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Background and Objective: The phase III ALFA-0701 study demonstrated the efficacy and safety of gemtuzumab ozogamicin (GO) versus standard of care (SOC) chemotherapy (daunorubicin and cytarabine) for the treatment of adult patients with de novo CD33+ acute myeloid leukaemia (AML). This study analysed the cost-effectiveness of GO from the perspective of the UK health care payer. Methods: A cohort state-transition model was developed to estimate direct health care costs and quality-adjusted life-years (QALYs) over a lifetime time horizon from AML diagnosis to death using monthly cycles. Data on complete remission, overall survival, relapse-free survival (RFS), haematopoietic stem-cell transplantation, and adverse events for GO plus SOC versus SOC were obtained from the ALFA-0701 study. Overall survival and RFS were extrapolated beyond the trial horizon using mixture cure models. Unit costs were obtained from standard national sources. Utilities were identified in a systematic literature review. Costs and outcomes were discounted at 3.5%. Analyses were performed for the base-case population, excluding patients with an unfavourable cytogenetic profile, and the overall population. Results: For the base-case and overall populations respectively, incremental per-patient costs (£13,456 and £14,773) and QALYs (0.99 and 0.68) for GO plus SOC versus SOC resulted in incremental cost-effectiveness ratios (ICERs) of £13,561 and £21,819 per QALY gained. The mean probabilistic ICERs were £14,217 and £23,245, respectively. Univariate sensitivity analyses supported the robustness of the results. Conclusions: The ICERs for both populations met NICE’s £20,000–£30,000 willingness-to-pay threshold for medicines and supported the current approval for GO.

Mason: Adelphi Values: Employment; Pfizer: Consultancy. Mamolo:Pfizer: Employment, Equity Ownership. Johnson:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Viqueira:Pfizer Inc: Employment, Equity Ownership. Gater:Pfizer Inc: Consultancy; Adelphi Values Ltd: Employment. Russell-Smith:Pfizer: Employment, Equity Ownership. Tatlock:Adelphi Values: Employment. Shah:Pfizer: Employment, Equity Ownership. Cappelleri:Pfizer: Employment, Equity Ownership.

Introduction:Tyrosine kinase inhibitors (TKIs) represent the standard of care for patients with chronic-phase chronic myeloid leukemia (CP-CML). The emergence and availability of second-generation TKIs (dasatinib, nilotinib and bosutinib) has led to increased choice for patients and physicians, with data from randomized clinical trials indicating improved efficacy of such therapies compared to the first-generation TKI (imatinib). Differences in the efficacy of second-generation TKIs are negligible; however, all have distinct dosing instructions and safety profiles.

Brümmendorf: Merck: Consultancy; Janssen: Consultancy; Novartis: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding; Takeda: Consultancy. Mamolo:Pfizer: Employment, Equity Ownership. Reisman:Pfizer: Employment, Equity Ownership. Bushmakin:Pfizer: Employment, Equity Ownership. Cappelleri:Pfizer: Employment, Equity Ownership. Crescenzo:Pfizer: Employment. DeAnnuntis:Pfizer Inc: Employment, Equity Ownership. Viquiera:Pfizer: Employment, Equity Ownership. Cortes:Novartis: Consultancy, Research Funding; Daiichi Sankyo: Consultancy, Research Funding; Astellas Pharma: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding; Arog: Research Funding.

Muresan: Ingress-health Nederland BV: Employment. Mamolo:Pfizer: Employment, Equity Ownership. Cappelleri:Pfizer: Employment, Equity Ownership. Mokgokong:Pfizer Inc: Equity Ownership. Palaka:Pfizer Inc: Employment, Equity Ownership. Hills:Daiichi Sankyo: Consultancy, Honoraria. Latimer:Astra Zeneca: Consultancy; Merck: Consultancy; Pfizer: Consultancy; BMS: Consultancy; Janssen: Consultancy; Bluebirdbio: Consultancy. Heeg:Ingress-health Nederland BV: Employment, Equity Ownership, Research Funding.

Muresan: Ingress-health Nederland BV: Employment. Mamolo:Pfizer: Employment, Equity Ownership. Cappelleri:Pfizer: Employment, Equity Ownership. Crescenzo:Pfizer: Employment. Leip:Pfizer: Employment, Equity Ownership. Viqueira:Pfizer: Employment, Equity Ownership. Heeg:Ingress-health Nederland BV: Employment, Equity Ownership, Research Funding.

Welch: Pfizer: Employment, Equity Ownership. Quon:Pfizer: Consultancy. Zou:Pfizer: Consultancy. Uyei:Pfizer: Consultancy. Wu:Pfizer: Consultancy. Xiao:Pfizer: Consultancy. Mamolo:Pfizer: Employment, Equity Ownership.

Introduction:In the ongoing phase 3 BFORE trial (NCT02130557), a significantly higher major molecular response rate at Month 12 was seen with bosutinib vs imatinib in patients with newly diagnosed chronic phase chronic myeloid leukemia (CP CML), and bosutinib treatment was accompanied by improvement or maintenance of health-related quality of life (HRQoL), as assessed by the patient-reported Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) instrument. Diarrhea is a common side effect of bosutinib and occurred more frequently in bosutinib- vs imatinib-treated patients in the primary analysis of the BFORE trial (all grades: 70.1% vs 33.6%). Here, the impact of diarrhea, specifically on HRQoL, was assessed in patients receiving bosutinib or imatinib in the BFORE trial.

Introduction:Tyrosine kinase inhibitors (TKIs) have transformed the treatment paradigm for chronic myeloid leukemia (CML). Although not curative, TKIs have increased the life expectancy of patients with CML to approximately that of the general population. There are currently 5 TKIs (imatinib, bosutinib, dasatinib, nilotinib, and ponatinib) approved in the United States to treat CML. Bosutinib was initially approved in September 2012 for the treatment of patients with Philadelphia chromosome-positive (Ph+) CML intolerant or resistant to prior therapy; the indication was expanded in December 2017 to patients with newly diagnosed chronic phase (CP) CML, based on data from the phase 3 BFORE trial demonstrating superior efficacy vs imatinib in this population. A budget impact model was developed to estimate the change in cost to US commercial payers following the addition of first-line (1L) bosutinib to the drug formulary for newly diagnosed Ph+ CP CML. As the introduction of bosutinib in the 1L setting is expected to have a halo effect on treatment pathways and utilization in subsequent treatment lines, the model also captured second- and third-line (2L and 3L) treatments for CML.

Introduction:Gemtuzumab ozogamicin (GO; MylotargTM) was approved by the US Food and Drug Administration in 2017 and the European Medicines Agency in 2018 in combination with standard of care (SOC) chemotherapy (daunorubicin/cytarabine) for the treatment of de novo acute myeloid leukemia (AML). Although initial remission rates for de novo AML are high, overall survival (OS) is low; however, a proportion of patients experience long-term remission (>5 years) and can be considered cured. Statistically cured populations can be estimated by fitting mixture cure models (MCMs) to survival data, a method that takes into account background survival rates. The objective of this research was to assess and compare the statistical cure rates with GO plus SOC vs SOC alone among adult patients with de novo AML.

Introduction

Introduction:Since many patients with chronic phase chronic myeloid leukemia (CP CML) achieve long-term survival, health-related quality of life (HR-QOL) as assessed by PROs is an important consideration. Bosutinib is a SRC/ABL tyrosine kinase inhibitor approved for Philadelphia chromosome-positive CML treatment in adults resistant or intolerant to prior therapy. In the BFORE trial (NCT02130557) of bosutinib versus imatinib a significantly higher major molecular response rate at 12 months (the primary endpoint) occurred with bosutinib. Here we report initial PRO results from BFORE.

INTRODUCTION:Acute myeloid leukemia (AML) is the most common form of acute leukemia. Induction therapy usually consists of a cytarabine-anthracycline combination, such as daunorubicin and cytarabine (DA), a regimen which has been in use for over 30 years. There have been few advances in treatment and thus there are few options for induction therapies that prolong remission. Gemtuzumab ozogamicin (GO) was approved in the United States in 2000 as a treatment for relapsed AML. Although withdrawn in 2010, data from recent randomized controlled trials (RCTs) showed a lower dose of GO (3 mg/m2) in combination with DA led to improved event-free survival (EFS), relapse-free survival (RFS), and overall survival (OS) in the newly-diagnosed setting.

Introduction:The care of adults with acute myeloid leukemia (AML) is complex, involving repeated courses of chemotherapy; many patients suffer from sequelae of cytopenias and treatment-related toxicities that impair their health-related quality of life (HRQoL). Because knowledge regarding symptom burden and HRQoL is largely derived from academic centers' experience, there are uncertainties regarding how this compares to the real-world experience.

Muresan: Ingress-health Nederland BV: Employment. Mamolo:Pfizer Inc: Employment, Equity Ownership. Cappelleri:Pfizer Inc: Employment, Equity Ownership. Shapiro:Pfizer Inc: Employment, Equity Ownership. Crescenzo:Pfizer Inc: Employment. Su:Pfizer: Employment, Equity Ownership; Bristol Myers Squibb: Equity Ownership. Heeg:Ingress-health Nederland BV: Employment, Equity Ownership, Research Funding.

Background: In clinical trials of second line and later line therapies for chronic myeloid leukemia (CML), to date only single arm trials have been conducted for the available treatments (i.e. Bosutinib, Dasatinib, Nilotinib and Ponatinib). These trials included heterogeneous patient populations in terms of line of treatment, stage of disease and other patient- and disease baseline characteristics that may impact the outcomes of the treatment. This hampers any direct or indirect comparisons of these second line CML treatments in terms of efficacy measures such as progression-free survival (PFS) and overall survival (OS). In this situation (i.e. lack of common comparator), matching-adjusted indirect treatment comparisons (MAICs) can be applied.