Budget impact analysis of trilaciclib for decreasing the incidence of chemotherapy-induced myelosuppression in patients with extensive-stage small cell lung cancer in the United States.

BACKGROUND: Chemotherapy-induced myelosuppression, which commonly manifests as neutropenia, anemia, and/or thrombocytopenia, is a frequent and severe complication of standard treatment regimens for patients with extensive-stage small cell lung cancer (ES-SCLC). Trilaciclib is a first-in-class myeloprotective therapy indicated to decrease the incidence of chemotherapy-induced myelosuppression when administered prior to a platinum-/etoposide-containing regimen or topotecan-containing regimen for ES-SCLC. OBJECTIVE: To estimate the budget impact of administering trilaciclib prior to chemotherapy to manage chemotherapy-induced myelosuppression in adults with ES-SCLC from a US payer perspective. METHODS: A budget impact model was developed to assess the impact of introducing trilaciclib to a hypothetical 1 million-member health insurance plan. The model compared 2 market scenarios: a current scenario of standard treatments for ES-SCLC without trilaciclib, and an alternative scenario of standard treatment plus trilaciclib. Population, clinical, and cost inputs were derived from published literature and trilaciclib clinical trial data. Model outcomes included the number of myelosuppressive adverse events (AEs), costs of treatment, costs of AE management, total cost, and per-member per-month (PMPM) costs. The budget impact of trilaciclib was calculated as the difference in cost (2021 US dollars) between the 2 scenarios over a 1- to 5-year time horizon. Scenario and deterministic sensitivity analyses were conducted to assess uncertainty around key model inputs. RESULTS: An estimated total of 301 patients were eligible for treatment with trilaciclib over a 5-year period. The use of trilaciclib was estimated to reduce the number of myelosuppressive AEs over a 5-year period (events avoided included 108 for neutropenia, 7 for febrile neutropenia, 23 for anemia, and 46 for thrombocytopenia) compared with the scenario without trilaciclib. The adoption of trilaciclib was associated with a cost saving of $801,254 ($0.013 PMPM) over 5 years. The acquisition cost for trilaciclib ($3,704,199) was offset by the reduction in AE management cost ($4,282,748) and reduction in prophylactic granulocyte colony-stimulating factor use ($222,704). The cost savings associated with trilaciclib began in year 1 (total $34,388; $0.003 PMPM) and accrued over time. CONCLUSIONS: The acquisition cost of trilaciclib is projected to be offset by a reduction in the costs of managing AEs related to myelosuppression when added to standard chemotherapy regimens for ES-SCLC. The net budget impact of trilaciclib is estimated to be a cost saving. DISCLOSURES: This research was funded by G1 Therapeutics, Inc., and implemented by ZS Associates, an independent consultancy that collated the model inputs and performed the budget impact analysis. The study sponsor was involved in the study design; collection, analysis, and interpretation of data; writing of the report; and the decision to submit the report for publication. The journal open access fee was funded by G1 Therapeutics, Inc. Moran, Chioda, and Huang are employed by G1 Therapeutics, Inc. Chioda and Huang report stocks and stock options for G1 Therapeutics, Inc. Goyal and Deniz are employed by ZS Associates. Goyal reports consulting fees from G1 Therapeutics, Inc. Abraham reports consulting fees from Coherus, G1 Therapeutics, Inc. (unrelated to this study and manuscript), Mylan/Viatris, and Sandoz and participation on a data safety monitoring board or advisory board for G1 Therapeutics, Inc. MacDonald reports consulting fees from Coherus, G1 Therapeutics, Inc. (unrelated to this study and manuscript), Mylan/Viatris, and Sandoz. Deniz reports no disclosures. A synopsis of the current study was presented in poster format at the Virtual AMCP Annual Meeting, April 12-16, 2021.

Short- and long-term real-world effectiveness of omalizumab in severe allergic asthma: systematic review of 42 studies published 2008-2018.

INTRODUCTION: Omalizumab is a recombinant monoclonal anti-IgE antibody approved in the US as add-on treatment in moderate-to-severe allergic asthma (in severe allergic asthma [SAA] in Europe). A 2016 review of 24 real-world effectiveness studies in SAA published between 2008-2015 concluded that omalizumab was associated with significant improvements in objective and subjective outcomes with benefits extending beyond 2 years. Several new real-world studies have been published since, bringing the total to 42 studies. Areas covered: This systematic review of 42 studies published since 2008 updates and extends the 2016 review on the real-word evidence on omalizumab in SAA. It offers greater granularity as to time windows within which outcomes are reported and includes studies extending well beyond 4 years post omalizumab initiation. Expert commentary: This review firmly establishes the short-term effectiveness of omalizumab in adolescent and adult patients with SAA at 1 year, and provides strong evidence of long-term effectiveness up to 4 years and emergent evidence of effectiveness beyond 4 years. In the aggregate, these 42 studies underscore the long-term effectiveness of omalizumab in terms of: reducing exacerbations and symptoms, achieving asthma control, improving lung function, enhancing quality of life, decreasing emergency department visits and hospitalizations, and promoting concomitant medication-sparing.

Maternal serum alpha-fetoprotein levels in fetal hydrocephalus: a retrospective population based study.

BACKGROUND: Although maternal serum alpha-fetoprotein (MSAFP) is a highly sensitive marker for certain congenital malformations such as open neural tube and ventral wall defects, its usefulness as a screening test for fetal hydrocephalus is uncertain. We wished to determine the distribution of maternal serum alpha-fetoprotein levels associated with fetal hydrocephalus in a population-based screening program in Manitoba, and their potential relationship to additional anomalies. METHODS: Cases of fetal hydrocephalus unrelated to neural tube defects were ascertained from multiple sources and reviewed. Cross-reference with the Manitoba Maternal Serum Screening Program database determined which mothers had undergone maternal serum screening. Mean MSAFP levels in both isolated and complex hydrocephalus were calculated and compared with the general population of screened pregnancies using Independent Samples T-tests. RESULTS: Mean MSAFP levels in 70 cases of fetal hydrocephalus were significantly higher than those of the general population of screened pregnancies (P = 0.029). This was due to the fact that mean MSAFP levels in those cases with other major anomalies were increased over those of the general population (P = 0.041); cases with hydrocephalus alone showed no significant difference (P = 0.203). Only seven cases (10%) had MSAFP levels > or = 2.3 multiples of the median, the cut-off used in Manitoba. However, six of these (86%) had additional major and/or minor malformations. CONCLUSION: MSAFP screening has low sensitivity for fetal hydrocephalus and is rarely elevated in isolated cases. However, when fetal hydrocephalus is detected, elevated MSAFP levels indicate that the fetus is at significant risk to have additional malformations and further investigations, including chromosome breakage studies, may be indicated.

Measuring the quality of nursing care to Alzheimer's patients.

Facilities that provide care to Alzheimer's disease patients are under unrelenting pressure to document the quality of nursing care they provide to various stakeholders. Unfortunately, little consensus exists nor is guidance given as to how to measure the quality of nursing care. Regulations and standards exist but are seldom translated into systematic outcome measures that assist nurses and facilities to measure, report,and manage the quality of care they provide to residents in general and Alzheimer's patients in particular. This article offers practical ad-vice on conceptualizing quality of nursing care to Alzheimer's patients and the selection of outcome measures to collect, analyze, use, and re-port quality of nursing care data.

Longitudinal observational studies to study the efficacy-effectiveness gap in drug therapy: application to mild and moderate dementia.

If well-designed, longitudinal observational studies (LOSs) can provide insights to the linkages between real-world outcomes and their multilevel determinants. In this article, some of the scientific and methodologic issues related to LOSs in pharmacotherapeutic evaluations are discussed. A case of such a study in the treatment of mild to moderate dementia is provided-a case in which a pharmaceutic sponsor addressing a medical question (long-term effectiveness) realized that caring for patients who have Alzheimer's disease involves the clinical community of caregivers, physicians, families, nurses, psychologists, and pharmacists, among others, and partnered with nurse researchers to design their inquiry. The authors conclude by presenting an argument for nurses to take the lead in effectiveness research.