title: Beyond patient benefit: clinical development in hemophilia.
abstract: Historically in hemophilia, outcome measures have not been collected systematically. Hence, there are insufficient clearly defined, evidence-based measures that can be applied consistently across hemophilia trials. This review focuses on some key challenges to evaluating patient outcomes  and performing trials identified by experts at the Fourth and Fifth Zurich Haemophilia Forums. As procedures appear inconsistent across Europe, guidelines require modification to be more appropriate and/or realistically achievable. The outcome measures utilized, and the timing  of their collection, should also be standardized, and more objective measures used where feasible. Implementation of outcome measures could be refined through greater understanding of patient heterogeneity, and tailored to differentiate between hemophilia- and aging-related disease effects.  Furthermore, robust outcome measures that can also inform health-economic decisions are increasingly needed. Lastly, as patient recruitment poses a challenge, the panel proposed a call for action to motivate physicians and patients to participate in clinical trials.
authors.affiliation: Klinikum Bremen-Mitte, Professor Hess Children's Hospital, Bremen, Germany. guenterauerswald@aol.com
authors.forename: Günter
authors.initials: G
authors.lastname: Auerswald
authors.forename: Silva Zupančić
authors.initials: SZ
authors.lastname: Šalek
authors.forename: Gary
authors.initials: G
authors.lastname: Benson
authors.forename: Ivo
authors.initials: I
authors.lastname: Elezović
authors.forename: Thierry
authors.initials: T
authors.lastname: Lambert
authors.forename: Massimo
authors.initials: M
authors.lastname: Morfini
authors.forename: John
authors.initials: J
authors.lastname: Pasi
authors.forename: Eduardo
authors.initials: E
authors.lastname: Remor
authors.forename: Elena
authors.initials: E
authors.lastname: Santagostino
authors.forename: Peter
authors.initials: P
authors.lastname: Salaj
authors.forename: Rolf
authors.initials: R
authors.lastname: Ljung
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D006467:Hemophilia A / Q000628:therapy*; D006801:Humans; D017063:Outcome Assessment, Health Care / Q000379:methods; D011788:Quality of Life