Pharmacoeconomics of Empirical Antifungal Use in Febrile Neutropenic Hematological Malignancy and Hematopoietic Stem Cell Transplant Patients

Stuart J Turner; Sharon CA Chen; Monica A Slavin; David CM Kong

Expert Rev Pharmacoeconomics Outcomes Res. 2013;13(2):227-235.

Five-year View

It is anticipated that newer antifungal agents will continue to become available, including the newest azoles and echinocandins and monoclonal antibody-based therapies.^[57] An increased interest in combination therapy is likely to occur owing to perceived, but not proven, better drug efficacy and to prevent antifungal resistance.^[58–60] These developments further highlight the need for pharmacoeconomic analyses to determine the relative merits of the alternatives available, and with respect to the focus of this review, the cost–effectiveness of these alternatives in empiric antifungal therapy. Finally, as diagnostic tests evolve to allow for increased predictive power, pre-emptive therapy may become a more popular treatment strategy compared with a prophylactic strategy. This will result in fewer patients exposed to unnecessary antifungal therapy.

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Acknowledgements
This article has greatly benefited from the review and advice of Jack Brown, PharmD, MS, BCPS.

Table 1. Summary of pharmacoeconomic studies of empiric antifungal treatment of invasive fungal infection.
Study (year)	Country	Alternatives evaluated	Type of economic analysis	Patient population studied	Outcome measures	Cost categories included	Conclusions	Ref.
Al-Badriyeh et al. (2009)	Australia	Voriconazole and LAmB	CEA	Hematology–oncology patients predominantly with AML as the underlying condition who have undergone chemotherapy or HSCT	5-point composite outcome measure^†	Drug acquisition, per diem costs, monitoring tests	LAmB net cost saving AU$1422 per patient treated	[40]
Al-Badriyeh et al. (2009)	Australia	Caspofungin and LAmB	CMA	Hematology–oncology patients predominantly with AML as the underlying condition who have undergone chemotherapy or HSCT	5-point composite outcome measure	Drug acquisition, per diem costs, monitoring tests	Caspofungin net cost saving AU$7245 per patient treated	[41]
Al-Badriyeh et al. (2012)	Australia	Caspofungin and voriconazole	CMA	Hematology–oncology patients predominantly with AML as the underlying condition who have undergone chemotherapy or HSCT	5-point composite outcome measure	Drug acquisition, per diem costs, monitoring tests	Caspofungin net cost saving AU$798 per patient treated	[42]
Bruynesteyn et al. (2007)	UK	Caspofungin and LAmB	CUA	Hematology–oncology patients predominantly with AML as the underlying condition who have undergone chemotherapy or HSCT	5-point composite outcome measure and QALY	Drug acquisition, per diem costs, mild toxicity costs (e.g., chills, nausea)	Caspofungin net cost saving GBP£2033 per patient treated, caspofungin 0.40 additional QALYs saved	[43]
Collins et al. (2007)	USA	Voriconazole and LAmB	CMA	Adult hematology patients receiving study drug and had chemotherapy for leukemia, lymphoma or other hematological cancers	Retrospective chart review of response rates and development of adverse effects	Drug acquisition, nephrotoxicity treatment cost, per diem costs	27% reduced treatment cost associated with voriconazole (US$14950 vs US$20591)	[44]
Kaskel et al. (2008)	Germany	Caspofungin and LAmB	CMA	Hematology–oncology patients predominantly with AML as the underlying condition who have undergone chemotherapy or HSCT	NNT for harm from nephrotoxicity and bed days reduced	Staff costs, all medication costs, infrastructure, microbiology	8.9 fewer patients (of 100 randomized) experienced worsening renal function. NNT for harm by LAmB of 12, 0.48 extra bed days per patient treated with LAmB worth €298 extra per patient receiving LAmB compared with caspofungin	[45]
Stam et al. (2008)	Italy	Caspofungin and LAmB	CUA	Hematology–oncology patients predominantly with AML as the underlying condition who have undergone chemotherapy or HSCT	5-point composite outcome measure and QALY	Drug acquisition, per diem costs, mild toxicity costs (e.g., chills, nausea)	Caspofungin net cost saving €3470 per patient treated, caspofungin 0.25 additional QALYs saved	[46]
Wingard et al. (2007)	USA	Caspofungin and LAmB	CMA	Hematology–oncology patients predominantly with AML as the underlying condition who have undergone chemotherapy or HSCT	Rate of impaired renal function	Drug acquisition, nephrotoxicity treatment cost	Overall treatment cost US$5326 less with caspofungin	[47]
Naik et al. (2011)	Sweden	Caspofungin and LAmB	CUA	Hematology–oncology patients predominantly with AML as the underlying condition who have undergone chemotherapy or HSCT	5-point composite outcome measure and QALY	Drug acquisition, per diem costs, mild toxicity costs (e.g., chills, nausea)	Caspofungin net cost saving SEK38,080, caspofungin 0.25 additional QALYs saved	[48]
Romá-Sánchez et al. (2008)	Spain	Voriconazole, caspofungin, LAmB and amphotericin B Lipid complex	CEA	Oncohematological patients at high risk of IFI and received empiric antifungal therapy	Partial or complete response with or without adverse drug reaction	Drug acquisition, per diem costs, diagnostic tests, monitoring tests	Voriconazole dominant over other alternatives (€144,794 net cost saving compared with caspofungin as next best alternative)	[49]
Shehab et al. (2007)	USA	Voriconazole and LAmB	CEA	Adult patients who received chemotherapy for leukemia, lymphoma or other hematologic cancers. Excluded baseline fungal infections within 24 h.	4-point composite outcome measure^‡	Drug acquisition, per diem costs	Voriconazole net cost saving of US$2551 per episode	[50]

†Included absence of a breakthrough fungal infection, survival for 7 days beyond end of therapy, no premature discontinuation of therapy, resolution of fever during the period of neutropenia and successfully treated for any baseline fungal infection.
‡Included resolution of fever during the period of neutropenia, absence of a breakthrough IFI within 7 days of the end of therapy, no discontinuation due to efficacy or safety concerns and survival within 7 days of the end of therapy.
AML: Acute myeloid leukemia; CEA: Cost–effectiveness analysis; CMA: Cost-minimization analysis; CUA: Cost–utility analysis; HSCT: Hematopoietic stem cell transplant; IFI: Invasive fungal infections; LAmB: Liposomal amphotericin B; NNT: Number needed to treat; QALY: Quality-adjusted life year; SEK: Swedish Krona.