From The Editor | July 18, 2016

Are Your Clinical Trials Prepared For The Coming mHealth Tsunami?

Ed Miseta

By Ed Miseta, Chief Editor, Clinical Leader

Mobile Devices

Excitement around mHealth technologies has been building for years. While initial apprehension kept many companies from jumping into the space, sensors are finally starting to make it onto patients. In this Q&A, Glen de Vries, president of Medidata Solutions, discusses progress the industry has made, concerns that remain, and what the future holds for mobile and wearable technologies.  

Ed Miseta: Where do you see the industry gaining traction in regard to incorporating mHealth technologies?

Glen de Vries: I think we are starting to see a shift that is driven by sponsors who are looking for two things. For some it is mobile health measurement. It will be an endpoint that is going to be more sensitive and more specific than a traditionally measured endpoint done in the clinic, like a 6-minute walk test or something requiring a more subjective assessment of a patient.

With these more sensitive and specific measures, there will be an opportunity for sponsors to create a more stringent environment by which their drugs are validated. This will allow them to do a better job of showing regulators why their drug should come to market, and showing payers why the compound should be reimbursed at a particular level.

The second is that we will increasingly see companies develop a digital component to their go-to market strategy. It might be something simple like an app that helps you take your medicine correctly and encourage adherence. Or it could be something that is more interactive and engages the patient, helping them to make certain lifestyle changes.

Miseta: Is there a right time to develop that digital component?

de Vries: Yes. I think the time to get the digital component of a go-to market strategy correct is not when you’re going to market. It has to be well before that. Digital strategies are exciting and you can look around and see many industries that have been significantly disrupted by companies like Uber and Airbnb. No one buys a house anymore without using a website or an app on their phone. In the life sciences it will become just as ubiquitous, where no one will take a new drug without making a website or app an integral part of that experience.

Still, companies should proceed with caution. There may have been 100,000 companies that were created to help people shop with an app. Only about a dozen of them became successful. Therefore, we need to start thinking about digital engagement strategies now, and figure out which ones are useful, effective, and will drive adherence or create a better patient experience.

Miseta: Do the companies pursuing these digital strategies tend to be large or small?

de Vries: It’s definitely a mix, but I would say it tends to be the larger companies. They have the resources to experiment more. That being said, I would also categorize the biotech market as one that is particularly aggressive with how they develop their compound and go-to-market strategies because their whole existence is typically built on one or two programs. 

Miseta: Was there a tipping point in the last 12 months that finally got more companies to start implementing mHealth in their trials? 

de Vries: I think there were a couple of things. Patient centricity is certainly a theme that people have been talking about for a long time. Unfortunately, companies haven’t found good ways to measure what is and isn’t patient centric. Thanks to things like Apple ResearchKit, companies can now measure patient centricity by looking at how much time you’re spending interacting with them and whether or not you’re measuring the things that are important to them. With the proliferation of smart phones around the world, it is now easier than ever to interact with patients. As soon as a few of these early adopter companies get drugs approved using new technologies, everyone else will have to follow suit very quickly.

Miseta: With mHealth, we know the amount of data collected will increase. Will we see the quality increase as well? 

de Vries: Absolutely. For years we have been talking about the parking lot effect, or the tendency of many patients to fill out their patient recorded outcomes right before they walk into a clinic. I think everyone would agree that is not reliable data. By replacing paper diaries with electronic patient reported outcomes, patients could carry an electronic device with them, and could be sent reminders when it is time to fill them out. 

We have already seen electronic patient reported outcomes having a positive impact on the quality of data collected. The quality will get even better as we start to measure these outcomes using improved instrumentation.

For example, if I ask a patient how they’re feeling, they can respond with how they are feeling at that exact moment. We can measure how patients are actually moving on a regular basis. If I’m measuring how well and how long they’re sleeping, I can have a cross-person comparison that has scientific value. In each of those cases, I am getting a much more objective and deterministic measurement, but also getting it in a much higher volume. Instead of getting this information once a week, once a month, or even once a day, I’m getting data feeds that are quite literally 24 hours a day, 7 days a week.

Miseta: Some executives may be wondering once they collect all this data, how do they manage it and glean valuable information from it. Could that be a stumbling point for companies?

de Vries: The requirements are changing in terms of how closely you need to manage the data. Companies no longer need to check every single data point. The FDA is more concerned with looking at data in the aggregate rather than making sure every single data point is correct. Machine learning is another new development that is being used to identify and understand outliers. Machines can see and understand patterns in data that humans might not be able to see. In the future we will continue to find new and better ways of viewing and managing the data.  

Miseta: We seem to have overcome all of the regulatory barriers to mHealth adoption, but there are still companies out there that are concerned about them. How do you help them overcome those concerns?

de Vries: I would first say that FDA is there to discuss specific study with them and to address their concerns. They can set up meetings long before the study starts to address any potential issues. Additionally, FDA is asking for input from sponsors as to how they should go about managing and reviewing this new process.

In general, everyone agrees we will get better efficacy and safety data using mobile health devices. But there’s this lack of clarity around regulatory approval. Nobody wants to be the guinea pigs. FDA understands that and is asking companies to work with them in devising how they regulate this data.

Miseta: Is the FDAs willingness to work with companies one of the reasons more sponsors are looking at mobile technologies?

de Vries: I think so. This is a gradual process. Sponsors will drive the innovation, but FDA has to tell them it’s okay. We had this same situation years ago when companies were moving to EDC. Technology has been improving quickly and FDA has always been open and receptive to innovation. I think we got to the point where sponsors and the FDA are working together because it’s what needed to happen.

Miseta: What do you see as the main barriers still holding up wide-scale adoption?

de Vries: I think one of the issues is that companies don’t realize how easy it can be. I’m not suggesting that picking the right mobile health endpoint is a trivial endeavor. It can be a challenge to decide what you want to measure and then be able to integrate it with more traditional clinical trial data. You also need to do it with the right regulatory framework so you can ultimately use the data for submission. But companies are now doing it and doing it successfully.

Having people who understand the process is important. Sponsors will need access to people who understand the mathematics, the statistics, and the techniques around using some of these new measurements to create reliable endpoints that we’ve proven to regulatory agencies are worthy of their attention. We need to get mobile health data to become substantial evidence to regulatory agencies.

Miseta: Are there therapeutic areas that seem to currently be a better fit for mHealth adoption?

de Vries: I think pain management is a really good one. It’s a space that is certainly worthy of additional measures for efficacy. Chronic disease management is another. I think there will also be opportunities in oncology. I was speaking to the founder of a biotech company that has yet to perform their first trial. He had a lot of questions on conducting the trial and I suggested he put an activity tracker on every patient. There is no reason we shouldn’t be as concerned about the quality of sleep in an oncology patient as we are with someone having a problem with their heart or endocrine system. While there are some obvious therapeutic areas, I think there will be some surprises that come along as well.

There are many opportunities that exist if we can get the right technology and the right patients together. The technology will help us to think differently about how we measure and manage the disease progression. But companies need to start thinking about this now. We want sponsors to drive the success of this technology, not get rolled over by it.

Miseta: Are there any sponsor companies out there that are aggressively implementing these technologies and blazing a path forward for others to follow?

de Vries: If I had to pick one company, I would say that GSK deserves to wear the mHealth superhero uniform. They were one of the first companies to push the limits of how well they could instrument a person. That’s the kind of innovative spirit that we need in this industry. Companies need to know it is possible to incorporate these technologies into trials. We are thankful to them for taking that initiative and showing what can be done.