Professor Hanns Lochmüller
As with any rare condition, one of the biggest hurdles in a neuromuscular trial is frequently patient recruitment. Particularly for large-scale pivotal studies, recruiting the numbers of patients necessary to adequately power the trial can take lengthy amounts of time and consume large quantities of resources. With therapies only addressing a certain mutation type or certain subset of a population, the difficulties are even more evident.
The TREAT-NMD patient registries were designed to address precisely this problem. The genetic and clinical information they contain enables filtering of patients by inclusion/exclusion criteria, which means that once those criteria have been established, you can find out patient numbers meeting all criteria country by country or region by region, and then reach out to those very patients through the registries as a trusted intermediary, knowing that:
- Patients in the registry have already consented to be contacted if they might be eligible for a trial
- Patient details are regularly updated, so you can be confident that those patients still meet the inclusion criteria
- Genetic data in the registries is accurate and reliable, so you are reaching out to the right patients
This recruitment effort through the registries can work in parallel with the recruitment efforts of the trial sites involved, ensuring that you reach out to all patients in a particular area even if their records are not held by the trial site.