OBJECTIVES:

Participation in RWD registries is often constrained to institutions where patients are receiving care and which participate as sites. This limits enrollment to a handful of geographies, and often to academic institutions within these geographies. In addition to the physical distance, multiple factors affect participation, including the need to take time off work, to care for dependents and to understand the language of the registry. The research community is increasingly committed to developing solutions that can improve access and inclusion of patients in research. This is particularly important for rare diseases. We report 2 cases where web-based and multilingual approaches have accelerated patient enrollment and increased geographical and cultural diversity.

METHODS:

The Poland Syndrome Community Register (PSCR) and the CDKL5 Registry are hosted on a flexible, cloud-based centralized platform that upholds regulatory, security, and privacy requirements to support worldwide collaboration. With PSCR, the decision was made to launch a registry that could support participation globally, while remaining compliant with data regulations. The decision with CDKL5 was made to expand the web-based registry to 9 additional languages to attract patients in non-English speaking countries.

RESULTS:

The PSCR web-based registry was initiated in January 2022, and currently includes 203 participants across 24 countries. As for the CDKL5 registry, 106 participants have been enrolled across 35 countries since its launch in July 2018, of which 56% were enrolled since March 2022, when additional languages were included. Similar trends were observed in other rare diseases such as pediatric narcolepsy.

CONCLUSIONS:

Inclusion and diversity are enabled through decentralized and multi-lingual approaches. Participants can access educational material and visualize their data on dashboards, which supports retention. These approaches lead to more generalizable data for researchers. Insights from this work will inform future registry design.