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Clinical trial design in haemophilia.

Dimichele, D M; Blanchette, V and Berntorp, Erik LU (2012) In Haemophilia 18 Suppl 4. p.18-23
Abstract
Summary:

Progress in the evidence-based care of haemophilia A and B worldwide has been historically challenged by the dearth of evaluable outcome data, including but not limited to the safety and effectiveness of therapeutic interventions. These challenges are partially rooted in the inherent difficulty of conducting prospective clinical trials and observational studies with statistically meaningful endpoints in a rare disease such as haemophilia. Despite the logistical barriers, the need for outcome data has never been more critical than in this time of expansive therapeutic advance tempered by the shrinking economic capacity to fund the rapidly increasing cost of treatment. Given that systematic analyses of published literature... (More)
Summary:

Progress in the evidence-based care of haemophilia A and B worldwide has been historically challenged by the dearth of evaluable outcome data, including but not limited to the safety and effectiveness of therapeutic interventions. These challenges are partially rooted in the inherent difficulty of conducting prospective clinical trials and observational studies with statistically meaningful endpoints in a rare disease such as haemophilia. Despite the logistical barriers, the need for outcome data has never been more critical than in this time of expansive therapeutic advance tempered by the shrinking economic capacity to fund the rapidly increasing cost of treatment. Given that systematic analyses of published literature have been largely unsuccessful in compensating for the lack of rigorous and purposeful data collection, new approaches to clinical study design and statistical modelling are urgently needed. However, even as these are considered, the lack of broadly accepted and well-defined clinical outcome endpoints poses an additional barrier to progress. The three presentations encompassed by this paper highlight the timely need for quality data from the perspectives of the clinicians, regulatory agencies and health care funders, and describe the ongoing coordinated efforts by the international haemophilia community to further understand and dismantle the barriers to harmonized and standardized data collection on a global scale using well-defined clinical outcome endpoints. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Haemophilia
volume
18 Suppl 4
pages
18 - 23
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • wos:000305605400003
  • pmid:22726077
  • scopus:84862889106
ISSN
1351-8216
DOI
10.1111/j.1365-2516.2012.02824.x
language
English
LU publication?
yes
id
22b33636-b16b-49c4-8b9a-0bc18fe7cf46 (old id 2858931)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/22726077?dopt=Abstract
date added to LUP
2012-07-04 19:46:52
date last changed
2017-10-01 04:57:25
@article{22b33636-b16b-49c4-8b9a-0bc18fe7cf46,
  abstract     = {Summary:<br/><br>
Progress in the evidence-based care of haemophilia A and B worldwide has been historically challenged by the dearth of evaluable outcome data, including but not limited to the safety and effectiveness of therapeutic interventions. These challenges are partially rooted in the inherent difficulty of conducting prospective clinical trials and observational studies with statistically meaningful endpoints in a rare disease such as haemophilia. Despite the logistical barriers, the need for outcome data has never been more critical than in this time of expansive therapeutic advance tempered by the shrinking economic capacity to fund the rapidly increasing cost of treatment. Given that systematic analyses of published literature have been largely unsuccessful in compensating for the lack of rigorous and purposeful data collection, new approaches to clinical study design and statistical modelling are urgently needed. However, even as these are considered, the lack of broadly accepted and well-defined clinical outcome endpoints poses an additional barrier to progress. The three presentations encompassed by this paper highlight the timely need for quality data from the perspectives of the clinicians, regulatory agencies and health care funders, and describe the ongoing coordinated efforts by the international haemophilia community to further understand and dismantle the barriers to harmonized and standardized data collection on a global scale using well-defined clinical outcome endpoints.},
  author       = {Dimichele, D M and Blanchette, V and Berntorp, Erik},
  issn         = {1351-8216},
  language     = {eng},
  pages        = {18--23},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Haemophilia},
  title        = {Clinical trial design in haemophilia.},
  url          = {http://dx.doi.org/10.1111/j.1365-2516.2012.02824.x},
  volume       = {18 Suppl 4},
  year         = {2012},
}