Clinical trial design – your key to successful proof-of-concept!

The goal of many drug development projects is to exit after phase I or phase II – the proof-of concept phase. Probably the main reason for this is the immense cost of drug development beyond phase I/II and that only the larger pharmaceutical companies have the muscles, financially and operationally, to take on phase III projects.
During early development, time and resources are spent on formulation development, preclinical planning and conduct of phase I/II studies. Nevertheless, at the end of the project, the crucial results for a successful exit is the phase II data (or phase I). All money and efforts spent on conducting the clinical study to the highest standard will be condensed into a final Clinical Study Report (CSR). Your CSR is your final product which needs to be perfect. One may say that all steps on the way towards the shining CSR is just means towards the goal.
There are a few things to consider carefully when planning your clinical study, to get the most out of your investments and efforts.

• Focus on trial design – an effective choice of design, suitable endpoints and planning with the desired end result in mind will give you a stronger proof-of-concept.

• Proof-of-concept studies usually use surrogate endpoints. The reason for this is that is that surrogate endpoints are chosen to generate earlier answers than hard clinical endpoints do and provide higher statistical power. A simple example is to measure proteinuria in diabetics as a measure of kidney function instead of waiting for kidney failure and need for transplantation.

• Statisticians may be focused on statistics and numbers, but they are your BEST FRIENDS when it comes to plan for a solid proof-of-concept. Seemingly insignificant choices with regards to endpoints and study design may have a considerable impact on the results.

• Planning is key to a successful analysis. In the best of worlds your Statistical Analysis Plan has been drafted before the launch of your project.

• Operational excellence is not so sexy. However, a study which fails to recruit patients because the study design is not well aligned with clinical practice is even less attractive.

If you are interested to hear how our experienced team of statisticians can work their magic for your trial design? Give us a call.