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Beyond patient benefit: clinical development in hemophilia

Auerswald, Guenter; Salek, Silva Zupancic; Benson, Gary; Elezovic, Ivo; Lambert, Thierry; Morfini, Massimo; Pasi, John; Remor, Eduardo; Santagostino, Elena and Salaj, Peter, et al. (2012) In Hematology 17(1). p.1-8
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... (More)
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. (Less)
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type
Contribution to journal
publication status
published
subject
keywords
Endpoints, Hemophilia, Inhibitors, Outcome measures, Health-related, quality of life
in
Hematology
volume
17
issue
1
pages
1 - 8
publisher
Informa Healthcare
external identifiers
  • wos:000303745700001
  • scopus:84863103599
ISSN
1607-8454
DOI
10.1179/102453312X13221316477372
language
English
LU publication?
yes
id
c7c6dd61-e35f-4bf5-8330-f6ec308bc13c (old id 2562876)
date added to LUP
2012-06-01 08:44:32
date last changed
2017-09-17 03:28:55
@article{c7c6dd61-e35f-4bf5-8330-f6ec308bc13c,
  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.},
  author       = {Auerswald, Guenter and Salek, Silva Zupancic and Benson, Gary and Elezovic, Ivo and Lambert, Thierry and Morfini, Massimo and Pasi, John and Remor, Eduardo and Santagostino, Elena and Salaj, Peter and Ljung, Rolf},
  issn         = {1607-8454},
  keyword      = {Endpoints,Hemophilia,Inhibitors,Outcome measures,Health-related,quality of life},
  language     = {eng},
  number       = {1},
  pages        = {1--8},
  publisher    = {Informa Healthcare},
  series       = {Hematology},
  title        = {Beyond patient benefit: clinical development in hemophilia},
  url          = {http://dx.doi.org/10.1179/102453312X13221316477372},
  volume       = {17},
  year         = {2012},
}