PD-MIND’s repurposing strategy offers significant time, cost and risk savings. Most importantly, the drug repurposing programme offered by PD-MIND is time-efficient. No pre-clinical research is involved, and a short follow-up period is sufficient taking into consideration the immediate effects expected on cognitive performance. The project follows and expands on the recommendations provided in a recent round-table meeting among Parkinson's experts, industry, and regulatory representatives.
The short trial duration will also positively affect timelines and costs for follow-up trials. PD-MIND proposes a considerably inexpensive trial set-up, as the primary outcome measures can be remotely assessed and analysed at a home setting, independent of the type and quality of the computer and internet connection used by participants, also providing a sensitive measurement of the most relevant cognitive domain, with a high signal-to-noise ratio. Potential risks are reduced by installation of a broad panel of cognition outcome measures, to optimise the detection rate of any cognitive improvement. Risk is further reduced by the implementation of a parallel design with 80 subjects in each arm, to reduce confounders and increase statistical power. Finally, biomarker studies may support the phase 3 clinical trial design by inclusion of biomarkers for patient (sub)diagnosis, stratification and outcome measurements, to focus this trial on target populations with highest anticipated effect sizes.