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Orphanet J Rare Dis. 2013 Sep 10;8:139. doi: 10.1186/1750-1172-8-139.

Methodological challenges in monitoring new treatments for rare diseases: lessons from the cryopyrin-associated periodic syndrome registry.

Author information

1
Novartis Pharma AG, Basel, Switzerland. paola.primatesta@novartis.com.

Abstract

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.

PMID:
24016338
PMCID:
PMC3848430
DOI:
10.1186/1750-1172-8-139
[Indexed for MEDLINE]
Free PMC Article
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