title: Issues in assessing products for the treatment of hemophilia - the intersection between efficacy, economics, and ethics.
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.
authors.affiliation: School of Surgery, QEII Medical Centre, The University of Western Australia (M509), Crawley, WA, Australia ; College of Medicine, Medicine and Environment, Australian National University, Canberra, WA, Australia.
authors.forename: Albert
authors.initials: A
authors.lastname: Farrugia
authors.affiliation: Irish Haemophilia Society, Dublin, Ireland.
authors.forename: Declan
authors.initials: D
authors.lastname: Noone
authors.affiliation: Deutsche Hämophiliegesellschaft (DHG), Hamburg, Germany.
authors.forename: Uwe
authors.initials: U
authors.lastname: Schlenkrich
authors.affiliation: Am Holzbiel, Erfurt-Töttleben, Germany.
authors.forename: Steffen
authors.initials: S
authors.lastname: Schlenkrich
authors.affiliation: Irish Haemophilia Society, Dublin, Ireland.
authors.forename: Brian
authors.initials: B
authors.lastname: O'Mahony
authors.affiliation: Faculty of Health, University of Canberra, Canberra, WA, Australia.
authors.forename: Josephine
authors.initials: J
authors.lastname: Cassar
keywords: clinical trials; hemophilia; reimbursement; standards; treatment products