Effectiveness, toxicity, and economic evaluation of ipilimumab for the treatment of patients with metastatic melanoma in the Spanish outpatient setting

Abstract

To evaluate the effectiveness and toxicity profile of ipilimumab treatment and to examine the cost-effectiveness relation in a real-world sample of patients with metastasic melanoma. This was a multicenter, observational, retrospective cohorts study. To assess the effectiveness and safety of ipilimumab treatment progression-free survival (PFS), overall survival (OS) and adverse events were registered. An economic evaluation was performed and cost-effectiveness ratios (CERs) were calculated. Eleven patients were included, mean age 59 (SD=11) years. The median PFS was 3.83 months (95% confidence interval 0.98-9.80) and the median OS was 5.15 months (95% confidence interval 1.70-8.48). None of the patients included in the study achieved an objective response. A stable disease was achieved in four (36%) patients. The most commonly reported analytical adverse event was anemia, with all patients developing anemia in any grade. The most severe adverse event was neutropenia (n=6; 55%), with three patients developing grade 4 neutropenia (3/11; 27%). The total cost of ipilimumab treatment was &OV0556;483 397, with a median of 43 033 (interquartile range=9555) euros per patient. The median-based CER was 136 675 (28 539-474 865) euros per progression-free year gained and the median-based CER was 100 112 (23 107-374 893) euros per life-year gained. PFS observed in real-world patients was higher than that reported in clinical trials and OS was lower. The incidence of adverse events was higher. The additional cost per progression-free year gained was ∼&OV0556;136 675. The data from this study fill an important need for information on the relative value of this treatment in terms of cost-effectiveness.

A systematic review of the cost and cost-effectiveness studies of immune checkpoint inhibitors

“…Of the 35 publications remaining, 15 were further eliminated; eight were unpublished abstracts and five were review articles. Two publications were ultimately removed owing to non-modeling and/or non-comparative designs that evaluated cost but not specifically CE [ 23 , 24 ]. Thus, 20 original investigations were found to have sufficient focus and relevance to be incorporated into the systematic review.…”

Ipilimumab in a real‐world population: A prospective Phase IV trial with long‐term follow‐up

“…Additionally, the cost of ipilimumab, when opening the markets postapproval, was perceived a substantial economic burden to the health care system, with an estimated cost‐effectiveness of €100 112 per life‐year gained 25 . Using drugs in daily clinical practice, there is a concern that efficacy may be decreased and toxicity increased compared to clinical trials due to less stringent eligibility criteria and less intensive monitoring.…”

“…19,24 Additionally, the cost of ipilimumab, when opening the markets postapproval, was perceived a substantial economic burden to the health care system, with an estimated cost-effectiveness of €100 112 per life-year gained. 25 Using drugs in daily clinical practice, there is a concern that efficacy may be decreased and toxicity increased compared to clinical trials due to less stringent eligibility criteria and less intensive monitoring. A number of reports on real-world data from expanded access, named patient and compassionate-use programmes exist, [15][16][17][18]22,[26][27][28][29][30][31] but are restrained by factors such as retrospective study design, patient selection, single centre experiences and/or limited follow-up.…”

Vinflunine in the treatment of advanced urothelial cancer: clinical evidence and experience

“…An interesting report was published by Guglieri-Lopez and colleagues. They performed an analysis of effectiveness, toxicity and economic evaluation of VFL for the treatment of patients with urothelial cancer in an outpatient setting in Spain [Guglieri-Lopez et al 2016]. In their retrospective multicenter observational cohort study 37 patients with mTCCU were treated with VFL in second line.…”

“…In addition to reproducing efficacy and tolerability data from previous trials the authors demonstrated a median-based cost-effectiveness ratio of €44,789 per progression-free year gained and €22,750 per life-year gained. Compared with other treatment approaches VFL seemed to have a higher cost at a lower OS effect [Guglieri-Lopez et al 2016].…”