The European Medicines Agency and the Brave New World of Access to Clinical Trial Data

Steinbrook, Robert

doi:10.1001/jamainternmed.2013.3842

Cited by 8 publications

(2 citation statements)

References 6 publications

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“…Finally, we note, the European Medicines Agency has stated its commitment to publishing clinical trial information on all drugs submitted as part of the licensing process, whether or not they are approved . As emphasized previously, this information would be invaluable when forming a surrogate outcome model in the MS setting and other disease areas.…”

Section: Discussionmentioning

confidence: 90%

A Bayesian hierarchical surrogate outcome model for multiple sclerosis

Pozzi

Schmidli

Ohlssen

2016

Pharmaceutical Statistics

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The development of novel therapies in multiple sclerosis (MS) is one area where a range of surrogate outcomes are used in various stages of clinical research. While the aim of treatments in MS is to prevent disability, a clinical trial for evaluating a drugs effect on disability progression would require a large sample of patients with many years of follow-up. The early stage of MS is characterized by relapses. To reduce study size and duration, clinical relapses are accepted as primary endpoints in phase III trials. For phase II studies, the primary outcomes are typically lesion counts based on magnetic resonance imaging (MRI), as these are considerably more sensitive than clinical measures for detecting MS activity. Recently, Sormani and colleagues in 'Surrogate endpoints for EDSS worsening in multiple sclerosis' provided a systematic review and used weighted regression analyses to examine the role of either MRI lesions or relapses as trial level surrogate outcomes for disability. We build on this work by developing a Bayesian three-level model, accommodating the two surrogates and the disability endpoint, and properly taking into account that treatment effects are estimated with errors. Specifically, a combination of treatment effects based on MRI lesion count outcomes and clinical relapse was used to develop a study-level surrogate outcome model for the corresponding treatment effects based on disability progression. While the primary aim for developing this model was to support decision-making in drug development, the proposed model may also be considered for future validation. Copyright © 2016 John Wiley & Sons, Ltd.

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Section: Discussionmentioning

confidence: 90%

A Bayesian hierarchical surrogate outcome model for multiple sclerosis

Pozzi

Schmidli

Ohlssen

2016

Pharmaceutical Statistics

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“…Therefore, it has taken the stance that trial results are nonproprietary and is moving steadily toward requiring proactive publication of all clinical trial data for drugs it reviews. 38 Even manufacturers are beginning to acknowledge that, far from undermining innovation, greater disclosure of clinical trial data represents a potential boon to their research and development efforts. 39,40 It is incumbent on Canada to move in the same direction.…”

Section: Outline Clear Limits To the Scope Of Proprietary Informationmentioning

confidence: 99%