Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm.

Area of Science:

• Neurology
• Pharmacology
• Health Informatics

Background:

• Patients with serious diseases often experiment with unapproved drugs.
• Online patient communities offer a platform for monitoring drug usage and outcomes.

Purpose of the Study:

• To analyze the effectiveness of lithium carbonate in amyotrophic lateral sclerosis (ALS) patients using online data.
• To assess the utility of patient-reported outcomes for clinical discovery.

Main Methods:

• Analysis of data from PatientsLikeMe for ALS patients treated with lithium carbonate.
• Development of an algorithm to match treated patients with disease-matched controls to minimize bias.

Main Results:

• No significant effect of lithium carbonate on ALS disease progression was observed at 12 months.
• The study's findings aligned with subsequent randomized controlled trials.

Conclusions:

• Patient-reported data from online communities can be valuable for accelerating clinical discovery.
• Internet-based observational studies can aid in evaluating drug effectiveness, complementing traditional trials.