. The objective of this work was to develop a microsimulation model for individuals with chronic psychotic disorders and to use the model to examine the impact of a smoking cessation initiative on patient outcomes. STUDY DESIGN: Using health records and survey data from Ontario, Canada, the PSY-SIM model was developed to simulate health and cost outcomes of individuals with chronic psychotic disorders. The model was then used to examine the impact of the Smoking Treatment for Ontario Patients (STOP) program from Ontario on the development of chronic conditions, life expectancy, quality of life, and lifetime health care costs. STUDY RESULTS: Individuals with chronic psychotic disorders had a lifetime risk of 63% for congestive heart failure and roughly 50% for respiratory disease, cancer and diabetes, and a life expectancy of 76 years. The model suggests the STOP program can reduce morbidity and lead to survival and quality of life gains with modest increases in health care costs. At a long-term quit rate of 4.4%, the incremental cost-effectiveness ratio of the STOP program was $41,936/QALY compared with status quo. CONCLUSIONS: Smoking cessation initiatives among individuals with chronic psychotic disorders can be cost-effective. These findings will be relevant for decision-makers and clinicians looking to improving health outcomes among this patient population.

Developing a cross-validation tool for evaluating economic evidence in rapid literature reviews.

Background: Rapid economic reviews efficiently summarize economic evidence. However, reporting main findings without assessing quality and credibility can be misleading. The objective of this study was to develop a rapid cross-validation screening tool to evaluate economic evidence when conducting rapid economic literature reviews. Methods: This article outlines our reasoning and the theoretical concepts for developing the screening tool. Results: This cross-validation tool is a qualitative approach under a Bayesian framework that uses prior health economic evidence to gauge the credibility of the rapid economic review's findings. This article describes an application of this tool and highlights practical considerations for its development and deployment. Conclusion: This tool can provide a valuable screening instrument to evaluate the quality and credibility of the economic evidence.