Potential health gains for patients with metastatic renal cell carcinoma in daily clinical practice: A real-world cost-effectiveness analysis of sequential first- and second-line treatments

Abstract

IntroductionRandomised controlled trials have shown that targeted therapies like sunitinib are effective in metastatic renal cell carcinoma (mRCC). Little is known about the current use of these therapies, and their associated costs and effects in daily clinical practice. We estimated the real-world cost-effectiveness of different treatment strategies comprising one or more sequentially administered drugs.MethodsAnalyses were performed using patient-level data from a Dutch population-based registry including patients diagnosed with primary mRCC from January 2008 to December 2010 (i.e., treated between 2008 and 2013). The full disease course of these patients was estimated using a patient-level simulation model based on regression analyses of the registry data. A healthcare sector perspective was adopted; total costs included healthcare costs related to mRCC. Cost-effectiveness was expressed in cost per life-year and cost per quality-adjusted life-year (QALY) gained. Probabilistic sensitivity analysis was conducted to estimate the overall uncertainty surrounding cost-effectiveness.ResultsIn current daily practice, 54% (336/621) of all patients was treated with targeted therapies. Most patients (84%; 282/336) received sunitinib as first-line therapy. Of the patients receiving first-line therapy, 30% (101/336) also received second-line therapy; the majority was treated with everolimus (40%, 40/101) or sorafenib (28%, 28/101). Current treatment practice (including patients not receiving targeted therapy) led to 0.807 QALYs; mean costs were €58,912. This resulted in an additional €105,011 per QALY gained compared to not using targeted therapy at all. Forty-six percent of all patients received no targeted therapy; of these patients, 24% (69/285) was eligible for sunitinib. If these patients were treated with first-line sunitinib, mean QALYs would improve by 0.062–0.076 (where the range reflects the choice of second-line therapy). This improvement is completely driven by the health gain seen amongst patients eligible to receive sunitinib but did not receive it, who gain 0.558–0.684 QALYs from sunitinib. Since additional costs would be €7,072–9,913, incremental costs per QALY gained are €93,107–111,972 compared to current practice.DiscussionHealth can be gained if more treatment-eligible patients receive targeted therapies. Moreover, it will be just as cost-effective to treat these patients with sunitinib as current treatment practice.

Augmenting the Randomized Controlled Trial with Real-World Data to Aid Clinical Decision Making in Metastatic Renal Cell Carcinoma: A Systematic Review and Meta-Analysis

“…Although no subgroup analyses were performed for these patient groups, efficacy outcomes appeared to be generally comparable with the overall estimates of the younger, predominantly white populations found in RCTs. Since 2005, the dramatic increase in targeted treatment options for mRCC has resulted in substantial improvements in health and survival [72,73]. The recent development of immune checkpoint inhibitors and their use in combination with TKIs, such as avelumab or pembrolizumab plus axitinib, look set to provide another leap forward in the treatment of mRCC [74,75].…”

A Generic Model for Follicular Lymphoma: Predicting Cost, Life Expectancy, and Quality-Adjusted-Life-Year Using UK Population–Based Observational Data

“…It is important to note, however, that others have used similar modeling techniques to explore the optimal treatment sequence for the management of conditions where options change as the disease progresses [15] , [39] , [40] , [41] , [42] . Like our approach, these studies modeled patients through their pathways; the main difference was that they simulated a set of key treatment sequences one at a time, rather than all treatment pathways/sequences at the same time [15] , [39] , [40] , [41] , [42] . The main benefit of modeling all treatment pathways at the same time is the ability to demonstrate impacts in the real-world setting, hence allowing policymakers to make more informed decisions.…”

Economic Burden of Metastatic Clear-Cell Renal Cell Carcinoma for French Patients Treated With Targeted Therapies

“…On the other hand, clinical and economic studies in daily practice are sparse [18][19][20][21][22][23][24][25][26][27]. A previous retrospective multicenter study was conducted to assess the effectiveness of targeted therapies in real life settings [28].…”