Artikel
Innovative Methodology to Translate Conditions of Daily Practice into an Evidence Based Study Design: Example of a randomized feasibility trial to compare a world where inhaled insulin (INH) is available with a world without INH
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Veröffentlicht: | 30. September 2004 |
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Gliederung
Text
Introduction
„Conventional" clinical trials are required to obtain regulatory approval for new medication. However, in these „head to head" clinical trials new medication shows often the same efficacy as standard treatment but may have beneficial effects under conditions of daily practice. So daily practice should be translated into clinical trials. Alternative study designs are needed. A clinical trial to compare a „world" where a new medication is available with a „world" without this option could help to show beneficial effects.
Aim of the Study
1. Create a evident study design to show beneficial effects of a new form of application in comparison to standard treatment under conditions of daily practice.
2. Test the design in a pre-study (feasibility trial)
3. Estimate the proportion of patients who prefer this novel treatment options in comparison to standard therapy (needed for clinical trial sample size calculation).
Methods
T2Ds failing OAD were randomly (patient based) assigned to 2 groups (A / B). Both groups received the same educational material regarding the available treatment options. In addition group A received information on INH. The decision regarding future treatment was made during an interactive discussion of patients and physicians.
Analysis
Proportions were analyzed using Fischer's exact test, and described using the odds ratio and theoretically exact 95% confidence intervals (CIs).
Results
779 patients (Group A = 391, Group B = 388) were recruited. 169 patients (43.2%) in group A opted for a treatment that included insulin, in comparison with 60 patients (15.5%) in group B; odds ratio 4.16 (95% CI 2.93 to 5.95; p < 0.0001). The proportions, together with published results of the different treatment options, permit sample size calculation. The limitation of the study is that the decision was only theoretical. There was no drug intervention.
Conclusion
The described method is suitable for translating conditions of daily practice into an evidence based study design (randomization). The results require confirmation in a large clinical trial including drug intervention. The study design seems to be suitable to show beneficial effects of new medication, not seen in conventional clinical trials, also in other therapeutic areas.
Conflict of interest: Sponsor of the Study were Pfizer Inc./USA and Aventis Inc./USA.