Crowdsourcing, citizen science and the future of clinical trials
Monday, October 24, 2016
The Pulse on Global Trials by Matthew Howes
The recent unveiling of OpenTrials.net marks an important milestone in the democratization of data. Bringing together multiple sources of clinical research information, from ClinicalTrials.gov, PubMed, EU CTR, WHO ICTRP and many others, this advanced search engine promises to bring the bourgeoning area of citizen science further into the mainstream.
Citizen science—scientific research conducted, in whole or in part, by amateur or nonprofessional scientists—represents a new frontier for clinical research. In a field that has well-established norms and methods, crowdsourcing necessitates the research sector to embrace a new mindset.
Today, data is ubiquitous, but it’s not visible to everyone. This limits the ability of patients, caregivers and healthcare professionals on the front lines of patient care from contributing good ideas to the advancement of science. In the future, clinical research will be powered by a far-reaching data infrastructure, much like an energy grid, where anyone plugged in can be both a consumer and generator of data.
SERMO, a professional online network of more than 600,000 healthcare professionals worldwide, is a good illustration of this model. In Canada, E. coli had killed a boy and two weeks later his sister presented with the same symptoms. Her doctor posted the case to SERMO, asking for input. Within hours, 46 doctors from around the world helped solve the case.
The ability to aggregate independent data points from multitudes of people has demonstrated effectiveness in other real-world applications. In 1999, world champion Garry Kasparov played a game of chess over the internet against a group of 50,000 people across 75 countries. A very close game, with Kasparov ultimately prevailing, the event revealed the power of crowdsourcing. While no single person could have competed that well against the expert, the combined knowledge of tens of thousands made for a formidable competitor. When responding to Kasparov’s moves, each person on the world team drew upon their individual experience. If one person had seen a particular pattern or configuration of the board, that knowledge rose to the top and was acted upon.
Over the course of the last two decades, clinical research has seen growing benefits from the application of crowdsourcing. In fact, PubMed now includes hundreds of crowdsourced studies. One example is an open challenge to find solutions to estimate the progression of Amyotrophic Lateral Sclerosis (ALS) or Lou Gehrig’s disease. The competition, with an award of $50,000, drew 37 groups from around the world—many with no ALS experience—to create better predictive tools that could make it easier to test new medications and their impact. The fresh thinking and alternative perspectives generated through crowdsourcing helped identify comparative strengths of competing methods.
In another study, scientists evaluated the viability of crowdsourcing to build data sets through the public health forums MedHelp and CureTogether. They found that crowd-labeled data was significantly more accurate than expert-labeled data (84% vs. 78%).
The most interesting changes, though, are when the public at large begins to harness the ubiquity of data to fuel humans’ ability to address unresolved scientific challenges. In this context, patients, caregivers and communities of shared purpose have found success initiating their own scientific inquiries.
Using the online platform PatientsLikeMe, two patients from Brazil and the U.S. with advanced-stage ALS recruited 149 patients with ALS to take lithium in order to test its effects on disease progression and symptom alleviation. The PatientsLikeMe ALS study, which was completed over eight months, was eventually published in Nature Biotechnology and provided the impetus for a standard clinical trial.
In another example, parents of children suffering from Niemann-Pick Type C, an extremely rare lipid storage disease, led extensive negotiations with the FDA for permission to administer cyclodextrin to their children, first intravenously, and then eventually directly into the spinal fluid. Their pursuit of a cure led to a phase I clinical trial of cyclodextrin.
Perhaps one of the most compelling stories is that of Dr. David Fajgenbaum, a young physician who became the patient, then lead researcher on a mission to cure Castleman’s disease. After nearly dying not once but five times from the disease, Fajgenbaum gave up his budding career as an OB-GYN to take residence at the University of Pennsylvania and form the Castleman Disease Collaborative Network (CDCN). Connecting hundreds of expert and non-expert researchers around the world through an online portal, he has been able to crowdsource a list of possible Castleman studies, prioritize them and fund about 20 different studies.
The implications of crowdsourcing for clinical trials are numerous. From developing study hypotheses, improving study design and optimizing analysis of data generated from study, to fundraising for underserved diseases, collaborating with peers and the public and increasing participation in clinical research, crowdsourcing represents a fundamental shift in global clinical research.
Matthew Howes is executive vice president, Strategy & Growth for PALIO, an inVentiv Health company. A leader in digital strategy, he has provided the fuel for digital businesses visited by more than 100 million people every month. Email email@example.com.
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