Abstract
Currently, there is a discrepancy between clinical trials designed to assess the efficacy and safety of a new medication under investigation and the real-life questions that need to be addressed regarding the clinical use of the medication by patients, healthcare professionals and society. The data necessary to obtain regulatory approval may be of limited relevance to policy makers when calculating economic parameters such as value for money or cost effectiveness. ‘Real-world’ studies examine questions relevant to health policy and reimbursement. There are many different forms of clinical trials, but in designing trials incorporating realistic budget impact estimates the important issue is to ensure we are asking a sensible question and attempting to answer it with an appropriate experimental design. As an example, a real-world trial currently underway that examines scenarios of introducing inhaled insulin into clinical practice is described.
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Notes
The use of trade names is for product identification purposes only and does not imply endorsement.
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Acknowledgements
The authors sit on the steering committee of the ‘Real World Trial of Inhaled Insulin’ sponsored by Pfizer Inc and sanofi-aventis Group. Bjorn Bolinder and Robert A Gerber are employees of sanofi-aventis Group and Pfizer Inc, respectively. Nick Freemantle, Lawrence Blonde, Richard Hobbs, Luc Martinez and Stuart Ross have received fees and expenses from the sponsors of the Real World Trial.
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Freemantle, N., Blonde, L., Bolinder, B. et al. Real-world trials to answer real-world questions. Pharmacoeconomics 23, 747–754 (2005). https://doi.org/10.2165/00019053-200523080-00001
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DOI: https://doi.org/10.2165/00019053-200523080-00001