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Issues in assessing products for the treatment of hemophilia – the intersection between efficacy, economics, and ethics

Authors Farrugia A, Noone D, Schlenkrich U, Schlenkrich S, O'Mahony B, Cassar J

Received 6 March 2015

Accepted for publication 21 April 2015

Published 15 June 2015 Volume 2015:6 Pages 185—195

DOI https://doi.org/10.2147/JBM.S79091

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 3

Editor who approved publication: Dr Martin Bluth

Video abstract presented by Albert Farrugia

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Albert Farrugia,1,2 Declan Noone,3 Uwe Schlenkrich,4 Steffen Schlenkrich,5 Brian O'Mahony,3 Josephine Cassar6

1School of Surgery, QEII Medical Centre, The University of Western Australia (M509), Crawley, WA, Australia; 2College of Medicine, Medicine and Environment, Australian National University, Canberra, WA, Australia; 3Irish Haemophilia Society, Dublin, Ireland; 4Deutsche Hämophiliegesellschaft (DHG), Hamburg, Germany; 5Am Holzbiel, Erfurt-Töttleben, Germany; 6Faculty of Health, University of Canberra, Canberra, WA, Australia

Abstract: Following the obviation of the pathogen safety threats posed by previous generations of clotting factor concentrates for the treatment of hemophilia, the principal issue facing the patient community is timely access to adequate supplies of continuously improving therapies. The application of evidence-based medicine has enhanced the basis of hemophilia therapy, while resulting in some challenges to patient care. Increasingly, the criteria used for the approval and payment of treatment products by regulatory and reimbursement agencies, respectively, are becoming inflexible and unrealistic. This is occurring particularly in the requirements for demonstrating product efficacy. Concurrently, emerging evidence of the interpatient variability in the clinical response to therapy has led to the proposed personalization of therapeutic regimens. Possible impediments to optimal care include competitive tensions among suppliers who seek to gain label claims for reimbursement purposes, which result in clinical trial designs of, arguably, unethical design, carried out in poor countries. We synthesize these converging developments to suggest some changes to the current hemophilia treatment paradigm, which should make it more patient-centric and enable speedier access to new therapies.

Keywords: hemophilia, treatment products, clinical trials, standards, reimbursement

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