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We assessed the relationship between the number of years since the initial approval date of medications and the utilization and expenditures represented by those medications in state Medicaid programs in 2017 based on publicly available data. Methods Prescription drug and expenditure data for 2017 was extracted from Centers for Medicare & Medicaid Services state drug utilization files for all states. [Extracted from the article]

Gene therapies are often curative one-time treatments that address the underlying cause of disease, helping patients avoid a lifetime of therapy and saving health systems the associated cost. Despite the potential of curative gene therapies, health systems cannot manage the short-term budgetary impact and risk associated with newly licensed high costs treatments and are increasingly demanding innovative pricing schemes. Therefore, gene therapy manufacturers must engage early with health providers and HTA authorities to agree innovative access schemes that balance equitable patient access with sustainable health system financing. [Extracted from the article]

Objectives: The development of accelerated approval programs for high morbidity and unmet need conditions has driven the use of single-arm studies in drug development. Regulatory and health technology assessment (HTA) agencies are recognizing that high-quality external control arms (ECAs), built using real-world data, can reduce uncertainties arising from single-arm studies. This review compared 7 case studies of regulatory and HTA agencies' evaluations of oncology ECAs.Methods: Food and Drug Administration multidisciplinary reviews for oncology submissions from 2014 to 2021 were screened to identify 7 cases (2 blinatumomab indications, avelumab, and erdafitinib, entrectinib, trastuzumab deruxtecan, and idecabtagene vicleucel) with ECAs to support efficacy claims. Regulatory (Food and Drug Administration, European Medicines Agency, Health Canada) and HTA (pan-Canadian Oncology Drug Review, National Institute for Health and Care Excellence, Federal Joint Committee, Haute Autorité de Santé, and Pharmaceutical Benefits Advisory Committee) submissions for these cases were reviewed. The decision makers' ECA critiques and the level of influence on the decision were analyzed and categorized.Results: Across case studies, selection bias and confounding were the most common ECA critiques. Nevertheless, agreement in critiques between and among regulators and HTA bodies was low. ECA influence on agencies' decisions also varied.Conclusions: Evaluating the same ECA evidence, agencies focused on methodologic issues (ie, selection bias and confounding), but were often not aligned on their critiques. Further research is needed to fully characterize how agencies evaluate ECAs. This study is a first step in critically evaluating agencies' critiques of ECAs and highlights the need for future guidance development around ECA design and generation. [ABSTRACT FROM AUTHOR]

Objectives: This study aimed to explore quality-adjusted life-year (QALY) and subsequent cost-effectiveness estimates based on the more physical health-focused EQ-5D 5-level version (EQ-5D-5L) value set for England or cross-walked EQ-5D 3-level version UK value set scores or more mental health recovery-focused Recovering Quality of Life Utility Index (ReQoL-UI), when using alternative within-trial statistical methods. We describe possible reasons for the different QALY estimates based on the interaction between item scores, health state profiles, preference-based scores, and mathematical and statistical methods chosen.Methods: QALYs are calculated over 8 weeks from a case study 2:1 (intervention:control) randomized controlled trial in patients with anxiety or depression. Complete case and with missing cases imputed using multiple-imputation analyses are conducted, using unadjusted and regression baseline-adjusted QALYs. Cost-effectiveness is judged using incremental cost-effectiveness ratios and acceptability curves. We use previously established psychometric results to reflect on estimated QALYs.Results: A total of 361 people (241:120) were randomized. EQ-5D-5L crosswalk produced higher incremental QALYs than the value set for England or ReQoL-UI, which produced similar unadjusted QALYs, but contrasting baseline-adjusted QALYs. Probability of cost-effectiveness <£30 000 per QALY ranged from 6% (complete case ReQoL-UI baseline-adjusted QALYs) to 64.3% (multiple-imputation EQ-5D-5L crosswalk unadjusted QALYs). The control arm improved more on average than the intervention arm on the ReQoL-UI, a result not mirrored on the EQ-5D-5L nor condition-specific (Patient-Health Questionnaire-9, depression; Generalized Anxiety Disorder-7, anxiety) measures.Conclusions: ReQoL-UI produced contradictory cost-effectiveness results relative to the EQ-5D-5L. The EQ-5D-5L's better responsiveness and "anxiety/depression" and "usual activities" items drove the incremental QALY results. The ReQoL-UI's single physical health item and "personal recovery" construct may have influenced its lower 8-week incremental QALY estimates in this patient sample. [ABSTRACT FROM AUTHOR]

Objectives: Approximately 20% of UK women aged 70+ with early breast cancer receive primary endocrine therapy (PET) instead of surgery. PET reduces surgical morbidity but with some survival decrement. To complement and utilize a treatment dependent prognostic model, we investigated the cost-effectiveness of surgery plus adjuvant therapies versus PET for women with varying health and fitness, identifying subgroups for which each treatment is cost-effective.Methods: Survival outcomes from a statistical model, and published data on recurrence, were combined with data from a large, multicenter, prospective cohort study of over 3400 UK women aged 70+ with early breast cancer and median 52-month follow-up, to populate a probabilistic economic model. This model evaluated the cost-effectiveness of surgery plus adjuvant therapies relative to PET for 24 illustrative subgroups: Age {70, 80, 90} × Nodal status {FALSE (F), TRUE (T)} × Comorbidity score {0, 1, 2, 3+}.Results: For a 70-year-old with no lymph node involvement and no comorbidities (70, F, 0), surgery plus adjuvant therapies was cheaper and more effective than PET. For other subgroups, surgery plus adjuvant therapies was more effective but more expensive. Surgery plus adjuvant therapies was not cost-effective for 4 of the 24 subgroups: (90, F, 2), (90, F, 3), (90, T, 2), (90, T, 3).Conclusion: From a UK perspective, surgery plus adjuvant therapies is clinically effective and cost-effective for most women aged 70+ with early breast cancer. Cost-effectiveness reduces with age and comorbidities, and for women over 90 with multiple comorbidities, there is little cost benefit and a negative impact on quality of life. [ABSTRACT FROM AUTHOR]

Access to care is a well-documented problem in patients with breast cancer (PwBC) from a payer/population perspective. The purpose of this study was to understand access to care issues in PwBC from their perspective and quantify the extent to which access to care issues are evident among PwBC in community oncology practices. Factor analysis determined the factor structure of access issues and multivariable regression examined the relationship between barriers and overall satisfaction with care. [Extracted from the article]

Chemotherapy dose reduction is a viable alternative that is not routinely recommended but may result in cost savings. The purpose of this study was to determine the prevalence and predictors of chemotherapy dose reduction in patients with metastatic colorectal cancer. Chemotherapy dose reduction was defined as >= 10% reduction in dose of at least one cytotoxic drug, compared to the standard or initial dose. [Extracted from the article]

Granulocyte Colony Stimulating Factors (G-CSF) are recommended by cancer consortia for the management of febrile neutropenia. This study aimed to compare survival outcomes among 1) G-CSF treated vs G-CSF untreated patients and 2) G-CSF treated vs CDR treated patients. [Extracted from the article]

Biologic Disease-Modifying Antirheumatic Drugs (bDMARDs) reduce joint damage and pain among rheumatoid arthritis (RA) patients. Proportion of patients with severe baseline DA by CDAI was highest among patients initiating certolizumab pegol (38.4%), followed by infliximab (36.4%), golimumab (36.2%), etanercept (32.2%), and adalimumab (26.3%). [Extracted from the article]