title: What Can Big Data Offer the Pharmacovigilance of Orphan Drugs?
abstract: The pharmacovigilance of drugs for orphan diseases presents problems related to the small patient population. Obtaining high-quality information on individual reports of suspected adverse reactions is of particular importance for the pharmacovigilance of orphan drugs. The possibility of mining "big data" to detect suspected adverse reactions is being explored in pharmacovigilance generally but may have limited application to orphan drugs. Sources of big data such as social media may be infrequently used as communication channels by patients with rare disease or their caregivers or by health care providers; any adverse reactions identified are likely to reflect what is already known about the safety of the drug from the network of support that grows up around these patients. Opportunities related to potential future big data sources are discussed.
authors.affiliation: Alexion Pharmaceuticals, Inc, New Haven, Connecticut. Electronic address: john.price@alexion.com.
authors.forename: John
authors.initials: J
authors.lastname: Price
mesh_terms: D016907:Adverse Drug Reaction Reporting Systems; D057225:Data Mining; D064420:Drug-Related Side Effects and Adverse Reactions / Q000517:prevention & control; D006801:Humans; D016247:Information Storage and Retrieval; D009965:Orphan Drug Production; D060735:Pharmacovigilance; D035583:Rare Diseases / Q000188:drug therapy*; D012944:Social Support
keywords: big data; drug safety; orphan drug; pharmacovigilance; rare disease; social media