N-of-1 trials for epilepsy: supported by mobile-health application and statistical software

Precision treatments In moNogenic ePilepsies: Observational regIstry and N-of-1 Trial recommendations (PINPOINT)

PINPOINT aims to create a shift in the therapeutic approach of (monogenic) epilepsies by providing an alternative to upgrade the quality of evidence on treatment efficacy using single-patient (n-of-1) trials. In PINPOINT, tailored n-of-1 methodology for epilepsy will facilitate evidence-based and individualised treatment selection. They go beyond proposing a methodology and partner with University of Amsterdam (JASP) and Neuroventis BV to deliver digital tools to support the implementation of n-of-1 trials.

Epilepsy is a debilitating brain condition affecting approximately 70 million individuals worldwide. More than 30% of patients with epilepsy suffer from treatment resistant seizures. While treatment guidelines are available for some forms of epilepsy, in practice individuals may respond differently to treatments or be burdened by comorbid symptoms. Personalised treatment selection, suited to the underlying cause of epilepsy or to specific symptoms, is a promising alternative requiring a systematic approach to assess therapy efficacy objectively.

In an n-of-1 trial each patient follows a series of treatment periods (cycle) with a randomly allocated treatment per cycle, thereby serving as their own control. Single-patient trials are well-suited for use in research and by practitioners searching for the optimal treatment for those with rare conditions, and when treatments effects or symptoms are highly variable between patients. The tailored n-of-1 methodology will provide the tools to conduct n-of-1 trials for a range of epilepsies to improve individualised treatment selection. To enhance the quality and relevance of n-of-1 trials we develop two user-friendly digital modules: (a) a patient-centered outcome measurement in a mobile-health application and (b) user-friendly statistical analysis software.

In PINPOINT they exemplify evidence-based, personalised treatment selection in proof-of-principle n-of-1 trials using tailored n-of-1

Summary

Efficacy of epilepsy therapies can differ per patient. N-of-1 studies are single-patient crossover trials using an individual as the sole unit of observation to establish efficacy and tolerability of a treatment. Here, we develop the tailored n-of-1 trial methodology and the supporting mobile-health application and user-friendly statistical software.

Technology Readiness Level (TRL)

2 - 5

Time period

18 months

Partners