#96 2006 · PatientsLikeMe (Heywood brothers) · Healthcare / patient datalegibility
Patients logging their own symptoms online did in months what a randomized clinical trial would have taken years to attempt — and it refuted a real drug claim
the problem
A rare disease produces too few patients and too slow a trial pipeline for any single doctor or study to see the real pattern across cases
background
Stephen Heywood was diagnosed with ALS in 1998, a disease with no cure and, at the time, no practical way for a patient or their family to see how their disease course compared to anyone else's — doctors saw only their own small caseload, and clinical trials moved far too slowly to help an individual patient make real-time treatment decisions. His brothers Jamie and Ben Heywood, with a former MIT classmate, watched this information vacuum firsthand and set out to build something doctors and trials weren't providing: a way for patients to see their disease in the context of thousands of others like them.
The standard tools for understanding a rare, slow-moving disease were the standard tools of clinical research generally — small trials, peer-reviewed publication, years-long timelines — none of which could answer an urgent question an individual patient actually needed answered quickly, like whether a promising new treatment showing up in early research was actually working for people taking it right now.
the move
PatientsLikeMe, founded in 2006, had patients themselves log structured symptom, treatment and side-effect data over time, building a dataset out of exactly the kind of unremarkable, individually invisible records that only reveal a pattern in aggregate. When a small 2008 Italian study suggested lithium might slow ALS progression, roughly 10% of PatientsLikeMe's ALS users started taking it on their own rather than wait for a formal trial — and the platform's researchers built an algorithm matching 149 lithium-treated patients against 447 non-treated controls with similar disease trajectories, using the patients' own logged data to run what amounted to an informal observational trial in real time.
the payoff
The matched-cohort analysis found no effect of lithium on ALS disease progression at 12 months, refuting the earlier published claim years before a formal randomized trial would have reached the same conclusion — the study, published in Nature Biotechnology in 2011, was among the first demonstrations that patient-reported data collected online could accelerate drug evaluation. By the time of that publication PatientsLikeMe had grown to more than 100,000 patients tracking over 500 conditions.
what came after
PatientsLikeMe's lithium study is cited as a landmark early proof that patient-generated real-world data can meaningfully contribute to clinical research, and the platform's model — patients as active data contributors rather than passive trial subjects — influenced the broader real-world-evidence movement in healthcare research and regulatory science that followed.
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references
- [1]Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithmNature Biotechnology (via PubMed), 2011pubmed.ncbi.nlm.nih.gov
- [2]Data from Patient Social Network Refutes Lithium for ALSMIT Technology Review, 2011technologyreview.com