A Targeted Review of Worldwide Indirect Treatment Comparison Guidelines and Best Practices.

OBJECTIVES: Controls and governance over the methodology and reporting of indirect treatment comparisons (ITCs) have been introduced to minimize bias and ensure scientific credibility and transparency in healthcare decision making. The objective of this study was to highlight ITC techniques that are key to conducting objective and analytically sound analyses and to ascertain circumstantial suitability of ITCs as a source of comparative evidence for healthcare interventions. METHODS: Ovid MEDLINE was searched from January 2010 through August 2023 to identify publicly available ITC-related documents (ie, guidelines and best practices) in the English language. This was supplemented with hand searches of websites of various international organizations, regulatory agencies, and reimbursement agencies of Europe, North America, and Asia-Pacific. The jurisdiction-specific ITC methodology and reporting recommendations were reviewed. RESULTS: Sixty-eight guidelines from 10 authorities worldwide were included for synthesis. Many of the included guidelines were updated within the last 5 years and commonly cited the absence of direct comparative studies as primary justification for using ITCs. Most jurisdictions favored population-adjusted or anchored ITC techniques opposed to naive comparisons. Recommendations on the reporting and presentation of these ITCs varied across authorities; however, there was some overlap among the key elements. CONCLUSIONS: Given the challenges of conducting head-to-head randomized controlled trials, comparative data from ITCs offer valuable insights into clinical-effectiveness. As such, multiple ITC guidelines have emerged worldwide. According to the most recent versions of the guidelines, the suitability and subsequent acceptability of the ITC technique used depends on the data sources, available evidence, and magnitude of benefit/uncertainty.

A retrospective study on the incidence, management and risk factors of skin rash in patients with advanced prostate cancer in Japan.

BACKGROUND: Worldwide, prostate cancer (PC) is the second most diagnosed cancer and the fifth leading cause of cancer death in men. Hormonal therapies, commonly used for PC, are associated with a range of treatment-emergent adverse events (TEAEs). The population from Japan seems to be at higher risk of developing TEAEs of skin rash compared to the overall global population. This study was conducted to get a better insight into the incidence, management, and risk factors for skin rash during active treatment for advanced PC in Japan. METHODS: A retrospective cohort of PC patients was identified and subsequently categorized, into non-metastatic and metastatic castration-resistant prostate cancer patients (nmCRPC and mCRPC), and metastatic castration-naïve prostate cancer patients (mCNPC). The analysis was based on a dataset from the Medical Data Vision (MDV) database. Descriptive statistics were determined, and a multivariate Cox proportional hazards model was used to the associated risk factors for the onset of rash. RESULTS: Overall, 1,738 nmCRPC patients, 630 mCRPC patients, and 454 mCNPC patients were included in this analysis. The median age was 78 years old and similar across the three cohorts. The skin rash incidence was 19.97% for nmCRPC cohort, 28.89% for mCRPC cohort, and 28.85% for mCNPC cohort. The median duration of skin rash ranged from 29 to 42 days. Statistically significant risk factors for developing skin rash included a history of allergy or hypersensitivity (all cohorts), increased age (nmCRPC and mCRPC), a body mass index (BMI) of < 18.5 (nmCRPC and mCRPC), and a PSA level higher than the median (nmCRPC). Skin rash was commonly managed with systemic and topical corticosteroids which ranged from 41.76% to 67.03% for all cohorts. Antihistamines were infrequently used. CONCLUSION: This study provides a better understanding of the real-world incidence, onset, duration, management and risk factors of skin rash in patients on active PC treatment in Japan. It was observed that approximately 20-30% of PC patients experience skin rash. Development of skin rash was associated with previous allergy or hypersensitivity, BMI of < 18.5, increased age and higher PSA levels, and was usually treated with corticosteroids.

Assessing the correlation between second progression-free survival (PFS2) and overall survival (OS) in advanced prostate cancer patients using medical data vision (MDV) claims database in Japan.

OBJECTIVE: To assess the correlation between PFS2 and OS among patients with advanced prostate cancer (PC) in a real-world setting for Japan. METHODS: This was a retrospective analysis using the Japanese MDV database. Patients with nmCRPC (non-metastatic Castration-Resistant PC), mCRPC (metastatic Castration-Resistant PC), and mCNPC (metastatic Castration-Naïve PC) were identified and their medical records were investigated for PFS2 and death. Association between PFS2 and OS was determined using the Pearson's, Spearman's, Kendall's Tau, and Fleischers' correlation coefficients. RESULTS: A total of 386,484 patients with PC were identified from the database, of which, 1,783 patients with nmCRPC, 630 with mCRPC, and 454 with mCNPC met the predefined eligibility criteria. Significant correlation between PFS2 and OS was observed in patients with nmCRPC (Pearson's r = 0.873; 95% CI: 0.849-0.897, Spearman's r = 0.909; 95% CI: 0.893-0.925; Kendall's Tau r = 0.831; 95% CI: 0.812-0.850, Fleischers' r = 0.682; 95% CI: 0.601-0.764), mCRPC (Pearson's r = 0.812; 95% CI: 0.758-0.865, Spearman's r = 0.895; 95% CI: 0.868-0.923, Kendall's Tau r = 0.789; 95% CI: 0.755-0.823, Fleischers' r= 0.439; 95% CI: 0.334-0.544), and mCNPC (Pearson's r = 0.931; 95% CI: 0.899-0.964, Spearman's r = 0.943; 95% CI: 0.922-0.964, Kendall's Tau r = 0.866; 95% CI: 0.836-0.896, Fleischers' r = 0.756; 95% CI: 0.624-0.888). CONCLUSIONS: The results of this study indicate a significant correlation between PFS2 and OS, which adds additional evidence to the existing literature of using PFS2 as a surrogate endpoint for OS in patients with PC.

Assessing the Burden of Type 2 Diabetes in China Considering the Current Status-Quo Management and Implications of Improved Management Using a Modeling Approach.

BACKGROUND: Recent estimates from the International Diabetes Federation Diabetes Atlas have quantified the total annual expenditure for diabetes in China to be between 354 and 611 billion Chinese yuan (¥) (2015). OBJECTIVES: To use a modeling approach to assess the current and possible future diabetes burden in China on the basis of the current standard of type 2 diabetes (T2D) management (status quo [SQ]) and a series of hypothetical improved management strategies. METHODS: The IQVIA CORE Diabetes Model was used to evaluate the economic burden of T2D in China on the basis of assumptions reflecting the current SQ of T2D management and a number of stepwise improvements. SQ was defined as a scenario in which T2D diagnosis is delayed by 4 years, treatment escalation to maintain glucose control occurs at a 9% glycated hemoglobin (HbA1c) threshold, and there is an overall 60% adherence rate. Stepwise improvements considered immediate diagnosis, declining levels of HbA1c escalation thresholds to 7.0%, and improvements in adherence rate to 80% and 100%. The CORE Diabetes Model was applied on per-capita level to project lifetime costs and clinical outcomes of newly diseased T2D individuals in the Chinese setting. Model outcomes were subsequently annualized and extrapolated to Chinese national level considering the total number of diagnosed individuals with T2D in China. RESULTS: The total annual direct costs attributable to diagnosed T2D in China reflecting current SQ management were estimated at ¥621 billion. Scenarios exploring stepwise improvements from SQ estimated annual net savings of ¥35, ¥35, ¥60, ¥71, ¥75, and ¥106 billion for scenarios exploring immediate diagnosis, HbA1c threshold reductions to 8.0% and 7.0%, adherence rate increase to 80% and 100%, and cardiovascular risk factor control in concordance with clinical guidelines, respectively. Net savings resulted from reduced costs to treat diabetes complications (¥38, ¥67, ¥124, ¥141, ¥161, and ¥212 billion) and excess treatment costs alongside stepwise management improvements (¥4, ¥32, ¥65, ¥69, ¥86, and ¥107 billion). Per-capita life expectancy was increased by 0.26, 0.68, 1.33, 1.47, 1.69, and 3.21 years, respectively. CONCLUSIONS: Improved T2D management strategies can help to decrease the financial burden of the disease and increase life expectancy of individuals with T2D.