Extracorporeal Photopheresis for Second-Line Treatment of Chronic Graft-versus-Host Diseases: Results from a Health Technology Assessment in Italy

Chiara de Waure; Stefano Capri; Maria Assunta Veneziano; Maria Lucia Specchia; Chiara Cadeddu; Francesco Di Nardo; Anna Maria Ferriero; Francesca Gennari; Colette Hamilton; Agostino Mancuso; Gianluigi Quaranta; Matteo Raponi; Luca Valerio; Gianfranco Gensini; Walter Ricciardi

doi:10.1016/j.jval.2015.01.009

Extracorporeal Photopheresis for Second-Line Treatment of Chronic Graft-versus-Host Diseases: Results from a Health Technology Assessment in Italy

Value Health. 2015 Jun;18(4):457-66. doi: 10.1016/j.jval.2015.01.009. Epub 2015 Apr 16.

Authors

Affiliations

¹ Section of Hygiene, Institute of Public Health, Catholic University of the Sacred Heart, Rome, Italy. Electronic address: chiara.dewaure@rm.unicatt.it.
² Section of Hygiene, Institute of Public Health, Catholic University of the Sacred Heart, Rome, Italy; School of Economics and Management, LIUC University, Castellanza (VA), Italy.
³ Section of Hygiene, Institute of Public Health, Catholic University of the Sacred Heart, Rome, Italy.
⁴ Therakos, Inc., West Chester, PA, USA.
⁵ Faculty of Medicine, Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy.

PMID: 26091600
DOI: 10.1016/j.jval.2015.01.009

Abstract

Objectives: To develop a comparative, cost-effectiveness, and budget impact analysis of Therakos online extracorporeal photopheresis (ECP) compared with the main alternatives used for the treatment of steroid-refractory/resistant chronic graft-versus-host disease (cGvHD) in Italy.

Methods: The current therapeutic pathway was identified by searching medical databases and from the results of a survey of practice in Italian clinical reference centers. A systematic review was performed to evaluate the efficacy and safety of second-line alternatives. Budget impact and cost-effectiveness analyses were performed from the Italian National Health Service perspective over a 7-year time horizon through the adaption of a Markov model. The following health states were considered: complete and partial response, stable disease, and progression. A discount rate of 3% was applied to costs and outcomes.

Results: The most common alternatives used in Italy for the management of steroid-refractory/resistant cGvHD were ECP, mycophenolate, pentostatin, and imatinib. The literature review highlighted that complete and partial responses are higher with ECP than with the alternatives while serious adverse events are less common. The economic analysis showed that Therakos online ECP represents the dominating alternative, in that it delivers greater benefit at a lower cost. In fact, according to the alternatives considered, cost saving ranged from €3237.09 to €19,903.51 per patient with 0.04 to 0.21 quality-adjusted life-year gained.

Conclusions: Therakos online ECP should be considered an effective, safe, and cost-effective alternative in steroid-refractory/resistant cGvHD. There is inequality in access, and a dedicated reimbursement tariff, however, should be introduced to overcome these barriers.

Keywords: Markov model; cost-effectiveness analysis; economic evaluation; graft-versus-host disease; photopheresis; technology assessment.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Chronic Disease
Cost-Benefit Analysis / methods
Cost-Benefit Analysis / standards
Female
Graft vs Host Disease / economics
Graft vs Host Disease / epidemiology*
Graft vs Host Disease / therapy*
Humans
Italy / epidemiology
Male
Photopheresis / economics
Photopheresis / methods*
Photopheresis / standards
Technology Assessment, Biomedical / methods*
Technology Assessment, Biomedical / standards
Treatment Outcome