A budget impact analysis of gilteritinib for the treatment of relapsed or refractory FLT3 mut+ acute myeloid leukemia in a US health plan.

AIMS: To estimate the economic impact of the introduction of gilteritinib for the treatment of relapsed/refractory (R/R) FLT3 mutation-positive (FLT3 mut+) acute myeloid leukemia (AML) from a US payer's perspective. METHODS: A budget impact model (BIM) was developed to evaluate the 3-year total budgetary impact of treating adults with R/R FLT3 mut+ AML eligible for gilteritinib in a hypothetical US health plan. Total costs (drugs/administration, hospitalization, monitoring, adverse events, transfusions, subsequent hematopoietic stem cell transplantation, post-progression, and FLT3 testing) were estimated before and after gilteritinib entry. The budget impact was the total cost difference between the two scenarios. The target population size and cost inputs were based on public data or published literature, drug market share was informed by market research data, and the model included recommended treatments for R/R FLT3 mut+ AML per clinical guidelines. Deterministic sensitivity analyses (DSAs) and scenario analyses varying key model inputs and assumptions were conducted to test for robustness. RESULTS: In a hypothetical health plan with 1 million members, 20.9 adults with R/R FLT3 mut+ AML were estimated to be eligible for gilteritinib. Of these, it was assumed 30.0% would be treated with gilteritinib in Year 1 following gilteritinib entry, increasing the total plan budget by $663,795 and the per-member-per-month (PMPM) cost by $0.055. In Years 2-3, the market share of gilteritinib increased to 45.0%, increasing the total plan budget impact by $1,078,371 and $1,087,230, and the PMPM cost by $0.090 and $0.091, respectively. The model results remained robust in DSAs and scenario analyses, with the largest impact observed when the projected uptake of gilteritinib was changed. LIMITATIONS: The results of this BIM are contingent upon the model's assumptions and inputs. CONCLUSIONS: Adding gilteritinib to the formulary for the treatment of adults with R/R FLT3 mut+ AML had a minimal budget impact from a US payer's perspective.

Characteristics of and trends in the late-stage biopharmaceutical pipeline.

OBJECTIVE: To quantify and characterize biopharmaceutical agents and new indications in late-stage development in the United States as of May 2006. STUDY DESIGN: Review of drug development databases and other secondary sources. METHODS: Biopharmaceutical was defined as "any biology-based therapeutic that structurally mimics compounds found within the body." Unique biopharmaceuticals, including new molecular entities or new indications in phase 2 or higher development, were identified and characterized through reviews of the literature, 5 drug development databases, a clinical trial database, and telephone inquiries with manufacturers. RESULTS: As of May 2006, there were 111 unique biopharmaceuticals in late-stage development for 190 indications. Of 111 unique agents in the pipeline, 87 are new molecular entities, and 24 are already approved for other indications. Overall, 38 disease categories were targeted, and at least 33 physician specialties are likely to be affected. The greatest proportion of agents (43 biopharmaceuticals and 83 indications) target cancer. More than 70% of agents in the pipeline will require administration by a healthcare provider. More than 50% of the indications in the pipeline will require long-term (chronic) treatment (defined as >1 year and excludes cancer). CONCLUSIONS: The steady growth of the US biopharmaceutical pipeline and consequent anticipated near-term approvals will increasingly affect third-party portfolio decision making. Cost of therapy, identifying the right drug for the right patient, and outcomes-based value should drive that decision process.