Amyotrophic lateral sclerosis, commonly known as ALS, is an enduring mystery for life scientists–even those who have dedicated their careers to understanding it and finding a cure. Terminal, essentially untreatable, and frustratingly hard to predict, the disease has eluded us for years–and has achieved a sort of “orphan” status that makes it hard to draw attention and research dollars to ALS.
But an innovative new approach, drawing on the disciplines of both medical science and data science, offers the promise of an unprecedented breakthrough in the search for a cure. For the first time, Big Pharma and Big Data are coming together to foster insights in ALS. Specifically, working with nonprofits like mine, Teva ($TEVA), Novartis ($NVS), Sanofi ($SNY), Regeneron ($REGN), Knopp Biosciences, and other companies have opened up their clinical trials databases to help create the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database, an open-access repository of ALS patient and clinical trial data made available to researchers, patients, and members of the public. Although recent efforts by Big Pharma’s two major trade groups, EFPIA and PhRMA, will increase the amount of clinical trial data available in the future, PRO-ACT has already vastly increased the breadth and depth of ALS clinical data available.
Why ALS needs an open-access database
But just because we’ve built it, does that mean the researchers–and the breakthroughs–will come?
We think the answer is “yes.” Here’s why: Previously, access to ALS clinical trials data was limited and piecemeal. Scientists interested in seeking a better understanding of past drug trials were frequently limited to a single trial or a handful of small studies, and even these data were often limited to placebo arms or a small subset of measures. This lack of data access meant that important questions about disease natural history and disease heterogeneity were unanswerable. Clinician-scientists interested in designing clinical trials of novel treatments were operating with the equivalent of one hand tied behind their backs with all of that valuable data out of reach.