Background: The Cryopyrin-Associated Periodic Syndromes (CAPS) are a group of rare hereditary autoinflammatory
diseases and encompass Familial Cold Autoinflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), and
Neonatal Onset Multisystem Inflammatory Disease (NOMID). Canakinumab is a monoclonal antibody directed
against IL-1 beta and approved for CAPS patients but requires post-approval monitoring due to low and short
exposures during the licensing process. Creative approaches to observational methodology are needed, harnessing
novel registry strategies to ensure Health Care Provider reporting and patient monitoring.
Methods: A web-based registry was set up to collect information on long-term safety and effectiveness of
canakinumab for CAPS.
Results: Starting in November 2009, this registry enrolled 241 patients in 43 centers and 13 countries by December
31, 2012. One-third of the enrolled population was aged < 18; the overall population is evenly divided by gender.
Enrolment is ongoing for children.
Conclusions: Innovative therapies in orphan diseases require post-approval structures to enable in depth
understanding of safety and natural history of disease. The rarity and distribution of such diseases and
unpredictability of treatment require innovative methods for enrolment and follow-up. Broad international practice-based
recruitment and web-based data collection are practical.