title: Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm.
abstract: Patients with serious diseases may experiment with drugs that have not received regulatory approval. Online patient communities structured around quantitative outcome data have the potential to provide an observational environment to monitor such drug usage and its consequences. Here we describe an analysis of data reported on the website PatientsLikeMe by patients with amyotrophic lateral sclerosis (ALS) who experimented with lithium carbonate treatment. To reduce potential bias owing to lack of randomization, we developed an algorithm to match 149 treated patients to multiple controls (447 total) based on the progression of their disease course. At 12 months after treatment, we found no effect of lithium on disease progression. Although observational studies using unblinded data are not a substitute for double-blind randomized control trials, this study reached the same conclusion as subsequent randomized trials, suggesting that data reported by patients over the internet may be useful for accelerating clinical discovery and evaluating the effectiveness of drugs already in use.
authors.affiliation: Research and Development, PatientsLikeMe Inc., Cambridge, Massachusetts, USA. pwicks@patientslikeme.com
authors.forename: Paul
authors.initials: P
authors.lastname: Wicks
authors.forename: Timothy E
authors.initials: TE
authors.lastname: Vaughan
authors.forename: Michael P
authors.initials: MP
authors.lastname: Massagli
authors.forename: James
authors.initials: J
authors.lastname: Heywood
mesh_terms: D000465:Algorithms; D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy*; D016022:Case-Control Studies; D005260:Female; D006801:Humans; D020407:Internet; D016651:Lithium Carbonate / Q000097:blood / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D019782:Riluzole / Q000008:administration & dosage; D012307:Risk Factors; D057566:Self Report; D017678:Sex Distribution; D016896:Treatment Outcome
chemical_list: D016651:Lithium Carbonate; D019782:Riluzole