What are cancer patients willing to pay for prophylactic epoetin alfa?—A cost‐benefit analysis

Abstract

The article by Ortega et al. provided further evidence to consider regarding recombinant human erythropoietin (r-HuEPO) use in the treatment of cancer patients.1 They addressed the economic aspects of r-HuEPO in cancer-related anemia and described the patients' perspective regarding the use of an expensive technology for their treatment. As the expensive drugs provided by high technologies gain wider areas of application, it seems that this discussion will be more popular among clinicians, patients, and health care providers who are supposed to pay for r-HuEPO. We believe the use of r-HuEPO, which is an expensive drug, will become wider in the near future, as is usually the case with high technology drugs that give the patient more comfort for higher cost. At this point, we have to stress the clinicians' responsibility for defining more clear-cut outlines of the therapeutic aspects of the drug in terms of cost-benefit advantage. In the case of r-HuEPO, this is quite complex. r-HuEPO is not simply a supplementary drug for cancer anemia. It is an endogenously secreted hormone that affects many systems. Chemotherapeutic agents may alter the endogenous secretion of EPO and may cause an EPO-resistant state.2, 3 High EPO levels after a renal injury and enhancement of recovery from cisplatin-induced acute renal failure have been shown, perhaps reflecting the role of EPO as part of the endogenous defense mechanism of the kidney.4, 5 The protective effect of EPO against neuronal damage is also under investigation.6, 7 These points need further investigation and must be considered in determining the potential therapeutic benefits of r-HuEPO therapy (especially for patients receiving cisplatin-based combinations that are potentially nephrotoxic and neurotoxic, because these side effects are being considered as important dose-limiting factors in clinical use). In this complexity, the clinicians' responsibility starts with investigating the benefits of r-HuEPO and identifying the patient population that will benefit most from the use of this drug. There is no consensus on the administration of this drug to cancer patients for reduction of transfusion requirements or for quality of life, whether the patient is receiving cytotoxic chemotherapy or not, although most studies point out the benefits.8-12 To determine the patient group that will benefit most from the use of this drug, we must first identify the patients who have the least tolerance of the complications of anemia and blood transfusions (i.e., older patients, who usually have a diminished reserve, and patients with underlying renal, pulmonary, and cardiovascular diseases). A history of previous cytotoxic treatment and frequent transfusions may also be important indicators; and rare blood groups, which are considered less often, must be regarded as important factors in patient selection for treatment with r-HuEPO. After determination of the patient group that is most prone to the complications of anemia and transfusion, we have to identify further the subsets of patients who will benefit from the treatment. These patients have low levels of endogenous EPO and have normal baseline leukocyte and platelet counts, indicating adequate bone marrow reserve. The criteria for the response to treatment have been defined previously and include increases in hemoglobin level more than 0.5 g/dL after 2 weeks, soluble transferrin receptor level after 2 weeks, and reticulocyte count after 4 weeks of r-HuEPO treatment.8, 13, 14 Another important issue is the dose: 150 U/kg 3 times a week seems to be an appropriate dose for patients not receiving cytotoxic chemotherapy.8 Patients treated with cytotoxic agents, especially cisplatin, seem to need higher doses.8, 15 Starting with the dose of 150 U/kg 3 times a week, and then doubling the dose after 2 weeks of therapy if there is not a response, seems to be an appropriate approach; still, dose schedule remains a subject of intensive research.8, 9, 11, 13, 14 We think this is the clinicians' strategy for extracting and treating the patients who will benefit most from r-HuEPO therapy. However, at this point, what we have learned from Ortega et al is to determine the willingness of the patient to receive a therapy for the correction or prevention of anemia at the expense of frequent injections, side effects such as hypertension, increased frequency of thrombotic events, high cost, and considerable incidence of treatment failure, instead of receiving blood products for the symptomatic management of anemia. Bülent Orhan M. D.*, Şuayib Yalçin M. D. , * Department of Medical Oncology, Uludag University School of Medicine, Bursa, Turkey, Department of Medical Oncology, Hacettepe University Institute of Oncology, Bursa, Turkey