Cost of implementing CAR-T activity and managing CAR-T patients: an exploratory study.

BACKGROUND: Chimeric antigen receptor T cells (CAR-T) represent an innovation but raise issues for healthcare payers because of the uncertainty on impact at market launch, high cost and important organisational impact. The literature has focused on their assessment, appraisal and market access solutions. No evidence on the costs sustained to implement CAR-T is available and a few studies reported the cost of the CAR-T clinical pathway, including the activities that are remunerated through inpatient or outpatient fee-for-service/episode. This paper aims at filling the information gap, assessing the cost of implementing CAR-T activity and the full cost of managing the CAR-T clinical pathway. METHODS: Cost analysis relied on the Activity Based Costing approach, which was applied to two Italian healthcare organisations, both CAR-T Centres authorized by the regional governments with a minimum of 20 patients treated with the first two CAR-T therapies launched on the market. RESULTS: The cost of implementing CAR-T was estimated at €1.31 million (calculated for one of the organizations with complete data). Most of these costs (77%) were generated by quality assurance activity. The mean cost per patient entering the CAR-T pathway (59 and 27) and surviving at follow-up (21 and 5) ranges from €48K to €57K and from €96K to €106K, respectively. Fees for hospitalization and infusion of gene therapy accounts for more than 70% of these costs. The actual hospitalisation cost varies greatly across patients and is in general lower than the fee-for-episode paid by the region to the hospital. CONCLUSIONS: Despite its limitations (exploratory nature; the time spent by staff on activities which are not remunerated through fees was estimated through interviews with the CAR-T coordinators; cost items are not fully comparable), this research highlighted the relevant organisational and economic impact of CAR-T and provided important insights for policy makers and healthcare managers: the necessity to invest resources in CAR-T implementation; the need for assessing activities which are not remunerated through fees for service / episode; the opportunity to shift from fee-for-episode / service to bundled payments for CAR-T clinical pathway.

Learning effect and diffusion of innovative medical devices: the case of transcatheter aortic valve implantation in Italy.

AIM: We investigated the diffusion of transcatheter aortic valve implantation (TAVI) since its introduction into the Italian market aimed at identifying the potential drivers of uptake and diffusion at hospital and regional levels. MATERIALS & METHODS: We estimated the determinants of TAVI diffusion in Italy from 2007 to 2015 with a regression analysis based on registry data. RESULTS: Since 2007, TAVI has shown significant diffusion rates in Italy. The diffusion is positively correlated with implanting centers' experience and with the presence of key opinion leaders. Regional recommendations on the use of TAVI negatively influence the diffusion. Reimbursement policies do not exert a relevant impact. CONCLUSION: Learning effect seems to be the major driver of TAVI diffusion in Italy.

Cost of care and social consequences of very low birth weight infants without premature- related morbidities in Italy.

Aim of this study was to estimate the cost that is borne by the Italian National Health Service, families, and social security due to very low birth weight infants (VLBWIs) without prematurity-related morbidities up to the age of 18 months. We followed up on 150 VLBWIs and 145 comparable full-term infants (FTIs) who were born in one of 25 different neonatal intensive care units upon discharge from the hospital and at six and 18 months of age. The average length of the primary hospitalisation of the VLBWIs was 59.7 days (SD 21.6 days), with a total cost of €20,502 (SD €8409), compared with three days (SD 0.4 days) with a total cost of €907 (SD €304) for the FTIs. The total societal cost of the VLBWIs for the first 18 months of life was €58,098 (SD €21,625), while the corresponding figure for FTIs was €24,209 (SD €15,557). Among VLBWIs, both low birth weight and gestational age were correlated with the length of hospitalisation after birth (r(2) = 0.61 and r(2) = 0.57, respectively; p values < 0.0005). Our findings highlight that the existing DRGs and tariffs inadequately reflect the actual costs for Italian National Health Service.

[Health technology assessment: a multidisciplinary approach for selecting innovations in the health service]. / Health technology assessment. Un approccio multidisciplinare per la scelta tra alternative in sanità

Technological evolution and the increasing requests of a more qualified health care have challenged politicians to evaluate the economical sustainability of proposed innovations. The objective of government health policies is to guarantee real advances in the quality of care to all citizens. Since 1965, independent research centers have analyzed this issue for the US Congress. In 1973, Congress endorsed the establishment of an Office of Technology Assessment (OTA) to discover the best strategies for evaluating such advances. OTA have proposed the following criteria to identify possible beneficial innovations to be introduced into routine health care: effectiveness, safeness, worth, costs, cost-effectiveness ratio and cost patient-benefit ratio. This review analyzes in detail the pathway that each medical innovation follows in order to identify which technological evolutions might prove to be truly beneficial and sustainable for the community.

Cost-effectiveness and cost-utility of beclomethasone/formoterol versus fluticasone propionate/salmeterol in patients with moderate to severe asthma.

BACKGROUND: Asthma is a chronic disease characterized by acute symptomatic episodes with variable severity and duration. Pharmacological asthma management aims to achieve and maintain control without side effects, thus improving quality of life and reducing the economic impact. Recently, a clinical trial showed the non-inferiority of beclomethasone/formoterol (BDP/F) versus fluticasone propionate/salmeterol (FP/S) in adults with moderate to severe persistent asthma. However, this study did not provide evidence on costs and did not quantify quality-of-life parameters. OBJECTIVE: The objective of the present study was to assess the cost effectiveness and cost utility of BDP/F versus FP/S in patients with moderate to severe asthma from the perspective of the Italian National Health Service (NHS). METHODS: A Markov model (MM) was used, with five health states for the different levels of asthma control: successful control, sub-optimal control, outpatient-managed exacerbation, inpatient-managed exacerbation, and death. Model data were derived from the ICAT SE study and from expert panels. Three outcomes were considered: time spent in successful control state, costs and quality-adjusted life-years (QALYs). RESULTS: The model shows that BDP/F treatment led to a slight increase of weeks in successful control compared with FP/S, with a lower cost. The probabilistic sensitivity analysis highlights that in 64% and 68% of the Monte Carlo simulations, BDP/F outperformed FP/S in terms of weeks in successful control and QALYs. Considering the expected cost of the two strategies, in 90% of simulations BDP/F was the least expensive choice. In particular, BDP/F was cost saving as compared with FP/S in about 63% and 59% of simulations as shown by the cost-utility and cost-effectiveness analysis, respectively. CONCLUSION: Overall, from the Italian NHS perspective, BDP/F treatment is associated with a reduction in cost and offers a slight increase of effectiveness in terms of weeks spent in successful control and QALYs.