Smart thinking: data management

Last year, for the first time in human history, there were more tablets, smartphones and conventional mobile phones in the world than there were people. According to calculations by digital analyst GSMA Intelligence, approximately 7.22 billion mobile devices were in use across the planet, compared with a total global population (as calculated by the US Census Bureau) of between 7.19 and 7.2 billon.

Given that mobile devices have only been around for roughly three decades, that is a phenomenal growth rate, although it doesn't necessarily mean every single person on the planet has, or even has access to, a mobile device, as Craig Lipset, head of clinical innovation at pharmaceutical giant Pfizer, concedes.

"I probably have three devices in my pocket right now," he points out. "But I do think, arguably, that 2014 was a tipping point for the sector. More consumers now access information via their mobile devices than through a desktop computer.

"We have begun to see a proliferation of devices as well as greater expectations on the part of the consumer - they expect to be able to access information from the palm of their hand. The whole landscape has changed, both in terms of the hardware and the consumers themselves," he adds.

Access to mobile devices is not only changing how we live our day-to-day lives, it also potentially has ramifications within the arena of clinical trials. The ubiquity and convenience of smartphones, and their potential for data collection, is encouraging pharmaceutical companies to look at how mobile devices and technologies - otherwise known as mHealth - could be used in a range of key trial areas such as patient recruitment and monitoring, data capture and interrogation, and even the way in which studies are carried out.

"The use of mobile within clinical trials has, historically, been quite limited; it's only really taken hold in a handful of areas, such as electronic patient-reporting outcome (ePRO) systems or electronic diaries, with ePRO systems and diaries often moving on to mobile devices and handsets, albeit well before the arrival of the smartphone," says Lipset.

However, the rise and proliferation of mobile devices, especially smartphones and tablets, is beginning to change this landscape.

"We are starting to see more use of apps and mobile devices within trials, engaging investigators and research participants, or as tools or resources for data interrogation," explains Lipset. "We are also seeing a lot of enthusiasm about wearable sensors and devices.

"Teams are increasingly looking to mobile to help modernise areas like informed consent, patient retention, patient self-reporting of data, capturing data from other sensors and remote monitors, or simply improving communications such as product information. Mobile technology can support and enhance study contact, information, design, planning and communication."

"Another important area is data capture; the efficacy and safety data you're capturing in this way must meet the high expectations we naturally have around data integrity and validation. We are gradually ascending a pathway from simply using these devices to support study conduct and information sharing, to data capture, and perhaps ultimately where mobile tools can be developed and utilised alongside medicine, as a companion to our therapeutics," he adds.

Regulatory challenges

But, while mobile technology presents new opportunities, it also brings with it new challenges. For example, it potentially throws up a whole new set of issues around consent, confidentiality and regulatory compliance.

"Mobile is a very broad category," says Lipset. "If it is just a question of wanting to support study conduct, then the barriers can be relatively modest from a regulatory perspective. But for things like capturing data the regulatory issues are, naturally, going to need to be more defined. Fortunately, a lot of guidance already exists."

The US Food and Drug Administration (FDA) issued updated guidance on mobile medical applications as recently as this February, superseding guidance first published in 2013. This discusses the devices FDA will regulate, apps that do not meet its definition of a medical device, and apps that may meet its definition but which, because they pose a low risk to the public, FDA intends only to "exercise enforcement discretion over".

Pfizer has been among pharmaceutical companies pioneering the use of mHealth within clinical trials. In 2011, it incorporated mobile technology into a virtual clinical study of its overactive bladder drug Detrol LA (tolterodine tartrate). This was the first randomised clinical trial patients could participate in entirely from home via their mobile phones or web-based technology.

But it is by no means the only example. Novartis has been running a 'Trials of the Future' programme, which uses mobile technology alongside designated medical devices to automate the collection of patient data from their homes during clinical trials. GlaxoSmithKline has also been investigating how it can combine mHealth devices with cloud-based technologies in a clinical trial setting.

"Different studies have different needs, but most studies have something where mobile can impact on the programme," explains Lipset. "Electronic consent, for example, crosses over and applies to many areas; allowing patients to stay at home for certain visits and transmit data or participate via video can have an impact on test retention. We are seeing more and more use of mobile at early and late stages."

So, where is this all going? One area attracting attention is the potential of wearable mobile devices - bands, sensors, watches and the like - within the context of clinical trials.

Lipset emphasises that this requires a disciplined approach. Many consumer-grade devices found in a local electronic store may well be better positioned for observational studies than for regulated clinical trials.

"There is a lot of enthusiasm, but also some legitimate concerns, about sensors and the topic of wearables," he explains. "Take something like an activity tracker you may find in a local electronics store - it may be very appropriate for a consumer looking to lose weight or be more active, but it can be very difficult to use in a research study if we do not know the algorithms it uses to calculate things. If I do not know it is calculating what a step is, it would be very difficult to use as a potential clinical trial endpoint.

"So what most will find is there are different sensors that are fit for purpose for different research needs; today, we need to isolate what we are measuring and if a consumer grade device is appropriate or whether a more sophisticated clinical grade will be more appropriate," Lipset adds.

"After selecting a sensor, we need to be sure we can deploy and support it in the study (such as training for patients), to pull data off of the devices and into a central database, and ultimately to be able to perform the analytics on that data to answer research questions."

Nevertheless, irrespective of where wearables ultimately fit within the mix, Lipset predicts that the use of mobile devices and technologies within clinical trials is likely to grow and accelerate.

"Mobile technologies illustrate just how accessible research participation can be made." he says. "I would expect to see more data coming from a combination of electronic sources - or eSources, where you pull data from an electronic medical record - coupled with more self-reported data from mobile, and use of diagnostic devices. I think e-source and mobile will continue down that trajectory."

Is your clinical trial a good fit for mHealth technology?

According to Abbe Steel, founder and CEO of HealthiVibe, a company that enables patients to contribute to the design of clinical trials, there are seven key things to look for when deciding if mobile technology could enhance or fit within your clinical trial.

• Protocol: does the protocol lend itself to the use of mHealth? It is important to review the schedule of assessments to see if trial data can be captured electronically without the patient going to the site and/or if mobile technology can be used for studies that require continual patient engagement for retention and increased compliance. But be aware that mobile technologies may not be cost-effective for studies that rely heavily on investigator assessments or specimen collection.
• Patient population: consider whether your trial will require patients to have remote or wireless access to the internet as, if so, this could exclude certain populations or demographics (such as more elderly patients). Some patient populations also have special needs that require more face time with the physician, and in those cases biometric and other data collection may be best done at the site.
• Data integrity: device-specific checkpoints and confirmations are a must. You need clean and quality data at the source.
• Adverse events: the system should include patient prompts to ensure adverse events are reported to the study's principle investigator as soon as they occur.
• Technology adoption and operations management: it is important to consider precisely how you are going use mobile technology to coordinate your trial operations most effectively.
• Device shipments: for global studies, it is important to ensure that device usage and delivery is customised as per in-country regulations. All relevant languages must be supported, and all country-specific privacy regulations must be observed.
• Remote monitoring: you will need to ensure you have the ability to monitor data remotely, via reports that sync device data to a master database. This will enable cost savings and increase efficiency.