Clinical trials are crucial to validating interventions that make health care treatments better, faster and cheaper. Yet, the trials themselves are anything but. Testing firms “recognize that running clinical trials the way they’re run today is too costly, takes too much time, takes too much effort, has too high of a failure rate and is an unsustainable business model,” says Rob Franco, a principal with consulting firm PwC.
Enter mHealth. Or not.
The tools of mobile health can make data more available and plentiful at a much lower cost, says Franco, whether it’s automatically monitoring and collecting the trial data, enabling frequent assessment of safety and efficacy, or providing convenience that makes it less likely enrollees will drop out. “The promise of adding new technology, especially informatics and mobile devices, is right now viewed as [an advance] that could dramatically change the way clinical trials are run.”
But doing things differently instead of following a long-standing, familiar pathway is always a risk, and the reaction to mHealth among contract research organizations, academic medical centers and pharmaceutical firms ranges from skittish to downright resistant, experts say. The high stakes inherent in a multimillion-dollar, eight- to 10-year proposition discourages taking a chance on some promising, but uncertain, new approach, says Donald Jones, chief digital officer of Scripps Translational Science Institute, La Jolla, Calif.
“There’s a basic, high-level, somewhat unspoken premise about regulatory trials, which is, ‘Ask the questions you need to, don’t ask the questions you don’t need to.’ Because the goal is to get approved, right?” Jones says. “The goal isn’t to find new theories you could pursue. When you use devices that collect a lot of data, it opens the possibility that it might raise more questions.” In addition to the burning questions about a new intervention, trials executives have to explain why a process that reduces face-to-face encounters and automates data collection is safe and sound.
One player in the trials field trying to piece together a track record for mHealth is also a big player in remote patient monitoring and other mobile device systems. AMC Health, based in New York City, currently is involved in five trials that include interventions for multiple sclerosis, Parkinson’s disease, pulmonary sarcoidosis and prostate cancer, says John Holland, senior vice president for research and business development.
A major obstacle to organizing trials for such specialized medical targets is recruitment and retention. It’s difficult for trial investigators to get enough enrollees initially and when they do, people drop out. Patients not only have to take medications dutifully, but also must travel to a trial site for periodic assessment and testing. “There’s sort of a rule of thumb that if somebody lives more than 20 miles or 20 minutes from a study site, they’re probably too far away to expect them to do monthly visits for a year or two,” Holland says. “So each study site has a rather narrow area to recruit from.”
Remote monitoring tools combined with patient contact through video “visits” can overcome that obstacle. For example, in AMC Health’s prostate cancer study based at Mount Sinai Hospital in New York City, six individuals who meet the study criteria are from Albany. But they only make the trip downstate once, Holland says, for an initial explain-everything visit, and then are managed through remote measurements and telemedicine. “If they had to come in six times, it’s unlikely that we would have gotten all those patients.”
Then there is the “adaptive” potential, or ability to modify protocols and procedures based on expeditious data collection, Franco says. The real-time availability of results via mHealth creates latitude to examine and analyze a steady stream of data to catch safety concerns early or adjust the trial based on early evidence of impact on patients [see case studies].
A number of biotechnology companies “have been exploring innovating the clinical trial process with the goal of trying to eliminate as much as half the cost of clinical trials,” says Christopher Wasden, an authority on mHealth and executive director of University of Utah’s Sorenson Center for Discovery and Innovation. “And when you break down where those costs are, they are in areas that lend themselves to digital health innovation.”
The breakthrough in adoption, Holland concludes, won’t stem as much from the capabilities of mHealth tools themselves as they will from evidence that they do what their advocates say they can, and that means successful trials properly proven and published. “Everybody says, ‘This is a great idea; when you’ve done four or five of them, give me a call.’ ”
Clinical trial participants are selected with great care to exhibit just the right physical traits and medical conditions that an investigational drug or specific clinical process is intended to make better. But the fact is that similar patients are affected differently, and that has implications for gauging effectiveness and preventing harm. The sooner these effects play out and become known, the better.
Using mobile devices to monitor and send information can quicken and sharpen the ability of academic medical institutions to modify trials, increase patient benefit and reduce patient risk, says Franco of PwC. For example, by detecting various human responses, investigators can identify patients with a particular physiological profile who are doing better on the tested drug than others. The criteria can be changed “to try to get more of those kinds of patients into the trial, exclude the patients who don’t do as well, in a real-time manner,” he says.
The drug company benefits by better pinpointing those for whom the medication is most effective. Providers serve their patients better. “Everyone has an interest in making sure that their patients get the best treatment, but the lowest risk,” Franco notes. “These kinds of adaptive technologies will help in that area and allow institutions to really give cutting-edge medical care with lower risk.”
Many clinical trials fail. The intervention doesn’t have the desired effect, or the side effects are too great. But some failure factors have to do with the behavior of patients, whether or not they follow the protocol. Under current information-gathering norms, serious problems with adherence often aren’t detected until it’s too late to recover — if they’re detected at all.
“The problem in most trials is that you don’t know when there’s nonadherence,” says Holland of AMC Health. “You can’t tell that this disappointing result is because people aren’t taking their meds.”
Regular daily monitoring of patients — for example, using “smart” pill bottles that report usage — can pick up their problems, temporary or chronic, in faithfully reaching into that pill bottle, he says. And the monitoring process itself is a compliance booster. “Knowing that a person’s adherence is being monitored improves adherence across the board, automatically.” And if patients are forgetful, aren’t up to it for health reasons or let a busy lifestyle get in the way, they get a reminder call from study staff.
If patients don’t keep up with protocols, they have to be dropped and replacements must be recruited, which adds to the length as well as cost of the study. “Delays are extremely costly. So, if you can make it easier for patients, you’re more likely to keep them enrolled.”
Where mHealth fits into clinical trials
• Collecting biometric data in homes of trial subjects Measurements don’t require time, cost of patient visit; frequency enables closer monitoring, richer analysis
• Conducting video visits with patients in their homeSTwo-way communication between patient and study staff confirms that protocols are being followed, monitors are used correctly
•Monitoring compliance with the use of tested intervention Trials are not productive or valid if the drug isn’t taken regularly, according to instructions
•Retaining people once the trial is underway Allows patients to participate without cost and effort of traveling to trial site for a year or two
Source: AMC Health, 2014